A DMD gene therapy has been placed on clinical hold

Following the occurrence of a safety incident, the FDA has placed on hold the clinical trial for SGT-001, the Solid’s gene therapy candidate for Duchenne muscular dystrophy (DMD). This clinical hold is the second bad news the DMD community has received this month. Last week, Swiss pharma giant Roche announced it was terminating its study of an investigational anti-myostatin adnectin protein in ambulatory boys with DMD. Roche said an analysis of the ongoing data indicated that its treatment RG6206 was “highly unlikely” to demonstrate clinical benefit in the trial.

 

La Force is sharing this press release provided by Solid Biosciences, Nov. 12, 2019,> PRESS RELEASE <

 

Solid Biosciences Provides SGT-001 Program Update

Solid Biosciences Inc. provided a clinical update on SGT-001, a microdystrophin gene transfer therapy, and reported that the U.S. FDA had notified the company that IGNITE DMD, its Phase I/II study of SGT-001, has been placed on clinical hold. 

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, who continue to do well and are being followed per the study protocol. Three patients were subsequently dosed in the second cohort. The first two of these patients are also doing well and being followed per study protocol.

The third patient in another 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 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 the next steps for IGNITE DMD. The company continues to plan to report additional biomarker data from the study before the year-end.

 

Ilan Ganot, Chief Executive Officer, President and Co-Founder of Solid Biosciences – “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. 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.”

 

Last year, the FDA placed a clinical hold on the trial following the report of a serious adverse event. Solid Biosciences Announces Clinical Hold On SGT-001 microdystrophin gene transfer Clinical Phase I/II Clinical Trial for Duchenne Muscular Dystrophy.  That hold was lifted in June 2018 after the company addressed the FDA’s concerns.

About SGT-001

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.

Learn more here

In case you don’t remember the specifics about the microdystrophin and gene therapy we invite you to watch the interview we conducted in London with Dr. Jeffrey Chamberlain PH.D.:  Here

About Solid Biosciences

Solid Biosciences is a life science company focused solely on finding meaningful therapies for Duchenne muscular dystrophy (DMD). Founded by those touched by the disease, Solid is a center of excellence for DMD, bringing together experts in science, technology and care to drive forward a portfolio of candidates that have life-changing potential. Currently, Solid is progressing programs across four scientific platforms: Corrective Therapies, Disease-Modifying Therapies, Disease Understanding and Assistive Devices. For more information, please visit www.solidbio.com.

 

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