UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
 
FORM
10-Q
FORM 10-Q
 
(Mark One)
☒
QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(D) OF THE SECURITIES EXCHANGE ACT OF 1934
For the quarterly period ended March 31,September 30, 2023
 
☐
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(D) OF THE SECURITIES EXCHANGE ACT OF 1934
For the transition period from
                    
to
                    
Commission File Number
001-39267
 
BENITEC BIOPHARMA INC.
(Exact name of registrant as specified in its charter)
 
Delaware
 
84-462-0206
(State or other jurisdiction of
incorporation or organization)
 
(IRS Employer
Identification No.)
3940 Trust Way, Hayward, California 94545
(Address of principal executive offices & zip code)
(510)
780-0819
(Registrant’s telephone number including area code)
 
Securities registered pursuant to Section 12(b) of the Act:
 
Title of each class
 
Trading
Symbol(s)
 
Name of each exchange
on which registered
Common Stock, par value $0.0001
 
BNTC
 
The Nasdaq Stock Market LLC
Indicate by check mark whether the registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.
Yes
    ☒  No    ☐
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation
S-T
(§ (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files).
Yes
    ☒  No    ☐
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a
non-accelerated
filer, smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule
12b-2
of the Exchange Act.
 
Large accelerated filer ☐  Accelerated filer ☐
    
Non-accelerated
filer		 ☒  Smaller reporting company ☒
    
Emerging growth company ☐     
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.  ☐
Indicate by check mark whether the registrant is a shell company (as defined in Rule
12b-2
of the Exchange Act). Yes    ☐  or  No    
☒
We had 27,981,1612,572,434 shares of our common stock outstanding as of the close of business on MaNovember
y 11, 27
023., 2023.
 
 

BENITEC BIOPHARMA INC.

INDEX TO FORM 10-Q

 

SPECIAL NOTE REGARDING FORWARD LOOKING STATEMENTS

   1 

PA RT PART I-FINANCIAL INFORMATION

   2 

ITEM 1.

 

Financial Statements

   2 
 

Consolidated Balance Sheets as of March 31,September 30, 2023 (Unaudited) and June 30, 20222023

   2 
 

Consolidated Statements of Operations and Comprehensive Loss for the Three and Nine Months Ended March 31,September 30, 2023 and 2022 (Unaudited)

   3 
 

Consolidated Statements of Stockholders’ Equity for the NineThree Months Ended March 31,September 30, 2023 and 2022 (Unaudited)

   4 
 

Consolidated Statements of Cash Flows for the NineThree Months Ended March 31,September 30, 2023 and 2022 (Unaudited)

   5 
 

Notes to Consolidated Financial Statements (Unaudited)

   6 

ITEM 2.

 

Management’s Discussion and Analysis of Financial Condition and Results of Operations

   1516 

ITEM 3.

 

Quantitative and Qualitative Disclosures About Market Risk

   2956 

ITEM 4.

 

Controls and Procedures

   2956 

PA RT PART II-OTHER INFORMATION

  

ITEM 1.

 

Legal Proceedings

   3057 

ITEM 1A.

 

Risk Factors

   3057 

ITEM 2.

 

Unregistered Sales of Equity Securities, and Use of Proceeds, and Issuer Purchases of Equity Securities

   3157 

ITEM 3.

 

Defaults Upon Senior Securities

   3157 

ITEM 4.

 

Mine Safety Disclosures

   3157 

ITEM 5.

 

Other Information

   3157 

ITEM 6.

 

Exhibits

   3257 

SIGNATURES

   3358 

SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS

This Report contains forward-looking statements that are subject to a number of risks and uncertainties, many of which are beyond our control. Our forward-looking statements relate to future events or our future performance and include, but are not limited to, statements concerning our business strategy, future commercial revenues, market growth, capital requirements, new product introductions, expansion plans and the adequacy of our funding. All statements, other than statements of historical fact included in this Report, are forward-looking statements. When used in this Report, the words “could,” “believe,” “anticipate,” “intend,” “estimate,” “expect,” “may,” “continue,” “predict,” “potential,” “project,” or the negative of these terms, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain such identifying words. These statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements.

Some of the risks and uncertainties that may cause our actual results, performance or achievements to differ materially from those expressed or implied by forward-looking statements include the following:

 

the success of our plans to develop and potentially commercialize our product candidates;

 

the timing of the initiation and completion of preclinical studies and clinical trials;

 

the timing and sufficiency of patient enrollment and dosing in any futureour clinical trials;

 

the timing of the availability of data from clinical trials;

 

the timing and outcome of regulatory filings and approvals;

 

unanticipated delays;

 

sales, marketing, manufacturing and distribution requirements;

 

market competition and the acceptance of our products in the marketplace;

 

regulatory developments in the United States of America, France, and Canada;

 

the development of novel AAV vectors;

 

the plans of licensees of our technology;

 

the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure;

 

our dependence on our relationships with collaborators and other third parties;

 

expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors, including our capital structure;

 

our ability to continue as a going concern;

 

our ability to meet the Nasdaq listing standards;

the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan;

 

our intellectual property position and the duration of our patent portfolio;

 

the impact of local, regional, and national and international economic conditions and events; and

 

•

the impact of the current COVID-19 pandemic, the disease caused by the SARS-CoV-2 virus, which may adversely impact our business and preclinical and future clinical trials;

the impact of the COVID-19 pandemic, the disease caused by the SARS-CoV-2 virus and similar events, which may adversely impact our business and preclinical and future clinical trials;

as well as other risks detailed under the caption “Risk Factors” in this Report and in other reports filed with the SEC. Although we believe that we have a reasonable basis for each forward-looking statement contained in this Report, we caution you that these statements are based on a combination of facts and important factors currently known by us and our expectations of the future, about which we cannot be certain. We have based the forward-looking statements included in this Report on information available to us on the date of this Report or on the date thereof. Except as required by law we undertake no obligation to revise or update any forward-looking statements, whether as a result of new information, future events or otherwise. You are advised to consult any additional disclosures that we may make directly to you or through reports that we, in the future, may file with the SEC, including annual reports on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K.

All forward-looking statements included herein or in documents incorporated herein by reference are expressly qualified in their entirety by the cautionary statements contained or referred to elsewhere in this Report.

 

1

PART I—FINANCIAL INFORMATION
ITEM 1.Financial Statements
ITEM 1. Financial Statements
BENITEC BIOPHARMA INC.
Consolidated Balance Sheets
(in thousands, except par value and share amounts)
   March 31,  June 30, 
   2023  2022 
   (Unaudited)    
Assets         
Current assets:         
   
Cash and cash equivalents  $6,551  $4,062 
Restricted cash   14   14 
Trade and other receivables   56   3 
Prepaid and other assets   774   741 
          
Total current assets   7,395   4,820 
Property and equipment, net   106   222 
Deposits   25   25 
Other assets   105   135 
Right-of-use
assets
   589   771 
          
Total assets  $8,220  $5,973 
          
Liabilities and stockholders’ equity         
Current liabilities:         
Trade and other payables  $2,455  $1,880 
Accrued employee benefits   429   400 
Lease liabilities, current portion   269   252 
          
Total current liabilities   3,153   2,532 
Lease liabilities, less current portion   354   559 
          
Total liabilities   3,507   3,091 
          
Commitments and contingencies (Note 11)         
Stockholders’ equity:         
Common stock, $0.0001 par
value-160,000,000
shares authorized; 27,981,161 shares and 8,171,690 shares issued and outstanding at March 31, 2023 and June 30, 2022, respectively
   3   1 
Additional
paid-in
capital
   168,791   152,453 
Accumulated deficit   (163,228)   (148,327) 
Accumulated other comprehensive loss   (853)   (1,245) 
          
Total stockholders’ equity   4,713   2,882 
          
Total liabilities and stockholders’ equity  $8,220  $5,973 
          
The accompanying notes are an integral part of these consolidated financial statements.
2
Table of Contents
BENITEC BIOPHARMA INC.
Consolidated Statements of Operations and Comprehensive Loss
(Unaudited)
(in thousands, except share and per share amounts)

 
   Three Months Ended  Nine Months Ended 
   March 31,  March 31, 
   2023  2022  2023  2022 
Revenue:                 
     
Licensing revenues from customers  $54  $48  $68  $73 
                  
Total revenues   54   48   68   73 
                  
Operating expenses                 
Research and development   3,167   2,171   9,588   8,096 
General and administrative   1,228   1,337   5,011   5,093 
                  
Total operating expenses   4,395   3,508   14,599   13,189 
                  
Loss from operations   (4,341)   (3,460)   (14,531)   (13,116) 
Other income (loss):                 
Foreign currency transaction gain (loss)   (45)   229   (391)   36 
Interest expense, net   (7)   (10)   (25)   (22) 
Other income, net   —     (29)   50   (29) 
Unrealized loss on investment   (4)   (5)   (4)   (10) 
                  
Total other income (loss), net   (56)   185   (370)   (25) 
                  
Net loss  $(4,397)  $(3,275)  $(14,901)  $(13,141) 
                  
Other comprehensive income:                 
Unrealized foreign currency translation gain (loss)   45   (233)   392   (51) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Total other comprehensive income (loss)   45   (233)   392   (51) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Total comprehensive loss  $(4,352)  $(3,508)  $(14,509)  $(13,192) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Net loss  $(4,397)  $(3,275)  $(14,901)  $(13,141) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Net loss per share:                 
Basic and diluted  $(0.16)  $(0.40)  $(0.67)  $(1.61) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Weighted average number of shares outstanding: basic and diluted   27,981,161   8,171,690   22,090,191   8,171,690 
 
  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
   September 30,
2023		  June 30,
2023		 
   (Unaudited)    
Assets
         
Current assets:
         
Cash and cash equivalents
  $25,864  $2,477 
Restricted cash
   13   13 
Trade and other receivables
   53   55 
Prepaid and other assets
   482   1,184 
   
 
 
  
 
 
 
Total current assets
   26,412   3,729 
Property and equipment, net
   68   87 
Deposits
   25   25 
Prepaid and other
 assets
   85   97 
Right-of-use assets
   463   526 
   
 
 
  
 
 
 
Total assets
  $27,053  $4,464 
   
 
 
  
 
 
 
Liabilities and stockholders’ equity
         
Current liabilities:
         
Trade and other payables
  $3,794  $3,231 
Accrued employee benefits
   460   472 
Lease liabilities, current portion
   280   275 
   
 
 
  
 
 
 
Total current liabilities
   4,534   3,978 
Lease liabilities, less current portion
   211   284 
   
 
 
  
 
 
 
Total liabilities
   4,745   4,262 
   
 
 
  
 
 
 
Commitments and contingencies (Note 10)
       
Stockholders’ equity:
         
Common stock, $0.0001 par value-160,000,000 shares authorized; 2,547,434 shares and 1,671,485 shares issued and outstanding at September 30, 2023 and June 30, 2023, respectively
   —    —  
Additional paid-in capital
   196,931   168,921 
Accumulated deficit
   (173,843)   (167,889) 
Accumulated other comprehensive loss
   (780)   (830) 
   
 
 
  
 
 
 
Total stockholders’ equity
   22,308   202 
   
 
 
  
 
 
 
Total liabilities and stockholders’ equity
  $27,053  $4,464 
   
 
 
  
 
 
 
The accompanying notes are an integral part of these consolidated financial statements.
 
3
2
Table of Contents

BENITEC BIOPHARMA INC.
Consolidated Statements of Stockholders’ EquityOperations and Comprehensive Loss
(Unaudited)
(in thousands, except share and per share amounts)

   Three Months Ended September 30, 
   2023  2022 
Revenue:
         
Licensing revenues from customers
         
   
 
 
  
 
 
 
Total revenues
  $—   $—  
   
 
 
  
 
 
 
Operating expenses:
         
Royalties and license fees
   (106)   —   
Research and development
   4,429   2,660 
General and administrative
   1,551   1,920 
   
 
 
  
 
 
 
Total operating expenses
   5,874   4,580 
   
 
 
  
 
 
 
Loss from operations
   (5,874)   (4,580) 
Other income (loss):
         
Foreign currency transaction loss
   (56)   (507) 
Interest expense, net
   (6)   (9) 
Other expense, net   (18)   —  
Unrealized gain on investment
   —    3 
   
 
 
  
 
 
 
Total other loss, net
   (80)   (513) 
   
 
 
  
 
 
 
Net loss
  $(5,954)  $(5,093) 
   
 
 
  
 
 
 
Other comprehensive income:
         
Unrealized foreign currency translation gain
   50   507 
   
 
 
  
 
 
 
Total other comprehensive income
   50   507 
   
 
 
  
 
 
 
Total comprehensive loss
  $(5,904)  $(4,586) 
   
 
 
  
 
 
 
Net loss
  $(5,954)  $(5,093) 
Net loss per share: basic and diluted
  $(2.76)  $(7.98) 
   
 
 
  
 
 
 
Weighted average number of shares outstanding: basic and diluted
   2,157,065   638,572 
   
 
 
  
 
 
 
   Common Stock   
Additional
Paid-in
  Accumulated  
Accumulated
Other
Comprehensive
  
Total
Stockholders’
 
   Shares   Amount   Capital  Deficit  Loss  Equity 
Balance at June 30, 2021   8,171,690   $1   $151,583  $(130,119)  $(1,455)  $20,010 
Share-based compensation   —      —      271   —     —     271 
Foreign currency translation gain   —      —      —     —     239   239 
Net loss   —      —      —     (5,045)       (5,045) 
                            
Balance at September 30, 2021   8,171,690    1    151,854   (135,164)   (1,216)   15,475 
                            
Share-based compensation   —      —      239   —     —     239 
Foreign currency translation loss   —      —      —     —     (57)   (57) 
Net loss   —      —      —     (4,821)   —     (4,821) 
                            
Balance at December 31, 2021   8,171,690    1    152,093   (139,985)   (1,273)   10,836 
Share-based compensation   —      —      192   —     —     192 
Foreign currency translation loss   —      —      —     —     (233)   (233) 
Net loss   —      —      —     (3,275)   —     (3,275) 
                            
Balance at March 31,2022   8,171,690   $1   $152,285  $(143,260)  $(1,506)  $7,520 
                            
Balance at June 30, 2022   8,171,690   $1   $152,453  $(148,327)  $(1,245)  $2,882 
Issuance of common stock,
pre-funded
warrants, and common warrants sold for cash, net of offering costs of $1,869
   17,637,843    2    16,013   —     —     16,015 
Share-based compensation   —      —      302   —     —     302 
Foreign currency translation gain   —      —      —     —     507   507 
Net loss   —      —      —     (5,093)   —     (5,093) 
   
 
 
   
 
 
   
 
 
  
 
 
  
 
 
  
 
 
 
Balance at September 30, 2022   25,809,533    3    168,768   (153,420)   (738)   14,613 
Exercise of
pre-funded
warrants
   2,171,628    —      —     —     —     —   
Share-based compensation   —      —      (48)   —     —     (48) 
Foreign currency translation loss   —      —      —     —     (160)   (160) 
Net loss   —      —      —     (5,411)   —     (5,411) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Balance at December 31, 2022   27,981,161    3    168,720   (158,831)   (898)   8,994 
Share-based compensation   —      —      71   —     —     71 
Foreign currency translation gain   —      —      —     —     45   45 
Net loss   —      —      —     (4,397)   —     (4,397) 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Balance at March 31, 2023   27,981,161   $3   $168,791  $(163,228)  $(853)  $4,713 
 
  
 
 
 
  
 
 
 
  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
The accompanying notes are an integral part of these consolidated financial statements.

4
3
Table of Contents

BENITEC BIOPHARMA INC.
Consolidated Statements of Cash FlowsStockholders’ Equity
(Unaudited)
(in thousands)thousands, except share amounts)
 
   Nine Months
Ended March 31,		 
   2023  2022 
Cash flows from operating activities:         
   
Net loss  $(14,901)  $(13,141) 
Adjustments to reconcile net loss to net cash used in operating activities:         
Depreciation and amortization   115   161 
Amortization of
right-of-use
assets
   182   168 
Unrealized loss on investment   4   10 
Share-based compensation expense   325   702 
Changes in operating assets and liabilities:         
Trade and other receivables   (50)   47 
Prepaid expense and other assets   (14)   609 
Trade and other payables   580   512 
Accrued employee benefits   30   28 
Lease liabilities   (188)   (140) 
          
Net cash used in operating activities   (13,917)   (11,044) 
          
Cash flows from financing activities:         
Proceeds from issuance of common stock,
pre-funded
warrants, and common warrants
   17,884   —   
Shares and
pre-funded
warrant issuance costs
   (1,869)   —   
          
Net cash provided by financing activities   16,015   —   
          
Effects of exchange rate changes on cash, cash equivalents, and restricted cash   391   (95) 
          
Net increase (decrease) in cash, cash equivalents, and restricted cash   2,489   (11,139) 
Cash, cash equivalents, and restricted cash, beginning of period   4,076   19,769 
          
Cash, cash equivalents, and restricted cash, end of period  $6,565  $8,630 
          
Supplemental disclosure of cash flow information:         
Re-measurement
of operating lease
right-of-use
assets and liabilities
  $—    $794 
          
   Common Stock   Additional
Paid-in		   Accumulated  Accumulated
Other
Comprehensive		  Total
Stockholders’		 
   Shares   Amount   Capital   Deficit  Loss  Equity 
Balance at June 30, 2022
   480,688   $—    $152,454   $(148,327)  $(1,245)  $2,882 
Issuance of common stock, pre-funded warrants, and common Warrants sold for cash, net of offering costs of $1,869
   1,037,520    —     16,015            16,015 
Share-based compensation
   —     —     302    —    —    302 
Foreign currency translation gain
   —     —     —     —    507   507 
Net loss
   —     —     —     (5,093)       (5,093) 
   
 
 
   
 
 
   
 
 
   
 
 
  
 
 
  
 
 
 
Balance at September 30, 2022
   1,518,208    —     168,771    (153,420)   (738)   14,613 
   
 
 
   
 
 
   
 
 
   
 
 
  
 
 
  
 
 
 
Balance at June 30, 2023
   1,671,485    —     168,921    (167,889)   (830)   202 
Issuance of common stock, pre-funded warrants, and common warrants sold for cash, net of offering costs of $2,964
   875,949    —     27,919    —    —    27,919 
Share-based compensation
   —     —     91    —    —    91 
Foreign currency translation gain
   —     —     —     —    50   50 
Net loss
   —     —     —     (5,954)   —    (5,954) 
   
 
 
   
 
 
   
 
 
   
 
 
  
 
 
  
 
 
 
Balance at September 30, 2023
   2,547,434   $—    $196,931   $(173,843)  $(780)  $22,308 
   
 
 
   
 
 
   
 
 
   
 
 
  
 
 
  
 
 
 
The accompanying notes are an integral part of these consolidated financial statements.
 
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Table of Contents

BENITEC BIOPHARMA INC.
Consolidated Statements of Cash Flows
(Unaudited)
(in thousands)
   Three Months Ended
September 30,		 
   2023  2022 
Cash flows from operating activities:
         
Net loss
  $(5,954)  $(5,093) 
Adjustments to reconcile net loss to net cash used in operating activities:
         
Depreciation and amortization
   19   42 
Amortization of right-of-use assets
   63   60 
Unrealized gain on investment
   —    (3) 
Share-based compensation expense
   91   302 
Changes in operating assets and liabilities:
         
Prepaid and other assets   709   220 
Trade and other payables
   566   500 
Accrued employee benefits
   (4)   (11) 
Lease liabilities
   (67)   (62) 
   
 
 
  
 
 
 
Net cash used in operating activities
   (4,577)   (4,045) 
   
 
 
  
 
 
 
Cash flows from investing activities:
         
Net cash used in investing activities
   —    —  
   
 
 
  
 
 
 
Cash flows from financing activities:
         
Proceeds from issuance of common stock, pre-funded warrants, and common warrants
   30,883   17,884 
Stock issuance costs

   (2,964)   (1,869) 
   
 
 
  
 
 
 
Net cash provided by financing activities
   27,919   16,015 
   
 
 
  
 
 
 
Effects of exchange rate changes on cash, cash equivalents, and restricted cash
   45   501 
   
 
 
  
 
 
 
Net increase in cash, cash equivalents, and restricted cash
   23,387   12,471 
Cash, cash equivalents, and restricted cash, beginning of period
   2,490   4,076 
   
 
 
  
 
 
 
Cash, cash equivalents, and restricted cash, end of period
  $25,877  $16,547 
   
 
 
  
 
 
 
The accompanying notes are an integral part of these consolidated financial statements.
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Table of Contents

