Riliprubart

Sanofi recently held its Q1 2026 earnings conference call, during which executives signaled a strategic pivot toward lunsekimig as its next respiratory franchise asset while defending amlitelimab’s path to regulatory submission despite an emerging safety signal. The call was held on April 23, 2026. Key Strategic Signals Lunsekimig Phase III Decision Decoupled from AIRLYMPUS Data: Sanofi confirmed it will not wait for results from the ongoing AIRLYMPUS high-risk asthma study before advancing lunsekimig to Phase III, following positive Phase IIb data in both moderate-to-severe asthma (AIRCULES) and chronic rhinosinusitis with nasal polyps (DUET). Head of R\&D Houman Ashrafian said progression “will solely be subject to internal portfolio decision-making and a regulatory conversation,” signaling high internal conviction. Amlitelimab was explicitly deprioritized in asthma to redirect resources toward lunsekimig. Amlitelimab Regulatory Submission Maintained for H2 2026 Despite Kaposi’s Sarcoma Cases: Sanofi confirmed the ESTUARY Phase III extension data required for filing are expected in H2 2026, with regulatory submissions planned for the same period. Management acknowledged two cutaneous Kaposi’s sarcoma cases across approximately 4,500 patients — one in Phase II ATLANTIS and one in the ESTUARY extension — but said overall malignancy rates were similar to placebo and that physician and payer enthusiasm had not been affected. Venglustat Mixed Phase III Outcome Reshapes Rare Disease Path: Venglustat met its primary endpoint in the LEAP2MONO Phase III study in Type 3 Gaucher’s disease and received U.S. Breakthrough Therapy designation. The PERIDOT Phase III study in Fabry’s disease did not meet its primary endpoint, leaving the ongoing CARAT Phase III as the sole remaining path in that indication. Analyst Pressure Points Amlitelimab Safety Signal Versus Competitor Discontinuation: Analysts questioned why Sanofi’s interpretation of the Kaposi’s sarcoma cases differed from Kyowa Kirin and Amgen, who discontinued their OX40-pathway program on similar grounds. Ashrafian declined to speculate on the competitor’s decision-making, citing low rates of pyrexia, chills and fever relative to other OX40 molecules, the cutaneous and resolving nature of both cases, and balanced adverse event rates across treatment and placebo arms as the basis for Sanofi’s continued confidence in amlitelimab’s benefit-risk profile. Itepekimab COPD Phase III Delay: Analysts pressed management on why the AERIFY Phase III study in COPD had not yet begun recruitment, noting that a competing IL-33 program at AstraZeneca had produced multiple positive Phase III results. Ashrafian acknowledged the program requires regulatory alignment and agreement from partner Regeneron before proceeding, and said the company is “taking the totality of data, not just AstraZeneca’s data” into