Eculizumab

--- Precision Bb Targeting, Robust Efficacy, Transfusion Independence, Excellent Safety in Phase II PNH Study, and Subcutaneous Administration Key Highlights: Monotherapy and Clinical Outcomes The 12-16-week open-label study evaluated Ruxoprubart as monotherapy in treatment-naïve PNH patients. Key findings include: Hemoglobin (Hb) Improvement: Hemoglobin levels increased by 1.5–2.7 g/dL, with an average rise of ~2.13 g/dL during weekly dosing, reflecting meaningful correction from the severely depressed baseline common in untreated PNH. Complete Transfusion Independence: Throughout the weekly dosing period, 100% of patients required no blood transfusions, demonstrating effective control of hemolysis and clinically meaningful correction of anemia. PNH Red Blood Cells (RBCs) Survival & Expansion: PNH RBC populations expanded to up to 94% of their maximum level. This is consistent with near-complete suppression of ongoing complement-mediated hemolysis, a core driver of anemia and fatigue. Biochemical Control (LDH): Lactate Dehydrogenase (LDH) levels were significantly reduced, reflecting biochemical control of intravascular hemolysis (IVH). Quality-of-Life (QoL) Gains: Patient‑reported outcomes, including the Functional Assessment of Chronic Illness Therapy (FACIT) and the European Organization for Research and Treatment of Cancer (EORTC) questionnaires, showed meaningful improvements across fatigue, pain, and overall functional capacity. Safety and Tolerability: The therapy was well-tolerated with no drug-related Adverse Events (AEs) or Serious Adverse Events (SAEs) observed. Orphan Drug Designation: Ruxoprubart holds U.S. FDA Orphan Drug Designation (ODD) for PNH, highlighting its potential to address this rare and life-threatening condition. CLEVELAND, March 09, 2026 (GLOBE NEWSWIRE) -- NovelMed Therapeutics Inc., a clinical-stage biopharmaceutical company specializing in alternative pathway (AP) complement-mediated therapies, today announced two major milestones in the clinical development of Ruxoprubart (NM8074) for Paroxysmal Nocturnal Hemoglobinuria (PNH). The company has received regulatory clearance to initiate a Phase II study for the subcutaneous (SC) route of administration, designed as a convenient, weekly, self-administered injection. Concurrently, NovelMed reported the full results of its Phase II intravenous (IV) monotherapy study in treatment-naïve adult patients with PNH. The study met all efficacy endpoints, including significant hemoglobin improvement and complete transfusion avoidance, with an exceptional safety profile. “These milestones validate both our clinical progress