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.
“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.
