Zavabresib

美国加利福尼亚州南旧金山，2026年1月21日——专注于发现和开发新型肿瘤疗法的临床阶段生物制药公司Opna Bio今日宣布，美国食品药品监督管理局（FDA）已授予其溴结构域和末端外结构域（BET）小分子抑制剂OPN-2853用于治疗骨髓纤维化（MF）的孤儿药资格（ODD）。此外，国际非专利药品名称（INN）已批准将zavabresib作为OPN-2853的通用名。 骨髓纤维化是一种罕见且严重的血液癌症，其特征为骨髓纤维化，会导致造血功能异常，并引发重度乏力、脾脏肿大、贫血等症状。美国约有25,000人受该疾病影响。 Opna Bio首席执行官雷纳尔多·迪亚兹（Reinaldo Diaz）表示：“zavabresib用于骨髓纤维化治疗的孤儿药资格获批，对Opna Bio而言是一项重要的监管里程碑，也凸显出这类患者对全新有效治疗方案的迫切需求。我们开展的zavabresib联合芦可替尼（ruxolitinib）研究者发起临床试验，迄今已取得令人瞩目的结果，包括为晚期骨髓纤维化患者实现持久的脾脏缩小。我们认为，选择性BET抑制联合JAK抑制，有望为骨髓纤维化患者提供一种极具前景的新治疗策略。近期与FDA的积极沟通也让我们备受鼓舞，公司将继续推进zavabresib的更多临床研究。” FDA授予孤儿药资格的对象，是用于治疗、诊断或预防美国患病人数不足20万人的罕见病的在研疗法。该资格可带来多项利好，包括临床试验费用税收抵免、免除部分FDA相关费用，以及药物获批后可享有七年市场独占权。 目前，由牛津大学亚当·米德（Adam Mead）教授主导、与英国癌症研究院（CRUK）合作开展的Ⅰ期PROMise研究正在进行中，该研究旨在评估zavabresib联合芦可替尼，用于治疗对芦可替尼不再响应的骨髓纤维化患者的疗效。2025年12月美国血液学会（ASH）年会上公布的数据显示，在26名可评估的联合治疗患者中，有16名患者的脾脏长度较基线缩小50%及以上。 关于Opna Bio Opna Bio是一家临床阶段生物制药公司，专注于新型肿瘤疗法的发现与开发。公司拥有丰富的研发管线，靶向多种癌症驱动因子，包括：处于多发性骨髓瘤Ⅰ期临床试验阶段的双靶点EP300/CBP抑制剂OPN-6602；正与芦可替尼联合开展骨髓纤维化Ⅰ期临床试验、具有潜在同类最优属性的BET溴结构域抑制剂OPN-2853。此外，公司还有一项针对多发性骨髓瘤和淋巴瘤的临床前多功能降解剂研发项目，靶点为EP300/CBP与伊卡洛斯/艾俄洛斯（Ikaros/Aiolos）。Opna Bio团队拥有深厚的科研实力和商业价值创造经验，已成功发现并开发出多款获FDA批准的药物。如需了解更多信息，敬请访问官网：opnabio.com。

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Opna Bio, a clinical-stage biopharmaceutical company focused on the discovery and development of novel oncology therapeutics, today announced that OPN-2853, a bromodomain and extra-terminal motif (BET) small molecule inhibitor, has been granted Orphan Drug Designation (ODD) for the treatment of myelofibrosis (MF) by the U.S. Food and Drug Administration (FDA). Additionally, the generic name of zavabresib for OPN-2853 has been approved by the International Nonproprietary Names (INN) for Pharmaceutical Substances. "Receiving Orphan Drug Designation for zavabresib in myelofibrosis is a significant regulatory milestone for Opna Bio and highlights the urgent need for new and effective treatment options for patients with this disease." Myelofibrosis is a rare and serious type of blood cancer characterized by bone marrow scarring, which leads to ineffective blood cell production and symptoms such as severe fatigue, enlarged spleen, and anemia. Myelofibrosis affects approximately 25,000 people in the U.S. “Receiving Orphan Drug Designation for zavabresib in myelofibrosis is a significant regulatory milestone for Opna Bio and highlights the urgent need for new and effective treatment options for patients with this disease,” said Reinaldo Diaz, chief executive officer of Opna Bio. “Our investigator-sponsored clinical trial with zavabresib and ruxolitinib has shown impressive results to date, including durable spleen reduction in patients with advanced myelofibrosis. We believe that selective BET inhibition alongside JAK inhibition offers a promising new therapeutic approach for patients with myelofibrosis. We are further encouraged by recent positive meetings with the FDA to continue to test zavabresib in additional clinical studies.” The FDA grants Orphan Drug Designation to investigational therapies intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. The designation provides several benefits, including tax credits for clinical trial costs, a waiver of certain FDA fees, and eligibility for seven years of market exclusivity upon approval. In the ongoing Phase 1 PROMise study led by Professor Adam Mead at the University of Oxford through a collaboration with Cancer Research UK (CRUK), zavabresib is being evaluated as an add-on to ruxolitinib in patients with myelofibrosis who are no longer responding to ruxolitinib alone. Data presented at the American Society of Hematology conference in December 2025 showed a 50% or greater reduction of spleen length in 16 of 26 evaluable patients on the combination treatment when compared to baseline. About Opna Bio Opna Bio is a clinical-stage biopharmaceutical company focused on the discovery and development of novel oncology therapeutics. The company’s broad portfolio targets multiple drivers of cancer, including OPN-6602, a dual EP300/CBP inhibitor, currently in a Phase 1 clinical trial in patients with multiple myeloma, and OPN-2853, a potentially best-in-class BET bromodomain inhibitor, in a combination Phase 1 clinical trial with ruxolitinib in patients with myelofibrosis. Our novel, preclinical multi-functional degrader program is focused on targeting EP300/CBP and Ikaros/Aiolos for the treatment of multiple myeloma and lymphomas. The Opna team has a proven track record of scientific expertise and commercial value creation, having discovered and developed multiple drugs that have achieved FDA approval. For more information, please visit opnabio.com.