Bayer AG and BlueRock Therapeutics have released new details on the Phase 3 exPDite-2 trial, which will evaluate the efficacy and safety of bemdaneprocel (BRT-DA01), an investigational stem cell-based therapy for Parkinson’s disease (PD). The study, set to begin in the first half of 2025, will be the first Phase 3 trial of an allogeneic pluripotent stem cell therapy in PD.
The randomized, double-blind, sham surgery-controlled trial will include 102 patients with moderate PD. The primary endpoint will measure changes in ON time without troublesome dyskinesia, adjusted for a 16-hour waking day over 78 weeks. This design builds on Phase 1 data, which showed a 21.9-point reduction in Unified Parkinson’s Disease Rating Scale (UPDRS) Part III scores for high-dose recipients.
Bemdaneprocel involves surgical implantation of neuron precursors into the brain, to rebuild lost neural networks and restore motor function. Prior findings from the exPDite Phase 1 trial indicated that the transplanted cells survived and engrafted after discontinuation of immunosuppressive therapy at 12 months.
The therapy has received Fast Track and Regenerative Medicine Advanced Therapy designations from the FDA.
According to Dr. Amit Rakhit, Chief Development and Medical Officer at BlueRock, “exPDite-2 is the first registrational Phase III clinical trial for an investigational pluripotent stem cell-derived therapy in Parkinson’s disease, and we look forward to working closely with clinical investigators and the PD community as we initiate this trial.”
Data from exPDite-2 may contribute to future regulatory submissions, potentially bringing a new stem cell therapy treatment to patients with PD.
