Shares of Sio Gene Therapies (NASDAQ: SIOX) are rallying premarket on Wednesday after the company announced positive six-month follow-up data from its Phase 1/2 trial of AXO-AAV-GM1, its gene therapy candidate for the treatment of GM1 gangliosidosis.
The company said that the drug was generally well-tolerated with a favourable safety profile.
The trial saw five patients (children aged between 32 to 68 months) enrolled with all showing signs of disease stability six months after the treatment was administered.
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“We are excited to report encouraging safety, tolerability, biomarker, and preliminary efficacy data for AXO-AAV-GM1, the first gene therapy evaluated in a clinical trial for GM1 gangliosidosis, a life-limiting disease caused by mutations in the GLB1 gene that impair beta-galactosidase enzyme activity,” said Gavin Corcoran Chief R&D Officer at Sio.
“Safety was the key measure in this first-in-human study, and we are pleased to see a favorable safety profile in the first five children treated with the low-dose,” continued Corcoran.
Sio said the company will collect additional data at the 12-month evaluation including several measures of the systemic manifestations of GM1 gangliosidosis.
Sio’s stock price rallied after the news and is currently trading at $4.07 per share, up 65.45% premarket.