Shares of Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) jumped 23% after the U.S. Food and Drug Administration lifted the clinical hold on the company’s pivotal Phase 2 trial of RP-A501 for treating Danon disease.
The FDA removed the hold in less than three months, confirming that Rocket had adequately addressed the agency’s prior concerns. The study is now authorized to resume with a recalibrated dose of 3.8 × 10¹³ GC/kg of RP-A501 in three patients, who will be treated sequentially with at least a four-week interval between each administration.
This new dose falls within the lower range of those previously tested in Phase 1, which demonstrated efficacy across multiple biomarkers and clinical endpoints. The company will also adopt a revised immunomodulatory regimen, discontinuing prophylactic use of a C3 complement inhibitor while continuing treatment with sirolimus, rituximab, and steroids.
So far, six patients have been treated in the Phase 2 study, which is a global, single-arm, multi-center pivotal trial targeting a total of 12 patients. The trial 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, with additional measures covering natriuretic peptides, Kansas City Cardiomyopathy Questionnaire scores, New York Heart Association class, event-free survival at 24 months, and treatment-emergent safety outcomes.
Rocket Pharmaceuticals stock price
This content is for informational purposes only and does not constitute financial, investment, or other professional advice. It should not be considered a recommendation to buy or sell any securities or financial instruments. All investments involve risk, including the potential loss of principal. Past performance is not indicative of future results. You should conduct your own research and consult with a qualified financial advisor before making any investment decisions.