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

Ropeginterferon alfa-2b versus anagrelide for the treatment of essential thrombocythemia: Topline results of the phase 3 SURPASS-ET trial. (ASCO 2025) - P3 | "Clinical Trial Registration Number: NCT04285086 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

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

Ropeginterferon alfa-2b for pre-fibrotic primary myelofibrosis and DIPSS low/intermediate-risk myelofibrosis. (ASCO 2025) - P2 | "Clinical Trial Registration Number: NCT04988815 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Myelofibrosis

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 TLR screened clinical comparative evidence on PV treatments from 5/2014-5/2024 from PubMed and relevant conference abstracts. 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

Navigating the peginterferon Alfa-2a shortage: practical guidance on transitioning patients to ropeginterferon alfa-2b. (PubMed, Ann Hematol) - No abstract available

Journal

Sequential combination with ropeginterferon alfa-2b and anti-PD-1 treatment as adjuvant therapy in HBV-related HCC: a phase 1 dose escalation trial. (PubMed, Hepatol Int) - "This phase I trial supports the safety and clinical efficacy of sequential treatment with ropeg and nivolumab in post-resection HBV-related HCC. This regimen holds promise for further adjuvant therapy trials in HCC, both HBV-related and other types."

Journal • P1 data • Hepatitis B • Hepatocellular Cancer • Hepatology • Infectious Disease • Oncology • Solid Tumor

[Announcement] PharmaChina's Phase III clinical trial application for Ropeginterferon alfa-2b (P1101) for early primary myelofibrosis or low or intermediate risk grade 1 significant primary myelofibrosis has been approved by the China National Medical Products Administration [Google translation] (stock.yahoo) - "PharmaChina's Phase III clinical trial application for Ropeginterferon alfa-2b (P1101) for early primary myelofibrosis or low or intermediate risk grade 1 significant primary myelofibrosis has been approved by the China National Medical Products Administration...The National Medical Products Administration (hereinafter referred to as the China National Medical Products Administration) has approved and agreed to conduct clinical trials....Estimated Completion Time: It is expected to be completed in 2025, but the actual schedule will be adjusted according to the execution progress."

New P3 trial • Trial completion date • Myelofibrosis

Pint Pharma Announces ANVISA’s Approval of BESREMi (ropeginterferon alfa-2b) for the Treatment of Polycythemia Vera (Businesswire) - "Pint Pharma and PharmaEssentia announced today that ANVISA (Brazilian Health Regulatory Agency) has approved BESREMi (ropeginterferon alfa-2b) for the treatment of adult patients with Polycythemia Vera (PV)....'Its approval was based on robust evidence from the PEGINVERA clinical study, which demonstrated superior and more sustained hematologic and clinical responses compared to standard therapy'."

Approval • Polycythemia Vera

Hematologic and molecular responses to ropeginterferon alfa-2b therapy of polycythemia vera: 48-week results from a prospective study. (PubMed, Int J Cancer) - "To prevent thrombosis in patients with polycythemia vera (PV), achieving a complete hematologic response (CHR) is highly recommended in practice. In the subgroup analysis, patients with hydroxyurea resistance or intolerance, and those who were hydroxyurea-naïve, had similar results in terms of the CHR. In conclusion, CHR and MR were observed to be associated in patients with PV treated with ropeginterferon."

Journal • Tumor mutational burden • Cardiovascular • Hematological Disorders • Polycythemia Vera • Thrombosis • JAK2 • TMB

ECLIPSE-PV: A Randomized, Multicenter Study to Assess Efficacy, Safety, and Tolerability of Two Dosing Regimens of Ropeginterferon alfa-2b-njft in Polycythemia Vera. (PubMed, Acta Haematol) - "The approved regimen requires an initial dose of 100 μg or 50 μg if transitioning from hydroxyurea (HU) and up-titrations of 50 μg every two weeks to 500 μg maximumly. The study is expected to be completed in the summer of 2025. This is the first prospective trial comparing two dosing regimens of ropeg. The results will inform the optimal treatment strategy for patients with PV."

