Becaplermin

ROCHESTER, Minn.--( BUSINESS WIRE )--RION, a clinical-stage regenerative medicine company at the forefront of exosome-based therapeutics, announced today that it has completed patient enrollment in its Phase 2 clinical trial for Purified Exosome Product™ (PEP™) in the treatment of Diabetic Foot Ulcers (DFUs). This milestone marks a significant step forward in the company’s mission to develop cutting-edge therapies addressing chronic wound care. The multi-center, prospective, open-label study enrolled 59 patients across the United States. The trial is designed to evaluate the safety and efficacy of PEP™ when applied topically in conjunction with standard wound care, compared to standard care alone. Over the course of up to 12 weekly applications, key endpoints, including wound closure rates, time to healing, and overall safety, will be assessed. DFUs remain a major complication of diabetes, affecting 18.6 million individuals globally each year, with significant associated morbidity, mortality, and financial burden 1 . The cost of DFU treatment in the United States alone exceeds $13 billion annually 2 . Despite this urgent need, no new biologics have been approved for diabetic wound care in over two decades 3 . PEP™ is a proprietary, lyophilized exosome-based powder derived from human platelets. Stabilized regenerative exosomes in PEP™ are engineered to enhance the body’s natural healing mechanisms by promoting cellular growth, angiogenesis, and inflammation reduction, while also protecting vulnerable tissues—key factors in achieving successful wound healing. “We are thrilled to announce the full enrollment of our Phase 2 study, a crucial milestone in advancing PEP™ and exosome-based therapeutics,” said Atta Behfar, MD, PhD, co-founder of RION. “This achievement reflects the commitment of our clinical partners and the trial participants who are helping to move the field of regenerative medicine forward. PEP™ has the potential to transform diabetic wound care by addressing a significant unmet need with a novel therapeutic approach.” The Phase 2 study builds on promising data from RION’s earlier Phase 1B trial conducted at Mayo Clinic, which demonstrated the safety and potential efficacy of PEP™. Upon successful completion of this trial, RION plans to initiate a pivotal Phase 3 study, with the goal of filing a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA). About RION RION was born out of Mayo Clinic after two decades of research and innovation. 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. Discovered by 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 Diabetic Foot Ulcers (DFU) Diabetic Foot Ulcers (DFUs) are a severe and common complication of diabetes, characterized by wounds on the feet that are slow to heal and susceptible to infection. DFUs significantly impact the quality of life due to pain, reduced mobility, and the potential for amputation. Approximately 15-25% of people with diabetes will develop a DFU in their lifetime 4 . Alarmingly, around 14-24% of these cases result in amputation 5 . Furthermore, every three and a half minutes in the US, there is an amputation due to DFU 6 , highlighting the severity of this condition. The management of DFUs is challenging, with many patients experiencing recurrent ulcers. Additionally, DFU patients face a high mortality rate, with about 40% succumbing to the condition 7 . The economic burden of DFUs is substantial, imposing significant costs on healthcare systems, from direct treatment expenses to indirect costs related to loss of productivity and long-term disability. References: 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.

ROCHESTER, Minn.--( BUSINESS WIRE )--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 year 1 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 1990s 3 . 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 lifetime 4 , and for almost a quarter of them also limb amputation 5 . In fact, every three and a half minutes in the US, a DFU becomes an amputation 6 . 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 condition 7 , 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. References 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.