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Rocket Pharmaceuticals (RCKT)

Rocket Pharmaceuticals, Inc. is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Company's platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia, and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rocket's first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.

Company profile

Ticker
RCKT
Exchange
CEO
Gaurav Shah
Employees
Incorporated
Location
Fiscal year end
Former names
INOTEK PHARMACEUTICALS CORP
SEC CIK
Subsidiaries
Rocket Pharmaceuticals, Ltd. • Rocket Foundation, Inc. • Spacecraft Seven, LLC ...

RCKT stock data

Analyst ratings and price targets

Last 3 months

Investment data

Data from SEC filings
Securities sold
Number of investors

Calendar

9 Aug 22
30 Sep 22
31 Dec 22
Quarter (USD) Jun 22 Mar 22 Dec 21 Sep 21
Revenue
Cost of revenue
Operating income
Operating margin
Net income
Net profit margin
Cash on hand
Change in cash
Diluted EPS
Annual (USD) Dec 21 Dec 20 Dec 19 Dec 18
Revenue
Cost of revenue
Operating income
Operating margin
Net income
Net profit margin
Cash on hand
Change in cash
Diluted EPS
Cash burn rate (est.) Burn method: Change in cash Burn method: Operating income Burn method: FCF (opex + capex)
Last Q Avg 4Q Last Q Avg 4Q Last Q Avg 4Q
Cash on hand (at last report) 188.89M 188.89M 188.89M 188.89M 188.89M 188.89M
Cash burn (monthly) (no burn) 6.42M 18.07M 15.9M 13.01M 11.38M
Cash used (since last report) n/a 19.4M 54.61M 48.04M 39.33M 34.4M
Cash remaining n/a 169.49M 134.28M 140.85M 149.57M 154.49M
Runway (months of cash) n/a 26.4 7.4 8.9 11.5 13.6

Beta Read what these cash burn values mean

Date Owner Security Transaction Code Indirect 10b5-1 $Price #Shares $Value #Remaining
12 Aug 22 Martin Wilson RSU Common Stock Grant Acquire A No No 0 28,763 0 28,763
12 Aug 22 Martin Wilson Stock Option Common Stock Grant Acquire A No No 17.47 20,318 354.96K 20,318
12 Aug 22 John Militello RSU Common Stock Grant Acquire A No No 0 14,310 0 14,310
12 Aug 22 Patel Kinnari RSU Common Stock Grant Acquire A No No 0 52,489 0 52,489
12 Aug 22 Jonathan David Schwartz RSU Common Stock Grant Acquire A No No 0 22,896 0 22,896
13 Jun 22 Yalamanchi Naveen Stock Option Common Stock Grant Acquire A No No 10.77 47,745 514.21K 47,745
13F holders Current Prev Q Change
Total holders 144 146 -1.4%
Opened positions 23 21 +9.5%
Closed positions 25 23 +8.7%
Increased positions 58 56 +3.6%
Reduced positions 39 45 -13.3%
13F shares Current Prev Q Change
Total value 949.1M 1.03B -7.8%
Total shares 66.93M 66.42M +0.8%
Total puts 0 14.7K EXIT
Total calls 29.2K 11.5K +153.9%
Total put/call ratio 1.3
Largest owners Shares Value Change
RTW Investments 15.8M $217.45M 0.0%
BLK Blackrock 4.01M $55.12M +20.2%
Wellington Management 3.41M $46.87M -1.9%
Vanguard 3.36M $46.17M +0.2%
Westfield Capital Management 3.35M $46.14M +52.9%
STT State Street 2.54M $34.9M -7.9%
Maverick Capital 2.53M $34.85M +18.7%
Tang Capital Partners 2.53M $0 0.0%
Ra Capital Management 2.11M $28.99M 0.0%
Assenagon Asset Management 1.69M $23.31M +28.3%
Largest transactions Shares Bought/sold Change
Westfield Capital Management 3.35M +1.16M +52.9%
TROW T. Rowe Price 1.41M -1.1M -43.8%
BEN Franklin Resources 661.72K -684.65K -50.9%
BLK Blackrock 4.01M +674.4K +20.2%
Perceptive Advisors 1.17M -511.21K -30.5%
GMT Capital 496.8K +496.8K NEW
Maverick Capital 2.53M +399.2K +18.7%
Healthcare Of Ontario Pension Plan Trust Fund 0 -391.7K EXIT
BCS Barclays 117.35K -388.19K -76.8%
Assenagon Asset Management 1.69M +373.2K +28.3%

