Axovant Gene Therapies Ltd (NASDAQ:AXGT) has reported its 6-month to follow-up data from the second group of patients who received a total dose of 1.4 x 107 TU of gene therapy in the SUNRISE-PD phase 2 study. The trial is an open-label dose-escalation study evaluating AXO-Lenti-PD in treating Parkinson’s Disease.
AXO-Lenti PD shows improved quality of life in PD patients
Gavin Corcoran, the Chief R & D Officer of the company, said that the data released shows more than 20 points of UPDRS Part II ‘OFF’ motor function score improvement and a significant quality of life improvement. This underpins AXO-Lenti-PD as a potentially best in class gene therapy in the management of Parkinson’s disease. Corcoran further indicated that the data’s totality reinforces the belief the AXO-Lenti-PD can potentially be a transformational Parkinson’s disease treatment as one-time gene therapy.
Based on the promising results, the company expects to commence dosing in the randomized Sham controlled EXPLORE-PD study in 2021. Corcoran said that the company will additionally evaluate the tolerability and safety of higher infusion volumes.
AXO-Lenti-PD gene therapy well tolerated
At 6 months, AXO-Lenti-PD showed favorable tolerability in 4 patients that were receiving the gene therapy. The 4 patients averaged 57 years having had Parkinson’s disease for around 13 years, and there were no adverse events related to the vector at 6 months after administration. There was a 21 point mean UPDRS Part III ‘OFF’ score improvement in 2 patients in Cohort 2. This represents a 40% improvement in the average motor function baseline score. Two of the four patients in the UK trial sites didn’t take part in the UPDRS assessment because of COVID-19 and the compulsory washout of levodopa therapy at 6 months.
Following the completion of the Cohort, Axovant expects to commence a safety and tolerability study if a higher volume and flow rate. This will enhance putaminal coverage and reduce cooperating room time in cohort 3. Following successful suspension-based manufacturing process development, the company anticipates the dosing of the first patients in the EXPLORE-PD study in 2021.