Besremi (ropeginterferon alfa-2b-njft) / PharmaEssentia, AOP Orphan Pharma, Pint Pharma, FORUS Therapeutics - LARVOL DELTA

Efficacy and Safety of Ropeginterferon in Myeloproliferative Neoplasms: A Systematic Review and Meta-Analysis (SOHO 2026) - "Hydroxyurea remains a mainstay of MPN treatment but lacks disease-modifying potential. This systematic review aims to evaluate the efficacy and safety of ropeginterferon alfa-2b, a novel pegylated interferon, in MPN patients across clinical trials and real-world studies... Ropeginterferon demonstrates durable hematologic and molecular efficacy with a favorable safety profile in MPNs, particularly PV. Long-term data suggest disease-modifying potential, supporting its integration into treatment strategies for PV and warranting further evaluation in ET and MF. Funding: AOP Health (AOP Orphan Pharmaceuticals GmbH) provided funding for journal fees through a medical grant."

Retrospective data • Review • Essential Thrombocythemia • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • CALR • JAK2 • MPL

Randomized phase 3 trial of Ropeginterferon alfa-2b versus surveillance after tyrosine kinase inhibitor discontinuation in chronic myeloid leukemia (ENDURE/CML-IX). (PubMed, Leukemia) - P3 | "Ropeg-IFN was well tolerated (median administered dose of 92 µg, range 3-104), and no new safety signals were observed. Ropeg-IFN maintenance did not improve the probability of sustained TFR after TKI discontinuation."

Journal • P3 data • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology

PMDA regulatory update on approval and revision of the precautions for use of anticancer drugs in Japan; sevabertinib for lung cancer, gemcitabine intravesical system and durvalumab for bladder cancer, azacitidine for leukemia, cemiplimab for skin cancer, and ropeginterferon alfa-2b for essential thrombocythemia. (PubMed, Int J Clin Oncol) - No abstract available

Japanese regulatory • Journal • Bladder Cancer • Essential Thrombocythemia • Genetic Disorders • Genito-urinary Cancer • Hematological Disorders • Hematological Malignancies • Leukemia • Lung Cancer • Myeloproliferative Neoplasm • Oncology • Skin Cancer • Solid Tumor • Thrombocytosis

Molecular Response, Event-Free Survival, and Treatment-Free Remission in Myeloproliferative Neoplasms: Update and Review with Insights from MPN Asia 2026. (PubMed, Curr Hematol Malig Rep) - "Clinical trials with ropeginterferon alfa-2b were central to the discussions given these studies have contributed to the evolving paradigm of disease modification in MPNs, underscoring the importance of molecular response and event-free survival (EFS) in MPN management...In MF, molecular profiling is already integral to prognostication and treatment selection, and disease modification assessment will likely require endpoints encompassing more than symptoms and spleen response, such as anemia response, bone marrow fibrosis change, clonal evolution, patient-reported outcomes, and importantly progression-free and overall survival. Molecular response, clonal suppression, prevention of vascular and progression events, survival outcomes such as EFS and TFR play increasingly important roles in MPN management."

JAK2V617F • Journal • Review • Cardiovascular • Essential Thrombocythemia • Fibrosis • Hematological Disorders • Immunology • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombocytosis • Thrombosis • ABL1 • CALR

Ropeginterferon alfa-2b versus anagrelide for the treatment of essential thrombocythemia: Topline results of the phase 3 SURPASS-ET trial. (ASCO 2025) - P3 | " SURPASS ET is an open-label, multicenter, Phase III trial comparing ropeg with anagrelide over 12 months in patients with ET who were hydroxyurea-resistant or-intolerant. Ropeg showed superior efficacy and safety compared to anagrelide as second-line therapy for ET. It represents a potential new therapeutic option for ET. Patient demographics and baseline characteristics.*n (%) presented for categorical variables."

P3 data • P3 data: top line • Essential Thrombocythemia • Hematological Disorders • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Polycythemia Vera • Thrombocytosis • ABL1

Real-World Patient Characteristics, Treatment Patterns, and Blood Counts of Patients With Essential Thrombocythemia Treated With Ropeginterferon Alfa-2b in the US Community Oncology Setting (SOHO 2026) - "In patients with ≥1 prior cytoreductive therapy (n = 49), the most common 1L was hydroxyurea (92%), and median time from ET diagnosis to ropeginterferon initiation was 13 months. In patients with ≥2 prior cytoreductive therapies (n = 21), next cytoreductive therapies after 1L included anagrelide (43%), peginterferon (24%), and ruxolitinib (19%)... This analysis provides preliminary insights into real-world use and outcomes of ropeginterferon in patients with ET. Ropeginterferon was largely initiated after 1L but also as 1L for younger patients. Platelets and response rates trended towards optimal levels regardless of cytoreductive therapy history."

