Ropeginterferon alfa-2b-NJFT

编者按 第66届美国血液学会（ASH）年会将于2024年12月7～10日在美国圣迭戈（San Diego）盛大召开。作为全球血液学领域规模最大、涵盖最全面的国际学术盛会之一，每年吸引着成千上万名来自世界各地的专家学者，分享最前沿的血液学进展和突破性临床数据。当地时间11月25日，大会官网公布了本次入选的最新重磅摘要（Late-Breaking Abstracts，LBA）共6项，将由研究者于当地时间2024年12月10日上午7:30至9:00，在会议现场Hall B (San Diego Convention Center)进行报告。本刊第一时间整理这6项LBA的核心要点，供大家提前阅览。 01 LBA-1 标题：Tafasitamab Plus Lenalidomide and Rituximab for Relapsed or Refractory Follicular Lymphoma: Results from a Phase 3 Study (inMIND) 中文标题：坦昔妥单抗联合来那度胺和利妥昔单抗治疗复发或难治性滤泡性淋巴瘤：III期研究（inMIND）结果 汇报人：Laurie H. Sehn（BC癌症中心淋巴瘤中心，加拿大） 研究结论 在来那度胺和利妥昔单抗的基础上加用坦昔妥单抗，显著且有临床意义地改善了无进展生存期（PFS），使复发/难治性滤泡性淋巴瘤（R/R FL）患者的疾病进展、复发或死亡风险降低了57%。尽管总生存期（OS）数据尚不成熟，但观察到了一种有利于塔坦昔妥单抗的趋势。其安全性特征可控，且与预期毒性一致。本研究首次验证了将两种单克隆抗体（抗CD19与抗CD20）联合用于淋巴瘤治疗的有效性。坦昔妥单抗+来那度胺+利妥昔单抗既可在社区环境中使用，也可在学术环境中使用，是R/R FL患者潜在的新标准治疗选择。 02 LBA-2 标题：Identification of Hepatic-like Erythropoietin with Enhanced Activity As a New Cause of Hereditary and Acquired Erythrocytosis 中文标题：识别具有增强活性的肝样促红细胞生成素作为遗传性和获得性红细胞增多症的新原因 汇报人：Betty Gardie[巴黎高等研究实践学院（EPHE），法国] 研究结论 本研究报告了一种名为肝样促红细胞生成素增多症（HEP）的新型继发性红细胞增多症，其特征是产生具有非典型糖基化模式和活性增强的肝样促红细胞生成素（EPO）。这种新型EPO在所有遗传性EPO突变型红细胞增多症患者、与肝病相关的红细胞增多症病例以及新生儿中均有观察到。有趣的是，新生儿体内从更活跃的肝型EPO向肾型EPO的转变，可能解释了生命最初几周内血红蛋白水平显著下降的典型现象。因此，在进行特发性和遗传性红细胞增多症的诊断时，将定量和定性EPO筛查作为生物学检查的一部分是至关重要的。 03 LBA-3 标题：Extended Treatment of Venous Thromboembolism with Reduced- Vs Full-Dose Direct Oral Anticoagulants in Patients at High Risk of Recurrence 中文标题：比较减剂量与全剂量直接口服抗凝药延长治疗高风险复发静脉血栓栓塞症 Francis Couturaud（布雷斯特大学、FCRIN INNOVTE网络，法国） 研究结论 在需要延长抗凝治疗的静脉血栓栓塞症患者中，尚无法证明使用减剂量与全剂量直接抗凝药物在预防静脉血栓栓塞症复发方面的非劣效性。在减剂量组，临床相关出血以及静脉血栓栓塞症复发或临床相关出血的复合终点发生率均低于全剂量组，且这一优势并未因死亡或动脉血栓栓塞事件风险增加而抵消。 04 LBA-4 标题：Andean Enriched NFKB1 Haplotype Reduces Inflammation and Improves Response to Ropeginterferon Alfa-2b in Polycythemia Vera (PV) and Essential Thrombocythemia (ET) 中文标题：富含安第斯特征的NFKB1单倍型可降低真性红细胞增多症（PV）和原发性血小板增多症（ET）患者的炎症水平，并提高对罗培干扰素α-2b的应答率 汇报人：Jihyun Song（亨斯迈癌症研究所，美国） 研究结论 本研究显示，与NFKB1野生型等位基因相比，富含艾马拉族特征的NFKB1 rs230511单倍型可降低真性红细胞增多症（PV）和原发性血小板增多症（ET）患者的炎症、促血栓形成及缺氧诱导因子（HIF）活性。此外，NFKB1 rs230511单倍型还能调节PV和ET患者对罗培干扰素的应答。携带NFKB1 rs230511 T等位基因的患者，其炎症和HIF靶向基因的表达降低，这可能潜在地减少PV和ET患者的炎症和血栓形成风险。携带C/T基因型的患者更有可能实现完全血液学缓解（CHR），这表明这种NFKB1单倍型可能影响罗培干扰素的治疗效果，并可作为评估PV和ET患者治疗应答的潜在生物标志物。这些新的见解对我们理解炎症和促血栓形成过程具有重要意义，并可能为PV和ET的新型疗法提供线索。 05 LBA-5 标题：Outcome of Cerebral Vasculopathy and Cognitive Performances 10 Years Post-Enrollment in the Drepagreffe Trial Comparing Allogeneic Stem Cell Transplantation to Standard-Care in Children with Sickle Cell Anemia and History of Abnormal Cerebral Velocities 中文标题：Drepagreffe试验中接受异体干细胞移植与标准治疗的镰状细胞贫血症伴异常脑血流速度病史患儿的10年脑血管病变情况及认知功能结局比较 汇报人：Françoise Bernaudin（巴黎东部大学，法国） 研究结论 这项首个前瞻性比较试验表明，异体干细胞移植（alloSCT）与标准治疗（SoC）之间的差异在10年后发生了显著变化，其中异体干细胞移植在无症状性脑梗死（SCI)方面提供了更好的一级预防效果，同时改善了患者的社会生活质量、工作记忆和处理速度。 06 LBA-6 标题：Lack of Benefit of Autologous Hematopoietic Cell Transplantation (auto-HCT) in Mantle Cell Lymphoma (MCL) Patients (pts) in First Complete Remission (CR) with Undetectable Minimal Residual Disease (uMRD): Initial Report from the ECOG-ACRIN EA4151 Phase 3 Randomized Trial 中文标题：自体造血干细胞移植（auto-HCT）对首次完全缓解（CR）且无法检测到微小残留病（uMRD）的套细胞淋巴瘤（MCL）患者（pts）无益处：ECOG-ACRIN EA4151 III期随机试验的初步报告 汇报人：Timothy S. Fenske（威斯康星医学院，美国） 研究结论 本次中期分析显示，在高效诱导和维持治疗方案时代，首次完全缓解（CR）且无法检测到微小残留病（uMRD）的套细胞淋巴瘤（MCL）患者并未从巩固性自体造血干细胞移植（auto-HCT）中获益。而诱导治疗后MRD仍为阳性的患者可能从auto-HCT中获益。更长时间的随访对于证实这些发现至关重要。 （来源：《肿瘤瞭望–血液时讯》编辑部） 声 明 凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。

