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

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

Rusfertide: “Rusfertide provided durable control of hematocrit through 3 years”; Polycythemia vera (Protagonist Therapeutics) - EHA 2024: “Rusfertide reduced the estimated average phlebotomy rate”

P2 data • Hematological Malignancies • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera

UPDATED LONG-TERM RESULTS FROM THE PHASE 2 REVIVE STUDY INVESTIGATING THE HEPCIDIN MIMETIC RUSFERTIDE IN POLYCYTHEMIA VERA PATIENTS (PTS): HEMATOCRIT CONTROL AND THERAPEUTIC PHLEBOTOMY (TP) FREQUENCY (EHA 2024) - P2, P3 | "In REVIVE, rusfertide added to TP with or without CRT provided long-term durable control of Hct anddecreased the need for TP compared with the pre-study period. REVIVE pts are eligible to roll over to the open-label phase 2 extension THRIVE study (NCT06033586), which will continue to assess the long-term safety andefficacy of rusfertide. The randomized phase 3 VERIFY study (NCT05210790) is also ongoing to evaluaterusfertide vs."

Clinical • P2 data • Acute Myelogenous Leukemia • Anemia • Dermatology • Fatigue • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Musculoskeletal Pain • Novel Coronavirus Disease • Oncology • Pain • Polycythemia Vera • Pruritus

ABSENCE OF QTC PROLONGATION WITH RUSFERTIDE, A HEPCIDIN MIMETIC FOR THE TREATMENT OF POLYCYTHEMIA VERA: A THOROUGH QT/QTC STUDY IN HEALTHY SUBJECTS (EHA 2024) - "Rusfertide did not result in a clinically relevant prolongation of the QTcF interval in healthy subjects. Rusfertidewas generally well tolerated."

Clinical • Cardiovascular • Polycythemia Vera

Protagonist Reports Updated Long Term Results from Rusfertide Phase 2 REVIVE Study at the EHA2024 Congress Showing Durable Hematocrit Control (ACCESSWIRE) - P2 | N=80 | REVIVE (NCT04057040) | Sponsor: Protagonist Therapeutics, Inc. | "Protagonist Therapeutics, Inc...announced details from two abstracts at the European Hematology Association 2024 Congress, including an oral presentation with long-term follow-up data from the Phase 2 REVIVE study with rusfertide, a mimetic of the natural hormone hepcidin with potential therapeutic value in the treatment of polycythemia vera (PV) and other disease indications....Long term durable control of hematocrit below the 45% threshold for up to 3 years; Decreased phlebotomy use, from a rate of 8.7 per year before study entry to 0.43 per year in Part 3 of REVIVE, which remained consistent with the rate of 0.36 per year observed for patients randomized to rusfertide in Part 2 of REVIVE; Decreased red blood cell counts and continued improvement and normalization of serum ferritin levels."

P2 data • Hematological Malignancies • Oncology • Polycythemia Vera

Protagonist Reports Updated Long Term Results from Rusfertide Phase 2 REVIVE Study at the EHA2024 Congress Showing Durable Hematocrit Control (ACCESSWIRE) - P=NA | N=60 | "A separate abstract accepted for publication...In this study, 60 healthy subjects were randomized to receive single doses of subcutaneous rusfertide (90mg), matching placebo, or oral moxifloxacin (400mg).Rusfertide was generally well tolerated and did not result in a clinically relevant prolongation of the QTcF interval."

Clinical data • Hematological Malignancies • Oncology • Polycythemia Vera

VERIFY: A randomized controlled phase 3 study of the hepcidin mimetic rusfertide (PTG-300) in patients with polycythemia vera (PV). (ASCO 2024) - P2, P3 | "VERIFY opened in January 2022 and aims to enroll approximately 250 patients globally. Reference: 1. Ritchie EK, et al."

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

Pharmacokinetics, pharmacodynamics, and tolerability of an aqueous formulation of rusfertide (PTG-300), a hepcidin mimetic, in healthy volunteers: A double-blind first-in-human study. (PubMed, Eur J Haematol) - "Rusfertide was well tolerated and showed dose-dependent pharmacokinetics and pharmacodynamics."

Journal • P1 data • PK/PD data • Polycythemia Vera

Contemporary Challenges in Polycythemia Vera Management From the Perspective of Patients and Physicians. (PubMed, Clin Lymphoma Myeloma Leuk) - "Phlebotomy needs can be reduced by utilizing cytoreductive therapy; however, standard first-line cytoreductive options (i.e., hydroxyurea and interferon) have not been shown to significantly improve symptom burden. Collectively, current PV management, while reducing thrombotic risk, often has a negative impact on patient quality of life. As researchers continue to advance towards the goal of developing a disease-modifying therapy for patients with PV, pursuit of nearer-term opportunities to shift the current treatment paradigm towards improving symptoms without compromising quality of life is also warranted, for example, by reducing or eliminating the frequent use of phlebotomy."

Journal • Review • Hematological Disorders • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera

Protagonist Therapeutics Announces Oral Presentation on Long-Term Follow-up of Rusfertide Phase 2 REVIVE Study Open Label Extension at the European Hematology Association 2024 Congress (ACCESSWIRE) - "Protagonist Therapeutics...announced that additional data from the rusfertide Phase 2 REVIVE open label extension study will be the focus of an oral presentation at the European Hematology Association (EHA) Congress being held in Madrid, Spain from June 13-16, 2024. There will also be a published abstract showing the absence of QTc prolongation with rusfertide based on the results from the thorough QT/QTc study conducted in healthy subjects."

Clinical data • P2 data • Polycythemia Vera