☒ | QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
☐ | TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
Delaware | 04-3475813 | |
(State or other jurisdiction of incorporation or organization) | (I.R.S. Employer Identification No.) |
9 Cedarbrook Drive, Cranbury, NJ | 08512 | |
(Address of principal executive office) | (Zip Code) |
Title of each class | Trading Symbol(s) | Name of each exchange on which registered |
Common Stock, $0.01 par value per share | RCKT | Nasdaq Global Market |
Large accelerated filer ☒ | Accelerated filer ☐ | |
Non-accelerated filer ☐ | Smaller reporting company ☐ | |
Emerging growth company ☐ |
Page | ||
PART I - FINANCIAL INFORMATION | ||
Item 1. | ||
4 | ||
5 | ||
6 | ||
7 | ||
8 | ||
9 | ||
Item 2. | 19 | |
Item 3. | ||
Item 4. | ||
PART II - OTHER INFORMATION | ||
Item 1. | 31 | |
Item 1A. | 31 | |
Item 2. | ||
Item 3. | ||
Item 4. | ||
Item 5. | ||
Item 6. | ||
Item 1. | Financial Statements |
September 30, | December 31, | |||||||
2021 | 2020 | |||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 245,644 | $ | 297,098 | ||||
Investments | 175,815 | 185,621 | ||||||
Prepaid expenses and other current assets | 3,610 | 4,626 | ||||||
Total current assets | 425,069 | 487,345 | ||||||
Property and equipment, net | 22,090 | 19,206 | ||||||
Goodwill | 30,815 | 30,815 | ||||||
Restricted cash | 1,334 | 1,568 | ||||||
Deposits | 455 | 455 | ||||||
Operating lease right-of-use assets | 1,467 | 914 | ||||||
Finance lease right-of-use asset | 49,011 | 50,521 | ||||||
Total assets | $ | 530,241 | $ | 590,824 | ||||
Liabilities and stockholders' equity | ||||||||
Current liabilities: | ||||||||
Accounts payable and accrued expenses | $ | 22,008 | $ | 25,472 | ||||
Convertible notes, net of unamortized discount, current | 0 | 4,875 | ||||||
Operating lease liabilities, current | 804 | 626 | ||||||
Finance lease liability, current | 1,677 | 1,644 | ||||||
Total current liabilities | 24,489 | 32,617 | ||||||
Convertible notes, net of unamortized discount, non-current | 0 | 35,066 | ||||||
Operating lease liabilities, non-current | 891 | 498 | ||||||
Finance lease liability, non-current | 19,109 | 18,988 | ||||||
Other liabilities | 94 | 136 | ||||||
Total liabilities | 44,583 | 87,305 | ||||||
Commitments and contingencies (Note 11) | 0 | 0 | ||||||
Stockholders' equity: | ||||||||
Preferred stock, $0.01 par value, authorized 5,000,000 shares: | ||||||||
Series A convertible preferred stock; 300,000 shares designated as Series A; 0 shares issued and outstanding | 0 | 0 | ||||||
Series B convertible preferred stock; 300,000 shares designated as Series B; 0 shares issued and outstanding | 0 | 0 | ||||||
Common stock, $0.01 par value, 120,000,000 shares authorized; 64,442,601 and 60,996,367 shares issued and outstanding at September 30, 2021 and December 31, 2020, respectively | 644 | 610 | ||||||
Additional paid-in capital | 932,776 | 825,794 | ||||||
Accumulated other comprehensive loss | (97 | ) | (42 | ) | ||||
Accumulated deficit | (447,665 | ) | (322,843 | ) | ||||
Total stockholders' equity | 485,658 | 503,519 | ||||||
Total liabilities and stockholders' equity | $ | 530,241 | $ | 590,824 |
June 30, 2022 | December 31, 2021 | |||||||
(unaudited) | ||||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 187,548 | $ | 232,694 | ||||
Investments | 133,820 | 156,046 | ||||||
Prepaid expenses and other current assets | 4,403 | 3,319 | ||||||
Total current assets | 325,771 | 392,059 | ||||||
Property and equipment, net | 24,851 | 22,299 | ||||||
Goodwill | 30,815 | 30,815 | ||||||
Restricted cash | 1,343 | 1,343 | ||||||
Deposits | 455 | 455 | ||||||
Operating lease right-of-use assets | 1,207 | 1,569 | ||||||
Finance lease right-of-use asset | 47,410 | 48,480 | ||||||
Total assets | $ | 431,852 | $ | 497,020 | ||||
Liabilities and stockholders’ equity | ||||||||
Current liabilities: | ||||||||
Accounts payable and accrued expenses | $ | 21,840 | $ | 19,615 | ||||
Operating lease liabilities, current | 737 | 863 | ||||||
Finance lease liability, current | 1,712 | 1,689 | ||||||
Total current liabilities | 24,289 | 22,167 | ||||||
Operating lease liabilities, non-current | 606 | 905 | ||||||
Finance lease liability, non-current | 19,210 | 19,144 | ||||||
Other liabilities | 51 | 80 | ||||||
Total liabilities | 44,156 | 42,296 | ||||||
Commitments and contingencies (Note 10) | ||||||||
Stockholders’ equity: | ||||||||
Preferred stock, $0.01 par value, authorized 5,000,000 shares: | ||||||||
Series A convertible preferred stock; 300,000 shares designated as Series A; 0 shares issued and outstanding | 0 | 0 | ||||||
Series B convertible preferred stock; 300,000 shares designated as Series B; 0 shares issued and outstanding | 0 | 0 | ||||||
Common stock, $0.01 par value, 120,000,000 shares authorized; 65,837,894 and 64,505,889 shares issued and outstanding at June 30, 2022 and December 31, 2021, respectively | 658 | 645 | ||||||
Additional paid-in capital | 977,099 | 946,152 | ||||||
Accumulated other comprehensive loss | (765 | ) | (161 | ) | ||||
Accumulated deficit | (589,296 | ) | (491,912 | ) | ||||
Total stockholders’ equity | 387,696 | 454,724 | ||||||
Total liabilities and stockholders’ equity | $ | 431,852 | $ | 497,020 |
Three Months Ended September 30, | Nine Months Ended September 30, | Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||||||||||||||||
2021 | 2020 | 2021 | 2020 | 2022 | 2021 | 2022 | 2021 | |||||||||||||||||||||||||
