rusfertide (PTG-300) / Protagonist Therap, Takeda - LARVOL DELTA

Results from VERIFY, a phase 3, double-blind, placebo (PBO)-controlled study of rusfertide for treatment of polycythemia vera (PV). (ASCO 2025) - P3 | "Clinical Trial Registration Number: NCT05210790 The abstract will be released to the public on June 1, 2025 at 7:00 AM CDT"

Clinical • Late-breaking abstract • P3 data • Polycythemia Vera

Protagonist Therapeutics Announces Plenary Presentation on VERIFY Phase 3 Study with Rusfertide at ASCO 2025 (ACCESSWIRE) - "Protagonist Therapeutics...announced that an abstract was accepted for presentation at the Plenary Session at the American Society of Clinical Oncology (ASCO) Annual Meeting....Presentation Details:...Title: Results From VERIFY, a Phase 3, Double-Blind, Placebo (PBO)-Controlled Study of Rusfertide for Treatment of Polycythemia Vera (PV); Abstract: LBA3;...Session Date: Sunday, June 1, 2025; Presentation Time: 2:09 PM - 2:21 PM CDT."

P3 data • Polycythemia Vera

Rusfertide: “Study met primary endpoint with a significantly higher proportion of clinical responders on rusfertide compared to placebo during weeks 20-32 (p<0.0001)”; Polycythemia Vera (Protagonist Therapeutics) - Investor Call and Webcast: “Phlebotomy rate - EU primary endpoint (p<0.0001)”

P3 data: top line • Hematological Malignancies • Oncology • Polycythemia Vera

Protagonist and Takeda Announce Positive Topline Results from Phase 3 VERIFY Study of Rusfertide in Patients with Polycythemia Vera (Businesswire) - P3 | N=250 | VERIFY (NCT05210790) | Sponsor: Protagonist Therapeutics, Inc. | "The primary endpoint of the study was met, with a significantly higher proportion of clinical responders among rusfertide-treated patients with PV (77%) compared to those who received placebo (33%) during weeks 20-32; p<0.0001....The first key secondary endpoint, which is the pre-specified primary endpoint for European Union (EU) regulators, was also met, with a mean of 0.5 phlebotomies per patient in the rusfertide arm compared to 1.8 phlebotomies per patient in the placebo arm during weeks 0-32; p<0.0001....'We plan to submit additional details of these promising results for presentation at upcoming medical conferences in 2025'....Under the license and collaboration agreement between Protagonist and Takeda, Protagonist earns a $25 million milestone payment following these positive results. The milestone is payable following completion of the VERIFY clinical study report."

Financing • P3 data • Polycythemia Vera

Rusfertide: Regulatory submission in US for polycythemia Vera (based on VERIFY trial) in Q4 2025 (Protagonist Therapeutics) - Corporate Presentation

FDA filing • Hematological Malignancies • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera

Takeda Delivers Strong Third-Quarter FY2024 Results; Raises Full Year Outlook, Forecasting Revenue and Core Operating Profit Margin Growth (Businesswire) - "Takeda...announced earnings results for the third quarter of fiscal year 2024 (nine months ended December 31, 2024) showing continued advancement of its Growth & Launch Products, which delivered double-digit growth of 14.6% at CER....Among the multiple late-stage programs presented, the company expects...Phase 3 data readouts in the calendar year 2025 with filings anticipated in FY2025-FY2026 for the following program...rusfertide for the treatment of polycythemia vera, a rare chronic blood disorder; additional indication filings for late-stage programs are on pace for FY2027-FY2029....elritercept for the treatment of anemia associated with myelodysplastic syndrome"

Commercial • Filing • P3 data • Anemia • Myelodysplastic Syndrome • Polycythemia Vera

Protagonist Therapeutics Receives FDA Breakthrough Therapy Designation for Rusfertide in Polycythemia Vera (PRNewswire) - "Protagonist Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its lead investigational new drug candidate, rusfertide, for the treatment of patients with polycythemia vera (PV) for the reduction of erythrocytosis in those patients who do not require further treatment for thrombocytosis and/or leukocytosis."

Breakthrough therapy • Polycythemia Vera

Multiple-Dose Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Subcutaneous Rusfertide, a Hepcidin Mimetic, in Healthy Subjects. (PubMed, Clin Pharmacol Drug Dev) - "While a higher incidence of treatment-emergent adverse events was noted in these healthy participants following multiple doses of 60 mg, rusfertide was generally well tolerated. There were no clinically meaningful differences in rusfertide pharmacokinetics or pharmacodynamics between injection sites."

