Luspatercept-AAMT

2026.10.13 2026年10月13日，来凯医药公告称，其自研 ActRIIA 单抗 LAE102 迎来24周单药治疗研究的最新进展。 图片来源：来凯医药公告 总体而言，核心结果可归纳为三大亮点： 令人鼓舞的减重效果。 观察到肌肉增加及脂肪减少。 为期24周的治疗耐受性良好，未报告任何严重不良事件，亦无腹泻、肌肉痉挛或痤疮等报告，与先前披露的 SAD 及 MAD 研究结果一致。 同时，来凯医药还提及，企业正积极寻求具备全球能力的合作伙伴，以加速 LAE102 的全球开发及商业化进程。 GLP-1 之后，ActRIIA 初露锋芒 就目前阶段而言，全球肥胖治疗领域正处于从“单纯减重”向“体成分优化”升级的关键节点，以 GLP-1 受体激动剂为代表的第一代减重药物虽已验证市场价值，但肌肉流失、代谢反弹等痛点日益凸显。 而 ActRII 通路凭借“减脂增肌”的双重效应，正成为下一代减重药物研发的核心赛道。 机制上，激活素 Ⅱ 型受体（ActRII）属于转化生长因子 β（TGF-β）受体超家族，主要包括 ActRIIA 和 ActRIIB 两种亚型，广泛表达于脂肪细胞与骨骼肌细胞表面，是调控机体能量代谢与肌肉稳态的关键信号枢纽。 靶点策略方面，ActRII 主要沿“受体侧阻断—配体侧中和—配体激活加工拦截”三条链路展开： 配体陷阱：可溶性 ActRIIA/ActRIIB 胞外域-Fc融合蛋白在细胞外广谱中和 Activin/Myostatin/GDF11 等配体，代表资产有默沙东的 Sotatercept 与 BMS 的 Luspatercept，在 PAH、骨髓增生异常综合征等适应症上已获批上市。 受体侧阻断：双特异性抗体同时结合 ActRIIA 与 ActRIIB（如 Bimagrumab），实现轴向全覆盖但需精细安全窗管理；或受体亚型选择性抗体（如 LAE102/LAE103）追求选择性与安全性平衡。 选择性配体阻断：仅靶向 Myostatin 或其潜伏型激活加工（如 Apitegromab/SRK-439），完全避开 Activin A 相关风险，安全窗最优但单一机制的效力天花板受限。 在肌少症/肥胖/代谢综合场景中，受体侧阻断与双配体阻断并行探索： 在脂肪细胞中，阻断激活素（Activins）等配体与 ActRII 受体结合，抑制脂质合成与储存，促进脂肪分解与白色脂肪棕色化，提升基础能量消耗，针对性减少内脏脂肪。 在骨骼肌细胞中，解除激活素、肌肉生长抑制素（Myostatin）等配体通过 ActRII 受体抑制肌肉蛋白合成作用，促进骨骼肌蛋白合成，增加瘦体重，改善肌肉功能。 这种“减脂不减肌、增肌促代谢”的双重效应，完美解决了 GLP-1 受体激动剂减重过程中约30%为瘦体重损失的行业难题，不仅能提升减重质量，更有助于维持长期代谢稳态，降低停药反弹风险。 更关键的是，临床前研究已证实，ActRIIA 单抗与 GLP-1 受体激动剂联用时可产生显著协同效应，在进一步放大减脂效果的同时，有效逆转 GLP-1 诱导的肌肉流失，实现“高效减脂+精准保肌”的最优组合。 ActRIIA 玩家 目前，ActRII 通路的巨大潜力吸引了全球药企争相布局，形成了 MNC 引领验证、Biotech 差异化突破的竞争格局。 其中，早期赛道内主要由 MNC 重金押注，但在 GLP-1 出现前，绝大多数研究方向集中于肌肉萎缩性疾病（如包涵体肌炎、杜氏肌营养不良等），逻辑即“缺啥补啥”，直到后来 GLP-1 爆火之后，其才重新于 MNC 管线中焕发新生。 礼来：2023年以超19亿美元收购 Versanis Bio，拿下核心产品 Bimagrumab（ActRIIA/B 双靶点单抗），成为该赛道的标杆。Ⅱ 期临床显示，Bimagrumab 治疗48周后患者全身脂肪含量平均下降20.5%，肌肉质量增加3.6%；其与替尔泊肽的联合疗法更是被寄予厚望，有望刷新减重疗效天花板。 阿斯利康：2026年4月正式启动 ActRII 抗体 AZD1043 的 I 期临床，快速切入这一赛道。 与 MNC 布局谨慎的态度相反，海内外 Biotech 则更像是技术疗法探索者，尤其是近年来相关管线数量更是大幅上涨。 来凯医药：凭借 LAE102 成为全球少数掌握选择性 ActRIIA 单抗技术的 Biotech，且已与礼来达成合作开展美国 I 期临床，研发进度处于全球第一梯队。 中国生物制药：TQF6422（ActRIIA/B 单抗）于2026年8月获 NMPA 临床试验批准，主打“减脂增肌”双重功效。 宜明昂科：IMC-003（ActRIIA-Fc 融合蛋白）聚焦肺动脉高压（PAH）适应症，临床前数据显示其结合活性优于已上市的 Sotatercept。 海外 Biotech：Arrowhead 等公司则从 siRNA 角度切入，通过沉默 INHBE 基因降低激活素 E 水平，实现内脏脂肪减少，但增肌效果弱于单抗类药物。 其中，来凯医药是全球为数不多对 ActRII 通路进行系统性、货架式布局的生物医药公司，围绕 ActRIIA、ActRIIB 及双靶点均构建了差异化产品矩阵（LAE102、LAE103 与 LAE123），实现了对不同适应症、不同人群的全方位覆盖。 图片来源：来凯医药官网 小结 综上所述，来凯医药 LAE102 24周单药治疗研究的积极进展，既验证了 ActRIIA 靶点“减脂增肌”的临床价值，也印证了公司在该领域的技术实力。 从行业层面看，ActRII 赛道正处于从概念验证到临床价值兑现的关键时期。随着礼来、诺和诺德等 MNC 的持续投入，以及来凯医药等 Biotech 的差异化突破，“减脂增肌”正从概念走向现实，有望重塑全球肥胖治疗格局。 作为全球少数实现 ActRII 通路系统化布局的生物医药企业，来凯医药通过 LAE102、LAE103、LAE123 的梯度产品矩阵，构建了深厚的竞争壁垒，有望在 ActRII 这一下一代减重赛道上，占据领先地位，并最终推动国内 ActRII 领域从跟跑到并跑甚至领跑的跨越。 博腾股份成立于2005年，为全球药企、生物科技公司及科研机构等提供从临床前研究到药品上市全生命周期的端到端 CDMO 服务，涵盖小分子药物、多肽与寡核苷酸药物、蛋白与偶联药物以及细胞与基因治疗药物的原料药到制剂全流程。截至2026年6月30日，公司拥有全球员工4,400余人，累计服务客户1,500余家，成功交付项目约6,000个。我们始终坚持以客户为中心，致力于提供创新可靠的全球化 CDMO 解决方案，让好药更早惠及大众。 免责声明：文中系整合内容，观点不代表博腾及其所属子公司立场，亦不对本文所提供信息做任何形式的保证。同时，本文不做治疗方案推荐，也不承担因使用或依赖本文信息所产生的任何直接或间接的后果。如需获得治疗方案指导，请前往正规医院就诊。