Revenue | $ | 0 | $ | 0 | $ | 0 | $ | 0 | $ | 0 | $ | 0 | $ | 0 | $ | 0 | ||||||||||||||||
Operating expenses: | ||||||||||||||||||||||||||||||||
Research and development | 39,975 | 21,657 | 93,315 | 55,345 | 41,356 | 24,530 | 72,150 | 52,839 | ||||||||||||||||||||||||
General and administrative | 9,671 | 5,730 | 29,600 | 19,720 | 12,854 | 9,518 | 24,624 | 20,431 | ||||||||||||||||||||||||
Total operating expenses | 49,646 | 27,387 | 122,915 | 75,065 | 54,210 | 34,048 | 96,774 | 73,270 | ||||||||||||||||||||||||
Loss from operations | (49,646 | ) | (27,387 | ) | (122,915 | ) | (75,065 | ) | (54,210 | ) | (34,048 | ) | (96,774 | ) | (73,270 | ) | ||||||||||||||||
Research and development incentives | 0 | 0 | 500 | 0 | 0 | 0 | 0 | 500 | ||||||||||||||||||||||||
Interest expense | (534 | ) | (1,967 | ) | (2,514 | ) | (5,326 | ) | (465 | ) | (251 | ) | (928 | ) | (1,980 | ) | ||||||||||||||||
Interest and other income - net | 806 | 518 | 2,218 | 1,913 | ||||||||||||||||||||||||||||
Interest and other income, net | 669 | 501 | 1,291 | 1,412 | ||||||||||||||||||||||||||||
Amortization of premium on investments - net | (744 | ) | (244 | ) | (2,111 | ) | (306 | ) | (396 | ) | (727 | ) | (973 | ) | (1,366 | ) | ||||||||||||||||
Net loss | $ | (50,118 | ) | $ | (29,080 | ) | $ | (124,822 | ) | $ | (78,784 | ) | $ | (54,402 | ) | $ | (34,525 | ) | $ | (97,384 | ) | $ | (74,704 | ) | ||||||||
Net loss per share attributable to common stockholders - basic and diluted | $ | (0.79 | ) | $ | (0.53 | ) | $ | (1.99 | ) | $ | (1.43 | ) | $ | (0.83 | ) | $ | (0.55 | ) | $ | (1.50 | ) | $ | (1.20 | ) | ||||||||
Weighted-average common shares outstanding - basic and diluted | 63,825,429 | 55,188,956 | 62,828,601 | 55,077,254 | 65,476,531 | 63,061,232 | 64,995,797 | 62,321,926 |
Three Months Ended September 30, | Nine Months Ended September 30, | Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||||||||||||||||
2021 | 2020 | 2021 | 2020 | 2022 | 2021 | 2022 | 2021 | |||||||||||||||||||||||||
Net loss | $ | (50,118 | ) | $ | (29,080 | ) | $ | (124,822 | ) | $ | (78,784 | ) | $ | (54,402 | ) | $ | (34,525 | ) | $ | (97,384 | ) | $ | (74,704 | ) | ||||||||
Other comprehensive loss | ||||||||||||||||||||||||||||||||
Net unrealized (loss) gain on investments | (16 | ) | (160 | ) | (55 | ) | 49 | |||||||||||||||||||||||||
Net unrealized loss on investments | (136 | ) | (6 | ) | (604 | ) | (39 | ) | ||||||||||||||||||||||||
Total comprehensive loss | $ | (50,134 | ) | $ | (29,240 | ) | $ | (124,877 | ) | $ | (78,735 | ) | $ | (54,538 | ) | $ | (34,531 | ) | $ | (97,988 | ) | $ | (74,743 | ) |
Accumulated | Common Stock | Additional Paid-In | Accumulated Other Comprehensive | Accumulated | Total Stockholders' | |||||||||||||||||||||||||||||||||||||||||||||||
Additional | Other | Total | Shares | Amount | Capital | Income/(Loss) | Deficit | Equity | ||||||||||||||||||||||||||||||||||||||||||||
Common Stock | Treasury | Paid-In | Comprehensive | Accumulated | Stockholders' | |||||||||||||||||||||||||||||||||||||||||||||||
Shares | Amount | Stock | Capital | Income/(Loss) | Deficit | Equity | ||||||||||||||||||||||||||||||||||||||||||||||
Balance at December 31, 2020 | 60,996,367 | $ | 610 | $ | 0 | $ | 825,794 | $ | (42 | ) | $ | (322,843 | ) | $ | 503,519 | |||||||||||||||||||||||||||||||||||||
Issuance of common stock pursuant to exercise of stock options | 991,432 | 9 | 0 | 8,783 | 0 | 0 | 8,792 | |||||||||||||||||||||||||||||||||||||||||||||
Balance at December 31, 2021 | 64,505,889 | $ | 645 | $ | 946,152 | $ | (161 | ) | $ | (491,912 | ) | $ | 454,724 | |||||||||||||||||||||||||||||||||||||||
Issuance of common stock pursuant to exercise of stock options and restricted stock units | 16,168 | 0 | 76 | 0 | 0 | 76 | ||||||||||||||||||||||||||||||||||||||||||||||
Unrealized comprehensive loss on investments | - | 0 | 0 | 0 | (33 | ) | 0 | (33 | ) | - | 0 | 0 | (468 | ) | 0 | (468 | ) | |||||||||||||||||||||||||||||||||||
Share-based compensation | - | 0 | 0 | 7,900 | 0 | 0 | 7,900 | - | 0 | 6,270 | 0 | 0 | 6,270 | |||||||||||||||||||||||||||||||||||||||
Net loss | - | 0 | 0 | 0 | 0 | (40,179 | ) | (40,179 | ) | - | 0 | 0 | 0 | (42,982 | ) | (42,982 | ) | |||||||||||||||||||||||||||||||||||
Balance at March 31, 2021 | 61,987,799 | 619 | 0 | 842,477 | (75 | ) | (363,022 | ) | 479,999 | |||||||||||||||||||||||||||||||||||||||||||
Balance at March 31, 2022 | 64,522,057 | 645 | 952,498 | (629 | ) | (534,894 | ) | 417,620 | ||||||||||||||||||||||||||||||||||||||||||||
Issuance of common stock pursuant to exercise of stock options | 133,838 | 2 | 0 | 1,113 | 0 | 0 | 1,115 | 2,387 | 0 | 3 | 0 | 0 | 3 | |||||||||||||||||||||||||||||||||||||||
Issuance of common stock pursuant to conversion of notes | 1,326,432 | 13 | 0 | 35,530 | 0 | 0 | 35,543 | |||||||||||||||||||||||||||||||||||||||||||||
Unrealized comprehensive loss on investments | - | 0 | 0 | 0 | (6 | ) | 0 | (6 | ) | |||||||||||||||||||||||||||||||||||||||||||
Issuance of common stock pursuant to the at-the-market offering program, net of issuance costs | 1,313,450 | 13 | 17,229 | 0 | 0 | 17,242 | ||||||||||||||||||||||||||||||||||||||||||||||
Unrealized comprehensive loss on marketable securities | - | 0 | 0 | (136 | ) | 0 | (136 | ) | ||||||||||||||||||||||||||||||||||||||||||||
Share-based compensation | - | 0 | 0 | 7,311 | 0 | 0 | 7,311 | - | 0 | 7,369 | 0 | 0 | 7,369 | |||||||||||||||||||||||||||||||||||||||