BENITEC BIOPHARMA INC.
Notes to Consolidated Financial Statements
(Unaudited)
1. Business
Benitec Biopharma Inc. (the “Company”) is a corporation formed under the laws of Delaware, United States of America, on November 22, 2019 and listed on the Nasdaq Capital Market (“Nasdaq”) under the symbol “BNTC”. Benitec Biopharma Inc. is the parent entity of a number of subsidiaries including the previous parent entity Benitec Biopharma Limited (“BBL”). BBL was incorporated under the laws of Australia in 1995 and was listed on the Australian Securities Exchange, or ASX, from 1997 until April 15, 2020. On August 14, 2020, BBL reorganized as a Proprietary Limited company and changed its name to Benitec Biopharma Proprietary Limited. The Company’s business focuses on the development of novel genetic medicines. Our proprietary platform, called
DNA-directed
RNA interference, or ddRNAi, combines RNA interference, or RNAi, with gene therapy to create medicines that facilitate sustained silencing of disease-causing genes.
During the year ended June 30, 2021, the Company completed an organization restructuring as part of the commercial desire to provide a more efficient structure for the future as the Company transitioned its operations to the United States.
The Company’s fiscal year end is June 30. References to a particular “fiscal year” are to our fiscal year end June 30 of that calendar year.
The consolidated financial statements of Benitec Biopharma Inc. are presented in United States dollars and consist of Benitec Biopharma Inc. and its wholly owned subsidiaries:subsidiaries as listed below. Aside from Benitec Biopharma Proprietary Limited, the international subsidiaries are dormant.
   Principal place of
business/country of
incorporation
Benitec Biopharma Proprietary Limited (“BBL”)
  Australia
Benitec Australia Proprietary Limited
  Australia
Benitec Limited
  United Kingdom
Benitec, Inc.
  USA
Benitec LLC
  USA
RNAi Therapeutics, Inc.
  USA
Tacere Therapeutics, Inc.
  USA
Benitec IP Holdings, Inc.
  USA
2. Basis of Presentation and Summary of Significant Accounting Policies
Basis of Presentation
The Company’s consolidated financial statements contained in this Quarterly Report on Form
10-Q
have been prepared in accordance with generally accepted accounting principles in the U.S. (“GAAP”) for interim financial information and with the instructions to Form
10-Q
and Article 8 of U.S. Securities and Exchange Commission (“SEC”) Regulation
S-X.
Accordingly, certain information and disclosures required by GAAP for annual financial statements have been omitted. In the opinion of management, all adjustments, consisting of normal recurring adjustments, considered necessary for a fair presentation have been included. Interim financial results are not necessarily indicative of results anticipated for the full year. These consolidated financial statements should be read in conjunction with the Company’s audited financial statements and accompanying notes included in the Company’s Annual Report on Form
10-K
for the year ended June 30, 2023.
On July 26, 2023, the Company effected a 1-for-17 reverse stock split (the “Reverse Stock Split”) of its common stock. In accordance with the Reverse Stock Split, 17 pre-split shares of the Company’s common stock were automatically converted into one issued and outstanding post-split share. Proportional adjustments were also made to all outstanding stock options, pre-funded warrants, and common warrants in accordance with their respective terms. The Reverse Stock Split did not change the par value of the Company’s common stock or the authorized number of shares. No fractional shares were issued in connection with the Reverse Stock Split. All fractional shares were rounded up to the nearest whole share with respect to outstanding shares of common stock.
All share and earnings per share amounts presented in this Form 10-Q reflect the impact of this reverse split as if it had taken effect on June 30, 2022.
Reference is frequently made herein to the Financial Accounting Standards Board (the “FASB”) Accounting Standards Codification (“ASC”). This is the source of authoritative GAAP recognized by the FASB to be applied to
non-governmental
entities.
Principles of Consolidation
The consolidated financial statements include the Company’s accounts and the accounts of its wholly owned subsidiaries. All intercompany transactions and balances have been eliminated.
Use of Estimates
The preparation of the Company’s consolidated financial statements requires management to make estimates and assumptions that impact the reported amounts of assets, liabilities and expenses and the disclosure of contingent assets and liabilities in the Company’s consolidated financial statements and accompanying notes. The most significant estimates and assumptions in the Company’s
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consolidated financial statements include the estimates of useful
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lives of property and equipment, valuation of the operating lease liability and related right-of-use asset, valuation of equity-based instruments issued for other than cash, the valuation allowance for uncollectable receivables, foreign currency translation due to certain average exchange rates applied in lieu of spot rates on transaction dates,deferred tax assets, and accrued research and development expenses.expense. These estimates and assumptions are based on current facts, historical experience and various other factors believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities and the recording of expenses that are not readily apparent from other sources. Actual results may differ materially and adversely from these estimates. To the extent there are material differences between the estimates and actual results, the Company’s future results of operations will be affected. 
Risks and Uncertainties
The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including, but not limitedlim
it
ed to, development by competitors of new technological innovations, protection of proprietary technology, dependence on key personnel, reliance on single- sourcesingle-source vendors and collaborators, availability of raw materials, patentability of the Company’s products and processes and clinical efficacy and safety of the Company’s products under development, compliance with government regulations and the need to obtain additional financing to fund operations.
There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained or maintained, that any products developed will obtain necessary government regulatory approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid technological change and substantial competition from other pharmaceutical and biotechnology companies. In addition, the Company is dependent upon the services of its employees, consultants and other third
parties.
Moreover, the COVID-19 pandemic, which impacted and may in the future impact worldwide economic activity, and similar events pose risks that the Company or its employees, contractors, suppliers, and other partners may be prevented or inhibited from conducting business activities for an indefinite period of time which may delay the start-up and conduct of the Company’s clinical trials, and negatively impact manufacturing and testing activities performed by third parties. Any significant delays may impact the use and sufficiency of the Company’s existing cash reserves, and the Company may be required to raise additional capital earlier than it had previously planned. The Company may be unable to raise additional capital if and when needed, which may result in delays or suspension of its development plans. The extent to which the pandemic will impact the Company’s business will depend on future developments that are highly uncertain and cannot be predicted at this time.
Segment Reporting
Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision-maker in making decisions regarding resource allocation and assessing performance. The Company views its operations and manages its business in one operating segment.
Foreign Currency Translation and Other Comprehensive Income (Loss)
The Company’s functional currency and reporting currency is the United States dollar. BBL’s functional currency is the Australian dollar (AUD). Assets and liabilities are translated at the exchange rate in effect at the balance sheet date. Revenues and expenses are translated at the average rate of exchange prevailing during the reporting period. Equity transactions are translated at each historical transaction date spot rate. Translation adjustments arising from the use of different exchange rates from period to period are included as a component of stockholders’ equity as “Accumulated other comprehensive loss.” Gains and losses resulting from foreign currency translation are included in the consolidated statements of operations and comprehensive loss as other comprehensive income (loss).
Other comprehensive income (loss) for all periods presented consists entirely of foreign currency translation gains and losses.
 
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Fair Value Measurements
The Company measures its financial assets and liabilities in accordance with GAAP using ASC 820,
Fair Value Measurements.Measurements
. For certain financial instruments, including cash and cash equivalents, accounts receivable, and accounts payable, the carrying amounts approximate fair value due to their short maturities.
The Company follows accounting guidance for financial assets and liabilities. ASC 820 defines fair value, provides guidance for measuring fair value and requires certain disclosures. The guidance utilizes a fair value hierarchy that prioritizes the inputs to valuation techniques used to measure fair value into three broad levels. The following is a brief description of those three levels:
 
Level 1:  Observable inputs such as quoted prices (unadjusted) in active markets for identical assets or liabilities.
  
Level 2:  Inputs, other than quoted prices that are observable, either directly or indirectly. These include quoted prices for similar assets or liabilities in active markets and quoted prices for identical or similar assets or liabilities in markets that are not active.
  
Level 3:  Unobservable inputs in which little or no market data exists, therefore developed using estimates and assumptions developed by us, which reflect those that a market participant would use.
Cash and Cash Equivalents
Cash and cash equivalents include cash on hand and at banks, short-term deposits with an original maturity of three months or less with financial institutions, and bank overdrafts. Bank overdrafts are reflected as a current liability on the consolidated balance sheets. There were no cash equivalents or bank overdrafts as of March 31,September 30, 2023 and June 30, 2022.2023.
Restricted cash balances of $13 thousand as of both September 30, 2023 and June 30, 2023 secure the Company’s credit cards.
Concentrations of Risk
Financial instruments that potentially subject the Company to significant concentration of credit risk consist primarily of cash and cash equivalents. The Company maintains deposits at federally insured financial institutions in excess of federally insured limits. The Company has not experienced any losses in such accounts, and management believes that the Company is not exposed to significant credit risk due to the financial position of the depository institutions in which those deposits are held.
Trade and Other Receivables
The Company adopted ASC 326—
Financial Instruments—Credit Losses (Topic 326).
as of July 1, 2023. As amounts become uncollectible, theysuch, the Company estimates current expected credit losses (CECL) on trade and other receivables on an ongoing basis, and will recognize those expected credit losses immediately. Estimates of current expected credit losses will be charged to an allowance and operations in the period when a determination of collectability is made. Any estimates of potentially uncollectible customer accounts receivable will be made based on an analysisanalyses of individual customer circumstances and historical
write-off
experience. The Company’s analysis includesanalyses will consider the ageaging of the receivable account,accounts, customer creditworthiness, of the customer and general economic conditions.
Property and Equipment
Property and equipment are stated at cost, net of accumulated depreciation and amortization. Expenditures for maintenance and repairs are expensed as incurred; additions, renewals, and improvements are capitalized. When property and equipment are retired or otherwise disposed of, the related cost and accumulated depreciation and amortization are removed from the respective accounts, and any gain or loss is included in operations. Depreciation and amortization of property and equipment is calculated using the straight-line basis over the following estimated useful lives:
 
Software  
3-4
years
Lab equipment  
3-7
years
Computer hardware  
3-5
years
Leasehold improvements  shorter of the lease term or estimated useful lives
Impairment of Long-Lived Assets
Property and equipment are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Recoverability of long-lived assets to be held and used is measured by a comparison of the carrying amount of an asset to the estimated undiscounted future cash flows expected to be generated by the asset. If the carrying amount of an asset exceeds its estimated undiscounted future cash flows, an impairment charge is recognized by the amount by which the carrying amount of the asset exceeds the fair value of the assets. Fair value is generally determined using the asset’s expected future discounted cash flows or market value, if readily determinable.
 
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Trade and other payables
These amounts represent liabilities for goods and services provided to the Company prior to the end of the period and which are unpaid. Due to their short-term nature, they are measured at amortized cost and are not discounted. The amounts are unsecured and are usually paid within 30 days of recognition.
Leases
At lease commencement, the Company records a lease liability based on the present value of lease payments over the expected lease term. The Company calculates the present value of lease payments using the discount rate implicit in the lease, unless that rate cannot be readily determined. In that case, the Company uses its incremental borrowing rate, which is the rate of interest that the Company would have to pay to borrow on a collateralized basis an amount equal to the lease payments over the expected lease term. The Company records a corresponding
right-of-use
lease asset based on the lease liability, adjusted for any lease incentives received and any initial direct costs paid to the lessor prior to the lease commencement date.
After lease commencement, the Company measures its leases as follows: (i) the lease liability based on the present value of the remaining lease payments using the discount rate determined at lease commencement; and (ii) the
right-of-use
lease asset based on the remeasured lease liability, adjusted for any unamortized lease incentives received, any unamortized initial direct costs and the cumulative difference between rent expense and amounts paid under the lease agreement. Any lease incentives received and any initial direct costs are amortized on a straight-line basis over the expected lease term. Rent expense is recorded on a straight-line basis over the expected lease term.
Basic and Diluted Net Loss Per Share
Basic net loss per share is calculated by dividing net loss by the weighted-average number of common shares outstanding during the period. Diluted net loss per share is calculated by dividing net loss by the weighted-average number of common shares outstanding plus potential common shares. Stock options, warrants and convertible instruments are considered potential common shares and are included in the calculation of diluted net loss per share using the treasury stock method when their effect is dilutive. Potential common shares are excluded from the calculation of diluted net income (loss) per share when their effect is anti-dilutive. As of March 31,September 30, 2023, and June 30, 2022,2023, there were 40,684,96533,584,433 and 845,1592,456,032 potential common shares, respectively, that were excluded from the calculation of diluted net loss per share because their effect was anti-dilutive.
Revenue Recognition
The Company recognizes revenue in accordance with ASC 606—
Revenue from Contracts with Customers
(“ASC 606”). The core principle of ASC 606 is that entities are to recognize revenue to depict the transfer of promised goods and services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services.
The Company recognizes revenue in accordance with that core principle by applying the following steps:
Step 1: Identify the contract(s) with a customer.
Step 2: Identify the performance obligations in the contract.
Step 3: Determine the transaction price.
Step 4: Allocate the transaction price to the performance obligations in the contract.
Step 5: Recognize revenue when (or as) the entity satisfies a performance obligation.
The Company applies judgement in determining whether contracts entered into fall within the scope of ASC 606, Revenue from Contracts with Customers (“ASC 606”).606. In doing so, management considers the commercial substance of the transaction and how risks and benefits of the contract accrue to the various parties to the contract.
Management has also made the judgement that the grant of the license and transfer of associated
know-how
and materials are accounted for as one performance obligation as they are not considered to be distinct; they are highly interrelated and could not provide benefits to the customer independently from each other. Judgements were made in relation to the transfer of the license and
know-how
and whether this should be recognized over time or a point in time. The point in time has been determined with regard to the point at which the transfer of
know-how
has substantially been completed and the customer has control of the asset and the ability to direct the use of and receive substantially all of the remaining benefits.
Licensing revenues
Revenue from licensees of the Company’s intellectual property reflects the transfer of a right to use the intellectual property as it exists at the point in time in which the license is transferred to the customer. Consideration can be variable and is estimated using the most likely amount method and is constrained to the extent that it is probable that a significant reversal will not occur. Revenue is recognized as or when the performance obligations are satisfied.
 
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The Company recognizes contract liabilities for consideration received in respect of unsatisfied performance obligations and reports these amounts as other liabilities in the consolidated balance sheet. Similarly, if the Company satisfies a performance obligation before it receives the consideration, the Company recognizes either a contract asset or a receivable in its consolidated balance sheet, depending on whether something other than the passage of time is required before the consideration is due.
Royalties
Revenue from licensees of the Company’s intellectual property reflect a right to use the intellectual property as it exists at the point in time in which the license is granted. Where consideration is based on sales of product by the licensee, revenue is recognized when the customer’s subsequent sales of products occur.
Services revenue
Revenue is earned (constrained by variable considerations) from the provision of research and development services to customers. Services revenue is recognized when performance obligations are either satisfied over time or at a point in time. Generally, the provision of research and development services under a contract with a customer will represent satisfaction of a performance obligation over time where the Company retains the right to payment for services performed but not yet completed.
Research and Development Expense
Research and development costs are expensed when incurred.
Research and development expenses relate primarily to the cost of conducting clinical and preclinicalpre-clinical trials. PreclinicalPre- clinical and clinical development costs are a significant component of research and development expenses. Estimates have been usedThe Company records accrued liabilities for estimated costs of research and development activities conducted by third-party service providers, which include the conduct of pre-clinical studies and clinical trials, and contract manufacturing activities. The Company records the estimated costs of research and development activities based upon the estimated amount of services provided but not yet invoiced and includes these costs in trade and other payables on the consolidated balance sheets and within research and development expenses on the consolidated statements of operations and comprehensive loss.
The Company accrues for these costs based on factors such as estimates of the work completed and in accordance with agreements established with its third-party service providers. The Company makes significant judgments and estimates in determining the expense liability under certain preclinical and clinical trial contracts where services have been performed butaccrued liabilities balance at the end of each reporting period. As actual costs become known, the Company adjusts its accrued liabilities. The Company has not yet invoiced. Generally, theexperienced any material differences between accrued costs and therefore estimates, associated with preclinical and clinical trial contracts are based on the number of animal subjects, samples or tissues requiring analyses, patients, drug administration cycles, the type of treatment and the outcome since the length of time before actual amounts can be determined will vary depending on the total samples requiring primary and repeated analyses, the length of the patient cycles and the timing of the invoices by the preclinical and clinical trial partners.costs incurred.
Share-based Compensation Expense
The Company records share-based compensation in accordance with ASC 718,
Stock Compensation.Compensation
. ASC 718 requires the fair value of all share-based compensation awarded to employees and
non-employees
to be recorded as an expense over the shorter of the service period or the vesting period. The Company determines employee and
non-employee
share-based compensation based on the grant-date fair value using the Black-Scholes Option Pricing Model.
Income Taxes
The Company is subject to Australia and United States income tax laws. The Company follows ASC 740,
Accounting for Income Taxes
, when accounting for income taxes, which requires an asset and liability approach to financial accounting and reporting for income taxes. Deferred income tax assets and liabilities are computed annually for temporary differences between the financial statements and tax bases of assets and liabilities that will result in taxable or deductible amounts in the future based on enacted tax laws and rates applicable to the periods in which the differences are expected to affect taxable income. Valuation allowances are established when necessary to reduce deferred tax assets to the amount more likely than not to be realized.
For uncertain tax positions that meet a “more likely than not” threshold, the Company recognizes the benefit of uncertain tax positions in the consolidated financial statements. The Company’s practice is to recognize interest and penalties, if any, related to uncertain tax positions in income tax expense in the consolidated statements of operations.
Recent Accounting Pronouncements
In June 2016, the FASB issued ASU
No. 2016-13:
Financial Instruments—Credit Losses (Topic 326).
This ASU represents a significant change in the accounting for credit losses model by requiring immediate recognition of management’s estimates of current expected credit losses (CECL). Under the prior model, losses were recognized only as they were incurred. The Company has determined that it has met the criteria of a smaller reporting company (“SRC”) as of November 15, 2019. As such, ASU
2019-10:
Financial Instruments-Credit Losses, Derivatives and Hedging, and Leases: Effective Dates amended the effective date for the Company to be for reporting periods beginning after December 15, 2022. The Company will adoptadopted this ASU effective July 1, 2023.2023 and determined that its impact on the accompanying consolidated financial statements is immaterial.
3. Going ConcernLiquidity
The accompanying consolidated financial statements have been prepared assuming that the Company will continue as a going concern. For the ninethree months ended March 31,September 30, 2023, and 2022, the Company incurred a net loss of $14.9$6.0 million and $13.1$5.1 million, and used net cash of $13.9$4.6 million and $11.0$4.0 million in operations, respectively. The Company expects to continue to incur additional operating losses in the foreseeable future.
 
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The Company’s business focuses on the development of novel genetic medicines and, at this stage in the Company’s development, the Company has not established a source of revenue to cover its full operating costs, and as such, is dependent on funding operations through capital financing activities. As of March 31,September 30, 2023, the Company had $6.6$25.9 million in cash and cash equivalents. The Company has performed a review of its cash flow forecasts and has concludedbelieves that substantial doubt exists as to its ability to continue asthe current funding will be sufficient for a going concern.period of at least twelve months from the date of this report.
The Company’s ability to continue as a going concern is dependent upon its ability to generate revenue and obtain adequate financing. While the Company believes in its ability to generate revenue and raise additional funds, there can be no assurances to that effect. The financial statements do not include any adjustments to reflect the possible future effects on the recoverability and classification of assets or the amounts and classification of liabilities that might be necessary if the Company is unable to continue as a going concern due to unsuccessful product development or commercialization, or the inability to obtain adequate financing in the future.
4. Cash, cash equivalents, and restricted cash
 
   March 31,   June 30, 
(US$’000)  2023   2022 
Cash at bank  $6,551   $4,062 
Restricted cash   14    14 
 
 
 
 
 
 
 
 
Total  $6,565   $4,076 
 
 
 
 
 
 
 
 
 
(US$’000)  September 30,
2023		   June 30,
2023		 
Cash at bank
  $25,864   $2,477 
Restricted cash
   13    13 
   
 
 
   
 
 
 
Total
  $25,877   $2,490 
   
 
 
   
 
 
 
5. Prepaid and other assets

(US$’000)  March 31,
2023		   June 30,
2022		 
Prepaid expenses  $332   $871 
Prepaid expenses—OPMD Program   546      
Market value of listed shares   1    5 
Total other assets   879    876 
Less:
non-current
portion
   (105)    (135) 
 
 
 
 
 
 
 
 
 
Current portion  $774   $741 
 
 
 
 
 
 
 
 
 
(US$’000)  September 30,
2023		   June 30,
2023		 
Prepaid expenses
  $565   $1,280 
Market value of listed shares
   2    1 
   
 
 
   
 
 
 
Total other assets
   567    1,281 
Less: non-current portion
   (85)    (97) 
Current portion
  $482   $1,184 
6. Pr
opertyProperty and equipment, net
 
(US$’000)  March 31,
2023		   June 30,
2022		   September 30,
2023		   June 30,
2023		 
Software
  $6   $6   $6   $6 
Lab equipment
   1,343    1,343    1,343    1,343 
Computer hardware
   30    31    31    32 
Leasehold improvements
   24    24    24    24 
  
 
   
 
   
 
   
 
 
Total property and equipment, gross
   1,403    1,404    1,404    1,405 
Accumulated depreciation and amortization
   (1,297)    (1,182)    (1,336)    (1,318) 
  
 
   
 
   
 
   
 
 
Total property and equipment, net
  $106   $222   $68   $87 
  
 
   
 
   
 
   
 
 
Depreciation and
amortization
expense was $32$19 thousand and $115$42 thousand and for the three and nine months ended March 31,September 30, 2023 and $54 thousand and $161 thousand, respectively, for the same periods in 2022.September 30, 2022, respectively.
7. Trade and other payables
 
(US$’000)  March 31,
2023		   June 30,
2022		 
Trade payable  $635   $422 
Accrued license fees   116    120 
Accrued professional fees   53    131 
Accrued OPMD project costs   1,560    1,089 
Accrued consultant fees   55    47 
Other payables   36    71 
           
Total  $2,455   $1,880 
 
 
 
 
 
 
 
 
 
(US$’000)  September 30,
2023		   June 30,
2023		 
Trade payable
  $1,528   $1,140 
Accrued license fees
   2    109 
Accrued professional fees
   49    75 
Accrued clinical development project costs

Accrued consultant fees
   2,088
61
 
 
   1,750
88
 
 
Accrued offering costs
   26    — 
Other payables
   40    69 
   
 
 
   
 
 
 
Total
  $3,794   $3,231 
   
 
 
   
 
 
 
 
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8. Leases
The Company has entered into an operating lease for office space under an agreement that expires in 2025. The lease requires the Company to pay utilities, insurance, taxes and other operating expenses. The Company’s lease does not contain any residual value guarantees or material restrictive covenants.
The tables below show the changes during the ninethree months ended March 31,September 30, 2023:
 
(US$’000)  Operating
lease right-
of-
use assets		 
Balance at July 1, 2022  $771 
Amortization of right of use asset   (182) 
      
Operating lease
right-of-use
asset at March 31, 2023
  $589 
      
(US$’000)  Operating
lease right-
of-
use assets		 
Balance at July 1, 2023
  $526 
Amortization of right of use asset
   (63) 
   
 
 
 
Operating lease right-of-use asset at September 30, 2023
  $463 
   
 
 
 
 
(US$’000)  Operating
lease
liabilities		 
Balance at July 1, 2022  $811 
Principal payments on operating lease liabilities   (188) 
      
Operating lease liabilities at March 31, 2023   623 
Less:
non-current
portion
   (354) 
      
Current portion at March 31, 2023  $269 
      
(US$’000)  Operating
lease
liabilities		 
Balance at July 1, 2023
  $559 
Principal payments on operating lease liabilities
   (68) 
 
 
 
 
 
Operating lease liabilities at September 30, 2023
   491 
Less: non-current portion
   (211) 
   
 
 
 
Current portion at September 30, 2023
  $280 
   
 
 
 
As of March 31,September 30, 2023, the Company’s operating lease has a remaining lease term of 2.211.71 years and a discount rate of 4.67%. The maturities of the operating lease liabilities are as follows:
 
(US$’000)  March 31,
2023		 
2023  $292 
2024   303 
2025   62 
      
Total operating lease payments   657 
Less imputed interest   (34) 
      
Present value of operating lease liabilities  $623 
      
(US$’000)  September 30,
2023		 
2024
  $221 
2025
   291 
   
 
 
 
Total operating lease payments
   512 
Less imputed interest
   (21) 
   
 
 
 
Present value of operating lease liabilities
  $491 
   
 
 
 
The Company recorded lease liabilities and
right-of-use
lease assets for the lease based on the present value of lease payments over the expected lease term, discounted using the Company’s incremental borrowing rate. Rent expense was $0.1 million and $0.2$0.1 million for the three and nine months ended March 31,September 30, 2023 respectively, and $0.1 million and $0.2 million for the same periods in 2022.September 30, 2022, respectively.
 
