Roche Discontinues Duchenne Hope At Interim Analysis
Anti-Myostatin Approach Disappoints Again
Executive Summary
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.
You may also be interested in...
Biohaven Returns To Well With Bristol License, Acquires Channel Biosciences
With Nurtec and zavegepant in hand from Bristol, Biohaven tries for another success with a spinal muscular atrophy asset. It will move into epilepsy indications with the buyout of Channel, a subsidiary of Knopp.
Sarepta Secures $1.15bn From Roche In Ex-US DMD Gene Therapy Deal
Including upfront payments, half of development costs for SRP-9001, $1.7bn in milestone fees and royalties on sales outside the US, Sarepta pegs deal’s value at $10bn-plus.
Adverse Event Concerns With Solid Biosciences' Candidate DMD Gene Therapy
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.