Efficacy and Safety of Ropeginterferon Alfa 2b (P1101) for Patients With Polycythemia Vera - A Randomized Open Label Global Multicenter Study (PARADIGM-PV)

This is a randomized, open-label, multicenter, two-arm study to assess the efficacy and safety of ropeginterferon alfa-2b for patients with PV. The entire study period is 60 weeks, including a main treatment phase (32 weeks), an extension treatment phase (24 weeks), and a safety follow-up phase (four weeks). However, the study may be extended for additional period of treatment after Week 60 pending the primary endpoint analysis at Week 32. Approximately 70 patients with PV will be enrolled.

开始日期2026-02-01

申办/合作机构

药华医药股份有限公司

NCT07047885

/ Recruiting临床1期IIT

A Phase I/IB Study of Ropeginterferon in Patients With Cutaneous T-Cell Lymphoma (CTCL)

This is a single-center, phase I/IB study to identify the recommended phase II dose of Ropeginterferon-alfa 2b (P1101) in patients with CTCL who have failed at least two prior lines of skin-directed therapy (Stage IA-IB) or have less than a complete response to phototherapy or extracorporeal photopheresis (ECP) or total skin electron beam therapy (TSET), or stable/progressive disease after at least two lines of topical therapy (Stage IIA-IIIB).

开始日期2025-08-27

申办/合作机构

H. Lee Moffitt Cancer Center & Research Institute, Inc.

[+1]

NCT06468033

/ Recruiting临床3期

A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase III Study to Assess Efficacy and Safety of Ropeginterferon Alfa-2b (P1101) in Adult Patients With Pre-fibrotic/Early Primary Myelofibrosis (PMF) or Overt PMF at Low or Intermediate-1 Risk According to DIPSS Plus (HOPE-PMF): The Core Study and Its Extension Study

This is a phase 3 double-blind clinical trial arm to test Ropeginterferon alfa-2b (P1101) in adult patients with Primary Myelofibrosis (PMF) at early stage or low to medium risk.
Participants will receive the study drug/placebo bi-weekly and have an assessment visit every 4 weeks. The ratio of study drug to placebo group is 2:1.

开始日期2025-07-18

申办/合作机构

药华医药股份有限公司

100 项与罗培干扰素α-2b 相关的临床结果

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100 项与罗培干扰素α-2b 相关的转化医学

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项与罗培干扰素α-2b 相关的文献（医药）

2025-12-01·Current Hematologic Malignancy Reports

Highlights from MPN Asia 2025: Advances in Molecular Pathogenesis and Therapeutic Strategies in Myeloproliferative Neoplasms

Review

作者: Yacoub, Abdulraheem ; Li, Bing ; Mascarenhas, John O ; Duan, Minghui ; Kiladjian, Jean-Jacques ; Hou, Hsin-An ; Hobbs, Gabriela S ; Lee, Sung-Eun ; Bose, Prithviraj ; Rampal, Raajit ; Tashi, Tsewang ; Qin, Albert ; Gill, Harinder ; Huang, Jian ; Hasselbalch, Hans C ; Mesa, Ruben A ; Kirito, Keita ; Yu, Lennex Hsueh-Lin ; Xiao, Zhijian ; Shimoda, Kazuya ; Prchal, Josef T

PURPOSE OF REVIEW:

This report summarizes key insights from the 8th Annual International Symposium on Myeloproliferative Neoplasms (MPN Asia 2025). The symposium brought together global experts to discuss advancements in MPN biology, diagnostics, and therapeutics, with a focus on emerging molecular understanding, novel treatment strategies and real-world data.

RECENT FINDINGS:

Molecular profiling has become essential in MPN risk stratification and therapeutic decision-making. High-risk mutations (e.g., ASXL1, TP53) and inflammatory pathways (e.g., IL-17, NF-κB) were shown to correlate with disease progression and transformation. Interferon-based therapy is increasingly used in younger, low-risk, or treatment-naïve patients, and is also being investigated in myelofibrosis and essential thrombocythemia. Ropeginterferon alfa-2b, a novel interferon-based therapy, demonstrated durable clinical efficacy in polycythemia vera. Its high initial-dose and accelerated titration (HIDAT) regimen led to fast achievement of complete hematologic response, rapid reductions in JAK2V617F allele burden, and high complete molecular response rate. Combination regimens involving ruxolitinib and agents such as pelabresib, selinexor, and interferon showed potential for enhanced efficacy. Population-based studies from Asia contributed regional epidemiological and treatment data, reinforcing the role of real-world evidence. Modern prognostic models such as MIPSS70+ v2.0 and GIPSS were discussed for more precise risk prediction. Preliminary findings also suggest ropeginterferon alfa-2b may be a safe option during pregnancy. MPN Asia 2025 highlighted the growing role of molecular diagnostics and targeted therapeutics in the management of MPNs. Ropeginterferon alfa-2b has emerged as a therapeutic potential across the MPN spectrum. 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.

