Every patient deserves long-lasting and transformative therapies. Throughout its existence, Sio Gene Therapies Inc. (NASDAQ: SIOX) has had one primary focus; to enhance the development of treatments for debilitating neurodegenerative diseases. Today, the company, formerly Axovant, prides itself in extensive collaborations with leading gene therapy institutions built from decades of research.
SIOX boasts a pipeline of treatments, the recent addition being AXO-AAV-GM1. The viral vector (AAV)9-based gene therapy on its Phase 1/2 study is a potential candidate for the treatment of GM1 gangliosidosis. In addition, the company says it has dosed the first Type I, or early infantile, patient in the low-dose cohort. This is one of the significant milestones for this candidate, according to Chief R&D Officer of Sio, Gavin Corcoran.
He explained, “Dosing the first Type I patient represents…the efforts by both Sio and our trial partners at the NIH are the culmination of our dedication to patients.”
Analyzing the Study and Possible Success
The clinical study (NCT03952637) has nine patients across low-dose and high-dose cohorts. It aims to evaluate the tolerability, safety, and potential efficacy of AXO-AAV-GM1. The Low-dose cohort is evaluating 1.5×1013 VG/kg, while the high-dose cohort is evaluating a dose of 4.5×1013 vg/kg in both disease subtypes. Corcoran says they are hopeful of positive 12-month clinical outcome data from the Type II low-dose cohort in October 2021. They are also expectant of meeting with the FDA in the first half of 2022 and possibly conclude about the registrational pathway in GM1 gangliosidosis
Several diagnoses of terminal illnesses do not have available treatments. Hence, approval of the AXO-AAV-GM1 by the FDA will be a step closer to the first approved gene therapy.
Meanwhile, Pavan Cheruvu, the CEO of SIOX, affirms that the team’s deep expertise and commitment have birthed a strong balance sheet. This gives the company an upper hand in its efforts to execute and advance in its pipeline of transformative gene therapies.