Giredestrant

GLOBAL PHARMA DAILY 全球药闻每日精选 2026年09月13日 · 收录于合集「全球药闻」 今日要点 ① FDA 批准首个靶向 SMA 肌肉流失疗法 Isembyld 注射剂 ② Scholar Rock 首个 SMA 肌萎缩靶向药 Isembyld 获 FDA 批准 ③ 阿斯利康口服 SERD Etcamah 一线乳腺癌试验失败，口服 SERD 赛道再添败绩 01 美洲 · AMERICAS 01 Ionis ASO 疗法获 FDA 突破性认定，强化罕见病管线支柱地位 来源：FiercePharma · fiercepharma.com 基因治疗罕见病FDA突破性认定 摘要：Ionis Pharmaceuticals 研发的针对 Alexander 病的反义寡核苷酸（ASO）疗法获得 FDA 突破性疗法认定，该病是一种罕见的遗传性神经系统疾病。此次获批标志着 Ionis 在罕见病领域的核心管线再添重要支柱，为该疾病的患者提供首个靶向治疗选择。Ionis 作为寡核苷酸药物领域的先行者，持续验证其 RNA 靶向治疗平台在罕见病中的商业化潜力。 BD 观察 BD 观察：Ionis ASO 疗法 FDA 突破性认定进一步巩固寡核苷酸赛道热度，提示中国药企可关注 RNA 靶向治疗平台的 License-in 机会及罕见病适应症布局。 02 BrainChild Bio 获 1.16 亿美元 C 轮，推进儿童脑瘤 CAR-T 疗法关键临床试验 来源：Endpoints News · endpoints.news CAR-T儿童肿瘤基因治疗 摘要：美国西雅图生物制药公司 BrainChild Bio 近日完成 1.16 亿美元大规模融资。该公司致力于将 CAR-T 细胞疗法开发为适用于脑肿瘤的小分子式疗法，降低治疗复杂性。本轮融资将用于支持其儿童脑瘤 CAR-T 疗法的关键性临床试验（pivotal trial），旨在推动该候选药物进入商业化阶段。 BD 观察 BrainChild Bio 聚焦 CAR-T 脑肿瘤递送突破，$116M 融资反映资本市场对实体瘤 CAR-T 疗法的持续看好。中国 ADC/CAR-T 赛道虽热，但脑瘤靶向递送仍是空白，可关注技术授权机会或差异化布局。 03 Roivant 肺动脉高压药数据惊艳，BMS 首创新型 CAR-T 疗法引领行业风向 来源：BioPharma Dive · biopharmadive.com CAR-T疗法肺动脉高压创新药 摘要：Roivant 公司肺动脉高压候选药物临床数据表现卓越，分析师预测未来销售额可能突破 100 亿美元。Bristol Myers 积极推广其首创新型 CAR-T 疗法。AstraZeneca 成功克服负面委员会投票障碍，Pharvaris 在关键研究中取得积极成果。 BD 观察 该 CAR-T 疗法代表细胞治疗前沿突破，中国药企可关注类似靶点 License-in 机会，或借鉴 Roivant 数据驱动策略加速研发布局。 04 Moonwalk Biosciences 融资 7000 万美元推进 siRNA 肥胖疗法临床开发 来源：Endpoints News · endpoints.news siRNA肥胖治疗基因疗法 摘要：Moonwalk Biosciences 宣布完成 7000 万美元融资，用于推进其 siRNA 肥胖治疗项目进入临床试验。该公司正调整基因疗法策略，从传统方法转向更高效的 siRNA 递送平台。这是肥胖赛道持续吸引资本关注的又一例证，siRNA 技术凭借靶向性强、半衰期长等优势成为热门选择。公司将利用本轮资金加速临床开发进程。 BD 观察 BD 观察：siRNA 肥胖疗法获资本青睐，提示中国药企关注该赛道差异化机会，尤其关注递送系统创新及临床数据验证进展。 05 FDA 批准首个靶向 SMA 肌肉流失疗法 Isembyld 注射剂 来源：FDA Drugs · fda.gov FDA批准SMA肌肉流失 摘要：FDA 今日批准 Isembyld（apitegromab-mstn）注射剂上市，用于治疗正在接受 SMN2 靶向治疗的脊髓性肌萎缩症（SMA）成人及 2 岁以上儿童患者。这是全球首个针对 SMA 肌肉流失适应症的疗法，为 SMA 治疗领域带来全新机制突破，填补了现有 SMN2 靶向药物无法改善肌肉萎缩的空白。 BD 观察 BD 观察：该疗法靶向肌肉生成素通路，机制独特，对国内 SMA 赛道构成潜在竞争压力；建议关注同类抗 MSTN 抗体管线进展及中国 SMA 患者用药可及性缺口。 06 Scholar Rock 首个 SMA 肌萎缩靶向药 Isembyld 获 FDA 批准 来源：STAT News · statnews.com SMAFDA批准myostatin抑制剂 摘要：Scholar Rock 公司旗下 myostatin 抑制剂 Isembyld（apitegromab）获 FDA 批准，用于治疗 2 岁及以上脊髓性肌萎缩（SMA）患者。该药联合现有 SMN2 靶向疗法使用，可与后者的作用机制形成互补。关键 II 期数据显示，联合用药一年后患者运动技能显著改善，而安慰剂组出现功能下降，差异具有统计学意义。Scholar Rock CEO 称这是 SMA 治疗领域数十年来首次成功验证 myostatin 抑制策略。 BD 观察 BD 观察：Isembyld 是中国已有 SMN2 靶向疗法（诺西那生/利司扑兰等）的潜在联合用药标的，其 myostatin 抑制新机制为中国生物企 License-in 或联合开发提供了差异化策略窗口。 07 阿斯利康口服 SERD Etcamah 一线乳腺癌试验失败，口服 SERD 赛道再添败绩 来源：FiercePharma · fiercepharma.com 口服SERD乳腺癌临床试验失败 摘要：阿斯利康口服 ER 降解剂 Etcamah 在 HR+/HER2-乳腺癌一线治疗 III 期临床试验中未达主要终点，正式加入罗氏竞品 giredestrant 同一失败的行列。