Clinical • Journal • Hematological Disorders • Polycythemia Vera

PharmaEssentia Highlights Availability of BESREMi (ropeginterferon alfa-2b-njft) for Patients Affected by Pegasys (peginterferon alfa-2a) Drug Shortage (Businesswire) - "PharmaEssentia USA Corporation...today announced that the latest National Comprehensive Cancer Network (NCCN) Guidelines (Version 1.2025, January 21, 2025) now highlight the substitution of ropeginterferon alfa-2b-njft (BESREMi) for treating polycythemia vera (PV) in the event that peginterferon alfa-2a (Pegasys) is unavailable....The updated NCCN Guidelines come at a crucial time, as an ongoing Pegasys shortage poses challenges in treatment continuity."

NCCN guideline • Polycythemia Vera

Research Progress of Ropeginterferon α-2b in Treatment of Myeloproliferative Neoplasm --Review (PubMed, Zhongguo Shi Yan Xue Ye Xue Za Zhi) - "It has a lower incidence and severity of adverse drug reactions than pegylated interferon alpha and hydroxyurea and a longer dosing interval. Some patients with lowrisk PV and myelofibrosis can benefit from it. This article reviews the latest progress of Ropeg in MPN."

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

NCCN has published updates to the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Myeloproliferative Neoplasms, Version 1.2025. (NCCN)

NCCN guideline • Myelofibrosis • Myeloproliferative Neoplasm

ICC Arbitral Tribunal Rules in Favor of AOP Health in Arbitration Proceedings Pertaining to BESREMi (Ropeginterferon alfa-2b) (Businesswire) - "Today, a tribunal under the auspices of the International Chamber of Commerce (ICC) served a partial final award in a dispute between PharmaEssentia Corp. ('PharmaEssentia') and AOP Orphan Pharmaceuticals GmbH ('AOP Health') in AOP Health’s favor. At the heart of the case were reciprocal claims for damages arising from a dispute regarding a licensing agreement between the parties...The conflict centers around ropeginterferon alfa-2b, a substance launched in 2019 and that AOP Health has developed into an innovative treatment (BESREMi) for rare blood cancers, particularly polycythemia vera, through a comprehensive clinical trial program....AOP Health welcomes this decision. It will continue to supply patients in need of ropeginterferon alfa-2b (BESREMi)."

Commercial • Polycythemia Vera

Besremi - ropeginterferon alfa-2b (European Medicines Agency) - "Polycythaemia vera is rare, and Besremi was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 9 December 2011."

Orphan drug • Polycythemia Vera

FDA Approves Treatment for Rare Blood Disease (FDA) - "Today, the U.S. Food and Drug Administration approved Besremi (ropeginterferon alfa-2b-njft) injection to treat adults with polycythemia vera, a blood disease that causes the overproduction of red blood cells. The excess cells thicken the blood, slowing blood flow and increasing the chance of blood clots."

FDA approval • Polycythemia Vera

Orphan Drug Designations and Approvals (FDA) - Generic Name: ropeginterferon alfa-2b-njft; Trade Name: Besremi; Date Designated: 04/02/2012; Orphan Designation: Treatment of polycythemia vera; Orphan Designation Status: Designated/Approved

Orphan drug • Polycythemia Vera

Cost-utility analysis of ropeginterferon alfa-2b to manage low-risk patients with polycythemia vera as compared to phlebotomy only in the Austrian healthcare system. (PubMed, Ann Hematol) - "RopegIFNα is a cost-effective treatment option for patients with low-risk PV. These findings suggest that early treatment with ropegIFNα could ensure optimal resource allocation by preventing costly thrombotic events and progression to MF whilst increasing patient quality of life."

HEOR • Journal • Acute Myelogenous Leukemia • Cardiovascular • Hematological Disorders • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombosis • IFNA1

Improving the management of polycythemia vera patients eligible for cytoreduction: report of a multidisciplinary advisory board. (PubMed, Curr Med Res Opin) - "The management of patients with Polycythemia Vera (PV) traditionally includes low-dose aspirin, phlebotomy, and cytoreductive therapy for high-risk individuals...Introducing new therapeutic agents, including ropeginterferon alfa-2b and ruxolitinib, enables a more personalized treatment approach tailored to individual patient characteristics...The advent of new criteria for starting cytoreduction and the approval of novel drugs for PV has increased the complexity of selecting appropriate cytoreductive therapies. A multidisciplinary approach is increasingly essential to ensure personalized care that maximizes tolerability and minimizes adverse events, particularly given the often chronic nature of the treatment."

