FDA Recognizes BlueRock’s Parkinson Disease Cell Therapy With RMAT Designation

The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BlueRockTherapeutics’s bemdaneprocel cell therapy for the potential treatment of Parkinson disease (PD).¹

“We are excited about the positive data from the bemdaneprocel phase I clinical trial and believe it has great potential to help patients living with Parkinson's disease regain functions they have lost to the disease,” Seth Ettenberg, President and CEO,BlueRock Therapeutics, said in a statement.¹ “Now with this RMAT designation in hand, we look forward to closely collaborating with the FDA to ready this program for phase II clinical studies.”

Bemdaneprocel is a dopaminergic neuron precursor cell therapy and is being evaluated in a phase 1/2 clinical trial (NCT04802733). The trial is primarily assessing the incidence and severity of adverse events (AEs) after administration of the cell therapy. Secondary outcome measures being assessed include cell survival, as measured by change in 18F-DOPA uptake using positron emission tomography (PET); changes in motor function, as measured by MDS-Unified Parkinson's Disease Rating Scale (UPDRS) motor sub-score in the "off" state; changes in waking hours in "Off" state; and long-term safety and tolerability.

“The RMAT designation for bemdaneprocel underscores the potential of this candidate to fundamentally change the way we think about Parkinson’s disease care,” Christian Rommel, PhD, Head of Research and Development, Bayer’s Pharmaceuticals Division, added.¹ “We are driven by our commitment to deliver breakthrough innovation for patients and are proud and excited to see bemdaneprocel continuing to clear hurdles in the development process.”

READ MORE: Where Are We: Development of Gene and Cell Approaches for Parkinson Disease

The trial recently reported data demonstrating tolerability and cell survival in March 2024. At 18 months posttreatment, bemdaneprocel remained well-tolerated and no major safety issues had occurred in the treated patients, which included 12 patients in total across the lower dose (0.9 million cells per putamen) cohort A (n = 5) and the higher dose (2.7 million cells per putamen) cohort B (n = 7). Furthermore, the transplanted cells survived and engrafted in the brain and following the cessation of immune suppression therapy at 12 months posttreatment as per study protocol F-DOPA signal was shown to continue increasing.²

At 18 months posttreatment, the patients treated at the high dose experienced a mean increase from baseline of 2.7 hours in the “ON” state and a 2.7 hour decrease for time in the “OFF” state. For patients treated at the low dose, a mean increase of 0.2 hours in the “ON” state compared to baseline was observed at 18 months posttreatment, with a corresponding mean decrease of 0.8 hours spent in the “OFF” state. The study also looks at the change from baseline in the MDS-Unified Parkinson's Disease Rating Scale Part III (MDS-UPDRS Part III) motor sub-score in the “OFF” state. At 18 months posttreatment, patients who received the high dose of bemdaneprocel achieved a 23-point reduction compared to baseline on the MDS-UPDRS Part III and patients who received the low dose achieved an 8.6-point reduction. The study is enrolling participants for its phase 2 arm.²

“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,” Claire Henchcliffe, MD, the chair of the University of California, Irvine, School of Medicine Department of Neurology and a principal investigator for the trial, said in a statement at that time.² “We should not overinterpret results of a phase 1 study, but this is a promising step that deserves to be followed up with further studies.”

REFERENCES

1. BlueRock Therapeutics receives FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease cell therapy candidate bemdaneprocel. News release. May 30, 2024. https://finance.yahoo.com/news/bluerock-therapeutics-receives-fda-regenerative-063000771.html
2. BlueRock Therapeutics phase I clinical trial for Parkinson’s disease continues to show positive trends at 18 months. News release. Bayer AG and BlueRock Therapeutics LP. March 6, 2024. Accessed March 22, 2024. https://www.bluerocktx.com/bluerock-therapeutics-phase-i-clinical-trial-for-parkinsons-disease-continues-to-show-positive-trends-at-18-months/