Why Solid Biosciences Is Under Fire From the FDA

By Chris Lange Updated Jan 12, 2:24AM EST · Published Mar 15, 10:25AM EDT

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Solid Biosciences Inc. (NASDAQ: SLDB) watched its shares get crushed on Thursday after the company announced some complications with the U.S. Food and Drug Administration (FDA). Specifically, the FDA notified the firm that its Phase 1/2 clinical trial for SGT-001 microdystrophin gene transfer in Duchenne muscular dystrophy (DMD) has been placed on clinical hold.

The study was designed to assess the safety and efficacy of SGT-001 in ambulatory and non-ambulatory children and adolescents with DMD. The first patient dosed in the clinical trial was a non-ambulatory adolescent who received SGT-001 on February 14, 2018. Several days after administration the patient was hospitalized due to laboratory findings that included a decrease in platelet count followed by a reduction in red blood cell count and evidence of complement activation.

At that time, the patient showed no signs or symptoms of coagulopathy (bleeding disorder) and no relevant changes from baseline in liver function tests. The patient responded well to medical treatment and is currently asymptomatic. All laboratory parameters have either improved or returned to normal, and he is continuing outpatient assessments per protocol.

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Solid Biosciences reported the event to the FDA and, because it was unexpected, classified it as a Suspected Unexpected Serious Adverse Reaction (SUSAR). The FDA informed the company that the clinical hold was due to the event.

The company has halted enrollment and dosing in the Ignite DMD study and is awaiting the formal Clinical Hold letter from the FDA to understand the requirements for resuming the clinical trial.

Shares of Solid Biosciences were last seen down more than 63% at $9.68, with a consensus analyst price target of $37.40 and a new 52-week range of $9.52 to $33.74.

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About the Author Chris Lange →

Chris Lange is a writer for 24/7 Wall St., based in Houston. He has covered financial markets over the past decade with an emphasis on healthcare, tech, and IPOs. During this time, he has published thousands of articles with insightful analysis across these complex fields. Currently, Lange's focus is on military and geopolitical topics.

Lange's work has been quoted or mentioned in Forbes, The New York Times, Business Insider, USA Today, MSN, Yahoo, The Verge, Vice, The Intelligencer, Quartz, Nasdaq, The Motley Fool, Fox Business, International Business Times, The Street, Seeking Alpha, Barron’s, Benzinga, and many other major publications.

A graduate of Southwestern University in Georgetown, Texas, Lange majored in business with a particular focus on investments. He has previous experience in the banking industry and startups.