RION Starts Phase 1b Study on PEP™ for Knee Osteoarthritis

ROCHESTER, MN, USA I September 10, 2024 I RION, a leader in exosome innovation and therapeutic development, has initiated a Phase 1b clinical trial to evaluate the safety and effectiveness of its Purified Exosome Product™ (PEP™) for treating knee osteoarthritis (Knee OA). Knee OA is a prevalent condition affecting around 364 million people globally and results in over a million hospitalizations annually in the United States, mainly for total joint replacements. The financial burden on U.S. healthcare systems from Knee OA is significant, estimated to range between $5.7 billion and $15 billion.

This trial is a crucial part of RION’s strategy to develop regenerative therapies for chronic diseases and unmet clinical needs, such as Knee OA. Notably, this is the first FDA-approved trial to assess the use of therapeutic exosomes in orthopedics. The Phase 1b study marks a major advancement in creating a regenerative biologic for Knee OA. It is an open-label, randomized, multi-center study involving 24 patients across the U.S. Participants will receive single intra-articular injections of PEP™ in increasing doses and will be monitored for safety and signs of efficacy.

In vitro peer-reviewed studies have demonstrated that RION's PEP™ is a potent chondroprotective and regenerative agent, promoting cell proliferation, reducing apoptosis, and modulating inflammation. Moreover, preclinical research in an animal model of post-traumatic osteoarthritis supported the potential effectiveness of PEP™ when combined with Hyaluronic Acid. A successful outcome in this Phase 1b trial will pave the way for future studies and the eventual submission of a Biologics License Application (BLA).

Dr. Atta Behfar, Co-founder of RION, emphasized the significance of this progress, stating, "The advancements with PEP™ reflect our commitment to developing powerful exosome therapies that harness the body's intrinsic healing capabilities." He noted that this study not only aims to address a critical unmet need in knee osteoarthritis but also represents a pivotal moment for RION. The company has overcome many barriers in the scaling and application of exosome therapies, aiming to disrupt the high costs associated with biologics-based technology. "We are establishing exosomes as a new generation of therapies with the potential to transform the treatment of complex and chronic diseases," he added. Under FDA guidance, RION is dedicated to bringing exosome-based therapies to patients swiftly and safely to transform the management of this debilitating condition.

RION originated from two decades of research and innovation at the Mayo Clinic, particularly within the Van Cleve Cardiac Regenerative Medicine Program. This program led to the discovery that exosome signaling is crucial for tissue repair. Located in Rochester, MN, RION is globally recognized for its advancements in isolating and mass-producing platelet-derived regenerative exosomes into the shelf-stable PEP™.

RION's innovative biomanufacturing platform is shaping the future of regenerative therapy by leveraging the potential of these cellular messengers. The company’s PEP™ technology is expected to be a cornerstone in the therapeutic exosome revolution.

PEP™ is a shelf-stable lyophilized powder derived from human platelets containing stabilized regenerative exosomes. Designed to promote cell growth, new blood vessels formation, reduce inflammation, and protect cells, PEP™ has shown promise in healing various damaged tissues. RION is currently evaluating PEP™ in multiple preclinical and clinical studies for several conditions, including musculoskeletal, cardiovascular, pulmonary diseases, women's health disorders, and drug delivery.

Knee OA is the most common form of arthritis, affecting over 32.5 million Americans and more than 350 million people worldwide. Injuries like ACL tears can significantly increase the risk of developing Knee OA. By 2040, an estimated 78 million Americans are expected to have osteoarthritis. Symptomatic knee osteoarthritis is particularly disabling, with a significant percentage of men and women likely to develop the condition in their lifetimes. The condition's burden is immense, highlighting the critical need for effective treatments to reduce pain and disability.

RION is determined to break the cycle of pain and economic burden associated with Knee OA through its pioneering PEP™ technology. The company remains committed to making PEP™ the first exosome biologics therapy approved for Knee OA treatment.

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