Journal • Cardiovascular • Polycythemia Vera • Thrombosis

PARADIGM-PV: a randomized, multicenter phase 4 study to assess the efficacy and safety of ropeginterferon alfa-2b in patients with low- or high-risk polycythemia vera. (PubMed, Ann Hematol) - "Phlebotomy (Phl) and aspirin are often used alone for low-risk PV patients...Patients will be randomized equally to receive either ropeg every two weeks or to continue their current treatment with Phl or other cytoreductive agents (e.g., hydroxyurea, other interferons, or ruxolitinib) as applicable...The study will enroll approximately 70 patients internationally, including patients in the US. This study will provide new efficacy data, measured as the ability of ropeg to reduce Phl eligibility and modify the disease."

Journal • P4 data • Hematological Disorders • Oncology • Polycythemia Vera • JAK2

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

Monotherapy • New P2 trial • Myelofibrosis • CALR

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 | Recruiting | Sponsor: PharmaEssentia | Trial completion date: Mar 2023 ➔ Mar 2026 | Trial primary completion date: Mar 2023 ➔ Dec 2025

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

Fedratinib combined with ropeginterferon alfa-2b in patients with myelofibrosis (FEDORA): study protocol for a multicentre, open-label, Bayesian phase II trial. (PubMed, BMC Cancer) - "FEDORA is using a Bayesian trial design and aims to provide evidence of the tolerability, safety, and activity of combining fedratinib with ropeginterferon alfa-2b upon which the decision as to whether a phase III trial is warranted will be based."

Journal • P2 data • Myelofibrosis

PharmaEssentia Announces Positive Topline Phase 3 Data from SURPASS-ET Study Evaluating Ropeginterferon Alfa-2b-njft (P1101) for Essential Thrombocythemia (Businesswire) - P3 | N=174 | SURPASS-ET (NCT04285086) | Sponsor: PharmaEssentia | "The trial successfully met its primary endpoint, demonstrating durable clinical response as measured using modified European Leukemia Net (ELN) criteria. Among the intent-to-treat (ITT) population, 42.9% (39/91) of participants in the P1101 group achieved durable responses at months 9 and 12, compared to 6.0% (5/83) in the comparator arm (administered Anagrelide) (p=0.0001). P1101 exhibited a manageable safety profile with no treatment-related serious adverse events....PharmaEssentia plans to present detailed clinical trial results, including additional pharmacokinetics and biomarker data, at a later date. The Company also intends to pursue regulatory discussions with the FDA to expand the existing label for P1101 to include a new potential indication of ET and anticipates regulatory submission by the end of 2025."

FDA filing • P3 data: top line • Essential Thrombocythemia

P1101 in ET patients in its EXCEED-ET clinical trial in North America (Businesswire) - "The Company is also evaluating P1101 in ET patients in its EXCEED-ET...clinical trial in North America. This Phase 2b clinical trial is a single-arm, multicenter trial designed to assess the efficacy, safety, and tolerability of P1101 in adult patients with ET. The company expects to present data from this clinical trial in the second half of 2025."

P2b data • Essential Thrombocythemia

Genetic Profile Linked with High-Altitude Andean Population Correlates with Better Response to Treatment for Myeloproliferative Neoplasms (PRNewswire) - P=NA | N=45 | "Our study suggests that with genotyping, the NFKB1 variant can be used as a biomarker for determining which patients may be more or less responsive to Ropeginterferon-α treatment....The researchers also analyzed the linkages between a person's NFKB1 genotype and their likelihood of achieving a complete hematologic response (a return to normal blood counts) after Ropeginterferon-α treatment....In this study, the researchers found that the T variant was associated with decreased inflammatory, prothrombotic, and HIF activity, and a better response to Ropeginterferon-α in PV and ET, which are characterized by chronic inflammation."

Biomarker • Clinical data • Essential Thrombocythemia • Polycythemia Vera