Clinical • Real-world • Real-world evidence • Essential Thrombocythemia • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera

Efficacy and Safety of Ropeginterferon Alfa-2b for Pre-Fibrotic Primary Myelofibrosis and DIPSS Low/Intermediate-1 Risk Myelofibrosis (ASH 2022) - P2 | "In summary, P1101 was well-tolerated, effective in cytoreduction and induced molecular responses. The recruitment of patients is ongoing."

Clinical • Alopecia • Anemia • Hematological Disorders • Musculoskeletal Pain • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Pain • Polycythemia Vera • Thrombocytosis • CALR

Real-World Phlebotomy Burden and Treatment Gaps in United States Patients With Polycythemia Vera: A Chart Review Across Physician Practice Settings (SOHO 2026) - "At the time of management change from phlebotomy, 47% were receiving concurrent CRT, whereas 45% initiated a new CRT thereafter (most commonly hydroxyurea [42%], ruxolitinib [36%], and ropeginterferon alfa-2b [21%]), and 15% did not receive CRT. Phlebotomy remains widely used in PV care but is associated with substantial procedural burden and frequent management changes. Reporting of inadequate hematocrit control as a driver of management change, treatment-related ID, fatigue, and continued need for management changes despite concurrent CRT collectively point to an urgent need for more effective, better-tolerated treatments."

Clinical • Real-world • Real-world evidence • Review • Oncology • Polycythemia Vera

JAK2V617F MOLECULAR RESPONSE CORRELATES WITH EVENT-FREE SURVIVAL IN AN EARLY POLYCYTHEMIA VERA POPULATION (EHA 2024) - "Recent prospective evidence suggests that decreasing the JAK2V617F allele burden could be a majoraim of PV treatment to modify the course of the disease, since molecular response (MR) correlates withprolonged event-free and overall survival in patients with high-risk, hydroxyurea resistant/intolerant PV(Harrison et al, JCO 2023). Depletion of JAK2V617F mutated cells improves event-free survival in patients with early-stage PV and can beeffectively achieved with ropeginterferon alfa-2b. Fig. 1: Time to first risk event (death, thromboembolic events, disease progression) by molecularresponse at last recorded time point"

Clinical • Acute Myelogenous Leukemia • Cardiovascular • Hematological Disorders • Hematological Malignancies • Leukemia • Myelofibrosis • Oncology • Polycythemia Vera • Thrombosis

Ropeginterferon alfa-2b for pre-fibrotic myelofibrosis and lower-risk myelofibrosis requiring cytoreduction. (PubMed, Blood Adv) - P2 | "Ongoing treatment with hydroxyurea was substituted with ropeg (week 0: 250 mcg; week 2: 350 mcg; week 4 onwards: 500 mcg every 2 weeks). In conclusion, ropeg was safe and induced CHCR associated with significant molecular responses in patients with early MF. ClinicalTrials.gov Identifier: NCT04988815."

JAK2V617F • Journal • Fibrosis • Hematological Disorders • Immunology • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • CALR

Efficacy and Safety of Ropeginterferon Alfa-2b in Pregnant Women With Myeloproliferative Neoplasms: Case Series of Three Subtypes and Review. (PubMed, J Korean Med Sci) - "Ropeginterferon was used during pregnancy or prior to conception (250-500 μg every 2-12 weeks) with aspirin and/or low-molecular-weight heparin. All pregnancies resulted in live births without major maternal or neonatal complications. Within the limits of a small retrospective series, ropeginterferon appeared to be a feasible cytoreductive option in these MPN pregnancies, including pre-conception and intra-pregnancy use; these preliminary observations warrant confirmation in larger prospective studies."

Journal • Review • Essential Thrombocythemia • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombocytosis

Real-World Experience With Ropeginterferon Alfa-2b in Polycythemia Vera and Essential Thrombocythemia: Practice Patterns, Tolerability, and Hematologic Response at a Single Academic Center (SOHO 2026) - "Our retrospective review demonstrated CHR and AE rates similar to those in published clinical trials. Most patients had PV, though some received off-label therapy for ET and unclassified MPNs. Importantly, our study supports ropeginterferon use for select patients with psychiatric conditions with close monitoring."