and our development strategy,” said Dr. Rekha Bansal, Chief Executive Officer of NovelMed. “Regulatory clearance of the SC route represents the next logical step toward a patient-friendly treatment option, while our monotherapy data demonstrate the strong, consistent efficacy and tolerability of targeting Bb. We are now progressing into the next phase of clinical development.” Advancing Two Complementary Tracks in PNH NovelMed is progressing Ruxoprubart through two distinct delivery methods to maximize patient access and convenience: Subcutaneous (SC) Administration: Cleared for Phase II Development The regulatory agency has cleared the SC route of administration, enabling a planned, weekly self-injection regimen at home. SC trials are planned but have not yet been initiated. This approach is expected to reduce patient and caregiver burden by eliminating the need for IV infusions in a clinical setting. Intravenous (IV) Monotherapy: Treatment-Naïve PNH Patients The successful completion of the intravenous Phase II study establishes Ruxoprubart as a potent monotherapy capable of addressing the core drivers of PNH without adjunctive treatment. “Ruxoprubart has proven its potential as a definitive monotherapy, delivering exceptional efficacy without compromise,” said Mr. Alex Kumar, Chief Strategy Advisor. “Coupled with FDA Orphan Drug Designation status and regulatory clearance for SC dosing, we have a clear, accelerated path forward. NovelMed is now uniquely positioned to lead the next generation of precision complement therapeutics on a global scale.” Precision Targeting: The Bb Mechanism and Differentiation PNH is an AP-driven hemolytic disorder. Ruxoprubart (NM8074) is a first-in-class monoclonal antibody engineered for precision targeting of Bb, the proteolytically activated catalytic fragment generated upon AP activation. By binding Bb rather than native Factor B, Ruxoprubart delivers precise AP-selective inhibition while preserving the Classical Pathway (CP), which remains essential for protecting patients from infections. “The Phase II intravenous results demonstrate a clinical efficacy and safety pro aligns well with FDA expectations for monotherapy development in PNH,” said Mr. Robert Bard. “With the SC route now cleared for evaluation under our Phase II program, we are advancing along a well-defined regulatory path. This progress brings us closer to enabling patient-friendly, self-administered treatment options supported by a mechanism designed for pathway-selective precision.” Competitive Landscape at a Glance Ruxoprubart (NovelMed): Targets activated Bb (FDA Orphan Drug Designation Holder). A