Balstilimab

本周大分子领域的主线是"交易"：诺华以最高78亿美元、赛诺菲以10亿美元预付款、阿斯利康以20亿美元投资，三笔大手笔接连落子。监管端，Teva的地舒单抗生物类似药拿下Xgeva全部适应症，一款不局限于RPE65突变亚群的视网膜色素变性基因治疗获中美试验许可。临床端则几家欢喜几家愁——亨廷顿病基因治疗四年随访未达统计学显著，卵巢癌领域却交出48%三年生存率与45.9%客观缓解率的两份成绩单。一、监管获批：一款生物类似药拿下Xgeva全部适应症1. Teva的Degevma作为Xgeva生物类似药获FDA批准 Teva的抗RANKL单克隆抗体生物类似药Denosumab-adet（Degevma）于 9 月 30 日获FDA批准，覆盖Xgeva全部3项适应症（肿瘤骨并发症等），为癌症骨并发症患者扩大低价治疗可及性。2. iRegene NouvSight001获美国与中国视网膜色素变性试验许可 基因疗法 NouvSight001 于 9 月 29 日获美国与中国临床试验许可，该疗法不局限于 RPE65 突变亚群，潜在覆盖全球约150万视网膜色素变性（RP）患者，突破现有基因疗法仅覆盖 RPE65 突变亚群的限制。3. Wave WVE-006 与 FDA 敲定 RNA 编辑药注册路径 GalNAc 偶联 RNA 编辑寡核苷酸 WVE-006 在 10 月 1 日与 FDA 会议后，计划开展单一项 2 年注册试验；1 年中期分析或可基于生物标志物支持 α-1 抗胰蛋白酶缺乏症（AATD）的加速批准。二、临床数据：亨廷顿病四年随访，卵巢癌交出三年生存率4. UniQure AMT-130 四年数据：放缓仍在，但"显著"没到 AAV 载体基因治疗 AMT-130（亨廷顿病）于 9 月 29–30 日公布 48 个月 cUHDRS 未达统计学显著（外部对照流失所致），但 36 个月 15 例高剂量显示 80% 放缓；FDA基于36个月数据审评其BLA。5. Agenus 双抗抗体组合复发卵巢癌三年生存率 48% Agenus 于 10 月 3 日在 IGCS 2026 公布 C-800-01 试验三年随访：botensilimab（Fc 增强型抗 CTLA-4 抗体）联合 balstilimab（抗 PD-1 抗体）用于复发卵巢癌，按铂敏感状态等分层显示生存获益，三年生存率 48%。三、ADC：一次撤回，两张成绩单6. 默克 / 第一三共撤回 I-DXd 肺癌加速批准申请 B7-H3 ADC（ifinatamab deruxtecan，I-DXd）于 9 月 28 日在 FDA 反馈后自愿撤回小细胞肺癌加速批准申请，失去相对多家快速推进竞品的先发优势。7. Genmab Rina-S 铂耐药卵巢癌客观缓解率 45.9% FRα 靶向 TOPO1 抑制剂 ADC（Rina-S）于 10 月 3 日公布 RAINFOL-01 C 部分结果：109 例铂耐药卵巢癌确认客观缓解率（ORR）45.9%（95% CI 36.3–55.7），含 5 例完全缓解。四、BD 与资本：三笔关键交易同一周落定8. 诺华最高 78 亿美元引进艾博生物 mRNA 编码双抗 诺华 10 月 2 日与 Abogen Biosciences（艾博生物）达成许可与选择权协议，获 mRNA 编码的 CD19×CD3 双特异性 T 细胞衔接器（ABO2203）全球独占许可，用于自身免疫疾病，预付款 5.75 亿美元、总交易额可达 78 亿美元。9. 赛诺菲预付再生元 10 亿美元，续寻 Dupixent 后继者 赛诺菲 10 月 1 日扩大与再生元的长期抗体合作，预付 10 亿美元、总交易额可达 80 亿美元，开发针对 2 型炎症同靶点的长效抗体，共 4 款超出 dupilumab 的长效下一代抗体。10. 阿斯利康 20 亿美元投资 Summit，ADC 联用同步铺开 阿斯利康 9 月 29 日投资 20 亿美元于 Summit Therapeutics（ivonescimab 为 PD-1/VEGF 双抗），获得与多款 AZ 药物（含 ADC）联用的临床合作，消除 Summit 财务不确定性；同期第一三共、阿斯利康与 Summit 就 TROP2 ADC（Datroway）联合 ivonescimab 达成临床试验合作，并规划一线三阴性乳腺癌三期试验。本周其他值得关注 罗氏因中期数据失望砍掉肌肉保留型肥胖药 emugrobart（9/29）；诺和诺德 denecimib（血友病 A 双抗）BLA 审评延期，公司计划 2027 年上半年在美上市（10/3）；礼来 lebrikizumab（抗 IL-13 单抗）16 周时过半手足特应性皮炎患者达"清除或几乎清除"、77% 满意（9/30）；Immunome IM-1340 获 FDA IND 许可（9/30）；罗氏一款小核酸肾病药物三期成功（9/28）；Atara 与 Pierre Fabre 在 FDA 立场再度变化后重新递交 tabelecleucel；Artiva 通用型 NK 疗法 AlloNK 将展示类 CAR-T 的 B 细胞耗竭数据；Capricor deramiocel 三期 HOPE-3（DMD）数据窗口内公布；BioNTech 关厂裁员、重心转回肿瘤（9/30）；又一家小核酸公司 IPO，首日市值约 20 亿美元（9/29）；拜耳 22 亿美元在美国 New Albany 建新生产基地（10/2–10/3）；Italfarmaco 与 JCR 就抗 IGF1R 抗体 JR-141（Hunter 综合征）达成美欧拉美独家许可（10/3）；Lonza 与 Asimov 合作体内慢病毒载体制造；Mirum brelovitug 丁肝三期阳性；Dyno 肌肉靶向衣壳、CaseBioscience 加入 730 万美元 ARPA-H 细胞保存团队。

Survival estimate remained at 48% between years two and three, extending evidence of durable benefit Results observed in heavily pretreated, difficult-to-treat population that received a median of four prior lines of therapy; nearly three-quarters of patients had platinum-resistant or refractory cancer LEXINGTON, Mass.--(BUSINESS WIRE)-- $AGEN #AGEN -- Agenus Inc. (Nasdaq: AGEN) , a leader in immuno-oncology innovation, today announced three-year follow-up results from the ovarian cancer cohort of its 400+ patient Phase 1b C-800-01 trial. The study evaluated botensilimab (BOT), Agenus’ multifunctional, Fc-enhanced anti-CTLA-4 antibody, in combination with balstilimab (BAL), Agenus’ anti-PD-1 antibody. The results, including three-year survival and outcomes by sensitivity to platinum-based chemotherapy and by prior treatment, were presented at the 2026 International Gynecologic Cancer Society (IGCS) Annual Global Meeting in Montréal, Canada. Among patients evaluable for efficacy, three-year overall survival was 48% (n=35), unchanged from the two-year