该结果对口服 SERD 类别构成重大打击，引发业界对该管线开发策略的重新审视。目前口服 SERD 赛道正面临口服制剂稳定性、疗效不足及联合用药方案优化等多重挑战。 BD 观察 BD 观察：Etcamah 失败印证口服 SERD 一线疗法开发难度超预期，中国口服 SERD 布局企业（如海思科、恩华药业）需重新评估该管线差异化竞争策略及联合用药开发路径。 08 阿斯利康乳腺癌药 camizestrant 关键试验失败，加速获批适应症市场受限 来源：STAT News · statnews.com 乳腺癌临床试验失败阿斯利康 摘要：阿斯利康宣布，其乳腺癌口服药 camizestrant（商品名 Etcamah）在 III 期关键试验 SERENA-4 中未达主要终点，未能证明在 ER 阳性/HER2 阴性晚期乳腺癌一线治疗中优于标准疗法。该药已于本月获 FDA 加速批准，用于携带耐药突变的晚期乳腺癌患者。此次试验失败意味着药物无法拓展至更广泛的一线治疗市场，商业化潜力受限。 BD 观察 BD 观察：AstraZeneca SERDE 类口服抑制剂首战受挫，提示 ERα靶向联合策略竞争激烈，中国同类管线需审慎评估临床差异化价值。 END 声明：本文由 AI制剂研发 整理汇编，原始报道版权归各来源媒体所有。个人观点，仅供参考。 AI制剂研发 AI 驱动的全球制药情报日更 AI制剂研发 · AI 驱动的全球制药情报日更

AstraZeneca's Etcamah (camizestrant) has fallen short in a Phase III study testing whether the oral selective oestrogen receptor degrader (SERD) could outperform an aromatase inhibitor when used as an upfront treatment for advanced ER-positive, HER2-negative breast cancer.In the SERENA-4 trial, AstraZeneca said Etcamah plus Pfizer's Ibrance (palbociclib) produced a "numerical improvement" on the primary endpoint of progression-free survival versus anastrozole plus Ibrance, but the difference wasn't statistically significant.The result comes a week after the FDA granted Etcamah accelerated approval for use as part of biomarker-triggered switching strategy in which patients with HR-positive, HER2-negative, locally advanced or metastatic breast cancer are given the drug when an ESR1 mutation emerges during aromatase inhibitor (AI) plus CDK4/6 inhibitor therapy (see – Physician Views Preview: Can AstraZeneca's Etcamah carve out a new treatment paradigm in breast cancer?). That decision was based on data from the Phase III SERENA-6 study."Whilst we are disappointed by the SERENA-4 outcome, it sharpens our focus on maximising the number of patients who can benefit from Etcamah today based on SERENA-6 and reinforces the importance of ESR1 testing for patients on first-line therapy," said Susan Galbraith, EVP for Oncology Haematology R&D at AstraZeneca, in a release Friday."Early breast cancer represents an important opportunity, and we remain confident in the long-term potential of Etcamah in the early setting as we advance our broader programme," she added.Echoes of Roche rivalSERENA-4 enrolled 1371 adult patients with newly diagnosed Stage IV de novo or recurrent disease who had not received systemic treatment for advanced disease. The trial was testing whether replacing the AI component of