Rocket Pharmaceuticals rises after FDA lifts clinical hold on gene therapy trial

Rocket Pharmaceuticals

20/08/2025 17:04

Updates

** Shares of drug developer Rocket Pharmaceuticals RCKT.O rise 16.3% to $3.38

** Company says the U.S. FDA has lifted a clinical hold placed on its mid-stage study testing its gene therapy RP-A501

** The hold was lifted in under three months after the FDA confirmed Rocket had satisfactorily addressed the outlined issues - company

** FDA in May paused RCKT's mid-stage gene therapy trial after a patient died from serious complications

** FDA cleared the trial to resume with a lower dose administered to three patients sequentially, with treatments spaced at least four weeks apart

     ** Company is testing RP-A501 for the treatment of Danon disease, a rare genetic disorder that leads to heart muscle damage and progressive muscle weakness

** "We are encouraged by the remarkably rapid resolution of the clinical hold, which further underscores the unmet need in Danon. However, the resumption of the study at a lower dose increases uncertainty around efficacy, given limited data at this dose" - Needham analysts

** Including session's move, RCKT stock down 72.9% YTD

(Reporting by Sneha S K and Padmanabhan Ananthan)

((Sneha.SK@thomsonreuters.com))