The IGNITE DMD study is assessing the company’s microdystrophin gene therapy asset SGT-001 as a potential treatment for DMD.
Shares of Solid Biosciences were down nearly 9% in premarket trading after the company announced late Friday that the U.S. Food and Drug Administration (FDA) has not yet lifted a clinical hold on its Phase I/II IGNITE Duchenne muscular dystrophy study.
The IGNITE DMD study is assessing the company’s microdystrophin gene therapy asset SGT-001 as a potential treatment for DMD, the most common and severe form of muscular dystrophy that primarily affects boys. The trial was placed on hold in November due to safety concerns. A patient experience a serious adverse event that was deemed to be related to SGT-001, a novel adeno-associated viral vector-mediated gene transfer. The patient had recovered from the event, but the clinical hold remains in place.
Before the clinical hold was placed in November, Solid Biosciences reported a previous adverse events-related issue in the IGNITE DMD trial in a quarterly financial report. Solid Biosciences reported that a patient treated with SGT-001 was diagnosed with a gastrointestinal infection. While it was believed that the issue was not related to the drug, it was classified as a serious adverse event.
In its announcement Friday, Solid Biosciences related a series of attempts to address the clinical hold over the past several months, including new information and measures to improve patient safety, as well as updated manufacturing process improvements. Solid Biosciences said the FDA’s latest response to the company not only maintains the clinical hold, but also requests further manufacturing information, updated safety and efficacy data for all patients dosed, and additional direction on total viral load to be administered per patient. The company said it is evaluating next steps and will provide an update as appropriate.
“Patient safety is our highest priority and we plan to continue our dialogue with the FDA to determine the appropriate path for SGT-001,” Ilan Ganot, co-founder, president and chief executive officer of Solid Biosciences said in a statement. “We are disappointed with the outcome today, however, we will continue to work with the FDA as we believe the clinical development of SGT-001 could offer meaningful benefits to patients with this devastating disease.”
The development of SGT-001 has been difficult. The clinical hold slapped on the company in November was the second for the DMD treatment. In 2018, the FDA placed a clinical hold on the trial following the report of a serious adverse event. That hold was lifted in June 2018 after the company addressed the FDA’s concerns.
DMD causes a progressive loss of muscle strength attributable to a loss of a protein called dystrophin, which normally protects muscle fibers from breaking down. Approximately 15,000 U.S. patients are affected with Duchenne, with a total of 300,000 patients worldwide. Solid Biosciences’ gene therapy drug SGT-001 is designed to deliver a synthetic dystrophin gene, called microdystrophin, to the body of DMD patients.
