Cell therapy bemdaneprocel gets FDA RMAT status

Designation paves way for more trials, potential early review

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to bemdaneprocel, a cell therapy BlueRock Therapeutics, a subsidiary of Bayer, is developing to treat Parkinson’s disease.

RMAT designation is granted to therapies that have shown potential to treat serious or life-threatening conditions. It provides a series of benefits, including early talks with the FDA about trial design, and potential for priority review of an eventual application seeking approval.

“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 at Bayer’s pharmaceuticals division, said in a Blue Rock press release. “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.”

Parkinson’s is caused by the loss of dopaminergic neurons, the nerve cells responsible for producing dopamine, a chemical messenger important for motor control. The loss of dopamine causes the disease’s typical motor symptoms, such as tremors, muscle rigidity, and slowness of movement, as well an nonmotor symptoms.

While levodopa and its derivatives are the mainstay treatment for Parkinson’s by providing cells with the raw material to produce dopamine, these treatments’ efficacy weakens over time.

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Therapy designed to generate new neurons

Bemdaneprocel, previously known as BRT-DA01, is designed to generate new dopaminergic neurons after being transplanted into the brain, specifically the putamen, a region involved in motor control. The cell therapy uses human embryonic stem cells to generate dopamine-producing cell precursors. The transplanted cells are expected to restore motor and non-motor function.

A Phase 1 clinical trial (NCT04802733) enrolled 12 people with Parkinson’s all of whom experienced off periods, when symptoms are not fully controlled by levodopa. The experimental cell therapy was transplanted at a low (0.9 million cells per putamen) or high dose (2.7 million cells per putamen).

Data obtained one year after the transplant showed that the cell therapy was safe and well-tolerated, without any serious side effects reported. Brain imaging scans showed that the transplanted cells produced dopamine, leading to an easing of motor symptoms and off time reduction.

These improvements were maintained 1.5 years after treatment, with cells surviving in the brain even after patients stopped a one-year immune-suppression regimen.

“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 Parkinson’s disease regain functions they have lost to the disease,” said Seth Ettenberg, PhD president and CEO of BlueRock Therapeutics. “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.”