Bayer-owned company says plans are now in progress for a Phase 2 Parkinson’s trial, set to begin in 2024.

Cell therapy company BlueRock Therapeutics has revealed promising results from the Phase 1 clinical trial of bemdaneprocel, an investigational stem cell therapy designed to treat Parkinson’s disease. BlueRock, a wholly owned independently operated subsidiary of pharma giant Bayer, is creating a pipeline of cell therapy treatments for neurological, cardiovascular, immunological, and ophthalmic conditions.

Bemdaneprocel is a stem cell therapy developed to replace dopamine-producing neurons that are lost in Parkinson’s disease. Derived from pluripotent stem cells, the therapy involves implanting neuron precursors into the brains of patients to potentially restore neural networks and improve motor and non-motor functions.

“The need for new therapies to help patients struggling with Parkinson’s disease is clear,” said Ahmed Enayetallah, Head of Development at BlueRock Therapeutics. “We are excited to be sharing the results of this Phase 1 and look forward to advancing bemdaneprocel to the next stage of clinical testing.”

The Phase 1 study included surgical transplantation of two different dose levels into the brains of 12 subjects, followed by a 1-year immunosuppression regimen. The surgeries were performed by experienced neurosurgeons, and assessments will continue for two years.

The trial demonstrated the therapy’s safety, tolerability, and potential effectiveness in addressing the symptoms of Parkinson’s disease. The study included 12 participants divided into low and high dose cohorts, with all participants completing the one-year trial duration. BlueRock reports the therapy was well tolerated, and no major safety concerns were observed.

‘Significant improvements’ for high dose cohort

Assessment of exploratory clinical endpoints showed improvements in both cohorts, with greater improvements observed in the high dose cohort. Using tools like the MDS-Unified Parkinson’s Disease Rating Scale Part III and the Hauser Diary, which evaluate disease severity and motor symptoms, participants in the high dose cohort experienced significant improvements compared with baseline.

“The data from this Phase 1 open label study are extremely encouraging,” said Dr Claire Henchcliffe, from 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.”

Furthermore, 18F-DOPA PET imaging scans provided evidence of cell survival and engraftment in both dose cohorts, indicating the potential for effective integration of the transplanted cells.

“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, Head of R&D at Bayer’s pharmaceuticals division. “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.”

Based on these positive results, plans are in progress for a Phase 2 study, set to begin enrolling participants in the first half of 2024. The Phase 2 study will build upon the Phase 1 findings to further evaluate the therapy’s safety and efficacy.