Net loss | - | 0 | 0 | 0 | 0 | (34,525 | ) | (34,525 | ) | - | 0 | 0 | 0 | (54,402 | ) | (54,402 | ) | |||||||||||||||||||||||||||||||||||
Balance at June 30, 2021 | 63,448,069 | 634 | 0 | 886,431 | (81 | ) | (397,547 | ) | 489,437 | |||||||||||||||||||||||||||||||||||||||||||
Issuance of common stock pursuant to exercise of stock options | 21,402 | 0 | 0 | 284 | 0 | 0 | 284 | |||||||||||||||||||||||||||||||||||||||||||||
Issuance of common stock pursuant to conversion of notes | 160,614 | 2 | 0 | 5,148 | 0 | 0 | 5,150 | |||||||||||||||||||||||||||||||||||||||||||||
Issuance of common stock, net of issuance costs | 812,516 | 8 | 0 | 26,346 | 0 | 0 | 26,354 | |||||||||||||||||||||||||||||||||||||||||||||
Warrant issuance | - | 0 | 0 | 7,578 | 0 | 0 | 7,578 | |||||||||||||||||||||||||||||||||||||||||||||
Unrealized comprehensive loss on investments | - | 0 | 0 | 0 | (16 | ) | 0 | (16 | ) | |||||||||||||||||||||||||||||||||||||||||||
Share-based compensation | - | 0 | 0 | 6,989 | 0 | 0 | 6,989 | |||||||||||||||||||||||||||||||||||||||||||||
Net loss | - | 0 | 0 | 0 | 0 | (50,118 | ) | (50,118 | ) | |||||||||||||||||||||||||||||||||||||||||||
Balance at September 30, 2021 | 64,442,601 | $ | 644 | $ | 0 | $ | 932,776 | $ | (97 | ) | $ | (447,665 | ) | $ | 485,658 | |||||||||||||||||||||||||||||||||||||
Balance at June 30, 2022 | 65,837,894 | $ | 658 | $ | 977,099 | $ | (765 | ) | $ | (589,296 | ) | $ | 387,696 |
Balance at December 31, 2019 | 54,773,061 | $ | 548 | $ | (53 | ) | $ | 489,925 | $ | 20 | $ | (183,143 | ) | $ | 307,297 | |||||||||||||||||||||||||||||||||||||
Common Stock | Additional Paid-In | Accumulated Other Comprehensive | Accumulated | Total Stockholders' | ||||||||||||||||||||||||||||||||||||||||||||||||
Shares | Amount | Capital | Loss | Deficit | Equity | |||||||||||||||||||||||||||||||||||||||||||||||
Balance at December 31, 2020 | 60,996,367 | $ | 610 | $ | 825,794 | $ | (42 | ) | $ | (322,843 | ) | $ | 503,519 | |||||||||||||||||||||||||||||||||||||||
Issuance of common stock pursuant to exercise of stock options | 386,974 | 4 | 0 | (4 | ) | 0 | 0 | 0 | 991,432 | 9 | 8,783 | 0 | 0 | 8,792 | ||||||||||||||||||||||||||||||||||||||
Share repurchase | (3,000 | ) | 0 | 0 | (72 | ) | 0 | 0 | (72 | ) | ||||||||||||||||||||||||||||||||||||||||||
Sale of treasury stock | 0 | 0 | 53 | 0 | 0 | 0 | 53 | |||||||||||||||||||||||||||||||||||||||||||||
Issuance of treasury stock pursuant to exercise of stock options | 0 | 0 | (429 | ) | 0 | 0 | 0 | (429 | ) | |||||||||||||||||||||||||||||||||||||||||||
Unrealized comprehensive loss on investments | - | 0 | 0 | 0 | (95 | ) | 0 | (95 | ) | - | 0 | 0 | (33 | ) | 0 | (33 | ) | |||||||||||||||||||||||||||||||||||
Share-based compensation | - | 0 | 0 | 3,961 | 0 | 0 | 3,961 | - | 0 | 7,900 | 0 | 0 | 7,900 | |||||||||||||||||||||||||||||||||||||||
Net loss | - | 0 | 0 | 0 | 0 | (24,664 | ) | (24,664 | ) | - | 0 | 0 | 0 | (40,179 | ) | (40,179 | ) | |||||||||||||||||||||||||||||||||||
Balance at March 31, 2020 | 55,157,035 | 552 | (429 | ) | 493,810 | (75 | ) | (207,807 | ) | 286,051 | ||||||||||||||||||||||||||||||||||||||||||
Balance at March 31, 2021 | 61,987,799 | 619 | 842,477 | (75 | ) | (363,022 | ) | 479,999 | ||||||||||||||||||||||||||||||||||||||||||||
Issuance of common stock pursuant to exercise of stock options | 12,968 | 0 | 0 | 290 | 0 | 0 | 290 | 133,838 | 2 | 1,113 | 0 | 0 | 1,115 | |||||||||||||||||||||||||||||||||||||||
Sale of treasury stock | 0 | 0 | 538 | 0 | 0 | 0 | 538 | |||||||||||||||||||||||||||||||||||||||||||||
Issuance of treasury stock pursuant to exercise of stock options | 0 | 0 | (109 | ) | 0 | 0 | 0 | (109 | ) | |||||||||||||||||||||||||||||||||||||||||||
Unrealized comprehensive gain on investments | - | 0 | 0 | 0 | 304 | 0 | 304 | |||||||||||||||||||||||||||||||||||||||||||||
Share-based compensation | - | 0 | 0 | 4,489 | 0 | 0 | 4,489 | |||||||||||||||||||||||||||||||||||||||||||||
Net loss | - | 0 | 0 | 0 | 0 | (25,040 | ) | (25,040 | ) | |||||||||||||||||||||||||||||||||||||||||||
Balance at June 30, 2020 | 55,170,003 | 552 | 0 | 498,589 | 229 | (232,847 | ) | 266,523 | ||||||||||||||||||||||||||||||||||||||||||||
Issuance of common stock pursuant to exercise of stock options | 34,124 | 0 | 0 | 587 | 0 | 0 | 587 | |||||||||||||||||||||||||||||||||||||||||||||
Issuance of treasury stock pursuant to exercise of stock options | 0 | 0 | (76 | ) | 0 | 0 | 0 | (76 | ) | |||||||||||||||||||||||||||||||||||||||||||
Issuance of common stock pursuant to conversion of notes | 1,326,432 | 13 | 35,530 | 0 | 0 | 35,543 | ||||||||||||||||||||||||||||||||||||||||||||||
Unrealized comprehensive loss on investments | - | 0 | 0 | 0 | (160 | ) | 0 | (160 | ) | - | 0 | 0 | (6 | ) | 0 | (6 | ) | |||||||||||||||||||||||||||||||||||
Share-based compensation | - | 0 | 0 | 4,047 | 0 | 0 | 4,047 | - | 0 | 7,311 | 0 | 0 | 7,311 | |||||||||||||||||||||||||||||||||||||||
Net loss | - | 0 | 0 | 0 | 0 | (29,080 | ) | (29,080 | ) | - | 0 | 0 | 0 | (34,525 | ) | (34,525 | ) | |||||||||||||||||||||||||||||||||||
Balance at September 30, 2020 | 55,204,127 | $ | 552 | $ | (76 | ) | $ | 503,223 | $ | 69 | $ | (261,927 | ) | $ | 241,841 | |||||||||||||||||||||||||||||||||||||
Balance at June 30, 2021 | 63,448,069 | $ | 634 | $ | 886,431 | $ | (81 | ) | $ | (397,547 | ) | $ | 489,437 |
Nine Months Ended September 30, | Six Months Ended June 30, | |||||||||||||||
2021 | 2020 | 2022 | 2021 | |||||||||||||
Operating Activities: | ||||||||||||||||
Net loss | $ | (124,822 | ) | $ | (78,784 | ) | $ | (97,384 | ) | $ | (74,704 | ) | ||||
