Solid Biosciences Rips Higher On Positive Biomarker Data For DMD Drug

Solid Biosciences Inc SLDB shares are advancing strongly Wednesday following on an update on its clinical development program for its SGT-001, its gene transfer investigational candidate for Duchenne muscular dystrophy, or DMD.

Biomarker Data Encouraging

Solid Biosciences said biomarker data from two patients dosed in the second cohort of the IGNITE DMD Phase 1/2 study indicated patients showed SGT-001 microdystrophin expression and associated neuronal nitric oxide synthase function. This, according to the company, suggested SGT-001 has the potential to produce therapeutic benefit for DMD patients.

DMD, caused by the absence of a protein called dystrophin that aids in keeping muscle cells intact, is a genetic disorder that causes progressive muscle weakness and degeneration, primarily in boys.

The company said the previously reported serious adverse event, or SAE, experienced by the third patient in the 2E14 vh/kg dose group has been fully resolved, allowing him to resume normal activities.

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Working Through Series Of Setbacks

The company announced Nov. 12 that the FDA placed the IGNITE DMD clinical trial on hold after a patient evaluated in the second cohort at a 2E14 vg/kg dose experienced a SAE deemed related to the study drug. The SAE manifested as complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency. The announcement led to a 74% plunge in the share price.

Incidentally, the study was placed on clinical hold in March 2018, citing a SAE, with the hold lifted in June of the same year. Subsequently, shares came under pressure in early February, when the company announced data from a Phase 1/2 dose-escalating study of SGT-001 that showed levels of microdystrophin expression.

In mid-May, shares tumbled yet again after the company said a patient in the treatment arm experienced a transient decline in platelet count, considered a SAE related to the study.

Reposing Faith In Therapy

Solid Biosciences said Wednesday it has received the FDA letter related to the second clinical hold imposed in November, and it will continue working internally, and with the FDA and other external experts to address the clinical hold and determine the path forward.

"We now have evidence that SGT-001 can lead to microdystrophin expression at levels that we believe are meaningful and warrant further clinical development," said Ilan Ganot, CEO of Solid Biosciences.

The race to market for DMD therapies is getting competitive. Sarepta Therapeutics Inc SRPT recently received FDA nod for its second DMD drug for patients with a confirmed mutation amenable to exon 53 skipping. The company is also evaluating a gene therapy candidate for DMD.

Solid Biosciences traded higher by 18% to $5.66 per share at time of publication.