precision monotherapy that provides selective AP inhibition while preserving the CP for host defense. Soliris® / Ultomiris® (AstraZeneca): Targets C5. A distal blocker of both AP and CP that fails to prevent C3b deposition, which leads to persistent anemia. Empaveli® / Syfovre® (Apellis): Targets C3. A proximal blocker that provides broad inhibition of both AP and CP, addressing C3b-mediated EVH but impacting overall complement surveillance. Fabhalta® (Novartis): Targets native Factor B. A proximal blocker that lacks precision by targeting the native protein rather than the proteolytically activated Bb fragment. Voydeya® (AstraZeneca): Targets Factor D. A proximal blocker, strictly approved as an add-on therapy to C5 inhibitors rather than a standalone monotherapy. Key Pillars of the Regulatory Strategy Monotherapy Treatment: Ruxoprubart has demonstrated the ability to meet all primary endpoints as a standalone treatment. This allows for a more direct regulatory path, as the drug does not need to be tested or approved alongside existing C5 inhibitors to prove its value. Subcutaneous (SC) Administration: The regulatory clearance for the SC route is a major milestone. Having passed rigorous safety and manufacturing reviews, Ruxoprubart is now cleared for human trials using at-home injections. This shifts the treatment model from clinical IV infusions to a more convenient, patient-controlled approach. Precision Targeting of the Bb: Ruxoprubart selectively targets the AP while keeping the CP intact. This "selective precision" is designed to maintain the body’s natural ability to fight infections, a differentiated safety pro supports the case for expedited regulatory designations like Fast Track or Breakthrough Therapy. Figure. Complement-mediated destruction of red blood cells in paroxysmal nocturnal hemoglobinuria (PNH). About Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare, acquired, and life-threatening hematologic disorder caused by a somatic mutation in the PIGA gene. This mutation results in the deficiency of GPI-anchored complement regulatory proteins, specifically CD55 and CD59, which act as protective shields for RBCs. Without these proteins, RBCs are highly vulnerable to persistent attack by the AP of the complement system, leading to chronic intravascular and extravascular hemolysis. This ongoing destruction manifests clinically as severe anemia, dangerously elevated Lactate Dehydrogenase (LDH) levels, and a significantly increased risk of life-threatening thrombosis and renal impairment. Effective treatment is measured by stabilization of hemoglobin and the expansion of the PNH RBC