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.

“We are very excited to share the 24-month data from the exPDite trial which shows that bemdaneprocel could be a potentially meaningful treatment option for individuals living with Parkinson’s disease,” said Amit Rakhit, Chief Development and Medical Officer at BlueRock Therapeutics. “The completion of this study marks an important milestone for bemdaneprocel and sets the stage for the next phase of clinical development.”

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.”

“The continued positive results of the exploratory clinical endpoints for bemdaneprocel after 24 months are encouraging and support our commitment in developing innovative therapies that can significantly improve patient lives,” said Christian Rommel, Head of Research and Development at Bayer’s Pharmaceuticals Division. “The emerging data confirm the potential for Parkinson's patients.”