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Solid Biosciences Provides Update regarding SGT-001 Phase I/II Clinical Hold on IGNITE DMD
CAMBRIDGE, Mass., May 07, 2020 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies
About this update from Solid Biosciences Inc.
[{"type":"text","content":"CAMBRIDGE, Mass., May 07, 2020 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy, announced that it received written communication from the U.S. Food and Drug Administration (FDA) regarding the clinical hold placed on the Company’s IGNITE DMD Phase I/II clinical trial. The program remains on clinical hold and the Company will continue to work with the FDA to address their requests. \n In April 2020, the Company submitted a response to the FDA that included changes to the clinical protocol designed to enhance patient safety, as well as information related to improvements to its manufacturing process. The FDA has responded by maintaining the clinical hold and requesting further data and analyses relating to this manufacturing process. The Company is in the process of generating these data and expects to submit this information to the FDA before the end of the third quarter of 2020. “We share the FDA’s commitment to patient safety and are working collaboratively with the agency to resolve the clinical hold,” said Ilan Ganot, Chief Executive Officer, President and Co-Founder of Solid Biosciences. “We consider patient safety the utmost priority and believe the clinical development of SGT-001 could offer meaningful benefits to patients with this devastating disease.” In addition, Solid Biosciences today filed its Quarterly Report on Form 10-Q for the quarter ended March 31, 2020 with the Securities and Exchange Commission. About SGT-001Solid’s SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy (Duchenne). Duchenne is caused by 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 Duchenne, regardless of genetic mutation or disease stage. SGT-001 is ba...