Ropeginterferon alfa-2b-NJFT

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 be presenting at the 67th American Society of Hematology (ASH) Annual Meeting, taking place December 6-9 in Orlando, Florida. The Company’s presentations will share new data exploring the clinical and biologic effects of BESREMI® ropeginterferon alfa-2b-njft in polycythemia vera (PV). “The new data presented at ASH build on the growing body of evidence showing deep and durable responses with ropeginterferon alfa-2b in PV, including molecular and transcriptomic findings that offer insight into potential mechanisms of disease modification,” said Ko-Chung Lin, Ph.D., Founder and CEO of PharmaEssentia. “These results expand the evidence supporting the potential of ropeginterferon alfa-2b-njft to transform outcomes for patients with PV.” Presentation Details Long-term efficacy and safety of ropeginterferon alfa-2b under its HIDAT regimen for the treatment of polycythemia vera Abstract: abs25-10438 (2039) Session Type: Poster Presenter: Shanshan Suo Room: OCCC - West Halls B3-B4 Date: Saturday, December 6, 5:30 PM - 7:30 PM EST Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster I Transcriptomic signatures associated with deep molecular response to ropeginterferon alfa-2b in polycythemia vera Abstract: abs25-10525 (2040) Session Type: Poster Presenter: Seug Yun Yoon Room: OCCC - West Halls B3-B4 Date: Saturday, December 6, 5:30 PM - 7:30 PM EST Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster I Rapid JAK2 V617F decline predicts response durability: kinetics of JAK2 V617F in ropeginterferon alfa-2b treated polycythemia vera Abstract: abs25-13127 (3819) Session Type: Poster Presenter: Sung-Eun Lee Room: OCCC - West Halls B3-B4 Date: Sunday, December 7, 6:00 PM - 8:00 PM EST Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster II Clinical significance of earlier CHR achievement and long-term CHR maintenance in polycythemia vera patients treated with ropeginterferon alfa-2b Abstract: abs25-13190 (5598) Session Type: Poster Presenter: Sung-Eun Lee Room: OCCC - West Halls B3-B4 Date: Monday, December 8, 6:00 PM - 8:00 PM EST Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster III About Polycythemia Vera Polycythemia vera, or PV, is a rare and chronic blood cancer. It’s part of a group of blood cancers called myeloproliferative neoplasms, or MPNs. PV starts in the bone marrow. Normally the body keeps all types of blood cells in a healthy balance, but with PV, the body produces too many red blood cells and may create excess white blood cells and platelets. About BESREMi® (ropeginterferon alfa-2b-njft) BESREMi® is the only FDA-approved treatment indicated for adults with polycythemia vera (PV) that targets the source of the disease. BESREMi® is not chemotherapy, it's an innovative immunotherapy. Ropeginterferon alfa-2b-njft is a preferred first-line cytoreductive