Rocket Pharmaceuticals (RCKT) investor relations material

Executive summary

• Achieved FDA accelerated approval for KRESLADI™ in March 2026 for severe LAD-I; commercial launch activities underway, but material revenue not expected near-term.

• Monetized a Priority Review Voucher for $180 million in April 2026, providing significant non-dilutive funding and extending operational runway.

• Strategic reorganization in July 2025 prioritized AAV cardiovascular gene therapy programs and reduced workforce by 30%.

• Reinitiated dosing in pivotal Phase 2 trial of RP-A501 for Danon disease after FDA lifted clinical hold in August 2025.

• Anticipates first patient dosing in Phase 1 study of RP-A701 for BAG3-related dilated cardiomyopathy in mid-2026 and ongoing engagement with FDA for RP-A601 pivotal Phase 2 trial design in PKP2 arrhythmogenic cardiomyopathy.

Financial highlights

• Net loss for Q1 2026 was $47.6 million ($0.42 per share), an improvement from $61.3 million ($0.56 per share) in Q1 2025.

• R&D expenses decreased to $31.5 million (from $35.9 million year-over-year); G&A expenses fell to $17.1 million (from $28.4 million year-over-year).

• Cash, cash equivalents, and investments totaled $144.4 million as of March 31, 2026, excluding PRV monetization.

• Pro forma cash position, including PRV proceeds, is $322.6 million.

• Operating expenses for Q1 2026 were $48.5 million, down from $64.4 million in Q1 2025.

Outlook and guidance

• Proceeds from PRV sale and current cash expected to fund operations into Q2 2028.

• No material product revenue expected from KRESLADI™ in the near term due to ultra-rare patient population and phased rollout.

• Continued focus on advancing AAV cardiovascular pipeline and exploring partnerships for non-core programs.

• Update on Danon program anticipated in the second half of 2026.