2025-09-01·INTERNATIONAL JOURNAL OF HEMATOLOGY

Successful treatment of myeloid blast phase chronic myelogenous leukemia with the JAK2 V617 F mutation by combination therapy with asciminib and ropeginterferon alfa-2b in an elderly patient

Article

作者: Mituyoshi, Takaya ; Akagi, Yuina ; Oka, Satoko ; Ono, Kazuo

Abstract:

The co-occurrence of JAK2 V617F mutations and the BCR::ABL1 translocation in the same patient is rare, and the current standard treatment for aggressive myeloid blast phase chronic myeloid leukemia (CML-myeloid BP) with JAK2 V617F mutations remains inadequate, particularly in transplant-ineligible patients. Asciminib, a first-in-class allosteric inhibitor of BCR::ABL1 kinase that specifically targets the ABL1 myristoyl pocket, has emerged as a novel alternative to standard tyrosine kinase inhibitor (TKI) therapy. Ropeginterferon alfa-2b (ropegIFNα2b) is a novel site-selective, monopegylated recombinant human IFN with long-term safety and efficacy in patients with polycythemia vera (PV). Here, we report a case of successful combination therapy with asciminib and ropegIFNα2b in a patient with CML-myeloid BP who had a long history of PV with JAK2 V617F refractory to induction chemotherapy with several TKIs. The combination of asciminib and ropegIFNα2b is a promising new treatment option for these patients.

2025-08-01·INTERNATIONAL JOURNAL OF CANCER

Hematologic and molecular responses to ropeginterferon alfa‐2b therapy of polycythemia vera: 48‐week results from a prospective study

Article

作者: Sohn, Sang Kyun ; Bae, Sung Hwa ; Oh, Suk Joong ; Shin, Ho‐Jin ; Choi, Eun‐Ji ; Park, Joon Seong ; Yang, Deok‐Hwan ; Lee, Gyeong‐Won ; Park, Yong ; Yoon, Seug Yun ; Bang, Soo‐Mee ; Choi, Chul Won ; Cheong, June‐Won ; Lee, Sung‐Eun ; Yoon, Sung‐Soo ; Park, Young Hoon

Abstract:

To prevent thrombosis in patients with polycythemia vera (PV), achieving a complete hematologic response (CHR) is highly recommended in practice. In addition, a reduced JAK2 V617F mutation burden is expected to have a disease‐modifying effect, and its molecular response (MR) is currently of significant interest. This study aimed to assess the association between CHR and MR in patients with PV following treatment with ropeginterferon alfa‐2b. This phase 2, single‐arm, open‐label, investigator‐initiated trial was conducted at 16 sites in South Korea. Ninety‐nine patients were treated with ropeginterferon alfa‐2b subcutaneously every 2 weeks, at doses of 250 μg (week 1), 350 μg (week 3), and 500 μg (week 5), until week 48. CHRs were 27% (25/94), 46% (40/87), 56% (47/84), and 63% (51/81) at 12, 24, 36, and 48 weeks, respectively. The MR rates were 32% (28/88), 36% (29/81), 49% (38/77), and 57% (42/74) at 12, 24, 36, and 48 weeks, respectively. The Phi Coefficient for the association between CHR and MR was 0.6146 (p < .0001) at 48 weeks. 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.

项与罗培干扰素α-2b 相关的新闻（医药）

2025-09-03

·PRNewswire

Myelofibrosis Market is Going to Grow at a CAGR of 9% in the Next Ten Years (2025-2034) | DelveInsight

