AskBio Inc., a gene therapy company and subsidiary of Bayer AG, is advancing its investigational gene therapy, AB-1005, for Parkinson's disease (PD) with two significant milestones. The U.S. Food and Drug Administration (FDA) has granted AB-1005 Regenerative Medicine Advanced Therapy (RMAT) designation, and the Phase II REGENERATE-PD clinical trial has enrolled and randomized its first participants. These developments signal progress in the search for effective treatments for this debilitating neurodegenerative disorder affecting over 10 million people worldwide.
RMAT Designation for AB-1005
The FDA's RMAT designation is reserved for regenerative therapies showing preliminary clinical evidence of addressing unmet medical needs for serious or life-threatening conditions. This designation provides AskBio with enhanced access to the FDA, potentially accelerating the development and review process for AB-1005 through intensive guidance, rolling Biologics License Application (BLA) review, and other expedited measures.
Gustavo Pesquin, CEO of AskBio, expressed optimism about the RMAT designation, stating, "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."
The RMAT designation was supported by data from AskBio’s Phase Ib trial, which demonstrated a favorable safety profile and continued positive trends in assessed clinical outcome measures of AB-1005 (formerly known as AAV2-GDNF) with no product-related serious adverse events.
REGENERATE-PD Phase II Trial
In addition to the RMAT designation, AskBio has announced the randomization of the first participants in the Phase II REGENERATE-PD clinical trial. This randomized, double-blind, sham-controlled trial is designed to evaluate the safety and efficacy of AB-1005 in adults aged 45-75 years with moderate-stage Parkinson's disease. The trial aims to enroll approximately 87 participants across clinical centers in the United States, Germany, Poland, and the United Kingdom.
AB-1005 is delivered directly to the putamen, a brain region involved in motor control, via neurosurgical injection. The gene therapy utilizes an adeno-associated viral vector serotype 2 (AAV2) to deliver the gene for glial cell line-derived neurotrophic factor (GDNF). GDNF is a protein that promotes the survival and function of dopaminergic neurons, which are progressively lost in Parkinson's disease.
Clinical Data Supporting AB-1005
Data from the Phase Ib trial of AB-1005, presented at the International Congress of Parkinson's Disease and Movement Disorders, showed that administration of AB-1005 was well tolerated with no attributed serious adverse events. The Moderate PD cohort showed trends for improvement or stability on several motor scales at 36 months, including Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) and motor diaries, and trends in reductions in Parkinson's medications (levodopa-equivalent daily dose [LEDD]). Most participants in the Mild PD cohort showed an overall stable clinical status with little change in MDS-UPDRS, the self-reported PD motor diary, or LEDD.
Addressing Unmet Needs in Parkinson's Disease
Parkinson's disease is a progressive neurodegenerative disorder characterized by the loss of dopaminergic neurons in the brain. This leads to motor symptoms such as tremors, rigidity, and slowness of movement, as well as non-motor symptoms like fatigue and depression. Current treatments primarily focus on managing symptoms, with no cure available. The global prevalence of PD has doubled in the last 25 years, with over 10 million people affected worldwide, highlighting the urgent need for new and effective therapies.
Christian Rommel, Executive Vice President, Global Head of Research and Development at Bayer, emphasized the potential of AB-1005, stating, "The RMAT designation for AB-1005 underscores the high unmet medical need and the potential of this investigational gene therapy to make a difference for patients with Parkinson's disease."
