ROCKET PHARMACEUTICALS, INC.

Rocket Pharmaceuticals reported Q1 2026 financial results, including a net loss of $47.6 million and cash, cash equivalents, and investments of $144.4 million as of March 31, 2026. The company announced the FDA's accelerated approval of KRESLADI for severe LAD-I and subsequently monetized its Rare Pediatric Disease Priority Review Voucher for $180 million. This transaction extends the company's operational runway into Q2 2028. Additionally, Rocket provided updates on its cardiovascular gene therapy pipeline, including reinitiating dosing for the pivotal Phase 2 trial of RP-A501 for Danon disease and anticipating first patient dosing in the Phase 1 study of RP-A701 for BAG3-related dilated cardiomyopathy in mid-2026.

Rocket Pharmaceuticals reported a reduced net loss of $(47.6) million for Q1 2026, down from $(61.3) million in Q1 2025. The company received FDA accelerated approval for KRESLADI™ in March 2026 for severe LAD-I, though material revenue is not expected in the near term. To support its cardiovascular gene therapy pipeline and extend its operational runway, Rocket entered an agreement in April 2026 to sell its Rare Pediatric Disease Priority Review Voucher for $180 million. The company also implemented a strategic reorganization in July 2025, reducing its workforce by 30% to focus on its AAV-based gene therapy platform.

Rocket Pharmaceuticals, Inc. announced it has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $180 million in cash. This non-dilutive capital infusion is expected to extend the company's cash runway into the second quarter of 2028. The proceeds will be used to advance Rocket's prioritized cardiovascular gene therapy pipeline, including programs for Danon disease, PKP2-associated arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy.