Hope Biosciences Awarded FDA RMAT Designation for Multiple Sclerosis Stem Cell Therapy
The FDA designation follows promising Phase II trial results for Hope Biosciences’ stem cell therapy in relapsing-remitting multiple sclerosis.
Hope Biosciences has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for its adipose-derived mesenchymal stem cells (HB-adMSCs) for the treatment of relapsing-remitting multiple sclerosis (RRMS).
This designation is based on research by the Hope Biosciences Research Foundation (HBRF), a non-profit clinical research organization.
RMAT designation is granted to therapies that demonstrate preliminary clinical evidence of addressing serious or life-threatening conditions with significant unmet medical needs. The designation offers eligibility for priority review or accelerated approval and involves enhanced collaboration with the FDA.
“Receiving the first RMAT designation for multiple sclerosis is a tremendous acknowledgment of our technology’s potential to impact the world of debilitating autoimmune conditions,” says Donna Chang, CEO, Hope Biosciences. “HBRF has been an instrumental partner, leveraging their unparalleled expertise in cell therapy as well as their proven ability to deliver compassionate care tailored to our community’s needs through pioneering, patient-centered protocols. It is a privilege to collaborate with them in advancing hope for patients.”
This designation follows positive top-line results from a Phase II clinical trial conducted at HBRF in 2023 and 2024. The trial, NCT05116540, was the first globally to administer pure mesenchymal stem cells in high quantities over repeated treatments to MS patients. The randomized, double-blind, single-center study enrolled 24 participants. Over 32 weeks, the treatment group received six intravenous infusions of 200 million stem cells each, totaling 1.2 billion cells.
“The trial successfully met its primary endpoint, and the results clearly demonstrate that HB-adMSCs can be effective in a complex, variable condition like RRMS,” explains Ridhima Vij, Ph.D., Clinical Research Scientist, HBRF. “There were statistically significant improvements in both physical and mental health for the treatment group compared to the placebo group as determined by the MS Quality of Life-54 Instrument. This tool is a multidimensional quality of life measure that combines general and MS-specific points related to fatigue, physical and cognitive function, sexual impairment, pain, energy, mobility, level of disability, and other considerations – all contributors to quality of life we keep front of mind, when we think about how a therapeutic might revolutionize how we approach chronic degenerative disease.”
Multiple sclerosis (MS) affects over 2 million people globally and about 400,000 in the United States. MS is an autoimmune disorder where the immune system attacks the myelin sheath (the protective covering around nerve fibers), leading to neurological symptoms that vary based on the nerves affected. Relapsing-remitting MS is characterized by alternating periods of relapse and remission, with progression and severity differing by patient. The disease is typically diagnosed in individuals in their 30s and 40s and disproportionately affects women. The estimated lifetime cost of MS approaches $5 million per patient.
“We are grateful to FDA for recognizing tremendous need for development of new solutions for multiple sclerosis,” concludes Chang. “We remain focused on getting treatment to people in need and will move rapidly to collaborate with FDA on advancing access, including filing our Biologics License Application. We also look forward to participation in the Chemistry, Manufacturing, and Controls Development and Readiness Pilot program, at FDA’s invitation.”
About HB-adMSCs
HB-adMSCs are Hope Biosciences’ proprietary adipose-derived mesenchymal stem cells. The company reports its process allows for sustained, high-volume administration with a high degree of cell viability. Over the past decade, thousands of patients, ranging from infants to older adults, have received HB-adMSCs through more than 50 FDA-authorized clinical trials and Expanded Access protocols, as well as under the Federal Right to Try Act.
About Hope Biosciences
Hope Biosciences is a cGMP-compliant biopharmaceutical research and manufacturing company focused on developing stem cell solutions for incurable conditions. The company also provides private stem cell banking services for both adults and newborns. More information is available at hope.bio.
About Hope Biosciences Research Foundation (HBRF)
HBRF is a non-profit clinical research organization specializing in adult cellular therapeutics for chronic degenerative and other challenging conditions. Since 2020, HBRF has obtained FDA authorization for more than 45 clinical protocols across a range of diseases. For more information, visit hopebio.org.
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