Adverse Event Concerns With Solid Biosciences' Candidate DMD Gene Therapy
Cardiopulmonary Symptoms May Have Triggered Clinical Hold
Executive Summary
The US FDA has for the second time placed a clinical hold on Solid Biosciences’ potential gene therapy for Duchenne muscular dystrophy, a move linked to a 70% decline in the company’s share price. The company had reported a serious adverse event in a patient treated with higher doses of the viral vector-delivered gene therapy.
You may also be interested in...
Solid Restarts DMD Gene Therapy Trial, With Encouraging Interim Data To Back It Up
The Phase I/II trial testing SGT-001 in patients with Duchenne muscular dystrophy resumed after FDA lifted a clinical hold on the trial; interim data from the first six patients were encouraging.
Finance Watch: Billions Flow Into Biopharma Via Three New Investment Funds
Blackstone raised $3.44bn of a $4.58bn life science fund, while HealthCare Royalty Partners raised $1.8bn and MVM closed a $325m fund. Also, 20 private drug developers raised $20m or more, but three public biopharma firms unveiled restructuring plans.
Roche Discontinues Duchenne Hope At Interim Analysis
Roche is to discontinue its RG6206 clinical development program in Duchenne muscular dystrophy after an interim analysis of the Phase II/III SPITFIRE study indicates the compound would be “highly unlikely” to show a clinical benefit.