The launch of emerging therapies like BESREMi (PharmaEssentia and AOP Orphan Pharmaceuticals), INCB057643 (Incyte), XPOVIO (Karyopharm Therapeutics), RYTELO (Geron), REBLOZYL (Bristol Myers Squibb), Navtemadlin (Kartos Therapeutics), Pelabresib (Novartis), and others is going to shift the myelofibrosis market. LAS VEGAS, Sept. 3, 2025 /PRNewswire/ -- DelveInsight's Myelofibrosis Market Insights report includes a comprehensive understanding of current treatment practices, emerging myelofibrosis drugs, market share of individual therapies, and current and forecasted myelofibrosis market size from 2020 to 2034, segmented into leading markets (the US, EU4, UK, and Japan). Myelofibrosis Market Summary The total myelofibrosis treatment market size in the leading markets (the US, EU4, UK, and Japan) was USD 2.2 billion in 2024. The United States accounts for the largest market size of myelofibrosis, in comparison to EU4 (Germany, Italy, France, and Spain) and the UK, and Japan. Based on DelveInsight's assessment in 2024, the 7MM had approximately 56K patient pool (prevalent cases) of myelofibrosis. Key myelofibrosis companies, including PharmaEssentia, AOP Orphan Pharmaceuticals, Incyte, Karyopharm Therapeutics, Geron, Bristol Myers Squibb, Kartos Therapeutics, Novartis, Merck, Telios Pharma, Ryvu Therapeutics, Sumitomo Pharma, Syntara, Disc Medicine, Menarini Group, and others, are actively working on innovative myelofibrosis drugs. Some of the key myelofibrosis therapies in clinical trials include BESREMi (ropeginterferon alfa-2b-njft/P-1101), INCB057643, XPOVIO (NEXPOVIO/selinexor/KPT-330), RYTELO (imetelstat), REBLOZYL (luspatercept/ACE-536), Navtemadlin (KRT-232), Pelabresib (DAK539), Bomedemstat (IMG-7289/MK-3543), TL-895, RVU120 ( SEL-120), TP-3654 (nuvisertib), SNT-5505 (PXS-5505), DISC-0974, ELZONRIS (tagraxofusp/SL-401), and others. These novel myelofibrosis therapies are anticipated to enter the myelofibrosis market in the forecast period and are expected to change the market. By 2034, among all the therapies, the highest revenue is expected to be generated by OJJAARA/OMJJARA. Discover which myelofibrosis therapies are expected to grab the largest market share @ Myelofibrosis Market Report Key Factors Driving the Growth of the Myelofibrosis Market Novel Therapies Drive Myelofibrosis Treatment Advancements Currently, four JAK inhibitors have been approved by the US FDA for the treatment of myelofibrosis, including JAKAFI (ruxolitinib), INREBIC (fedratinib), VONJO (pacritinib), and OJJAARA (momelotinib). No drug therapy can cure myelofibrosis. As myelofibrosis primarily affects older adults, stem cell transplantation is not a treatment option for most myelofibrosis patients. The launch of another JAK inhibitor, BESREMi, will further change the dynamics of the myelofibrosis market. Rich clinical pipeline and emerging non-JAK approaches driving the myelofibrosis landscape Beyond JAK inhibitors, active clinical development includes BET inhibitors (Incyte's INCB057643; Novartis' Pelabresib), XPO1 inhibitor (Karyopharm Therapeutics' XPOVIO), Telomerase inhibitor (Geron's RYTELO), MDM2 protein inhibitor (Kartos Therapeutics' Navtemadlin), Tyrosine kinase inhibitors (Telios Pharma's TL-895), PIM1 kinase inhibitor (Sumitomo Pharma's TP-3654), LOX inhibitor (Syntara's SNT-5505), and others, raising market potential by promising new label expansions, second-line options, and higher-value therapies should any prove to be disease-modifying. An aging population & increasing incidence with better survival Myelofibrosis primarily affects older adults; population aging increases the absolute number of patients. As treatments improve symptoms and (in some cases) survival, prevalent patient pools grow, supporting longer-term market demand. In the US in 2024, the 70+ years of age group had the highest number of cases, accounting for approximately 60% of the total prevalent cases, while the ≤39 years of age group accounted for just ~2%. Diagnostic & genomic testing improvements are driving myelofibrosis patient pool, leading to the growth of myelofibrosis market Wider use of molecular profiling (JAK2, CALR, MPL, and others) enables earlier and more accurate MF diagnosis and better patient stratification for targeted therapies. This both expands the diagnosed population and helps match patients to appropriate, often higher-value, treatments. Myelofibrosis Market Analysis JAK inhibitors have become the cornerstone of treatment for patients with myelofibrosis, offering significant benefits such as spleen reduction, symptom relief, and improved quality of life, which can also extend survival in those with advanced disease. All approved JAK inhibitors primarily target JAK2, particularly the wild-type form, but they differ in their activity against other JAK family members. For instance, JAKAFI myelofibrosis