Rocket Pharmaceuticals' RP-A501 Phase 2 Trial Undergoes Adjusted Protocol

Rocket Pharmaceuticals' pivotal Phase 2 trial for RP-A501 has been cleared by the FDA after a clinical hold was imposed due to a serious adverse event. However, the trial will now proceed with a lower dose and adjusted immune suppression regimen, introducing uncertainty about the therapy's efficacy and safety. Analyst Michael Ulz of Morgan Stanley maintains a Hold rating and price target of $7.00, citing cautious optimism and the need for further data to confirm safety and effectiveness.

The U.S. Food and Drug Administration (FDA) has lifted the clinical hold on Rocket Pharmaceuticals, Inc.'s (NASDAQ: RCKT) pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease. The FDA's decision, made in less than three months, follows Rocket's satisfactory response to the agency's concerns [1].

The trial, which was temporarily halted due to a serious adverse event, will now resume with a recalibrated dose of 3.8 × 10¹³ GC/kg of RP-A501 in three patients. This dose is within the lower range of those previously tested in Phase 1, demonstrating efficacy across multiple biomarkers and clinical endpoints [2]. Additionally, the company will adopt a revised immunomodulatory regimen, discontinuing prophylactic use of a C3 complement inhibitor while continuing treatment with sirolimus, rituximab, and steroids [1].

The Phase 2 trial is a global, single-arm, multi-center study targeting 12 patients, with six patients already treated. The study evaluates efficacy using a biomarker-based co-primary endpoint, focusing on improvements in LAMP2 protein expression and reductions in left ventricular mass. Key secondary endpoints include changes in troponin levels, natriuretic peptides, Kansas City Cardiomyopathy Questionnaire scores, New York Heart Association class, event-free survival at 24 months, and treatment-emergent safety outcomes [2].

Analyst Michael Ulz of Morgan Stanley maintains a Hold rating and a price target of $7.00, citing cautious optimism and the need for further data to confirm the safety and effectiveness of the therapy [1].

References:
[1] https://investorshub.advfn.com/market-news/article/15017/rocket-pharmaceuticals-surges-as-fda-lifts-clinical-hold-on-phase-2-trial
[2] https://www.businesswire.com/news/home/20250820073531/en/Rocket-Pharmaceuticals-Announces-FDA-Has-Lifted-the-Clinical-Hold-on-the-Pivotal-Phase-2-Trial-of-RP-A501-for-the-Treatment-of-Danon-Disease