RION Announces First Patient Enrolled in Phase 1b Clinical Study Evaluating Purified Exosome Product™ (PEP™) for Knee Osteoarthritis

ROCHESTER, Minn.--(BUSINESS WIRE)--RION, a clinical-stage regenerative medicine company at the forefront of exosome-based therapeutics, today announced the enrollment of the first patient in its Phase 1b clinical study evaluating Purified Exosome Product™ (PEP™) for the treatment of Knee Osteoarthritis (OA). This milestone highlights RION’s commitment to advancing innovative regenerative therapies that address unmet medical needs.

Knee Osteoarthritis is the most common form of arthritis, affecting approximately 364 million people worldwide each year (1). In the United States alone, Knee OA is associated with more than one million hospitalizations annually, primarily for total joint replacement, contributing to healthcare costs ranging from $5.7 billion to $15 billion (2). Despite its prevalence, current treatment options primarily manage symptoms rather than addressing the underlying biology of the disease, leaving a critical need for novel therapies that can restore joint function.

Phase 1b Study Overview

RION's Phase 1b study represents a major step forward in developing regenerative therapies for Knee OA. This open-label, multi-center trial will enroll 24 patients across the United States. Participants with symptomatic unilateral Knee OA will receive single intra-articular injections of PEP™ in escalating doses to evaluate safety, tolerability, and preliminary efficacy, including signals of cartilage regeneration and inflammation reduction.

“The enrollment of the first patient in this trial represents an exciting milestone for RION and the field of regenerative medicine,” said Atta Behfar, MD, PhD, co-founder of RION. “PEP™ offers a groundbreaking approach to addressing the unmet needs of knee osteoarthritis patients by leveraging the body’s natural repair mechanisms. This trial is the first step in advancing PEP™ as a transformative therapy that goes beyond symptom management to target the underlying causes of this debilitating condition.”

About RION

RION was established out of Mayo Clinic after two decades of research and innovation. RION, located in Rochester, MN, is internationally recognized for its pioneering advancements in isolating and mass-producing platelet-derived regenerative exosomes into shelf stable PEP™.

RION is rewriting the regenerative medicine playbook. Our cutting-edge proprietary biomanufacturing platform crafts the future of regenerative therapy, unlocking the potent secrets within these tiny cellular messengers. RION’s regenerative PEP™ technology will be integral to the therapeutic exosome revolution.

About PEP™

RION's Purified Exosome Product™ (PEP™) is a shelf stable product in a lyophilized powder derived from human platelets that contains stabilized platelet-derived regenerative exosomes. Discovered at the Mayo Clinic Van Cleve Cardiac Regenerative Medicine Program, PEP™ is an exosome therapeutic that is designed to promote cell growth and formation of new blood vessels, while also reducing inflammation and protecting cells. RION and its scientific collaborators have performed extensive research showing the potential of PEP™ to heal a wide array of damaged tissue. The company is currently evaluating PEP™ in preclinical and clinical studies for several indications. While our focus remains on wound healing, multiple IND-enabling efforts in musculoskeletal, cardiovascular disease, pulmonary disease and women’s health disorders are creating new solutions where current standards of care cannot address unmet clinical needs.

About Knee Osteoarthritis

Osteoarthritis is the most common form of arthritis, impacting more than 350 million individuals globally, including over 32.5 million Americans (3). Symptomatic Knee OA is highly prevalent, with 40% of men and 47% of women developing the condition during their lifetimes (4). The disease often leads to significant pain, disability, and healthcare costs, highlighting the urgent need for therapies that address both symptoms and the underlying drivers of joint degeneration.

References

1. https://acrjournals.onlinelibrary.wiley.com/doi/abs/10.1002/acr.25158
2. https://pubmed.ncbi.nlm.nih.gov/34054301/
3. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8248516/#:~:text=Osteoarthritis%20(OA)%20is%20the%20most,sites%20being%20knee%20and%20hip
4. https://www.sciencedirect.com/science/article/pii/S1063458413007607#:~:text=The%20lifetime%20risk%20of%20developing,weight%20or%20are%20underweight18