BlueRock's Parkinson's Therapy in Phase III
Bayer AG and BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, announced plans to initiate a Phase III clinical trial for bemdaneprocel, its investigational cell therapy for Parkinson’s disease
The registrational trial, named exPDite-2, is expected to begin in the first half of 2025 and will represent a significant milestone in the development of allogeneic cell-based therapies for neurodegenerative disorders.
In a Phase I study with 12 participants, bemdaneprocel demonstrated tolerability, with no serious adverse events related to drug product at 24 months post-surgery. Further, encouraging trends were observed in secondary endpoints related to motor impairments at 24 months post-surgery. Building on these results, exPDite-2 is a double-blind trial that will assess the efficacy, safety and overall impact of bemdaneprocel compared to a sham surgery control. The trial is designed to enroll approximately 102 participants with moderate Parkinson’s disease. The primary endpoint of the study is change from baseline to week 78 in PD diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day. In addition, the trial will incorporate secondary endpoints designed to capture objective measures of movement, safety and tolerability, and instruments that capture activities of daily living and quality of life.
The advancement to this registrational trial follows discussions with the FDA under the Regenerative Medicine Advanced Therapy (RMAT) designation granted by the FDA in May 2024. Depending upon the outcome, the results from this trial are intended to be part of a robust data package to support regulatory submissions for marketing authorization.
“With the planned initiation of the Phase III clinical trial, we are committed to bringing bemdaneprocel faster to patients in need,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division, and Global Head of Research and Development. “It represents a significant milestone in our efforts to advance our cell and gene therapy pipeline and deliver on our ambition to be an industry leader in this space.”
"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 Parkinson’s disease community as we initiate this trial.” said Dr. Amit Rakhit, Chief Development and Medical Officer at BlueRock Therapeutics.
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