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注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。2025年4月7日，药融圈此前长文撰写过的纳斯达克上市公司Zenas BioPharma, Inc.(以下简称"Zenas"或"公司")(代码:ZBIO)宣布任命Haley Laken博士为首席科学官(CSO)。（引进模式妙手：泽纳仕生物申请纳斯达克IPO，成立3年吸金超4亿美元）在加入Zenas之前，Laken博士曾担任Tabby Therapeutics公司的首席运营官，这是一家专注于B细胞工程的私营生物技术公司，她在该公司领导所有的研发和运营部门。在此之前，Laken博士在Jounce Therapeutics公司担任多项职务，最后担任首席开发官和高管团队成员。在此之前，她在TESARO公司担任高级项目管理领导职务，领导多个生物制剂开发项目从研究到早期临床开发的全过程。Laken博士还曾在Rhythm Pharmaceuticals、辉瑞和惠氏担任药物开发战略领导职务。Laken博士拥有迪堡大学生物学学士学位和约翰斯·霍普金斯大学人类遗传学与分子生物学博士学位，目前她是InCephalo Therapeutics公司的董事会成员。此前在3月中旬，Zenas宣布任命医学博士Lisa von Moltke为其研发负责人兼首席医学官 (CMO)。加入Zenas之前，von Moltke 博士在Seres Therapeutics公司担任CMO，负责临床开发、科学与运营、注册与医疗事务。在加入Seres之前，von Moltke博士在 Alkermes公司任职，在职期间的职责不断扩大，最后担任高级副总裁兼临床开发负责人。在加入Alkermes之前，von Moltke博士曾担任赛诺菲/健赞公司的临床与探索性药理学副总裁，还曾担任美国临床与探索性药理学(CEP)和早期开发负责人，后来又担任日本和中国的CEP负责人。在其职业生涯早期，von Moltke博士曾担任Millennium公司(武田肿瘤公司)的转化医学部负责人。von Moltke 博士曾是一名执业医师，并在塔夫茨大学医学院完成了临床药理学研究，在药理学和开发治疗学领域拥有长达15年的学术生涯。2021年3月23日，Zenas BioPharma成立，不少中国资本押注其中。如再鼎相关的泉创资本、WuXi Biologics HealthCare Venture、维梧资本。截至2024年12月31日，公司拥有约3.5亿美元的现金、现金等价物和短期投资，预计这些资金可满足公司至2026年第四季度的运营支出和资本支出需求*，因此公司有充足的资本实现2025年的主要里程碑。（*这一数额是初步的，未经审计，有待公司完成财务结算程序。因此，该金额可能与公司截至2024年12月31日的年度合并财务报表中反映的金额存在差异。）核心产品Obexelimab作用机制Obexelimab 2025该公司预期的临床里程碑Obexelimab是一种双功能单克隆抗体，可同时结合CD19和FcγRIIb(广泛存在于B细胞系中)，从而抑制与许多自身免疫性疾病有关的细胞的活性，而不会耗竭它们。这种独特的作用机制和每周一次的皮下注射方案可广泛而有效地解决B细胞系在慢性自身免疫性疾病中的致病作用。Obexelimab已在五项已完成的临床试验中进行了评估，共有198名患者通过静脉输注或皮下注射接受了Obexelimab治疗。在这五项临床试验中，Obexelimab的耐受性和临床活性均表现良好，为公司提供了一个初步的临床概念验证，即Obexelimab是一种有效的B细胞抑制剂，可用于治疗某些自身免疫性疾病。（2021年11月21日，Zenas宣布从Xencor获得Obexelimab的全球独家权利。此前还有与Amgen的爱恨纠葛）来源：摩熵医药数据pharma.bcpmdata.com（原药融云数据）2025年，公司预计将实现以下重要临床里程碑：在2025年第三季度获得针对RMS患者的MoonStone 2期试验的12周主要终点结果。其他公司的抗CD20 B细胞靶向疗法，包括OCREVUS®(ocrelizumab)和KESIMPTA®(ofatumumab)的成功临床开发、批准和临床应用证明了B细胞在包括RMS在内的多发性硬化症发病机制中的作用，这些疗法可选择性地清除CD20表达的B细胞。公司认为，obexelimab具有独特的作用机制，能有效抑制CD20更广泛的B细胞系但不消耗B细胞，其非临床数据和皮下注射方案都支持其治疗RMS的潜力。o 经过初步筛选后，MoonStone试验的患者将按2:1的比例随机分配到250毫克的Obexelimab或安慰剂中，每七天皮下注射一次，治疗期为12周。o 该试验双盲部分的主要目标是评估T1加权磁共振成像(MRI)中发现的钆(Gd)增强病灶的累积数量与基线相比的变化。o 12周疗程结束后，患者将进入开放标签期，在此期间，服用安慰剂的患者将接受至少3个月的Obexelimab治疗，而最初随机接受Obexelimab治疗的患者将继续接受治疗。o 开放标签期的重要次要终点包括使用标准化评估、新型三维成像和生物标志物(包括血清神经丝轻链(NfL))来评估Obexelimab对疾病进展的影响。2025年年末公布针对IgG4-RD患者的INDIGO 3期试验的初步结果IgG4-RD是一种慢性免疫介导纤维炎性疾病，几乎可影响所有器官系统，包括胰腺、胆道、唾液腺和泪腺、肺和肾。IgG4-RD患者可能表现为单一器官症状，但更常见的是多器官受影响。随着病情的发展，患者会出现新的或恶化的症状(即复发)，病变会在更多器官中发展，早期疾病的细胞炎症会向纤维化阶段发展，这可能会导致严重的不可逆转的组织损伤，最终导致器官衰竭。我们估计，目前美国确诊的IgG4-RD患者约有20,000人，全球发病率相当。尽管人们对IgG4-RD的认识在不断提高，对其病理生理学的理解也在不断进步，但治疗这种疾病尚未有合适疗法，仍有大量治疗需求未得到满足。目前的标准疗法是使用糖皮质激素(GCs)治疗。尽管糖皮质激素最初是有效的，但使用糖皮质激素治疗往往会导致各种并发症和合并症。大多数患者会在停止GCs治疗后的12个月内复发，而GCs的维持治疗尚未证明能防止疾病复发。IgG4-RD的发病机制表明，B细胞靶向疗法可能会带来治疗效果。虽然还未有得到批准的产品用于治疗IgG4-RD，但某些B细胞耗竭剂(如rituximab)偶尔也会用于IgG4-RD患者。然而，B细胞耗竭剂药物通常与感染有关，包括严重机会性感染，而且会损害患者对疫苗接种产生反应的能力。有证据表明B细胞在IgG4-RD发病机制中的作用，B细胞靶向药物在既往的 IgG4-RD试验中观察到的效果，我们用Obexelimab进行的IgG4-RD 2期试验的数据，以及它独特的非耗竭机制和每周一次的皮下注射方案，都支持在IgG4-RD患者中开发这种药物。o INDIGO是针对IgG4-RD患者开展的规模最大的临床试验，旨在评估Obexelimab在约190名活动性IgG4-RD患者中的安全性和有效性，目前正在20个国家的约100个研究中心进行。o 经过初步筛选后，患者按1:1随机分配到250毫克的Obexelimab或安慰剂，每七天皮下注射一次，持续 52 周，随后符合条件的患者有机会继续接受开放标签延长期治疗，所有患者都将接受Obexelimab治疗。o INDIGO的主要疗效终点是首次IgG4-RD复发的时间，由研究者和裁定委员会根据方案确定。o 次要终点包括年复发率、获得完全缓解的患者比例以及补救药物(包括 GCs)的使用情况和数量。