FDA Grants Regenerative Medicine Advanced Therapy Designation to Parkinson Cell Therapy Candidate Bemdaneprocel

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Following presented phase 1 clinical trial findings at AAN 2024, bemdaneprocel receives regenerative medicine advanced therapy designation by the FDA for the treatment of Parkinson disease.

Seth Ettenberg, PhD

(Credit: BlueRock Therapeutics)

According to a recent announcement, the FDA has granted BlueRock Therapeutics’ bemdaneprocel, an investigational cell therapy for Parkinson disease (PD), a regenerative medicine advanced therapy (RMAT) designation.¹ Bemdaneprocel is designed to reform neural networks that have been severely impacted by the disease and currently is the most clinically advanced investigational cell therapy in the United States for the treatment of PD.

The company recently presented positive 18-month data on bemdaneprocel from its phase 1 study (NTC04802733) at the 2024 International Conference on Alzheimer’s and Parkinson’s Disease (AD/PD), held March 5-9, in Lisbon, Portugal. Conducted by lead author Claire Henchcliffe, MD, chair of the department of neurology at the University of Califorina, Irvine, and colleagues, the results showed that the agent was safe and well-tolerated, with treated patients demonstrating improvements in ON and OFF time over that period.²

“We are excited about the positive data from the bemdaneprocel phase 1 clinical trial and believe it has great potential to help patients living with PD regain functions they have lost to the disease,” Seth Ettenberg, president and CEO at 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 2 clinical studies.”

The multicenter, open-label, nonrandomized, noncontrolled phase 1 trial had 12 patients with PD who received surgical transplantation of 1 of 2 different dose levels of bemdaneprocel cells to the post-commissural putamen bilaterally, along with an immunosuppression regimen for 1 year. Using the Hauser Diary, those in the high-dose cohort demonstrated a mean increase of 2.7 hours in good ON time compared with baseline following 18 months of treatment. Additionally, this treated patient group demonstrated a mean reduction of 2.7 hours in time spent in the OFF state.

Patients on high-dose bemdaneprocel (n = 7) had a dose of 2.7 million cells per putamen whereas those in the low-dose group (n = 5) had a dose of 0.9 million cells per putamen. The initial 12-month data of the trial, presented at the 2023 International Congress of Parkinson’s Disease and Movement Disorders, held August 27-31, in Copenhagen, Denmark, highlighted the efficacy and safety of this treatment. At 1 year, the participants in the high-dose cohort displayed a reduction of 13.0 points in MDS-UPDRS-III scores compared with the baseline.

Additional findings were less pronounced in the low-dose cohort, with reductions of 7.6 points after 1 year. Imaging analysis using 18F-DOPA PET, a technique used to visualize and assess dopaminergic activity, revealed evidence of cell survival and engraftment in both the high- and low-dose cohorts. At 1-year follow-up investigators observed no reports of serious adverse events (AEs) attributable to bemdaneprocel; however, they did note 2 unrelated serious AEs of seizure attributed to the surgical procedure and 1 COVID case where both resolved without sequelae.

“The RMAT designation for bemdaneprocel underscores the potential of this candidate to fundamentally change the way we think about PD care,” Christian Rommel, PhD, head of research and development at Bayer’s Pharmaceuticals Division, said in a statement.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.”

At the 2024 American Academy of Neurology (AAN) Annual Meeting, held April 13-18, in Denver, Colorado, NeurologyLive^® sat down with PD expert Daniel Kremens, MD, JD, an associate professor of neurology and codirector of the Parkinson’s Disease and Movement Disorders Center at Thomas Jefferson University, to discuss some of the top data being presented at the meeting on novel approaches in development for the treatment of the movement disorder. Kremens discussed these various presentations, offering his perspective on the clinical landscape. In this segment, Kremens discussed new data from an early-stage trial assessing bemdaneprocel in patients with PD. He gave his reaction to the findings presented at AAN and how they will impact clinical care going forward.

REFERENCES
1. BlueRock Therapeutics receives FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease cell therapy candidate bemdaneprocel. News Release. BlueRock Therapeutics. Published May 30, 2024. Accessed June 6, 2024. https://www.bluerocktx.com/bluerock-therapeutics-receives-fda-regenerative-medicine-advanced-therapy-designation-for-parkinsons-disease-cell-therapy-candidate-bemdaneprocel/
2.Henchcliffe C, Sarva H, Lozano A, et al. Dopaminergic neuronal cell therapy for Parkinson’s disease: results from a phase 1 study of bemdaneprocel. Presented at 2023 MDS Congress; August 27-31; Copenhagen, Denmark. Abstract 65.