standard first-line treatment with Etcamah could improve outcomes, with all patients also receiving Ibrance. Secondary endpoints include overall survival and PFS2.AstraZeneca said the safety profile was consistent with the known profiles of the individual drugs, with no new safety concerns, and that full data would be shared in due course.The result puts Etcamah in similar territory to Roche's rival oral SERD giredestrant, which also failed to significantly improve PFS in the Phase III persevERA trial toplined in March. That study compared giredestrant plus Ibrance with letrozole plus Ibrance in 992 patients receiving first-line treatment; it had also shown a numerical PFS improvement, but missed statistical significance.At this year's American Society of Clinical Oncology meeting, Roche reported median PFS was 33.1 months with giredestrant versus 28.2 months with letrozole, corresponding to a PFS benefit of 11% in favour of giredestrant (see – KOL Views Q&A: Breast cancer at ASCO – first take on AZ and Roche oral SERD debates, Gilead's Trodelvy TNBC data).'Manageable setback'RBC Capital analyst Trung Huynh called the SERENA-4 results a "manageable setback" that largely removes a near-term uncertainty without fundamentally altering the long-term investment case for Etcamah.James Gordon of Barclays likewise described the update as "well anticipated by the market," citing the earlier Roche failure as well as AstraZeneca's cautious commentary on SERENA-4.Earlier this year, Galbraith had highlighted a number of factors that could favour SERENA-4 over persevERA, including a first-line population enriched for endocrine sensitivity and a much larger patient population "sized for an effect size that will still be clinically meaningful in that population." For more, see – Spotlight On: First-line and adjuvant opportunities still beckon for AstraZeneca's camizestrant despite FDA setback.Meanwhile, AstraZeneca is also continuing to test Etcamah in earlier-stage disease in the Phase III CAMBRIA-1 and CAMBRIA-2 trials, which together are enrolling about 10,000 patients. The trials are designed for patients at both intermediate and high risk of recurrence in the adjuvant setting, evaluating Etcamah as monotherapy, together with CDK4/6 inhibitors and following CDK4/6 inhibitor treatment.According to Jefferies analysts, AstraZeneca estimates the drug could generate more than $5 billion in peak annual sales, with early breast cancer expected to account for most of that opportunity.