RCKT Completes Enrollment in Rare Heart Disease Study, Stock Rises

Core Viewpoint - Rocket Pharmaceuticals has completed patient enrollment in a pivotal phase II study for its gene therapy candidate RP-A501, aimed at treating male patients with Danon disease, a rare genetic disorder [1][3]. Company Developments - The company estimates that 15,000 to 30,000 patients in the U.S. and Europe suffer from Danon disease, with cardiac transplantation being the only current treatment option, which is not curative [2]. - Following the announcement of the study completion, Rocket's shares increased by 13.5% on September 17, although they have declined by 27.2% year-to-date compared to a 1% increase in the industry [2]. Study Details - The global multi-center phase II study focuses on the safety and efficacy of RP-A501, with co-primary endpoints including improvements in LAMP2 protein expression and reductions in left ventricular mass [3][4]. - Key secondary endpoints include changes in troponin levels and natriuretic peptides event-free survival to 24 months, along with treatment-emergent safety events [4]. Regulatory Designations - RP-A501 has received regenerative medicine advanced therapy designation from the FDA and PRIME scheme designation from the European Medical Agency, indicating its potential significance in treating Danon disease [5]. Other Pipeline Activities - Rocket is also developing Kresladi for severe leukocyte adhesion deficiency-I (LAD-I), although the FDA issued a complete response letter regarding its biologics license application, requesting additional information on Chemistry Manufacturing and Controls [6]. - Additionally, the company is evaluating two other gene therapies, RP-L102 and RP-L301, for Fanconi anemia and pyruvate kinase deficiency, respectively, both in mid-stage development [7].