Bayer gains ground in two-fronted Parkinson's offensive

 Bayer is preparing to start a phase 3 trial of its cell therapy candidate bemdaneprocel for Parkinson's disease after discussing phase 1 results with the FDA, while its experimental gene therapy is also advancing into a phase 2 study.

According to Bayer's BlueRock Therapeutics subsidiary – which is running the exPDite-2 trial of bemdaneprocel due to get underway in the first half of this year – it will be the first phase 3 study of an allogeneic pluripotent stem cell-based therapy to treat Parkinson's.

Bemdaneprocel is administered by surgical transplantation into a part of the brain known as the putamen, in the hope of grafting dopamine-producing cells that can bolster levels of the neurotransmitter, which is depleted in Parkinson's patients. The study will compare the cell therapy to a sham surgical procedure.

The move into phase 3 follows positive 24-month data from the phase 1 exPDite study in 12 patients, reported last September, which backed up the safety and tolerability of the therapy and showed that the transplanted cells were surviving and growing in the brain.

Moreover, the results also revealed trends towards improvement in motor function assessed using the MDS-UPDRS Part II and III scales and the Hauser PD Diary.

exPDite-2 will enrol a target of 102 patients with moderate Parkinson's symptoms, measuring the change from baseline to week 78 in Hauser PD Diary measure of 'on' time without dyskinesia – in other words, the period where muscle symptoms associated with Parkinson's are controlled without involuntary, uncontrolled movements.

The FDA awarded bemdaneprocel a regenerative medicine advanced therapy (RMAT) designation last year, which can accelerate the development of new treatments by offering increased flexibility in clinical trials, early FDA engagement, and priority review.

First patient randomised in gene therapy trial

Meanwhile, another Bayer subsidiary – AskBio – has started randomising the first patients in its phase 2 trial of AB-1005 (AAV2-GDNF), which delivers a gene into the putamen region of the brain that expresses a growth factor called GDNF that is thought to promote the survival of dopaminergic neurons.

The start of a phase 2 REGENERATE-PD study follows 36-month results from a phase 1b trial of AB-1005, reported last year, which demonstrated a favourable safety profile and continued positive trends in assessed clinical measures of AB-1005 with no associated serious adverse events.

Once again, there were trends towards improved MDS-UPDRS and diary scores, along with signs that patients needed lower levels of the standard Parkinson's medication levodopa to control motor symptoms.

AskBio is also exploring AB-1005 beyond Parkinson's and is currently enrolling participants in the US with the Parkinsonian subtype of multiple system atrophy (MSA-P) into a phase 1 trial.

Bayer agreed a $600 million deal in 2019 to take full control of BlueRock, which was formed in 2016 as a joint venture between the drugmaker's Leaps by Bayer venture capital group and private equity firm Versant.

It followed that deal with a $4 billion agreement to acquire AskBio a year later, cementing its ambition in the regenerative medicines category. Bayer has said it wants both companies to operate independently in order to preserve their entrepreneurial culture.

https://pharmaphorum.com/news/bayer-gains-ground-two-fronted-parkinsons-offensive