clone, indicating that these cells are successfully protected from complement-mediated attack. About NovelMed Therapeutics NovelMed Therapeutics Inc. is a clinical-stage biopharmaceutical company dedicated to developing next-generation immunotherapies that selectively target the complement AP. The company has pioneered a site-specific approach by engineering monoclonal antibodies, such as its lead candidates Ruxoprubart (NM8074) and NM5072, to target proteolytically activated fragments, such as Bb, rather than native proteins. This precision enables potent blockade of the disease-driving AP while intentionally preserving the CP’s critical role in host defense and immune surveillance. Supported by a robust portfolio of intellectual property and promising Phase II data, NovelMed is advancing a differentiated pipeline of first-in-class biologics designed to achieve durable clinical outcomes without compromising the patient’s ability to fight infections. Strategic Partnership Opportunity Following the achievement of 100% transfusion avoidance in its Phase II monotherapy study and the recent regulatory clearance for SC administration, NovelMed is actively pursuing a strategic partnership to propel Ruxoprubart through late-stage global development. This program is exceptionally well-positioned for expansion, supported by U.S. FDA Orphan Drug Designation (ODD) and a validated, precision mechanism that addresses significant unmet needs in the PNH market. NovelMed seeks a partner with extensive clinical and commercial expertise to accelerate the regulatory path towards approval and support the global launch of this patient-friendly, self-administered therapy. By combining NovelMed’s innovative science with a partner's scale, the company aims to redefine the treatment landscape for rare complement-mediated diseases. Media Contact: Ya Gao, MS NovelMed Therapeutics, Inc. media@novelmed.com (216) 440 2696 Forward-Looking Statements This press release contains forward-looking statements that reflect NovelMed’s current expectations and projections about future events. Ruxoprubart (NM8074) is an investigational therapy and has not been approved by any regulatory authority for commercial use. The subcutaneous administration route has received regulatory clearance for further study; no subcutaneous clinical trials have been initiated. Forward-looking statements include expectations about clinical development, regulatory interactions, future study plans, and potential outcomes. NovelMed is a privately held, clinical stage biotechnology company which undertakes no obligation to update these statements except as required by law. A photo accompanying this announcement is available at

