Voyager Therapeutics will soon initiate a global Phase 2/3 clinical trial program to evaluate its VY-AADC gene therapy for the treatment of advanced Parkinson’s disease.
The trial follows approval by the U.S. Food and Drug Administration of the company’s investigational new drug (IND) application for VY-AADC.
“Following institutional review board approval and patient screening at clinical referral and surgical sites, we continue to plan to dose the first patient in our pivotal program during the second quarter of this year, representing a very important milestone for both the program and the company,” Bernard Ravina, chief medical officer at Voyager Therapeutics, said in a press release.
The IND application included information demonstrating that Voyager’s baculovirus/Sf9 therapy manufacturing process is comparable, and not inferior, to the standard production system based on mammalian cells. The new baculovirus manufacturing process uses insect-derived cells, which allows for the production of greater amounts of VY-AADC with a reduced risk of introducing mammalian cell-derived impurities.
“Our baculovirus manufacturing process is designed for production of AAV [adeno-associated virus] vectors at clinical and commercial scale, with the potential for increased yields and efficient scalability compared with mammalian-based systems,” Ravina said. “We are pleased to initiate our pivotal program and begin dosing patients with the baculovirus-produced vector.”
VY-AADC is a therapy designed to deliver the AADC gene directly to brain cells in the putamen region. This enzyme is responsible for converting the standard of care treatment levodopa into dopamine, the signaling molecule that is missing in Parkinson’s patients.
The approach is expected to bypass the effects of degenerating dopamine neurons in the substantia nigra, a part of the midbrain, by increasing dopamine levels in the putamen.
Preliminary data have shown that after a single administration, the therapy induced a durable, dose-dependent, and time-dependent response across multiple measures of motor function. The treatment also effectively increased AADC enzyme activity and enhanced the response of levodopa. To date, no serious adverse events have been reported due to VY-AADC.
Voyager is recruiting participants for the Phase 1 trial, which will be testing a new VY-AADC delivery method, and expects to provide an update from these studies during the first quarter of 2018.