BlueRock Therapeutics has dosed the first patient in its pivotal phase 3 trial of bemdaneprocel, an investigational cell therapy for Parkinson’s disease. The exPDite-2 study is the first phase 3 trial of an allogeneic pluripotent stem cell derived therapy for the condition.
The multicentre, double-blind trial will enrol around 102 participants. It compares bemdaneprocel with sham surgery control and will assess efficacy, safety and overall impact. The primary endpoint is change in ON-time without troublesome dyskinesia at week 78, adjusted for a 16-hour waking day.
Secondary endpoints will evaluate movement, non-motor symptoms, safety and tolerability, alongside measures of daily living and quality of life. The trial builds on phase 1 data showing good tolerability and encouraging trends in motor function at 24 months post-surgery.
Amit Rakhit MD, MBA, Chief Development and Medical Officer at BlueRock, said: “The initiation of the exPDite-2 trial represents a major step forward toward advancing bemdaneprocel and we are excited to build on the momentum of our earlier data.”
Christian Rommel PhD, Head of Research and Development at Bayer’s Pharmaceuticals Division, added: “Bemdaneprocel aims to sustainably restore lost physiologic function in the dopaminergic system impacted by the disease, ultimately to enhance the quality of life for patients.”
Depending on results, the trial could support future regulatory submissions for marketing authorisation. Bemdaneprocel has received Fast Track and RMAT designation from the US FDA but is not yet approved for any condition.
BlueRock, a subsidiary of Bayer AG, is focused on developing cellular medicines for neurological and ophthalmic diseases. Its pipeline includes bemdaneprocel for Parkinson’s and OpCT-001 for photoreceptor disorders.