therapy option for both low-risk (symptomatic) and high-risk PV in the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®). National Comprehensive Cancer Network® (NCCN®)–preferred interventions are based on efficacy, safety, and evidence, and when appropriate, affordability. BESREMi® holds orphan drug designation in the United States for the treatment of polycythemia vera (PV) in adults. It has received regulatory approval in over 40 countries, including from the European Medicines Agency (2019), the U.S. Food and Drug Administration (2021), and the Pharmaceuticals and Medical Devices Agency in Japan (2023). The product was developed by PharmaEssentia and is manufactured at the company’s facility in Taichung. PharmaEssentia retains full global intellectual property rights across all indications. Indication BESREMi® is indicated for the treatment of adults with polycythemia vera. Important Safety Information Boxed 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 depression, suicidal ideation, or suicide attempt Hypersensitivity to interferons or any inactive ingredients Moderate or severe hepatic impairment History or presence of active serious or untreated autoimmune disease History of transplantation and receiving immunosuppressant agents Warnings and Precautions Depression and Suicide: Closely monitor patients for any symptoms of psychiatric disorders and, if needed, consider psychiatric consultation and treatment or dosage modifications as listed in the full prescribing information. Endocrine Toxicity: 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: 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 or recent stroke or myocardial infarction. Decreased Peripheral Blood Counts: Monitor complete blood counts at baseline, during titration, and every 3-6 months during the maintenance phase. Pancreatitis/Colitis/Pulmonary Toxicity: Interrupt BESREMi® treatment in patients with possible pancreatitis, colitis, or pulmonary toxicity and evaluate promptly. Consider discontinuation of BESREMi® in patients with confirmed pancreatitis or who show signs or symptoms of serious ulcerative or hemorrhagic colitis, or who develop pulmonary infiltrates or pulmonary function impairment. Ophthalmologic Toxicity: Advise patients to have eye examinations before and during treatment. Discontinue BESREMi® in patients who develop new or worsening eye disorders. Hyperlipidemia/Hepatotoxicity/Renal Toxicity: Monitor serum triglycerides, liver enzymes, hepatic function, and serum creatinine at baseline and during therapy. Avoid use of BESREMi® in patients with eGFR <30 mL/min. Discontinue BESREMi® in patients with persistently marked elevated triglycerides, evidence of hepatic decompensation, or if renal impairment develops during treatment. Dental and Periodontal Toxicity: Patients should have good oral hygiene and regular dental examinations. Dermatologic