有关INDIGO 3期试验(NCT05662241)的更多信息，请访问clinicaltrials.gov于2025年完成系统性红斑狼疮患者SunStone 2期试验的入组工作，并于2026年上半年报告初步结果从产生自身抗体到异常调节免疫反应，B细胞在系统性红斑狼疮发病机制中的关键作用已得到认证。此外，系统性红斑狼疮是一种自身免疫性疾病，其特点是B细胞功能障碍、产生针对细胞和核成分的自身抗体，以及受影响组织内免疫复合物沉积和炎症引起的多器官损伤。目前的治疗方法数量有限，疗效一般。Obexelimab在之前的一项2期双盲随机试验中显示出了临床活性，在整个试验人群中证明了概念，并在保持较高的Obexelimab全身暴露量的患者和生物标志物定义的亚人群中增加了反应。加上迄今为止获得的安全性数据，我们相信这些数据为在系统性红斑狼疮患者中开发Obexelimab 提供了支持。o 经研究者和裁定委员会筛选确定为活动性系统性红斑狼疮患者，按1:1随机分配到Obexelimab 250毫克或安慰剂，每七天皮下注射一次，共注射24周。o在之前的系统性红斑狼疮2期试验中，较高的全身暴露量(Ctrough)与较高的临床活性相关，因此选择了每周一次的250毫克皮下注射剂量，以最大限度地提高临床活性。o 主要终点是在第24周时系统性红斑狼疮疾病活动减少的应答者百分比，该百分比由基于BILAG的狼疮综合评估确定。o 计划对所有患者进行生物标志物分析，包括基线RNA表达谱，以对患者进行免疫分型，并评估他们对治疗的不同反应。参考：NMPA/CDE；摩熵医药数据pharma.bcpmdata.com（原药融云数据）；FDA/EMA/PMDA；clinicaltrials.gov；www.zenasbio.com；https://pharma.bcpmdata.com/database/24/detail/441/ThZHD5YBg-Rn_cM4_t1Z；等等。 此文仅用于向医疗卫生专业人士提供科学信息，不代表平台立场，不作任何用药推荐。更多药企信息交流或合作可后台留下名片。植根上海、辐射全球,药融圈作为生物医药产业级战略平台,以"让智慧与人脉无界流动"为使命,构建覆盖药物研发、生产、流通、商业化的全产业链赋能体系。通过智能化云服务、精准资源链接、产业智库与生态社群四大核心引擎,打造中国医药价值流动的基础设施。在全球化与数字化双重浪潮下,药融圈正重新定义医药资源的流通范式--这里不仅是信息与人脉的枢纽站,更是催生产业变革的化学反应器。我们以中国为原点,编织全球医药智慧网络,让每一次精准链接都成为企业跨越式增长的催化剂。点点赞点分享点推荐

CORAL GABLES, Fla., April 08, 2025 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare and difficult-to-treat diseases, today reported that its sub-licensee in Canada, Kye Pharmaceuticals, Inc. (Kye), has announced that Health Canada has accepted the New Drug Submission (NDS) for AGAMREE®, a novel corticosteroid treatment for Duchenne muscular dystrophy (DMD), for review. The submission has been granted Priority Review, expediting the regulatory process with the potential for marketing authorization before the end of 2025. If approved, AGAMREE would be the first and only treatment option indicated for patients diagnosed with DMD in Canada. “We are pleased that Health Canada has accepted the New Drug Submission for AGAMREE with a Priority Review, recognizing its potential to address a significant unmet need for patients with Duchenne muscular dystrophy in Canada,” stated Richard J. Daly, President and CEO of Catalyst. “In collaboration with our Canadian sub-licensee, Kye, we remain committed to bringing this innovative therapy to DMD patients and ensuring access, particularly for those in underserved communities.” AGAMREE (40 mg/mL oral suspension) received U.S. Food and Drug Administration (FDA) approval on October 26, 2023, for the treatment of DMD in patients aged two years and older and became commercially available in the U.S. on March 13, 2024. Kye currently markets FIRDAPSE®, Catalyst’s flagship product for the treatment of Lambert-Eaton myasthenic syndrome (LEMS), in Canada. LEMS is a rare neuromuscular disorder characterized by debilitating and progressive muscle weakness and fatigue. About Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects males, with symptoms typically appearing in early childhood, around ages three to five. DMD is caused by mutations in the gene that encodes dystrophin, a protein that plays a crucial role in maintaining the structure and function of muscle fibers. Without dystrophin, muscle cells become fragile and easily damaged, leading to progressive muscle degeneration. Symptoms of DMD usually begin with difficulty in walking, frequent falls, and muscle weakness, particularly in the legs and pelvis. As the disease progresses, individuals may experience difficulty standing, climbing