Financial report summary

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Risks
  • The outbreak of the novel strain of coronavirus, SARS-CoV-2, which causes COVID-19, could adversely impact our business, including our preclinical and clinical studies.
  • We have a history of operating losses, and we may not achieve or sustain profitability. We anticipate that we will continue to incur losses for the foreseeable future. If we fail to obtain additional funding to conduct our planned research and development efforts, we could be forced to delay, reduce, or eliminate our product development programs or commercial development efforts.
  • Changes in tax law could adversely affect our business and financial condition.
  • The amount of and our ability to use net operating losses and research and development credits to offset future taxable income may be subject to certain limitations and uncertainty.
  • We may need to raise additional funding, which may not be available on acceptable terms, or at all. Failure to obtain this necessary capital when needed may force us to delay, limit or terminate certain of our licensing activities, product development efforts or other operations.
  • We have never generated any revenue from product sales and may never be profitable.
  • We may encounter substantial delays in commencement, enrollment or completion of our clinical trials or may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities, which could prevent us from commercializing our current and future product candidates on a timely basis, if at all.
  • If we have difficulty enrolling a sufficient number of patients to conduct our clinical trials as planned, we may need to delay, limit or terminate planned clinical trials, the occurrence of any of which would harm our business, financial condition, results of operations and prospects.
  • We have not completed any clinical studies of our current product candidates. Preliminary, interim or topline results in our ongoing clinical studies may not be indicative of results obtained when these studies are completed. Furthermore, success in early clinical studies may not be indicative of results obtained in later studies.
  • Our gene therapy product candidates are based on novel technology, which makes it difficult to predict the time and cost of product candidate development and subsequently obtaining regulatory approval. Currently, only a few gene and cell therapy products have been approved in the United States and the European Union.
  • Even though we have obtained orphan designation for certain of our product candidates, we may not be able to realize the benefits of such designation, including potential marketing exclusivity of our product candidates, if approved.
  • A Fast Track or regenerative medicine advanced therapy, or RMAT, designation by the FDA, or a PRIority MEdicines, or PRIME, designation by the EMA, even if granted for any of our current or future product candidates, may not lead to a faster development or regulatory review or approval process, and does not increase the likelihood that our current product candidate and any future product candidates will receive marketing approval.
  • We have received rare pediatric disease designation for RP-A501 for Danon disease, RP-L102 for FA, and RP-L201 for LAD-I. However, a marketing application for these product candidates, if approved, may not meet the eligibility criteria for a rare pediatric disease priority review voucher.
  • Even if we successfully complete the necessary preclinical studies and clinical trials, we cannot predict when, or if, we will obtain regulatory approval to commercialize a product candidate and the approval may be for a narrower indication than we seek.
  • Even if we obtain regulatory approval for a product candidate, we will remain subject to ongoing regulatory obligations and continued regulatory scrutiny.
  • We may never obtain FDA or EMA approval for any of our product candidates in the U.S. or the EU, and even if we do, we may never obtain approval for or commercialize any of our product candidates in any other jurisdiction, which would limit our ability to realize our full market potential.
  • Healthcare legislative reform measures may have a material adverse effect on our business and results of operations.
  • If we are successful in commercializing any product, our relationships with customers and third-party payors will be subject to applicable anti-kickback, fraud and abuse and other healthcare laws and regulations, which could expose us to criminal sanctions, civil penalties, exclusion from government healthcare programs, contractual damages, reputational harm and diminished profits and future earnings.
  • We are subject to environmental, health and safety laws and regulations, and we may become exposed to liability and substantial expenses in connection with environmental compliance or remediation activities.
  • Our manufacturing facilities are subject to significant government regulations and approvals, which are often costly and could result in adverse consequences to our business if we fail to comply with the regulations or maintain the approvals.
  • Our ability to successfully develop and commercialize our product candidates will substantially depend upon the availability of reimbursement for the costs of the resulting drugs and related treatments.
  • Even if approved, we may not successfully commercialize our product candidates.
  • We face intense competition and rapid technological change and the possibility that our competitors may develop therapies that are more advanced or effective than ours, which may adversely affect our financial condition and our ability to successfully commercialize our product candidates.
  • We may not be successful in our efforts to expand our pipeline of additional product candidates for development.
  • The success of our research and development activities, clinical testing and commercialization, upon which we primarily focus, is uncertain.
  • We expect to rely on third parties to conduct some or all aspects of our drug product manufacturing, research and preclinical and clinical testing, and these third parties may not perform satisfactorily.
