Rilzabrutinib

Eli Lilly has struck a deal to acquire Merida Biosciences for up to $2.875 billion, establishing a beachhead in its efforts to treat disease through the selective degradation of pathogenic autoantibodies. Merida exited stealth last year with $121 million and a vision for a new way of treating autoimmune and allergic diseases. While many existing therapies work by suppressing the immune system, Merida’s drug candidates target disease-driving antibodies that interact inappropriately with healthy cells. Eliminating the antibodies could treat disease without disrupting normal processes. Lilly sees promise in Merida’s technology. The biotech’s approach aligns with the Big Pharma’s focus on changing the course of disease, not just its downstream effects, Francisco Ramírez-Valle, M.D., Ph.D., senior vice president at Lilly immunology research and early clinical development, said in a statement. The $2.875 billion offer includes an upfront fee and milestone payments. In return for the outlay, Lilly will acquire a pipeline led by a potential Graves’ disease (GD) treatment. Merida began a phase 1 trial of its GD drug candidate, MER511, in December. Participants are receiving intravenous or subcutaneous doses of MER511. In GD, autoantibodies bind thyroid-stimulating hormone receptors (TSHRs). The autoantibodies trigger thyroid hormone production, accelerating metabolic activity, and can lead to thyroid eye disease (TED). Targeting the autoantibodies could treat the underlying cause of GD while preserving healthy signaling. Initial phase 1 data show robust reductions in thyroid-stimulating antibodies, Lilly said. Current GD treatments include medicines that stop the thyroid from making hormones or block their activity. Amgen’s Tepezza is among the treatment options for patients who develop TED. Investigational GD treatments include FcRn blockers from argenx and Immunovant and a BTK inhibitor, rilzabrutinib, from Sanofi. Biohaven is developing an IgG degrader, BHV-1300, while Lycia Therapeutics has designed LCA-0321 to bind and eliminate TSHR autoantibodies. Merida’s R&D ambitions extend beyond GD. The biotech is moving a treatment for IgE-mediated allergic disease, MER769, and a primary membranous nephropathy program toward the clinic. Lilly noted the potential for MER769 to treat food allergy, asthma and chronic spontaneous urticaria. The deal feeds Lilly’s voracious appetite for assets. Propelled by sales of its GLP-1 drugs, the company has inked a series of buyouts and licensing pacts this year, penning multibillion-dollar deals with biotechs including AtaiBeckley , Centessa Pharmaceuticals and Kelonia Therapeutics .

8月24日强生宣布，美国食品药品监督管理局(FDA)已批准公司的IMAAVY(nipocalimab-aahu)用于治疗成人和12岁及以上儿科患者的温抗体型自身免疫性溶血性贫血(wAIHA)。这是头个获批专门用于wAIHA的疗法。 wAIHA是一种罕见的自身免疫性血液疾病，患者体内的致病性免疫球蛋白 G(IgG)自身抗体错误地附着并破坏红细胞，导致严重贫血、极度疲劳，并显著增加发病和病亡风险。 据悉，IMAAVY于2025年4月还获批用于治疗全身型重症肌无力(gMG)。此外，该药在风湿性疾病、Sjögren综合征、系统性红斑狼疮以及母胎疾病(如胎儿和新生儿溶血病)等领域正在推进临床研究。 强生预计该药峰值销售额可超过50亿美元。 wAIHA是一种罕见但致命的自体抗体驱动疾病，数据显示，全球每年每10万人中约有1至3例新发病例，患病率约为八千分之一。虽然患者群体不大，但是数十年来始终没有一款获批的特异性药物。强生的MAAVY作为一款免疫选择性FcRn阻断剂，它通过阻断新生儿Fc受体来减少致病性IgG自身抗体。 此次FDA批准主要基于 II/III 期 ENERGY 研究(NCT04119050)的积极结果。该研究共随机入组 115 例受试者，主要终点为持续性血红蛋白应答，次要终点包括第 24 周 FACIT 乏力量表较基线的均值变化，以及糖皮质激素剂量变化等。 在wAIHA领域，还有多家药企也在积极开发中。 如赛诺菲于今年2月宣布，FDA已授予rilzabrutinib(瑞扎布鲁替尼)突破性疗法认定，用于治疗温型自身免疫性溶血性贫血(wAIHA)。该产品是一种口服可逆性布鲁顿酪氨酸激酶(BTK)抑制剂，有望通过多重免疫调节恢复免疫平衡，成为治疗多种罕见免疫介导或炎症性疾病的有效新药。FDA授予rilzabrutinib突破性疗法认定，是基于正在进行的开放标签2b期LUMINA 2研究的数据。该研究评估了口服可逆性布鲁顿酪氨酸激酶抑制剂rilzabrutinib在wAIHA患者中的疗效、安全性和药代动力学。研究结果显示，rilzabrutinib治疗可带来持久的血红蛋白反应，且该反应在长期随访中得以维持。基于这些结果，赛诺菲启动了3期LUMINA 3试验。 在中国，和黄医药于今年4月宣布，索乐匹尼布(sovleplenib)用于治疗既往至少对糖皮质激素治疗反应不佳的成人温抗体型自身免疫性溶血性贫血(wAIHA)的患者的新药上市申请已获中国国家药品监督管理局受理，并获纳入优先审评。资料显示，索乐匹尼布是一种开发用于治疗自身免疫性疾病和血液恶性肿瘤的新型、高选择性的口服脾酪氨酸激酶(「Syk」)抑制剂。 展望未来，随着更多临床数据积累、注册审批推进，以及药物可及性政策落地，wAIHA 患者有望摆脱过去 “缺药可用” 的困境，迎来属于罕见血液病的靶向治疗新时代。 文章参考：制药网 👉扫码进入会议小程序  查看同期所有论坛全议程 报名后可解锁大咖约见名额 👉扫码进入会议小程序  查看同期所有论坛全议程 报名后可解锁大咖约见名额 本届展会彻底重构往届对接模式，依托全新升级的1V1约见系统，打造会前、会中、会后全流程闭环的深度对接服务，直击“找品种、找资本、找供应链、找海外渠道”匹配难、沟通浅的行业痛点： 重磅企业矩阵 特设10间专属对接房，每间入驻一家行业标杆企业。助您一站式锁定产业链关键节点。 *重磅企业持续增加中，敬请期待... 甲方需求直连 现场发布真实甲方企业需求清单，涵盖原料药采购、创新管线授权、CXO合作、投融资及设备耗材采购等方向，让合作诉求一目了然。 *上下滑动查看 *以上为部分公司呈现，排名不分先后... 嘉宾面对面洽谈 汇聚产业决策层与学术带头人，为您提前勾勒现场高端人脉网络。 *嘉宾排名不分先后，按姓名首字母排序，持续增加中... 自由约见，高效会晤 所有参会人员均可通过1V1约见系统自由发起洽谈邀约。系统基于智能匹配机制，特别针对合成生物学、CGT、中药、宠物药等热门细分赛道设立专属匹配通道，集中汇聚同赛道供需双方，让企业高效锁定垂直领域的关键合作伙伴。到场即直面精准匹配的决策层，大幅提升转化效率，精准实现“找方向、搞增长、寻长期战略伙伴”的目标。  👉扫码进入会议小程序 报名后即可约见