Rocket Pharmaceuticals Halts Danon Study Following Serious Adverse Event In Participant, Stock Tanks

(RTTNews) - Rocket Pharmaceuticals, Inc. (RCKT), Tuesday announced its decision to voluntarily halt the Phase 2 pivotal trial of RP-A501, its investigational gene therapy for Danon disease, which is caused by mutations in the LAMP2 gene.

The decision comes as a participant of the trial experienced an unexpected serious adverse event related to a capillary leak syndrome. The company reported that the patient has passed away after an acute systemic infection.

Following the initial event, the biotechnology company voluntarily paused further dosing in the study. Later, the U.S. Food and Drug Administration placed a clinical hold on the trial to allow for further evaluation.

Notably, Rocket is working with the FDA, the Independent Data Safety Monitoring Committee, clinical investigators, and scientific experts to ensure safety of all participants. However, due to the clinical hold, the company is unable to provide guidance on the anticipated timing for completion of the Phase 2 trial.

Additionally, the company stated that it now expects its existing resources to be sufficient to fund operations into 2027, excluding any potential proceeds from the sale of Priority Review Vouchers that could follow the approval of therapies from the hematology portfolio.

As of March 31, 2025, Rocket had cash, cash equivalents, and investments of $318.2 million.

In the pre-market hours, Rocket's stock is plummeting 63.32 percent, to $2.30 on the Nasdaq.