Adjustments to reconcile net loss to net cash used in operating activities: | ||||||||||||||||
Accretion of discount on convertible notes | 752 | 2,085 | 0 | 705 | ||||||||||||
Depreciation and amortization expense | 2,188 | 515 | ||||||||||||||
Write down of property and equipment | 0 | 62 | ||||||||||||||
Depreciation and amortization of property and equipment | 1,798 | 1,380 | ||||||||||||||
Right of use asset | 1,070 | 0 | ||||||||||||||
Write down of property and equipment, net | 177 | 0 | ||||||||||||||
Stock-based compensation | 22,200 | 12,497 | 13,639 | 15,211 | ||||||||||||
Accretion of discount on investments, net | 2,089 | 306 | 977 | 1,345 | ||||||||||||
Expense in connection with warrant issuance | 7,578 | 0 | ||||||||||||||
Unrealized loss on marketable securities | 0 | 1 | ||||||||||||||
Changes in operating assets and liabilities: | ||||||||||||||||
Prepaid expenses and other assets | 1,016 | (1,415 | ) | (1,084 | ) | 1,030 | ||||||||||
Accounts payable and accrued expenses | (2,842 | ) | 1,752 | 2,548 | (8,982 | ) | ||||||||||
Operating lease liabilities | 17 | (114 | ) | (64 | ) | 50 | ||||||||||
Finance lease liability | 1,644 | 1,565 | 90 | 1,184 | ||||||||||||
Other long term liabilities | (42 | ) | 0 | (29 | ) | (29 | ) | |||||||||
Net cash used in operating activities | (90,222 | ) | (61,531 | ) | (78,262 | ) | (62,809 | ) | ||||||||
Investing activities: | ||||||||||||||||
Purchases of investments | (226,484 | ) | (132,101 | ) | (143,023 | ) | (158,571 | ) | ||||||||
Proceeds from maturities of investments | 234,146 | 104,111 | 163,669 | 180,584 | ||||||||||||
Payments made to acquire right of use asset | (18 | ) | (6,539 | ) | 0 | (61 | ) | |||||||||
Purchases of property and equipment | (4,907 | ) | (6,954 | ) | (4,852 | ) | (1,774 | ) | ||||||||
Purchases of internal use software | (748 | ) | (646 | ) | ||||||||||||
Net cash provided by (used in) investing activities | 1,989 | (42,129 | ) | |||||||||||||
Net cash provided by investing activities | 15,794 | 20,178 | ||||||||||||||
Financing activities: | ||||||||||||||||
Issuance of common stock, pursuant to exercise of stock options | 10,191 | 874 | 79 | 9,907 | ||||||||||||
Issuance of common stock, net of issuance costs | 26,354 | 0 | ||||||||||||||
Common stock repurchase | 0 | (72 | ) | |||||||||||||
Proceeds from sale of treasury stock | 0 | 591 | ||||||||||||||
Proceeds from exercise of options | 0 | 4 | ||||||||||||||
Payment of withholding tax on option exercises | 0 | (614 | ) | |||||||||||||
Convertible notes refinancing costs to the lender | 0 | (237 | ) | |||||||||||||
Issuance of common stock pursuant to the at-the-market offering program, net of issuance costs | 17,243 | 0 | ||||||||||||||
Net cash provided by financing activities | 36,545 | 546 | 17,322 | 9,907 | ||||||||||||
Net change in cash, cash equivalents and restricted cash | (51,688 | ) | (103,114 | ) | (45,146 | ) | (32,724 | ) | ||||||||
Cash, cash equivalents and restricted cash at beginning of period | 298,666 | 186,908 | 234,037 | 298,666 | ||||||||||||
Cash, cash equivalents and restricted cash at end of period | $ | 246,978 | $ | 83,794 | $ | 188,891 | $ | 265,942 | ||||||||
Supplemental disclosure of non-cash financing and investing activities: | ||||||||||||||||
Accrued purchases of property and equipment | $ | 1,106 | $ | 5,627 | $ | 405 | $ | 1,394 | ||||||||
Accrued purchases of internal use software | $ | 26 | $ | 100 | ||||||||||||
Unrealized (loss) gain on investments | $ | (55 | ) | $ | 49 | |||||||||||
Conversion of 2021 and 2022 convertible notes into common stock | $ | (40,693 | ) | $ | 0 | |||||||||||
Finance lease right of use asset and lease liability | $ | 0 | $ | 20,179 | ||||||||||||
Reclassification of construction in process to finance right of use asset | $ | 39 | $ | 26,465 | ||||||||||||
Unrealized loss on investments | $ | (604 | ) | $ | (39 | ) | ||||||||||
Conversion of convertible notes into common stock | $ | 0 | $ | 35,544 | ||||||||||||
Reclassification of construction in process from finance right of use asset | $ | 0 | $ | (5 | ) | |||||||||||
Supplemental cash flow information: | ||||||||||||||||
Cash paid for interest | $ | 148 | $ | 2,960 | $ | 0 | $ | 1,347 |
1. | Nature of Business |
2. | Risks and Liquidity |
3. | Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies |
September 30, 2021 | December 31, 2020 | June 30, 2022 | December 31, 2021 | |||||||||||||
Cash and cash equivalents | $ | 245,644 | $ | 297,098 | $ | 187,548 | $ | 232,694 | ||||||||
Restricted cash | 1,334 | 1,568 | 1,343 | 1,343 | ||||||||||||
$ | 246,978 | $ | 298,666 | $ | 188,891 | $ | 234,037 |
4. | Fair Value of Financial Instruments |
Fair Value Measurements as of September 30, 2021 Using: | Fair Value Measurements as of June 30, 2022 Using: | |||||||||||||||||||||||||||||||
Level 1 | Level 2 | Level 3 | Total | Level 1 | Level 2 | Level 3 | Total | |||||||||||||||||||||||||
Assets: | ||||||||||||||||||||||||||||||||
Cash and cash equivalents: | ||||||||||||||||||||||||||||||||
Cash equivalents: | ||||||||||||||||||||||||||||||||
Money market mutual funds | $ | 180,084 | $ | 0 | $ | 0 | $ | 180,084 | $ | 142,290 | $ | 0 | $ | 0 | $ | 142,290 | ||||||||||||||||
180,084 | 0 | 0 | 180,084 | 142,290 | 0 | 0 | 142,290 | |||||||||||||||||||||||||
Investments: | ||||||||||||||||||||||||||||||||
United States Treasury securities | 67,686 | 0 | 0 | 67,686 | 80,793 | 0 | 0 | 80,793 | ||||||||||||||||||||||||
