Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) provided an update on the phase 2 clinical trial of RP-A501 for Danon disease. The company announced an unexpected serious adverse event (SAE) experienced by a patient participating in the trial, leading to the FDA placing a clinical hold on the trial for further evaluation.
As of March 31, 2025, Rocket had cash, cash equivalents, and investments of $318.2 million, and the company is prioritizing investment in its AAV platform while assessing ways to optimize value for the rest of the pipeline.
The global, single-arm, multi-center phase 2 pivotal trial evaluates the efficacy and safety of RP-A501 in 12 patients with Danon disease, including a pediatric safety run-in (n=2), and a dose level of 6.7 x 1013 gc/kg.
RP-A501 is Rocket’s investigational gene therapy product for the treatment of Danon disease and is the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies. It consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a full-length, wild-type version of the human lamp2b transgene (AAV9.LAMP2B).
Danon disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), leading to heart failure and frequent death during adolescence or early adulthood for male patients.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Their innovative multi-platform approach allows for the optimal gene therapy for each indication, creating potentially transformative options for individuals living with rare diseases. The market has reacted to these announcements by moving the company's shares -4.42% to a price of $6.27. Check out the company's full 8-K submission here.