inhibits both JAK1 and JAK2; INREBIC selectively inhibits JAK2 while sparing JAK1 and also affects FLT3 and other targets; VONJO myelofibrosis inhibits JAK2 while sparing JAK1 but additionally impacts FLT3, IRAK, and ACVR1; and OJJAARA, approved through a different pathway, targets JAK1/JAK2 and ACVR1, mainly for myelofibrosis patients with anemia. These varying mechanisms lead to distinct patient outcomes. JAKAFI myelofibrosis continues to see strong demand and is expected to grow further, maintaining its position as the standard of care in myelofibrosis. Myelofibrosis will remain the largest segment of JAKAFI's patient population until polycythemia vera cases eventually increase. However, market growth may be limited due to patent expirations of key therapies, with JAKAFI patents set to expire in 2027 for Novartis and 2028 for Incyte, presenting potential opportunities for competitors. In response, Incyte is exploring combination trials with novel drugs to extend JAKAFI's therapeutic lifespan. Learn more about the treatment options for myelofibrosis @ Myelofibrosis Therapy The myelofibrosis treatment landscape offers significant opportunities for innovation, focusing on several key areas: Treating Lower-Risk Patients: Developing safe and effective therapies for patients with lower-risk myelofibrosis, allowing for earlier and more favorable interventions. Enhancing First-Line Treatments: Improving existing first-line therapies for intermediate- to high-risk patients through novel drugs or combination strategies. Managing Cytopenia: Developing targeted therapies to address cytopenia, a significant unmet need in patient care. Myelofibrosis Competitive Landscape The myelofibrosis clinical trial pipeline includes several drugs in mid- and late-stage development that are expected to enter the market during the forecast period. The emerging landscape offers a diverse range of therapeutic alternatives for treatment, including XPOVIO (Karyopharm Therapeutics), Elitracet (Takeda/Keros Therapeutics), Navtemadlin (Kartos Therapeutics), Pelabresib (Novartis), INCB057643 (Incyte), Bomedemstat (Merck), and others, all of which are used in various lines of treatment. The expected launch of these therapies is expected to have a further positive impact on the market. INCB57643 is an orally administered small molecule. Bromodomain and extra-terminal (BET) proteins act as epigenetic readers that control the expression of key oncoproteins implicated in the development of myelofibrosis and other hematologic malignancies, including B-lymphoma-2, nuclear factor kappa, and c-Myc. In a prior Phase I/II clinical trial, the oral BET inhibitor INCB057643, tested both as a monotherapy and in combination with ruxolitinib, demonstrated favorable tolerability and promising clinical activity in patients with advanced cancers. In its Q2 2025 financial report, the company announced that the combination of INCB057643 with ruxolitinib and INCB57643 (a JAK1/JAK2 and BET inhibitor) is currently being evaluated in a Phase II trial for myelofibrosis. RYTELO (imetelstat) is an investigational telomerase inhibitor that targets telomerase, selectively eliminating malignant stem and progenitor cells in the bone marrow, which drive diseases like myelodysplastic syndromes (MDS) and myelofibrosis. By blocking the proliferation of these malignant cells, imetelstat supports the recovery of healthy bone marrow and blood cell production and has shown disease-modifying effects and clinical benefits in Phase III trials for myelofibrosis. This mechanism sets imetelstat apart from other approved or investigational therapies for these blood cancers. In January 2025, Geron reported achieving 75% enrollment in the Phase III IMpactMF trial, which is comparing imetelstat to Best Available Therapy (BAT) in intermediate-2 or high-risk myelofibrosis patients who have relapsed or are refractory to JAK inhibitor treatment. Elritercept is a late-stage investigational activin inhibitor aimed at treating anemia associated with hematologic malignancies, including MDS and myelofibrosis. It is currently undergoing Phase II evaluation in patients with myelofibrosis. In December 2024, Keros Therapeutics presented updated data from this ongoing Phase II trial at ASH 2024. Additionally, in December 2024, Takeda announced an exclusive licensing agreement with Keros to further develop, manufacture, and commercialize elritercept globally, excluding mainland China, Hong Kong, and Macau. The anticipated launch of these emerging myelofibrosis therapies are poised to transform the myelofibrosis market landscape in the coming years. As these cutting-edge myelofibrosis therapies continue to mature and gain regulatory approval, they are expected to reshape the myelofibrosis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about new myelofibrosis treatment, visit @ Myelofibrosis Treatment Market Recent Developments in the Myelofibrosis Market In July 2025, Incyte announced that the Phase I data in patients with myelofibrosis as monotherapy and in combination with ruxolitinib are anticipated in the second half of 2025. In June 2025, QIAGEN and Incyte announced a new global collaboration to develop a novel diagnostic panel to support Incyte's extensive portfolio of investigational therapies for patients with myeloproliferative neoplasms (MPNs), including Incyte's monoclonal antibody INCA033989. In January 2025, Karyopharm Therapeutics stated that it expects to report top-line results from the Phase III SENTRY trial in the second half of 2025, which could represent a potentially transformative opportunity to establish a new treatment paradigm in myelofibrosis. What is Myelofibrosis? Myelofibrosis is a rare blood cancer marked by the accumulation of scar tissue, or 'fibrosis', in the bone marrow. This excess scar tissue prevents the bone marrow from producing enough healthy blood cells. It belongs to a group of blood cancers called 'myeloproliferative neoplasms (MPNs)', in which the blood cells produced by the bone marrow grow and function abnormally. When myelofibrosis arises independently, without being caused by another bone marrow disorder, it is referred to as primary myelofibrosis. In other cases, it can develop from another MPN, such as polycythemia vera or essential thrombocythemia. When this occurs, it is called secondary myelofibrosis, sometimes specifically termed post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. Myelofibrosis Epidemiology Segmentation The myelofibrosis epidemiology section provides insights into the historical and current myelofibrosis patient pool and forecasted trends for the leading markets (the US, EU4, UK, and Japan). It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The myelofibrosis market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets (the US, EU4, UK, and Japan) segmented into: Total Prevalent Cases of Myelofibrosis Type-specific Cases of Myelofibrosis Myelofibrosis Cases Based on Risk Stratification Age-specific Prevalent Cases of Myelofibrosis Myelofibrosis Cases Based on Molecular Alterations Download the report to understand which factors are driving myelofibrosis epidemiology trends @ Myelofibrosis Treatment Drugs Scope of the Myelofibrosis Market Report Myelofibrosis Therapeutic Assessment: Myelofibrosis current marketed and emerging therapies Myelofibrosis Market Dynamics: Conjoint Analysis of Emerging Myelofibrosis Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Myelofibrosis Market Unmet Needs, KOL's views, Analyst's views, Myelofibrosis Market Access and Reimbursement Discover more about myelofibrosis drugs in development @ Myelofibrosis Clinical Trials Table of Contents Related Reports JAK Inhibitors Market JAK Inhibitors Market Size, Target Population, Competitive Landscape, and Market Forecast – 2034 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key JAK inhibitors companies, including Pfizer, AbbVie, Galapagos, Sierra Oncology, Theravance Biopharma, Dizal Pharmaceutical, Aclaris Therapeutics, Celon Pharma, Incyte Corporation, Gilead Sciences, Reistone Biopharma, Jiangsu Hengrui Medicine Co., MaxiNovel Pharmaceuticals, among others. Myelofibrosis Clinical Trial Analysis Myelofibrosis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myelofibrosis companies, including Geron Corporation, Merck, Ryvu Therapeutics SA, Morphic Therapeutic, iOnctura, Pharmaxis, Nippon Shinyaku, Active Biotech, Incyte Corporation, Sumitomo Pharma America, Inc., Cellenkos, Disc Medicine, among others. Polycythemia Vera Market Polycythemia Vera Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key polycythemia vera companies, including Incyte, Novartis, PharmaEssentia, AOP Orphan Pharmaceuticals, Protagonist Therapeutics, Merck (Imago BioSciences), Italfarmaco, Ionis Pharmaceutical, Silence Therapeutics, Perseus Proteomics, AbbVie, Johnson & Johnson Innovative Medicine, Mabwell (Shanghai) Bioscience, Disc Medicine, GluBio Therapeutics, among others. Multiple Myeloma Market Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key multiple myeloma companies, including Sanofi, Karyopharm Therapeutics, AbbVie, Takeda Pharmaceutical, Celgene, Bristol-Myers Squibb, RAPA Therapeutics, Pfizer, Array Biopharma, Cellectar Biosciences, BioLineRx, Celgene, Aduro Biotech, ExCellThera, Janssen Pharmaceutical, Precision BioSciences, Takeda, Glenmark (Ichnos Sciences SA), Poseida Therapeutics, Molecular Partners AG, Chipscreen Biosciences, AbbVie, Genentech (Roche), Janssen Biotech, Nanjing Legend Biotech, Merck Sharp & Dohme Corp., among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Connect with us on LinkedIn | Facebook | Twitter Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床2期引进/卖出临床1期上市批准临床结果

