AskBio Begins Phase 2 Parkinson's Disease Trial Recruitment

RESEARCH TRIANGLE PARK, NC; USA – June 25, 2024 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company under the ownership of Bayer AG, has announced the start of participant recruitment for its REGENERATE-PD clinical trial. This Phase 2 study aims to evaluate the efficacy and safety of AB-1005, an investigational gene therapy using adeno-associated virus 2 (AAV2) to deliver glial cell line-derived neurotrophic factor (GDNF) for the treatment of moderate-stage Parkinson’s disease.

Dr. Alan Whone, a Consultant Senior Lecturer in Movement Disorder at Bristol Medical School and Honorary Consultant Neurologist at North Bristol Trust, UK, emphasized the importance of GDNF gene therapy. "By boosting the levels of naturally occurring GDNF, this gene therapy aims to support the survival and functioning of brain cells that deteriorate in Parkinson’s disease," he stated. Dr. Whone will serve as the European Lead Principal Investigator for the REGENERATE-PD study once it is active in the UK.

According to the Parkinson’s Foundation, over 10 million people globally are afflicted by Parkinson’s disease. Following promising results from the Phase 1b trial, which were presented at the American Association of Neurology (AAN) meeting in April, AskBio is now advancing AB-1005 to this larger Phase 2 study. Krystof Bankiewicz, MD, PhD, Scientific Chair for Parkinson’s and MSA at AskBio, expressed enthusiasm about the trial's progression. He highlighted the potential significant impact AB-1005 could have on patients with Parkinson’s disease.

Earlier in January, AskBio announced that the Phase 1b trial of AB-1005 had met its primary endpoint, focusing on the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. The therapy was well tolerated, with no serious adverse events related to AB-1005 reported in the 11 patients followed for 18 months.

Beyond Parkinson’s disease, AskBio is also investigating the application of GDNF gene therapy for other conditions. The company is currently enrolling patients in the US with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase 1 trial to evaluate the preliminary safety, tolerability, and efficacy of GDNF therapy for this rapidly progressing disorder.

AB-1005 remains an investigational gene therapy, not yet approved by any regulatory authority, with its full efficacy and safety still under evaluation.

Parkinson’s disease is a progressive neurological disorder characterized by the loss of nerve cells in the brain, leading to reduced dopamine levels. Upon diagnosis, it is estimated that patients have already lost 50-80% of their dopamine-producing neurons. This loss results in a progressive decline in motor function, manifesting as tremors, muscle rigidity, and slow movements. Even with medication, Parkinson’s symptoms can vary throughout the day. There is currently no cure for Parkinson’s disease, and the effectiveness of existing treatments often diminishes over time.

The REGENERATE-PD study is a Phase 2, randomized, double-blind, surgery-controlled trial to evaluate the efficacy and safety of intraputaminal AAV2-GDNF in adults aged 45-75 with moderate-stage Parkinson’s disease. Participants will receive either a bilateral, image-guided infusion of AAV2-GDNF into the putamen or undergo a control surgery. The trial plans to enroll approximately 87 participants across sites in the US, UK, and Europe.

AB-1005, which utilizes an adeno-associated viral vector to deliver the GDNF gene, aims to achieve stable and continuous expression of GDNF in specific brain regions following direct neurosurgical injection with MRI-monitored delivery. GDNF is known to enhance the survival and differentiation of dopaminergic neurons and increase dopamine uptake, making it a potential treatment for conditions marked by the degeneration of these neurons.

AskBio, a pioneer in gene therapy and a subsidiary of Bayer AG, focuses on developing transformative treatments for various diseases. The company operates globally, with headquarters in Research Triangle Park, North Carolina, and Edinburgh, Scotland.