AskBio Garners RMAT Designation for Parkinson Disease Gene Therapy AB-1005
The FDA’s decision was based on a review of findings from a phase 1b clinical trial.
Bayer subsidiary Asklepios BioPharmaceutical (AskBio)'s AB-1005 (AAV2-GDNF), an investigational AAV vector serotype 2 (AAV2)-based gene therapy intended to treat Parkinson disease (PD), has received regenerative medicine advanced therapy (RMAT) designation from the FDA.1
The FDA’s decision was based on a review of findings from a phase 1b clinical trial (NCT04167540), which has passed its primary completion date but is continuing to collect long-term follow-up data. Notably, AB-1005 is also now being evaluated for PD in a sham-controlled phase 2 clinical trial (REGENERATE-PD; NCT06285643),which recently began the process of randomly assigning enrolled participants in the United States in January 2025.2 AskBio anticipates that REGENERATE-PD will open additional sites for enrollment in the US, Germany, Poland, and the United Kingdom before the midway point of 2025.
“The FDA’s decision to grant RMAT designation to AB-1005 is exciting news for people living with PD and their loved ones,” Gustavo Pesquin, MBA, the chief executive officer of AskBio, said in a statement.1 “This milestone could potentially expedite the development of our important investigational gene therapy program, and it highlights our promising data and the potential of AB-1005 for patients and the medical community. We look forward to working closely with the FDA to accelerate our program.”
At the International Congress of Parkinson’s Disease and Movement Disorders, held in Philadelphia, PA, from September 27 to October 1, 2024, AskBio presented updated data from the phase 1b clinical trial evaluating AB-1005. The company highlighted that at 36 months posttreatment the data showed trends for improvement in patients in the moderate PD cohort on the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and motor diaries. At the same time point, patients in this cohort also demonstrated trends for reductions in Parkinson’s medications (levodopa-equivalent daily dose [LEDD]). Not much change was recorded on the MDS-UPDRS, self-reported PD motor diary, or LEDD for the majority of patients in the mild PD cohort, leading AskBio to describe an “overall stable clinical status” for these participants. In terms of safety, no serious adverse events occurred that were deemed related to AB-1005 itself.
CGTLive® previously covered the design of the REGENERATE-PD trial, which was launched on June 11, 2024. The randomized, double-blind, sham surgery-controlled clinical trial is expected to enroll approximately 87 patients in total and will utilize masking for participants, care providers, investigators, and outcomes assessors. One primary end point for the trial consists of the results of Motor symptoms-MDS-UPDRS parts 2 and 3 and another primary end point consists of PD Motor Diary Non-Motor symptoms-MDS-UPDRS part 1. A third primary end point is the Parkinson's Disease Questionnaire (PDQ-39) LEDD.
In addition to AB-1005, AskBio is also developing AB-1002, a gene theraphy product intended to treat congestive heart failure.3 Notably, earlier February of this year, the company announced that the first patient in Europe had been randomly assigned in the phase 2 GenePHIT clinical trial (NCT05598333) evaluating AB-1002. The first patient in the US had previously been randomly assigned last year.
“Cardiovascular disease is the most common cause of death in Europe, claiming nearly 4 million lives each year, and it accounts for 45 percent of all deaths in females and 39 percent of all deaths in males,” GenePHIT principal investigator and steering committee member Timothy D. Henry, MD, MSCAI, said in a February 2025 statement.3 “As society ages, the burden heart failure places on individuals and healthcare systems will only increase unless a solution is found. Today’s announcement is important as it represents meaningful progress in the development of a potentially disease-modifying therapy.”