Corporate Bonds | 0 | 92,817 | 0 | 92,817 | 0 | 53,027 | 0 | 53,027 | ||||||||||||||||||||||||
Municipal Bonds | 0 | 6,000 | 0 | 6,000 | ||||||||||||||||||||||||||||
Agency Bonds | 0 | 9,312 | 0 | 9,312 | ||||||||||||||||||||||||||||
67,686 | 108,129 | 0 | 175,815 | 80,793 | 53,027 | 0 | 133,820 | |||||||||||||||||||||||||
$ | 247,770 | $ | 108,129 | $ | 0 | $ | 355,899 | $ | 223,083 | $ | 53,027 | $ | 0 | $ | 276,110 |
Fair Value Measurements as of December 31, 2020 Using: | Fair Value Measurements as of December 31, 2021 Using: | |||||||||||||||||||||||||||||||
Level 1 | Level 2 | Level 3 | Total | Level 1 | Level 2 | Level 3 | Total | |||||||||||||||||||||||||
Assets: | ||||||||||||||||||||||||||||||||
Cash and cash equivalents: | ||||||||||||||||||||||||||||||||
Cash equivalents: | ||||||||||||||||||||||||||||||||
Money market mutual funds | $ | 193,312 | $ | 0 | $ | 0 | $ | 193,312 | $ | 179,900 | $ | 0 | $ | 0 | $ | 179,900 | ||||||||||||||||
United States Treasury securities | 62,497 | 0 | 0 | 62,497 | ||||||||||||||||||||||||||||
Corporate Bonds | 0 | 501 | 0 | 501 | ||||||||||||||||||||||||||||
Agency Bonds | 0 | 8,015 | 0 | 8,015 | ||||||||||||||||||||||||||||
255,809 | 8,516 | 0 | 264,325 | 179,900 | 0 | 0 | 179,900 | |||||||||||||||||||||||||
Investments: | ||||||||||||||||||||||||||||||||
United States Treasury securities | 112,328 | 0 | 0 | 112,328 | 44,045 | 0 | 0 | 44,045 | ||||||||||||||||||||||||
Corporate Bonds | 0 | 63,710 | 0 | 63,710 | 0 | 96,696 | 0 | 96,696 | ||||||||||||||||||||||||
Municipal Bonds | 0 | 6,000 | 0 | 6,000 | 0 | 6,000 | 0 | 6,000 | ||||||||||||||||||||||||
Agency Bonds | 0 | 3,583 | 0 | 3,583 | 0 | 9,305 | 0 | 9,305 | ||||||||||||||||||||||||
112,328 | 73,293 | 0 | 185,621 | 44,045 | 112,001 | 0 | 156,046 | |||||||||||||||||||||||||
$ | 368,137 | $ | 81,809 | $ | 0 | $ | 449,946 | $ | 223,945 | $ | 112,001 | $ | 0 | $ | 335,946 |
5. | Property and Equipment, Net |
September 30, 2021 | December 31, 2020 | |||||||
Laboratory equipment | $ | 12,120 | $ | 7,807 | ||||
Machinery and equipment | 10,065 | 9,933 | ||||||
Computer equipment | 218 | 218 | ||||||
Furniture and fixtures | 1,955 | 1,880 | ||||||
Leasehold improvements | 106 | 29 | ||||||
Internal use software | 1,860 | 1,385 | ||||||
26,324 | 21,252 | |||||||
Less: accumulated depreciation and amortization | (4,234 | ) | (2,046 | ) | ||||
$ | 22,090 | $ | 19,206 |
June 30, 2022 | December 31, 2021 | |||||||
Laboratory equipment | $ | 16,349 | $ | 12,600 | ||||
Machinery and equipment | 10,692 | 10,432 | ||||||
Computer equipment | 244 | 218 | ||||||
Furniture and fixtures | 2,123 | 1,963 | ||||||
Leasehold improvements | 458 | 407 | ||||||
Internal use software | 1,936 | 1,902 | ||||||
31,802 | 27,522 | |||||||
Less: accumulated depreciation and amortization | (6,951 | ) | (5,223 | ) | ||||
$ | 24,851 | $ | 22,299 |
6. | Accounts Payable and Accrued Expenses |
September 30, | December 31, | |||||||||||||||
2021 | 2020 | June 30, 2022 | December 31, 2021 | |||||||||||||
Research and development | $ | 14,617 | $ | 14,962 | $ | 15,926 | $ | 12,082 | ||||||||
Property and equipment | 1,106 | 1,456 | 405 | 725 | ||||||||||||
Employee compensation | 3,815 | 4,875 | 3,007 | 4,533 | ||||||||||||
Accrued interest | 0 | 1,122 | ||||||||||||||
Government grant payable | 597 | 590 | 597 | 597 | ||||||||||||
Professional fees | 1,196 | 1,332 | 1,260 | 1,196 | ||||||||||||
Internal use software | 26 | 300 | ||||||||||||||
Other | 651 | 835 | 645 | 482 | ||||||||||||
$ | 22,008 | $ | 25,472 | $ | 21,840 | $ | 19,615 |
7. |
September 30, 2021 | December 31, 2020 | |||||||
Principal amount | $ | 0 | $ | 5,150 | ||||
Discount | 0 | (275 | ) | |||||
Carrying value | $ | 0 | $ | 4,875 |
8. | Stock Based Compensation |
Nine Months Ended September 30, | Six Months Ended June 30, | |||||||||||||||
2021 | 2020 | 2022 | 2021 | |||||||||||||
Risk-free interest rate | 0.78 | % | 1.07 | % | 2.32 | % | 0.74 | % | ||||||||
Expected term (in years) | 5.84 | 5.83 | 5.79 | 5.84 | ||||||||||||
Expected volatility | 69.31 | % | 76.74 | % | 73.26 | % | 69.19 | % | ||||||||
Expected dividend yield | 0.00 | % | 0.00 | % | 0.00 | % | 0.00 | % | ||||||||
Exercise price | $ | 53.98 | $ | 21.97 | $ | 15.93 | $ | 57.18 | ||||||||
Fair value of common stock | $ | 53.98 | $ | 21.97 | $ | 15.93 | $ | 57.18 |
Number of Shares | Weighted Average Exercise Price | Weighted Average Contractual Term (Years) | Aggregate Intrinsic Value | |||||||||||||
Outstanding as of December 31, 2020 | 11,050,931 | $ | 9.10 | 6.55 | $ | 504,079 | ||||||||||
Granted | 1,506,009 | 53.98 | 8.96 | |||||||||||||
Exercised | (1,146,672 | ) | 8.84 | 53,871 | ||||||||||||
Cancelled | (302,026 | ) | 33.88 | |||||||||||||
Outstanding as of September 30, 2021 | 11,108,242 | $ | 14.55 | 6.15 | $ | 203,781 | ||||||||||
Options vested and exercisable as of September 30, 2021 | 8,468,030 | $ | 6.88 | 5.25 | $ | 194,593,951 | ||||||||||
Options unvested as of September 30, 2021 | 2,640,212 | $ | 39.11 | 9.04 |
Number of Shares | Weighted Average Exercise Price | Weighted Average Contractual Term (Years) | Aggregate Intrinsic Value | |||||||||||||
Outstanding as of December 31, 2021 | 11,143,761 | $ | 14.51 | 5.95 | $ | 128,817 | ||||||||||
Granted | 1,896,748 | 15.93 | 7.13 | |||||||||||||
Exercised | (8,387 | ) | 9.39 | $ | 49 | |||||||||||
Forfeited | (505,371 | ) | 33.65 | |||||||||||||
Outstanding as of June 30, 2022 | 12,526,751 | $ | 13.96 | 5.85 | $ | 72,718 | ||||||||||
