Bayer’s experimental Parkinson’s disease therapy made from stem cells will soon enter late-stage studies, marking a key test for regenerative medicine in neurologic conditions.

The therapy, known as bemdaneprocel, is being developed by BlueRock Therapeutics, a Bayer subsidiary. The company said Monday that it is no longer doing a previously planned Phase 2 study, opting instead to test the cell therapy directly in a Phase 3 trial that’s expected to begin in the first half of this year. BlueRock has “start-up activities already ongoing,” BlueRock chief development and medical officer Amit Rakhit said in an interview.

Rakhit, who joined BlueRock six months ago, said the company decided to go directly into Phase 3 for three reasons: There are few treatment options for the condition, which impacts more than 10 million people globally; the therapy had a “very acceptable safety profile” in its Phase 1 study; and the FDA granted the therapy a regenerative medicine advanced therapy designation — meaning its approval could be expedited.

“It’s exciting, right? We have been trying for a long time to do a therapy like this,” said Mount Sinai neurologist Joohi Jimenez-Shahed, the principal investigator for the Phase 3 study. Jimenez-Shahed served on the data safety monitoring board for the Phase 1 study and has received compensation from BlueRock.

BlueRock is moving forward with the high dose that it tested in its Phase 1 study, surgically implanting three million cells in each side of the brain. In a Phase 1 trial, seven patients who received the high dose saw a mean reduction of 21.9 points at two years compared with baseline on a measure of Parkinson’s motor symptoms called the MDS-Unified Parkinson’s Disease Rating Scale Part III.

Including those patients who received the low dose of the therapy, the 12 participants in the Phase 1 study saw no adverse events related to the therapy at two years, and transplanted cells continued to survive even after patients stopped taking immunosuppressive drugs at one year, BlueRock reported in September when it shared long-term follow-up results from the study.

The Phase 3 trial, called exPDite-2, will enroll around 102 patients with moderate Parkinson’s and randomize them to receive either bemdaneprocel or sham surgery. However, reaching the primary endpoint will take time. The study will look at change from baseline to week 78, or one and a half years, using a common clinical trial tool in Parkinson’s studies called the Parkinson’s disease diary. The study’s key measure looks at PD Diary “on” time without troublesome dyskinesia, meaning time when the treatment is working without involuntary and uncontrolled muscle movement.

The current treatment options for Parkinson’s include levodopa — but many who take the drug eventually develop dyskinesia — as well as deep brain stimulation. Meanwhile, bemdaneprocel, which is made from embryonic stem cells, is meant to replace dopamine-producing neurons in the brain.

“This will be the first dopaminergic stem cell therapy that really gets to this phase of investigation, so I think it’s going to be really interesting to see how it works; what works; what — if there are any — pitfalls,” Jimenez-Shahed said. “We’re going to learn a lot from it.”

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