今天要跟大家分享一个医药圈的重磅消息——海思科医药集团（股票代码：002653.SZ）的子公司四川海思科制药有限公司，近日收到了国家药品监督管理局下发的《受理通知书》，其自主研发的创新药HSK39297片的上市许可申请正式获受理！ 这意味着什么？简单说，一款针对罕见血液病——阵发性睡眠性血红蛋白尿症（PNH）的全新口服治疗药物，离正式上市又近了一大步。 对于PNH患者来说，这可能是改变命运的好消息。因为HSK39297不是普通的仿制药，而是 化药1类创新药 ，属于“全球新”的原创药物。更关键的是，它是 口服制剂 ，相比目前需要每两周去医院静脉注射一次的现有疗法，简直是天壤之别。 一、什么是PNH？为什么叫“睡眠性”血红蛋白尿？ 先来科普一下这个听起来有点拗口的疾病。 阵发性睡眠性血红蛋白尿症（PNH），是一种罕见的获得性造血干细胞克隆性疾病。名字里的“睡眠性”其实是个美丽的误会——并不是说这个病只在睡觉时发作，而是因为患者 晨起时尿液颜色会像酱油一样深 ，古人以为是“夜间睡眠时发作”，所以起了这个名字。 实际上，PNH是一种 补体介导的溶血性疾病 。通俗点说，就是患者体内的免疫系统“补体”会错误地攻击自己的红细胞，导致红细胞在血管内被破坏（血管内溶血），血红蛋白被释放到尿液中。 这种病有多可怕？数据显示，PNH全球患病率约为16-20/100万，中国发病率在1/10万左右。它好发于 20-40岁的青壮年 ，77%的患者在这个年龄段发病。 如果没有有效治疗，PNH患者的生存质量极差，5年死亡率高达35%。传统治疗手段下（包括糖皮质激素、免疫抑制、输血支持等），患者10年生存率仅70.7%-77.6%。 患者常常被三大问题困扰： 血红蛋白尿 ：尿液呈酱油色或浓茶色 严重贫血 ：因为红细胞被大量破坏 血栓形成 ：这是PNH患者死亡的主要原因之一 二、现有治疗的困境：每两周就要跑一次医院 目前，PNH的标准治疗是 补体C5抑制剂 ，代表药物就是依库珠单抗（商品名：舒立瑞®）。 依库珠单抗的工作原理是“末端拦截”——它靶向补体系统的最后一个环节C5蛋白，阻止膜攻击复合物（MAC）的形成，从而控制血管内溶血。 听起来不错，但现实很骨感： 第一，给药方式极其不便 。依库珠单抗需要 每两周静脉输注一次 ，每次输注时间25-45分钟。这意味着患者每半个月就要跑一次医院，风雨无阻。 第二，治疗效果有限 。虽然依库珠单抗能控制血管内溶血，但对 血管外溶血 基本无效。血管外溶血是由C3b介导的，而C5抑制剂管不到这个环节。结果就是， 仍有25%-35%的在治患者依赖红细胞输注 ，生活质量大打折扣。 第三，存在突破性溶血风险 。研究显示，9例仅接受1次依库珠单抗治疗的患者中，在随访28天时3例出现突破性溶血。 第四，感染风险高 。所有患者必须在接受依库珠单抗给药前至少2周接种脑膜炎球菌疫苗，并且必须根据国家疫苗接种指南进行疫苗接种或再接种。紧急治疗未接种疫苗者需要预防性使用抗生素。 三、HSK39297：从“末端拦截”到“源头控制”的革命 HSK39297的出现，可能彻底改变这个局面。 1. 作用机制：补体系统的“上游调控者” 如果把补体系统比作一条河流，依库珠单抗是在 下游建水坝 （抑制C5），而HSK39297是在 上游控制水源 （抑制补体B因子）。 HSK39297是海思科自主研发的一种 高效、高选择性的口服补体B因子（FB）小分子抑制剂 。它通过抑制FB活性，从而阻断旁路途径（AP）的激活与补体扩增循环，进而抑制整个补体通路的活性。 更具体地说： 抑制C3转化酶形成 ：补体旁路途径的激活始于C3转化酶（C3bBb）的形成，HSK39297通过抑制因子B与C3b的结合，阻止C3转化酶的生成 阻断C5转化酶形成 ：C3转化酶进一步催化C5转化为C5a和C5b，HSK39297通过抑制C3转化酶的活性，间接阻断C5转化酶的形成 抑制C3正反馈扩增 ：补体系统的正反馈扩增是导致过度激活的关键，HSK39297通过抑制因子B的循环利用，阻断这一扩增过程 这种“上游控制”的优势非常明显： 既能控制血管内溶血，又能控制血管外溶血 ，实现双重保护。 2. 临床数据：疗效令人振奋 在2025年9月的中华医学会第十九次全国血液学学术会议上，海思科公布了HSK39297治疗PNH的II期临床研究结果。 这项多中心、随机、开放的II期研究共纳入47例PNH患者，这些患者都伴有显著血管内溶血（基线乳酸脱氢酶均值1964.8 U/L）及贫血（基线血红蛋白均值仅69.3 g/L），其中63.8%的患者在筛选前12个月内曾有输血史。 研究结果相当亮眼： 疗效方面 ： 快速起效 ：受试者在接受HSK39297片治疗后 1周 即可观察到LDH下降及Hb升高，治疗 2周 起LDH已下降至接近正常水平 显著改善贫血 ：治疗24周，受试者的Hb均值从69.3 g/L升至 120.1 g/L 高比例脱离输血 ： 95.7%的受试者实现脱离输血依赖 主要终点达标率高 ：200 mg QD组 93.8% 的受试者达到主要终点（Hb升高≥20 g/L且4~24周不输血），与国内已上市同类药物伊普可泮的治疗应答率相当（92.2%） 安全性方面 ： 治疗期间受试者安全耐受性良好 药物不良反应无剂量相关性 未导致停药或减量 3. 相比依库珠单抗的四大优势 根据海思科的公告，HSK39297片在改善贫血、减少输血需求、缓解疲劳症状等方面 较依库珠单抗均有显著的治疗优势，且疗效可长期维持 。 具体优势体现在： 优势一：口服给药，患者依从性大幅提升 从每两周跑一次医院静脉输注，变成每天在家口服一片药，这对患者的生活质量是质的飞跃。 优势二：双重控制，疗效更全面 HSK39297通过抑制补体B因子，同时控制血管内溶血和血管外溶血，而依库珠单抗只能控制血管内溶血。 优势三：摆脱输血依赖比例更高 II期数据显示95.7%的患者脱离输血依赖，而依库珠单抗治疗后仍有25%-35%的患者需要输血。 优势四：可能降低突破性溶血风险 HSK39297的III期研究方案中，明确将突破性溶血（BTH）发生率写进了次要终点观察项，说明研究者对这一指标的重视。 