Rocket Pharmaceuticals Inc

Rocket Pharmaceuticals said it has a clear path to complete a pivotal phase 2 trial for its Danon disease asset RP-A501. Twelve male patients will receive treatment, with co-primary endpoints of myocardial LAMP2 protein expression of at least Grade 1 and a reduction in left ventricular mass index of at least 10% from baseline after one year. If those endpoints are met, Rocket could seek a potential accelerated approval pathway. Phase 1 results showed that at around 12 months, the six patients in the main phase 1 analysis had myocardial LAMP2 protein expression and at least a 10% reduction in left ventricular mass index. At follow-up of 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. Danon disease is a rare genetic disorder characterized by cardiomyopathy and muscle weakness.

Rocket Pharmaceuticals completed the sale of its Rare Pediatric Disease Priority Review Voucher for gross proceeds of $180 million. The voucher was awarded by the FDA in March 2026 following the approval of Kresladi, a gene therapy for severe leukocyte adhesion deficiency-I. Before the transaction, the company reported cash, cash equivalents, and investments of $144.4 million as of March 31, 2026, and the non-dilutive proceeds increased its pro forma cash position to approximately $322.6 million, extending its expected operational runway through the second quarter of 2028. BofA Securities analyst Jason Zemansky recently raised the price target on Rocket Pharmaceuticals to $10 from $9 and maintained a Buy rating, citing the restart of RP-A501 dosing as a positive step in the company's near-term recovery strategy.