Asklepios BioPharmaceutical (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer, has announced initiation of recruitment to REGENERATE-PD, a Phase 2 clinical study assessing efficacy and safety of AB-1005, an investigational adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of moderate-stage Parkinson’s disease.
AskBio
“By enhancing levels of a naturally occurring growth factor, glial cell line-derived neurotrophic factor (GDNF) gene therapy is intended to promote the survival and functioning of vulnerable brain cells that degenerate in Parkinson’s disease”, said Alan Whone, MD, PhD, consultant senior lecturer in Movement Disorder, Bristol Medical School, and Honorary Consultant Neurologist at North Bristol Trust, UK. “The advancement of AB-1005 is a significant milestone in the development of a gene therapy for Parkinson’s disease and has the potential to bring an effective treatment one step closer to patients.” Dr Whone will act as European Lead Principal Investigator (PI) on REGENERATE-PD once the programme becomes active in the UK.
According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease.
“Following the encouraging results from the Phase 1b study and the presentation of 18-month data at the American Association of Neurology (AAN) meeting in April, we are excited to be progressing AB-1005 to this larger, Phase 2 study,” said Krystof Bankiewicz, MD, PhD, ccientific chair, Parkinson’s and MSA, AskBio. “This latest advancement highlights our confidence in the potential of AB-1005 to provide a transformative impact for patients with Parkinson’s disease.”
In January, AskBio announced that the Phase 1b trial of AB-1005 met its primary endpoint, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. The investigational gene therapy for the treatment of Parkinson’s disease was well tolerated with no serious adverse events that were considered related to AB-1005 in all 11 patients at 18 months.
AskBio is also exploring GDNF therapy beyond Parkinson’s disease and is currently enrolling patients in the US with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase 1 trial to assess the preliminary safety, tolerability, and efficacy of GDNF therapy for this rapidly progressing condition.
AB-1005 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.