Options vested and exercisable as of June 30, 2022 | 9,468,501 | $ | 10.21 | 4.76 | $ | 71,260 | ||||||||||
Options unvested as of June 30, 2022 | 3,059,712 | $ | 25.53 | 9.21 |
Number of Shares | Weighted Average Grant Date Fair Value | |||||||
Unvested as of December 31, 2021 | 23,500 | $ | 30.61 | |||||
Granted | 552,483 | $ | 16.35 | |||||
Exercised | (10,168 | ) | $ | 62.32 | ||||
Forfeited | (38,511 | ) | $ | 16.67 | ||||
Unvested as of June 30, 2022 | 527,304 | $ | 16.55 |
Three Months Ended September 30, | Nine Months Ended September 30, | Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||||||||||||||||
2021 | 2020 | 2021 | 2020 | 2022 | 2021 | 2022 | 2021 | |||||||||||||||||||||||||
Stock options | $ | 6,831 | $ | 4,047 | $ | 21,872 | $ | 12,497 | $ | 6,677 | $ | 7,215 | $ | 12,638 | $ | 15,041 | ||||||||||||||||
Restricted stock units | 158 | 0 | 328 | 0 | 692 | 96 | 1,001 | 170 | ||||||||||||||||||||||||
Total share based compensation expense | $ | 6,989 | $ | 4,047 | $ | 22,200 | $ | 12,497 | ||||||||||||||||||||||||
Total stock-based compensation expense | $ | 7,369 | $ | 7,311 | $ | 13,639 | $ | 15,211 |
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
2021 | 2020 | 2021 | 2020 | |||||||||||||
Research and development | $ | 3,084 | $ | 1,780 | $ | 9,148 | $ | 5,194 | ||||||||
General and administrative | 3,905 | 2,267 | 13,052 | 7,303 | ||||||||||||
Total share based compensation expense | $ | 6,989 | $ | 4,047 | $ | 22,200 | $ | 12,497 |
Number of Shares | Weighted Average Grant Date Fair Value | |||||||
Unvested as of December 31, 2020 | 20,000 | $ | 25.06 | |||||
Granted | 3,500 | 62.32 | ||||||
Unvested as of September 30, 2021 | 23,500 | 30.61 |
Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
2022 | 2021 | 2022 | 2021 | |||||||||||||
Research and development | $ | 2,889 | $ | 3,148 | $ | 5,207 | $ | 6,064 | ||||||||
General and administrative | 4,480 | 4,163 | 8,432 | 9,147 | ||||||||||||
Total stock-based compensation expense | $ | 7,369 | $ | 7,311 | $ | 13,639 | $ | 15,211 |
Exercise Price | Outstanding | Grant Date | Expiration Date | ||||||
24.42 | 7,051 | June 28, 2013 | June 28, 2023 | ||||||
57.11 | 603,386 | December 21, 2020 | December 21, 2030 | ||||||
33.63 | 301,291 | August 9, 2021 | August 9, 2031 | ||||||
22.51 | 153,155 | December 17, 2021 | December 17, 2031 | ||||||
22.51 | 153,155 | December 17, 2021 | December 17, 2031 | ||||||
Total | 1,218,038 |
Number of Warrant Shares Outstanding and Exercisable | Exercise Price per Share | |||||||
Balance as of December 31, 2021 | 1,218,038 | |||||||
Granted | 0 | $ | 0 | |||||
Exercised | 0 | $ | - | |||||
Balance as of June 30, 2022 | 1,218,038 |
9. |
Net Loss Per Share |
Three Months Ended September 30, | Nine Months Ended September 30, | Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||||||||||||||||
2021 | 2020 | 2021 | 2020 | 2022 | 2021 | 2022 | 2021 | |||||||||||||||||||||||||
Numerator: | ||||||||||||||||||||||||||||||||
Net loss attributable to common stockholders | $ | (50,118 | ) | $ | (29,080 | ) | $ | (124,822 | ) | $ | (78,784 | ) | $ | (54,402 | ) | $ | (34,525 | ) | $ | (97,384 | ) | $ | (74,704 | ) | ||||||||
Denominator: | ||||||||||||||||||||||||||||||||
Weighted-average common shares outstanding - basic and diluted | 63,825,429 | 55,188,956 | 62,828,601 | 55,077,254 | 65,476,531 | 63,061,232 | 64,995,797 | 62,321,926 | ||||||||||||||||||||||||
Net loss per share attributable to common stockholders - basic and diluted | $ | (0.79 | ) | $ | (0.53 | ) | $ | (1.99 | ) | $ | (1.43 | ) | $ | (0.83 | ) | $ | (0.55 | ) | $ | (1.50 | ) | $ | (1.20 | ) |
Three and Nine Months Ended September 30, | Three and Six Months Ended June 30, | |||||||||||||||
2021 | 2020 | 2022 | 2021 | |||||||||||||
Shares issuable upon conversion of the 2021 Convertible Notes | 0 | 160,536 | 0 | 160,536 | ||||||||||||
Shares issuable upon conversion of the 2022 Convertible Notes | 0 | 1,460,412 | ||||||||||||||
Warrants exercisable for common shares | 911,728 | 14,102 | 1,218,038 | 610,437 | ||||||||||||
Restricted stock units exercisable for common shares | 23,500 | 0 | 527,304 | 23,500 | ||||||||||||
Options to purchase common shares | 11,108,242 | 10,922,678 | 12,526,751 | 11,040,697 | ||||||||||||
12,043,470 | 12,557,728 | 14,272,093 | 11,835,170 |
Commitments and Contingencies |
Lease cost | September 30, 2021 | June 30, 2022 | ||||||
Operating lease cost | $ | 457 | $ | 394 | ||||
Finance lease cost | ||||||||
Amortization of right of use assets | 1,605 | 1,070 | ||||||
Interest on lease liabilities | 1,382 | |||||||
Interest on lease liablities | 929 | |||||||
Total lease cost | $ | 3,444 | $ | 2,393 |
Maturity of operating lease liabilities | September 30, 2021 | June 30, 2022 | ||||||
2021 | $ | 215 | ||||||
2022 | 860 | 459 | ||||||
2023 | 488 | 548 | ||||||
2024 | 207 | 269 | ||||||
2025 | 64 | |||||||
2026 | 55 | |||||||
Total lease payments | $ | 1,770 | $ | 1,395 | ||||
Less: interest | (75 | ) | (52 | ) | ||||
Total operating lease liabilities | $ | 1,695 | $ | 1,343 |
Maturity of finance lease liability | September 30, 2021 | June 30, 2022 | ||||||
2021 | $ | 416 | ||||||
2022 | 1,689 | 850 | ||||||
2023 | 1,736 | 1,736 | ||||||
2024 | 1,791 | 1,791 | ||||||
2025 | 1,856 | 1,856 | ||||||
2026 | 1,912 | |||||||
Thereafter | 46,912 | 45,001 | ||||||
Total lease payments | $ | 54,400 | $ | 53,146 | ||||
Less: interest | (33,614 | ) | (32,224 | ) | ||||
