Dive Brief:

• Following last month's announcement of a full clinical hold on Solid Biosciences Inc.'s Phase 1/2 Duchenne muscular dystrophy study, IGNITE DMD, the Food and Drug Administration is seeking further clinical information and measures to address patient safety.
• The biotech said it plans to submit a response "in the coming weeks" on the trial, which was evaluating a gene therapy known as SGT-001. The hold came after a patient was hospitalized with low platelet and red blood cell counts. The treated patient is now doing well.
• Meanwhile, an additional, although unconnected, partial clinical hold on the high dose of SGT-001 has been resolved. Solid said the manufacturing-related questions behind the hold have been "satisfactorily addressed."

Dive Insight:

Solid hasn't had an easy start for its only clinical-stage program. Just days after receiving a first dose of the microdystrophin gene therapy, the initial patient in the study ended up in the hospital with reduced platelet and red blood cell counts. Following this serious adverse event, the FDA placed a full clinical hold on the study, which was already on a partial clinical hold following a manufacturing issue with the highest dose.

"We are working with the FDA to better understand the unexpected event that resulted in the clinical hold and put a plan in place to monitor and manage potential events in the future. I am happy to share that the treating physician has reported the patient is doing well," Solid's CEO Ilan Ganot said in a statement. "We are also pleased that we were able to lift the earlier partial clinical hold on the high dose of SGT-001."

The company has sent out an update to the DMD community, stating that the team is "committed to maintaining dialogue with the FDA with the hope of resuming IGNITE DMD as soon as possible."

The gene therapy space is a challenging one, offering great hope for patients balanced with high levels of risk for companies. Novartis AG is pushing forward with its purchase of AveXis Inc. for $8.7 billion. At the same time, GlaxoSmithKline plc is pulling out, divesting its gene therapy portfolio to U.K. biotech Orchard Therapeutics Ltd. in return for a near-20% stake in the smaller company.