Rocket Pharma Q1 Results; KRESLADI Approved; PRV Sold for $180M
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.
Key Highlights
- KRESLADI™ granted FDA accelerated approval for the treatment of severe LAD-I.
- Rare Pediatric Disease Priority Review Voucher monetized for $180 million.
- Pro forma cash, cash equivalents, and investments of approximately $322.6 million, extending operational runway into Q2 2028.
- Cash, cash equivalents, and investments were $144.4 million as of March 31, 2026 (excluding PRV monetization).
- Net loss was $47.6 million ($0.42 per share) for Q1 2026, compared to $61.3 million ($0.56 per share) for Q1 2025.
- Research and development expenses decreased by $4.4 million to $31.5 million in Q1 2026.
- General and administrative expenses decreased by $11.3 million to $17.1 million in Q1 2026.
- Dosing reinitiated in pivotal Phase 2 trial of RP-A501 for Danon disease.
Price Impact
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