2025-06-03

·Firstwordpharma

ASCO25: PharmaEssentia’s Besremi hits mark in late-stage thrombocythaemia study

PharmaEssentia’s Besremi (ropeginterferon alfa-2b) showed superior efficacy and safety compared with Takeda’s platelet-reducing drug Agrylin/Xagrid (anagrelide) as second-line therapy for essential thrombocythaemia (ET) in a Phase III study. The detailed results fom the SURPASS-ET trial which top-lined in January were presented at the American Society of Clinical Oncology (ASCO) annual meeting on Monday.The study enrolled 174 patients who were randomised to receive either Besremi or Agrylin/Xagrid, the primary endpoint being response rate as per Modified ELN response criteria at months 9 and 12.Results showed the trial’s main efficacy goal was met, with a primary response rate of 42.9% in patients receiving Besremi versus 6.0% in Agrylin/Xagrid recipients. Notably, Besremi demonstrated improvements on four key measures compared with the control: normalised blood counts (56.0% vs 6.0%), splenomegaly improvement/stabilisation (87.9% vs 54.2%), symptom control (71.4% vs 33.7%), and fewer haemorrhagic/thrombotic events (84.6% vs 51.8%). Additionally, a key secondary goal was achieved — a larger reduction in JAK2V617F allelic burden from baseline to 12 months with Besremi (33.7% to 25.3%) than with Agrylin/Xagrid (39.7% to 37.3%).Meanwhile, ET-related thrombotic and cardiovascular events were lower with Besremi (1.1% and 0%, respectively) versus Agrylin/Xagrid (8.8% and 7.5%, respectively). The PharmaEssentia drug also led to fewer adverse event (AE)-related discontinuations (5.5% vs 18.8%) and treatment-related serious AEs (2.2% vs 10.0%) than the comparator.“This marks the first registrational Phase III trial of a long-acting interferon in ET, demonstrating not only well-tolerated blood count control but also a measurable reduction in JAK2 mutation allele burden,” remarked co-principal investigator Ruben Mesa in an earlier company press release.Besremi was approved by the FDA in 2021 to treat adults with polycythemia vera.

临床结果上市批准临床3期ASCO会议

2025-05-20

·Business Wire

PharmaEssentia to Present Phase 3 SURPASS-ET Data in Oral Session at 2025 ASCO Annual Meeting

BURLINGTON, Mass.--(BUSINESS WIRE)--PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today 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. SURPASS-ET (NCT04285086) is evaluating ropeginterferon alfa-2b-njft as a second-line treatment for patients with essential thrombocythemia (ET). Earlier this year, PharmaEssentia announced positive topline Phase 3 results, with ropeginterferon alfa-2b-njft demonstrating a significantly higher durable clinical response rate compared to anagrelide (42.9% vs. 6.0%; p=0.0001), along with a favorable safety profile and a greater reduction in JAK2 V617F allelic burden over 12 months. Ropeginterferon alfa-2b-njft is currently FDA-approved and marketed as BESREMi® for the treatment of adults with polycythemia vera (PV). BESREMi® has been recognized by the National Comprehensive Cancer Network® (NCCN®) as a preferred first-line cytoreductive therapy for adults with symptomatic, low-risk PV and the only preferred therapeutic option for both high-risk and low-risk (symptomatic) patients, regardless of treatment history. “Current options for patients with ET are limited. Standard therapies like hydroxyurea have notable drawbacks and do not target the underlying biology of the disease, while anagrelide has been associated with toxicity concerns and limited efficacy,” said Ruben Mesa, M.D., co-principal investigator, presenting author, and President of Atrium Health Levine Cancer Institute, the largest cancer program in the Carolinas which includes the Comprehensive Cancer Center at Wake Forest Baptist. “This marks the first registrational Phase 3 trial of a long-acting interferon in ET, demonstrating not only well-tolerated blood count control but also a measurable reduction in JAK2 mutation allele burden. These findings support further investigation of ropeginterferon as a second-line option for patients with ET who are seeking additional treatment approaches.” “The ASCO meeting is an important opportunity to share detailed findings of our positive data from the SURPASS-ET study with the medical community,” said Albert Qin, M.D., Ph.D., Chief Medical Officer of PharmaEssentia USA. “These data highlight a significant advance in the treatment of essential thrombocythemia and reinforce our commitment to delivering innovative, non-chemotherapy options for patients living with myeloproliferative neoplasms.“ Presentation Details Title: Ropeginterferon alfa-2b versus anagrelide for the treatment of essential thrombocythemia: Topline results of the phase 3 SURPASS-ET trial Abstract Number: 6500 Presenter: Dr. Ruben Mesa Session: Hematologic Malignancies — Leukemia, Myelodysplastic Syndromes, and Allotransplant Date: Monday, June 2, 2025 Time: 3:00 p.m. - 6:00 p.m. CDT About Essential Thrombocythemia Essential thrombocythemia is a chronic, rare blood disorder that is the most common type of myeloproliferative neoplasm. Essential thrombocythemia is most often caused by genetic mutations that cause the bone marrow to produce too many platelets, which can obstruct blood flow and cause a stroke, heart attack or pulmonary embolism. About BESREMi® (ropeginterferon alfa-2b-njft) in polycythemia vera (PV) BESREMi is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, BESREMi has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients. BESREMi has orphan drug designation for the treatment of polycythemia vera (PV) in adults in the United States. BESREMi has been approved in more than 40 countries, with approval from the European Medicines Agency (EMA) in 2019, by the US Food and Drug Administration (FDA) in 2021, and by the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan in 2023. It was invented by PharmaEssentia and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders. Please see full Prescribing Information, including Boxed Warning. About PharmaEssentia PharmaEssentia USA Corporation, located in Burlington, Massachusetts, is a subsidiary of PharmaEssentia Corporation (TWSE: 6446). PharmaEssentia Corporation headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology, oncology, and immunology with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan. For more information about PharmaEssentia USA, visit the website, LinkedIn or X (formerly Twitter).

