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

Efficacy and Safety of Ropeginterferon Alfa-2b in the Treatment of Polycythemia Vera: A Systematic Review and Single Arm Meta-Analysis (ESMO 2025) - No abstract available

Retrospective data • Review • Oncology • Polycythemia Vera

Highlights from MPN Asia 2025: Advances in Molecular Pathogenesis and Therapeutic Strategies in Myeloproliferative Neoplasms. (PubMed, Curr Hematol Malig Rep) - "Ropeginterferon alfa-2b, a novel interferon-based therapy, demonstrated durable clinical efficacy in polycythemia vera...Combination regimens involving ruxolitinib and agents such as pelabresib, selinexor, and interferon showed potential for enhanced efficacy...Its early use and personalized strategies are increasingly recognized. Real-world data and regional insights are shaping a more nuanced, globally informed approach to MPN care."

Journal • Review • Essential Thrombocythemia • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombocytosis • ASXL1 • IL17A

Effective Use of Ropeginterferon alpha-2b and PUVA in CD30+ Large Cell Transformed Mycosis Fungoides Refractory to BV-CHP (EADV 2025) - No abstract available

Cutaneous T-cell Lymphoma • Dermatology • Mycosis Fungoides • Oncology • TNFRSF8

Real-World Evidence on Outcomes and Safety of Ropeginterferon Alfa-2b in Patients With Myeloproliferative Neoplasms: A Retrospective Cohort Study. (PubMed, Clin Lymphoma Myeloma Leuk) - "RopegIFN demonstrated hematological and molecular efficacy across Ph- MPN subtypes and was generally well tolerated. However, its effectiveness appears limited in patients with MF, particularly those with high-molecular-risk (HMR) mutations. These findings support the potential disease-modifying role of ropegIFN and highlight the need for prospective multicenter studies to validate its long-term impact on disease progression and survival."

HEOR • Journal • Real-world evidence • Retrospective data • Essential Thrombocythemia • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombocytosis • JAK2

Ropeginterferon alfa-2b versus anagrelide for the treatment of essential thrombocythemia: Topline results of the phase 3 SURPASS-ET trial. (BASCO-MN 2025) - No abstract available

P3 data • P3 data: top line • Essential Thrombocythemia • Hematological Disorders • Myeloproliferative Neoplasm • Thrombocytosis

Ropeginterferon alfa-2b as a promising alternative to conventional interferon in CDA type 1: a case report of two siblings. (PubMed, Clin Exp Med) - "Our findings suggest that ropeginterferon alfa-2b may serve as a more tolerable and effective long-term treatment. Further prospective large-scale studies are needed to validate its broader clinical applicability."

Journal • Genetic Disorders • Hematological Disorders • IFNA1

Phase I Open-Label Study of P1101 Followed by P1801 in Advanced Solid Tumors (clinicaltrials.gov) - P1 | N=54 | Enrolling by invitation | Sponsor: PharmaEssentia

New P1 trial • Solid Tumor

Ropeginterferon in Patients w/Cutaneous T-Cell Lymphoma (CTCL) (clinicaltrials.gov) - P1 | N=38 | Recruiting | Sponsor: H. Lee Moffitt Cancer Center and Research Institute

New P1 trial • Cutaneous T-cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma

UNMET NEEDS & TREATMENT GAPS AMONG PATIENTS WITH POLYCYTHEMIA VERA: A RETROSPECTIVE MEDICAL CHART AUDIT STUDY (EHA 2025) - "A retrospective medical chart review was conducted in October-December 2023 among US healthcare providers (HCPs) managing adult patients with a confirmed diagnosis of PV in the prior 12 months, receiving phlebotomy alone (PLB), or cytoreductive therapies (CT) with or without concomitant phlebotomy: hydroxyurea (HU), ruxolitinib (RUX), peginterferon alfa-2a (PEG), and ropeginterferon alfa-2b (ROP). Data suggests a diagnosis delay in low and high-risk PV and current CT is commonly initiated with HU. Despite ROP being NCCN preferred as a first-line CT in low and high-risk patients, it was underutilized. Instead, ROP appears to be used in later lines."