Toxicity: 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. Obtain a pregnancy test in females of reproductive potential prior to initiating 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. Advise women not to breastfeed during treatment and for 8 weeks after the final dose. Adverse Reactions The most common adverse reactions reported in >40% of patients were influenza-like illness, arthralgia, fatigue, pruritus, nasopharyngitis, and musculoskeletal pain. 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. Avoid use with myelosuppressive agents, narcotics, hypnotics, or sedatives, and monitor patients receiving the combination for effects of excessive CNS toxicity. 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).

第一章开场白：当医学突破告别 “一刀切”，生物治疗为何成新希望？ 在人类与疾病抗争的漫长历史中，手术、化疗、放疗曾是对抗重症的 “三驾马车”，但 “杀敌一千，自损八百” 的局限，让无数患者在治疗中承受着身心双重煎熬。如今，随着生命科学的飞速发展，一种名为 “生物治疗” 的新型疗法正悄然改写医学版图 —— 它不再依赖外力强行清除病灶，而是唤醒人体自身的免疫细胞，或是借助基因工程改造的 “生物武器”，精准狙击病魔。据《自然・医学》2024 年全球生物治疗报告显示，过去五年全球生物治疗市场规模年均增速达 18.7%，2023 年更是突破 4800 亿美元，越来越多的患者正通过这种 “智慧疗法” 重获新生。今天，我们就一起揭开生物治疗的神秘面纱，看看它如何让医学从 “对抗” 走向 “协同”。第二章免疫治疗：唤醒体内 “健康卫士”，让癌细胞无处遁形 提及生物治疗，免疫治疗无疑是最受关注的领域之一。我们的免疫系统本就像一支训练有素的 “军队”，免疫细胞便是其中的 “士兵”，负责识别并清除癌细胞等 “入侵者”。但狡猾的癌细胞会通过表达 PD - L1 蛋白等方式，与免疫细胞表面的 PD - 1 受体结合，就像给免疫细胞戴上 “枷锁”，使其失去战斗力。而免疫检查点抑制剂的出现，打破了这一僵局。以帕博利珠单抗为例，《新英格兰医学杂志》2023 年发表的 Ⅲ 期临床试验数据显示，在晚期非小细胞肺癌患者中，接受帕博利珠单抗联合化疗治疗的患者，中位生存期达 24.8 个月，较单纯化疗组延长了 8.3 个月，5 年生存率更是提升至 31.9%，远超传统疗法的 15.6%。除了免疫检查点抑制剂，CAR - T 细胞治疗也展现出惊人潜力。2024 年美国临床肿瘤学会（ASCO）年会上公布的数据显示，针对复发难治性弥漫大 B 细胞淋巴瘤患者，阿基仑赛注射液治疗后的客观缓解率达82.5%，完全缓解率为 58.4%，部分患者甚至实现了长期无瘤生存，真正实现了 “细胞抗癌” 的突破。第三章基因治疗：修复 “生命密码”，攻克遗传病与罕见病 如果说免疫治疗是 “唤醒士兵”，那么基因治疗就是直接 “修复生命蓝图”。当人体基因出现缺陷或突变时，就可能引发遗传病、罕见病等疑难病症。基因治疗通过病毒载体、基因编辑等技术，将正常基因导入患者体内，或修正突变基因，从根源上治愈疾病。以脊髓性肌萎缩症（SMA）为例，这是一种因 SMN1 基因缺失导致的罕见遗传病，患儿常因肌肉无力失去行走能力，甚至危及生命。2023 年《柳叶刀・神经病学》发表的长期随访研究显示，接受onasemnogene abeparvovec 基因治疗的 SMA 患儿，在治疗后 5 年，92% 的患儿能够独立行走，83% 的患儿无需依赖呼吸支持，而未接受治疗的传统组患儿，仅12% 能实现独立行走。在基因编辑领域，CRISPR 技术更是让精准治疗成为可能。2024 年《科学》杂志报道，美国研究团队利用 CRISPR - Cas9 技术，成功修正了镰状细胞贫血患者造血干细胞中的基因突变，在接受治疗的 25 名患者中，23 名患者的血红蛋白水平恢复正常，随访 18 个月无严重不良反应，这一成果为遗传病治疗开辟了全新路径。第四章细胞因子与抗体药物：精准靶向病灶，降低治疗副作用 除了免疫治疗和基因治疗，细胞因子疗法与单克隆抗体药物也是生物治疗的重要组成部分。细胞因子是免疫系统分泌的 “信号分子”，能调节免疫细胞活性，增强免疫应答。比如干扰素 α - 2b，在慢性乙型肝炎治疗中，《中华肝脏病杂志》2023 年发布的多中心研究显示，使用干扰素 α - 2b 治疗 48 周的患者，乙肝表面抗原清除率达 12.3%，较核苷类似物治疗组提升了 7.8 个百分点，且停药后持续应答率更高，有效降低了肝硬化和肝癌的发生风险。单克隆抗体药物则像 “精准导弹”，能特异性结合病灶部位的抗原，精准杀伤病变细胞。以曲妥珠单抗为例，它专门针对 HER2 阳性乳腺癌细胞，《临床肿瘤学杂志》2024 年的数据显示，HER2 阳性早期乳腺癌患者接受曲妥珠单抗辅助治疗后，复发风险降低 34%，死亡风险降低 26%，10 年无病生存率达 73.7%，显著改善了患者预后。与传统化疗药物相比，这些生物制剂因靶向性强，对正常细胞损伤小，副作用发生率大幅降低，患者生活质量得到明显提升。第五章结尾：生物治疗的未来，离我们并不遥远 从免疫治疗让癌症患者长期生存成为可能，到基因治疗为遗传病患者点亮希望，再到抗体药物精准降低治疗副作用，生物治疗正以惊人的速度改变着疾病治疗的格局。当然，它并非完美无缺，部分疗法存在价格较高、适应症有限等问题，但随着技术的不断突破和医保政策的逐步覆盖，这些障碍正在被一一打破。据《全球生物治疗发展白皮书（2024）》预测，到 2030 年，全球将有超过 300 种生物治疗产品上市，覆盖癌症、遗传病、自身免疫病等多个领域，惠及数十亿患者。生物治疗的时代已经来临，它不仅是医学科技的奇迹，更是人类对抗疾病的新希望。如果这篇文章让你对生物治疗有了新的认识，不妨分享给身边的人，让更多人了解这份 “健康守护力量”，共同期待医学带来的更多惊喜。