BURLINGTON, Mass.--( BUSINESS WIRE )--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 a slate of nine new abstracts exploring the use of ropeginterferon alfa-2b-njft, known in market as BESREMi ® , will be presented during the 66 th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego, CA. Presentations at ASH include a quality-of-life evaluation for patients with polycythemia vera (PV) or essential thrombocythemia (ET) and treated with ropeginterferon alfa-2b compared to other best available therapies; identifying the efficacy and safety of ropeginterferon alfa-2b in low-risk PV patients and the potential of an AI-powered approach to discovering potential new indications for ropeginterferon alfa-2b. “With new clinical findings, AI-powered results and real-world analyses, we are excited to continue to advance our knowledge on the potential of ropeginterferon alfa-2b in treating chronic and life-threatening myeloproliferative neoplasms,” said Ko-Chung Lin, Ph.D., Founder and CEO of PharmaEssentia. “PharmaEssentia maintains its commitment to educating providers and patients to make informed decisions to improve care and outcomes for people living with myeloproliferative neoplasms such as PV and ET.” Ropeginterferon Alfa-2b presentations at ASH include: Ropeginterferon Alfa-2b Induces Apoptosis and Differentiation of Leukemia Stem Cells and Separates GVL Effects from GVHD after Allogeneic Hematopoietic Cell Transplantation Oral Presentation Abstract #902: Monday, December 9, 2024, 2:45-4:15 PM PT Quality of Life Evaluation for Patients on Ropeginterferon Alpha-2b Versus Other Best Available Therapies Poster Presentation Abstract #1791: Saturday, December 7, 2024, 5:30-7:30 PM PT Ropeginterferon in Low-risk Patients with Polycythemia Vera Poster Presentation Abstract #1799: Saturday, December 7, 2024, 5:30-7:30 PM PT HOPE-PMF: 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 Poster Presentation Abstract #3191.1: Sunday, December 8, 2024, 6:00-8:00 PM PT Predicting Molecular Response through Half Reduction of Neutrophil-to-Lymphocyte ratio (NLR) in Polycythemia Vera Treatment with Ropeginterferon Poster Presentation Abstract #3170: Sunday, December 8, 2024, 6:00-8:00 PM PT Remarkable molecular response in Chinese PV patients treated with Ropeginterferon alfa-2b Poster Presentation Abstract #4562: Monday, December 9, 2024, 6:00-8:00 PM PT Ropeginterferon Alfa-2b (ROPEG) and Peginterferon Alfa-2a (PEG) at Low Dose with Response-Based Titration (LDRT) Have Comparable Efficacy and Tolerability in Polycythemia Vera (PV) Poster Presentation Abstract #4568: Monday, December 9, 2024, 6:00-8:00 PM PT Accelerating Drug Discovery: AI-Powered Indication Exploration for Ropeginterferon Alfa-2b Poster Presentation Abstract #4982: Monday, December 9, 2024, 6:00-8:00 PM PT Treatment Patterns of Ropeginterferon Alfa-2b-njft in the real-world setting Poster Presentation Abstract #5184: Monday, December 9, 2024, 6:00-8:00 PM PT About PharmaEssentia PharmaEssentia (TWSE: 6446), 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 Twitter . About Polycythemia Vera (PV) Polycythemia vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells, and platelets. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2. 1 About Essential Thrombocythemia (ET) Essential thrombocythemia (ET) is a myeloproliferative neoplasm (MPN) characterized by an overproduction of platelets in the blood that results from a genetic mutation; data indicates a JAK2 gene mutation is present in approximately half of diagnosed patients. ET is estimated to affect up to 57 per 100,000 people in the U.S. The disease is most commonly diagnosed through routine blood work and is most common in people over the age of 50, with women 1.5 more times more likely to be diagnosed than men. As a chronic, progressive disease, ET requires regular monitoring and appropriate treatment. Over time, the disease may progress into more deadly conditions such as myelofibrosis or acute leukemia. 