Rusfertide

MIMRYLO™ is the First and Only Hepcidin Mimetic Peptide Therapy Approved for the Treatment of Erythrocytosis in Adults with Polycythemia Vera, Offering a Novel Mechanism to Maintain Hematocrit Control, Meaningfully Reduce Phlebotomy Burden, and Improve Fatigue Approval Supported by Phase 3 VERIFY Results Showing 76.9% of Patients Achieved Response During Weeks 20-32 FDA Approval Triggers $275M in Milestones, 14%-29% Royalties, with Up to an Additional $875M in Potential Future Milestones Investor call to be held Monday, August 31st at 8:30 am ET August 28, 2026 / Protagonist Therapeutics, Inc. (NASDAQ:PTGX) ("Protagonist" or "the Company") announced today that Takeda received U.S. Food and Drug Administration (FDA) approval for MIMRYLO (rusfertide), a subcutaneously delivered first-in-class hepcidin mimetic peptide for the treatment of erythrocytosis in adults with polycythemia vera (PV). MIMRYLO is the first and only hepcidin mimetic approved for polycythemia vera (PV), a blood cancer characterized by excessive production of red blood cells. "Today's FDA approval of MIMRYLO represents a paradigm shifting approach to management of PV. MIMRYLO mimics the action of hepcidin, the body's natural master regulator of iron availability thereby controlling hematocrit levels and helping restore iron balance. MIMRYLO was conceived fourteen years ago with the idea that a mimetic of hepcidin, the body's primary regulator of iron homeostasis, could address the underlying biology of erythrocytosis in PV. The approved label also reflects improvement in fatigue as measured by PROMIS Fatigue Short Form 8a." said Dinesh V. Patel, PhD, President and Chief Executive Officer of Protagonist Therapeutics. "I am very proud of what the team at Protagonist, in partnership with investigators and patients, have accomplished in bringing this drug over the finish line, and we have full confidence in Takeda as the ideal partner to bring MIMRYLO to patients who need it most." "This is Protagonist's second FDA approval in 2026, reflecting years of disciplined investment in the right type of innovative projects, and working with the right partners at the right time," Patel continued. "With two approved products validating our peptide-centric drug discovery and development acumen and a strong financial foundation, the Company is now focused on leveraging its expertise to advance the next generation of wholly owned product candidates in IL-17, obesity, and beyond with the intent of addressing unmet needs of patients and creating long-term value for our shareholders." MIMRYLO will be commercialized by Takeda under the worldwide license and collaboration agreement entered into in 2024 between Protagonist and Takeda. Protagonist discovered rusfertide (PTG-300) and had primary responsibility for its development through Phase 3, with Takeda assuming responsibility for NDA submission and commercialization following Protagonist's opt-out election in April 2026. FDA approval of MIMRYLO triggers $275 million in payments to Protagonist, consisting of a $200 million opt-out fee and a separate $75 million approval milestone. Protagonist is eligible to receive up to an additional $875 million in total potential milestone payments, as well as tiered royalties ranging from 14% to 29% on worldwide net sales, corresponding to approximately 21% on a weighted-average basis at $1.5 billion in annual sales. Clinical evidence summary MIMRYLO was approved on the basis of the Phase 3 VERIFY study, a randomized, double-blind, placebo-controlled trial in patients with polycythemia vera. In VERIFY, patients in the MIMRYLO arm achieved statistically significant benefit over the placebo arm, including the proportion of responders (with absence of phlebotomy eligibility), mean number of phlebotomies, proportion of participants maintaining hematocrit 15%) adverse reactions were injection site reactions (56%) and anemia (16%). PV is characterized by the excessive production of red blood cells (erythrocytosis), leading to elevated hematocrit which can increase blood viscosity, or thickness. This has the potential to result in life-threatening thrombotic events, including stroke, deep vein thrombosis and pulmonary embolism. Patients with PV may also experience burdensome symptoms, including severe fatigue, pruritus (itching), difficulty concentrating and night sweats. Patients with PV experiencing uncontrolled hematocrit have a four times higher risk of cardiovascular death or major cardiovascular events compared to patients who achieve a hematocrit of less than 45%1. An estimated 78% of patients still experience uncontrolled hematocrit with current standard of care, including phlebotomy and cytoreductive therapies. MIMRYLO is the first and only hepcidin mimetic peptide approved for the treatment of erythrocytosis in adults with polycythemia vera (PV). Rusfertide mimics the action of the natural hormone hepcidin, the body's natural master regulator of iron availability, suppressing red blood cell production by reducing iron availability to erythroid progenitor cells, thereby controlling hematocrit levels. Unlike current standards of care, which include phlebotomy and cytoreductive therapy and do not address the mechanism of iron dysregulation in PV, MIMRYLO directly targets red blood cell overproduction in PV. In the Phase 3 VERIFY study, 76.9% of patients receiving MIMRYLO plus current standard of care achieved a clinical response during Weeks 20-32, compared with 32.9% of patients