BlueRock Therapeutics, a Bayer subsidiary, has begun its pivotal phase III trial (exPDite-2) of bemdaneprocel, an investigational cell therapy for Parkinson’s disease (PD).
This is the first phase III trial for an allogeneic pluripotent stem cell-derived therapy in PD.
Why It Matters
Parkinson’s disease affects over 10 million people worldwide. It is the second most prevalent neurodegenerative disease and the most common movement disorder. Current therapies manage symptoms but do not alter disease progression.
About the Trial
- Design: Multicenter, randomized, double-blind, sham-controlled.
- Enrollment: ~102 patients with Parkinson’s disease.
- Primary endpoint: Change from baseline to week 78 in “ON-time” without troublesome dyskinesia (16-hour waking day).
- Secondary endpoints: Movement measures, non-motor symptoms, safety, quality of life.
In phase I, bemdaneprocel showed good tolerability and encouraging trends in motor improvement at 24 months. No serious adverse events related to the therapy were reported.
How Bemdaneprocel Works
Bemdaneprocel (BRT-DA01) is designed to replace dopamine-producing neurons lost in PD.
- Derived from human embryonic pluripotent stem cells.
- Implanted surgically into the brain.
- Mature into dopamine neurons.
- Potential to re-form neural networks and restore motor and non-motor function.
The FDA has granted Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations.
Leadership Voices
“People with Parkinson’s urgently need therapies that alter the disease course,”
- Amit Rakhit, MD, MBA, Chief Development & Medical Officer at BlueRock.
“This milestone advances our commitment to transform treatment through innovative therapies,”
- Christian Rommel, PhD, Head of R&D, Bayer Pharmaceuticals.
The Bigger Picture
If successful, the exPDite-2 trial could provide data for regulatory submissions and move PD care toward disease-modifying therapy.
