Parkinson Disease Neuronal Cell Therapy Demonstrates Safety, Cell Survival

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Detailed topline data will be presented at the 2023 International Congress on Parkinson’s Disease and Movement Disorders in August.

BlueRock Therapeutics has announced that its phase 1/2 clinical trial (NCT04802733) of bemdaneprocel (BRT-DA01) cell therapy has yielded positive topline results in treated participants with Parkinson disease (PD).

“We are on a mission to harness the power of cell therapy with the aim to help people with PD regain control of their lives by restoring the functions that they have lost to this disease,” Ahmed Enayetallah, Senior Vice President and Head of Development, BlueRock Therapeutics, said in a statement. “The safety profile of bemdaneprocel was encouraging along with early evidence of cell survival and engraftment, marking a very important step in the development of a potential new therapy for patients with this disease. These topline data provide a strong rationale for initiating the next phase study, and we look forward to advancing this clinical program.”

The detailed data will be presented at the 2023 International Congress on Parkinson’s Disease and Movement Disorders taking place in Copenhagen from August 27-31, but BlueRock has so far reported that bemdaneprocel was well-tolerated in all 12 patients treated to date and no serious adverse events have been observed, meeting the study’s primary endpoints. The study’s secondary endpoints, exploring feasibility of transplantation and evidence of cell survival, were also met through one year of follow-up. BlueRock is planning for a phase 2 study expected to begin enrolling patients in the first half of 2024. Another secondary endpoint the study is assessing is motor effects at 1 and 2 years posttransplant.

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Bemdaneprocel is comprised of dopamine producing neurons derived from pluripotent stem cells and is designed to be surgically implanted into the brain to reform neural networks in patients with PD. Transplant surgeries have been performed by neurosurgeons at Memorial Sloan Kettering Cancer Center, Krembil Brain Institute, and University of Toronto (UoT). Participants were followed at clinical sites including Weill Cornell Medicine, University of California, Irvine, University Health Network, and UoT.

“At Bayer, we are committed to advancing cell and gene therapy innovations for patients with PD, a neurodegenerative disorder with debilitating effects on people’s lives for which there is currently no cure and only limited treatment options,” Christian Rommel, member, executive committee, pharmaceuticals division and head, research and development, Bayer, added. “The positive outcome of our first cell therapy clinical trial for PD is encouraging not only for the bemdaneprocel development program but also our entire pluripotent stem cell-based platform and warrants further investigation in larger groups of patients.”

REFERENCE
BlueRock’s neuronal stem cell therapy for Parkinson’s disease is first to show positive results in Phase I clinical study. News release. BlueRock Therapeutics. June 28, 2023.
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