Retrospective data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • JAK2

The Safety and Efficacy of Sequential Treatment of Ropeginterferon Alfa-2b (P1101) and Anti-PD1 in Interferon-Naive Adults With Chronic Hepatitis B or D Infection (clinicaltrials.gov) - P1 | N=20 | Completed | Sponsor: PharmaEssentia | Recruiting ➔ Completed | Trial completion date: Mar 2026 ➔ May 2025 | Trial primary completion date: Dec 2025 ➔ May 2025

Trial completion • Trial completion date • Trial primary completion date • Hepatitis B • Infectious Disease • Inflammation

PARADIGM-PV: Efficacy and Safety of Ropeginterferon Alfa 2b (P1101) for Patients With Polycythemia Vera (clinicaltrials.gov) - P4 | N=70 | Not yet recruiting | Sponsor: PharmaEssentia | Trial completion date: Jun 2026 ➔ Jun 2027 | Trial primary completion date: Dec 2025 ➔ Dec 2026

Trial completion date • Trial primary completion date • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera

ROPEGINTERFERON ALFA-2B DEMONSTRATES SUPERIOR HEMATOLOGIC RESPONSE IN HYDROXYUREA-RESISTANT OR -INTOLERANT POLYCYTHEMIA VERA: PROPENSITY-MATCHED REAL-WORLD ANALYSIS (EHA 2025) - "Additionally, 14% of patients required multiple cytoreductive agents, while 6% received ruxolitinib, either alone or in combination with other treatments. This real-world comparison provides evidence supporting ropeginterferon alfa-2b as an effective second-line option for HU-R/I PV patients, demonstrating superior hematologic control compared to conventional salvage therapies. Further investigation into long-term outcomes is warranted."

Clinical • Real-world • Real-world evidence • Cardiovascular • Hematological Disorders • Polycythemia Vera • Thrombosis

Efficacy and Safety of Ropeginterferon Alfa-2b in Low-Risk Polycythemia Vera. (PubMed, Cancer Res Treat) - "Conventional management of low-risk polycythemia vera (PV), consisting of phlebotomy and aspirin, often fails to adequately control symptoms and hematologic parameters...The JAK2V617F allele burden decreased, and hydroxyurea-naïve patients showed better responses...Most adverse events were mild or moderate, with no grade 4 or 5 events reported. These findings suggest that Ropeg is a promising treatment option for low-risk PV by effectively reducing the need for phlebotomy and demonstrating efficacy and safety."

Journal • Alopecia • Hematological Disorders • Immunology • Polycythemia Vera

HIGH-INITIAL DOSE AND ACCELERATED TITRATION REGIMEN OF ROPEGINTERFERON ALFA-2B IN PATIENTS WITH ESSENTIAL THROMBOCYTHEMIA (EHA 2025) - "Its extended dosing interval, and improved tolerability comparable to hydroxyurea (HU), has generated increasing clinical and research interest. Our findings provide preliminary evidence that ropegIFN is a safe and effective therapeutic option for ET. Additionally, this study suggests that treatment regimens initiated at 250 mcg and maintained at 500 mcg are feasible."

Clinical • Alopecia • Asthma • Dermatology • Essential Thrombocythemia • Fatigue • Hematological Disorders • Immunology • Myeloproliferative Neoplasm • Pain • Polycythemia Vera • Pruritus • Respiratory Diseases • Thrombocytosis

IMPACT OF DNMT3A MUTATION ON TREATMENT RESPONSE TO ROPEGINTERFERON ALPHA-2B IN PATIENTS WITH POLYCYTHEMIA VERA AND ESSENTIAL THROMBOCYTHEMIA (EHA 2025) - "Preexisting DNMT3A mutations in MPN that do not result in complete loss of the DNMT3A gene function may still respond to ropeginterferon alpha-2b therapy. However, our sample size is small, and further studies are needed."

Clinical • Chronic Eosinophilic Leukemia • Essential Thrombocythemia • Fatigue • Hematological Disorders • Hematological Malignancies • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombocytosis • CALR • DNMT3A • IFNA1 • JAK2

REAL-WORLD EVIDENCE ON CLINICAL OUTCOMES AND SAFETY OF ROPEGINTERFERON ALFA-2B IN PATIENTS WITH MYELOPROLIFERATIVE NEOPLASMS: A RETROSPECTIVE COHORT STUDY (EHA 2025) - "Current treatment focuses on thrombosis prevention and symptomatic control rather than disease midifications.Ropeginterferon alfa-2b-njft (ropegIFN), a monopegylated interferon alpha, has demonstrated superior efficacy over hydroxyurea in polycythemia vera (PV), with a potential of disease modification. This study demonstrates the efficacy, safety, and molecular impact of ropegIFN in Ph- MPNs, highlighting its robust responses and the need for prospective studies to confirm its long-term impact on disease progression and survival."

