Solid Biosciences Inc. (NASDAQ: SLDB) shares jumped on Wednesday after the firm provided an update from its midstage study in Duchenne muscular dystrophy (DMD). Specifically, the data are coming from the Ignite DMD Phase 1/2 study of SGT-001.
The data from these patients showed SGT-001 microdystrophin expression and associated neuronal nitric oxide synthase function, providing evidence that SGT-001 has the potential to result in therapeutic benefit for patients with DMD.
The company also announced that the previously reported serious adverse event experienced by the third patient in the 2E14 vg/kg dose group has fully resolved, and the patient has resumed his normal activities.
Solid Biosciences has received the clinical hold letter from the U.S. Food and Drug Administration (FDA) and will continue working internally, and with the FDA and other external experts, to address the clinical hold and determine the path forward.
Ilan Ganot, CEO, president and co-founder of Solid Biosciences, commented:
We now have evidence that SGT-001 can lead to microdystrophin expression at levels that we believe are meaningful and warrant further clinical development. I’m also pleased to say that the patient who experienced the event announced in November is doing well. We are steadfast in our commitment to bringing a transformative and safe therapy to the Duchenne community and are working diligently to resolve the clinical hold and resume dosing with SGT-001.
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Shares of Solid Biosciences were last seen down about 8% at $4.40, in a 52-week range of $2.75 to $32.85. The consensus price target is $7.63.
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