Clinical • Real-world • Real-world evidence • Essential Thrombocythemia • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • CALR • JAK2 • MPL

Ropeginterferon alfa-2b in mycosis fungoides: A multicentre cohort. (PubMed, J Eur Acad Dermatol Venereol) - No abstract available

Journal • Cutaneous T-cell Lymphoma • Dermatology • Hematological Malignancies • Lymphoma • Mycosis Fungoides • Oncology

Event-free survival in early polycythemia vera patients correlates with molecular response to ropeginterferon alfa-2b or hydroxyurea/best available therapy (PROUD-PV/CONTINUATION-PV). (PubMed, Hemasphere) - No abstract available

Journal • Polycythemia Vera

Additionally, PharmaEssentia will present real-world evidence from a retrospective cohort study of 55 patients with ET evaluating the use of BESREMi in community oncology settings (Businesswire) - "PharmaEssentia to Present at the Society of Hematologic Oncology (SOHO) 2026 Annual Meeting....The analysis found that BESREMi was most commonly initiated after first-line therapy, while first-line use was also observed, predominantly in younger patients. Platelet counts and response rates trended toward optimal levels regardless of prior cytoreductive therapy history."

Retrospective data • Essential Thrombocythemia

Disease-Modifying Potential of Ropeginterferon Alfa-2b Across Myeloproliferative Neoplasms: A Systematic Review and Meta-Analysis (SOHO 2026) - "Ropeginterferon alfa-2b demonstrates progressive hematologic control, molecular remission, low discontinuation rates, and signals of disease modification across MPNs. The strongest effects were seen in sustained 24-month CHR and JAK2 V617F reduction. These findings support ropeginterferon alfa-2b as a disease-modifying therapeutic strategy beyond cytoreduction, although larger disease-specific randomized studies are needed."

Retrospective data • Review • Essential Thrombocythemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • JAK2

BETTER SAFETY AND EFFICACY WITH ROPEGINTERFERON ALFA-2B OVER ANAGRELIDE AS SECOND-LINE TREATMENT OF ESSENTIAL THROMBOCYTHEMIA IN THE TOPLINE RESULTS OF THE RANDOMIZED PHASE 3 SURPASS-ET TRIAL (EHA 2025) - "SURPASS ET is an active-controlled open-label multicenter phase III investigation comparing ropeg to anagrelide over 1 months in ET patients who have shown resistance or intolerance to hydroxyurea. Ropeg outperformed anagrelide in terms of efficacy and safety with respect to the primary and secondary endpoints. These findings indicate that ropeg represents a superior second-line therapy for patients with ET in addition to its established role in treating PV."

Clinical • P3 data • P3 data: top line • Acute Myelogenous Leukemia • Essential Thrombocythemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Psychiatry • Thrombocytosis

Hematocrit Control, Symptom Burden, and Time Toxicity Among Phlebotomy-Treated Patients With Polycythemia Vera in the United States (SOHO 2026) - "At survey, PHL+cyto patients were receiving hydroxyurea (64%), ruxolitinib (22%), ropeginterferon alfa-2b (10%), or hydroxyurea plus ruxolitinib combination (4%). In this real-world US cohort, hematocrit control was poor, and symptom burden persisted among phlebotomy-treated patients, including those receiving cytoreductive therapy. Patient-reported mild fatigue and time toxicity indicated a multifactorial burden for phlebotomy-treated patients, with patients losing 3–4 hours of their day per phlebotomy. These findings highlight a clear unmet need for therapies that improve hematocrit and symptom control while reducing time burden."

Clinical • Oncology • Polycythemia Vera

PharmaEssentia will present two-year results from the Phase 3 SURPASS-ET study (NCT04285086) and its ongoing extension in adults with high-risk essential thrombocythemia (ET) who were resistant to or intolerant of hydroxyurea (Businesswire) - "PharmaEssentia to Present at the Society of Hematologic Oncology (SOHO) 2026 Annual Meeting....In the extension, it was observed that study participants who continued BESREMi (ropeginterferon alfa-2b-njft) maintained modified ELN response rates and platelet and white blood cell control through two years, with continued reduction in JAK2V617F variant allele burden and a safety profile consistent with prior reports. Patients followed in the extension study who transitioned from anagrelide to BESREMi after the 52-week core study showed improved hematologic control and reduced JAK2V617F allele burden relative to their prior anagrelide therapy. Long-term efficacy and safety analyses are ongoing."