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9. Stockholders’ equity
Common Stock
On December 8, 2021, the stockholders of the Company approved an amendment (the “Charter Amendment”) to the Company’s Amended and Restated Certificate of Incorporation to increase the total number of authorized shares of common stock of the Company from 10,000,000 to 40,000,000. The Charter Amendment was filed with the Secretary of State of the State of Delaware and became effective on December 17, 2021. On December 7, 2022, the stockholders of the Company approved another amendment to the Company’s Amended and Restated CertificateArticles of Incorporation to increase the number of authorized shares of common stock from 40,000,000 to 160,000,000. The Charter Amendment was filed with the Secretary of State of the State of Delaware and became effective December 9, 2022.
Warrants
On December 6, 2019, certain investors (the “Investors”)the Investors were issued 4 Purchase Warrants that were exercisable into 214,19012,600 fully paid shares of common stock should the Purchase Warrants be exercised in full (“Purchase Warrants”). The exercise price for the Purchase Warrants is US$10.50178.50 per share issued on exercise of a Purchase Warrant. The Purchase Warrants are exercisable, in whole or in part, any time from the date of issue until the fifth anniversary of the date of issue (December 6, 2024). On April 22, 2020, the Company issued 37,4172,201 shares of common stock in connection with a cashless exercise of Purchase Warrants exercisable for 107,0956,300 shares of common stock. The Company did not have an effective registration statement registering the resale of the Warrant Shares by the Holder at the time the Holder wanted to exercise the warrant; therefore, the Holder carried out a cashless exercise. The formula for conducting a cashless exercise was outlined in the Warrant agreement. Based on this formula, the Holder would have been entitled to receive 6,300 shares of common stock if they had exercised the Purchase Warrants for cash. Because of the cashless exercise, the holder received 2,201 shares.
The activity related to warrants during the nine months ended March 31, 2023, is summarized as follows:
       Weighted- 
       average 
       Exercise 
   Common   Price 
   Stock from   (per 
   Warrants   share) 
Outstanding at July 1, 2022   107,095   $10.50 
Pre-funded
warrants issued September 15, 2022
   12,171,628   $0.0001 
Series 2 Warrants issued September 15, 2022   29,809,471   $0.66 
           
Outstanding at September 30, 2022   42,088,194   $0.49 
Pre-funded
warrants exercised
   2,171,628   $0.0001 
           
Outstanding at March 31, 2023   39,916,566   $0.52 
           
Exercisable at March 31, 2023   39,916,566   $0.52 
           
On September 15, 2022, we closed an underwritten public offering in which we issued and sold (i) 17,637,8431,037,520 shares of the Company’s common stock, (ii) 12,171,628
pre-funded
warrants, with each
pre-funded
warrant immediatelywhich, after giving effect to the Reverse Stock Split, are currently exercisable for one shareinto 715,979 shares of common stock at an exercise price of $0.0001
$0.0017 per share until exercised in full and (iii) 29,809,471 common warrants, the
Series 2 Warrants, with each common warrant accompanying each issued share of common stock and/orwarrants (the “Series 2 Warrants”),
pre-fundedwhich, after 
warrant andgiving effect to the Reverse Stock Split, are currently exercisable for one shareinto
1,753,503
shares of common stock at an exercise price of $0.66
$11.22 per share. The Series 2 warrants sold in the offering became exercisable commencing December 9, 2022, the date on which the Company had both (a) received approval from its stockholders to increase the number of shares of common stock it is authorized to issue and (b) effected such stockholder approval by filing with the Secretary of State of the State of Delaware a certificate of amendment to its
Amended and Restated Certificate of Incorporation, and will expire on the fifth anniversary of such
initial exercise date. The combined purchase price for each share of common stock and accompanying common warrant was $0.60,$10.20, which was allocated as $0.59$10.03 per share of common stock and $0.01$0.17 per common warrant. The Series 2 Warrants contain an exercise price adjustment mechanism providing that certain issuances of common stock (or common stock equivalents), if made at a price lower than the then existing exercise price of such Series 2 Warrants would reset the exercise price to such lower price. As a result of the August 11, 2023 public offering, the exercise price of the Series 2 Warrants has been automatically reset as of the closing time of such public offering to $1.9299.
On October 17, 2022 and October 27, 2022, investors exercised 2,004,961117,939 and 166,667
9,804 pre-funded
warrants, respectively, at an exercise price of $0.0017 per share.
On August 11, 2023 we closed an underwritten public offering in which we sold 875,949 shares of common stock, 15,126,226 pre-funded warrants to purchase 15,126,226 shares of common stock, and 16,002,175 common warrants to purchase up to 16,002,175 shares of common stock. The combined purchase price for each share of common stock and accompanying common warrant was $1.93, which was allocated as $1.9299 per share of common stock and $0.0001 per share.common warrant. Each pre-funded warrant was sold together with one common warrant at a combined price of $1.9299, which was allocated as $1.9298 per pre-funded warrant and $0.0001 per common warrant. The pre-funded warrants were immediately exercisable until exercised in full at an exercise price of $0.0001 per share of common stock. The common warrants were immediately exercisable at an exercise price of $3.86 per share of common stock and will expire on the fifth anniversary of such initial exercisable date. In addition, the Company granted the underwriter an option to purchase up to 2,331,606 additional shares of common stock and/or up to 2,331,606 additional common warrants. As of September 
30
, 2023, the underwriter had partially exercised this option and purchased 458,134 additional shares of common stock and 458,134 additional common warrants. Net proceeds from the offering, including the impact of the underwriter’s partial exercise of its option and net of underwriting discounts, commissions, and other offering expenses, totaled $27.9 million.
As of March 31,September 30, 2023, there were 39,916,56633,476,440 warrants outstanding.
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Table of Contents

The activity related to warrants for the three months ended September 30, 2023, is summarized, after giving effect to the Reverse Stock Split, as follows:
   Common
Stock from
Warrants		   Weighted-
average
Exercise
Price
(per
share)		 
Outstanding at July 1, 2023
   2,348,039   $1.92 
Pre-funded warrants issued August 11, 2023
   15,126,226   $0.0001 
Common warrants issued August 11, 2023   16,002,175   $3.86 
Outstanding at September 30, 2023
   33,476,440   $1.98 
Exercisable at September 30, 2023
   33,476,440   $1.98 
Equity Incentive Plan
Employee Share Option Plan
In connection with its
re-domiciliation
to the United States, in April 2020, the Company assumed BBL’s obligations with respect to the settlement of options that were issued by BBL prior to the
re-domiciliation
Re- domiciliation pursuant to the Benitec Officers’ and Employees’ Share Option Plan (the “Share Option Plan”“Plan”). This includes the Company’s assumptionassumptions of the Share Option Plan and all award agreements pursuant to which each of the options were granted. Each option when exercised entitles the option holder to one share in the Company. Options are exercisable on or before an expiry date, do not carry any voting or dividend rights and are not transferable except on death of the option holder or in certain other limited circumstances.circumstances . Employee options vest
one-third
one third on each anniversary of the applicable grant date for three years. If an employee dies, retires or otherwise leaves the Companyorganization, and certain exerciseother conditions have been satisfied, generally the employee has 12 months to exercise their options, or the options are cancelled. SinceAfter the
re-domiciliation,
Re-domiciliation, no new options have been or will be issued under the Share Option Plan.
Equity and Incentive Compensation Plan
On December 9, 2020, the Company’s stockholders approved the Company’s 2020 Equity and Incentive Compensation Plan and, on December 8, 2021, the Company’s stockholders approved an amendment to increase the maximum number of shares that may be issued under such plan to 1,850,000 (as amended, the(the “2020 Plan”). The 2020 Plan provides for the grant of various equity awards. Currently, only stock options are issuedoutstanding under the 2020 Plan. Each option when exercised entitles the option holder to one share of the Company’s common stock. Options are exercisable on or before an expiry date, do not carry any voting or dividend rights, and are not transferable except on death of the option holder or in certain other limited circumstances. Employee stock options vest in increments of
one-third
on each anniversary of the applicable grant date forover three years.
Non-employee
director options vest in increments of
one-third
on the day prior to each of the Company’s next three annual stockholder meetings following the grant date. If an option holder dies or terminates employment or service due to Disability (as defined in the 2020 Plan) and certain exercise conditions have been satisfied, generally,, the option holder generally has 12 months to exercise their vested options, or the options are cancelled. If an option holder otherwise leaves the Company, other than for a termination by the Company for Cause (as defined in the 2020 Plan) and certain exercise conditions have been satisfied, generally,, the option holder generally has 90 days to exercise their vested options, or the options are cancelled. FutureThe maximum contractual term of options granted under the 2020 Plan is ten years. Upon the consummation of a Change in Control (as defined in the 2020 Plan), all unvested stock options will immediately vest as of immediately prior to the Change in Control.
On December 8, 2021, the Company’s stockholders approved an amendment to the 2020 Plan, which increased the number of shares of the Company’s common stock reserved under the 2020 Plan to 1,850,000. For the fiscal year ended June 30, 2023, our named executive officers (“NEO’s”) were each granted equity grants will be madeincentive awards under the 2020 Plan.
 
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14
Table of Contents
Equity Awards
The activity related to equity awards, which are comprised of stock options during the ninethree months ended March 31,September 30, 2023 is summarized as follows:
 
           Weighted-     
       Weighted-   average     
       average   Remaining   Aggregate 
   Stock   Exercise   Contractual   Intrinsic 
   Options   Price   Term   Value 
Outstanding at June 30, 2022   738,064   $6.95    7.18 years   $—   
                     
Expired   5,665   $45.92           
                     
Granted   36,000   $0.17    9.93 years      
Outstanding at March 31, 2023   768,399   $6.35    6.64 years   $1,501 
                     
Exercisable at March 31, 2023   456,688   $8.58    6.46 years   $—   
                     

   
Stock
Options
   
Weighted-
average
Exercise
Price
   
Weighted-
average
Remaining
Contractual
Term
   
Aggregate
Intrinsic
Value
 
Outstanding at June 30, 2023
   107,993   $31.88    8.96 years   $11,888 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Outstanding at September 30, 2023
   107,993   $31.88    8.70 years   $342 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Exercisable at September 30, 2023
   27,135   $88.79    6.97 years   $—  
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Share-Based Compensation Expense
The classification of share-based compensation expense is
summarized
as follows:
 
   Three Months Ended   Nine Months Ended 
   March 31, 2023 
(US$’000)  2023   2022   2023   2022 
Research and development  $27   $63   $87   $224 
General and administrative   44    129    238    478 
Total share-based compensation expense  $71   $192   $325   $702 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
   Three Months Ended
September 30,		 
(US$’000)  2023   2022 
Research and development
  $34   $30 
General and administrative
   57    272 
   
 
 
   
 
 
 
Total share-based compensation expense
  $91   $302 
   
 
 
   
 
 
 
As of March 31,September 30, 2023, there was $0.2$0.3 million of unrecognized share-based compensation expense related to stock options issued under the Share Option Plan and the 2020 Plan.Plan, which is expected to be recognized over a weighted average period of 2.3 years.
10. Income taxes
For the three and nine months ended March 31,September 30, 2023, and March 31, 2022 respectively, the Company did not recognize a provision or benefit for income taxes as it has incurred net losses. In addition, the net deferred tax assets generated from net operating losses are fully offset by a valuation allowance as the Company believes it is not more likely than not that the benefit will not be realized.

11. Commitments and contingencies
Contract commitments
The Company enters into contracts in the normal course of business with third-party contract research organizations, contract development and manufacturing organizations and other service providers and vendors. These contracts generally provide for termination on notice and, therefore, are cancellable contracts and not considered contractual obligations and commitments.
Contingencies
From time to time, the Company may become subject to claims and litigation arising in the ordinary course of business. The Company is not a party to any material legal proceedings, nor is it aware of any material pending or threatened litigation.
12. Related party transactions
During the nine monthsthree month periods ended March 31,September 30, 2023 and September 30, 2022, the Company did not enter into any related party transactions. As of March 31, 2022,
13. Subsequent events
There were
no
subsequent events for the Company had entered into a related party transaction with Francis Abourizk Lightowlers for legal fees totaling $1 thousand. Peter Francis, aperiod end September 30, 2023.
non-executive
director at the Company, is a partner at Francis Abourizk Lightowlers. As of March 31, 2022 amounts due to this related party of $1 thousand were included in trade and other payables on the accompanying consolidated balance sheet.
 
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15

Item 2.

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

You should read the following discussion and analysis of financial condition and operating results together with our consolidated financial statements and the related notes and other financial information included elsewhere in this document.

Company Overview

We endeavor to become the leader in discovery, development, and commercialization of therapeutic agents capable of addressing significant unmet medical need via the application of the silence and replace approach to the treatment of genetic disorders.

Benitec Biopharma Inc. (“Benitec” or the “Company” or in the third person, “we” or “our”) is a development-stageclinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary platform, called DNA-directed RNA interference, or ddRNAi, combines RNA interference, or RNAi, with gene therapy to create medicines that facilitate sustained silencing of disease- causingdisease-causing genes following a single administration. The unique therapeutic constructs also enable the simultaneous delivery of wildtype replacement genes, facilitating the proprietary “silence and replace” approach to the treatment of genetically defined diseases. The Company is developing a ddRNAi-basedsilence and replace-based therapeutic (BB-301) for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD), a chronic, life-threatening genetic disorder.

BB-301 is a ddRNAi-basedsilence and replace-based genetic medicine currently under development by Benitec. BB-301 is an AAV-based gene therapy designed to simultaneouslypermanently silence the expression of the mutant, disease-causing gene (to slow, or halt, the biological mechanisms underlying disease progression in OPMD) and to simultaneously replace the mutant gene with a wild typewildtype gene (to drive restoration of function in diseased cells). This fundamental therapeutic approach to disease management is called “silence and replace”.replace.” The silence and replace mechanism offers the potential to restore the normative physiology of diseased cells and tissues and to improve treatment outcomes for patients suffering from the chronic, and potentially fatal, effects of OPMD. BB-301 has been granted Orphan Drug Designation in the United States and the European Union.

The targeted gene silencing effects of RNAi, in conjunction with the durable transgene expression achievable via the use of modified viral vectors, imbues the silence and replace approach with the potential to produce long-termpermanent silencing of disease-causing genes along with simultaneous replacement of the wild type gene function following a single administration of the proprietary genetic medicine. We believe that this novel mechanistic profile of the current and future investigational agents developed by Benitec could facilitate the achievement of robust and durable clinical activity while greatly reducing the frequency of drug administration traditionally expected for medicines employed for the management of chronic diseases.

Additionally, the achievement of long-termpermanent gene silencing and gene replacement may significantly reduce the risk of patient non-compliance during the course of medical management of potentially fatal clinical disorders.

We will require additional financing to progress our product candidates through to key inflection points.

Our proprietary technology platforms are designated as DNA-directed RNA interference, or “ddRNAi”, and “silence and replace.” ddRNAi is designed to produce permanent silencing of disease-causing genes, by combining RNA interference, or RNAi, with viral delivery agents typically associated with the field of gene therapy (i.e., viral vectors). Modified AAV vectors are employed to deliver genetic constructs which encode short hairpin RNAs that are, then, serially expressed and processed to produce siRNA molecules within the transduced cell for the duration of the life of the target cell. These newly introduced siRNA molecules drive

16

permanent silencing of the expression of the disease-causing gene. The silence and replace approach further bolsters the biological benefits of permanent silencing of disease-causing genes by incorporating multifunctional genetic constructs within the modified AAV vectors to create an AAV-based gene therapy agent that is designed to silence the expression of disease-causing genes (to slow, or halt, the underlying mechanism of disease progression) and to simultaneously replace the mutant genes with normal, “wildtype” genes (to drive restoration of function in diseased cells). This fundamentally distinct therapeutic approach to disease management offers the potential to restore the underlying physiology of the treated tissues and, in the process, improve treatment outcomes for patients suffering from the chronic and, potentially, fatal effects of diseases like Oculopharyngeal Muscular Dystrophy (OPMD).

Traditional gene therapy is defined by the introduction of an engineered transgene to correct the pathophysiological derangements derived from mutated or malfunctioning genes. Mutated genes can facilitate the intracellular production of disease-causing proteins or hamper the production of critical, life-sustaining, proteins. The introduction of a new transgene can facilitate the restoration of production of normal proteins within the diseased cell, thus restoring natural biological function. Critically, the implementation of this traditional method of gene therapy cannot eliminate the expression, or the potential deleterious effects of, the underlying mutant gene (as mutant proteins may be continually expressed and aggregate or drive the aggregation of other native proteins within the diseased cell). In this regard, the dual capabilities of the proprietary silence and replace approach to silence a disease-causing gene via ddRNAi and simultaneously replace the wild type activity of a mutant gene via the delivery of an engineered transgene could facilitate the development of differentially efficacious treatments for a range of genetic disorders.

COVID-19Overview of RNAi and the siRNA Approach

COVID-19The mutation of a single gene can cause a chronic disease via the resulting intracellular production of a disease-causing protein (i.e., an abnormal form of the protein of interest), and many chronic and/or fatal disorders are known to result from the inappropriate expression of a single gene or multiple genes. In some cases, genetic disorders of this type can be treated exclusively by “silencing” the intracellular production of the disease-causing protein through well-validated biological approaches like RNA interference (“RNAi”). RNAi employs small nucleic acid molecules to activate an intracellular enzyme complex, and this biological pathway temporarily reduces the production of the disease-causing protein. In the absence of the disease-causing protein, normal cellular function is restored and the chronic disease that initially resulted from the presence of the mutant protein is partially or completely resolved. RNAi is potentially applicable to over 20,000 human genes and a large number of disease-causing microorganism-specific genes.

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Figure 1

LOGO

A small double stranded RNA, or dsRNA, molecule (A, Figure 1), comprising one strand known as the sense strand and another strand known as the antisense strand, which are complementary to each other, is synthesized in the laboratory. These small dsRNAs are called small interfering RNAs, or siRNAs. The sequence of the sense strand corresponds to a short region of the target gene mRNA. The siRNA is delivered to the target cell (B, Figure 1), where a group of enzymes, referred to as the RNA-Induced Silencing Complex, or RISC, process the siRNA (C, Figure 1), where one of the strands (usually the sense strand) is released (D, Figure 1). RISC uses the antisense strand to find the mRNA that has a complementary sequence (E, Figure 1) leading to the cleavage of the target mRNA (F, Figure 1). As a consequence, the output of the mRNA (protein production) does not occur (G, Figure 1). Several companies, including Alnylam Pharmaceuticals Inc. (“Alnylam”), utilize this approach in their RNAi product candidates.

Importantly, many genetic disorders are not amenable to the traditional gene silencing approach outlined in Figure 1, as the diseased cells may produce a mixture of the wild type protein of interest and the disease-causing mutant variant of the protein, and the underlying genetic mutation may be too small to allow for selective targeting of the disease-causing variant of the protein through the use of siRNA-based approaches exclusively. In these cases, it is extraordinarily difficult to selectively silence the disease-causing protein without simultaneously silencing the wild type intracellular protein of interest whose presence is vital to the conduct of normal cellular functions.

Our proprietary silence and replace technology utilizes the unique specificity and robust gene silencing capabilities of RNAi while overcoming many of the key limitations of siRNA-based approaches to disease management.

In the standard RNAi approach, double-stranded siRNA is produced synthetically and, subsequently, introduced into the target cell via chemical modification of the RNA or alternative methods of delivery. While efficacy has been declared a pandemic bydemonstrated in several clinical indications through the World Health Organization and has spread to nearly every country, including Australia and the United States. The impactuse of this pandemic has been,approach, siRNA-based approaches maintain a number of limitations, including:

Clinical management requires repeat administration of the siRNA-based therapeutic agent for multiple cycles to maintain efficacy;

18

Long-term patient compliance challenges due to dosing frequencies and will likely continuetreatment durations;

Therapeutic concentrations of siRNA are not stably maintained because the levels of synthetic siRNA in the target cells decrease over time;

Novel chemical modifications or novel delivery materials are typically required to be, extensiveintroduce the siRNA into the target cells, making it complicated to develop a broad range of therapeutics agents;

Potential adverse immune responses, resulting in many aspectsserious adverse effects;

Requirement for specialized delivery formulations for genetic disorders caused by mutations of society, which has resulted in,multiple genes; and will likely continue

siRNA acts only to result in, significant disruptions to businesses and capital markets around the world. The extent to which the coronavirus impacts us will depend on future developments, which are highly uncertainsilence genes and cannot be predicted, including new information which may emerge concerningused to replace defective genes with normally functioning genes.

Our Approach to the severityTreatment of the coronavirusGenetic Diseases—ddRNAi and its variants,Silence and the actions to contain the coronavirus or treat its impact, including the effectiveness and adoption of vaccines for the virus, among others.Replace

Certain elements of our research and development efforts are conducted globally, including the ongoing development of ourOur proprietary silence and replace approach to the treatment of genetic diseases combines RNAi with wild type gene replacement to drive permanent silencing of disease-causing genes and concomitant restoration of functional wild type genes following a single administration of the therapeutic agent. Benitec employs ddRNAi in combination with classical gene therapy (i.e., transgene delivery via viral vectors) to overcome several of the fundamental limitations of RNAi.

The silence and replace approach to the treatment of genetic disorders employs adeno-associated viral vectors (“AAVs”) to deliver genetic constructs which may, after a single administration to the target tissues:

Chronically express RNAi molecules inside of the target, diseased, cells (to serially silence the intracellular production of mutant, disease- causing, protein and the wild type protein of interest);

Simultaneously drive the expression of a wild type variant of the protein of interest (to restore native intracellular biological processes); and

AAV vectors can accommodate the multi-functional DNA expression cassettes containing the engineered wild type transgenes and the novel genes encoding short hairpinRNA/microRNA molecules (shRNA/miRNA) that are required to support the development of therapeutic agents capable of the achievement of the goals of the silence and replace approach to therapy.

Our silence and replace technology utilizes proprietary DNA expression cassettes to foster continuous production of gene silencing shRNAs and wild type proteins (via expression of the wild type transgene). A range of viral and non-viral gene therapy vectors can be used to deliver the DNA construct into the nucleus of the target cell and, upon delivery, shRNA molecules are expressed and subsequently processed by intracellular enzymes into siRNA molecules that silence the expression of the mutant, disease-causing protein (Figure 2).

In the silence and replace approach (Figure 2):

A DNA construct is delivered to the nucleus of the target cell by a gene therapy vector (A) such as an AAV vector;

Once inside of the nucleus, the DNA construct drives the continuous production of shRNA molecules (B) which are processed by an enzyme called Dicer into siRNAs (C);

The processed siRNA is incorporated into RISC and silences the target gene using the same mechanism shown in Figure 1; and

When the DNA expression cassette is additionally comprised of a wild type transgene, upon entry of the DNA construct into the nucleus of the target cell via the use of the AAV vector, the DNA construct also drives the continuous production of wild type protein (to restore native intracellular biological processes).

19

Figure 2

LOGO

Our strategy is to discover, develop and commercialize treatments that leverage the capabilities of ddRNAi and the silence and replace approach to disease management.

For selected product candidates, at the appropriate stage, we may collaborate with large biopharmaceutical companies to further co-develop and, if approved, commercialize our ddRNAi-based and silence and replace-based products to achieve broad clinical and commercial distribution. For specific clinical indications that we deem to be outside of our immediate areas of focus, we will continue to out-license, where appropriate, applications of our ddRNAi and silence and replace technology to facilitate the development of differentiated therapeutics, which could provide further validation of our proprietary technology and approach to disease management.

Our cash and cash equivalents will be deployed for the advancement of our product candidate BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD), and will be dependent upon our ability to complete preclinical studies and initiate clinical studies despite the ongoing COVID-19 pandemic.

