
AskBio, a gene therapy subsidiary of Bayer has received the US Food and Drug Administration (FDA) Fast Track Designation for its investigational Parkinson’s disease (PD) drug, AB-1005.
The experimental drug also received the Innovation Passport, the UK Medicines and Healthcare Products Regulatory Agency (MHRA’s) innovative medicine designation, to treat PD.
AB-1005 is a gene therapy developed based on adeno-associated viral vector serotype 2 containing the human glial cell line-derived neurotrophic factor (AAV2-GDNF) transgene.
It works by enabling a stable and continuous expression of GDNF in the regions of the brain after direct neurosurgical injection with MRI-monitored convection-enhanced delivery.
GDNF is a homodimer that is a remotely related member of the growth factor-β superfamily, which has been evaluated as a potential treatment for diseases like PD.
AskBio Parkinson’s and MSA scientific chair Krystof Bankiewicz said: “These designations clearly underscore the importance of developing innovative therapies for those living with Parkinson’s disease, where a significant unmet need still exists.
“They further highlight the willingness of key regulatory bodies to support the accelerated development of AB-1005 with a focus on the potential benefit to patients.”
AskBio chief development officer and chief medical officer Canwen Jiang said: “We look forward to advancing our Phase II REGENERATE-PD clinical trial, which is currently enrolling patients in the US with sites in the European Union and UK planned to open later this year.”
Earlier this year, AskBio reported positive results for AB-1005, from the 18-month Phase 1b clinical trial, which met its primary objective of evaluating the safety of the drug.
The gene therapy company is currently enrolling participants in REGENERATE-PD, a Phase 2, randomised, double-blind, surgery-controlled clinical trial in the US.
The Phase 2 trial will evaluate the efficacy and safety of intraputaminal AB-1005 in 87 adults, for the treatment of moderate-stage PD.
Subjects will be randomised to receive either bilateral image-guided infusion of AB-1005 into the putamen, single dose (active treatment arm), or bilateral partial twist holes (control arm).
Furthermore, AskBio is enrolling patients for a Phase 1 trial in the US, to assess the preliminary safety, tolerability, and efficacy of GDNF therapy in multiple system atrophy (MSA-P).
Bayer pharmaceuticals division R&D global head Christian Rommel said: “We are excited about the opportunity to potentially accelerate the development of AB-1005, leveraging the frequent interaction with relevant regulatory bodies.
“The granted designations for AB-1005 highlight the demand to advance novel therapeutic modalities, like gene therapy, for people living with the debilitating effects of Parkinson’s disease.”