RION Announces Initiation of Phase 2A Clinical Study for Purified Exosome Product™ (PEP™) in Diabetic Foot Ulcers

 // News

January 30  

  • RION’s Phase 2A clinical trial focuses on evaluating the safety and efficacy of Platelet Exosome Product™ (PEP™) combined with TISSEEL fibrin sealant for treating Diabetic Foot Ulcers (DFU).
  • Diabetic Foot Ulcers, affecting 18.6 million people worldwide each year, significantly impact quality of life and impose substantial economic burdens on healthcare systems.
  • The trial is a key part of RION’s broader efforts in regenerative medicine, aiming to address unmet needs in the treatment of chronic conditions such as DFU.

ROCHESTER, Minn. — RION, a clinical-stage regenerative medicine company, at the forefront of the exosome therapeutic revolution, has officially commenced a Phase 2A study aimed at assessing the efficacy and safety of its exclusive exosome regenerative therapeutic, known as Purified Exosome Product™ (PEP™), for the management of Diabetic Foot Ulcers (DFU).

Diabetic Foot Ulcers affect approximately 18.6 million people worldwide each year1 and are associated with increasing rates of amputation and death​​. The economic burden of DFUs is substantial, imposing $13 billion in cost on the US public and private healthcare systems.2 The trial is a key part of RION’s broader efforts in regenerative medicine, aiming to address unmet needs in the treatment of chronic conditions such as DFU.

Building on the success of the Phase 1B study conducted at Mayo Clinic, RION’s Phase 2A trial represents a significant step forward in the development of a regenerative biologic for Diabetic Foot Ulcers. This prospective, randomized, multi-center study will involve 40 patients across the United States. Patients will be divided into two cohorts: one receiving standard care and the other treated with PEP™ exosomes, enabling a thorough comparison of outcomes.

A positive outcome in the Phase 2A trial has the potential to pave the way for a pivotal study followed by the submission of a Biologics License Application (BLA). PEP™ addresses a significant unmet medical need in the realm of diabetic wound care.

Dr. Atta Behfar, Co-founder of RION, said, “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. Not only will this study address a critical unmet need in chronic wound care, but it’s a pivotal moment for RION. We are disrupting the boundaries of exosome technology, to develop a new generation of therapies endowed with the potential to truly transform the way we treat complex and incurable diseases.”

In the United States, the treatment of DFUs remains a significant challenge in the medical community, with no new biologics approved by the US FDA for diabetic wounds since the late 1990s3. RION is committed to bringing exosome-based therapies to patients as quickly and safely as possible to transform the management and outcomes of this devastating condition​​​​.

About RION

RION was born out of the Mayo Clinic after two decades of research and innovation at that institution. RION is located in Rochester, MN and 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. 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 Diabetic Foot Ulcers (DFU)

Diabetic foot ulcers (DFUs) are not just wounds. These slow-healing sores on the feet are a brutal consequence of diabetes, inflicting a triple threat: excruciating pain, crippling mobility, and the specter of amputation. The numbers paint a grim picture. Up to 25% of people with diabetes will face a DFU in their lifetime4, and for almost a quarter of them also limb amputation5. In fact, every three and a half minutes in the US, a DFU becomes an amputation6. This isn’t just a statistic, it’s a story of lives shattered, livelihoods lost, and families forever changed. Many patients endure the agony of recurrences, their lives trapped in a cycle of healing and reopening wounds. And sadly, the shadow of mortality looms large. 40% of DFU sufferers will succumb to the condition7, making it a pervasive silent killer of diabetic communities. The economic cost is equally staggering. DFUs drain healthcare systems, piling on direct treatment expenses and crippling indirect costs from lost productivity and long-term disability.

RION’s journey is paved with unwavering determination. We seek to break the cycle of pain, loss, and economic burden through application of our next-generation regenerative exosome technology. We will remain undeterred in our pursuit to have PEP™ become the first biologic to be approved for the treatment of DFU in over 25 years.

1.Armstrong DG, Tan TW, Boulton AJM, Bus SA. Diabetic Foot Ulcers: A Review. JAMA. 2023 Jul 3;330(1):62-75. doi: 10.1001/jama.2023.10578. PMID: 37395769; PMCID: PMC10723802.
2.Rice JB, Desai U, Cummings AK, Birnbaum HG, Skornicki M, Parsons NB. Burden of diabetic foot ulcers for medicare and private insurers. Diabetes Care. 2014;37(3):651-8. doi: 10.2337/dc13-2176. Epub 2013 Nov 1. Erratum in: Diabetes Care. 2014 Sep;37(9):2660. PMID: 24186882.
3.Fang RC, Galiano RD. A review of becaplermin gel in the treatment of diabetic neuropathic foot ulcers. Biologics. 2008 Mar;2(1):1-12. doi: 10.2147/btt.s1338. PMID: 19707423; PMCID: PMC2727777.
4.Yazdanpanah L, Shahbazian H, Nazari I, Arti HR, Ahmadi F, Mohammadianinejad SE, Cheraghian B, Hesam S. Incidence and Risk Factors of Diabetic Foot Ulcer: A Population-Based Diabetic Foot Cohort (ADFC Study)-Two-Year Follow-Up Study. Int J Endocrinol. 2018 Mar 15;2018:7631659. doi: 10.1155/2018/7631659. PMID: 29736169; PMCID: PMC5875034.
5.Liao X, Li SH, El Akkawi MM, Fu XB, Liu HW, Huang YS. Surgical amputation for patients with diabetic foot ulcers: A Chinese expert panel consensus treatment guide. Front Surg. 2022 Nov 8;9:1003339. doi: 10.3389/fsurg.2022.1003339. PMID: 36425891; PMCID: PMC9679004.
6.Amputation Prevention Alliance. (n.d.). Amputation prevention alliance. Amputation Prevention Alliance | ADA.
7.Jeyaraman K, Berhane T, Hamilton M, Chandra AP, Falhammar H. Mortality in patients with diabetic foot ulcer: a retrospective study of 513 cases from a single Centre in the Northern Territory of Australia. BMC Endocr Disord. 2019 Jan 3;19(1):1. doi: 10.1186/s12902-018-0327-2. PMID: 30606164; PMCID: PMC6318899.
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