Journal • PK/PD data • Dermatology • Fatigue • Pain • Polycythemia Vera • Pruritus

Rusfertide: 32-week primary endpoint data from P3 VERIFY trial (NCT05210790) for polycythemia vera in March 2025 (Protagonist Therapeutics) - Corporate Presentation

P3 data • Hematological Malignancies • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera

Takeda Spotlights High-Value, Late-Stage Pipeline Accelerating the Development of Potential Transformative Treatments for Patients in Multiple Therapeutic Areas (Businesswire) - "Expected Phase 3 Data Readouts in 2025 for...Rusfertide (TAK-121); Regulatory Filings for...Rusfertide (Polycythemia Vera) on Track for Fiscal Years 2025 - 2026; Five Additional Filings Anticipated in Fiscal Years 2027 - 2029 Including First Indication Submissions for...Elritercept (TAK-226)....elritercept, an investigational activin inhibitor designed to treat anemia associated with certain hematologic cancers, including myelodysplastic syndromes (MDS)."

FDA filing • P3 data • Myelodysplastic Syndrome • Oncology • Polycythemia Vera

Final Results from the Phase 2 Revive Study Investigating the Hepcidin Mimetic Rusfertide in Patients with Polycythemia Vera (PV) (ASH 2024) - P2, P3 | "Patients are eligible to roll over to the phase 2 THRIVE OLE study (NCT06033586), which will continue to assess the long-term safety and efficacy of rusfertide for a total period of up to 5.8 years. The randomized phase 3 VERIFY study (NCT05210790) is ongoing and will evaluate rusfertide vs placebo in PV patients."

Clinical • P2 data • Acute Myelogenous Leukemia • Anemia • Dermatology • Fatigue • Genetic Disorders • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Musculoskeletal Pain • Myeloproliferative Neoplasm • Novel Coronavirus Disease • Oncology • Pain • Polycythemia Vera • Pruritus • Skin Cancer • CRP • IL6

Rusfertie: “In REVIVE, rusfertide added to TP with or without CRT controlled erythrocytosis, provided long-term durable control of Hct, and decreased the need for TP in patients with PV”; Polycythemia vera (Protagonist Therapeutics) - ASH 2024: “Rusfertide was well-tolerated; the most common adverse events were Grade 1 or 2 injection site reactions, fatigue, pruritus, COVID-19, arthralgia, dizziness, nausea, headache, anemia, paresthesia, and diarrhea”

P2 data • Hematological Malignancies • Oncology • Polycythemia Vera

Protagonist Reports Final Results From Rusfertide Phase 2 REVIVE Study Showing Durable Hematocrit Control at the ASH 2024 Annual Meeting (ACCESSWIRE) - P2 | N=80 | REVIVE (NCT04057040) | Sponsor: Protagonist Therapeutics, Inc. | "Final results show that rusfertide, when added to therapeutic phlebotomy with or without cytoreductive therapy achieved long term durable control of hematocrit below the 45% threshold for over 3 years. Prior to enrollment, the estimated mean phlebotomy rate (EPHL) in patients who enrolled on study was >5/year: In Part 1, the EPHL was <1/year in patients who received rusfertide (N=70). In Part 2 (randomized withdrawal phase), the EPHL was <1/year and approximately 6.1/year in the rusfertide and placebo groups, respectively. For patients who continued to Part 3 (Week 42+), the EPHL remained at <1/year....Overall, 18 (26%) patients experienced serious adverse events (SAEs); most SAEs were unrelated and likely associated with the underlying disease. One patient developed acute myeloid leukemia after treatment discontinuation."

P2 data • Polycythemia Vera

Takeda to Showcase Latest Research at the 66th American Society of Hematology (ASH) Annual Meeting (Takeda Press Release) - "The presentations will include five-year follow-up of the Phase 2 OPTIC study providing updated long-term efficacy and safety data of ICLUSIG (ponatinib) in patients with chronic-phase...CML....Results from two post-hoc analyses from the Phase 3 PhALLCON trial evaluating ICLUSIG versus imatinib plus reduced-intensity chemotherapy will also be presented, providing additional data for patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Additionally, four presentations evaluating data from the Phase 3 HD21 study will be featured, including the German Hodgkin Study Group’s (GHSG) oral presentation of results from a single-arm cohort evaluating ADCETRIS (brentuximab vedotin) with etoposide, cyclophosphamide, doxorubicin, dacarbazine and dexamethasone (BrECADD) in elderly patients with advanced-stage Hodgkin lymphoma....Additional presentations will feature the Phase 2 REVIVE final study results evaluating rusfertide..."

