Positive 24-Month Data for BlueRock's Bemdaneprocel in Parkinson’s Therapy

BERLIN, Germany & CAMBRIDGE, MA, USA I September 27, 2024 I Bayer AG and BlueRock Therapeutics LP, a clinical stage cell therapy company and subsidiary of Bayer AG, have announced promising 24-month results from exPDite, a Phase 1 clinical trial of bemdaneprocel, an investigational cell therapy aimed at treating Parkinson’s disease.

Bemdaneprocel is currently the most advanced investigational cell therapy for Parkinson's disease in the U.S. The exPDite trial, which aimed to evaluate the safety and tolerability of bemdaneprocel, has reached completion, and the latest data will be shared at the International Congress of Parkinson’s Disease and Movement Disorders in Philadelphia on September 28, 2024.

Amit Rakhit, Chief Development and Medical Officer at BlueRock Therapeutics, expressed enthusiasm about the data, stating that the results indicate bemdaneprocel could be a valuable treatment for Parkinson’s patients. The completion of this study is a significant milestone and lays the groundwork for the next phase of clinical development.

The 24-month safety profile remains consistent with earlier findings, showing that bemdaneprocel is well-tolerated with no adverse events reported. Transplanted cells have survived and integrated into the brain even after immunosuppression therapy was stopped at 12 months. Secondary clinical endpoints related to motor symptoms have shown positive trends from the baseline throughout the follow-up period, with the high dose cohort exhibiting more favorable trends than the low dose cohort. These trends were evaluated using the MDS-Unified Parkinson’s Disease Rating Scale Part II and III and the Hauser PD Diary, which assess the severity of motor symptoms in Parkinson’s disease. The persistent positive trends suggest that bemdaneprocel could offer sustained benefits for movement impairments.

Dr. Claire Henchcliffe, chair of the UCI School of Medicine Department of Neurology and a Principal Investigator of the study, emphasized the importance of these results in advancing the concept of restoring dopamine inputs in the brain using transplanted cells. The study shows that the transplanted cells survive, and there are early indications that bemdaneprocel could help patients better manage their motor symptoms. These promising results merit further exploration in a next-phase placebo-controlled study.

In the high dose cohort, there was a mean reduction of 21.9 points in MDS-UPDRS Part III scores in the "OFF"-medication state compared to baseline, while the low dose cohort showed a mean decrease of 8.3 points. Using the Hauser PD Diary, the high dose cohort showed a mean increase of 1.8 hours in "Good ON" state time without troublesome dyskinesias and a mean decrease of 1.9 hours in "OFF" state time after 24 months. The low dose cohort displayed a mean decrease of 0.8 hours in "Good ON" state time and a mean increase of 0.4 hours in "OFF" state time.

Furthermore, the high dose cohort showed a mean reduction of 3.4 points in MDS-UPDRS Part II scores, which measure activities of daily living, compared to baseline, while the low dose cohort showed a mean increase of 2.0 points.

Christian Rommel, Head of Research and Development at Bayer’s Pharmaceuticals Division, stated that the continued positive results support their commitment to developing innovative therapies that significantly improve patient lives. The emerging data reinforce the potential benefits for Parkinson’s patients.

Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine-producing neurons lost in Parkinson’s disease. Derived from human embryonic stem cells, these dopaminergic neuron precursors are implanted into the brain to potentially reform neural networks and restore motor and non-motor functions. Bemdaneprocel received a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA in May 2024, facilitating expedited development and review.

The exPDite trial, a multi-center, open-label, non-randomized, non-controlled phase 1 study, involved 12 subjects with Parkinson’s disease undergoing surgical transplantation of bemdaneprocel at two different dose levels. After assessing safety, tolerability, cell survival, and motor effects at 12 months, final assessments were made at 24 months.

Transplant surgeries were performed by Dr. Viviane Tabar and Dr. Andres Lozano, with participants monitored by clinical specialists at various institutions including Weill Cornell Medicine, University of California, Irvine, and the University Health Network.

Parkinson’s disease is a progressive neurodegenerative disorder characterized by the loss of dopamine-producing neurons, leading to motor function decline and symptoms such as tremors and muscle rigidity. Current treatments become less effective over time, highlighting the need for new therapies. More than 10 million people worldwide suffer from Parkinson’s, with about one million in the U.S.