  • Our reliance on third parties requires us to share our trade secrets, which increases the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed.
  • Disruptions at the FDA and other government agencies caused by funding shortages or global health concerns could hinder their ability to hire, retain or deploy key leadership and other personnel, or otherwise prevent new or modified products from being developed, approved, or commercialized in a timely manner or at all, which could negatively impact our business.
  • Our rights to intellectual property for the development and commercialization of our product candidates are subject to the terms and conditions of licenses granted to us by others.
  • If we breach our license agreements, it could have a material adverse effect on our commercialization efforts for our product candidates.
  • If we are unable to protect the confidentiality of our trade secrets, our business and competitive position may be harmed.
  • If we are unable to obtain or protect intellectual property rights related to our product candidates, we may not be able to compete effectively in our markets.
  • We may not be successful in obtaining or maintaining necessary rights to gene therapy product components and processes for our development pipeline through acquisitions and in-licenses.
  • Issued patents covering our product candidates could be found invalid or unenforceable if challenged in court.
  • Changes in U.S. patent law could diminish the value of patents in general, thereby impairing our ability to protect our products.
  • We may not be able to protect our intellectual property rights throughout the world.
  • Patent terms may be inadequate to protect our competitive position on our product candidates for an adequate amount of time.
  • We may incur substantial costs as a result of litigation or other proceedings relating to patent and other intellectual property rights and we may be unable to protect our rights to, or use, our technology.
  • We may be subject to claims challenging the inventorship or ownership of our patents and other intellectual property.
  • Third-party claims of intellectual property infringement may prevent or delay our development and commercialization efforts.
  • Our business could suffer if it loses the services of, or fails to attract, key personnel.
  • We may be subject to claims that our employees, consultants, or independent contractors have wrongfully used or disclosed confidential information of third parties or that our employees have wrongfully used or disclosed alleged trade secrets of their former employers.
  • We may need to expand our organization and may experience difficulties in managing this growth, which could disrupt our operations.
  • Future acquisitions of businesses or products, formations of strategic alliances or joint ventures with third parties could disrupt our business and harm our financial condition and operating results.
  • Future sales of our common stock in the public market could cause the market price of our common stock to drop significantly, even if our business is performing well.
  • If securities analysts do not publish research or reports about our business or if they publish negative evaluations of our stock, the price of our stock could decline.
  • The price of our common stock may be volatile and fluctuate substantially, which could result in substantial losses for our stockholders.
  • RTW Investments, LP, our largest stockholder, may have the ability to significantly influence all matters submitted to stockholders for approval.
  • Because we do not anticipate paying any cash dividends on our capital stock in the foreseeable future, capital appreciation, if any, will be stockholders’ sole source of gain.
  • As of December 31, 2020, we were no longer an “emerging growth company,” as defined in the JOBS Act. or a “smaller reporting company” as defined in the Exchange Act and were a “large accelerated filer” which subjects us to increased disclosure and compliance requirements.
  • If we fail to maintain proper and effective internal control over financial reporting, our ability to produce accurate and timely financial statements could be impaired, which could harm our operating results, investors’ views of us and, as a result, the value of our common stock.
  • Provisions in our corporate charter documents and under Delaware law could make an acquisition of us, which may be beneficial to our stockholders, more difficult and may prevent attempts by our stockholders to replace or remove our current management.
  • Our internal computer systems, or those of our third-party collaborators or other contractors, may fail or suffer security breaches, which could result in a material disruption of our development programs.
  • The increasing use of social media platforms presents new risks and challenges.
Management Discussion
  • R&D expenses increased $16.8 million to $41.3 million for the three months ended June 30, 2022 compared to the three months ended June 30, 2021. The increase in R&D expenses was primarily driven by an increase in manufacturing and development costs of $11.3 million, an increase in compensation and benefits of $1.9 million due to increased R&D headcount and an increase in laboratory supplies of $1.4 million.
  • G&A expenses increased $3.3 million to $12.9 million for the three months ended June 30, 2022, compared to the three months ended June 30, 2021. The increase in G&A expenses was primarily driven by an increase in commercial preparation expenses which consists of commercial strategy, medical affairs, market development and pricing analysis of $1.4 million, an increase in compensation and benefits of $0.9 million due to increased G&A headcount, an increase in legal expense of $0.5 million, and an increase in stock compensation expense of $0.3 million.
  • Other expense, net decreased by $0.3 million to $0.2 million for the three months ended June 30, 2022, compared to the three months ended June 30, 2021. The decrease in other expense, net was primarily driven by reduced interest expense of $0.2 million associated with the 2022 Convertible Notes that were redeemed in April 2021 and the 2021 Convertible Notes that were converted in August 2021.

Content analysis

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