Clinical data • Acute Lymphocytic Leukemia • Chronic Myeloid Leukemia • Hodgkin Lymphoma • Polycythemia Vera

Pharmacokinetics and Pharmacodynamics of Rusfertide, a Hepcidin Mimetic, Following Subcutaneous Administration of a Lyophilized Powder Formulation in Healthy Volunteers. (PubMed, Drugs R D) - "Rusfertide was well tolerated; the pharmacokinetic and pharmacodynamic results indicate that lyophilized rusfertide is suitable for once-weekly or twice-weekly administration."

Journal • PK/PD data • Dermatology • Polycythemia Vera • Pruritus

Iron deficiency and phlebotomy in patients with polycythemia vera. (PubMed, Int J Hematol) - "Among them, rusfertide has been shown to control hematocrit levels without requiring phlebotomy. Further studies are needed to identify new treatment strategies for PV that also consider iron deficiency."

Journal • Review • Cardiovascular • Fatigue • Hematological Disorders • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombosis

Protagonist Reports Third Quarter 2024 Financial Results and Provides Corporate Update (ACCESSWIRE) - "Upcoming Milestones - Rusfertide: Subcutaneous Injectable Hepcidin Mimetic for Polycythemia Vera (PV): The top line data for the Phase 3 VERIFY study in polycythemia vera, which has a 32-week primary efficacy endpoint, is expected in Q1 2025."

P3 data: top line • Polycythemia Vera

Protagonist Therapeutics Announces Poster Presentation on Final REVIVE Phase 2 Study Data with Rusfertide at the 66th Annual ASH Meeting (ACCESSWIRE) - "Protagonist Therapeutics, Inc...announced that final data from the Phase 2 REVIVE study with rusfertide in polycythemia vera will be the focus of a poster presentation at the 66th Annual American Society of Hematology (ASH) Annual Meeting being held in San Diego from December 7-10, 2024."

P2 data • Polycythemia Vera

Updated Long-term Results from the Phase 2 REVIVE Study Investigating the Hepcidin Mimetic Rusfertide in Polycythemia Vera (PV) Patients: Hematocrit (Hct) Control and Therapeutic Phlebotomy (TP) Frequency. (MPN 2024) - No abstract available

Clinical • P2 data • Polycythemia Vera

VERIFY: A randomized, controlled, phase 3 trial of the hepcidin-mimetic experimental drug rusfertide (PTG-300) in patients with polycythemia vera (PV) (DGHO 2024) - P2, P3 | "VERIFY began enrolling patients in January 2022 and met its 250-patient enrollment target at the end of March 2024. Conclusions : Top-line data are anticipated at the end of the first quarter of 2025."

Clinical • P3 data • Cardiovascular • Hematological Disorders • Oncology • Polycythemia Vera • Thrombosis

Rusfertide: Regulatory approval in US for polycythemia vera in Q4 2026 (Protagonist Therapeutics) - Corporate Presentation: Launch in US for polycythemia vera in 2027

FDA approval • FDA filing • Launch US • Hematological Malignancies • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera

Rusfertide: Primary endpoint data from P3 VERIFY trial (NCT05210790) for polycythemia vera in Q1 2025 (Protagonist Therapeutics) - Corporate Presentation

P3 data • Hematological Malignancies • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera

Rusfertide: Launch in US for polycythemia vera in 2026 (Protagonist Therapeutics) - Corporate Presentation

Launch US • Hematological Malignancies • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera

The evolving landscape of polycythemia vera therapies. (PubMed, Expert Opin Pharmacother) - "The treatment landscape of polycythemia vera (PV) has seen major advancements within the last decade including approval of ruxolitinib in the second line setting after hydroxyurea, ropegylated interferon-α2b, and advanced clinical development of a novel class of agents called hepcidin mimetics...The discovery of hepcidin mimetics has come as a breakthrough in restoring iron homeostasis, achieving phlebotomy-independence and may lead to improved thrombosis-free survival with stricter hematocrit control. On the other hand, emerging data with IFN- α and ruxolitinib as well as combination of the two agents suggests the potential for achieving molecular remission in a subset of PV patients and long-term follow-up is awaited to validate the correlation of molecular responses with clinically relevant outcomes of progression-free and thrombosis-free survival."

Journal • Review • Cardiovascular • Hematological Disorders • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombosis • IFNA1

VERIFY: A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera (clinicaltrials.gov) - P3 | N=250 | Active, not recruiting | Sponsor: Protagonist Therapeutics, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Polycythemia Vera