Axovant’s gene therapy yields favourable data in Parkinson’s trial

June 10, 2019

AXO-Lenti-PD is an investigational gene therapy being developed for the treatment of Parkinson’s disease. Credit: Colin Behrens from Pixabay.

Axovant Gene Therapies has reported six-month follow-up results from the first cohort of the ongoing Phase II SUNRISE-PD clinical trial investigating AXO-Lenti-PD in Parkinson’s disease patients.

AXO-Lenti-PD is an investigational gene therapy that delivers the tyrosine hydroxylase, cyclohydrolase 1 and aromatic L-amino acid decarboxylase genes through a single lentiviral vector.

It encodes a set of enzymes necessary for dopamine synthesis in order to minimise variability and restore steady dopamine levels in the brain.

In the open-label, dose-escalation part of the study, the gene therapy was generally well-tolerated, without any serious adverse events associated with the product or the procedure.

Participants also sustained improvement from baseline across various measurements.

According to the six-month data, patients demonstrated an average improvement of 17 points or 29% in Unified Parkinson’s Disease Rating Scale (UPDRS) Part III (motor) OFF score from baseline.

Average improvement from baseline in the UPDRS Part II (activities of daily living) OFF score was around 20 points, while the UPDRS Part IV (complications of therapy) OFF score improved by three points.

The average levodopa equivalent daily dose (LEDD) showed a decrease of 21% from baseline, and the Rush Dyskinesia Rating Scale had a mean improvement of 18% in functional disability during daily living activities while on oral levodopa.

Axovant Gene Therapies chief research and development officer Dr Gavin Corcoran said: “Our patient-focussed goal of improving motor function, reducing dyskinesia, lowering the requirement for oral levodopa, and improving quality of life is made possible by the continuous dopamine replacement strategy of AXO-Lenti-PD gene therapy.

“These data at six months highlight the potential for a clinically meaningful improvement over the currently available standard of care for those patients with moderate to advanced Parkinson’s disease.”

Dosing in the second cohort of the SUNRISE-PD trial was commenced in April and initial three-month data is expected to be available in the fourth quarter of the year.

https://www.drugdevelopment-technology.com/news/axovant-parkinsons-gene-therapy-trial/