Bayer's BRT-DA01 Stem Cell Therapy for Parkinson’s Disease Meets Primary Endpoint in Phase 1 Clinical Trial

▎WuXi

Edited by Kant Content Team

Bayer and its subsidiary BlueRock Therapeutics announced today that their investigational stem cell-derived therapy, bemdaneprocel (BRT-DA01), met the primary endpoint in a Phase 1 clinical trial for the treatment of Parkinson's disease. Detailed data were presented at the International Congress of Parkinson's Disease and Movement Disorders. Based on these results,The Phase 2 clinical trial of this therapy is in the planning stages and is expected to begin patient recruitment in the first half of 2024.

Parkinson's disease is the most common neurodegenerative movement disorder, affecting more than 10 million people worldwide. It is caused by damage to nerve cells in the brain, leading to decreased dopamine levels, a neurotransmitter involved in processes such as memory or movement. The disease often begins with a tremor in one hand, and other symptoms include muscle stiffness, spasms, and dyskinesia (involuntary twisting movements of the face, arms, legs, or trunk). Common dopamine replacements, such as levodopa, are used to alleviate disease symptoms, but their effectiveness diminishes as the disease progresses. By targeting the root cause of the disease, cell and gene therapies aim to go beyond merely alleviating symptoms.

Bemdaneprocel is an investigational cell therapy composed of pluripotent stem cell-derived, dopamine-producing neurons, which can be surgically implanted into the brains of patients with Parkinson's disease.When these cells are transplanted, they have the potential to rebuild the damaged neural networks in the brains of Parkinson's disease patients, thereby restoring their motor and non-motor functions.

The study met its primary objective, demonstrating that bemdaneprocel was safe and well-tolerated in all 12 subjects across both low-dose and high-dose groups, with no serious adverse events related to the therapy reported within one year.Two serious adverse events unrelated to bemdaneprocel (including one seizure triggered by surgery and one case of COVID-19) have been resolved without sequelae. Additionally, 18F-DOPA PET imaging scans demonstrated evidence of cell survival and engraftment in both the low-dose and high-dose groups. 18F-DOPA PET imaging is a neuroimaging technique used to visualize and evaluate dopaminergic activity in Parkinson’s disease.

Using Hauser Diary to classify patients, being in the "On" state indicates well-controlled symptoms; being in the "Off" state indicates symptom worsening.Compared with the baseline, participants in the high-dose group of the bemdaneprocel clinical trial spent an additional 2.16 hours in the "on" state without dyskinesia after one year, and the time in the "off" state was correspondingly reduced by 1.91 hours.Subjects in the low-dose group spent 0.72 more hours in the "on" state compared to baseline without experiencing dyskinesia, and correspondingly, their time in the "off" state decreased by 0.75 hours.

In the high-dose group, the one-year efficacy of bemdaneprocel in the "off-medication" state was assessed using Part III of the MDS-UPDRS scale for Parkinson's disease, showing a reduction of 13.0 points from baseline. The low-dose group showed a reduction of 7.6 points from baseline.