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Bayer AG and BlueRock Therapeutics, a wholly-owned subsidiary of Bayer that operates independently and is a clinical-stage cell therapy company, announced positive 24-month data from the exPDite study, an investigational cell therapy using bemdaneprocel forTreatment of Parkinson's DiseasePhase 1 clinical trial.
Bemdaneprocel is the most advanced clinical research cell therapy used in the United States for treating patients with Parkinson's disease.In May 2024, bemdaneprocel received the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation, enabling senior management at the FDA's Center for Biologics Evaluation and Research (CBER) to expedite development review and provide guidance on development planning.The exPDite trial, designed to evaluate the safety and tolerability of bemdaneprocel, has now been completed, and the 24-month data will be presented on September 28, 2024, at the International Parkinson and Movement Disorder Society meeting in Philadelphia.
Using the Hauser PD Diary (which classifies patients as "ON" when symptoms are well-controlled and "OFF" when symptoms worsen), 7 participants in the high-dose group showed an average increase of 1.8 hours in "Good ON" state time without troublesome dyskinesia at 24 months compared to baseline. At 24 months, the time spent in the "OFF" state decreased by an average of 1.9 hours compared to baseline. In the low-dose group, 5 participants showed "Good ON"The 'ON' state time decreased by an average of 0.8 hours, while the 'OFF' state time increased by an average of 0.4 hours."In the high-dose cohort, the impact on motor symptoms was measured over 24 months using Part III of the MDS-UPDRS in the “OFF” state, showing results compared to baseline.bemdaneprocelThe average reduction in motor symptoms was 21.9 points.The average reduction in the low-dose group was 8.3 points.In the high-dose cohort, usingMDS-UPDRSPart IICorrectbemdaneprocelThe 24-month measurement of impact on activities of daily living showed an average reduction of 3.4 points compared to baseline.The low-dose group increased by an average of 2.0 points.The 24-month safety profile is consistent with previous research findings, indicating that bemdaneprocel continues to be well-tolerated in patients, with no adverse events related to bemdaneprocel reported. The transplanted cells remained viable and engrafted in the brain 12 months after the cessation of immunosuppressive therapy. Additionally, secondary clinical endpoints associated with motor symptoms continued to show a positive trend from baseline throughout the follow-up period, with a more encouraging trend observed in the high-dose cohort compared to the low-dose cohort. These trends were assessed using MDS-UPDRS Part II and Part III and the Hauser PD Diary. These consistently positive trends suggest that bemdaneprocel may provide sustained benefits for disease-induced motor impairments.Amit Rakhit, Chief Development and Medical Officer of BlueRock Therapeutics, said："We are pleased to share with you the 24-month data from the exPDite trial, which indicate that bemdaneprocel could be a meaningful treatment option for patients with Parkinson's disease. The completion of this study is an important milestone for bemdaneprocel and lays the groundwork for the next phase of clinical development."Claire Henchcliffe, M.D., Chair of the Department of Neurology at the UCI School of Medicine, University of California, Irvine, and one of the principal investigators of the study, said"The concept of restoring dopamine input in the brain using transplanted cells is gaining significant momentum, with the positive results of the exPDite trial leading the way. Completed studies have shown that the transplanted cells can survive, and there are early indications that bemdaneprocel may help patients achieve better control of motor symptoms. These results are encouraging and warrant further exploration in the next phase of placebo-controlled studies."Source of the article: Company's official website
