ROCHESTER, Minn.--(BUSINESS WIRE)--RION, at the forefront of exosome innovation and therapeutic application, has officially commenced a Phase 1b study aimed at assessing the efficacy and safety of Purified Exosome Product™ (PEP™), its exclusive exosome regenerative therapeutic, for the management knee osteoarthritis (Knee OA).
Each year, Knee OA affects approximately 364 million people worldwide and is associated with over one million hospitalizations annually in the U.S., primarily for total joint replacement. Knee OA represents a substantial burden on U.S. public and private healthcare systems, with estimates ranging from $5.7 billion to $15 billion in cost2. This trial serves as a key component of RION's broader efforts aimed at developing regenerative therapies in areas of unmet clinical need and chronic disease, such as Knee OA. This is the first FDA approved trial evaluating therapeutic exosomes in the orthopedic space.
RION's Phase 1b trial represents a significant step forward in the development of a regenerative biologic for Knee OA. This open label, randomized, multi-center study will involve 24 patients across the United States. Patients will receive single intra-articular injections of PEP™ in escalating doses and followed for safety and signals of efficacy.
Peer-reviewed in vitro data found RION’s PEP™ is an efficient chondroprotective and regenerative agent by enhancing proliferation, attenuating apoptosis and modulating inflammation. Further, a preclinical study in a validated animal model of post-traumatic osteoarthritis confirmed the potential efficacy of PEP™ combined with Hyaluronic Acid in support of RION’s osteoarthritis clinical program3. A positive outcome in the Phase 1b trial will pave the way for future studies with PEP™ in Knee OA followed by the submission of a Biologics License Application (BLA).
“The progress we've made in advancing PEP™, reflects our deep commitment to developing powerful exosome therapies that unleash the body’s ability to heal from within,” said Dr. Atta Behfar, Co-founder of RION. “Not only will this study address a critical unmet need in knee osteoarthritis, but it serves as a pivotal moment for RION. From manufacturing to therapeutic innovation, we have been able to disrupt barriers in scaling and application of exosome therapies. With focus on disrupting paradigms that drive up the cost of biologics-based technology, we are establishing exosomes as a new generation of therapies endowed with the potential to truly transform the way we treat complex and chronic diseases. Guided by the FDA, RION is committed to bringing exosome-based therapies to patients as quickly and safely as possible to transform the management of this debilitating condition.”
About RION
RION was born out of the Mayo Clinic after two decades of research and innovation, in the Van Cleve Cardiac Regenerative Medicine Program, leading to the discovery that the driving force behind tissue repair was exosome signaling. 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 penitential 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. 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. As our efforts in wound healing advance, multiple IND-enabling efforts in musculoskeletal, cardiovascular disease, pulmonary disease, women’s health disorders and drug delivery are creating solutions where current standards of care cannot address unmet clinical needs.
About Knee Osteoarthritis
Osteoarthritis is by far the most common form of arthritis, affecting more than 32.5 million Americans and more than 350 million individuals worldwide4. About half of knees with ACL injuries develop osteoarthritis within 5 to 15 years. 78 million Americans are projected to have osteoarthritis by the year 20405. Symptomatic knee osteoarthritis is particularly prevalent and disabling, with 40% of men and 47% of women developing knee osteoarthritis in their lifetimes6. The burden of osteoarthritis is enormous, and the need for treatments that reduce pain and attendant disability for persons with osteoarthritis is critical.
RION's journey is paved with unwavering determination. We seek to break the cycle of pain, loss, and economic burden through application of PEP™, our pioneering regenerative exosome technology. We will remain undeterred in our pursuit to have PEP™ become the first exosome biologics therapy to be approved for the treatment of Knee Osteoarthritis.
References
- https://acrjournals.onlinelibrary.wiley.com/doi/abs/10.1002/acr.25158
- https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8153072/
- https://pubmed.ncbi.nlm.nih.gov/36255073/
- https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8248516/#:~:text=Osteoarthritis%20(OA)%20is%20the%20most,sites%20being%20knee%20and%20hip
- https://www.arthritis.org/osteoarthritis-patient-education
- https://www.sciencedirect.com/science/article/pii/S1063458413007607#:~:text=The%20lifetime%20risk%20of%20developing,weight%20or%20are%20underweight18