As we endeavor to complete our development programs, including the ongoing Toxicology and Biodistribution and Natural History studies for BB-301, we are in close contact with our principal investigators, contract research organizations, and preclinical trial sites, which are located in the United States, Canada, and France, and are assessing the impact of COVID-19 on our studies and the expected development timelines and costs on an ongoing basis. In light of developments relating to the COVID-19 global pandemic since the beginning of the outbreak, the focus of healthcare providers and hospitals on fighting the virus, and consistent with the FDA’s industry guidance for conducting clinical trials, we have experienced delays to the original timeline regarding the initiation and anticipated completion of the ongoing BB-301 Clinical Trial Application (CTA)-enabling and Investigational New Drug Application (IND)-enabling development work. The initiation of the BB-301 Pilot Dosing Study in Beagle dogs, which represents a key component of the CTA- enabling and IND-enabling work, was delayed by several months, however, the study has been completed without incident. The acquisition of chemical reagents, biological reagents and laboratory supplies which are essential for the conduct of basic laboratory research, the conduct of nonclinical studies and the

15

completion of GMP manufacturing of BB-301, has also become challenging due to the disruption of global supply chains inherent to the production of these materials. We will continue to evaluate the impact of the COVID-19 pandemic on our business and we expect to reevaluate the timing of our anticipated preclinical and clinical milestones as we learn more and the impact of COVID-19 on our industry becomes clearer.

We have also implemented a halt of non-essential business travel and a rotation system whereby staff work from home and attend the laboratory on designated days which may result in a reduction of laboratory work. As we transition our employees back to our premises, there is a risk that COVID-19 infections occur at our offices or laboratory facilities and significantly affect our operations. Additionally, if any of our critical vendors are impacted, our business could be affected if we become unable to procure essential equipment in a timely manner or obtain supplies or services in adequate quantities and at acceptable prices.

Public Equity Offering

On September 15, 2022, we closed an underwritten public offering in which we issued and sold (i) 17,637,843 shares of the Company’s common stock, (ii) 12,171,628 pre-funded warrants, with each pre-funded warrant immediately exercisable for one share of common stock at an exercise price of $0.0001 per share until exercised in full and (iii) 29,809,471 common warrants, with each common warrant accompanying each issued share of common stock and/or pre-funded warrant and exercisable for one share of common stock at an exercise price of $0.66 per share (the “September 2022 Capital Raise”). The common warrants sold in the offering became exercisable on December 9, 2022, the date by which the Company (a) received approval from its stockholders to increase the number of shares of common stock it is authorized to issue and (b) effected such stockholder approval by filing with the Secretary of State of the State of Delaware a certificate of amendment to its amended and restated certificate of incorporation, and will expire on the fifth anniversary of such initial exercise date. The combined purchase price for each share of common stock and accompanying common warrant was $0.60, which was allocated as $0.59 per share of common stock and $0.01 per common warrant.

The net proceeds to the Company from the public offering were approximately $16 million, after deducting underwriting discounts and commissions and public offering expenses payable by the Company, and excluding any proceeds the Company may receive upon exercise of the pre-funded warrants or the common warrants. The Company currently intends to use the net proceeds for the clinical development of BB-301,OPMD-derived dysphagia, including the natural history lead-in study and the Phase 1b/2a BB-301 treatment study, for the continued advancement of development activities for other existing and new product candidates, for general corporate purposes and for strategic growth opportunities.

Oculopharyngeal Muscular Dystrophy—OPMD

OPMD is an insidious, autosomal-dominant, late-onset degenerative muscle disorder that typically presents in patients at 40-to-50 years of age. The Company willdisease is characterized by progressive swallowing difficulties (dysphagia) and eyelid drooping (ptosis). OPMD is caused by a specific mutation in the poly(A)-binding protein nuclear 1, or PABPN1, gene. OPMD is a rare disease; however, patients have broad discretionbeen diagnosed with OPMD in determining howat least 33 countries. Patient populations suffering from OPMD are well-identified, and significant geographical clustering has been noted for patients with this disorder, which could simplify clinical development and global commercialization efforts.

BB-301 is an AAV-based gene therapy designed to silence the proceedsexpression of disease-causing genes (to slow, or halt, the underlying mechanism of disease progression) and to simultaneously replace the mutant genes with normal, “wildtype” genes (to drive restoration of function in diseased cells). This fundamental therapeutic approach to disease management is called “silence and replace” and this biological mechanism offers the potential to restore the underlying physiology of the public offeringtreated tissues and, in the process, improve treatment outcomes for patients suffering from the chronic and, potentially, fatal effects of Oculopharyngeal Muscular Dystrophy (OPMD). BB-301 has been granted Orphan Drug Designation in the United States and the European Union.

20

Our Strengths

We believe that the combination of our proprietary ddRNAi and silence and replace technology, and our deep expertise in the design and development of genetic medicines, will be used,enable us to achieve and its discretion is not limitedmaintain a leading position in gene silencing and gene therapy for the treatment of human disease. Our key strengths include:

A first mover advantage for silence and replace-based therapeutics;

A proprietary ddRNAi-based silence and replace technology platform that may potentially enable the serial development of single-administration therapeutics capable of facilitating sustained, long-term silencing of disease-causing genes and concomitant replacement of wild type gene function;

A proprietary AAV vector technology which improves the endosomal escape capability of virus produced in insect cells using a baculovirus system. This technology has broad application in AAV-based gene therapies;

The capabilities to drive the development of a pipeline of programs focused on chronic diseases with either large patient populations, or rare diseases, which may potentially support the receipt of Orphan Drug Designation, including OPMD; and

A growing portfolio of patents protecting improvements to our ddRNAi, and silence and replace, technology and product candidates through at least 2036, with additional patent life anticipated through at least 2040.

Our Strategy

We endeavor to become the leader in discovery, development, and commercialization of silence and replace-based therapeutic agents. We apply the following general strategy to drive the Company towards these goals:

Selectively develop proprietary and partnered programs; and

Continue to explore and secure research and development partnerships with global biopharmaceutical companies supported by the aforementioned possible uses.differentiated nature of our scientific platform and intellectual property portfolio.

Nasdaq Listing

On September 6, 2022,Our senior leadership team will continue to explore partnership opportunities with global biopharmaceutical companies, as we received a letter fromexpect that the Listing Qualifications Departmentunique attributes of the Nasdaq Stock Market notifying us thatproprietary ddRNAi and silence and replace approaches, and the minimum bid price per share forbreadth of potential clinical indications amenable to our common stock fell below $1.00 forproprietary methods, to support the formation of collaborations over a periodbroad range of 30 consecutive business daysdiseases with significant unmet medical need.

We seek to actively protect our intellectual property and therefore we did not meetproprietary technology. These efforts are central to the minimum bid price requirement set forth in Nasdaq Listing Rule 5550(a)(2).

The letter also states that we will be provided 180 calendar days, or until March 6, 2023, to regain compliance with the minimum bid price requirement. In accordance with Rule 5810(c)(3)(A), we can regain compliance if at any time during the 180-day period the closing bid pricegrowth of our common stock is at least $1.00 for a minimum of 10 consecutive business days. On March 7, 2023, Nasdaq notifiedand include:

Seeking and maintaining patents claiming our ddRNAi and silence and replace technologies and other inventions relating to our specific products in development or that are otherwise commercially and/or strategically important to the Company in writing that while the Company had not regained compliance with the minimum bid price requirement, it was eligible for an additional 180 calendar day period, or until September 5, 2023, to regain compliance with the minimum bid price requirement. Nasdaq’s determination was based on the Company having met the continued listing requirement for market value of publicly held shares and all other applicable requirements for initial listing on The Nasdaq Capital Market, with the exception of the minimum bid price requirement and on the Company’s written notice to Nasdaq of its intention to cure the deficiency during the second compliance period by effecting a reverse stock split, if necessary. If at any time during this additional time period the closing bid price of the Company’s common stock is at least $1.00 per share for a minimum of 10 consecutive business days, Nasdaq will provide written confirmation of compliance and the matter will be closed.

We intend to monitor the closing bid pricedevelopment of our common stockbusiness;

Protecting and may, if appropriate, consider implementing available optionsenforcing our intellectual property rights; and

Strategically licensing intellectual property from third parties to regain compliance with the minimum bid price requirement. These options include completing a reverse stock splitadvance development of our common stock for the purpose of meeting the closing bid price requirement. We received stockholder approval to effect a reverse stock split at our Annual Meeting of Stockholders on December 7, 2022 but currently have no plans to proceed. Such approval allows our board of directors, in its discretion, to elect to implement such reverse stock split at any time prior to December 7, 2023. Completing a reverse stock split will not, in of itself, cause us to remain in compliance with Nasdaq’s listing standards.product candidates.

Development Programs21

Our Pipeline

The following table sets forth theour current product candidate and the development status:

Table 1. Pipeline: Oculopharyngeal Muscular Dystrophy

 

LOGO

BB-301LOGO

We are developing BB-301 for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD). The Investigational New Drug (IND) application for BB-301 was approved to proceed by the U.S. Food and Drug Administration in June 2023. BB-301 is the lead investigational agent under development by Benitec, and the key attributes of OPMD and BB-301 are outlined in Figure 3.

Figure 3. Overview of the BB-301 Program3

 

LOGOLOGO

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BB-301 is a first-in-class genetic medicine employing the “silence and replace” approach for the treatment of OPMD. OPMD is an insidious, autosomal- dominant,autosomal-dominant, late-onset, degenerative muscle disorder that typically presents in patients at 40-to-50 years of age. The disease is characterized by progressive swallowing difficulties (dysphagia) and eyelid drooping (ptosis). OPMD is caused by a specific mutation in the poly(A)-binding protein nuclear 1 gene (PABPN1).

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OPMD is a rare disease, however, patients have been diagnosed with OPMD in at least 33 countries. Patient populations suffering from OPMD are well- identified,well-identified, and significant geographical clustering has been noted for patients with this disorder. Each of these attributes could facilitate efficient clinical development and global commercialization of BB-301.

PABPN1 is a ubiquitous factor that promotes the interaction between the poly(A) polymerase and CPSF (cleavage and polyadenylation specificity factor) and, thus, controls the length of mRNA poly(A) tails, mRNA export from the nucleus, and alternative poly(A) site usage. The characteristic genetic mutation underlying OPMD results in trinucleotide repeat expansion(s) within exon 1 of PABPN1 and results in an expanded poly-alanine tract at the N-terminal end of PABPN1. The mutation generates a protein with an N-terminal expanded poly-alanine tract of up to 18 contiguous alanine residues, and the mutant protein is prone to the formation of intranuclear aggregates designated as intranuclear inclusions (INIs). The INIs that sequester wildtype PABPN1 may contribute to the “loss of function” phenotype associated with OPMD.

No therapeutic agents are approved for the treatment of OPMD. Additionally, there are no surgical interventions available to OPMD patients that modify the natural history of the disease, which is principally comprised of chronic deterioration of swallowing function. BB-301 has received Orphan Drug Designation in the United States and the European Union and, upon achievement of regulatory approval for BB-301 in these respective jurisdictions, the Orphan Drug Designations would provide commercial exclusivity independent of intellectual property protection. While OPMD is a rare medical disorder, we believe the commercial opportunity for a safe and efficacious therapeutic agent in this clinical indication exceeds $1 billion over the course of the commercial life of the product.

Investigational therapies that have been explored, unsuccessfully, in the past include:

Intravenous administration of trehalose; and

The use of autologous myoblast transplant.

BB-301 is our Lead, Silence and Replace-Based, OPMD Therapeutic Agent

BB-301 is composed of a modified AAV serotype 9 (AAV9) capsid that expresses a bifunctional construct under the control of a single muscle specific Spc5-12 promoter to achieve co-expression of both the codon-optimized PABPN1 mRNA and two shmiR molecules directed against wild type and mutant PABPN1. BB-301 is designed to correct the genetic defect underlying OPMD following a single localized administration.

BB-301—Design and Mechanism of Action

BB-301 is designed to target two distinct regions of the PABPN1 mRNA to accomplish gene silencing via the concomitant expression of two distinct shmiRs from a single DNA construct (Figure 4). BB-301 is also engineered to drive the simultaneous expression of a codon-optimized, siRNA-resistant, version of the wild type PABPN1 gene (Figure 4).

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Figure 4

LOGO

In collaboration with researchers at the Royal Holloway University of London and the Institut de Myologie in Paris, we developed a ddRNAi construct expressing three shRNAs against three distinct regions of PABPN1 mRNA and observed effective silencing of the PABPN1 gene in vitro using this ddRNAi construct. Furthermore, as part of this collaboration, we have generated a gene expression construct that produces a siRNA-resistant version of the wild type PABPN1 gene.

In subsequent studies undertaken exclusively by Benitec, has previously outlineda second set of target regions within PABPN1 were identified for therapeutic development and shmiRs designed against these regions. Additional shmiRs have also been designed for the core CTA-enablingoriginal shRNA developed in collaboration with Royal Holloway University of London and IND-enablingthe Institut de Myologie. The ‘silence and replace’ construct, designated BB-301, incorporates the two best performing shmiRs, and the gene expression construct that produces a siRNA-resistant version of the wild type PABPN1 gene, under the control of a muscle-specific promoter. The mechanism of action of BB-301 is shown in Figure 5.

Figure 5

LOGO

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In initial in vivo studies requiredevaluating the use of direct intramuscular injection of AAV-based constructs with the potential to facilitate the desired silence and replace approach in the A17 transgenic mouse model of OPMD at the Royal Holloway University of London and the Institut de Myologie, we observed decreases in muscle fibrosis, increases in cross sectional area of the treated muscles, decreases in intranuclear inclusions, and normalization of muscle strength. These nonclinical results were published in Nature Communications in April

2017.

In subsequent studies, Benitec demonstrated in a key nonclinical model (the A17 mouse model) that a single intramuscular injection of BB-301 results in robust intracellular silencing of PABPN1 protein production and concomitant expression of the normal, biologically functional PABPN1 protein. In the A17 mouse model, the treatment restores muscle strength and muscle weight to wild type levels and improves other physiological hallmarks of the disease (Figure 6a, Figure 6b, Figure 6c, Figure 6d):

Multiple A17 animal cohorts received single doses of BB-301 (over a range of doses spanning 4x108 vg/muscle-to-7.5x1011 vg/muscle) and, following BB-301 administration, each cohort was observed for 14-weeks

BB-301 was injected into the Tibialis Anterior (TA) muscle of 10 week old-to-12 week old animals and, 14-weeks post administration, each A17 cohort was anesthetized and the contractile properties of the injected TA muscles were analyzed via in-situ muscle electrophysiology

Intermediate doses of BB-301 resulted in 75% silencing of PABPN1 and 26% replacement of wild type PABPN1 activity, leading to full restoration of muscle strength, clearance of INIs, and a reduction of fibrosis

An additional experiment conducted over the course of 20-weeks demonstrated that more modest doses of BB-301 (which supported only partial resolution of the disease phenotype at week-14) were, surprisingly, able to facilitate significant benefit at 20-weeks, as evidenced by global regulatory agenciesrestoration of parameters relating to supportmuscle strength, weight and INI formation

Figure 6a. Dose-Dependent shRNA Expression

LOGO

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Figure 6b. Dose-Dependent PABPN1 Inhibition and Transgene Expression

LOGO

Figure 6c. Dose-Dependent Decreases in Intranuclear Inclusions

LOGO

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Figure 6d. Dose-Dependent Increases in Muscle Force

LOGO

Restoration of muscle strength was assessed by muscle contractility measurements in response to a series of induced impulses that ranged from 10 to 180 Hz

Ongoing Development Activities for BB-301

On July 8, 2020, Benitec announced the initiation of BB-301 clinical trials in OPMD patients, and these studies include a BB-301 Pilot Dosing Study (the “Pilot Dosing Study”) in large animals and a classical 12-week GLP Toxicology and Biodistribution Study for BB-301. In these large animal studies, BB-301 is directly injected into the pharyngeal muscles known to underlie the morbidity and mortality which characterizes the natural history of OPMD in human subjects.

As referenced above, the BB-301 Pilot Dosing Study in large animalsanimal subjects.

The BB-301 Pilot Dosing Study was the first of two planned CTA-enabling and IND-enabling studies that were designed to be conducted by Benitec. This studyin large animals. The BB-301 Pilot Dosing Study was carried out under the guidance of the scientific team at Benitec, with key elements of the study design and execution of the study conducted in close collaboration with a team of leading experts in both medicine and surgery that have been deeply engaged in the treatment of OPMD patients for several decades. The BB-301 Pilot Dosing Study, andalong with the subsequent GLP Toxicology and Biodistribution Study, for BB-301were conducted in canine subjects in order to:

•

Support the validation and optimization of the newly designed route and method of BB-301 administration,

Confirmand were carried out to support the validation and optimization of the newly designed method of BB-301 administration, confirm the efficiency of vector transduction and transgene expression in the key tissue compartments underlying the morbidity and mortality that comprises the natural history of OPMD,

•

Confirm the optimal BB-301 doses in advance of initiation of human clinical studies,

Facilitate confirm the optimal drug doses in advance of initiation of human clinical studies, and facilitate observation of key toxicological data-points.

The BB-301 Pilot Dosing Study was designed as an 8-week study in Beagle dogs to confirm the transduction efficiency of BB-301 upon administration via direct intramuscular injection into specific anatomical regions of the pharynx through the use of an open surgical procedure. This new method and route of BB-301 administration was developed in collaboration with key surgical experts in the field of Otolaryngology, and this novel method of BB-301 dosing willwas implemented to significantly enhance the ability of a treating physiciansphysician to accurately administer the AAV-based investigational agent to the muscles that underlie the characteristic deficits associated with diseasethe progression inof OPMD. It is important to note that prior nonclinical studies of BB-301 non-clinical studies have reproducibly validated the robust biological activity achieved following direct intramuscular injection of the AAV-based agent.injection. As an example, direct injection of BB-301 into the tibialis anterior muscles of A17 mice facilitated robust transduction of the targeted skeletal muscle cells and supported complete remission of the OPMD disease phenotype in this animal model.

Benitec conducted the BB-301 Pilot Dosing Study in Beagle dog subjects to demonstrate that direct intramuscular injection of BB-301 via the use of a proprietary dosing device in an open surgical procedure could safely achieve the following goals:

 

•

Biologically significant and dose-dependent levels of BB-301 tissue transduction (i.e., delivery of the multi-functional BB-301 genetic construct into the target pharyngeal muscle cells),

Biologically significant and dose-dependent levels of BB-301 tissue transduction (i.e., delivery of the multi-functional BB-301 genetic construct into the target pharyngeal muscle cells);

 

•

Broad-based and dose-dependent expression of the three distinct genes comprising the BB-301 gene construct within the pharyngeal muscle cells, and

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Broad-based and dose-dependent expression of the three distinct genes comprising the BB-301 gene construct within the pharyngeal muscle cells; and

 

BiologicallyDurable and biologically significant levels of target gene knock-down (i.e., inhibition of the expression of the gene of interest) within the pharyngeal muscle cells.

The Pilot Dosing Study evaluated the safety and biological activity of two concentrations of BB-301 (1.0+E13 vg/mL and 3.0+E13 vg/mL) across three distinct doses (1.0+E13 vg/mL, and 3.0+E13 vg/mL with a low injection volume, and 3.0+E13

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vg/mL with a high injection volume) following direct intramuscular injection into the HypopharyngeusHypopharyngeal (HP) muscles and the ThyropharyngeusThyropharyngeal (TP) muscles of Beagle dogs via the use of a proprietary delivery device employed in an open surgical procedure. The HP muscle in Beagle dogs corresponds to the Middle Pharyngeal Constrictor muscle in human subjects, and the TP muscle in Beagle dogs corresponds to the Inferior Pharyngeal Constrictor muscle in human subjects. Atrophy, fibrosis, and the presence of intranuclear inclusions characterize the Middle Pharyngeal Constrictor muscles and the Inferior Pharyngeal Constrictor muscles of human subjects diagnosed with OPMD. BB-301 was injected into the pharyngeal muscles of the Beagle dog subjects only on Day 1 of the Pilot Dosing Study, and the corresponding canine pharyngeal muscles were harvested for molecular analysesanalysis after 8 weeks of observation post-injection. post-dosing. BB-301 dosing was carried out independently by both a veterinary surgeon and ana practicing Otolaryngologist withwho has extensive experience regardingwith the provision of palliative surgical care for OPMD patients.

Molecular analyses have been completed for the canine subjects treated in the BB-301 Pilot Dosing Study. Key interim data-sets derived from the analyses of pharyngeal muscle tissues isolated from 16 Beagle dog subjects (of the 24-subject Beagle dog study population) are highlighted below. The final data-set derived from the completed molecular analyses of the pharyngeal muscle tissues of the canine subjects treated on the Pilot Dosing Study will be presented in a peer-reviewed format.

The key interim data-setsresults are summarized below:here:

Figure 4.7. Pharyngeal Muscle Tissue Transduction Levels Achieved by for BB-301

 

LOGOLOGO

Regarding Gene Expression Levels Observed for BB-301 Within the Pharyngeal Muscle Tissues (Figure 5,8, Figure 6,9, Figure 7)10):

 

•

BB-301 encodes two distinct siRNA species (i.e., siRNA13 and siRNA17) which are each, independently, capable of inhibiting (i.e., “silencing”) the expression of the mutant form of the PABPN1 protein and the wild type (i.e., endogenous) form of the PABPN1 protein (importantly, the mutant form of the PABPN1 protein underlies the development, and progression, of OPMD).

BB-301 encodes two distinct siRNA species (i.e., siRNA13 and siRNA17) which are each, independently, capable of inhibiting (i.e., “silencing”) the expression of the mutant form of the PABPN1 protein and the wild type (i.e., endogenous) form of the PABPN1 protein (importantly, the mutant form of the PABPN1 protein underlies the development and progression of OPMD).

•

BB-301 also codes for a wild type version of the PABPN1 protein whose intracellular expression is unaffected by the inhibitory activities of siRNA13 and siRNA17; this “codon optimized” transcript drives the expression of a PABPN1 protein (i.e., coPABPN1) which serves to replenish the endogenous form of the PABPN1 protein and to replace the mutant form of PABPN1 that underlies the development and progression of OPMD in diseased tissues.

•

For comparative purposes, it should be noted that the average range of expression for wild type PABPN1 within the pharyngeal muscle cells of Beagle dogs is 4.5 copies per cell-to-7.8 copies per cell.

 

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BB-301 also codes for a wild type version of the PABPN1 protein whose intracellular expression is unaffected by the inhibitory activities of siRNA13 and siRNA17, and this codon optimized PABPN1 protein (i.e., coPABPN1) serves to replenish the endogenous form of the PABPN1 protein and to replace the mutant form of PABPN1 that underlies the development and progression of OPMD in diseased tissues.

For comparative purposes, it should be noted that the average range of expression for wild type PABPN1 within the pharyngeal muscle cells of Beagle dogs is 4.5 copies per cell-to-7.8 copies per cell.

