Rocket Pharmaceuticals, Inc. reported first quarter 2026 results and highlighted major pipeline and financing milestones. The company received FDA accelerated approval for KRESLADI for severe LAD-I and monetized a Rare Pediatric Disease Priority Review Voucher for $180 million.
Pro forma cash, cash equivalents and investments were approximately $322.6 million, providing an expected operational runway into the second quarter of 2028. For the three months ended March 31, 2026, Rocket recorded a net loss of $47.6 million compared with $61.3 million a year earlier, with total operating expenses declining to $48.5 million from $64.4 million. Cash, cash equivalents and investments were $144.4 million as of March 31, 2026.
Rocket Pharmaceuticals, Inc. has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher for $180 million in cash, payable at closing. The voucher was granted after FDA accelerated approval of KRESLADI, Rocket’s gene therapy for severe LAD-I.
Rocket states that monetizing the voucher provides meaningful non-dilutive capital and extends its cash runway into the second quarter of 2028. The company plans to use the proceeds to support its prioritized cardiovascular gene therapy pipeline, including clinical programs in Danon disease, PKP2-associated arrhythmogenic cardiomyopathy and BAG3-associated dilated cardiomyopathy.
Rocket Pharmaceuticals is calling a virtual 2026 annual stockholder meeting on May 20, 2026, to vote on four key items: electing seven directors, ratifying EisnerAmper LLP as auditor, a non-binding advisory vote on executive pay, and approving a stock option exchange program.
Stockholders of record on March 24, 2026, when 109,122,837 common shares were outstanding, may vote online or by proxy. The proxy also describes a 2025 strategic reorganization that cut headcount by about 30% and is expected to lower 12‑month operating expenses by nearly 25%, along with a pay-for-performance executive compensation framework that received about 98% support in the 2025 advisory vote.
Rocket Pharmaceuticals Inc reported an amendment to institutional holdings showing 0 shares beneficially owned by The Vanguard Group after an internal realignment. The filing states that, following the realignment described in January 12, 2026, certain Vanguard subsidiaries will report holdings separately.
The Schedule 13G/A lists Amount beneficially owned: 0 and Percent of class: 0%. The filing is signed by Ashley Grim on March 27, 2026.
Rocket Pharmaceuticals, Inc. has filed a shelf registration on Form S-3 to offer, from time to time, up to $400,000,000 of common stock, preferred stock, debt securities, warrants and/or units, in one or more offerings, as set forth in prospectus supplements.
The prospectus states the offering is a shelf registration that permits multiple issuances in amounts, at prices and on terms to be determined at the time of each offering. Authorized capital stock is 180,000,000 shares of common stock; shares outstanding were 108,569,143 as of March 2, 2026.
Rocket Pharmaceuticals reported fourth-quarter 2025 net loss of $42,538, improving from $60,327 a year earlier. Full-year 2025 net loss was $223,123, compared with $258,746 in 2024, with net loss per share of $2.01 versus $2.73.
Total operating expenses for 2025 were $231,747, down from $273,205 in 2024, reflecting lower research and development and general and administrative spending and recorded restructuring charges of $3,231. Cash, cash equivalents and investments were $188,929 at December 31, 2025, versus $372,336 a year earlier, which the company says supports an operational runway into the second quarter of 2027.
Operationally, Rocket highlighted progress across its cardiovascular gene therapy programs. The pivotal Phase 2 trial of RP‑A501 for Danon disease is expected to resume in the first half of 2026, the KRESLADI™ BLA for severe LAD‑I has a March 28, 2026 PDUFA date, and first dosing in a Phase 1 study of RP‑A701 for BAG3‑related dilated cardiomyopathy is anticipated in mid‑2026.
Rocket Pharmaceuticals is a late-stage biotech company developing gene therapies for rare, often fatal genetic diseases, with a strategic focus on inherited cardiomyopathies. It runs both AAV and lentiviral platforms and operates a large cGMP manufacturing facility in Cranbury, New Jersey.
In 2025 Rocket implemented a corporate reorganization, including a workforce reduction of about 30% and cost-saving initiatives expected to lower 12‑month operating expenses by roughly 25%, to extend its cash runway and concentrate resources on its AAV cardiovascular pipeline. Development of Fanconi anemia and pyruvate kinase deficiency programs is being de‑prioritized while the company explores partnerships.
Lead programs include RP‑A501 for Danon disease, where positive long‑term Phase 1 data supported a pivotal Phase 2 trial that was temporarily paused after two serious adverse events, including one death, then allowed to resume at a lower dose with added safeguards. RP‑A601 for PKP2‑related arrhythmogenic cardiomyopathy has early Phase 1 data showing tolerability and increased PKP2 expression, and RP‑A701 for BAG3‑related dilated cardiomyopathy entered Phase 1 after FDA IND clearance and Fast Track designation.
In hematology, ex vivo gene therapy KRESLADI for severe LAD‑I has published long‑term data and a resubmitted BLA with a new PDUFA date of March 28, 2026. Across programs, Rocket highlights extensive regulatory designations, complex licensing arrangements, and a wide array of risks, including ongoing losses, financing needs, clinical and regulatory uncertainty, manufacturing and data‑privacy obligations, intense competition, and protection of its intellectual property.
Rocket Pharmaceuticals, Inc. officer John Militello reported an open-market sale of 1,157 shares of common stock at an average price of $3.338 per share. According to the filing, the shares were sold to cover tax withholding obligations from vesting restricted stock units, and he now holds 91,019 shares, including RSUs that convert one-for-one into common stock.
Rocket Pharmaceuticals CEO Gaurav Shah reported a tax-related stock sale. On February 18, 2026, he sold 5,990 shares of common stock at $3.338 per share to cover tax withholding obligations tied to vesting restricted stock units. After this transaction, he directly holds 1,046,055 common shares, with additional indirect holdings of 207,897 shares held by his spouse and 198,341 shares held by the Gaurav D. Shah Irrevocable Trust.