receiving placebo plus current standard of care. The study also demonstrated improvement in fatigue as measured by PROMIS Fatigue Short Form 8a. For patients who have spent years managing a chronic, burdensome disease through frequent blood draws and ongoing cytoreductive therapy, MIMRYLO represents a fundamentally different approach, leveraging the body's natural regulatory systems to control hematocrit and help restore iron balance. MIMRYLO is self-administered as a weekly subcutaneous injection. MIMRYLO is currently approved in the U.S. for the treatment of erythrocytosis in adults with PV. Protagonist Therapeutics is a biopharmaceutical company built on a proprietary peptide technology platform that has now produced two FDA-approved, first-in-class medicines, generating royalties and milestones from global commercial partnerships with Johnson and Johnson and Takeda Pharmaceutical Company Limited. ICOTYDE™ (icotrokinra), licensed to Johnson & Johnson company Janssen Biotech, Inc., is the first and only targeted oral peptide that precisely blocks the Interleukin-23 receptor. ICOTYDE was launched in the U.S. in March 2026 for the treatment of moderate-to-severe plaque psoriasis in adults and pediatric patients 12 years of age or older and is in Phase 3 development for psoriatic arthritis, ulcerative colitis and Crohn's disease. ICOTYDE was jointly discovered by Protagonist and Johnson & Johnson scientists, with Protagonist having primary responsibility for the development of ICOTYDE through Phase 1, and Johnson & Johnson assuming responsibility for further clinical and regulatory development and commercialization. Protagonist also discovered and led research and development through Phase 3 for MIMRYLO™ (rusfertide), a first-in-class hepcidin mimetic peptide licensed to Takeda Pharmaceutical Company Limited for worldwide commercialization. MIMRYLO is approved in the U.S. for the treatment of erythrocytosis in adults with PV. The Company also has a number of clinical and preclinical programs based on clinically and commercially validated targets and addressing unmet medical needs, including an oral IL-17 antagonist peptide, anti-obesity dual and triple agonists, an oral hepcidin functional mimetic, and the recently announced IL-4 and amylin discovery programs. 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MIMRYLO, a First-in-Class Medicine with a Unique Mechanism of Action, is Approved for the Treatment of Erythrocytosis in Adults with Polycythemia Vera (PV) MIMRYLO Has Been Shown to Maintain Hematocrit Control, the Primary Treatment Goal in PV, as Well as Reduce Phlebotomy Burden and Improve Fatigue Approval Supported by Phase 3 VERIFY Results Showing 76.9% of Patients Achieved Clinical Response During Weeks 20-32 Takeda (TSE:4502/NYSE:TAK) announced U.S. Food and Drug Administration (FDA) approval of the New Drug Application (NDA)* for MIMRYLO™ (rusfertide) for the treatment of erythrocytosis in adults with polycythemia vera (PV), a blood cancer. MIMRYLO is a first-in-class hepcidin mimetic designed to regulate iron distribution in the body and red blood cell overproduction to control hematocrit levels, which is the ratio of red blood cells to the total amount of blood in the body. Maintaining controlled hematocrit levels below 45% is the primary treatment goal in PV.1 “For patients living with PV, uncontrolled hematocrit can have serious consequences, including an elevated risk of life-threatening thrombotic events," said Andrew T. Kuykendall, M.D., VERIFY lead investigator and Associate Member in the Department of Hematology at Moffitt Cancer Center. “Current treatments, such as phlebotomy, leave a significant gap for too many patients and can pose challenges to daily life and routines. The approval of MIMRYLO offers clinicians and patients a novel, first-in-class therapy that targets erythrocytosis, which drives excess red blood cell production in PV. The strength and consistency of the VERIFY data give me real confidence in MIMRYLO’s potential to advance how we treat PV in everyday practice and to maintain hematocrit control.” Uncontrolled Hematocrit is a Challenge in the Treatment of PV Affecting approximately 90,000 people in the U.S., PV is characterized by the overproduction of red blood cells (erythrocytosis), leading to elevated hematocrit which can increase blood viscosity, or thickness.2,3 This has the potential to result in life-threatening thrombotic events, including stroke, deep vein thrombosis and pulmonary embolism.4 Maintaining hematocrit levels consistently below 45% can prevent thrombotic events and alleviate burdensome symptoms, including severe fatigue, pruritus (itching), difficulty concentrating and night sweats.4 An estimated 78% of patients still experience uncontrolled hematocrit with current standard of care, including phlebotomy and cytoreductive therapies.5 Patients with PV experiencing uncontrolled hematocrit have a four times higher risk of cardiovascular death or major cardiovascular events.4 “People living with PV often experience complex and invisible symptoms, from extreme fatigue to the emotional strain of living with a chronic blood cancer,” said Kapila Viges, Chief Executive Officer, MPN Research Foundation. “At the same time, we know that every patient’s experience with PV is different, underscoring the need to continue to listen closely to the community to understand what matters most. There remains a need for treatments that better address these daily challenges. This meaningful