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FDA Again Keeps Solid Biosciences’ Duchenne Trial On Clinical Hold

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Solid Biosciences (SLDB) has announced that it has been notified by the U.S. Food and Drug Administration (FDA) that the company’s IGNITE DMD Phase I/II clinical trial remains on clinical hold.

In November 2019, the FDA placed IGNITE DMD on clinical hold as the result of a serious adverse event (SAE) that occurred in the sixth patient dosed in the trial; as previously reported the SAE has fully resolved.

In April 2020, Solid provided the FDA with information and measures intended to improve patient safety and in May 2020, Solid received written communication from the FDA that the trial remained on hold.

In June 2020, the company submitted a response to the FDA that provided data related to manufacturing process improvements. However the FDA has now responded by maintaining the clinical hold and requesting further manufacturing information, updated safety and efficacy data for all patients dosed, and providing direction on total viral load to be administered per patient.

Solid 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,” said Ilan Ganot, CEO of Solid Biosciences. “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.”

Solid’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.

Shares in Solid Biosciences have plunged 36% year-to-date, and analysts have a cautiously optimistic Moderate Buy take on the stock's outlook. That's with a $5.50 average analyst price target for upside potential of over 90%. (See SLDB stock analysis on TipRanks).

"We view SLDB as among the riskiest names our analyst covers, as it is a company we believe is more likely to fail to get its product to market than to succeed" comments Chardan Capital analyst Gbola Amusa.

"However, increases in the market's and our view of the potential for SGT-001 to succeed could lead to >5x or even >10x share price upside, e.g. if SLDB returns to even half its peak highs of >$50/sh, thus exposing positive risk-reward dynamics that merit our Buy rating for investors with the appropriate risk appetite" he explained. Amusa has a buy rating on the stock with a $5 price target.

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