JAK2V617F • P3 data • Essential Thrombocythemia

Ropeginterferon-α2b in mycosis fungoides and Sézary syndrome: Safety and efficacy in 41 patients. (PubMed, J Dtsch Dermatol Ges) - " This study provides initial evidence that ropeginterferon-α2b may be effective and well tolerated in patients with CTCL. Although interpretation is limited by the small retrospective cohort and short follow-up, the observed outcomes appear comparable to those previously reported for other interferon-α formulations."

Journal • Cutaneous T-cell Lymphoma • Dermatology • Fatigue • Hematological Disorders • Hematological Malignancies • Lymphoma • Mycosis Fungoides • Neutropenia • Oncology • Sezary Syndrome • T Cell Non-Hodgkin Lymphoma

ROPEGINTERFERON ALFA-2B FOR PRE-FIBROTIC PRIMARY MYELOFIBROSIS AND DIPSS LOW/INTERMEDIATE-RISK MYELOFIBROSIS (EHA 2025) - "Ropeg-IFN-α2b was well-tolerated and induced clinical, hematologic and molecular responses in patients with pre-PMF and low/intermediate-1-risk MF."

Alopecia • Anemia • Fibrosis • Immunology • Myelofibrosis • Myeloproliferative Neoplasm • Neutropenia • Thrombocytopenia • Thrombocytosis • CALR • IFNA1

Ropeginterferon Alfa-2b in Polycythemia Vera: A PRISMA-Compliant Systematic Review and Meta-Analysis (SOHO 2026) - "Ropeginterferon alfa-2b demonstrates noninferiority to hydroxyurea and superiority over phlebotomy in short-term efficacy for PV management. Its favorable molecular and hematologic outcomes support its role as a disease-modifying therapy, particularly in low-risk patients. However, larger and longer-term trials are needed to confirm these benefits across broader populations."

Retrospective data • Review • Oncology • Polycythemia Vera • JAK2

Efficacy and Molecular Dynamics of Ropeginterferon Alfa-2b in High-Risk Essential Thrombocythemia: Two-Year Results From the Phase 3 SURPASS-ET Trial (SOHO 2026) - P3 | "The study enrolled adults with high-risk ET and leukocytosis (WBC, >10 × 109/L) following hydroxyurea failure. Early start and continuous ropeginterferon alfa-2b therapy provides durable clinical and molecular benefits in patients with high-risk ET. The progressive reduction in JAK2 V617F VAF suggests that early and sustained ropeginterferon alfa-2b exposure may significantly impact the underlying disease burden. Anagrelide-treated patients showed VAF increase, which could be reversed by starting ropeginterferon alfa-2b."

Clinical • P3 data • Essential Thrombocythemia • Hematological Malignancies • Leukemia • Myeloproliferative Neoplasm • Oncology • JAK2

A randomized, double-blind, placebo-controlled phase 3 study to assess efficacy and safety of ropeginterferon alfa-2b in patients with early/lower-risk primary myelofibrosis. (PubMed, Ann Hematol) - "Ropeg will be administered subcutaneously with a tolerable, higher starting-dose regimen. The study will provide important data for the treatment of early/lower-risk PMF for which an anti-clonal, disease-modifying agent is highly needed."

Clinical • Journal • P3 data • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Oncology

Ropeginterferon Alfa-2b vs Hydroxyurea in Polycythemia Vera: A Propensity Score-Matched Real-World Analysis of Thrombotic Events and Phlebotomy Burden (SOHO 2026) - " Using the TriNetX Global Collaborative Network (172 health care organizations), we identified adults with confirmed PV (ICD-10: D45) who initiated RPI (n = 315) or HU (n = 3263) as their first cytoreductive agent, excluding prior exposure to the comparator agent, ruxolitinib, and patients with antecedent thromboembolic events and prior myeloid malignancy/myelofibrosis. In this propensity-matched real-world analysis, RPI was associated with numerically lower thrombotic event rates and phlebotomy burden vsHUover 3 years. The magnitude and direction of the observed differences are consistent with the mechanistic rationale for interferon-based disease modification and support prospective evaluation. These findings provide the first real-world comparative thrombotic data between these two agents in PV."

Clinical • Real-world • Real-world evidence • Hematological Malignancies • Myelofibrosis • Oncology • Polycythemia Vera