Solid Biosciences Inc (NASDAQ: SLDB) shares pulled back to all-time lows Tuesday after the company said its lead gene therapy candidate to treat Duchenne muscular dystrophy has been placed on clinical hold by the FDA yet again.
FDA Seeks Study Suspension On Safety Scare
Solid Biosciences said it has been notified by the FDA that the IGNITE DMD Phase 1/2 study of SGT-001 has been placed on clinical hold.
SGT-001 is the company's investigational microdystrophin gene therapy for treating DMD.
A clinical hold is an order issued by the FDA to the sponsor of a trial to delay a proposed clinical investigation or to suspend an ongoing investigation.
Solid Biosciences said that of the three patients dosed in the second cohort at a 2E14 vg/kg dose, the third patient dosed in late October experienced a serious adverse event, or SAE, that was deemed to be related to the drug.
The SAE manifested as complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury and cardio-pulmonary insufficiency, according to Solid Biosciences.
After the company notified the FDA and the study Data Safety Monitoring Board, the regulatory agency placed the program on hold.
The trial suffered a setback back in March 2018 when the FDA imposed a clinical hold after a patient dosed in the trial had to be hospitalized due to laboratory findings of a decrease in platelet count and a reduction in RBC count as well as evidence of complement activation.
The FDA subsequently lifted the hold in June 2018, satisfied with the remedial measures undertaken by the company.
Solid Biosciences had made changes to the trial protocol related to the lifting of the clinical hold by adding intravenous glucocorticoids in the initial weeks after administration of SGT-001, as well as enhanced monitoring measures.
SGT-001 is Solid Biosciences' only pipeline asset in human trials.
The stock was tanking 68.55% to $3.46 at the time of publication Tuesday.
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