Solid Biosciences Inc. has resumed the Phase I/II IGNITE DMD trial of the gene therapy SGT-001, dosing a seventh patient with Duchenne muscular dystrophy in the study. Meanwhile, the company also announced on 15 March encouraging interim data from the first six patients dosed in the study, improving the outlook for the gene therapy, though not eliminating uncertainty in what is a challenging area of drug development.
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.
More from Clinical Trials
• By
Aldeyra’s dry eye candidate reproxalap received a second FDA complete response letter, but the firm expressed confidence about refiling quickly based on two ongoing studies.
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
• By
By allowing it to enter the brain more easily, trontinemab’s brain shuttle brings more patients to ‘amyloid zero’ levels faster, and with fewer brain swelling side effects.
• By
Compass' bispecific antibody tovecimig hits primary efficacy endpoint in Phase II/III top-line data in advanced biliary tract cancer, and may have class side-effect advantages. But additional survival data may be needed to support US approval.
More from R&D
• By
Compass' bispecific antibody tovecimig hits primary efficacy endpoint in Phase II/III top-line data in advanced biliary tract cancer, and may have class side-effect advantages. But additional survival data may be needed to support US approval.
• By
AstraZeneca remains committed to investing in R&D and alliances in China, where Susan Galbraith, the UK major’s head of oncology R&D, sees innovation eventually reaching parity with the US and Europe.
• By
BeiGene’s Phase III ociperlimab joins the list of failed TIGIT inhibitors, as candidates from Roche, Merck & Co. and others have failed late-stage studies.