Is This DMD Study Sunk After FDA Update?

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Solid Biosciences Inc. (NASDAQ: SLDB) shares were absolutely crushed on Tuesday after the firm announced that the U.S. Food and Drug Administration (FDA) has placed its Ignite DMD Phase 1/2 study of SGT-001 on hold.

So far, six patients have been dosed with SGT-001, Solid Bio’s gene transfer candidate under investigation for Duchenne muscular dystrophy (DMD).

This includes three patients in the first cohort, who continue to do well and are being followed per the study protocol. Three patients were subsequently dosed in the second cohort. The first two of these patients are also doing well and being followed per study protocol. However, the third patient in the second cohort experienced a serious adverse event deemed related to the study.

The company reported the event to the FDA and the study Data Safety Monitoring Board. The FDA has notified the company that the study has been placed on clinical hold. As a result, Solid Bio will work with the FDA in an effort to resolve the hold and determine next steps for Ignite DMD.

Ilan Ganot, CEO, president and co-founder of Solid Bio, commented:

We are encouraged that this patient is recovering. I would like to thank both the patient and his family for their participation in our study, as well as the team at the University of Florida for the excellent care they provide. We remain committed to bringing meaningful new therapies to the Duchenne community and continue to believe in the differentiated construct of SGT-001 and the potential benefits it may offer to patients. In the coming weeks, we anticipate that we will have a better understanding of the biological activity and potential benefit of SGT-001. We look forward to sharing this additional data and working with the FDA to resolve the clinical hold and determining next steps for the program.

Shares of Solid Bio traded down about 70% on Tuesday, at $3.33 in a 52-week range of $3.15 to $37.50. The consensus price target is $12.38.


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