Leader in Global Drug Development of T Cell Therapies PHILADELPHIA, Oct. 01, 2026 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, today announced the appointment of Noah Berkowitz, M.D., Ph.D., as Chief Medical Officer (CMO), effective September 30, 2026. “We are pleased to welcome Noah to Iovance as we prepare for important development and regulatory milestones for our TIL platform across multiple solid tumor cancers,” said Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer. “Noah brings deep experience in global drug development that has led to regulatory approvals across cancer, blood disorders, and cell therapy. His track record will be instrumental as we work to bring TIL therapy to many more patients in new markets, earlier treatment settings, and additional solid tumor cancers.” As Chief Medical Officer of Arvinas Pharmaceuticals, Dr. Berkowitz led the clinical strategy that resulted in the first-ever U.S. Food and Drug Administration (FDA) approval of a targeted protein degradation therapy and expanded the company’s clinical programs across cancer, neurological, and rare diseases. Previously, Dr. Berkowitz served as Senior Vice President and Therapeutic Area Head, Hematology at Bristol Myers Squibb, where he oversaw the full scope of drug development for the company’s benign and malignant hematology programs and, on an interim basis, its cell therapy programs. His global development teams achieved regulatory approvals for the chimeric antigen receptor (CAR) T cell therapies Abecma and Breyanzi, two of the first cell therapies approved for multiple myeloma and large B cell lymphoma, respectively. Clinical programs under his leadership helped drive recent annual sales of Reblozyl above $2 billion. Earlier, Dr. Berkowitz held leadership positions of increasing responsibility in clinical development across multiple cancer indications at Novartis. Dr. Berkowitz completed a clinical investigation fellowship in medical oncology at the National Cancer Institute (NCI). He holds an M.D. and Ph.D. in molecular immunology from Columbia University. “I am excited to join Iovance at this critical inflection point to explore broader applications for TIL technology in new indications and treatment settings,” stated Dr. Berkowitz. “The Iovance TIL platform demonstrates remarkable clinical results and represents the new frontier across solid tumors, including next-generation approaches. I look forward to contributing to the Iovance vision to expand the reach of this transformative treatment to patients who need it most.” In connection with Dr. Berkowitz’s appointment, Iovance approved the grant of inducement stock options and restricted stock units covering an aggregate of up to 450,000 shares of Iovance’s common stock to Dr. Berkowitz. The awards were granted on September 30, 2026 (the date of grant), under Iovance’s Amended and Restated 2021 Inducement Plan, which provides for the granting of equity awards to new employees of Iovance by the Company’s compensation committee in accordance with Nasdaq Listing Rule 5635(c)(4). Each of the stock options granted has an exercise price of $14.82, the closing price of Iovance’s common stock on the date of grant. Each stock option and each restricted stock unit vests over a three-year period, with one-third of the shares vesting on the first anniversary of Dr. Berkowitz’s start date (the first vesting date) and the remaining shares vesting in eight quarterly installments over the next two years, commencing with the first quarter following the first vesting date. In addition, certain of these restricted stock units may vest upon the achievement of performance milestones based on specified regulatory results rather than time-based vesting. The foregoing vesting is subject to continued employment with Iovance through the applicable vesting dates. About Iovance Biotherapeutics, Inc. Iovance