Bayer AG and BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned independently operated subsidiary of Bayer AG, announced today details of the positive data from the Phase I clinical trial for bemdaneprocel (BRT-DA01), a stem cell derived investigational therapy for treating Parkinson’s disease. The data were presented at the International Congress of Parkinson's Disease and Movement Disorders® in Copenhagen, Denmark.

The study met the primary objective of demonstrating safety and tolerability in all 12 participants in the study’s low and high dose cohorts, with no serious adverse events (SAEs) reported related to bemdaneprocel through one year. There were two SAEs reported that were unrelated to bemdaneprocel, one seizure attributed to the surgical procedure and one COVID case. Both resolved without sequelae. In addition, 18F-DOPA PET imaging scans demonstrated evidence of cell survival and engraftment in both low and high dose cohorts. 18F-DOPA PET imaging is a neuroradiological technique used to visualize and assess dopaminergic activity in Parkinson’s disease.

Secondary exploratory clinical endpoints improved in both cohorts, with participants in the high dose cohort showing greater improvement, as assessed by the MDS-Unified Parkinson’s Disease Rating Scale Part III (MDS-UPDRS Part III) and the Hauser Diary, which are tools used to assess Parkinson’s disease severity in motor symptoms.

“The data from this Phase I open label study are extremely encouraging,” 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. “While this is a small open label study, meeting the study’s primary objective for safety and tolerability along with initial improvements seen in clinical outcomes represents a great step forward. The hope now is that these trends continue and translate into meaningful benefit for people with Parkinson’s disease in controlled clinical trials.”

Using the Hauser 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, participants in the high dose cohort showed an improvement of 2.16 hours in time spent in the “ON” state without troubling dyskinesia compared with baseline after one year. Time spent in the “OFF” state showed a corresponding decrease of 1.91 hours after one year. Participants in the low dose cohort showed an improvement of 0.72 hours in the “ON” state without troubling dyskinesia time compared with baseline and a corresponding decrease of 0.75 hours in “OFF” state time.

In the high dose cohort, a one-year measurement of the effects of bemdaneprocel using MDS-UPDRS Part III measured in the “OFF”-medication state, showed a reduction of 13.0 points compared with baseline. The low dose cohort showed a reduction of 7.6 points.

“The need for new therapies to help patients struggling with Parkinson’s disease is clear,” said Ahmed Enayetallah, Senior Vice President and Head of Development, BlueRock Therapeutics. “We are excited to be sharing the results of this Phase I and look forward to advancing bemdaneprocel to the next stage of clinical testing.”

 Based on these results, planning is underway for a Phase II study that is expected to begin enrolling patients in H1 (first half) 2024.

“The standard of care for millions of people living with Parkinson’s disease has only marginally improved in the past decades, and the existing unmet medical need will only become higher due to the growing aging population,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development. “The positive outcome of this Phase I clinical trial is a clear step forward, and it brings us closer to delivering new treatment options to patients.”