Clinical data • HEOR • Real-world • Real-world evidence • Retrospective data • Cardiovascular • Essential Thrombocythemia • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombocytosis • Thrombosis • CALR • JAK2

DEEP MOLECULAR RESPONSES WITH LOW-FIXED DOSE OF ROPEGINTERFERON ALFA-2B IN A ROLLOVER COHORT OF THE LOW-PV TRIAL (EHA 2025) - "In this long-term follow-up of low-risk PV patients from the Low-PV trial, low-dose Ropeginterferon alfa-2b provided sustained hematologic control with no thrombotic events. Although the sample size was small, a significant proportion achieved deep molecular responses, comparable to dose-escalation strategies. These findings support the potential of tailored, lower-dose regimens to effectively suppress JAK2-mutant clones while optimizing efficacy, tolerability, and cost."

Acute Kidney Injury • Hematological Disorders • Infectious Disease • Polycythemia Vera • Renal Calculi • Renal Disease • Septic Shock • JAK2

MOLECULAR DETERMINANTS OF RESISTANCE TO ROPEGINTERFERON ALFA-2B IN PV AND ET (EHA 2025) - "Our findings indicate that non-responsiveness to Ropeg is associated with increased expression levels of inflammatory, HIF-targeted, and prothrombotic genes. These data suggest that therapeutic strategies, such as the use of HIF inhibitors or anti-inflammatory agents, aimed at reducing the elevated expression of these genes prior to Ropeg administration, may be beneficial."

Cardiovascular • Essential Thrombocythemia • Fatigue • Hematological Disorders • Inflammation • Myelofibrosis • Myeloproliferative Neoplasm • Polycythemia Vera • Thrombocytosis • Thrombosis • CXCL8 • IL15 • IL1B • IL6 • LDHA • SELP • SLC2A1

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

FACTORS INFLUENCING CHR AND MR ACHIEVEMENT AND MAINTENANCE IN POLYCYTHEMIA VERA PATIENTS WITH HYDROXYUREA RESISTANCE/INTOLERANCE TREATED WITH ROPEGINTERFERON ALFA-2B (EHA 2025) - "Ropeginterferon alfa-2b demonstrated promising efficacy in achieving and maintaining CHR and MR in HU R/I PV patients. A substantial proportion of patients attained durable responses, with female gender and shorter disease duration favoring earlier CHR, while higher platelet counts were associated to a longer time to first MR. These findings provide insights on optimizing therapeutic strategies for HU R/I PV management."

Clinical • Polycythemia Vera • JAK2

NEUTROPHIL-TO LYMPHOCYTE RATIO AS A SURROGATE INFLAMMATORY BIOMARKER FOR JAK2V617F SUPPRESSION AND EVENT-FREE SURVIVAL IN POLYCYTHEMIA VERA (EHA 2025) - "Aims: To evaluate in the ECLAP study the extent to which hydroxyurea (HU) modifies leukocyte-derived biomarkers compared to phlebotomy (PHL) alone and determine whether these changes are associated with thrombosis and survival outcomes.To examine the effects of Ropeginterferon alfa-2b (Ropeg) on inflammatory biomarkers relative to HU in the Low-PV Phase II trial vs ECLAP study and explore correlations between biomarker changes and JAK2V617F VAF.To validate these findings within the PROUD-PV/CONTINUATION-PV studies, which randomized PV patients to HU or Ropeg. In a propensity score matched analysis, we compared 207 HU-treated PV patients with 207 PHL-treated matched controls from the ECLAP study. These data underscore NLR as a simple, widely accessible biomarker that reflects JAK2V617F VAF dynamics and is associated with event-free survival in PV."

Biomarker • Cardiovascular • Hematological Disorders • Inflammation • Polycythemia Vera • Thrombosis

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

Phase 3 Data From EHA 2025 Support New Option for High-Risk Blood Cancer (Oncology News Central) - P3 | N=174 | SURPASS ET (NCT04285086) | Sponsor: PharmaEssentia | "Treatment with ropeginterferon alfa-2b (ropeg; Besremi) showed superior efficacy to anagrelide as a second-line treatment for patients with high-risk essential thrombocythemia (ET) who were intolerant or resistant to hydroxyurea, according to results of the phase 3 SURPASS ET trial presented at the European Hematology Association 2025 Congress (Abstract S102)...Significantly more participants assigned to ropeg achieved durable modified ELN criteria response rate at nine and 12 months compared with patients assigned to anagrelide (42.9% vs. 6.0%; P=.0001)....An important secondary endpoint was overall molecular response rate at 12 months. Partial molecular response was achieved in 27.8% of patients, and complete molecular response was achieved in 2.8% of patients assigned to ropeg compared with no patients assigned anagrelide."

P3 data • Essential Thrombocythemia

Ropeginterferon Alfa 2b Plus Ruxolitinib for Myelofibrosis (clinicaltrials.gov) - P2 | N=20 | Recruiting | Sponsor: The University of Hong Kong | Not yet recruiting ➔ Recruiting

Enrollment open • Monotherapy • Myelofibrosis

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