Total finance lease liability | $ | 20,786 | $ | 20,922 |
Leases | September 30, 2021 | June 30, 2022 | ||||||
Operating right-of-use assets | $ | 1,467 | $ | 1,207 | ||||
Operating current lease liabilities | 804 | 737 | ||||||
Operating noncurrent lease liabilities | 891 | 606 | ||||||
Total operating lease liabilities | $ | 1,695 | $ | 1,343 | ||||
Finance right-of-use assets | $ | 49,011 | $ | 47,410 | ||||
Finance current lease liability | 1,677 | 1,712 | ||||||
Finance noncurrent lease liability | 19,109 | 19,210 | ||||||
Total finance lease liability | $ | 20,786 | $ | 20,922 |
Other information | ||||||||
Cash paid for amounts included in the measurement of lease liabilities: | ||||||||
Operating cash flows from operating leases | $ | 439 | $ | 458 | ||||
Cash flows from finance lease | $ | 1,227 | $ | 839 | ||||
Weighted-average remaining lease term - operating leases | 2.3 years | 2.1 years | ||||||
Weighted-average remaining lease term - finance lease | 22.9 years | 22.2 years | ||||||
Weighted-average discount rate - operating leases | 4.67 | % | 4.17 | % | ||||
Weighted-average discount rate - finance lease | 8.96 | % | 8.96 | % |
Agreements Related to Intellectual Property |
CIRM Grants |
Related Party Transactions |
Nine Months Ended September 30, 2021 | Year Ended December 31, 2020 | |||||||
Risk-free interest rate | 1.33 | % | 0.95 | % | ||||
Expected term (in years) | 10.00 | 10.00 | ||||||
Expected volatility | 69.40 | % | 74.20 | % | ||||
Expected dividend yield | 0.00 | % | 0.00 | % | ||||
Exercise price | $ | 33.63 | $ | 57.11 | ||||
Fair value of common stock | $ | 33.63 | $ | 57.11 |
401(k) Savings Plan |
Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
2022 | 2021 | 2022 | 2021 | |||||||||||||
Direct Expenses: | ||||||||||||||||
Danon Disease (AAV) RP-A501 | $ | 9,568 | $ | 4,507 | $ | 15,942 | $ | 8,307 | ||||||||
Leukocyte Adhesion Deficiency (LVV) RP-L201 | 7,044 | 3,864 | 10,095 | 10,270 | ||||||||||||
Fanconi Anemia (LVV) RP-L102 | 4,557 | 2,013 | 9,087 | 5,608 | ||||||||||||
Pyruvate Kinase Deficiency (LVV) RP-L301 | 632 | 562 | 1,486 | 2,421 | ||||||||||||
Infantile Malignant Osteopetrosis (LVV) RP-L401 (1) | - | 449 | 190 | 1,245 | ||||||||||||
Other product candidates | 3,776 | 1,179 | 7,030 | 1,871 | ||||||||||||
Total direct expenses | 25,577 | 12,574 | 43,830 | 29,722 | ||||||||||||
Unallocated Expenses | ||||||||||||||||
Employee compensation | $ | 6,964 | $ | 5,078 | $ | 12,511 | $ | 9,741 | ||||||||
Stock based compensation expense | 2,889 | 3,148 | 5,207 | 6,064 | ||||||||||||
Depreciation and amortization expense | 1,060 | 1,194 | 1,887 | 2,370 | ||||||||||||
Laboratory and related expenses | 1,291 | 835 | 2,518 | 1,482 | ||||||||||||
Legal and patent fees | - | (16 | ) | - | 41 | |||||||||||
Professional Fees | 629 | 358 | 1,190 | 827 | ||||||||||||
Other expenses | 2,946 | 1,359 | 5,007 | 2,592 | ||||||||||||
Total other research and development expenses | 15,779 | 11,956 | 28,320 | 23,117 | ||||||||||||
Total research and development expense | $ | 41,356 | $ | 24,530 | $ | 72,150 | $ | 52,839 |
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
2021 | 2020 | 2021 | 2020 | |||||||||||||
Direct Expenses: | ||||||||||||||||
Danon Disease (AAV) | $ | 3,497 | $ | 5,131 | $ | 11,804 | $ | 13,575 | ||||||||
Leukocyte Adhesion Deficiency (LVV) | 7,896 | 1,568 | 18,167 | 3,784 | ||||||||||||
Fanconi Anemia (LVV) | 6,379 | 4,661 | 11,986 | 11,318 | ||||||||||||
Pyruvate Kinase Deficiency (LVV) | 1,109 | 1,606 | 3,530 | 3,406 | ||||||||||||
Infantile Malignant Osteopetrosis (LVV) | 729 | 704 | 1,975 | 1,882 | ||||||||||||
Other product candidates | 2,127 | 262 | 3,998 | 349 | ||||||||||||
Total direct expenses | 21,738 | 13,932 | 51,460 | 34,314 | ||||||||||||
Unallocated Expenses | ||||||||||||||||
Employee compensation | 5,524 | 3,380 | 15,265 | 9,662 | ||||||||||||
Share based compensation expense | 3,084 | 1,780 | 9,148 | 4,960 | ||||||||||||
Depreciation and amortization expense | 1,258 | 755 | 3,627 | 1,633 | ||||||||||||
Laboratory and related expenses | 651 | 726 | 2,133 | 1,770 | ||||||||||||
Legal and patent fees | 386 | 328 | 937 | 751 | ||||||||||||
Professional Fees | 400 | 357 | 1,217 | 977 | ||||||||||||
Other expenses | 6,934 | 399 | 9,526 | 1,278 | ||||||||||||
Total other research and development expenses | 18,236 | 7,725 | 41,854 | 21,031 | ||||||||||||
Total research and development expense | $ | 39,975 | $ | 21,657 | $ | 93,315 | $ | 55,345 |
(1) | Effective December 2021, a decision was made to no longer pursue Rocket-sponsored clinical evaluation of RP-L401; this program was returned to academic innovators. |
Three Months Ended June 30, | ||||||||||||||||||||||||
Three Months Ended September 30, | 2022 | 2021 | Change | |||||||||||||||||||||
2021 | 2020 | Change | ||||||||||||||||||||||
Operating expenses: | ||||||||||||||||||||||||
Research and development | $ | 39,975 | $ | 21,657 | $ | 18,318 | $ | 41,356 | $ | 24,530 | $ | 16,826 | ||||||||||||
General and administrative | 9,671 | 5,730 | 3,941 | 12,854 | 9,518 | 3,336 | ||||||||||||||||||
Total operating expenses | 49,646 | 27,387 | 22,259 | 54,210 | 34,048 | 20,162 | ||||||||||||||||||
Loss from operations | (49,646 | ) | (27,387 | ) | (22,259 | ) | (54,210 | ) | (34,048 | ) | (20,162 | ) | ||||||||||||
Research and development incentives | - | - | - | - | - | - | ||||||||||||||||||
Interest expense | (534 | ) | (1,967 | ) | 1,433 | (465 | ) | (251 | ) | (214 | ) | |||||||||||||