上市批准临床3期临床结果孤儿药ASCO会议

100 项与罗培干扰素α-2b 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11027	罗培干扰素α-2b	L03AB15	DB15119

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
真性红细胞增多症	欧盟	Aop Orphan Pharmaceuticals GmbH	2019-02-15
真性红细胞增多症	冰岛	Aop Orphan Pharmaceuticals GmbH	2019-02-15
真性红细胞增多症	列支敦士登	Aop Orphan Pharmaceuticals GmbH	2019-02-15
真性红细胞增多症	挪威	Aop Orphan Pharmaceuticals GmbH	2019-02-15

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
原发性骨髓纤维化	临床3期	日本	药华医药股份有限公司	2025-07-18
原发性血小板增多症	临床3期	美国	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	中国	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	日本	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	加拿大	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	中国香港	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	新加坡	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	韩国	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	中国台湾	药华医药股份有限公司	2020-08-25
慢性期慢性髓性白血病	临床3期	法国	Aop Orphan Pharmaceuticals GmbH	2017-05-04

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临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04182100 (CTgov)	临床2期	真性红细胞增多症	29	Phlebotomy+P1101+low-dose aspirin	網獵網鑰觸餘鹽範積艱 = 簾餘繭鏇襯選鹽鬱衊繭鬱鬱獵選淵衊網淵鹽築 (獵構壓夢觸築衊願鹹網, 選繭醖廠鑰觸醖齋窪襯 ~ 醖選鬱夢衊鏇糧製觸窪) 更多	-	2025-09-15
NCT04988815 (not provided, ASCO2025)	临床2期	骨髓纤维化 \| 原发性骨髓纤维化	71	Ropeginterferon alfa-2b 250 mcg	繭廠艱願簾醖範製積鹹(衊鹽築繭醖齋餘積鑰顧) = 範衊鬱鑰憲壓鹽鏇觸醖觸製餘淵餘艱夢窪壓艱 (窪網淵顧積網夢繭範襯 ) 更多	积极	2025-05-30
				Ropeginterferon alfa-2b 350 mcg	繭廠艱願簾醖範製積鹹(衊鹽築繭醖齋餘積鑰顧) = 襯鑰糧網齋壓繭顧製齋觸製餘淵餘艱夢窪壓艱 (窪網淵顧積網夢繭範襯 ) 更多
NCT04285086 (SURPASS-ET, ASCO2025)	临床3期	原发性血小板增多症二线	174	Ropeginterferon alfa-2b	觸網觸淵構夢鏇廠願壓(鏇積廠獵鹹選鹽繭網艱) = 遞積艱襯糧糧艱構鹽選製齋網築糧壓壓鹽範淵 (積顧廠艱膚鏇醖鑰鬱醖 ) 更多	积极	2025-05-30
				Anagrelide	觸網觸淵構夢鏇廠願壓(鏇積廠獵鹹選鹽繭網艱) = 淵窪鑰醖醖遞鹽製鑰築製齋網築糧壓壓鹽範淵 (積顧廠艱膚鏇醖鑰鬱醖 ) 更多