Figure 5.8. siRNA13 Expression Levels Achieved by for BB-301 within Pharyngeal Muscle Tissues

 

LOGOLOGO

Figure 6.9. siRNA17 Expression Levels Achieved by for BB-301 within Pharyngeal Muscle Tissues

 

LOGOLOGO

 

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Figure 7.10. coPABPN1 Expression Levels Achieved by for BB-301 within Pharyngeal Muscle Tissues

 

LOGOLOGO

Regarding Wild Type PABPN1 Silencing (i.e., target “knock-down”) Observed for BB-301 Within the Pharyngeal Muscle Tissues (Figure 8)11):

 

•

As noted above, BB-301 encodes two distinct siRNA species (i.e., siRNA13 and siRNA17) which are each, independently, capable of inhibiting (i.e., “silencing”) the expression of all forms of the PABPN1 protein (siRNA13 and siRNA17 silence the expression of both wild type PABPN1 [wtPABPN1] and mutant PABPN1).

As noted above, BB-301 encodes two distinct siRNA species (i.e., siRNA13 and siRNA17) which are each, independently, capable of inhibiting (i.e., “silencing”) the expression of all forms of the PABPN1 protein (siRNA13 and siRNA17 silence the expression of both wild type PABPN1 [wtPABPN1] and mutant PABPN1).

 

•

While the Beagle dog subjects treated in the BB-301 Pilot Dosing Study do not express mutant PABPN1, the level of BB-301-driven gene silencing for the PABPN1 target can be indirectly assessed in these study subjects due to the equivalent inhibitory effects of siRNA13 and siRNA17 on both wtPABPN1 and mutant PABPN1.

While the Beagle dog subjects treated in the BB-301 Pilot Dosing Study did not express mutant PABPN1, the level of BB-301-driven gene silencing for the PABPN1 target can be accurately assessed due to the equivalent inhibitory effects of siRNA13 and siRNA17 on both wtPABPN1 and mutant PABPN1.

 

•

Thus, the wtPABPN1 silencing activity observed in the BB-301 Pilot Dosing Study serves as a surrogate for the silencing activity that would be anticipated in the presence of mutant PABPN1.

Thus, the wtPABPN1 silencing activity observed in the BB-301 Pilot Dosing Study served as a surrogate for the activity that would be anticipated in the presence of mutant PABPN1.

 

•

BB-301 has been evaluated in prior non-clinical studies in animals that express mutant PABPN1 and, as a result, manifest the symptomatic phenotype of OPMD; in the symptomatic animal model of OPMD (i.e. the A17 mouse model), the achievement of PABPN1 silencing levels of 31% inhibition (or higher) following BB-301 administration led to resolution of OPMD disease symptoms and the elimination of the histopathological hallmarks of OPMD.

BB-301 has been evaluated in prior nonclinical studies in animals that express mutant PABPN1 and manifest the key signs and symptoms of OPMD and, in these animal models of OPMD, the achievement of PABPN1 silencing levels of 31% inhibition or higher led to resolution of OPMD disease symptoms and correction of the histological hallmarks of OPMD.

 

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Figure 8.11. PABPN1 Silencing (i.e., “target knock-down”) Achieved by BB-301within Pharyngeal Muscle Tissues

 

LOGOLOGO

There areFinally, it is critical to highlight the key methodological distinctions between the current recently completed BB-301 Pilot Dosing Study in Beagle dogs conducted by Benitec as compared to(i.e., the study described above) and the priorBB-301 Beagle dog dosing study carried out independently by the previous BB-301 licensee. licensee of Benitec. The BB-301 dosing study conducted by the prior BB-301 licensee employed non-ideal routes and methods of BB-301 administration to the target pharyngeal muscle tissues and employed similarly limited analytical methods at the completion of the dosing phase of the study. Subsequently, theThe Benitec team worked to optimize the route and method of administration of BB-301 and to refine the core analytical methods employed following the completion of dosingdosing.

Following the implementation of the large animal subjects.

The current proprietary method of BB-301 delivery to the key pharyngeal muscles of study subjects, and the proprietary molecular analytical methods employed to assay the pharyngeal muscle tissues of study subjects, with both methods having been developed by thethese methodological modifications, Benitec team, led to the observation of broad-based transduction of the targeted pharyngeal muscle tissues (Figure 9, represents individual sections of the TP muscle following BB-301 dosing). Critically, the Benitec-developed methods also facilitated the achievement ofdemonstrated a 228-fold248-fold improvement (+22,647%24,650%) in BB-301 transduction of the HP muscle and a 113-fold111-fold improvement (+11,163%11,027%) in BB-301 transduction of the TP muscle relative to the levels of BB-301 transduction observed by the previous BB-301 licensee at identical BB-301 doses in identical canine study populations (Figure 10)12).

Figure 9. BB-301 Transduction Levels Achieved for Individual Sections of the TP Muscle Following BB-301 dosing

 

LOGO

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Figure 10.12. Impact of theBenitec-Initiated Methodological Improvements to the BB-301 Large Animal Dosing Study Design on the Relative Pharyngeal Muscle Tissue Transduction Levels Achieved by Benitec vs. the Former for BB-301 Licensee

 

LOGOLOGO

Following the disclosure of the positive interim BB-301 Pilot Dosing Study results, Benitec completed pre-CTA

and pre-IND meetings with regulatory agencies in France, Canada, and the United States.

Summary of Regulatory Interactions:

 

Benitec successfully completedIn June 2023 the regulatory interactions requiredU.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for BB-301 which allows dosing of BB-301 to support initiationbegin for OPMD subjects that are eligible for enrollment into the Phase 1b/2a treatment study (described below).

Operational Updates

The key milestones related to the development of BB-301 for the treatment of OPMD, along with other corporate updates, are outlined below:

BB-301 Clinical Development Program Overview:

The BB-301 clinical development program will be conducted in 2022the United States and Canada, and the primary elements of the program are summarized below:

 

Successful regulatory engagement comprised the completionThe program will comprise approximately 76 weeks of the following meetings:follow-up which we anticipate will consist of:

 

 • 

Pre-Clinical Trial Application (Pre-CTA) Consultation Meeting with Health Canada

Scientific Advice Meeting with The National Agency for the Safety of Medicines and Health Products in France (L’Agence nationale de sécurité du médicament et des produits de santé or “ANSM”)

Type C Meeting with the U.S. Food and Drug Administration (“FDA”)

In December 2022 Benitec began screening OPMD subjects at the first clinical trial site in the United States in support of the OPMD clinical development program. In January 2023 Benitec disclosed the enrollment of the first OPMD subject into the Natural History Study (i.e., the 6-month pre-treatment observation period described below). Enrollment of OPMD subjects into the Natural History Study at the U.S. clinical trial site is proceeding at a rapid pace and supports our central clinical development goals of: (1) administering BB-301 to OPMD subjects in 2H2023, and (2) disclosing interim safety and efficacy data in 2H2023 for one or more subjects that have received BB-301.

Summary of the BB-301 Clinical Development Program:

•

The BB-301OPMD Natural History (NH) Study: clinical development program began6-month pre-treatment observation periods for the evaluation of baseline disposition and natural history of OPMD-derived dysphagia (swallowing impairment) in 2022, and the conduct of the development program will comprise approximately 76-weeks of follow-up for each OPMD study participant, inclusive of:participant.

 

 • 

6-month pre-treatmentDosing with BB-301: observation periods employing quantitative radiographic imaging techniques for evaluation1-day of the baseline disposition and natural history of OPMD-derived dysphagia in each study participant

•

1 day of BB-301 dosing to initiate participation in the Phase 1b/2a single-arm, open-label, sequential, dose escalationdose-escalation cohort study. BB-301 will be delivered directly to the pharyngeal muscles of each study subject.

 

 • 

Phase 1b/2a Treatment Evaluation:52-weeks of post-dosing follow-up for conclusive evaluation of the primary and secondary endpoints of the BB-301 Phase 1b/2a BB-301treatment study, with interim safety and efficacy results expected to be available at the end of each 90-day period following the administration of BB-301.

 

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The screeningOPMD NH Study will characterize the level of OPMD subjects for enrollment into the OPMD Natural History Study began in December 2022, with the firstdysphagia borne by each OPMD subject being formally enrolled in January 2023 and brisk enrollment having continued over the subsequent months. This observational study will facilitate the characterization of OPMD patient disposition at baseline and assess subsequent rates of progression of dysphagia (swallowing impairment) in subjects with OPMD via the use of the following quantitative radiographic measures (i.e., videofluoroscopic swallowing studies or “VFSS”). The VFSS outlined below collectively provide objective assessments of global swallowing function and the function of the pharyngeal constrictor muscle function along with clinical assessments and patient-reported self-assessments of swallowing functionmuscles (i.e., the muscles whose functional deterioration drives disease progression in OPMD):

 

•

Total Pharyngeal Residue %(C2-4)2

Videofluoroscopic Swallowing Studies (VFSS) will be conducted to complete the following methodological assessments:Pharyngeal Area at Maximum Constriction (PhAMPC)

 

Dynamic Imaging Grade of Swallowing Toxicity Scale (DIGEST)

 

Pharyngeal Area at Maximum Constriction (PhAMPC)

Pharyngeal Constriction Ratio (PCR)

Clinical measures of global swallowing capacity and oropharyngeal dysphagia

Patient-reported measures of oropharyngeal dysphagia

 • 

The natural history of dysphagia observed for each OPMD study participant in the Natural History Study, as characterized by the quantitative radiographic measuresVallecular Residue %(C2-4)2, Pyriform Sinus Residue %(C2-4)2, and the clinical and patient self-reported assessments outlined above, will serve as the baseline for comparative assessments of safety and efficacy of BB-301 upon rollover of OPMD study subjects from the Natural History Study onto the Phase 1b/2a BB-301 treatment study

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Upon the achievement of 6-months of follow-up in the Natural History Study, OPMD Natural History Study participants can become eligible for enrollment onto the Phase 1b/2a treatment study with the investigational genetic medicine, BB-301, which uses an AAV9-based gene therapy approach for the treatment of OPMD-derived dysphagiaOther Pharyngeal Residue %(C2-4)2

 

 • 

This first-in-human (FIH) clinical trial will be a Phase 1b/2a, open-label, dose escalation study to evaluate the safety and clinical activity of intramuscular doses of BB-301 administered to the pharyngeal muscles of subjects with OPMDNormalized Residue Ratio Scale (NRRSv, NRRSp)

 

Pharyngeal Construction Ratio (PCR)

The NH study will also employ clinical measures of global swallowing capacity and oropharyngeal dysphagia, along with two distinct patient-reported outcome instruments targeting the assessment of oropharyngeal dysphagia.

Upon the achievement of 6-months of follow-up in the NH Study, participants will, potentially, be eligible for enrollment into the BB-301 Phase 1b/2a treatment study.

BB-301 Phase 1b/2a Treatment Study:

This first-in-human (FIH) study will evaluate the safety and clinical activity of intramuscular doses of BB-301 administered to subjects with OPMD.

The primary endpoint of the FIH study will be safety.

Secondary endpoints are designed to determine the impact of BB-301 on swallowing efficiency, swallowing safety, and pharyngeal constrictor muscle function in subjects diagnosed with OPMD with dysphagia via the use of serial clinical and videofluoroscopic assessments. Critically, each of the clinical and videofluoroscopic assessments employed in the FIH study will be equivalent to those employed for the NH study to facilitate comparative clinical and statistical analyses for each study subject.

The primary and secondary endpoints will be evaluated during each 90-day period following BB-301 intramuscular injection (Day 1).

The NH of dysphagia observed for each OPMD study participant, as characterized by the VFSS and clinical swallowing assessments carried out during the NH Study, will serve as the baseline for comparative assessments of safety and efficacy of BB-301 upon rollover from the NH Study onto the BB-301 Phase 1b/2a Treatment Study.

In December 2022, Benitec began screening OPMD subjects at the lead clinical study site in the United States.

In January 2023, Benitec announced the enrollment of the first OPMD subject into the NH Study in the United States.

As of October 2023, 19 subjects have been enrolled into the NH study in the United States.

The pace of enrollment of OPMD subjects into the NH Study at the U.S. clinical study site supports our central clinical development goals of: (1) administering BB-301 to OPMD subjects in December 2023 or January 2024, and (2) disclosing the initial interim safety and efficacy data over the next 12 months for subjects that have received BB-301.

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Additional Regulatory Updates for the Clinical Development Program:

North America:

Formal submission of the comprehensive NH Study trial package to the Research Ethics Board (REB) for the lead clinical study site in Canada was completed, and Benitec awaits the formal response from the REB.

Approval of the NH Study trial package by the REB is required for clinical study site activation and OPMD patient screening and enrollment to begin in Canada.

Intellectual Property

Benitec seeks to actively procure rights to and protect the intellectual property and proprietary technology that it believes is important to its business. Such intellectual property rights include patents claiming our ddRNAi and silence and replace technologies, as well as know-how and trade secrets related to our product candidates and proprietary technology.

ddRNAi-based treatment for OPMD

Benitec’s patent portfolio for OPMD includes five patent families relating to shRNA and shmiRs targeting PABPN1 (the causative gene for OPMD), as well as ‘silence and replace’ therapeutics and treatment strategies for OPMD. These five families cover the OPMD therapeutic candidate, BB-301, under development at Benitec, treatment strategies for OPMD that silence PABPN1 which is causative for OPMD and replace with functional PABPN1, and Benitec’s AAV patent family which covers the delivery system for BB-301. BB-301 is a ‘silence and replace’ construct encoding two shmiRs targeting the endogenous PABPN1 (including variants causative of OPMD) internally designated shmiR-13 and shmiR-17, as well as a codon-optimized PABPN1 replacement construct, the transcript of which is not targeted by shmiR-13 and shmiR-17. Both shmiRs and the codon-optimized PABPN1 replacement construct are under the control of a muscle-specific promoter.

The first patent family, entitled “Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof (OPMD family #1)”, arose out of a collaboration with Royal Holloway University of London (RHUL) and relates to three shRNA target regions within PABPN1. RHUL assigned its ownership interests in this patent family to Benitec, and the PCT application and the related U.S. priority document were filed solely in the name of Benitec. This patent family is directed to RNAi agents targeting specific regions within mutant PABPN1 variants causative of OPMD, as well as use of those RNAi agents in combination with PABPN1 replacement constructs to treat OPMD. More specifically, this family includes claims covering shmiR17 of BB-301 This patent family entered the national/regional phase in October/November 2018.

The second patent family, entitled “Reagents for treatment of oculopharyngeal muscular dystrophy (OPMD) and use thereof (OPMD family #2)” relates to a second set of target gene sequences within PABPN1 as well as ‘silence and replace’ construct BB-301 under development at Benitec. The PCT application and the related U.S. priority document were filed solely in the name of Benitec, and this family entered the national/regional phase in June/July 2019. This patent family is directed to RNAi agents targeting specific regions within mutant PABPN1 variants causative of OPMD, as well as ‘silence and replace’ constructs and use of same for treatment of OPMD. More specifically, this family includes claims covering shmiR13 and shmiR17 of BB-301 separately, as well as the full BB-301 ‘knockdown and replacement’ construct.

A third patent family, entitled “Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD) (OPMD family #3)” has been filed to pursue claims which are broadly directed to the ‘silence and replace’ treatment concept for OPMD, relying on RNAi agents to knockdown PABPN1 and replacement with functional PABPN1 which is not targeted by the RNAi agents. The claims in this application are not limited to BB-301. This patent family exists as a PCT application and was filed solely in the name of Benitec.

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A fourth patent family, entitled “Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD) (OPMD family #4)” has been filed to specifically claim the OPMD therapeutic candidate developed by Benitec, BB-301 (described herein). This patent family exists as a PCT application and was filed solely in the name of Benitec.

AAV with modified phospholipase domain

The Benitec patent portfolio includes a single patent family, entitled “Adeno-associated virus (AAV) with modified phospholipase domain,” which relates to an AAV having a modified phospholipase (PLA2) domain in the capsid. The modified AAV will be used as the delivery system for the OPMD therapeutic.

We are aware of a third party patent directed to AAV vectors that expires in 2026. In the event we receive regulatory marketing approval before the expiration date it may be necessary for us to obtain a license to the patent in order to commercialize. We cannot guarantee the availability of the license or that it can be obtained on commercially reasonable terms.

Know-How

In addition to patent protection of ddRNAi and other technology and our product candidates, we also rely on proprietary know-how that is not patentable or that we elect not to patent, as valuable intellectual property for our business. This know-how is related to the areas of, among others, identifying nucleic acid targets for ddRNAi technology and designing ddRNAi constructs for targeting preferred genes. We have implemented a number of security measures designed to safeguard our know-how including limiting access to our research facilities, databases and networks. We also seek to protect our know-how by way of confidentiality agreements when engaging with external providers for progressing our pipeline of therapeutic candidates.

Laws and Regulations Regarding Patent Terms

The term of individual patents depends upon the legal terms of the patents in the countries in which they are obtained. In most countries in which we file, the patent term is 20 years from the earliest date of filing a non-provisional patent application. In the United States, a patent term may be shortened if a patent is terminally disclaimed over another patent or as a result of delays in patent prosecution by the patentee. A patent’s term may be lengthened by a patent term adjustment, which compensates a patentee for administrative delays by the USPTO in granting a patent. The patent term of a European patent is 20 years from its filing date, which, unlike in the United States, is not subject to patent term adjustments.

The term of a patent that covers an FDA-approved biologic may also be eligible for patent term extension, which permits patent term restoration as compensation for the patent term lost during the FDA regulatory review process. The Drug Price Competition and Patent Term Restoration Act of 1984, or the Hatch-Waxman Act, permits a patent term extension of up to five years beyond the expiration of the patent. The length of the patent term extension is related to the length of time the biologic is under clinical testing regulatory review. Patent extension cannot extend the remaining term of a patent beyond a total of 14 years from the date of product approval and only one patent applicable to an approved biologic may be extended. Similar provisions are available in Europe and other jurisdictions to extend the term of a patent that covers an approved biologic although the eligibility requirements for any duration of such extension vary. In the future, if and when our products receive FDA approval, or approval from an equivalent regulatory body in another jurisdiction in which patent protection is sought or obtained, we expect to apply for patent term extensions on patents covering those products.

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Trademarks

Our trademarks include registrations for company branding and product names for our pipeline in development. The trademarks that we use in connection with our business include the following:

Country or Territory

Trademark (program)

Application or

Registration number

Status

USABENITEC
BIOPHARMA86190065Registered
USASILENCING GENES86488147Registered
FOR LIFE
AustraliaSILENCING GENES1448041Registered
FOR LIFE
AustraliaBENITEC
BIOPHARMA1448046Registered
AustraliaBENITEC—logo1448052Registered
AustraliaNervarna1526478Registered
AustraliaTRIBETARNA1526479Registered
AustraliaHEPBARNA1526483Registered
International BureauGIVING DISEASE THE1389399Registered
(WIPO) –SILENT TREATMENT
designating EU; UK and US
USABENITEC86795296Registered
USAGIVING DISEASE THE79226988Registered
SILENT TREATMENT
European UnionBENITEC14680003Registered
AustraliaBENITEC1728797Registered
AustraliaBENITEC1103049Registered
AustraliaBENITEC1103300Registered
AustraliaGIVING DISEASE THE1851660Registered
SILENT TREATMENT
United KingdomBENITEC3238275Registered

Manufacturing

The manufacture of the biological products required for gene therapy is complex and difficult. We do not currently own or operate manufacturing facilities for the production of preclinical, clinical or commercial quantities of any of our product candidates. We are exploring long-term manufacturing alliances with a number of potential partners to investigate manufacturing processes in order to produce materials at reasonable scale and cost of goods to support future commercialization efforts. We do not have a long-term agreement with any third-party manufacturer, but we plan to establish such a relationship with an appropriate manufacturer to serve our long-term needs.

Manufacturing is subject to extensive regulations that impose various procedural and documentation requirements, which govern record keeping, manufacturing processes and controls, personnel, quality control and quality assurance, among others. Our contract manufacturing organizations manufacture our product candidates under cGMP conditions. cGMP is a regulatory standard for the production of pharmaceuticals that will be used in humans.

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Sales and Marketing

We have not yet established sales, marketing or product distribution operations because our product candidates are in preclinical or clinical development. If we receive marketing and commercialization approval for any of our product candidates, we intend to market the product through strategic alliances and distribution agreements with third parties. In certain cases, we may market an approved product directly worldwide or in selected geographical segments. The ultimate implementation of our strategy for realizing the financial value of our product candidates is dependent on the results of clinical trials for our product candidates, the availability of funds and the ability to negotiate acceptable commercial terms with third parties.

Competition

The biopharmaceutical industry is characterized by intense and dynamic competition to develop new technologies and proprietary therapies.

Any product candidates that we successfully develop and commercialize will have to compete with existing therapies and new therapies that may become available in the future. While we believe that our proprietary technology and scientific expertise in gene silencing using ddRNAi provide us with competitive advantages, we face potential competition from many different sources, including larger and better-funded pharmaceutical, specialty pharmaceutical and biotechnology companies, as well as from academic institutions and governmental agencies and public and private research institutions that may develop potentially competitive products or technologies. We are aware of several companies focused on developing gene therapy or gene silencing product candidates.

We are not aware of any companies developing a gene therapy or gene silencing approach for OPMD. Our product candidates, if approved, would also compete with treatments that have already been approved and accepted by the medical community, patients and third-party payers.

Many of our competitors and potential competitors, alone or with their strategic partners, have substantially greater financial, technical and human resources than we do and significantly greater experience in the discovery and development of product candidates, obtaining FDA and other regulatory approvals of treatments and the commercialization of those treatments. Mergers and acquisitions in the biotechnology and pharmaceutical industries may result in even more resources being concentrated among a smaller number of our competitors. These competitors also compete with us in recruiting and retaining qualified scientific and management personnel and establishing clinical study sites and subject registration for clinical studies, as well as in acquiring technologies complementary to, or necessary for, our programs. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies.

We anticipate that we will face intense and increasing competition as new products enter the market and advanced technologies become available. We expect any treatments that we develop and commercialize to compete on the basis of, among other things, efficacy, safety, convenience of administration and delivery, price, the level of competition and the availability of reimbursement from government and other third party-payers.

Our commercial opportunity could be reduced or eliminated if our competitors develop and commercialize products that are safer, more effective, have fewer or less severe side effects, are more convenient or are less expensive than any products that we may develop. Our competitors also may obtain FDA or other regulatory approval for their products more rapidly than we may obtain approval for ours, which could result in our competitors establishing a strong market position before we are able to enter the market. In addition, we expect that our therapeutic products, if approved, will be priced at a significant premium over competitive products and our ability to compete may be affected in many cases by insurers or other third-party payers seeking to encourage the use of competitive products including biosimilar or generic products.

This increasingly competitive landscape may compromise the development of our product candidates.

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Government Regulation

As a pharmaceutical and biological product company that wishes to conduct clinical trials and ultimately obtain marketing approval in the United States, we are subject to extensive regulation by the FDA, and other federal, state, and local regulatory agencies. The Federal Food, Drug, and Cosmetic Act, or the FDC Act, the Public Health Service Act, or PHS Act, and their implementing regulations set forth, among other things, requirements for the research, testing, development, manufacture, quality control, safety, effectiveness, approval, labeling, storage, record keeping, reporting, distribution, import, export, advertising and promotion of our products. A failure to comply explicitly with any requirements during the product development, approval, or post-approval periods, may lead to administrative or judicial sanctions. These sanctions could include the imposition by the FDA or an IRB, of a suspension on clinical trials, refusal to approve pending marketing applications or supplements, withdrawal of approval, warning letters, product recalls, product seizures, total or partial suspension of production or distribution, injunctions, fines, civil penalties or criminal prosecution.

