BlueRock Therapeutics’ investigational cell therapy bemdaneprocel for Parkinson’s disease shows positive data at 24-months.
Bayer AG and BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned independently operated subsidiary of Bayer AG today announced positive 24-month data from exPDite, a Phase 1 clinical trial of bemdaneprocel, an investigational cell therapy for the treatment of Parkinson's disease.
Bemdaneprocel is the most clinically advanced investigational cell therapy in the U.S. for treating patients living with Parkinson’s disease. The exPDite trial, designed to assess the safety and tolerability of bemdaneprocel, is now complete and the 24-month data will be presented at the International Congress of Parkinson's Disease and Movement Disorders in Philadelphia on September 28, 2024.
The safety profile at 24 months is consistent with earlier findings, demonstrating that bemdaneprocel continues to be well-tolerated by patients, with no adverse events reported related to bemdaneprocel. Transplanted cells continue to survive and engraft in the brain after discontinuing immunosuppression therapy at 12 months as outlined in the study’s protocol. In addition, secondary clinical endpoints related to motor symptoms continue to show positive trends from baseline through the duration of follow-up, with more encouraging trends in the high dose cohort than those in the low dose cohort. These were assessed by the MDS-Unified Parkinson’s Disease Rating Scale Part II and III (MDS-UPDRS Part II & III) and the Hauser PD Diary, tools used to assess Parkinson’s disease severity in motor symptoms. These consistent positive trends suggest that bemdaneprocel could potentially offer sustained benefit for movement impairments caused by the disease.
“There is considerable momentum in the concept of restoring dopamine inputs in the brain using transplanted cells, and the positive results from the exPDite trial leads the drive forward,” said Claire Henchcliffe, MD, chair of the UCI School of Medicine Department of Neurology at the University of California, Irvine and one of the study’s Principal Investigators. “The completed study demonstrates that the transplanted cells survive and there are early signs that bemdaneprocel can potentially help patients to better control their motor symptoms. These are exciting results that warrant further exploration in a next phase placebo-controlled study.”
In the high dose cohort, the 24-month measurement of the effects of bemdaneprocel on motor symptoms using MDS-UPDRS Part III measured in the “OFF”-medication state, showed a mean reduction of 21.9 points compared with baseline. The low dose cohort showed a mean decrease of 8.3 points. Using the Hauser PD Diary, which categorizes patients as being in the “ON” state when their symptoms are well controlled and in the “OFF” state when they experience a worsening of their symptoms, the 7 participants in the high dose cohort showed a mean increase of 1.8 hours in time spent in the “Good ON” state without troublesome dyskinesias compared with baseline after 24 months. Time spent in the “OFF” state showed a mean decrease of 1.9 hours from baseline after 24 months. The 5 participants in the low dose cohort showed a mean decrease of 0.8 hours in the “Good ON” state time compared with baseline and a corresponding mean increase of 0.4 hours in “OFF” state time.
In the high dose cohort, the 24-month measurement of the effects of bemdaneprocel on activities of daily living using MDS-UPDRS Part II measured a mean reduction of 3.4 points compared with baseline. The low dose cohort showed a mean increase of 2.0 points.
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