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

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

Ropeginterferon alfa-2b for pre-fibrotic primary myelofibrosis and DIPSS low/intermediate-risk myelofibrosis. (ASCO 2025) - P2 | "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 • Neutropenia • Thrombocytopenia • Thrombocytosis • CALR • IFNA1

PharmaEssentia Announces Presentations at EHA 2025 Highlighting Clinical Advances in Myeloproliferative Neoplasms (Businesswire) - "The company’s results from the Phase 3 SURPASS-ET trial (NCT04285086) have been selected for an oral presentation during the plenary session—one of six top-ranked abstracts chosen by the EHA 2025 Scientific Program Committee....In addition to the plenary presentation, Dr. Gill will also present results from the University of Hong Kong’s Phase 2 trial (NCT05731245) evaluating ropeginterferon alfa-2b in pre-fibrotic primary myelofibrosis (PMF) and DIPSS low/intermediate-risk myelofibrosis during an oral session focused on innovative treatment approaches in myeloproliferative neoplasms (MPNs)."

P2 data • P3 data: top line • Essential Thrombocythemia • Myelofibrosis • Myeloproliferative Neoplasm

Evaluating the Feasibility of a Network Meta-Analysis Comparing Treatment Options in Polycythemia Vera (ISPOR 2025) - "Therefore, a targeted literature review (TLR) and subsequent feasibility of indirectly comparing ropeginterferon alfa-2b to peginterferon alfa-2a or ruxolitinib using standard of care as the common comparator was performed. A NMA for a rare disease like PV was not feasible due to heterogeneity among studies including the population of interest, treatments, outcome definitions, length of follow-up and potential treatment effect modifiers."

Retrospective data • Hematological Disorders • Hematological Malignancies • Oncology • Polycythemia Vera • Rare Diseases

Ropeginterferon-α2b discontinuation after long-term exposure: four cases from a single institution. (PubMed, Int J Hematol) - "Continuous therapy remains the standard approach due to the lack of predictive models to identify patients who can safely discontinue RopegIFN. In the future, it will be necessary to verify whether patients who have achieved an "operational cure" can remain treatment free."

Journal • Hematological Disorders • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • JAK2

PharmaEssentia to Present Phase 3 SURPASS-ET Data in Oral Session at 2025 ASCO Annual Meeting (Businesswire) - "PharmaEssentia USA Corporation...announced it will present results from the Phase 3 SURPASS-ET clinical trial in an oral session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place May 30-June 3 in Chicago."

P3 data • Essential Thrombocythemia

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

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

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

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

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

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

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 • Cardiovascular • Essential Thrombocythemia • Gastrointestinal Disorder • 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 • Hematological Disorders • Polycythemia Vera • JAK2

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

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

Novel treatment strategies for polycythemia vera: focus on ropeginterferon alfa-2b (PubMed, Rinsho Ketsueki) - "A phase 3 randomized study (PROUD-PV/CONTINUATION-PV) revealed that treatment with ropeg continuously decreased the JAK2V617F allele burden compared to hydroxyurea and improved event-free survival (with events defined as thromboembolic events, disease progression, or death). A phase 2 study in Japanese patients with PV further confirmed the efficacy of ropeg."

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

Management of common autoimmune diseases in patients with myeloproliferative neoplasms treated with pegylated interferon alfa-case report, review of the literature and multidisciplinary clinical practice recommendations. (PubMed, Ther Adv Hematol) - "Ropeginterferon alfa-2b has been approved for patients with polycythemia vera (PV) with no symptomatic splenomegaly as first-line cytoreductive therapy, based on the results of the PROUD-PV/CONTINUATION-PV studies, documenting significantly higher rates of complete hematologic response (CHR) compared with hydroxyurea from 2-year timepoint onward...The other patient developed widespread psoriatric skin lesions, which were treated with moderate effect with topical therapy with calcipotriene and betamethasone dipropionate foam, antidry calm lotion and in the course of the disease with ultraviolet light therapy (UVB). Only with methotrexate she achieved a nearly complete remission of the psoriasis...We review the pertinent literature on the management of interferon-mediated autoimmune diseases in patients with MPN. As there is little published evidence on that topic, we propose multidisciplinary clinical practice recommendations based on available evidence and clinical..."

Journal • Dermatology • Hematological Disorders • Immunology • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Psoriasis • Sarcoidosis

3 new blood disease drugs eligible for health insurance coverage in Korea (Korea Biomedical Review) - "The Health Insurance Review and Assessment Service (HIRA) held its fifth Pharmaceutical Reimbursement Evaluation Committee (PREC) meeting for 2025 on Thursday and announced the results of its deliberations. First, the PREC recognized the appropriateness of Besremi's proposed reimbursement. Although the drug has been available in Korea for three and a half years, this marks a major step forward in securing reimbursement. Besremi, approved by the Ministry of Food and Drug Safety (MFDS) in October 2021 after being designated an orphan drug, has been used to treat polycythemia vera (PV)....Following the PREC’s approval, Besremi is expected to gain reimbursement coverage after pricing negotiations with the National Health Insurance Service (NHIS) and final approval by the Health Insurance Policy Evaluation Committee."

Reimbursement • Polycythemia Vera

A multicenter study to assess efficacy, safety, and tolerability of ropeginterferon alfa-2b-njft in patients with essential thrombocythemia in the US and Canada: EXCEED-ET trial. (PubMed, Front Med (Lausanne)) - No abstract available

Clinical • Journal • Essential Thrombocythemia • Hematological Disorders • Myeloproliferative Neoplasm • Thrombocytosis

Patient group urges quick reimbursement of Besremi for polycythemia vera (Korea Biomedical Review) - "The Korea Leukemia Patients Organization (KLPO) has called for the reimbursement of Besremi Inj. (ropeginterferon alfa-2b), the only treatment alternative for patients with polycythemia vera (PV), ahead of the upcoming reimbursement review committee meeting on Thursday this week. The patient group issued a statement last Friday, urging the government and pharmaceutical companies to expedite the reimbursement process for Besremi....Besremi is a third-generation mono-pegylated proline interferon that selectively eliminates JAK2-mutated hematopoietic stem cells, the underlying cause of PV. It can be administered once every two weeks initially, and once every four weeks with long-term use. However, the drug is not covered by insurance and costs 4.25 million won (approximately $3,054) per dose, resulting in annual treatment expenses amounting to tens of millions of won."

Reimbursement • Polycythemia Vera

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