Bayer's billon-dollar Parkinson's disease bets land in the clinic

Brain image from Parkinson's study
Parkinson's disease typically begins with a tremor in one hand and progresses to symptoms including rigidity, cramping and involuntary movement of the face, arms, legs or trunk. (Radiological Society of North America)

Bayer’s Parkinson’s disease stem cell therapy has officially landed in the clinic while another treatment for the neurodegenerative movement disorder is seeking patients.

The first dose of DA01, which the German Big Pharma refers to as pluripotent stem cell-derived dopaminergic neurons, was given out in a phase 1 test through its biotech subsidiary, BlueRock Therapeutics.

Bayer has also kicked off a gene therapy trial in Parkinson’s disease, this time through Asklepios BioPharmaceutical, an adeno-associated virus (AAV) gene therapy unit also known as AskBio. That trial is a phase 1b.

“For the first time, it might be possible to stop and reverse this degenerative disease and truly help patients with their high unmet medical need,” said Wolfram Carius, head of cell and gene therapy at Bayer. “The start of clinical trials represents the beginning towards a truly breakthrough treatment option to dramatically improve the lives of patients.”

RELATED: Bayer to pay $2B upfront for AskBio to drive gene therapy expansion

Parkinson’s impacts about 10 million people worldwide, according to Bayer. The disease typically begins with a tremor in one hand and progresses to symptoms including rigidity, cramping and involuntary movement of the face, arms, legs or trunk.

Few treatments are available, and the ones that are diminish in effectiveness over time. There are no therapies that actually treat the underlying cause of the disease.

Bayer is hoping its stem cell and gene therapy programs might change that and address the root cause of the devastating disorder, and they build on its gene and stem cell therapy outlook that has seen it dig deep into its pockets over the past few years.

BlueRock’s trial will eventually enroll 10 patients across the U.S. Patients will have dopamine-producing cells surgically implanted into a part of the brain called the putamen. The trial will primarily examine the safety and tolerability of DA01 cell transplantation a year after the procedure. Secondary goals include assessing evidence of cell survival following transplant and motor effects one and two years after surgery. Further safety and tolerability data will be collected at the two-year mark to determine whether the transplantation is a feasible treatment.

The AskBio trial, meanwhile, will also target the putamen with an AAV gene therapy. The virus will deliver a gene to boost GDNF gene expression, which has been shown in preclinical models to promote regeneration of neurons and motor recovery. The study has so far enrolled 10 patients since August 2020.

RELATED: Biogen bets $1B-plus on Denali's anti-LRRK2 Parkinson's program

Gene therapy veteran AskBio was acquired in 2020 for a whopping $2 billion upfront and another $2 billion in milestones. The unit has candidates in Pompe disease and congestive heart failure as well. Technology from AskBio was used to develop Novartis’ approved gene therapy Zolgensma.

BlueRock is a joint venture between Bayer and Versant Ventures that launched in 2016 to develop new stem cell therapies. Bayer bought out the remaining shares of the venture in August 2019 for $240 million.

Many Big Pharmas are making plays in Parkinson’s, hoping to bring a disease-modifying treatment to market. However, like all CNS research, the path has been a rocky one with some, like Sanofi, falling to the wayside, and will require more than a little luck to win out.