Leading U.S. stem cell banking and cellular therapeutic manufacturer Hope Biosciences announces the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for Hope Biosciences’ adipose-derived mesenchymal stem cells (HB-adMSCs) for treatment of patients living with relapsing-remitting multiple sclerosis (RRMS), based on research conducted by Sugar Land-based non-profit clinical research organization Hope Biosciences Research Foundation (HBRF).

To qualify for RMAT, a therapy must demonstrate preliminary clinical evidence of addressing a serious or life-threatening condition with significant unmet medical needs. The process requires compelling proof that the therapeutic has potential to substantially improve patient outcomes where existing treatments fall short. RMAT designation grants eligibility for priority review or accelerated approval, including enhanced FDA collaboration.

“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.”

RMAT designation for HB-adMSCs in RRMS comes after positive top-line results in a Phase II clinical trial conducted at HBRF throughout 2023 and 2024, the first trial globally to administer pure mesenchymal stem cells to MS patients in such high quantities over repeat treatments. That trial (NCT05116540) was a balanced randomized, double-blind, single center study that enrolled 24 participants. Over the course of 32 weeks, the treatment group received six intravenous infusions of 200 million stem cells, for a total of 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.”

More than 2 million people worldwide and about 400,000 people in the United States suffer from MS, a condition in which the immune system attacks a protective sheath around nerve fibers, called “myelin.” As myelin deteriorates, the brain and spinal cord are affected. Symptoms are diverse, depending on which nerves are damaged and to what degree. RRMS is a subtype characterized by periods of remission and periods of relapse. Developmental timelines also vary widely, ranging from periods of remission to loss of ability to walk. Typically diagnosed in the 30’s and 40’s, MS disproportionately affects women. Economically, MS is second only to heart failure in fiscal burden, with an estimated cost of nearly $5 million over the course of a lifetime.

“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.”