2,3 About BESREMi ® (ropeginterferon alfa-2b-njft) 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. Indication BESREMi is indicated for the treatment of adults with polycythemia vera. Important Safety Information WARNING: RISK OF SERIOUS DISORDERS Interferon alfa products may cause or aggravate fatal or life-threatening neuropsychiatric, autoimmune, ischemic, and infectious disorders. Patients should be monitored closely with periodic clinical and laboratory evaluations. Therapy should be withdrawn in patients with persistently severe or worsening signs or symptoms of these conditions. In many, but not all cases, these disorders resolve after stopping therapy. CONTRAINDICATIONS Existence of, or history of severe psychiatric disorders, particularly severe depression, suicidal ideation, or suicide attempt Hypersensitivity to interferons including interferon alfa-2b or any of the inactive ingredients of BESREMi. Moderate (Child-Pugh B) or severe (Child-Pugh C) hepatic impairment History or presence of active serious or untreated autoimmune disease Immunosuppressed transplant recipients WARNINGS AND PRECAUTIONS Depression and Suicide: Life-threatening or fatal neuropsychiatric reactions have occurred in patients receiving interferon alfa-2b products, including BESREMi. These reactions may occur in patients with and without previous psychiatric illness. Other central nervous system effects, including suicidal ideation, attempted suicide, aggression, bipolar disorder, mania and confusion have been observed with other interferon alfa products. Closely monitor patients for any symptoms of psychiatric disorders and consider psychiatric consultation and treatment if such symptoms emerge. If psychiatric symptoms worsen, it is recommended to discontinue BESREMi therapy. Endocrine Toxicity: These toxicities may include worsening hypothyroidism and hyperthyroidism. Do not use BESREMi in patients with active serious or untreated endocrine disorders associated with autoimmune disease. Evaluate thyroid function in patients who develop symptoms suggestive of thyroid disease during BESREMi therapy. Discontinue BESREMi in patients who develop endocrine disorders that cannot be adequately managed during treatment with BESREMi. Cardiovascular Toxicity: Toxicities may include cardiomyopathy, myocardial infarction, atrial fibrillation and coronary artery ischemia. Patients with a history of cardiovascular disorders should be closely monitored for cardiovascular toxicity during BESREMi therapy. Avoid use of BESREMi in patients with severe or unstable cardiovascular disease, (e.g., uncontrolled hypertension, congestive heart failure (≥ NYHA class 2), serious cardiac arrhythmia, significant coronary artery stenosis, unstable angina) or recent stroke or myocardial infarction. Decreased Peripheral Blood Counts: These toxicities may include thrombocytopenia (increasing the risk of bleeding), anemia, and leukopenia (increasing the risk of infection). Monitor complete blood counts at baseline, during titration and every 3-6 months during the maintenance phase. Monitor patients for signs and symptoms of infection or bleeding. Hypersensitivity Reactions: Toxicities may include serious, acute hypersensitivity reactions (e.g., urticaria, angioedema, bronchoconstriction, anaphylaxis). If such reactions occur, discontinue BESREMi and institute appropriate medical therapy immediately. Transient rashes may not necessitate interruption of treatment. Pancreatitis: Pancreatitis has occurred in 2.2% of patients receiving BESREMi. Symptoms may include nausea, vomiting, upper abdominal pain, bloating, and fever. Patients may experience elevated lipase, amylase, white blood cell count, or altered renal/hepatic function. Interrupt BESREMi treatment in patients with possible pancreatitis and evaluate promptly. Consider discontinuation of BESREMi in patients with confirmed pancreatitis. Colitis: Fatal and serious ulcerative or