Pozelimab

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。 8月26日，再生元（Regeneron Pharmaceuticals）宣布，C5 siRNA新药Cemdisiran治疗全身型重症肌无力（gMG）的三期临床BIMBLE达到主要终点和关键次要终点。再生元计划在与FDA沟通后，于2026年第一季度提交cemdisiran单药的监管申请。 分析显示，每三个月皮下注射一次的cemdisiran单药显示出平均74%的补体活性抑制率。而cemdisiran与C5抗体pozelimab的联合疗法则达成接近99%的补体活性抑制。此外，cemdisiran单药在日常生活活动量表（MG-ADL）总分（主要终点）方面，实现了相较安慰剂2.30分的改善（p=0.0005）。而联合疗法在MG-ADL总分上，则实现了相较安慰剂1.74分的改善（p=0.0086）。 NIMBLE临床3期试验结果摘要 cemdisiran 最初由 Alnylam 研发，再生元于去年修改合作协议后获得授权。根据新协议，Alnylam 收到 1000 万美元首付款，并在 cemdisiran 单药获批时有机会获得额外付款；此外，如 cemdisiran 作为联合疗法上市销售，Alnylam 最高可获得 3.25 亿美元的商业里程碑付款。 Pozelimab为再生元开发的C5抗体，2023年获得FDA批准上市，用于治疗蛋白丢失性肠病(CHAPLE)。cemdisiran为一款C5靶向siRNA疗法，再生元还在探索PNH、地图样萎缩等多个适应症。 参考资料： 公司官网

iStock, beast01 Regeneron’s cemdisiran, used alone or in combination with its complement inhibitor Veopoz, significantly improved activities of daily living in patients with generalized myasthenia gravis.   Regeneron’s investigational small interfering RNA therapy cemdisiran significantly improved disease activity in certain patients with generalized myasthenia gravis, opening up a path for regulatory filing early next year. Results from the Phase III NIMBLE study were published Tuesday , demonstrating a 2.3-point placebo-adjusted improvement in Myasthenia Gravis Activities of Daily Living (MG-ADL) scores, a patient-reported scale used to measure the daily impacts of the disease. Cemdisiran also achieved a 74% reduction of complement factor 5 (C5), a part of the complement system that is inappropriately activated in gMG. Regeneron also tested a combination regimen of cemdisiran with the company’s complement inhibitor Veopoz, which elicited a significant MG-ADL improvement of 1.74 points over placebo. Veopoz is approved for the treatment of patients 1 year and up with CD55-deficient protein-losing enteropathy. In premarket trading Wednesday, Regeneron’s stock was up 2.67%. Writing to investors on Tuesday, analysts at Truist Securities noted that cemdisiran  monotherapy  “surpassed our expectations,” given the “historical performance” of other anti-C5 therapies. NIMBLE did not detect instances of meningitis, a known side effect of this drug class, but Truist still expects cemdisiran to come with a boxed warning if approved, “based on the class precedent.” Aside from its apparent efficacy and safety edge, Truist also pointed to cemdisiran’s “convenient administration” as another point for Regeneron. The drug is given subcutaneously every 12 weeks, whereas its most notable competitors—AstraZeneca’s Soliris and Ultomiris—are delivered intravenously. This administration advantage could help cemdisiran “capture a significant share of the C5 gMG market,” according to the analysts. With NIMBLE data in hand, Regeneron is planning an FDA submission for the first quarter of 2026, the pharma noted in its Tuesday release. Full findings from the study will be shared at an upcoming meeting. Designed to target and destroy C5 mRNA, cemdisiran is a small interfering RNA therapy that helps suppress the complement cascade, which in gMG plays a role in damaging muscle nerves. The therapy was developed by Alnylam as part of a research collaboration with Regeneron, for which Regeneron in April 2019 paid $800 million upfront . The deal gave the pharma access to Alnylam’s RNAi experience for diseases of the eyes and central nervous system, a push that has cemdisiran at its core. Milestone payments for this agreement could reach up to $200 million. Elsewhere in the gMG arena, argenx on Monday reported positive Phase III data for its FcRn inhibitor Vyvgart, positioning it up for expansion into patients who are AChR seronegative. While argenx did not provide specific data, it emphasized a “statistically significant and clinically meaningful improvement” in disease activity in patients treated with Vyvgart, adding that it is preparing for a label expansion application into this specific patient population.