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Solid Biosciences Surges on DMD Gene Therapy Study Amendments

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Shares of Solid Biosciences Inc. (SLDB - Free Report) have gained 70.4% since Aug 14 as investors cheered the amendments made to a gene therapy study protocol evaluating its lead candidate, SGT-001 as a potential treatment for Duchenne muscular dystrophy (“DMD”). The company announced the update in its second-quarter earnings release.

The company is evaluating SGT-001, a novel adeno-associated viral (AAV) vector-mediated gene therapy, in a phase I/II study – IGNITE DMD – for treating DMD by increasing level of dystrophin protein in patients. Per the amended protocol, the company has limited new patient selection up to a weight of 25kg. The company has also removed the matched patient control arm in the second cohort of the study. These changes are expected to expedite the data readout from the IGNITE DMD study. Faster progress of SGT-001 is necessary for growth of Solid Biosciences as several companies are developing gene therapies for treating DMD with Sarepta Therapeutics (SRPT - Free Report) leading the space.

The company also stated that it has initiated dosing of the second patient in the higher dose (2E14 vg/kg) cohort in the IGNITE DMD study. It anticipates to announce updated data from the study later this year.

Meanwhile, Solid Biosciences’ shares have declined 67.1% so far this year compared with the industry’s decrease of 2.5%.

The development of SGT-001 for DMD has faced multiple hurdles so far this year. In February, the company announced preliminary data from IGNITE DMD study, which showed that lower dose (5E13 vg/kg) of SGT-001 was unable to achieve expected level of dystrophin protein in patients. Based on this result, the company expedited dose escalation activities to evaluate the higher dose in patients. In March 2018, one patient receiving the lower dose of SGT-001 was hospitalized following an adverse event, which led the FDA to put the study on clinical hold. Investors remain skeptical of the dose escalation as higher dose of the candidate can result in more severe adverse events. However, the company has not reported any such adverse event from the higher as well the lower dose cohort since June 2018. The clinical hold on the study was removed in June 2018.

Meanwhile, Solid Biosciences also announced in its earnings release that it has raised $60 million from a private placement. The company expects its cash resources as of Jun 30, 2019 along with the raised fund to be able to support the company’s operations into the fourth quarter of 2020 from the previously expected timeline of first quarter of 2020.

The company is developing SGT-001 in several early-stage studies for indications other than DMD. Please note that the gene therapy space is getting crowded with several pharma/biotech companies developing their pipeline, targeting multiple indications. Apart from Solid Biosciences and Sarepta, companies developing gene therapy for DMD include Pfizer (PFE - Free Report) , Audentes Therapeutics (BOLD - Free Report) , Wave Life Sciences and others.

Zacks Rank

Solid Biosciences currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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