Although the discussion below focuses on regulation in the United States, we anticipate seeking approval for the testing and marketing of our products in other countries. Generally, our activities in other countries will be subject to regulation that is similar in nature and scope as that imposed in the United States, although there can be important differences. Additionally, some significant aspects of regulation in the European Union are addressed in a centralized way through the EMA, but country-specific regulation remains essential in many respects.

Government regulation may delay or prevent testing or marketing of our products and impose costly procedures upon our activities. The testing and marketing approval process, and the subsequent compliance with appropriate statutes and regulations, requires substantial time, effort, and financial resources, and we cannot be certain that the FDA or any other regulatory agency will grant marketing approvals for our products or any future products on a timely basis, if at all. The FDA’s or any other regulatory agency’s policies may change and additional governmental regulations may be enacted that could prevent or delay regulatory approval of our products or any future products or approval of new indications or label changes. We cannot predict the likelihood, nature or extent of adverse governmental regulation that might arise from future legislative, judicial, or administrative action, either in the United States or abroad.

Recent Developments in Regulation of Gene Therapy

Government Regulation in the United States

The FDA has provided guidance for the development of gene therapy products. For example, the FDA has established the Office of Tissues and Advanced Therapies (formerly Office of Cellular, Tissue and Gene Therapies) within the Center for Biologics Evaluation and Research (CBER), to consolidate the review of gene therapy and related products, and the Cellular, Tissue and Gene Therapies Advisory Committee to advise CBER on its reviews. In addition, the FDA has issued a growing body of clinical guidelines, chemical, manufacturing and control, or CMC, guidelines, regenerative medicine guidelines and other guidelines, all of which are intended to facilitate industry’s development of gene therapy products.

In 2016, Section 3033 of the 21st Century Cures Act created a new product category called “regenerative medicine advanced therapy”, or the RMAT designation. The RMAT designation gives the sponsor of a new investigational biologic access to increased meeting opportunities with the FDA, in a manner comparable to those offered to sponsors of therapies designated as “breakthrough therapies” by the FDA. Because the designated products meet the criteria for unmet medical need in the treatment of a serious condition, they may be eligible for priority review, in which the initial assessment of the BLA is reduced from 12 months to eight months, and accelerated approval, which bases approval on an effect on a predictive surrogate endpoint or an intermediate clinical endpoint. RMATs qualifying for such accelerated approval may be able to satisfy licensing requirements through commitment to post-approval clinical studies as well as real-world data such as patient registries and health record analysis. The eligibility of the RMAT-designated product for these expedited

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programs can be discussed with the FDA at specific development meetings, but we do not know whether any of our current or future product candidates will be eligible for RMAT designation. We believe the increased access to the FDA during early development is a benefit for sponsors, because the typical Type B development meetings are normally restricted to one each at the stages of pre-IND, end of Phase II/pre-Phase III and pre-BLA submission. In addition, the option to qualify for a fast-track program, also based on the potential to serve an unmet medical need in the treatment of a serious condition, allows for a so-called “rolling review” of parts of the BLA, which can be submitted for assessment following agreement of a review timetable with CBER.

The FDA plans to include certain gene therapy products that permanently alter tissue and produce a sustained therapeutic benefit as part of the products that will meet the definition of being eligible to come under the pathway enabled by RMAT designation. RMAT designation enables gene therapy products to access the FDA’s existing expedited programs to help foster the development and approval of gene therapy products. Our product candidates may not be eligible for RMAT designation now or in the future.

In May 2016, the EMA approved a second gene therapy product called Strimvelis, the first approved ex vivo stem cell gene therapy, to treat patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency).

In August 2017, the FDA approved the first gene therapy product in the United States. The FDA approved Kymriah (tisagenlecleucel) for the treatment of certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). Kymriah is a genetically-modified autologous T-cell immunotherapy. Because of the risk of cytokine release syndrome and neurological events, Kymriah is being approved with a REMS. In December 2017, the FDA approved Luxturna (voretigene neparvovec-rzyl), a gene therapy to treat children and adult patients with an inherited form of vision loss that may result in blindness. Luxturna is the first directly administered gene therapy approved in the United States that targets a disease caused by mutations in a specific gene.

Marketing Approval

In the United States, for premarket approval purposes, the FDA regulates gene therapy products as biologics under the FDC Act, the PHS Act and related regulations.

The steps required before a new biologic may be marketed in the United States generally include:

nonclinical pharmacology and toxicology laboratory and animal tests according to good laboratory practices, or GLPs, and applicable requirements for the humane use of laboratory animals or other applicable regulations;

submission of an IND application which must become effective before human clinical trials may begin;

adequate and well-controlled human clinical trials according to GCPs and any additional requirements for the protection of human research subjects and their health information to establish the safety and efficacy of the investigational product for each targeted indication;

submission of a biologics license application, or BLA, to the FDA;

FDA’s pre-approval inspection of manufacturing facilities to assess compliance with cGMPs and, if applicable, the FDA’s good tissue practices, or GTPs, for the use of human cellular and tissue products to prevent the introduction, transmission, or spread of communicable diseases;

FDA’s audit of clinical trial sites that generated data in support of the BLA; and

FDA approval of a BLA, which must occur before a product can be marketed or sold.

Product Development Process

Before testing any biologic in humans, the product enters the nonclinical, or preclinical, testing stage. Nonclinical tests include laboratory evaluations of product chemistry, toxicity, and formulation, as well as animal studies to assess the potential safety and activity of the product. The conduct of nonclinical tests must comply with federal regulations and requirements including GLPs.

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Where a gene therapy trial is conducted at, or sponsored by, institutions receiving NIH funding for recombinant DNA research, prior to the submission of an IND to the FDA, a protocol and related documentation is submitted to and the trial is registered with the NIH Office of Science Policy, or OSP.

The product sponsor then submits the results of the nonclinical testing, together with manufacturing information, analytical data, any available clinical data or literature, and a proposed clinical protocol, to the FDA in an IND, which is a request for authorization from the FDA to administer an investigational product to humans. Some nonclinical testing may continue even after the IND application is submitted. IND authorization is required before interstate shipping and administration of any new product to humans that is not the subject of an approved BLA. The IND automatically becomes effective 30 days after receipt by the FDA unless the FDA, within the 30-day time period, raises concerns or questions about the conduct of the clinical trial and places the clinical trial on a clinical hold. In such case, the IND sponsor must resolve any outstanding concerns with the FDA before the clinical trial may begin. Further, an IRB for each site proposing to conduct the clinical trial must review and approve the plan for any clinical trial before it commences at that site. If the site has an IBC, it may also have to review and approve the proposed clinical trial. Clinical trials involve the administration of the investigational product to patients under the supervision of qualified investigators following GCPs, requirements meant to protect the rights and health of patients and to define the roles of clinical trial sponsors, investigators, and monitors. Clinical trials are conducted under protocols that detail, among other things, the objectives of the clinical trial, dosing procedures, subject selection and exclusion criteria, the parameters to be used in monitoring safety, including stopping rules that assure a clinical trial will be stopped if certain adverse events should occur, and the efficacy criteria to be evaluated. Each protocol involving testing on U.S. patients and subsequent protocol amendments must be submitted to the FDA as part of the IND. The informed written consent of each participating subject is required and the form and content of the informed consent must be approved by each IRB.

The clinical investigation of an investigational product is generally divided into three phases. Although the phases are usually conducted sequentially, they may overlap or be combined in some cases. The three phases of an investigation are as follows:

Phase I includes the initial introduction of an investigational product into humans. Phase I clinical trials may be conducted in patients with the target disease or condition or on healthy volunteers. These studies are designed to evaluate the safety, metabolism, pharmacokinetics and pharmacologic actions of the investigational product in humans, the side effects associated with increasing doses, and if possible, to gain early evidence on effectiveness. During Phase I clinical trials, sufficient information about the investigational product’s pharmacokinetics and pharmacological effects may be obtained to permit the design of Phase II clinical trials. The total number of participants included in Phase I clinical trials varies, but is generally in the range of 20 to 80.

Phase II includes the controlled clinical trials conducted to evaluate the effectiveness of the investigational product for a particular indication(s) in patients with the disease or condition under study, to determine dosage tolerance and optimal dosage, and to identify possible adverse side effects and safety risks associated with the product. Phase II clinical trials are typically well- controlled, closely monitored, and conducted in a limited patient population, usually involving no more than several hundred participants. Phase IIa trials provide information on the impact of dose ranging on safety, biomarkers and proof of concept, while Phase IIb trials are patient dose-ranging efficacy trials.

Phase III clinical trials are controlled clinical trials conducted in an expanded patient population at geographically dispersed clinical trial sites. They are performed after preliminary evidence suggesting effectiveness of the investigational product has been obtained, and are intended to further evaluate dosage, clinical effectiveness and safety, to establish the overall benefit-risk relationship of the product, and to provide an adequate basis for product approval. Phase III clinical trials usually involve several

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 •

Upon rollover fromhundred to several thousand participants. In most cases, the Natural History Study ontoFDA requires two adequate and well controlled Phase III clinical trials to demonstrate the efficacy of the product. FDA may accept a single Phase 1b/2a BB-301 treatment study,III trial with other confirmatory evidence in rare instances where the follow-uptrial is a large multicenter trial demonstrating internal consistency and a statistically very persuasive finding of OPMD study participants will continue for 52-weeks,a clinically meaningful effect on mortality, irreversible morbidity or prevention of a disease with a potentially serious outcome and confirmation of the primary endpoints (safety) and secondary endpoints (comprising the quantitative radiographic measures of global swallowing function and pharyngeal constrictor muscle function, and the clinical and patient-reported assessments noted above) willresult in a second trial would be evaluated during each 90-day period following Day 1 (Day 1 represents the day of BB-301 intramuscular injection).practically or ethically impossible.

Annual progress reports detailing the results of the clinical trials must be submitted to the FDA. Written IND safety reports must be promptly submitted to the FDA and the investigators for serious and unexpected adverse events; any findings from other studies, tests in laboratory animals or in vitro testing that suggest a significant risk for human subjects; or any clinically important increase in the rate of a serious suspected adverse reaction over that listed in the protocol or investigator brochure. The sponsor must submit an IND safety report within 15 calendar days after the sponsor determines that the information qualifies for reporting. The sponsor also must notify the FDA of any unexpected fatal or life-threatening suspected adverse reaction within seven calendar days after the sponsor’s initial receipt of the information. The FDA typically recommends that sponsors observe subjects for potential gene therapy-related delayed adverse events for a 15-year period, including a minimum of five years of annual examinations followed by 10 years of annual queries, either in person or by questionnaire, of trial subjects.

The decision to terminate a clinical trial of an investigational biologic may be made by the FDA or other regulatory authority, an IRB, an IBC, or institutional ethics committee, or by a company for various reasons. The FDA may place a clinical hold and order the temporary, or permanent, discontinuation of a clinical trial at any time, or impose other sanctions, if it believes that the clinical trial either is not being conducted in accordance with FDA requirements or presents an unacceptable risk to the clinical trial patients. If the FDA imposes a clinical hold, trials may not recommence without FDA and IRB authorization and then only under terms authorized by the FDA and IRB. In some cases, clinical trials are overseen by an independent group of qualified experts organized by the trial sponsor, or the clinical monitoring board or DSMB. This group provides authorization for whether or not a trial may move forward at designated check points. These decisions are based on the limited access to data from the ongoing trial. The suspension or termination of a clinical trial can occur during any phase of clinical trials if it is determined that the participants or patients are being exposed to an unacceptable health risk. In addition, there are requirements for the registration of ongoing clinical trials of drugs and biologics on public registries and the disclosure of certain information pertaining to the trials as well as clinical trial results after completion.

Assuming successful completion of all required testing in accordance with all applicable regulatory requirements, detailed investigational product information is submitted to the FDA in the form of a BLA for a biologic to request marketing approval for the product in specified indications.

Human gene therapy products are a new category of therapeutics. Because this is a relatively new and expanding area of novel therapeutic interventions, there can be no assurance as to the length of the trial period, the number of patients the FDA will require to be enrolled in the trials in order to establish the safety, efficacy, purity and potency of human gene therapy products, or that the data generated in these trials will be acceptable to the FDA to support marketing approval. The NIH and the FDA have a publicly accessible database, the Genetic Modification Clinical Research Information System, which includes information on gene transfer trials and serves as an electronic tool to facilitate the reporting and analysis of adverse events on these trials. Over the last several years the FDA has issued helpful guidance on development of gene therapy products and shown a willingness to work closely with developers, especially with those working in orphan disease areas.

Biologics License Application Approval Process

In order to obtain approval to market a biologic in the United States, a BLA must be submitted to the FDA that provides data from nonclinical studies and clinical trials and manufacturing information establishing to the FDA’s satisfaction the safety, purity, and potency or efficacy of the investigational product for the proposed indication. The BLA must be accompanied by a substantial user fee payment unless a waiver or exemption applies.

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The FDA will initially review the BLA for completeness before it accepts it for filing. Under the FDA’s procedures, the agency has 60 days from its receipt of a BLA to determine whether the application will be accepted for filing based on the agency’s threshold determination that the application is sufficiently complete to permit substantive review. After the BLA submission is accepted for filing, the FDA reviews the BLA to determine, among other things, whether the proposed product is safe, pure and potent, which includes determining whether it is effective for its intended use, and whether the product is being manufactured in accordance with cGMP, to assure and preserve the product’s identity, safety, strength, quality, potency and purity, and in accordance with biological product standards. The FDA will inspect the facilities at which the product is manufactured to ensure the manufacturing processes and facilities are in compliance with cGMP requirements and are adequate to assure consistent production of the product within required specifications. For a human cellular or tissue product, the FDA also will not approve the product if the manufacturer is not in compliance with the GTPs. These are FDA regulations that govern the methods used in, and the facilities and controls used for, the manufacture of human cells, tissues, and cellular and tissue based products, which are human cells or tissue intended for implantation, transplant, infusion, or transfer into a human recipient. The primary intent of the GTP requirements is to ensure that cell and tissue based products are manufactured in a manner designed to prevent the introduction, transmission, and spread of communicable disease. Additionally, before approving a BLA, the FDA may inspect one or more clinical sites to assure compliance with GCP.

If the FDA determines the application, manufacturing process or manufacturing facilities are not acceptable, it typically will outline the deficiencies and often will request additional testing or information, or corrective action for a manufacturing facility. This may significantly delay further review of the application. If the FDA finds that a clinical site did not conduct the clinical trial in accordance with GCP, the FDA may determine the data generated by the clinical site should be excluded from the primary efficacy analyses provided in the BLA. Additionally, notwithstanding the submission of any requested additional information, the FDA ultimately may decide that the application does not satisfy the regulatory criteria for approval.

The FDA may refer applications for novel products or products that present difficult questions of safety or efficacy to an advisory committee. The FDA also may determine a REMS is necessary to assure the safe use of the biologic, in which case the BLA sponsor must submit a proposed REMS. The REMS may include, but is not limited to, a Medication Guide, a communications plan, and other elements to assure safe use, such as restrictions on distribution, prescribing, and dispensing.

After the FDA completes its initial review of a BLA, it will either license, or approve, the product, or issue a complete response letter to communicate that it will not approve the BLA in its current form and to inform the sponsor of changes that the sponsor must make or additional clinical, nonclinical or manufacturing data that must be received before the FDA can approve the application, with no implication regarding the ultimate approvability of the application. If a complete response letter is issued, the sponsor may either resubmit the BLA, addressing

all of the deficiencies identified in the letter, or withdraw the application.

The testing and approval process for both a drug and biologic requires substantial time, effort and financial resources and this process may take several years to complete. Data obtained from clinical trials is not always conclusive and may be susceptible to varying interpretations, which could delay, limit or prevent regulatory approval. The FDA may not grant approval on a timely basis, or at all. We may encounter difficulties or unanticipated costs in our efforts to secure necessary governmental approvals, which could delay or preclude us from marketing our products.

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Orphan Drug Designation

Under the Orphan Drug Act, the FDA may grant orphan designation to a drug or biological product candidate intended to treat a rare disease or condition, which is generally a disease or condition that affects fewer than 200,000 individuals in the United States, or more than 200,000 individuals in the United States and for which there is no reasonable expectation that the cost of developing and making a drug or biological product available in the United States for this type of disease or condition will be recovered from sales of the product candidate. Orphan product designation must be requested before submitting a BLA. After the FDA grants orphan product designation, the identity of the therapeutic agent and its potential orphan use are disclosed publicly by the FDA. Orphan product designation does not convey any advantage in or shorten the duration of the regulatory review and approval process.

If a product candidate that has orphan designation subsequently receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications to market the same drug or biological product for the same indication for seven years, except in limited circumstances, such as a showing of clinical superiority to the product with orphan exclusivity. Competitors, however, may receive approval of different products for the indication for which the orphan product has exclusivity or obtain approval for the same product but for a different indication than the one for which the orphan product has exclusivity. Orphan product exclusivity also could block the approval of one of our products for seven years if a competitor obtains approval of the same biological product as defined by the FDA or if our product candidate is determined to be contained within the competitor’s product for the same indication or disease. If a drug or biological product designated as an orphan product receives marketing approval for an indication broader than what is designated, it may not be entitled to orphan product exclusivity. Orphan drug status in the European Union has similar, but not identical, benefits.

Expedited Development and Review Programs

The FDA has a fast track program that is intended to expedite or facilitate the process for reviewing new drugs and biological products that meet certain criteria. Specifically, new drugs and biological products are eligible for fast track designation if they are intended to treat a serious or life-threatening condition and demonstrate the potential to address unmet medical needs for the condition. Fast track designation applies to the combination of the product and the specific indication for which it is being studied. The sponsor of a new biologic or drug may request the FDA to designate the biologic or drug as a fast track product at any time during the clinical development of the product. Unique to a fast track product, the FDA may consider for review sections of the marketing application on a rolling basis before the complete application is submitted, if the sponsor provides a schedule for the submission of the sections of the application, the FDA agrees to accept sections of the application and determines that the schedule is acceptable, and the sponsor pays any required user fees upon submission of the first section of the application.

Any product submitted to the FDA for marketing, including under a fast track program, may be eligible for other types of FDA programs intended to expedite development and review, such as priority review and accelerated approval. Any product is eligible for priority review if it would, if approved, be a significant improvement in the safety, effectiveness, treatment, diagnosis or prevention of a disease compared to marketed products. The FDA will attempt to direct additional resources to the evaluation of an application for a new biological or drug product designated for priority review in an effort to reduce the review period from 12 to eight months. Additionally, a product may be eligible for accelerated approval. Biological or drug products studied for their safety and effectiveness in treating serious or life-threatening illnesses and that provide meaningful therapeutic benefit over existing treatments may receive accelerated approval, which means that they may be approved on the basis of adequate and well-controlled clinical trials establishing that the product has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit, or on the basis of an effect on an intermediate clinical endpoint. As a condition of approval, the FDA may require that a sponsor of a biological or drug product receiving accelerated approval perform adequate and well-controlled post-marketing clinical trials. In addition, the FDA

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currently requires as a condition for accelerated approval pre-approval of promotional materials, which could adversely impact the timing of the commercial launch of the product. Lastly, under the provisions of the new Food and Drug Administration Safety and Innovation Act, enacted in 2012, a sponsor can request designation of a product candidate as a “breakthrough therapy.” A breakthrough therapy is defined as a drug or biological that is intended, alone or in combination with one or more other drugs or biological, to treat a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or biological may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. Drugs and biologicals designated as breakthrough therapies receive the same benefits as drugs and biologicals with Fast Track designation. In addition, the FDA must take certain additional actions, such as intensive guidance on an efficient drug development program (beginning as early as Phase 1), and organizational commitment involving senior managers, intended to expedite the development and review of an application for approval of a breakthrough therapy.

Fast Track designation and breakthrough therapy designation may expedite the product development and approval process, and priority review may expedite the approval process. However, these three paths do not change the standards for approval. Accelerated approval designation changes the standards for product approval and thus may expedite the development and/or approval process.

FDA Additional Requirements

The FDA may require, or companies may pursue, additional clinical trials after a product is approved. These so-called Phase 4 clinical trials may be made a condition to be satisfied for continuing drug and biologic approval. The results of Phase 4 clinical trials can confirm the efficacy of a product candidate and can provide important safety information. In addition, the FDA has expressed statutory authority to require sponsors to conduct post-market studies to specifically address safety issues identified by the agency.

Even if a product candidate receives regulatory approval, the approval may be limited to specific disease states, patient populations and dosages, or might contain significant limitations on use in the form of warnings, precautions or contraindications, or in the form of an onerous REMS, restrictions on distribution, or post-marketing study requirements. Further, even after regulatory approval is obtained, later discovery of previously unknown problems with a product may result in restrictions on the product or even complete withdrawal of the product from the market. In addition, we cannot predict what adverse governmental regulations may arise from future U.S. or foreign governmental action.

Medical Device Requirements

Our contemplated diagnostics, for use with certain of our therapeutic products, are regulated by FDA as in vitro diagnostic, or IVD, medical devices. Such IVD devices must comply with applicable FDA IVD-specific regulations as well as FDA regulations applicable more broadly to medical devices. These FDA regulations include requirements for registering establishments with FDA; listing IVD devices with FDA; reporting certain adverse events related to IVD devices to FDA; complying with the Quality System Regulation (current good manufacturing practices for devices); labeling IVD devices; and obtaining premarket approval or clearance prior to marketing IVD devices (unless exempt). There are also regulations covering the requirements for investigational devices and the conduct of clinical investigations of devices. Like drugs and biologics, failure to comply with applicable device/IVD requirements can result in legal or administrative enforcement actions against an IVD device firm, its officers or employees, and/or its products.

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FDA Post-Approval Requirements

Any products manufactured or distributed by us or on our behalf pursuant to FDA approvals are subject to continuing regulation by the FDA, including requirements for record-keeping, reporting of adverse experiences with the biologic or drug, and submitting biological product deviation reports to notify the FDA of unanticipated changes in distributed products. Manufacturers are required to register their facilities with the FDA and certain state agencies, and are subject to periodic announced or unannounced inspections by the FDA and certain state agencies for compliance with cGMP requirements, which impose certain quality processes, manufacturing controls and documentation requirements upon us and our third-party manufacturers in order to ensure that the product is safe, has the identity and strength, and meets the quality, purity and potency characteristics that it purports to have. In November 2013, the Drug Quality and Security Act, or DQSA, became law and establishes requirements to facilitate the tracing of prescription drug and biological products through the supply distribution chain. This law includes a number of new requirements that are being implemented over time and require us to devote additional resources to satisfy these requirements, including serializing the product and using new technology and data storage to electronically trace the product from manufacturer to dispenser. If our products are not covered by the serialization and tracing requirements of the DQSA, they may be subject to state pedigree and traceability requirements. We cannot be certain that we or our present or future suppliers will be able to comply with the cGMP and other FDA regulatory requirements. If our present or future suppliers are not able to comply with these requirements, the FDA may halt our clinical trials, refuse to approve any BLA, force us to recall a product from distribution, shut down manufacturing operations or withdraw approval of the applicable BLA. Noncompliance with cGMP or other requirements can result in issuance of warning or untitled letters, civil and criminal penalties, seizures, and injunctive action.

