Solid biosciences provides sgt-001 program update

Solid biosciences inc. provided a clinical update on sgt-001 and reported that the u.s. food and drug administration (fda) has notified the company that ignite dmd, its phase i/ii study of sgt-001, has been placed on clinical hold. the company will hold a webcast conference call this morning to discuss this update. to date, six patients have been dosed with sgt-001, solid’s gene transfer candidate under investigation for duchenne muscular dystrophy (dmd). this includes three patients in the first cohort at a 5e13 vg/kg dose, who continue to do well and are being followed per the study protocol. three patients were subsequently dosed in the second cohort at a 2e14 vg/kg dose. the first two of these patients are also doing well and being followed per study protocol. the third patient in the 2e14 vg/kg cohort, dosed in late october, experienced a serious adverse event (sae) deemed related to the study drug that was characterized by complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency. neither cytokine- nor coagulopathy-related abnormalities were observed. currently the patient is being closely followed by his care team. he is recovering and continues to improve. the company reported the event to the fda and the study data safety monitoring board (dsmb). the fda has notified the company that the study has been placed on clinical hold. solid will work with the fda in an effort to resolve the hold and determine next steps for ignite dmd. the company continues to plan to report additional biomarker data from the study before year end.
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