hemorrhagic/ischemic colitis have occurred in patients receiving interferon alfa products, some cases starting as early as 12 weeks after start of treatment. Symptoms may include abdominal pain, bloody diarrhea, and fever. Discontinue BESREMi in patients who develop these signs or symptoms. Colitis may resolve within 1 to 3 weeks of stopping treatment. Pulmonary Toxicity: Pulmonary toxicity may manifest as dyspnea, pulmonary infiltrates, pneumonia, bronchiolitis obliterans, interstitial pneumonitis, pulmonary hypertension, and sarcoidosis. Some events have resulted in respiratory failure or death. Discontinue BESREMi in patients who develop pulmonary infiltrates or pulmonary function impairment. Ophthalmologic Toxicity: These toxicities may include severe eye disorders such as retinopathy, retinal hemorrhage, retinal exudates, retinal detachment and retinal artery or vein occlusion which may result in blindness. During BESREMi therapy, 23% of patients were identified with an eye disorder. Eyes disorders ≥5% included cataract (6%) and dry eye (5%). Advise patients to have eye examinations before and during BESREMi therapy, specifically in those patients with a retinopathy-associated disease such as diabetes mellitus or hypertension. Evaluate eye symptoms promptly. Discontinue BESREMi in patients who develop new or worsening eye disorders. Hyperlipidemia: Elevated triglycerides may result in pancreatitis. Monitor serum triglycerides before BESREMi treatment and intermittently during therapy and manage when elevated. Consider discontinuation of BESREMi in patients with persistently, markedly elevated triglycerides. Hepatotoxicity: These toxicities may include increases in serum alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT) and bilirubin. Liver enzyme elevations have also been reported in patients after long-term BESREMi therapy. Monitor liver enzymes and hepatic function at baseline and during BESREMi treatment. Discontinue BESREMi in patients who develop evidence of hepatic decompensation (characterized by jaundice, ascites, hepatic encephalopathy, hepatorenal syndrome or variceal hemorrhage) during treatment. Renal Toxicity: Monitor serum creatinine at baseline and during therapy. Avoid use of BESREMi in patients with eGFR <30 mL/min. Discontinue BESREMi if severe renal impairment develops during treatment. Dental and Periodontal Toxicity: These toxicities may include dental and periodontal disorders, which may lead to loss of teeth. In addition, dry mouth could have a damaging effect on teeth and mucous membranes of the mouth during long-term treatment with BESREMi. Patients should have good oral hygiene and regular dental examinations. Dermatologic Toxicity: These toxicities have included skin rash, pruritus, alopecia, erythema, psoriasis, xeroderma, dermatitis acneiform, hyperkeratosis, and hyperhidrosis. Consider discontinuation of BESREMi if clinically significant dermatologic toxicity occurs. Driving and Operating Machinery: BESREMi may impact the ability to drive and use machinery. Patients should not drive or use heavy machinery until they know how BESREMi affects their abilities. Patients who experience dizziness, somnolence or hallucination during BESREMi therapy should avoid driving or using machinery. Embryo-Fetal Toxicity: Based on the mechanism of action, BESREMi can cause fetal harm when administered to a pregnant woman. Pregnancy testing is recommended in females of reproductive potential prior to treatment with BESREMi. Advise females of reproductive potential to use an effective method of contraception during treatment with BESREMi and for at least 8 weeks after the final dose. ADVERSE REACTIONS The most common adverse reactions reported in > 40% of patients in the PEGINVERA study (n=51) were influenza-like