The FDA and other federal and state agencies closely regulate the labeling, marketing and promotion of drugs and biologics. Government regulators, including the Department of Justice and the Office of the Inspector General of the Department of Health and Human Services, as well as state authorities, recently have increased their scrutiny of the promotion and marketing of drugs and biologics. While doctors are free to prescribe any product approved by the FDA for any use, a company can only make claims relating to safety and efficacy of a product that are consistent with FDA approval, and the company is allowed to market a product only for the particular use and treatment approved by the FDA. In addition, any claims we make for our products in advertising or promotion must, among other things, be appropriately balanced with important safety information and otherwise be adequately substantiated. Failure to comply with these requirements can result in adverse publicity, warning or untitled letters, corrective advertising, injunctions, potential civil and criminal penalties, criminal prosecution, and agreements with governmental agencies that materially restrict the manner in which a company promotes or distributes products.

Pediatric Research Equity Act

Under the Pediatric Research Equity Act, or PREA, as amended, a BLA or supplement must contain data to assess the safety and efficacy of the product for the claimed indications in all relevant pediatric subpopulations and to support dosing and administration for each pediatric subpopulation for which the product is safe and effective. Manufacturers must submit a pediatric study plan to the IND not later than 60 days after the end-of-phase 2 meeting with the FDA; if there is no such meeting, before the initiation of any phase 3 studies or a combined phase 2 and phase 3 study; or if no such study will be conducted, no later than 210 days before a marketing application or supplement is submitted. The intent of PREA is to compel sponsors whose products have pediatric applicability to study those products in pediatric populations, rather than ignoring pediatric indications for adult indications that could be more economically desirable. The FDA may grant deferrals for submission of data or full or partial waivers. By its terms, PREA does not apply to any product for an indication for which orphan designation has been granted, unless the FDA issues regulations stating otherwise. Because the FDA has not issued any such regulations, submission of a pediatric assessment is not required for an application to market a product for an orphan-designated indication. In a July 2018 guidance, the FDA announced that it does not expect to grant any additional orphan drug designations to drugs for pediatric subpopulations of common diseases (i.e., diseases or conditions with an overall prevalence of 200,000 or greater). Pediatric subpopulation orphan designations that have already been granted will not be affected by this change.

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Patent Term Restoration and Marketing Exclusivity

Depending on the timing, duration and specifics of FDA marketing approval of our product candidates, some of our U.S. patents may be eligible for limited patent term extension under the Hatch-Waxman Amendments. The Hatch-Waxman Amendments permit a patent restoration term of up to five years as compensation for patent term lost during product development and the FDA regulatory review process. However, patent term restoration cannot extend the remaining term of a patent beyond a total of 14 years from the product’s approval date. The patent term restoration period is generally one-half the time between the effective date of an IND and the submission date of a BLA plus the time between the submission date of a BLA and the approval of that application. Only one patent applicable to an approved biological is eligible for the extension and the application for the extension must be submitted prior to the expiration of the patent and within sixty days of approval of the biological product. The USPTO, in consultation with the FDA, reviews and approves the application for any patent term extension or restoration.

The Biologics Price Competition and Innovation Act of 2009, which was included within the Patient Protection and Affordable Care Act, created an abbreviated approval pathway for biological products shown to be similar to, or interchangeable with, an FDA-licensed reference biological product, and grants a reference biologic twelve years of exclusivity from the time of first licensure. Biosimilarity, which requires that there be no clinically meaningful differences between the biological product and the reference product in terms of safety, purity, and potency, can be shown through analytical studies, animal studies, and a clinical study or studies. Interchangeability requires that a product is biosimilar to the reference product and the product must demonstrate that it can be expected to produce the same clinical results as the reference product and, for products administered multiple times, the biologic and the reference biologic may be switched after one has been previously administered without increasing safety risks or risks of diminished efficacy relative to exclusive use of the reference biologic. However, complexities associated with the larger, and often more complex, structure of biological products, as well as the process by which such products are manufactured, pose significant hurdles to implementation that are still being worked out by the FDA.

Pediatric exclusivity is another type of exclusivity in the United States. Pediatric exclusivity, if granted, provides an additional six months of exclusivity to be attached to any existing marketing exclusivity, e.g., twelve year exclusivity, or patent protection for a drug. This six month exclusivity, which runs from the end of other exclusivity protection or patent delay, may be granted based on the voluntary completion of a pediatric trial in accordance with an FDA-issued “Written Request” for such a trial.

Government regulation outside the United States

In addition to regulations in the United States, we will be subject to a variety of regulations in other jurisdictions governing, among other things, clinical trials and any commercial sales and distribution of our products.

Whether or not we obtain FDA approval for a product, we must obtain the requisite approvals from regulatory authorities in foreign countries prior to the commencement of clinical trials or marketing of the product in those countries. Certain countries outside of the United States have a similar process that requires the submission of a clinical trial application much like the IND prior to the commencement of human clinical trials. In the European Union, for example, a request for a clinical trial authorization, or CTA, must be submitted to each country’s national health authority and an independent ethics committee, much like the FDA and the IRB, respectively. Once the CTA is approved in accordance with a country’s requirements, clinical trial development may proceed.

The requirements and process governing the conduct of clinical trials, product approval or licensing, pricing and reimbursement vary from country to country. In all cases, the clinical trials are conducted in accordance with GCP and the applicable regulatory requirements and the ethical principles that have their origin in the Declaration of Helsinki.

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To obtain regulatory approval of a biological product under European Union regulatory systems, we must submit a marketing authorization application. The application required in the European Union is similar to a BLA in the United States, with the exception of, among other things, country-specific document requirements. The European Union also provides opportunities for market exclusivity. For example, in the European Union, upon receiving marketing authorization, a new biological generally receives eight years of data exclusivity and an additional two years of market exclusivity. If granted, data exclusivity prevents regulatory authorities in the European Union from referencing the innovator’s data to assess a biosimilar application. During the additional two-year period of market exclusivity, a biosimilar marketing authorization can be submitted, and the innovator’s data may be referenced, but no biosimilar product can be marketed until the expiration of the market exclusivity. The innovator may obtain an additional one year of market exclusivity if the innovator obtains an additional authorization during the initial eight year period for one or more new indications that demonstrate significant clinical benefit over existing therapies. This data and market exclusivity regime in the European Union of a total of 10 or 11 years protects against generic competition, but does not protect against the launch of a competing product if the competitor, rather than referencing the clinical data of the originator, has conducted its own clinical trials to support its marketing authorization application.

Orphan drugs in the European Union are eligible for 10-year market exclusivity. This 10-year market exclusivity may be reduced to six years if, at the end of the fifth year, it is established that the product no longer meets the criteria for orphan designation, for example, if the product is sufficiently profitable not to justify maintenance of market exclusivity. Additionally, marketing authorization may be granted to a similar product for the same indication at any time if:

the second applicant can establish that its product, although similar, is safer, more effective or otherwise clinically superior;

the applicant consents to a second orphan medicinal product application; or

the applicant cannot supply enough orphan medicinal product.

If we fail to comply with applicable foreign regulatory requirements, we may be subject to, among other things, fines, suspension or withdrawal of regulatory approvals, product recalls, seizure of products, operating restrictions and criminal prosecution.

Pharmaceutical Coverage, Pricing and Reimbursement

Sales of our products, when and if approved for marketing, will depend, in part, on the extent to which our products will be covered by third-party payers, such as federal, state, and foreign government healthcare programs, commercial insurance and managed healthcare organizations. These third-party payers are increasingly reducing reimbursements for medical products, biologicals, drugs and services. In addition, the U.S. government, state legislatures and foreign governments have continued implementing cost containment programs, including price controls, restrictions on coverage and reimbursement and requirements for substitution of interchangeable products. Adoption of price controls and cost containment measures, and adoption of more restrictive policies in jurisdictions with existing controls and measures, could further limit our net revenue and results. Decreases in third-party reimbursement for our product candidates or a decision by a third-party payer not to cover our product candidates could reduce physician usage of our products once approved and have a material adverse effect on our sales, results of operations and financial condition.

The containment of healthcare costs has become a priority of federal, state and foreign governments. Third-party payers are increasingly challenging the prices charged for drug products and medical services, examining the medical necessity and reviewing the cost effectiveness of drug products and medical services, in addition to questioning safety and efficacy. If these third-party payers do not consider our products to be cost-effective compared to other available therapies, they may not cover our products after FDA approval or, if they do, the level of payment may not be sufficient to allow us to sell our products at a profit.

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In the United States and some foreign jurisdictions, there have been, and likely will continue to be, a number of legislative and regulatory changes and proposed changes regarding the healthcare system directed at broadening the availability of healthcare and containing or lowering the cost of healthcare. In March 2010, the Patient Protection and Affordable Care Act, as amended by the Health Care Education Reconciliation Act, or the ACA, was enacted. The ACA includes measures that have significantly changed, and are expected to continue to significantly change, the way healthcare is financed by both governmental and private insurers. Other legislative changes have been proposed and adopted in the United States since the ACA was enacted. In August 2011, the Budget Control Act of 2011, among other things, created measures for spending reductions by Congress. A Joint Select Committee on Deficit Reduction, tasked with recommending a targeted deficit reduction of at least $1.2 trillion for the years 2013 through 2021, was unable to reach required goals, thereby triggering the legislation’s automatic reduction to several government programs. This includes aggregate reductions of Medicare payments to providers up to 2% per fiscal year, which went into effect in April 2013 and will remain in effect through 2024 unless additional Congressional action is taken. In January 2013, former President Obama signed into law the American Taxpayer Relief Act of 2012, which, among other things, further reduced Medicare payments to several providers, including hospitals, imaging centers and cancer treatment centers, and increased the statute of limitations period for the government to recover overpayments to providers from three to five years. Additionally, in August, 2022, President Biden signed into law the Inflation Reduction Act of 2022, which includes, in part, provisions to lower certain products’ costs for Medicare beneficiaries. Beginning in 2023, if a manufacturer of any of our products increases the prices for the products faster than inflation for such products used by Medicare beneficiaries, then the manufacturer would be required to pay rebates to Medicare. If we establish any relationships with manufacturers for our products, and if our products are used by Medicare beneficiaries, manufacturers would be required to pay rebates to Medicare or else face a penalty fine representing the difference between the actual and the inflated price. Depending on the financial aspects of our relationships with manufacturers for such products, we may incur additional compliance costs which could impact our financial results.

Other Healthcare Laws

Although we currently do not have any products on the market, we may be subject to additional healthcare regulation and enforcement by the federal government and by authorities in the states and other countries in which we conduct our business. Such laws include, without limitation, state and federal anti-kickback, fraud and abuse, false claims, privacy and security and physician sunshine and open payment laws and regulations, many of which may become more applicable to us if our product candidates are approved and we begin commercialization. If our operations are found to be in violation of any of such laws or any other governmental regulations that apply to us, we may be subject to penalties, including, without limitation, administrative, civil and criminal penalties, damages, fines, disgorgement, the curtailment or restructuring of our operations, exclusion from participation in federal and state healthcare programs and imprisonment, any of which could adversely affect our ability to operate our business and our financial results.

Employees

As of November 1, 2023, we had 17 full-time employees, 6 of whom have a Ph.D., 4 have Masters Degrees and 2 have Biotechnology Certificates, for a total of 12 with post-graduate degrees. Of these full-time employees, 12 are engaged in research and development activities and 5 are engaged in finance, legal, human resources, facilities, and general management. None of our employees are represented by any labor union. As of November 1, 2023, 16 employees were located in the United States of America, and 1 employee was located in Australia

Corporate Information

We were incorporated as a Delaware corporation on November 22, 2019 and completed the Re-domiciliation April 15, 2020. Our predecessor, Benitec Limited, was incorporated under the laws of Australia in 1995. Our principal executive offices are located at 3940 Trust Way, Hayward, California 94545.

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Re-domiciliation

On April 15, 2020, or the Implementation Date, the re-domiciliation, or the Re-domiciliation, of Benitec Biopharma Limited, a public company incorporated under the laws of the State of Western Australia, or Benitec Limited, was completed in accordance with the Scheme Implementation Agreement, as amended and restated as of January 30, 2020, between Benitec Limited and us. As a result of the Re-domiciliation, our jurisdiction of incorporation was changed from Australia to Delaware, and Benitec Limited became our wholly owned subsidiary.

The Re-domiciliation was effected pursuant to a statutory scheme of arrangement under Australian law, or the Scheme, whereby on the Implementation Date, all of the issued and outstanding ordinary shares of Benitec Limited were exchanged for newly issued shares of our common stock, on the basis of one share of our common stock, par value $0.0001 per share, for every 300 ordinary shares of Benitec Limited issued and outstanding. Holders of Benitec Limited’s American Depository Shares, or ADSs (each of which represented 200 ordinary shares), received two shares of our common stock for every three ADSs held.

Our common stock began trading on The Nasdaq Capital Market, or Nasdaq, at the start of trading on the Implementation Date under the symbol “BNTC.”

COVID-19

COVID-19 has been declared a pandemic by the World Health Organization and has spread to nearly every country, including Australia and the United States. The impact of this pandemic has been and may continue to be extensive in many aspects of society, which has resulted in and may continue to result in significant disruptions to businesses and capital markets around the world. The extent to which the coronavirus continues to impact us will depend on future developments, which are highly uncertain and cannot be predicted, including new information which may emerge concerning the severity of the coronavirus and its variants, and the actions to contain the coronavirus or treat its impact, including the effectiveness and adoption of vaccines for the virus, among others.

Certain of our research and development efforts are conducted globally, including the ongoing development of our silence and replace therapeutic for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD), and will be dependent upon our ability to continue our preclinical and clinical studies and related work despite the COVID-19 pandemic and any similar events.

Reverse Stock Split

On July 26, 2023, the Company effected a 1-for-17 reverse stock split (the “Reverse Stock Split”) of its common stock. In accordance with the Reverse Stock Split, 17 pre-split shares of the Company’s common stock were automatically converted into one issued and outstanding post-split share. Proportional adjustments were also made to all outstanding stock options, pre-funded warrants, and common warrants in accordance with their respective terms. The Reverse Stock Split did not change the par value of the Company’s common stock or the authorized number of shares. No fractional shares were issued in connection with the Reverse Stock Split. All fractional shares were rounded up to the nearest whole share with respect to outstanding shares of common stock. All share and earnings per share amounts presented in this Form 10-Q reflect the impact of this reverse split.

Available Information

Our telephone number is (510) 780-0819, and our Internet website is www.benitec.com. The information on, or that can be accessed through, our website is not part of this Quarterly Report on Form 10-Q and is not incorporated by reference herein.

Royalties, milestone payments and other license fees

We are required to pay royalties, milestone payments and other license fees in connection with our licensing of intellectual property from third parties, including as discussed below.

We have collaborated with Biomics Biotechnologies Co., Ltd., or Biomics, pursuantForeign Currency Translation and Other Comprehensive Income (Loss)

The Company’s functional currency and reporting currency is the United States dollar. BBL’s functional currency is the Australian dollar (AUD). Assets and liabilities are translated at the exchange rate in effect at the balance sheet date. Revenues and expenses are translated at the average rate of exchange prevailing during the reporting period. Equity transactions are translated at each historical transaction date spot rate. Translation adjustments arising from the use of different exchange rates from period to several collaboration agreements in relation to single-stranded RNAperiod are included as a component of stockholders’ equity as “Accumulated other comprehensive loss.” Gains and shRNA sequences for treatment of hepatitis B. In July 2015, we entered into an earn-out agreement with Biomics which confirmed Benitec’s ownership of certain patentslosses resulting from foreign currency translation are included in the collaboration in exchangeconsolidated statements of operations and comprehensive loss as other comprehensive income (loss).

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Other comprehensive income for an upfront paymentall periods presented consists entirely of foreign currency translation gains and equity issuance to Biomics and a share of certain future licensing revenue received by Benitec.

October 2020 Capital Raise

On October 6, 2020, the Company announced the closing of an underwritten public offering of common stock and common stock equivalents. The Company received gross proceeds of approximately $11.5 million and net proceeds of approximately $9.9 million from the offering.losses.

April 2021 Capital Raise

On April 30, 2021, the Company announced the closing of an underwritten public offering of common stock and common stock equivalents. The Company received gross proceeds of approximately $14.3 million and net proceeds of approximately $12.7 million from the offering.

September 2022 Capital Raise

On September 15,16, 2022, the Company announced the closing of an underwritten public offering of common stock and common stock equivalents. The Company received gross proceeds of approximately $17.9 million and net proceeds of approximately $16.0 million from the offering.

August 2023 Capital Raise

On August 11, 2023 we closed an underwritten public offering in which we sold 875,949 shares of common stock, 15,126,226 pre-funded warrants to purchase 15,126,226 shares of common stock, and 16,002,175 common warrants to purchase up to 16,002,175 shares of common stock.The pre-funded warrants were immediately exercisable until exercised in full at an exercise price of $0.0001 per share of common stock. The common warrants were immediately exercisable at a price per share of common stock of $3.86 and expire on the fifth anniversary of such initial exercisable date. The combined purchase price for each share of common stock and accompanying common warrant was $1.93, which was allocated as $1.9299 per share of common stock and $0.0001 per common warrant. Each pre-funded warrant was sold together with one common warrant at a combined price of $1.9299, which was allocated as $1.9298 per pre-funded warrant and $0.0001 per common warrant. In addition, the Company granted the underwriter an option to purchase up to 2,331,606 additional shares of common stock and/or up to 2,331,606 additional common warrants. As of September 30, 2023 the underwriter had partially exercised this option and purchased 458,134 additional shares of common stock and 458,134 additional common warrants. Net proceeds from the offering, including the impact of the underwriter’s partial exercise of its option and net of underwriting discounts, commissions, and other offering expenses, totaled $27.9 million.

The Company has 29,809,471 outstanding Series 2 warrants (the “Series 2 Warrants”) which are currently exercisable into 1,753,503 shares of common stock after giving effect to the Reverse Stock Split. The Series 2 Warrants contain an exercise price adjustment mechanism providing that certain issuances of common stock (or common stock equivalents) if made at a price lower than the existing exercise price of $11.22 of such Series 2 Warrants, would reset the exercise price to such lower price. As a result of the August 11, 2023 public offering, the exercise price of the Series 2 Warrants has been automatically reset as of the closing time of such public offering to $1.9299.

Results of Operations

Revenues

The Company has not generated any revenues from the sales of products. Revenues from licensing fees are included in the revenue from customers line item on our consolidated statements of operations and comprehensive loss. Our licensing fees have been generated through the licensing of our ddRNAi technology to biopharmaceutical companies. The Company recognized licensing fee revenues totaling $68 thousand and $73 thousanddid not recognize any revenue during the ninethree months ended March 31,September 30, 2023 and March 31, 2022, respectively. Licensing fee revenue for the three-month periods ended March 31, 2023 and March 31, 2022 totaled $54 thousand and $48 thousand, respectively.September 30, 2022.

 

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Royalties and License Fees

Royalties and license fees consist primarily of payments we are required to remit for royalties and other payments related to in-licensed intellectual property. Under our in-license agreements, we may pay up-front fees and milestone payments and be subject to future royalties. We cannot precisely predict the amount, if any, of royalties we will owe in the future, and if our calculations of royalty payments are incorrect, we may owe additional royalties, which could negatively affect our results of operations. As our product sales increase, we may, from time to time, disagree with our third-party collaborators as to the appropriate royalties owed, and the resolution of such disputes may be costly, may consume management’s time, and may damage our relationship with our collaborators. Furthermore, we may enter into additional license agreements in the future, which may also include royalty, milestone and other payments.

Expenses

Research and Development Expenses

Research and development expenses relate primarily to the cost of conducting clinical and preclinicalpre-clinical trials. PreclinicalPre-clinical and clinical development costs are a significant component of research and development expenses. The Company records accrued liabilities for estimated costs of research and development activities conducted by third-party service providers, which include the conduct of preclinicalpre-clinical studies and clinical trials, and contract manufacturing activities. The Company records the estimated costs of research and development activities based upon the estimated amount of services provided but not yet invoiced and includes these costs in trade and other payables on the consolidated balance sheets and within research and development expenses on the consolidated statements of operations and comprehensive loss.

The Company accrues for these costs based on factors such as estimates of the work completed and in accordance with agreements established with its third-party service providers. The Company makes significant judgments and estimates in determining the accrued liabilities balance at the end of each reporting period. As actual costs become known, the Company adjusts its accrued liabilities. The Company has not experienced any material differences between accrued costs and actual costs incurred.

General and Administrative Expenses

General and administrative expenses consist primarily of salaries, related benefits, travel, and equity-based compensation expense. General and administrative expenses also include facility expenses, professional fees for legal, consulting, accounting and audit services and other related costs.

We anticipate that our general and administrative expenses may increase as the Company focuses on the continued development of the preclinicalpre-clinical OPMD program. The Company also anticipates an increase in expenses relating to accounting, legal and regulatory-related services associated with maintaining compliance with exchange listing and SEC requirements, director and officer insurance premiums and other similar costs.

On September 13, 2023, the Compensation Committee (the “Compensation Committee”) of the Company’s Board of Directors approved increases of the base salaries of Dr. Jerel Banks, the Company’s Executive Chairman and Chief Executive Officer, and Megan Boston, the Company’s Executive Director, to $655,200 and $350,784 (Ms. Boston’s salary as noted has been converted from AUD $1.00 to USD $0.64, which was the conversion rate as of September 13, 2023) respectively, each adjustment being effective as of October 1, 2023.

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Operating Expenses

The following tables sets forth a summary of our expenses for each of the periods:

 

  Three Months Ended 
  Three Months Ended   Nine Months Ended   September 30, 
  March 31,   March 31,   2023   2022 
  2023   2022   2023   2022         
  (US$’000)   (US$ 000) 

Operating Expenses:

            

Royalties and license fees

   (106)    —   

Research and development

  $3,167   $2,171   $9,588   $8,096    4,429    2,660 

General and administrative

   1,228    1,337    5,011    5,093    1,551    1,920 
  

 

   

 

   

 

   

 

   

 

   

 

 

Total operating expenses

  $4,395   $3,508   $14,599   $13,189   $5,874   $4,580 
  

 

   

 

   

 

   

 

   

 

   

 

 

During the three and nine months ended March 31,September 30, 2023 and September 30, 2022, we incurred $3.2royalties and license fees expenses of ($106) thousand and zero, respectively. The credit to expense during the quarter ended September 30, 2023 reflects the reversal of an accrual for license fees no longer due.

During the three months ended September 30, 2023 and September 30, 2022, respectively, we incurred $4.4 million and $9.6$2.7 million in research and development expenses, respectively, as compared to $2.2 million and $8.1 million for the comparable periods ended March 31, 2022.respectively. Research and development expenses relate primarily to the OPMD project. The year-over-year increasesongoing clinical development of BB-301 for the nine-month period reflect conclusiontreatment of the BB-301 Regulatory Toxicology Study and the Parallel Assay Method Development, Qualification, and Validation project, and the continuation of the GMP manufacturing project and Natural History Study.OPMD.

