BlueRock Therapeutics’ Cell Therapy for Parkinson’s Gains Special Designation in Japan

Key findings

• Bayer’s BlueRock Therapeutics bemdaneprocel received Japan’s SAKIGAKE designation for Parkinson’s disease.
• SAKIGAKE can enable more frequent consultations with Japan’s MHLW and may support accelerated approval and priority review if criteria are met.
• The companies say a pivotal Phase III trial (exPDite-2) is enrolling and will compare bemdaneprocel to sham surgery.

Bayer and its subsidiary BlueRock Therapeutics say Japan’s Ministry of Health, Labor and Welfare (MHLW) has granted Pioneering Regenerative Medical Product designation (SAKIGAKE) to bemdaneprocel, an investigational cell therapy for Parkinson’s disease.

SAKIGAKE can allow more frequent interaction with the MHLW to discuss the development plan and may support accelerated approval and priority review if relevant criteria are met.

What is bemdaneprocel?
Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace dopamine-producing neurons that are lost in Parkinson’s disease. The treatment uses dopaminergic neuron precursors derived from human embryonic pluripotent stem cells (stem cells that can develop into many cell types), which are developed into mature dopamine neurons after brain implantation.

BlueRock says the transplanted cells have the potential to re-form neural networks affected by Parkinson’s disease and may help restore motor and non-motor function, although it has not been approved yet.

Clinical and regulatory status
BlueRock says bemdaneprocel received Fast Track designation from the FDA in 2021, and Regenerative Medicine Advanced Therapy (RMAT) designation in 2024.

A pivotal Phase III clinical trial, exPDite-2, is currently enrolling participants, and they’ll evaluate the efficacy, safety, and overall impact of bemdaneprocel compared to sham surgery control.

exPDite-2 trial design
BlueRock describes exPDite-2 as the first Phase III pivotal clinical trial for an investigational allogeneic (donor-derived) pluripotent stem cell-derived therapy to treat Parkinson’s disease. It’s a multicenter, double-blind trial designed to enroll approximately 102 participants with Parkinson’s disease in the US, Canada, and Australia.

• Primary endpoint: Change from baseline to week 78 in a PD motor diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day.
• Secondary endpoints: Measures of movement, non-motor symptoms, safety and tolerability, activities of daily living, and quality of life.

BlueRock’s earlier Phase I study enrolled 12 participants and noted that bemdaneprocel was well tolerated, with no serious adverse events related to the drug product at 36 months post-surgery. The company added that “encouraging trends” were observed in secondary endpoints related to motor impairments at 36 months post-surgery.

Parkinson’s disease in context
Parkinson’s disease (PD) is a progressive neurodegenerative disease marked by the loss of dopamine-producing nerve cells in the brain, leading to ongoing motor impairment. Symptoms can include tremors, muscle rigidity, and slowed movement, along with non-motor symptoms such as fatigue, cognitive issues, and depression.

The companies estimate PD affects more than 10 million people worldwide and about 250,000 people in Japan.

Company snapshot
BlueRock Therapeutics is a clinical-stage cell therapy company focused on neurological and ophthalmic diseases. Its clinical-stage programs include bemdaneprocel for Parkinson’s disease and OpCT-001 for primary photoreceptor diseases. BlueRock says OpCT-001 has Fast Track designation from the FDA and is being evaluated in a Phase I clinical trial (CLARICO).

BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, and became a wholly owned, independently operated subsidiary of Bayer in late 2019, according to the company. More information is available at www.bluerocktx.com.

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