四、国产口服补体抑制剂“三小只”的竞争 HSK39297并不是孤军奋战。目前国内还有另外两款口服补体B因子抑制剂也在研发中，被病友圈称为国产PNH口服补体抑制剂“三小只”。 维度 MY008211A（朗来科技） HRS-5965（恒瑞医药） HSK39297（海思科） 剂型 片剂 胶囊剂 片剂 适应症 既往未接受补体抑制剂的PNH成人 既往未接受补体抑制剂的PNH成人 既往未接受补体抑制剂的PNH成人 关键III期设计 既有单臂III期（C5单抗疗效不佳人群） 随机、开放、阳性对照III期（对比依库珠单抗） 随机、开放、阳性对照III期（对比依库珠单抗） 主要终点 Hb在第126-128天的持续达标 第18–24周，无输血前提下多次Hb≥12g/dL的比例 第18–24周，无输血前提下多次Hb≥120g/L的比例 监管进度 NDA于2025-10-17获受理，纳入优先审评 进入拟纳入优先审评程序 上市申请获受理（受理号CXHS2600002），纳入优先审评 额外布局 公开信息显示在其他领域也有布局 企业披露：IgA肾病等也在开展研究 狼疮肾炎II期、IgA肾病III期准备启动 从进度上看，MY008211A的NDA受理更早（2025年10月17日），但HSK39297的临床数据已经相当扎实。 更重要的是，HSK39297的适应症布局更广。除了PNH，它还在开展 狼疮肾炎患者的Ⅱ期临床试验 ，同时也在启动 原发性IgA肾病Ⅲ期临床研究 。这意味着如果这些适应症都能获批，HSK39297的市场空间将大大扩展。 五、海思科：从仿制药巨头到创新药领军者 可能有些朋友对海思科不太熟悉，这家公司可是中国创新药领域的“隐形冠军”。 海思科医药集团成立于2000年，2012年在深交所上市（股票代码：002653.SZ）。公司最初以特色仿制药起家，但现在已成功转型为 创新药研发企业 。 1. 研发投入：五年砸下43.58亿元 创新药研发是烧钱的游戏，海思科在这方面毫不手软。2020年—2024年，公司研发投入分别为7.09亿元、8.15亿元、9.58亿元、8.75亿元、10.01亿元， 五年累计达43.58亿元 。 2024年，海思科研发投入占总营收比例达 26.90% ，这个比例在医药企业中属于很高水平。 2. 创新药管线：已进入收获期 海思科已经构建起多元化的创新药管线： 已上市创新药 ： 环泊酚注射液（思舒宁®） ：中国首个自主研发且具有全球自主知识产权的1类静脉麻醉药，2024年销售收入 12.32亿元 ，同比增长44.96%，占总营收的33.10% 苯磺酸克利加巴林胶囊（思美宁®） ：中国大陆首个获批用于治疗糖尿病周围神经病变疼痛的新药 考格列汀片（倍长平®） ：全球首创双周超长效口服降糖药 安瑞克芬注射液（思舒静®） ：全球首个获批镇痛适应症且无需纳入麻醉精神药品管理的白处方阿片类药物 在研重磅产品 ： HSK31858 ：DPP-1抑制剂，非囊性纤维化支气管扩张症适应症处于临床III期 HSK39297 ：就是本文主角，补体B因子抑制剂 还有HSK45019、HSK46575、HSK36357等多个在研项目 截至2025年6月30日，海思科的商业化产品及进入临床阶段的1类新药共有 17个 ，还有处于筛选阶段的项目40余项。 3. 国际化布局：从中国走向世界 海思科的野心不限于国内市场。2020年，公司在美国成立全资子公司美国海思科。2024年，向美国海思科增资4000万美元，专项用于创新药临床研究和日常运营维护。 环泊酚注射液已向美国FDA递交全麻诱导适应症的NDA申请，且已申请中国、美国、加拿大、日本、欧洲等20多个国家和地区的专利。 2022年，海思科与意大利Chiesi公司达成协议，将DPP1小分子抑制剂（HSK31858）大中华区以外的全球范围内开发、生产和商业化的独家权利以 4.62亿美元 价款转让。 六、市场前景：百亿蓝海等待开拓 PNH虽然属于罕见病，但治疗费用高昂，市场空间不容小觑。 依库珠单抗在国内的年治疗费用约 100-150万元 ，这对大多数家庭来说是沉重的负担。HSK39297作为口服制剂，如果定价合理，有望大幅降低患者的治疗负担。 更重要的是，HSK39297的适应症不止PNH。它还在开展狼疮肾炎、原发性IgA肾病的临床研究。 狼疮肾炎 是系统性红斑狼疮最严重的并发症之一，约50%-70%的狼疮患者会出现肾脏受累。 原发性IgA肾病 是全球最常见的原发性肾小球疾病，中国是IgA肾病的高发区，患者数量庞大。 如果这些适应症都能获批，HSK39297的市场潜力将是巨大的。 七、风险提示：创新药研发的不确定性 当然，创新药研发从来不是一帆风顺的。海思科在公告中也明确提示：“由于药品审评周期长、环节多，容易受到一些不确定性因素的影响，敬请广大投资者谨慎决策，注意防范投资风险。” 从受理到最终获批，HSK39297还需要经过： 技术审评 ：CDE专家对申报资料进行全面审查 现场核查 ：对生产现场、临床试验机构等进行核查 审评审批 ：综合评估后做出是否批准的决定 虽然HSK39297已被纳入优先审评程序（审评时限目标从常规200个工作日缩短到130个工作日），但具体获批时间仍有不确定性。 八、结语：中国创新药的春天来了 HSK39297上市申请获受理，不仅对PNH患者是个好消息，也标志着中国创新药研发正在从“跟跑”向“并跑”甚至“领跑”转变。 从海思科的发展轨迹，我们可以看到中国创新药企的成长路径： 从仿制到创新 ：从特色仿制药起家，逐步建立自主创新能力 从国内到国际 ：不仅满足国内需求，还要走向全球市场 从单点到全面 ：构建多元化的产品管线，降低风险 对于PNH患者来说，HSK39297可能意味着： 从每两周跑一次医院，变成每天在家服药 从可能还要输血，到基本摆脱输血依赖 从只控制血管内溶血，到血管内外溶血双重控制 从高昂的治疗费用，到可能更经济的治疗方案 当然，这一切还要等待最终的审评结果。但无论如何，HSK39297的进展让我们看到了希望——中国药企正在用实实在在的研发投入，为患者带来更好的治疗选择。 创新药的研发之路漫长而艰辛，但每一次突破都值得期待。HSK39297能否成为PNH治疗的新标杆？让我们拭目以待！ 注 ：本文基于公开信息整理，不构成任何投资建议。药品具体疗效和安全性以最终说明书为准，患者用药请遵医嘱。