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Investing.com -- Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) stock surged 23% after the FDA lifted a clinical hold on the company’s pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease.
The hold was removed in under three months, with the FDA confirming that Rocket had satisfactorily addressed the outlined issues. The agency has authorized the study to resume with a recalibrated dose of 3.8 x 10¹³ GC/kg of RP-A501 in three patients, who will be treated sequentially with a minimum four-week interval between each treatment.
This adjusted dose aligns with the lower range of administered doses that showed efficacy across multiple biomarkers and clinical endpoints in the Phase 1 study. The company will also implement a revised immunomodulatory regimen that discontinues prophylactic use of a C3 complement inhibitor while maintaining sirolimus, rituximab, and steroids.
To date, six patients with Danon disease have been treated in the Phase 2 study. The global, single-arm, multi-center pivotal trial aims to evaluate 12 patients total. The study assesses efficacy through a biomarker-based co-primary endpoint consisting of improvements in LAMP2 protein expression and reductions in left ventricular mass.
The key secondary endpoint is change in troponin, with additional secondary endpoints including natriuretic peptides, Kansas City Cardiomyopathy Questionnaire, New York Heart Association class, event-free survival to 24 months, and treatment emergent safety events.
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