Sio Gene Therapies Stock Jumps After FDA Granted Fast Track Designation for Investigational Gene Therapy in Patients with GM1 Gangliosidosis

Oct 21, 2021 By MarketDepth

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FDA Approved

Sio Gene Therapies Inc. (NASDAQ: SIOX) shares jumped more than 20% on Thursday after the clinical-stage company announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for the treatment of Type I (early infantile-onset) and Type II (late infantile-onset and juvenile-onset) GM1 gangliosidosis. The Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

“Receiving Fast Track Designation is a critical step in our mission to develop the first potential treatment for all pediatric forms of this rare, terminal disease. This designation joins both the Orphan Drug Designation and Rare Pediatric Disease Designation assigned to AXO-AAV-GM1 by the FDA, which we believe further demonstrates the potential impact of this work on the patient community. Building on the recently presented data at ESGCT demonstrating normalization of key disease biomarkers in the high-dose cohort with no serious adverse events attributed to AXO-AAV-GM1, this designation will help us accelerate clinical development of this promising investigational therapy for children and families.”

Pavan Cheruvu, M.D., Chief Executive Officer of Sio Gene Therapies

Sio Gene Therapies focuses on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases. It has a license agreement with Oxford BioMedica (UK) Ltd. to develop and commercialize AXO-Lenti-PD and related gene therapy products; and The University of Massachusetts Medical School to develop and commercialize gene therapy product candidates, including AXO-AAV-GM1 and AXO-AAV-GM.

Safety Being Evaluated

The current Phase 1/2 study (NCT03952637) is designed to evaluate the safety, tolerability, and potential efficacy of AXO-AAV-GM1 gene therapy delivered intravenously in children with early infantile, or Type I, and late infantile and juvenile, or Type II, GM1 gangliosidosis. Stage 1 of the study is a dose-escalation study in which the low-dose cohort is evaluating 1.5×1013 vg/kg and the high-dose cohort is evaluating a dose of 4.5×1013 vg/kg. Stage 2 of the trial will then evaluate the efficacy and safety of the optimal dose identified in Stage 1.