General and administrative expense totaled $1.2$1.6 million and $5.0$1.9 million for the three and nine months ended March 31,September 30, 2023 compared to $1.3 million and $5.1 million for the comparable periods ended March 31, 2022.September 30, 2022, respectively. The year-over-year decrease for the three-month periods ended March 31period relates primarily to lower listingdecreases in stock-based compensation ($215 thousand), legal fees ($203 thousand), and filing fees and stock-based compensation. See Note 9 of the Notes to Consolidated Financial Statements included in this Quarterly Report on Form 10-Q.insurance premiums ($37 thousand); partially offset by higher travel expense ($54 thousand).

 

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Other Income (Loss)(Expense)

The following tables sets forth a summary of our other income (loss) for each of the periods:

 

   Three Months Ended   Nine Months Ended 
   March 31,   March 31, 
   2023   2022   2023   2022 
   (US$’000) 

Other Income (Loss):

        

Foreign currency transaction gain (loss)

  $(45)   $229   $(391)   $36 

Interest expense, net

   (7)    (10)    (25)    (22) 

Other income, net

   —      (29)    50    (29) 

Unrealized loss on investment

   (4)    (5)    (4)    (10) 
  

 

 

   

 

 

   

 

 

   

 

 

 

Total other income (loss), net

  $(56)   $185   $(370)   $(25) 
  

 

 

   

 

 

   

 

 

   

 

 

 
   Three Months Ended 
   September 30, 
   2023   2022 
         
   (US$’000) 

Other Income (Loss):

    

Foreign currency transaction loss

  $(56)   $(507) 

Interest expense, net

   (6)    (9) 

Other expense, net

   (18)    —   

Unrealized gain on investment

   —      3 
  

 

 

   

 

 

 

Total other loss, net

  $(80)   $(513) 

Other income (loss),The other loss, net during the three and nine months ended March 31,September 30, 2023 and September 30, 2022, respectively, totaled $80 thousand and $513 thousand, which consists of foreign currency transaction gain (loss),losses, interest expense, other income,expense, net and unrealized lossgain on investment, totaled $(56) thousand and $(370) thousand, respectively. During the three and nine months ended March 31, 2022, respectively, other income (loss) totaled $185 thousand and $(25) thousand. The swings to foreigninvestment. Foreign currency transaction losses during the three- and nine-month month periods ended March 31 reflect changeshave decreased due to a change in foreign exchange rates. Other income for the nine-month period ended March 31, 2023expense, net relates to recognition of a refund.state income taxes. Unrealized lossgain on investment decreased for the three- and nine- month periodsthree months ended March 31,September 30, 2023 compared to the same prior year periods.three months ended September 30, 2022.

Liquidity and Capital Resources

The Company has incurred cumulative losses and negative cash flows from operations since our predecessor’s inception in 1995. The Company had accumulated losses of $163$173.8 million as of March 31,September 30, 2023. We expect that our research and development expenses may increase due to the continued development of the OPMD program. It is also likely that there will be an increase in the general and administrative expenses due to the obligations of being a domestic public company in the United States.

We had no borrowings as of March 31,September 30, 2023 and do not currently have a credit facility.

As of March 31,September 30, 2023, we had cash and cash equivalents of approximately $6.6$25.9 million. Cash in excess of immediate requirements is invested in accordance with our investment policy, primarily with a view to liquidity and capital preservation. Currently, our cash and cash equivalents are held in bank accounts.

The following table sets forth a summary of the net cash flow activity for each of the periods set forth below:

 

  Three Months Ended 
  Nine Months Ended   September 30, 
  March 31,   2023   2022 
  2023   2022         
  (US$’000)   (US$’000) 

Net cash provided by (used in):

        

Operating activities

  $(13,917)   $(11,044)   $(4,577)   $(4,045) 

Investing activities

   —      —   

Financing activities

   16,015    —      27,919    16,015 

Effects of exchange rate changes on cash and cash equivalents

   391    (95)    45    501 
  

 

   

 

   

 

   

 

 

Net increase (decrease) in cash, cash equivalents, and restricted cash

  $2,489   $(11,139) 

Net increase in cash

  $23,387   $12,471 
  

 

   

 

   

 

   

 

 

Operating activities

Net cash used in operating activities for the ninethree months ended March 31,September 30, 2023 and 2022 was $13.9$4.6 million and $11.0$4.0 million, respectively. Net cash used in operating activities was primarily the result of our net loss, partially offset by non-cash expenses, and changes in working capital, including an increase in payables.payables and a decrease in prepaid expenses.

 

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Financing activities

Net cash provided by financing activities was $27.9 million and $16.0 million and $0 for the ninethree months ended March 31,September 30, 2023 and 2022, respectively. Cash from financing activities during the nine months ended March 31in 2023 was related to the issuance of common stock, pre-funded warrants, and Series 2common warrants, including $17.9 million inwith gross proceeds from the September 2022 Capital Raise,of $30.9 million, partially offset by $1.9$2.9 million in share issuance costs.

The future of the Company as an operating business will depend on its ability to manage operating costs and budgeted amounts and obtain adequate financing. While we continue to progress discussions and advance opportunities to engage with pharmaceutical companies and continue to seek licensing partners for ddRNAi in disease areas that are not our focus, there can be no assurance as to whether we will enter into such arrangements or what the terms of any such arrangement could be.

While we have established some licensing arrangements, we do not have any products approved for sale and have not generated any revenue from product sales. We do not know when, or if, we will generate any revenue from product sales. We do not expect to generate significant revenue from product sales unless and until we obtain regulatory approval of and commercialize one of our current or future product candidates.

Unless and until we establish significant revenues from licensing programs, strategic alliances or collaboration arrangements with pharmaceutical companies, or from product sales, we anticipate that we will continue to generateincur losses for the foreseeable future, and we expect the losses to increase as we continue the development of product candidates and begin to prepare to commercialize any product that receives regulatory approval. We are subject to the risks inherent in the development of new gene therapy products, and we may encounter unforeseen expenses, difficulties, complications, delays, and other unknown factors that may adversely affect our business. The Company has performed a review of itsWe estimate that our cash flow forecasts and has concluded that substantial doubt exists as to its ability to continue as a going concern. The Company does not havecash equivalents will be sufficient liquidity to fund itsthe Company’s operations for at least the next twelve months without raising additional funds and the success of raising such additional capital is not solely within the Company’s control.months.

We have based our projections of operating capital requirements on assumptions that may prove to be incorrect and we may use all of our available capital resources sooner than we expect. Because of the numerous risks and uncertainties associated with research, development, and commercialization of pharmaceutical products, we are unable to estimate the exact amount of our operating capital requirements. Our future funding requirements will depend on many factors, including, but not limited to:

 

the timing and costs of our planned clinical trials for our ddRNAi and silence and replace product candidates;

 

the timing and costs of our planned preclinical studies for our ddRNAi and silence and replace product candidates;

 

the number and characteristics of product candidates that we pursue;

 

the outcome, timing, and costs of seeking regulatory approvals;

 

revenue received from commercial sales of any of our product candidates that may receive regulatory approval;

 

the terms and timing of any future collaborations, licensing, consulting, or other arrangements that we may establish;

 

the amount and timing of any payments we may be required to make, or that we may receive, in connection with the licensing, filing, prosecution, defense and enforcement of any patents or other intellectual property rights;

 

the costs of preparing, filing and prosecuting patent applications, maintaining and protecting our intellectual property rights and defending against intellectual property related claims; and

 

•

the extent to which we need to in-license or acquire other products and technologies.

the extent to which we need to in-license or acquire other products and technologies.

Contractual Obligations and Commercial Commitments

On October 1, 2016, the Company entered into an operating lease for office space in Hayward, California that originally expired in April 2018. The Company has entered into lease amendments that extended the lease through June 2025. See Note 8 of the Notes to Consolidated Financial Statements included in this Quarterly Report on Form 10-Q.

The Company enters into contracts in the normal course of business with third-party contract research organizations, contract development and manufacturing organizations and other service providers and vendors. These contracts generally provide for termination on notice and, therefore, are cancellable contracts and not considered contractual obligations and commitments.

Critical Accounting Policies and Significant Accounting Estimates

The preparation of consolidated financial statements and related disclosures in conformity with accounting principles generally accepted in the United States of America requires management to make judgments, assumptions and estimates that affect the amounts reported. Note 2 of the Notes to Consolidated Financial Statements included in this Quarterly Report on Form 10-Q describes the significant accounting policies used in the preparation of the consolidated financial statements. Certain of these significant accounting policies are considered to be critical accounting policies.

 

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A critical accounting policy is defined as one that is both material to the presentation of the Company’s consolidated financial statements and requires management to make difficult, subjective, or complex judgments that could have a material effect on the Company’s financial condition or results of operations. Specifically, these policies have the following attributes: (1) the Company is required to make assumptions about matters that are highly uncertain at the time of the estimate; and (2) different estimates the Company could reasonably have used, or changes in the estimate that are reasonably likely to occur, would have a material effect on the Company’s financial condition or results of operations.

Estimates and assumptions about future events and their effects cannot be determined with certainty. The Company bases its estimates on historical experience and on various other assumptions believed to be applicable and reasonable under the circumstances. These estimates may change as new events occur, as additional information is obtained and as the Company’s operating environment changes. These changes have historically been minor and have been included in the consolidated financial statements as soon as they became known. In addition, management is periodically faced with uncertainties, the outcomes of which are not within its control and will not be known for prolonged periods of time. These uncertainties are discussed in the section above entitled “Risk Factors.” Based on a critical assessment of its accounting policies and the underlying judgments and uncertainties affecting the application of those policies, management believes that the Company’s consolidated financial statements are fairly stated in accordance with accounting principles generally accepted in the United States of America and provide a meaningful presentation of the Company’s financial condition and results of operations.

Management believes that the following are critical accounting policies:

Research and Development Expense

Research and development expenses relate primarily to the cost of conducting clinical and preclinicalpre-clinical trials. PreclinicalPre-clinical and clinical development costs are a significant component of research and development expenses. The Company records accrued liabilities for estimated costs of research and development activities conducted by third-party service providers, which include the conduct of preclinicalpre-clinical studies and clinical trials, and contract manufacturing activities. The Company records the estimated costs of research and development activities based upon the estimated amount of services provided but not yet invoiced and includes these costs in trade and other payables on the consolidated balance sheets and within research and development expenses on the consolidated statements of operations and comprehensive loss.

The Company accrues for these costs based on factors such as estimates of the work completed and in accordance with agreements established with its third-party service providers. The Company makes significant judgments and estimates in determining the accrued liabilities balance at the end of each reporting period. As actual costs become known, the Company adjusts its accrued liabilities. The Company has not experienced any material differences between accrued costs and actual costs incurred.

Share-based Compensation Expense

The Company records share-based compensation in accordance with ASC 718, Stock Compensation.Compensation. ASC 718 requires the fair value of all share-based employee compensation awarded to employees and non-employees to be recorded as an expense over the shorter of the service period or the vesting period. The Company determines employee and non-employee share-based compensation based on grant-date fair value using the Black-Scholes Option Pricing Model.

Recent Accounting Pronouncements

Accounting Standards recently adopted

None.

New Accounting Standards and Interpretations not yet mandatory or early adopted

ASU 2016-13-In2016-13 – In June 2016, the FASB issued ASU No. 2016-13: “Financial“Financial Instruments-Credit Losses (Topic 326)”. This ASU represents a significant change in the accounting for credit losses model by requiring immediate recognition of management’s estimates of current expected credit losses (CECL). Under the prior model, losses were recognized only as they were incurred. The Company has determined that it has met the criteria of a smaller reporting company (“SRC”) as of November 15, 2019. As such, ASU 2019-10: “Financial Instruments-Credit Losses, Derivatives and Hedging, and Leases: Effective Dates” amended the effective date for the Company to be for reporting periods beginning after December 15, 2022. The Company will adoptadopted this ASU effective July 1, 2023.2023 and determined that its impact on the accompanying consolidated financial statements is immaterial.

New Accounting Standards and Interpretations not yet mandatory or early adopted

None.

 

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Item 3.

Item 3. Quantitative and Qualitative Disclosures About Market Risk

As a smaller reporting company, we are not required to provide the information pursuant to this Item.

Item 4.

Item 4. Controls and Procedures

We have established disclosure controls and procedures (as defined in Rules 13a-15(e)13a-15€ and 15d-15(e) under the Securities Exchange Act of 1934, as amended). As of the end of the period covered by this Report we carried out an evaluation under the supervision and with the participation of our management, including our principal executive officer and principal financial and accounting officer, of the effectiveness of our disclosure controls and procedures pursuant to Rule 13a-15 of the Securities and Exchange Act of 1934, as amended. Based upon that evaluation, our principal executive officer and principal financial and accounting officer concluded that our disclosure controls and procedures are effective.

There were no changes in our internal controls over financial reporting during the quarter ended March 31,September 30, 2023 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

We do not expect that our disclosure controls and procedures or our internal controls will prevent all errors and all fraud. A control system, no matter how well conceived and operated, can provide only reasonable, not absolute, assurance that the objectives of the control system are met. Further, the design of a control system must reflect the fact that there are resource constraints, and the benefits of controls must be considered relative to their costs. Because of the inherent limitations in all control systems, no evaluation of controls can provide absolute assurance that all control issues and instances of fraud, if any, have been detected.

 

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PART II

OTHER INFORMATION

Item 1.

Item 1. Legal Proceedings

We are currently not a party to any material legal proceedings.

Item 1A.

Item 1A. Risk Factors

There have been no material changes to the risk factors disclosed in Item 1A of the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2022 other than as set forth below.2023.

We may fail to continue to meet the listing standardsItem 2. Unregistered Sales of The Nasdaq Capital MarketEquity Securities, Use of Proceeds, and our failure to maintain the listingIssuer Purchases of our common stock with a U.S. national securities exchange could adversely affect the liquidity off our common stock.Equity Securities

Our common stock is currently listed on The Nasdaq Capital Market. In order to maintain that listing, we must satisfy minimum financial and other continued listing requirements and standards, including maintaining a minimum share price. For example, the current continued listing requirements of Nasdaq provide, among other things, that a company may be delisted if the bid price of its stock drops below $1.00 for a period of 30 consecutive business days.

On September 6, 2022, we received a letter from the Listing Qualifications Department of the Nasdaq Stock Market notifying us that the minimum bid price per share for our common stock fell below $1.00 for a period of 30 consecutive business days and that therefore we did not meet the minimum bid price requirement set forth in Nasdaq Listing Rule 5550(a)(2). The letter stated that we will be provided 180 calendar days, or until March 6, 2023 to regain compliance with the minimum bid price requirement.

On March 7, 2023, Nasdaq notified the Company in writing that while the Company had not regained compliance with the minimum bid price requirement, it was eligible for an additional 180 calendar day period, or until September 5, 2023, to regain compliance with the minimum bid price requirement. Nasdaq’s determination was based on the Company having met the continued listing requirement for market value of publicly held shares and all other applicable requirements for initial listing on The Nasdaq Capital Market, with the exception of the minimum bid price requirement and on the Company’s written notice to Nasdaq of its intention to cure the deficiency during the second compliance period by effecting a reverse stock split, if necessary. If at any time during this additional time period the closing bid price of the Company’s common stock is at least $1.00 per share for a minimum of 10 consecutive business days, Nasdaq will provide written confirmation of compliance and the matter will be closed.

If the Company does not regain compliance by September 5, 2023, then Nasdaq will provide written notification that the Company’s common stock will be delisted, at which point the Company may appeal Nasdaq’s determination to a Hearings Panel. The Company would remain listed on Nasdaq pending the Hearings Panel’s decision. There can be no assurance that, if the Company does appeal the delisting determination by Nasdaq to the Hearings Panel, that such appeal would be successful.

In the event of a delisting, we would take actions to restore our compliance with Nasdaq’s listing requirements, but we can provide no assurance that any such action taken by us would allow our common stock to become listed again, stabilize the market price or improve the liquidity of our securities, prevent our common stock from dropping below the Nasdaq minimum share price requirement or prevent future non-compliance with Nasdaq’s listing requirements.

We intend to monitor the closing bid price of our common stock and may, if appropriate, consider implementing available options to regain compliance with the minimum bid price requirement. These options include completing a reverse stock split of our common stock for the purpose of meeting the closing bid price requirement. Our stockholders approved a reverse stock split at our stockholder meeting on December 7, 2022.

If our common stock were to be delisted from Nasdaq, our common stock could begin to trade on one of the markets operated by OTC Markets Group, including OTCQX, OTCQB or OTC Pink (formerly known as the “pink sheets”), as the case may be. In such event, our common stock could be subject to the “penny stock” rules which, among other things, require brokers or dealers to approve investors’ accounts, receive written agreements and determine investor suitability for transactions and disclose risks relating to investing in the penny stock market. Any such delisting of our common stock could have an adverse effect on the market price of, and the efficiency of the trading market for our common stock, not only in terms of the number of shares that can be bought and sold at a given price, but also through delays in the timing of transactions and less coverage of us by securities analysts, if any. Also, if in the future we were to determine that we need to seek additional equity capital, it could have an adverse effect on our ability to raise capital in the public or private equity markets. In addition, there can be no assurance that our common stock would be eligible for trading on any such alternative exchange or markets.

30

We will need to continue our efforts to raise additional funding, which may not be available on acceptable terms, or at all. Failure to obtain capital when needed may negatively impact our ability to continue as a going concern.

Developing ddRNAi products is expensive, and we expect our research and development expenses to increase substantially in connection with our ongoing activities, particularly as we advance our product candidates in preclinical studies and in future clinical trials and as we undertake preclinical studies of new product candidates. As of March 31, 2023, our cash and cash equivalents were $6.6 million. We estimate that without additional financing, our existing cash and cash equivalents will not be sufficient to fund our operations for the next twelve months and the success of raising such additional capital is not solely within the Company’s control. Our operating plan may require changes and delays as a result of such liquidity issues. We may need to seek additional funds sooner than planned, through public or private equity or debt financings, government grants or other third-party funding, strategic alliances and licensing arrangements or a combination of these approaches. In addition, because the length of time and activities associated with successful development of our product candidates is highly uncertain, we are unable to estimate the actual funds we will require for development and any approved marketing and commercialization activities. In any event, we will require additional capital to obtain regulatory approval for our product candidates and to commercialize any product candidates that receive regulatory approval.

Any additional fundraising efforts may divert our management from their day-to-day activities, which may compromise our ability to develop and commercialize our product candidates. In addition, we cannot guarantee that future financing will be available in sufficient amounts or on terms acceptable to us, if at all. Moreover, the terms of any financing may adversely affect the holdings or the rights of our shareholders, and the issuance of additional securities, whether equity or debt, by us, or the possibility of such issuance, may cause the market price of our common stock to decline. If we incur indebtedness we may be required to agree to restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire, sell or license intellectual property rights and other operating restrictions that could compromise our ability to conduct our business. We could also seek financing through arrangements with collaborative partners at an earlier stage than would otherwise be desirable and we may be required to relinquish rights to some or all of our technologies or product candidates or otherwise agree to terms unfavorable to us.

If we are unable to obtain funding on a timely basis or on acceptable terms, we may be required to significantly curtail, delay or discontinue one or more of our research or development programs or the commercialization of any approved product candidates.

Item 2.

Unregistered Sales of Equity Securities and Use of Proceeds

None.

Item 3.

Item 3. Defaults Upon Senior Securities

None.

Item 4.

Item 4. Mine Safety Disclosures

None.

Not applicable.

Item 5.

Item 5. Other Information

None.

During the three-month period ended September 30, 2023, none of our directors or officers adopted, modified, or terminated a “Rule 10b5-1 trading arrangement” or a “non-Rule 10b5-1 trading arrangement” as such terms are defined under Item 408 of Regulation S-K.

31

Item 6.

Item 6. Exhibits.

 

Number  Description of Document
    3.1Amended and Restated Certificate of Incorporation of Benitec Biopharma Inc. (incorporated by reference in Exhibit 3.1 to the Registrant’s Form 8-K filed on April 15, 2020 (File No. 001-39267))
    3.2Certificate of Amendment to the Amended and Restated Certificate of Incorporation of Benitec Biopharma Inc., effective December 17, 2021 (incorporated by reference in Exhibit 3.1 to the Registrant’s Form 8-K filed on December 21, 2021 (File No. 001-39267))
    3.3Certificate of Amendment to the Amended and Restated Certificate of Incorporation of Benitec Biopharma Inc., effective December 9, 2022 (incorporated by reference in Exhibit 3.1 to the Registrant’s Form 8-K filed on December 12, 2022 (File No. 001-39267))
    3.4Certificate of Amendment to the Amended and Restated Certificate of Incorporation of Benitec Biopharma Inc., dated July 25, 2023 (incorporated by reference in Exhibit 3.1 to the Registrant’s Form 8-K filed on July 25, 2023 (File No. 001-39267))
    4.1Form of Pre-Funded Warrant (incorporated by reference to Exhibit 4.1 to the Company’s Form 8-K filed on August 11, 2023 (File. No. 001-39267))
    4.2Form of Common Warrant (incorporated by reference to Exhibit 4.2 to the Company’s Form 8-K filed on August 11, 2023 (File No. 001-39267))
    4.3Warrant Agency Agreement, dated August 11, 2023, by and between Benitec Biopharma Inc. and Computershare Trust Company, N.A. (incorporated by reference to Exhibit 4.3 to the Company’s Form 8-K filed on August 11, 2023 (File. No. 001-39267))
    1.2Underwriting Agreement, dated August 8, 2023, by and between Benitec Biopharma Inc. and Citizens JMP Securities LLC (incorporated by reference to Exhibit 1.1 to the Company’s Form 8-K filed on August 11, 2023 (File No. 001-39267))
  31.1  Statement of CEO Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002*
  31.2  Statement of CFO Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002*
  32.1  Statement of CEO Pursuant to Section 906 of the Sarbanes-Oxley Act of 2002**
  32.2  Statement of CFO Pursuant to Section 906 of the Sarbanes-Oxley Act of 2002**
101.INS  Inline XBRL Instance Document*
101.SCH  Inline XBRL Taxonomy Extension Schema Document*
101.CAL  Inline XBRL Calculation Linkbase Document*
101.DEF  Inline XBRL Taxonomy Extension Definition Linkbase Document*
101.LAB  Inline XBRL Label Linkbase Document*
101.PRE  Inline XBRL Taxonomy Presentation Linkbase Document*
104  Cover Page Interactive Data File—the cover page interactive data file does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document

 

*

Filed herewith.

**

Furnished, not filed.

 

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SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on our behalf by the undersigned thereunto duly authorized.

 

   Benitec Biopharma Inc.
Dated: November 13, 2023
Dated: May 15, 2023   

/s/ Jerel Banks

   Jerel Banks
   Executive Chairman and Chief Executive Officer
   (principal executive officer)
   

/s/ Megan Boston

   Megan Boston
   Executive Director (principal financial and accounting officer)

 

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