Interest and other income net | 806 | 518 | 288 | |||||||||||||||||||||
Interest and other income, net | 669 | 501 | 168 | |||||||||||||||||||||
Amortization of premium on investments - net | (744 | ) | (244 | ) | (500 | ) | (396 | ) | (727 | ) | 331 | |||||||||||||
Total other expense, net | (472 | ) | (1,693 | ) | 1,221 | (192 | ) | (477 | ) | 285 | ||||||||||||||
Net loss | $ | (50,118 | ) | $ | (29,080 | ) | $ | (21,038 | ) | $ | (54,402 | ) | $ | (34,525 | ) | $ | (19,877 | ) |
Six Months Ended June 30, | ||||||||||||
2022 | 2021 | Change | ||||||||||
Operating expenses: | ||||||||||||
Research and development | $ | 72,150 | $ | 52,839 | $ | 19,311 | ||||||
General and administrative | 24,624 | $ | 20,431 | 4,193 | ||||||||
Total operating expenses | 96,774 | 73,270 | 23,504 | |||||||||
Loss from operations | (96,774 | ) | (73,270 | ) | (23,504 | ) | ||||||
Research and development incentives | - | 500 | (500 | ) | ||||||||
Interest expense | (928 | ) | (1,980 | ) | 1,052 | |||||||
Interest and other income, net | 1,291 | 1,412 | (121 | ) | ||||||||
Amortization of premium on investments - net | (973 | ) | (1,366 | ) | 393 | |||||||
Total other expense, net | (610 | ) | (1,434 | ) | 824 | |||||||
Net loss | $ | (97,384 | ) | $ | (74,704 | ) | $ | (22,680 | ) |
Nine Months Ended September 30, | ||||||||||||
2021 | 2020 | Change | ||||||||||
Operating expenses: | ||||||||||||
Research and development | $ | 93,315 | $ | 55,345 | $ | 37,970 | ||||||
General and administrative | 29,600 | 19,720 | 9,880 | |||||||||
Total operating expenses | 122,915 | 75,065 | 47,850 | |||||||||
Loss from operations | (122,915 | ) | (75,065 | ) | (47,850 | ) | ||||||
Research and development incentives | 500 | - | 500 | |||||||||
Interest expense | (2,514 | ) | (5,326 | ) | 2,812 | |||||||
Interest and other income net | 2,218 | 1,913 | 305 | |||||||||
Amortization of premium on investments - net | (2,111 | ) | (306 | ) | (1,805 | ) | ||||||
Total other expense, net | (1,907 | ) | (3,719 | ) | 1,812 | |||||||
Net loss | $ | (124,822 | ) | $ | (78,784 | ) | $ | (46,038 | ) |
Nine Months Ended September 30, | ||||||||
2021 | 2020 | |||||||
Cash used in operating activities | $ | (90,222 | ) | $ | (61,531 | ) | ||
Cash provided by (used in) investing activities | 1,989 | (42,129 | ) | |||||
Cash provided provided by financing activities | 36,545 | 546 | ||||||
Net change in cash, cash equivalents and restricted cash | $ | (51,688 | ) | $ | (103,114 | ) |
Six Months Ended June 30, | ||||||||
2022 | 2021 | |||||||
Cash used in operating activities | $ | (78,262 | ) | $ | (62,809 | ) | ||
Cash provided by investing activities | 15,794 | 20,178 | ||||||
Cash provided by financing activities | 17,322 | 9,907 | ||||||
Net change in cash, cash equivalents and restricted cash | $ | (45,146 | ) | $ | (32,724 | ) |
Item 3 | Quantitative and Qualitative Disclosures About Market Risk |
Exhibit | ||
Number | Description of Exhibit | |
Agreement and Plan of Merger and Reorganization, dated as of September 12, 2017, by and among Inotek Pharmaceuticals Corporation, Rocket Pharmaceuticals, Ltd., and Rome Merger Sub (incorporated by reference to Exhibit 2.1 to the Company’s Current Report on Form | ||
Seventh Amended and Restated Certificate of Incorporation of Rocket Pharmaceuticals, Inc., effective as of February 23, 2015(incorporated by reference to Exhibit 3.1 to the Company’s Annual Report on Form 10-K (001-36829), filed with the SEC on March 31, 2015) | ||
Certificate of Amendment (Reverse Stock Split) to the Seventh Amended and Restated Certificate of Incorporation of the Registrant, effective as of January 4, 2018 (incorporated by reference to Exhibit 3.1 to the Company’s Current Report on Form 8-K (001-36829), filed with the SEC on January 5, 2018) | ||
Certificate of Amendment (Name Change) to the Seventh Amended and Restated Certificate of Incorporation of the Registrant, effective January 4, 2018 (incorporated by reference to Exhibit 3.2 to the Company’s Current Report on Form 8-K (001-36829), filed with the SEC on January 5, 2018) | ||
Certificate of Amendment to the Seventh Amended and Restated Certificate of Incorporation of the Registrant, effective as of June 25, 2018 (incorporated by reference to Exhibit 3.1 to the Company’s Current Report on Form 8-K (001-36829), filed with the SEC on June 25, 2019 | ||
Amended and Restated By-Laws of Rocket Pharmaceuticals, Inc., effective as of March 29, 2018 (incorporated by reference to | ||
Certification of Principal Executive Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 | ||
Certification of Principal Financial Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 | ||
Certification of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C. Section 1350, as adopted 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 Taxonomy Extension Calculation Document. | |
101.DEF | Inline XBRL Taxonomy Extension Definition Linkbase Document. | |
101.LAB | Inline XBRL Taxonomy Extension Labels Linkbase Document. | |
101.PRE | Inline XBRL Taxonomy Extension Presentation Link Document. | |
104 | Cover Page Interactive Data File (the cover page XBRL tags are embedded within the Inline XBRL document) |
ROCKET PHARMACEUTICALS, INC. | |||
August 9, 2022 | By: | /s/ Gaurav Shah, MD | |
Gaurav Shah, MD | |||
Chief Executive Officer and Director | |||
(Principal Executive Officer) | |||
August 9, 2022 | By: | /s/ | |
John Militello | |||
(Interim Principal Financial Officer | |||