BlueRock Therapeutics advances investigational cell therapy bemdaneprocel for treating Parkinson’s disease to registrational Phase III clinical trial

BlueRock Therapeutics is advancing its investigational cell therapy, bemdaneprocel, into a Phase 3 clinical trial named exPDite-2, set to start in early 2025. The trial will evaluate the safety and efficacy of bemdaneprocel in treating moderate Parkinson’s disease. This decision follows positive results from a Phase 1 trial (exPDite, NCT04802733), which showed that bemdaneprocel helped patients spend more time with symptoms under control and reduced "off" periods when symptoms reemerge as medication effects wear off. Patients from this trial could also join a five-year extension study (NCT05897957). Although a Phase 2 trial was initially planned for late 2024, the company is now accelerating development into Phase 3 with the U.S. Food and Drug Administration’s agreement on the exPDite-2 trial design. Bemdaneprocel is a cell therapy that uses human embryonic stem cells to generate dopamine-producing cell precursors. These cells are surgically transplanted into the brain to replace the dopamine-producing neurons lost in Parkinson’s disease. The therapy aims to restore motor function by forming new neural networks. The Phase 3 exPDite-2 trial will involve 102 patients with moderate Parkinson’s. It will compare bemdaneprocel to a sham surgery over 78 weeks (1.5 years) to measure changes in "on time"—periods when symptoms are well controlled without dyskinesia, a side effect of long-term levodopa use. Secondary goals include assessing safety, movement improvements, daily activity levels, and quality of life. The trial will use a double-blind, randomized design, ensuring neither patients nor researchers know who receives the therapy. “This marks a significant milestone as we investigate a potential new treatment option for people with Parkinson’s disease,” said Dr. Amit Rakhit, Chief Development and Medical Officer at BlueRock, a Bayer subsidiary. Christian Rommel, Bayer’s global head of research and development, emphasized the company’s commitment to bringing this therapy to patients more quickly. Parkinson’s disease results from the loss of dopamine-producing neurons, leading to motor symptoms like slowed movements, tremors, and stiffness. While levodopa is the standard treatment, its effects diminish over time, causing "off" periods. Bemdaneprocel aims to address this challenge by replacing lost neurons, potentially offering a new way to manage the disease.