Biotherapeutics, Inc. is the global leader in innovating, developing, and delivering tumor infiltrating lymphocyte (TIL) cell therapies for patients with solid tumors. Amtagvi® (li ) is the first FDA-approved, one-time treatment for previously treated advanced melanoma, now approved in three global markets and available at nearly 100 authorized treatment centers (ATCs). The Iovance TIL platform spans registrational trials and next-generation programs in additional solid tumors, including gene-edited and IL-12 tethered TIL therapies, next-generation IL-2, and precision immuno-oncology approaches. As the first and only company to take TIL therapy from concept to a broadly accessible commercial treatment, Iovance operates as an end-to-end cell therapy company, anchored by fully owned, centralized U.S.-based manufacturing that is scaled to serve thousands of cancer patients worldwide each year. Headquartered in Pennsylvania with offices and laboratories in California and Florida, Iovance serves patients at ATCs and clinical sites across almost the entire U.S. and in many countries around the world. For more information, please visit . Amtagvi® and its accompanying design marks, Proleukin ® , Iovance ® , and IovanceCares™ are trademarks and registered trademarks of Iovance Biotherapeutics, Inc. or its subsidiaries. All other trademarks and registered trademarks are the property of their respective owners. Information on Iovance’s broad, industry-leading patent portfolio is available on the Intellectual Property page on . Forward-Looking Statements Certain matters discussed in this press release are “forward-looking statements” of Iovance Biotherapeutics, Inc. (hereinafter referred to as the “Company,” “we,” “us,” or “our”) within the meaning of the Private Securities Litigation Reform Act of 1995 (the “PSLRA”). Without limiting the foregoing, we may, in some cases, use terms such as “predicts,” “believes,” “potential,” “achievable,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “forecast,” “guidance,” “outlook,” “may,” “can,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes and are intended to identify forward-looking statements. Forward-looking statements are based on assumptions and assessments made in light of management’s experience and perception of historical trends, current conditions, expected future developments, and other factors believed to be appropriate. Forward-looking statements in this press release are made as of the date of this press release, and we undertake no duty to update or revise any such statements, whether as a result of new information, future events or otherwise. Forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties, and other factors, many of which are outside of our control, that may cause actual results, levels of activity, performance, achievements, and developments to be materially different from those expressed in or implied by these forward-looking statements. Important factors that could cause actual results, developments, and business decisions to differ materially from forward-looking statements are described in the sections titled “Risk Factors” in our filings with the U.S. Securities and Exchange Commission, including our most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. CONTACTS Investors IR@iovance.com 650-260-7120 ext. 150 Media PR@iovance.com 650-260-7120 ext. 150