Axovant has reported positive interim results from the first cohort of patients in the ongoing SUNRISE-PD Phase II clinical trial of its gene therapy AXO-Lenti-PD, which is being evaluated for Parkinson’s disease treatment.
According to the data, one-time administration of the lowest dose of the gene therapy was generally well-tolerated without any reports of serious adverse events.
Patients also achieved an average Unified Parkinson’s Disease Rating Scale (UPDRS) Part III score of 25 points at three months post-treatment, indicating an average improvement of 42% from baseline.
UPDRS Part III score is a physician-rated scale evaluating motor function and low scores represent improvement.
Patients also experienced a mean 18% improvement in dyskinesia, while the average levodopa equivalent daily dose (LEDD) was decreased by 208mg at month three.
Based on these findings, Axovant plans to advance to the planned second dose cohort of AXO-Lenti-PD.
AXO-Lenti-PD is an investigational therapy designed to deliver tyrosine hydroxylase, cyclohydrolase 1 and aromatic L-amino acid decarboxylase genes through a single lentiviral vector.
The therapy is intended to encode a set of key enzymes involved in dopamine synthesis in order to reduce variability and restore stable dopamine levels in the brain.
Axovant Research and Development executive vice-president Gavin Corcoran said: “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.
“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.”
The company also reported positive three-month data from an investigator-initiated study of its AXO-AAV-GM2 gene therapy in infantile Tay-Sachs disease, a paediatric neurodegenerative genetic disorder.
AXO-AAV-GM2 was found to be generally well-tolerated and no serious adverse events have been reported. No clinically relevant laboratory abnormalities were observed following treatment with the gene therapy.