Solid Biosciences IGNITE DMD placed on clinical hold
Official press release
CAMBRIDGE, Mass., Nov. 12, 2019 (GLOBE NEWSWIRE) — Solid Biosciences Inc. (Nasdaq: SLDB) today provided a clinical update on SGT-001 and reported that the U.S. Food and Drug Administration (FDA) has notified the company that IGNITE DMD, its Phase I/II study of SGT-001, has been placed on clinical hold. The company will hold a webcast conference call this morning to discuss this update.
To date, six patients have been dosed with SGT-001, Solid’s gene transfer candidate under investigation for Duchenne muscular dystrophy (DMD). This includes three patients in the first cohort at a 5E13 vg/kg dose, who continue to do well and are being followed per the study protocol. Three patients were subsequently dosed in the second cohort at a 2E14 vg/kg dose. The first two of these patients are also doing well and being followed per study protocol.
The third patient in the 2E14 vg/kg cohort, dosed in late October, experienced a serious adverse event (SAE) deemed related to the study drug that was characterized by complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency. Neither cytokine- nor coagulopathy-related abnormalities were observed. Currently the patient is being closely followed by his care team. He is recovering and continues to improve.
The company reported the event to the FDA and the study Data Safety Monitoring Board (DSMB). The FDA has notified the company that the study has been placed on clinical hold. Solid will work with the FDA in an effort to resolve the hold and determine next steps for IGNITE DMD. The company continues to plan to report additional biomarker data from the study before year end.
“We are encouraged that this patient is recovering. I would like to thank both the patient and his family for their participation in our study, as well as the team at the University of Florida for the excellent care they provide,” said Ilan Ganot, Chief Executive Officer, President and Co-Founder of Solid Biosciences. “We remain committed to bringing meaningful new therapies to the Duchenne community and continue to believe in the differentiated construct of SGT-001 and the potential benefits it may offer to patients. In the coming weeks, we anticipate that we will have a better understanding of the biological activity and potential benefit of SGT-001. We look forward to sharing this additional data and working with the FDA to resolve the clinical hold and determining next steps for the program.”
Conference Call Information
The company will host a conference call and webcast at 8:30 a.m. ET today to discuss the program update. Participants are invited to listen by dialing +1 866-763-0341 (domestic) or +1 703-871-3818 (international) five minutes prior to the start of the call and providing the passcode 2277849. A listen-only webcast of the conference call can also be accessed through the “Investors” tab on the Solid Biosciences website, www.solidbio.com, and a replay of the call will be available for approximately six weeks after the call.
Solid’s lead candidate, SGT-001, is a novel adeno-associated viral (AAV) vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of DMD, mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). Data from Solid’s preclinical program suggests that SGT-001 has the potential to slow or stop the progression of DMD, regardless of genetic mutation or disease stage.
SGT-001 is based on pioneering research in dystrophin biology by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, in the United States and Orphan Drug Designations in both the United States and European Union.