Rocket Pharmaceuticals Inc (RCKT) News & EventsRCKTRocket Pharmaceuticals Inc

Rocket Pharmaceuticals Highlights Danon Disease Research Progress

Rocket Pharmaceuticals highlighted key findings and development milestones presented during its October 6 Danon disease investor webinar. The update outlined a defined path to completing the FDA-aligned pivotal Phase 2 trial, highlighted sustained biological activity in longer-term Phase 1 follow-up and reported encouraging initial safety observations in the first three patients treated under the modified protocol. Key highlights included: Pivotal Phase 2 trial: The FDA-aligned pivotal efficacy population includes 12 male patients receiving commercial-grade RP-A501 at the recalibrated dose. The first three patients treated under the modified protocol count toward this population. Trial success requires at least seven patients to meet both response criteria at 12 months: myocardial LAMP2 protein expression of at least Grade 1 and a reduction in left ventricular mass index of at least 10% from baseline. These co-primary endpoints are intended to support a potential accelerated approval pathway. Modified protocol: The first three patients treated at the recalibrated dose completed the protocol-specified initial observation period without clinical or laboratory evidence of thrombotic microangiopathy or capillary leak syndrome, as reported during the webinar. Follow-up is ongoing, and these initial observations are preliminary. Phase 1 results: At approximately 12 months, all six patients in the principal Phase 1 analysis demonstrated myocardial LAMP2 protein expression and at least a 10% reduction in left ventricular mass index. At follow-up of approximately three to seven years, left ventricular mass index was below baseline in five of six patients, with reductions of at least 10% in four patients. All six patients also had sustained reduction of cardiac biomarkers and improvements of quality of life. Original-dose Phase 2 results: Three of four patients with longitudinal efficacy assessments achieved at least a 10% reduction in left ventricular mass index. Safety findings from the original-dose study, including thrombotic microangiopathy and a fatal serious adverse reaction, informed the recalibrated dose, revised immunomodulation regimen and enhanced safety monitoring now in place. Patient population: Genetic and clinical analyses converged on a modeled U.S. Danon disease population of approximately 10,000-11,000, including 4,000 males. Rocket's initial focus is males with symptomatic hypertrophic cardiomyopathy. Upcoming milestones: Rocket expects to complete pivotal trial dosing by mid-2027, with topline results anticipated mid-2028.

Rocket Pharmaceuticals Enters $150 Million Credit Facility with Hercules Capital

Rocket Pharmaceuticals (RCKT) "announced that it has entered into a credit facility agreement with Hercules Capital (HTGC) for up to $150 million. The facility strengthens Rocket's financial position and provides access to additional capital to support execution of its pivotal Phase 2 study of RP-A501 for Danon disease and continued advancement of its broader cardiovascular pipeline."

Rocket Pharmaceuticals Aligns with FDA on RP-A501 Clinical Trial

Rocket Pharmaceuticals announced alignment with the U.S. Food and Drug Administration, FDA, on continued enrollment and dosing and key elements of the global pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease, including the recalibrated dose, 12-patient pivotal population and established 12-month co-primary endpoints. The clinical data reviewed by FDA included results from the first three patients treated under the modified protocol. All three completed at least four weeks of follow-up and had been discharged following protocol-specified observation. No clinical or laboratory evidence of thrombotic microangiopathy or capillary leak syndrome had been observed. Following its review of the available safety data, the FDA confirmed continued enrollment and dosing under the modified protocol at the recalibrated dose.