BlueRock readies for Phase 2 trial of neuron replacement therapy as exploratory clinical endpoints from Phase 1 continue to improve.
Cell therapy company BlueRock Therapeutics has unveiled encouraging 18-month data from its ongoing Phase 1 clinical trial of its lead stem cell-derived therapy targeting Parkinson’s disease. The company, which became a wholly owned subsidiary of pharma giant Bayer in 2019, develops cellular medicines to address neurological, cardiovascular, immunological, and ophthalmic diseases.
BlueRock’s lead clinical program, bemdaneprocel, is designed to replace lost dopamine-producing neurons in Parkinson’s disease. Derived from pluripotent stem cells, it aims to restore neural networks severely affected by the disease.
The Phase 1 trial of bemdaneprocel, which assessed safety, tolerability, cell survival, and motor effects, involved 12 participants in low and high dose cohorts, and demonstrated sustained positive trends without major safety concerns. The study, which began in surgical transplantation with a one-year immunosuppression regimen, will continue assessments over two years. Notably, after discontinuing the 12-month immune suppression regimen, assessment at 18 months revealed cells continued to survive and engraft, and scans showed that patients’ F-DOPA signal (a measure of dopamine function in the brain) continued to increase.
Exploratory clinical endpoints, measured by the MDS-Unified Parkinson’s Disease Rating Scale Part III (MDS-UPDRS Part III) and the Hauser Diary, also showed improvement from 12 to 18 months. Participants in the high dose cohort exhibited greater enhancements compared to the low dose cohort.
Using the Hauser Diary, participants in the high dose cohort demonstrated a mean increase of 2.7 hours in the “Good ON” state time and a corresponding mean decrease of 2.7 hours in the “OFF” state time after 18 months. The low dose cohort also showed improvement, albeit to a lesser extent.
In the high dose cohort, an 18-month measurement using MDS-UPDRS Part III in the “OFF”-medication state revealed a mean reduction of 23 points compared to baseline, whereas the low dose cohort showed a mild improvement.
A Phase 2 study of the effects of bemdaneprocel in Parkinson’s patients is slated to begin enrolling patients later this year.
“It’s exciting that bemdaneprocel met safety and tolerability criteria at 12 months, and now the 18-month results suggest that these allogeneic cells survive and have potentially positive effects even after discontinuation of immunosuppressants,” said Dr Claire Henchcliffe from the University of California, Irvine and one of the study’s Principal Investigators. “We should not overinterpret results of a phase I study, but this is a promising step that deserves to be followed up with further studies.”