account. No timeline for a final decision was provided. Forward-Looking Catalysts H2 2026: Amlitelimab regulatory submission in atopic dermatitis is the most near-term major pipeline catalyst, contingent on ESTUARY extension data. A hyaluronidase-based co-formulation of Dupixent with an undisclosed partner is also expected to enter clinical studies in the second half of 2026, representing a key pillar of the loss-of-exclusivity defense strategy ahead of the U.S. compound patent expiry in March 2031. 2027: Phase III readouts for frexalimab in relapsing-multiple sclerosis and secondary-progressive multiple sclerosis, and for riliprubart in chronic inflammatory demyelinating polyneuropathy, are expected next year. Management acknowledged riliprubart’s Phase III recruitment had been slower than anticipated due to screening challenges identified after study initiation, but said enrollment is now accelerating. Leave a Reply Cancel reply Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

点击“蓝字”关注我们 近日，FDA 批准了全球首个完全基于血管化类器官疗效数据进入临床试验的抗癌药物联合疗法，标志着类器官技术在药物研发中的独立性获得监管认可。这一里程碑事件，再次印证了类器官模型在转化医学与精准医疗中不可替代的价值。 随着国内外监管机构对类器官用于非临床研究的政策逐步开放与规范化，这一技术正在彻底释放其在药效评估与毒性测试中的潜力，成为推动创新药，尤其是肿瘤新药研发的加速引擎。 01 国际前沿： 类器官数据支撑IND获批已成趋势 在国际舞台上，类器官平台已成为说服监管机构的有力工具。 2022 年，荷兰 Merus 公司利用大规模患者来源类器官（PDO）库进行高通量筛选，成功发现并验证了双特异性抗体 MCLA-158 在靶向 EGFR 和 LGR5、特异性杀伤结直肠癌等上皮肿瘤干细胞方面的卓越效果。该研究发表于《Nature Cancer》，其类器官药效学数据直接支持了该药物的 IND 获批，展现了类器官在药物发现阶段的强大筛选能力。 在罕见病领域，类器官及器官芯片技术更是解决了动物模型缺失的难题。赛诺菲针对罕见自身免疫性神经病变的药物 SAR445088，基于人源神经细胞构建的脑神经芯片模型所提供的药效与安全性数据，成功获得 FDA 豁免动物试验，直接进入临床。 02 本土实践： 中国药企多元创新， 类器官应用遍地开花 中国药企在类器官技术的应用上同样呈现出多元化与爆发力。 2023 年，恒瑞医药的 HRS-1893 片凭借心脏器官芯片的体外评价数据获批临床，成为国内首个利用微生理系统支持 IND 的案例。同年，艺妙神州的 CAR-T 产品 IM83 注射液，成为中国首个依靠类器官数据支持申报并获批的细胞治疗药物。 2024 年，易慕峰生物的靶向 EpCAM 的 CAR-T 产品 IMC001 在中美获批 IND。其临床前研究利用结直肠癌类器官与正常类器官共培养平台，清晰证明了 CAR-T 细胞对肿瘤的特异性杀伤及对正常组织的低损伤性，为药物的安全性提供了关键证据。 03 范式跃迁： 全球首个完全基于 类器官数据获批的IND案例 2025 年 10 月，韩国 SillaJen 公司的 BAL0819 与免疫检查点抑制剂的联合疗法 IND 修正案获得 FDA 批准。该案例之所以引发行业地震，在于其完全依赖人体血管化类器官的体外药效数据，FDA 豁免了全部传统的动物体内药效实验。 这标志着类器官模型完成了从“辅助工具”到“独立评估标准”的历史性跨越，为整个新药研发行业指明了新的方向——高度拟人化的体外模型数据，足以支撑药物进入人体试验的决策。 可普睿 HycopGel工程化基质胶 如何赋能肿瘤类器官培养 基质胶是类器官三维生长的支架，其成分、纯度、力学性能及批次稳定性直接影响类器官的形成率、形态、功能及实验可重复性。 可普睿生物推出的 HycopGel 工程化基质胶，针对肿瘤类器官培养的痛点进行了多重优化，展现出显著优势： 成分明确，批次稳定：传统 EHS 基质胶成分复杂，批次差异大。HycopGel 通过工程化重组技术，实现对核心功能蛋白（如层粘连蛋白、胶原等）的精准调控，确保每一批次产品性能高度一致，极大提升了实验数据的可靠性与可重复性，满足药物研发的工业化需求。 力学性能可调，适配不同肿瘤：不同来源的肿瘤细胞对微环境硬度的需求不同。HycopGel 允许在一定范围内灵活调整凝胶硬度，研究人员可借此模拟从软组织到较硬组织的生理或病理微环境，助力肺癌、乳腺癌、结直肠癌等多种肿瘤类器官的高效形成与生长。 高生物活性，提升成球效率：产品保留了细胞外基质蛋白的关键活性位点，能更有效地与细胞表面整合素结合，激活下游信号通路，促进细胞聚集、存活与增殖，从而提高肿瘤类器官的成球成功率和生长速度，尤其适用于珍贵临床样本的建模。 低生长因子残留，背景清晰：HycopGel 在生产中严格控制内源性生长因子的残留，为研究外源性药物或生长因子对类器官的作用提供了更“干净”的背景，使药效学数据解读更为清晰准确。 卓越的兼容性与操作性：产品在 4°C 时呈液态，便于与细胞混合；在 37°C 下快速成胶，形成稳定的三维结构。其良好的光学透明度便于后续显微观察与成像分析，与主流类器官培养流程及下游检测技术完美兼容。 往期回顾 从实验室到临床：肿瘤类器官的构建逻辑与未来标准化浪潮 文献解读 | 揭开肿瘤类器官的“硬壳”秘密：基质力学如何操控癌细胞命运？ 文献解读 | 肝硬化微环境如何“助长”肝癌？最新研究揭示ECM组成与硬度对肿瘤行为的关键影响！ 探索更多