Axovant Releases Positive Interim Updates for Parkinson's and Tay-Sachs Gene Therapy Trials

Axovant has been busy, releasing interim results from two different clinical trials, one for Tay-Sachs, the other for Parkinson’s disease. For Parkinson’s disease, Axovant released positive interim results from the first cohort of its SUNRISE-PD Phase II clinical trial of AXO-Lenti-PD. AXO-Lenti-PD is a gene therapy that delivers three genes, tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase, by way of a single lentiviral vector. These genes code for a group of enzymes required for dopamine synthesis. The cohort involved two patients with advanced Parkinson’s disease. They both received a single administration of the lowest dose of the therapy. It was well-tolerated, and no serious adverse events were observed. At three months after receiving the therapy, improvements of an average of 55 percent from baseline were observed, based on the Unified Parkinson’s Disease Rating Scale (UPDRS) Total OFF score. “Our focus in this first cohort of the SUNRISE-PD study was on the safety and tolerability of AXO-Lenti-PD, as well as the evaluation of efficacy using well-validated, objective measures,” stated Gavin Corcoran, Axovant’s executive vice president of Research and Development. “These early data support the safety of the lowest dose of AXO-Lenti-PD, similar to what was observed with the earlier generation construct, ProSavin, and also suggest substantially greater biological activity than the highest dose of ProSavin previously tested. These findings are highly encouraging, and we look forward to advancing to higher dose cohorts where we will explore the full clinical potential of AXO-Lenti-PD in patients with Parkinson’s disease.”