approval reflects important progress and brings forward a new treatment option in a disease where patients have long needed innovation and more choices. We are encouraged by MIMRYLO’s potential to help patients meet their treatment goals.” The approval was supported by data from the global randomized Phase 3 VERIFY study (NCT05210790) that included 293 patients with PV, showing that MIMRYLO met all efficacy endpoints and demonstrated a favorable safety profile. In the study, patients receiving MIMRYLO plus current standard of care demonstrated a higher response rate compared to placebo plus current standard of care. This included hematocrit control, a reduction in the need for phlebotomy and improvement in fatigue as measured by PROMIS Fatigue Short Form 8a. MIMRYLO was generally well-tolerated through 52 weeks of treatment in the VERIFY trial. The most common treatment-emergent adverse events in MIMRYLO-treated patients were injection site reactions and anemia. Learn more about the Phase 3 data results here. “The approval of MIMRYLO underscores the strength of Takeda’s late-stage pipeline and our focus on developing genuinely differentiated therapies for patients who are urgently waiting for new options,” said Julie Kim, President and Chief Executive Officer, Takeda. “We are at an important inflection point as we prepare to deliver three new medicines, which have the potential to drive our future growth and are a reflection of our commitment to advancing innovation that doesn’t just add to the treatment landscape, but reshapes it. We are grateful to the patients, care partners, advocates and investigators who helped make this approval possible.” The open-label extension of the VERIFY trial is ongoing and Takeda will share further findings at upcoming medical conferences. Takeda is working with regulators outside of the U.S. to potentially bring MIMRYLO to more patients worldwide. This approval does not result in any changes to Takeda’s consolidated financial forecast for the fiscal year ending March 31, 2027 (FY2026). MIMRYLO™ is a first-in-class subcutaneous treatment that mimics the action of hepcidin, a natural hormone that regulates iron homeostasis and erythrocytosis. By targeting the underlying mechanism of iron dysregulation in polycythemia vera, MIMRYLO aims to reduce excess red blood cell production and help patients maintain hematocrit control. MIMRYLO is administered once weekly via subcutaneous injection and has been generally well-tolerated in clinical trials to date. Protagonist discovered MIMRYLO and led its development through Phase 3. Takeda now has exclusive global development and commercialization rights for MIMRYLO. About VERIFY The Phase 3 VERIFY study (NCT05210790) is an ongoing, three-part, global, randomized, placebo-controlled study evaluating MIMRYLO in 293 patients with polycythemia vera over a 156-week period, with treatment extension for participants who are continuing to derive benefit from MIMRYLO beyond the 156-week treatment period. The study is evaluating the efficacy and safety of once-weekly, subcutaneously self-administered MIMRYLO in patients with uncontrolled hematocrit who are phlebotomy-dependent despite current standard of care treatment, which could include phlebotomy, hydroxyurea, interferon and/or ruxolitinib. The primary endpoint of the study was the proportion of patients achieving a response during Weeks 20-32, which was defined as the absence of “phlebotomy eligibility.” To meet phlebotomy eligibility, patients in the study were required to have: confirmed hematocrit ≥45% that was ≥3% higher than their baseline hematocrit value, or hematocrit ≥48%. Key secondary endpoints evaluated at Week 32 included mean number of phlebotomies, proportion of patients maintaining hematocrit <45%, mean change in fatigue score as measured by PROMIS Fatigue Short Form 8a and total symptom burden as measured by Myelofibrosis Symptom Assessment Form (MFSAF) Version 4.0. All patients have completed their participation in the randomized, placebo-controlled portion of the study evaluating the efficacy and safety of MIMRYLO plus current standard of care versus placebo plus current standard of care and are now in the open-label portions of the study. About Polycythemia Vera (PV) Polycythemia vera (PV) is a chronic blood cancer characterized by the overproduction of red blood cells (erythrocytosis), which increases blood viscosity, or thickness, and can result in life threatening thrombotic events such as stroke, deep vein thrombosis and pulmonary embolism. Hematocrit is the ratio of red blood cells to the total amount of blood in the body. Achieving and maintaining controlled hematocrit levels of less than 45% is the primary treatment goal in PV to prevent thrombotic events and alleviate burdensome symptoms, including severe fatigue, difficulty in concentrating, night sweats and pruritus. Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. References Barbui T, et al. Philadelphia chromosome-negative classical myeloproliferative neoplasms: revised management recommendations from European LeukemiaNet. Leukemia 2018; 32(5), 1057-1069. Vachhani PJ. Estimated prevalence of polycythemia vera in the United States (2025-2030): SEER analysis with modeled reporting delay. J Clin Oncol.2026;44(suppl 16):e18589. Lu X, Chang R. Polycythemia Vera. [Updated 2023 Apr 24]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan-. The content above comes from the network. if any infringement, please contact us to modify.