stairs, and eventually, complete loss of mobility. Other complications can include respiratory and cardiac issues due to muscle weakness. About AGAMREE® (vamorolone) AGAMREE's unique mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity. As such, it is considered a novel corticosteroid designed to achieve dissociative properties while maintaining efficacy that has the potential to demonstrate comparable efficacy to steroids, with the potential for a better-tolerated side effect profile. This mechanism of action may allow AGAMREE to emerge as an effective alternative to the current standard of care corticosteroids in children, adolescents, and adult patients with DMD. In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, psychiatric disorders, vomiting, weight increases, and vitamin D deficiency. Adverse events were generally of mild to moderate severity. About Kye Pharmaceuticals Kye Pharmaceuticals is a growth-stage Canadian specialty pharmaceutical company committed to bringing value to Canadians by identifying, licensing, and commercializing novel prescription medicines that may not otherwise be available to patients across Canada. With a growing pipeline of novel medicines, Kye's portfolio spans a range of therapeutic areas, including cardiology, psychiatry, pediatrics, rare diseases, hematology, and neurology. Kye Pharmaceuticals is a private company headquartered in Toronto focused on bringing medications to the Canadian market which fulfill clinically significant unmet needs. Kye is committed to licensing and launching medicines that matter by delivering better outcomes to our partners, Canadian healthcare professionals, and, most importantly, patients across Canada. For more information about the company, its management, portfolio and pipeline, please visit www.kyepharma.com About Catalyst Pharmaceuticals Catalyst Pharmaceuticals, Inc. (Nasdaq: CPRX) is a biopharmaceutical company committed to improving the lives of patients with rare diseases. With a proven track record of bringing life-changing treatments to the market, we focus on in-licensing, commercializing, and developing innovative therapies. Guided by our deep commitment to patient care, we prioritize accessibility, ensuring patients receive the care they need through a comprehensive suite of support services designed to provide seamless access and ongoing assistance. Catalyst maintains a well-established U.S. presence while actively seeking to expand its global commercial footprint through strategic partnerships. Catalyst, headquartered in Coral Gables, Fla., was recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America’s Fastest-Growing Companies. For more information, please visit Catalyst's website at www.catalystpharma.com. Forward-Looking Statements This press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including (i) whether Health Canada will approve AGAMREE either in the anticipated timeline or at all, (ii) if approved, whether Kye can successfully commercialize AGAMREE in Canada, and (iii) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2024 and its subsequent filings with the U.S. Securities and Exchange Commission (“SEC”), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date. Source: Catalyst Pharmaceuticals, Inc. Source: Catalyst Pharmaceuticals, Inc.