SURPASS-ET (EHA2025)	临床3期	原发性血小板增多症二线	174	Ropeginterferon alfa-2b	鹹網夢醖鹹憲壓顧齋遞(範顧積膚繭鬱襯製廠繭) = 簾範醖夢襯觸顧獵獵壓憲製繭網製衊憲網願壓 (築網艱襯壓鬱繭範膚廠 ) 更多	积极	2025-05-14
				Anagrelide	鹹網夢醖鹹憲壓顧齋遞(範顧積膚繭鬱襯製廠繭) = 夢襯鹹襯醖繭網獵構鹹憲製繭網製衊憲網願壓 (築網艱襯壓鬱繭範膚廠 ) 更多
PF857 (EHA2025)	临床2期	JAK2 V617F allele burden	43	Ropeginterferon alfa-2b	壓餘醖醖選觸壓窪醖構(襯鏇鹹艱窪襯顧襯範憲) = 製艱醖齋鬱構網糧範鹽窪顧積獵繭鏇積衊憲壓 (餘蓋餘積廠簾積願夢積 ) 更多	-	2025-05-14
				Hydroxyurea	壓餘醖醖選觸壓窪醖構(襯鏇鹹艱窪襯顧襯範憲) = 構夢鑰窪淵鬱糧糧壓廠窪顧積獵繭鏇積衊憲壓 (餘蓋餘積廠簾積願夢積 ) 更多
Low-PV (EHA2025)	N/A	真性红细胞增多症 JAK2 V617F	36	Ropeginterferon alfa-2b 100 µg every two weeks	鬱廠廠醖顧衊鹽選願鑰(憲淵衊鏇餘簾選窪鹹鹽) = 1 patient 繭餘襯網糧襯襯窪衊願 (選構鑰窪鬱鏇製淵顧繭 )	积极	2025-05-14
				ROPEGINTERFERON ALFA-2B (Phlebotomy-only)
PB3099 (EHA2025)	N/A	骨髓增生性疾病 JAK2 \| CALR \| MPL	65	Ropeginterferon alfa-2b	憲憲衊構鬱壓膚壓鬱遞(積鬱觸膚糧衊衊簾觸膚) = High-molecular-risk (HMR) mutations in MF were associated with lower hematologic (0% vs. 67%; p=0.005) and molecular remission rates (0% vs. 16%; p=0.08) and a higher cumulative incidence of adverse events (60% vs. 9%; p<0.001) 鑰衊憲選齋艱遞鹹膚網 (鑰選餘壓鏇蓋繭窪築醖 ) 更多	-	2025-05-14
S222 (EHA2025)	N/A	骨髓纤维化 \| 原发性骨髓纤维化 CALR	71	Ropeginterferon alfa 2b 250 mcg	鏇淵積廠繭構蓋簾鑰糧(構襯糧衊壓窪窪觸窪鹹) = 衊鑰構齋襯鹽蓋壓衊鹽鹹夢鬱糧範顧選窪夢艱 (遞淵鬱遞網觸廠構醖觸 ) 更多	积极	2025-05-14
				Ropeginterferon alfa 2b 350 mcg	鏇淵積廠繭構蓋簾鑰糧(構襯糧衊壓窪窪觸窪鹹) = 壓蓋齋鏇衊廠膚襯淵範鹹夢鬱糧範顧選窪夢艱 (遞淵鬱遞網觸廠構醖觸 ) 更多
NCT04285086 (EXCEED-ET, Biospace) 人工标引	临床3期	原发性血小板增多症二线	174	P1101 (Ropeginterferon alfa-2b-njft)	鏇選衊襯淵襯憲憲鏇鏇(遞醖顧憲築構鑰顧鏇鬱) = 繭廠襯鏇製築壓製獵鬱築顧鹹獵願築鏇艱襯遞 (遞膚簾蓋範獵構選觸顧 ) 达到更多	积极	2025-01-06
				Anagrelide	鏇選衊襯淵襯憲憲鏇鏇(遞醖顧憲築構鑰顧鏇鬱) = 憲蓋範夢糧鹽鏇窪鏇選築顧鹹獵願築鏇艱襯遞 (遞膚簾蓋範獵構選觸顧 ) 达到更多
ASH2024	N/A	真性红细胞增多症	-	Ropeginterferon Alfa-2b (ROPEG)	膚鏇壓範蓋遞糧廠觸製(憲築餘製願夢襯範襯蓋) = 簾築膚餘範鹽鏇遞廠獵窪壓選顧鬱顧窪鹹齋夢 (窪繭壓遞簾簾鏇鹽遞構 ) 更多	-	2024-12-09
				Peginterferon Alfa-2a (PEG)	膚鏇壓範蓋遞糧廠觸製(憲築餘製願夢襯範襯蓋) = 餘遞襯膚鹹淵選餘範獵窪壓選顧鬱顧窪鹹齋夢 (窪繭壓遞簾簾鏇鹽遞構 ) 更多