ROCKET PHARMACEUTICALS, INC.
For the period ended Jun 30, 2025. Filed Aug 7, 2025.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Jun 30, 2025 · Filed Aug 7, 2025
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LAD-I CIRM Grant
On April 30, 2019, CIRM awarded the Company up to $7.5 million under a CLIN2 grant to support the clinical development of its LV-based gene therapy, RP-L201. Proceeds from the grant helped fund clinical trial costs as well as manufactured drug product for Phase 1/2 patients enrolled at the U.S. clinical site, University of California, Los Angeles Mattel Children’s Hospital, led by principal investigator Donald Kohn, M.D., UCLA Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. As of June 30, 2025, the Company has received $5.9 million in total RP-L201 grants from CIRM. The Company received a final milestone grant of $0.05 million on January 2, 2024, andno additional payments are available under the grant awards program.
DD CIRM Grant
On August 18, 2024, CIRM awarded the Company up to $5.8 million under a CLIN2 grant award to support the clinical development of its AAV-based gene therapy, RP-A501 for the treatment of DD. Proceeds from the grant would help fund clinical trial costs as well as manufactured drug product for Phase 1/2 patients. During thesix months ended June 30, 2025, the Company received grants of $2.7 million, which were recorded as a reduction of R&D expenses. Through June 30, 2025, the Company has received RP-A501 grants of $5.0 million from CIRM.
Related Party Transactions
In June 2023, the Company entered into a consulting agreement with the spouse of one of the Company’s executive officers for information technology advisory services. The Company incurred expenses of approximately $0 and $2,000 for thesix months ended June 30, 2025 and 2024, respectively, for services provided under this agreement.
In December 2024, in connection with a public offering, the Company sold 400,000 pre-funded warrants to purchase shares of the Company’s common stock to funds affiliated with RTW, the Company’s largest shareholder (see Note 8 “Stockholders’ Equity”).
In February 2025, the Company entered into a consulting agreement with one of the Company’s board members, effective March 3, 2025, for services related to the Company’s R&D activities. As compensation for services rendered during 2025, the consultant will receive $125,000 to be paid in equal monthly installments and $125,000 of RSU’s valued as of the closing price on March 3, 2025, which will cliff vest on December 31, 2025. The agreement will terminate on December 31, 2025, unless terminated earlier by the Company for cause or voluntarily by the consultant. The board member was paid approximately $50,000 for thesix months ended June 30, 2025, for services provided under the consulting agreement.
In June 2025, the Company received a total of approximately $215,000, representing return of short-swing profits from the sale of common stock by beneficial owners under Section 16(b) of the Securities and Exchange Act of 1934, as amended. The Company recognized these proceeds as a capital contribution and reflected a corresponding increase to additional paid-in capital.
401(k) Savings Plan
The Company has a defined contribution savings plan (the “Plan”) under Section 401(k) of the Internal Revenue Code of 1986. This Plan covers substantially all employees who meet the minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pre-tax basis. Company contributions to the Plan may be made at the discretion of the Company’s Board of Directors. The Company has elected the safe harbor match of 4% of employee contributions to the Plan, subject to certain limitations. The Company’s matching contribution for thethree and six months ended June 30, 2025, was $0.7 million and $1.2 million, respectively. The Company’s matching contribution for the three and six months ended June 30, 2024, was $0.4 million and $0.8 million, respectively.
Segment Reporting
The Company hasone reportable segment related to R&D and commercial readiness of its gene therapies.
The Company’s CODM is its Chief Executive Officer and the senior leadership team. The CODM manages the Company’s operations on an integrated basis for the purpose of allocating resources. When evaluating the Company’s financial performance, the CODM regularly reviews total expenses and expenses by significant areas to make decisions on a company-wide basis. Included in these expenses are R&D expenses by program.
The table below is a summary of the segment loss, including significant segment expenses:
| Three Months Ended June 30, | Six Months Ended June 30, | |||
|---|---|---|---|---|
| 2025 | 2024 | 2025 | 2024 | |
| Revenue | $ | - | $ | - |
| Operating expenses: | ||||
| Research and development | 42,658 | 46,345 | 78,600 | 91,572 |
| Non-commercial general and administrative | 17,042 | 16,777 | 37,657 | 32,553 |
| Commercial general and administrative | 7,978 | 10,590 | 15,809 | 16,962 |
| Restructuring | 3,471 | - | 3,471 | - |
| Total operating expenses | 71,149 | 73,712 | 135,537 | 141,087 |
| Loss from operations | (71,149) | (73,712) | (135,537) | (141,087) |
| Interest expense | (473) | (471) | (945) | (942) |
| Interest and other income, net | 483 | 2,294 | 1,819 | 5,323 |
| Accretion of discount and amortization of premium on investments, net | 2,220 | 2,243 | 4,410 | 5,006 |
| Net Segment loss and Net loss | $(68,919) | $(69,646) | $(130,253) | $(131,700) |
The Company’s CODM uses net loss to evaluate past spending and to guide decisions about future spending. Net loss is used to monitor the budget versus actual results. The CODM also uses net loss in analysis of programs and along with the monitoring of budgeted versus actual results in assessing performance of the segment and in establishing manager’s compensation.
Restructuring
In June 2025, the Company’s Board of Directors approved a restructuring plan to prioritize investments in its AAV platform and reduce overall cash spending, which was communicated to employees before the end of June 2025. The restructuring includes a reduction of the Company’s workforce by approximately 80 employees.
As a result of the restructuring, the Company incurred aggregate charges of approximately $3.5 million in restructuring costs related to severance and employee termination costs to be paid out over multiple months through the second half of 2025.
The following table summarizes the accrued liabilities activity in connection with the restructuring plan for thethree and six months ended June 30, 2025:
| Three and Six Months, | |
|---|---|
| Ended June 30, | |
| Beginning balance · $ · - | |
| Restructuring charges incurred during the period | 3,471 |
| Restructuring charges paid during the period | - |
| Remaining accrual at June 30, 2025 | $3,471 |
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