RCKT biotech catalysts & pipeline

ROCKET PHARMACEUTICALS INC · Data as of 2026-10-25

Upcoming catalysts

RP-A501 — Regulatory decision

H2 2026

Rocket is actively engaging with the FDA to seek alignment on the path to dosing additional patients and completing the pivotal Phase 2 trial and expects to provide an update on the regulatory pathway in the second half of 2026.

Source filing

RP-A601 — Regulatory decision

H2 2026

Rocket continues to engage with the FDA regarding alignment on the design of a potential pivotal study of RP-A601 in PKP2-ACM with regulatory update expected in the second half of 2026.

Source filing

RP-A701 — Initial data

H2 2026

Patient screening and enrollment has begun, with initial patient dosing anticipated in the second half of 2026.

Source filing

RP-A501 — Topline data

mid-2028

Rocket expects to complete pivotal trial dosing by mid-2027, with topline results anticipated mid-2028.

Source filing

RP-A501 — NDA/BLA submission

LATE-2028

LATE-2028 BLA regulatory filing PRV eligible; Accelerated Approval; Priority review

Source filing

RP-A501 — Conference presentation

H2 2026

Rocket is on track to host an investor webinar in the second half of 2026 to provide a comprehensive Danon disease program update.

Source filing

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