Rocket Pharma outlines FDA-aligned pivotal Phase 2 trial path for Danon gene therapy RP-A501
RCKT•Rocket Pharmaceuticals outlined an FDA-aligned pivotal Phase 2 path for its Danon disease gene therapy RP-A501 during an Oct. 6 investor webinar. Dosing is expected to finish by mid-2027, with topline results expected in mid-2028.
1. Trial timeline
Rocket Pharmaceuticals said its pivotal Phase 2 study of RP-A501 is intended to target accelerated approval. The company expects dosing to finish by mid-2027 and topline results in mid-2028.
2. Early observations
The company cited durable signs of benefit in Phase 1 follow-up but did not detail measurements. Early safety observations from the first three patients on a revised dosing protocol showed no initial signs of key prior risks.