illness, arthralgia, fatigue, pruritis, nasopharyngitis, and musculoskeletal pain. In the pooled safety population (n=178), the most common adverse reactions greater than 10%, were liver enzyme elevations (20%), leukopenia (20%), thrombocytopenia (19%), arthralgia (13%), fatigue (12%), myalgia (11%), and influenza-like illness (11%). DRUG INTERACTIONS Patients on BESREMi who are receiving concomitant drugs which are CYP450 substrates with a narrow therapeutic index should be monitored to inform the need for dosage modification for these concomitant drugs. Avoid use with myelosuppressive agents and monitor patients receiving the combination for effects of excessive myelosuppression. Avoid use with narcotics, hypnotics or sedatives and monitor patients receiving the combination for effects of excessive CNS toxicity. USE IN SPECIFIC POPULATIONS Pregnancy: Based on mechanism of action and the role of interferon alfa in pregnancy and fetal development, BESREMi may cause fetal harm and should be assumed to have abortifacient potential when administered to a pregnant woman. There are adverse effects on maternal and fetal outcomes associated with polycythemia vera in pregnancy. Advise pregnant women of the potential risk to a fetus. Lactation: There are no data on the presence of BESREMi in human or animal milk, the effects on the breastfed child, or the effects on milk production. Because of the potential for serious adverse reactions in breastfed children from BESREMi, advise women not to breastfeed during treatment and for 8 weeks after the final dose. Females of Reproductive Potential: BESREMi may cause embryo-fetal harm when administered to a pregnant woman. Pregnancy testing prior to BESREMi treatment is recommended for females of reproductive potential. Advise female patients of reproductive potential to use effective contraception during treatment with BESREMi and for at least 8 weeks after the final dose. Pediatric Use: Safety and effectiveness in pediatric patients have not been established. Geriatric Use: In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function and of concomitant disease or other therapy. Forward Looking Statement This press release may contain forward looking statements, including statements regarding the clinical benefits to be derived from ropeginterferon alfa-2b, the commercial opportunity and competitive positioning, new indications or labeling for ropeginterferon alfa-2b, and business prospects for ropeginterferon alfa-2b. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 and similar legislation and regulations under Taiwanese law. These forward-looking statements are based on management expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward looking statements as a result of various factors. These factors include whether BESREMi is successfully commercialized and adopted by physicians and patients, the extent to which reimbursement is available for BESREMi, and the ability to receive FDA and other regulatory approvals for additional indications for BESREMi. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. © 2024 PharmaEssentia Corporation. All rights reserved. PharmaEssentia, the PharmaEssentia logo, and BESREMi are trademarks or registered trademarks of PharmaEssentia Corporation. 1 Cerquozzi S, Tefferi A. Blast transformation and fibrotic progression in polycythemia vera and essential thrombocythemia: a literature review of incidence and risk factors. Blood Cancer J . 2015;5, e366; DOI:10.1038/bcj.2015.95 2 Mehta J, Wang H, Iqbal SU, Mesa R. Epidemiology of myeloproliferative neoplasms in the United States. Leuk Lymphoma. 2014 Mar;55(3):595-600 3 “What is Essential Thrombocythemia?” MPN Research Foundation. 2020. Available at: http://www.mpnresearchfoundation.org/Essential-Thrombocythemia