FDA Grants RMAT Designation to AskBio’s Parkinson’s Gene therapy
Berlin, Germany, and Research Triangle Park, N.C.,February 19,2025 – AskBio Inc.,a Bayer AG subsidiary,announced its investigational gene therapy,AB-1005,for parkinson’s disease (PD) received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S.Food and Drug Management (FDA).
This designation is a significant milestone for AB-1005, a gene therapy designed to slow disease progression and improve motor function in PD patients. The FDA’s decision follows a review of data from AskBio’s Phase Ib trial, a 36-month open-label, uncontrolled study.
The FDA’s decision to grant RMAT designation to AB-1005 is exciting news for people living with Parkinson’s disease and their loved ones,said Gustavo Pesquin, CEO, AskBio.This milestone could perhaps expedite the development of our crucial 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.
The Phase Ib data showed AB-1005 was well-tolerated,with no product-related serious adverse events.1 In the moderate PD cohort,the study showed trends toward advancement or stability on several clinical scales at 36 months compared to baseline. These included the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and self-reported motor diaries, along with trends in reduced Parkinson’s medications (levodopa-equivalent daily dose [LEDD]).1 Most participants in the mild PD cohort maintained stable clinical status with minimal changes in MDS-UPDRS, self-reported motor diaries, or LEDD.1
RMAT designation, granted by the FDA to regenerative therapies, including gene therapies, accelerates the development of treatments for serious or life-threatening diseases.2 The designation provides enhanced access to the FDA,potentially leading to faster drug development,rolling Biologics License Submission (BLA) review,and other expedited review processes.2
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,said Christian Rommel, Executive Vice President, Global Head of Research and Development and Member of the Pharmaceuticals Leadership team at Bayer.This is the latest exmaple of what can be achieved through the joint commitment of AskBio and Bayer to deliver breakthrough innovation for patients.
The AB-1005 Phase II REGENERATE-PD clinical trial, currently recruiting participants in the United states, is expanding. Additional sites in Germany, Poland, and the United Kingdom are expected to open in the first half of 2025.3 This multicenter trial will involve an estimated 87 subjects with moderate-stage Parkinson’s disease.
It is crucial to note that AB-1005 has not yet received regulatory approval, and its efficacy and safety remain under evaluation.
Understanding Parkinson’s Disease
Parkinson’s disease (PD) is a progressive neurodegenerative disorder significantly impacting daily life.4 The death of dopamine-producing nerve cells in the brain leads to continuous motor function loss.4 Symptoms include tremors,muscle rigidity,and slowness of movement.4 Non-motor symptoms such as fatigue, congestive issues, and depression are also common.4 The prevalence of PD has doubled in the last 25 years,5 with over 10 million people worldwide currently affected,6 making it the world’s second most prevalent
Headline: A Groundbreaking Leap in Parkinson’s Treatment: Unveiling the Promise of AB-1005 Gene Therapy
Editor: Our world today is on the brink of a revolutionary breakthrough in Parkinson’s disease treatment. Could gene therapy be the key to unlocking new hope for millions? Let’s dive into the details of AskBio’s RMAT-designated therapy, AB-1005.
Interview with Dr.Maria Thompson, Parkinson’s Disease Specialist and Gene Therapy Expert
Editor: Welcome, Dr. Thompson. We’re thrilled to discuss the recent FDA RMAT designation for AskBio’s AB-1005 gene therapy. Could you explain what this designation means for the future of Parkinson’s treatment?
Dr. Thompson: Absolutely! The RMAT designation stands for Regenerative Medicine Advanced Therapy. It’s a pivotal acknowledgment by the FDA, indicating that AB-1005 has the potential to address the ample unmet needs in Parkinson’s disease management. Essentially, this means that the therapy can benefit from an expedited review process. This acceleration could lead to quicker access to a treatment that may significantly slow disease progression and improve quality of life for patients suffering from Parkinson’s. The designation highlights the FDA’s recognition of gene therapy’s transformative potential in treating neurodegenerative disorders.
Editor: The Phase Ib trial results for AB-1005 have shown promising trends in clinical metrics. Could you elaborate on what these metrics signify for patients with Parkinson’s?
Dr. Thompson: The trial results are indeed encouraging. in the Phase Ib trial, measures such as the Movement disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and self-reported motor diaries indicated trends toward stabilization or enhancement in motor functions for patients, especially those in the moderate stage of Parkinson’s. This suggests that AB-1005 could help maintain or even improve motor skills, reducing dependence on medications like levodopa.Having well-tolerated treatment with no serious adverse events reported further bolsters confidence in its developmental trajectory.
Editor: Moving to the Phase II REGENERATE-PD clinical trial, what does the expansion into Europe mean for the global reach of this therapy?
Dr. Thompson: The expansion into countries like Germany, Poland, and the UK is a crucial step toward understanding AB-1005’s efficacy across diverse populations. It also supports Bayer and AskBio’s commitment to global innovation in regenerative medicine. by incorporating a broader demographic, we can gain insights into the therapy’s performance in varied genetic and environmental contexts, which is vital for its ultimate success. This international reach solidifies the partnership between AskBio and Bayer, ensuring a comprehensive collection of data that could expedite global regulatory approvals if the therapy proves efficacious.
Editor: With such high hopes pinned on AB-1005, what are the challenges it might face before achieving regulatory approval?
Dr. Thompson: The road to approval is complex. Key challenges include repeated validation of results across larger, more diverse populations to confirm safety and effectiveness. Regulatory bodies will need thorough data that confirm consistent benefits without significant risks. Additionally,as this involves gene therapy,long-term monitoring for unforeseen effects will be essential to ensure patient safety. The ability to adapt production and distribution mechanisms to meet potential demand, if approved, is another balance to strike.
editor: In light of the doubling prevalence of Parkinson’s over the last quarter-century, what lasting impact can gene therapies like AB-1005 have on society?
Dr. Thompson: Parkinson’s disease has significantly impacted millions of lives, doubling in prevalence and thereby straining healthcare systems and affecting families globally. Gene therapies like AB-1005 have the potential to alter this trajectory. By slowing disease progression and improving motor function,we can reduce symptoms that hinder daily living and lessen healthcare burdens. The societal implications are profound: perhaps increased productivity,reduced caregiving demands,and improved quality of life. However, long-term success will depend on accessibility and affordability, ensuring these advanced therapies become a common remedy, not a privilege.
Editor: Concluding,what advice would you give to those eagerly awaiting or advocating for breakthroughs in Parkinson’s treatment?
Dr. Thompson: I would encourage patience balanced with optimism. The path to groundbreaking treatments is frequently enough a marathon rather than a sprint, involving rigorous testing and careful consideration. Advocacy is crucial—supporting research funding and awareness can significantly influence the success of new therapies. Patience and engagement help ensure therapies like AB-1005, with their promising capabilities, reach their full potential in transforming lives.
Editor: Thank you, Dr. Thompson, for providing such insightful perspectives. Readers, we’re hopeful that continuous breakthroughs will bring us closer to conquering Parkinson’s disease. Share your thoughts and experiences with gene therapy innovations in the comments!