estimate, suggesting durable clinical benefit in this population. Responses and long-term survival were observed in both platinum-sensitive and platinum-resistant or refractory disease. At the last follow-up, 25% of patients who received at least one dose of treatment (11 of 44) were alive and off all therapy. These results come from a population with substantial prior treatment. Patients had received a median of four prior lines of therapy, and 66% had received at least four. All had previously received platinum chemotherapy, 77% had received bevacizumab and 57% had received a PARP inhibitor. Nearly three-quarters had platinum-resistant or refractory disease, meaning their cancer had progressed during platinum treatment or returned within six months of it. “In recurrent ovarian cancer, conventional checkpoint immunotherapy has rarely produced lasting benefit, particularly after the disease stops responding to platinum chemotherapy,” said Rebecca L. Porter, MD, of Dana-Farber Cancer Institute, who presented the data . “What stands out with longer follow-up is the survival plateau in patients who had already received multiple treatments. That pattern suggests that a different approach to activating the immune system may offer some patients lasting benefit, even when treatment options have narrowed.” Three-year follow-up findings Overall efficacy results (n=35) Overall survival (OS): Estimated survival was 48% at both two and three years. Median OS was 14.8 months. Objective response rate (ORR): 23% of patients (8/35) had a response, including one complete response and seven partial responses. Duration of response: Responses lasted a median of 9.7 months. Clinical benefit: 31% of patients (11/35) had a response or stable disease lasting at least 24 weeks. Alive and off treatment: At last follow-up, 25% (11 of all 44 patients who received at least one dose of treatment) were alive and off all therapy. Results by patient subgroup Platinum-resistant or refractory disease (n=25): Estimated three-year survival was 47%. ORR was 20% (5/25), including one complete and four partial responses. Platinum-sensitive disease (n=10): Estimated three-year survival was 45%. Thirty percent (3/10) of patients had a partial response. Four or more prior lines of therapy (n=23): Estimated three-year survival was 48%. ORR was 17% (4/23), with all four responses being partial responses. Disease progression on a PARP inhibitor (n=17): I n patients whose cancer had progressed during PARP inhibitor treatment or maintenance, estimated three-year survival was 61%. Two patients (12%) had partial responses. Safety remained consistent with the known BOT+BAL profile. No new safety signals or treatment-related deaths were reported. “The most compelling finding is that the survival curve extends beyond two years in women who had already received multiple treatments, including those with platinum-resistant or refractory disease,” said Steven J. O’Day, MD, Chief Medical Officer of Agenus . “We have seen a similar pattern of durable survival in other difficult-to-treat cancers studied with BOT+BAL. This three-year ovarian update strengthens our conviction that the combination can deliver meaningful, lasting benefit to a subset of patients who have had few effective immunotherapy options.” The three-year ovarian results add to a pattern of long-term survival across difficult-to-treat cancers studied with BOT+BAL. In the broader 400+ patient Phase 1b pan-tumor analysis, estimated two-year survival was 39%. In a separate cohort of patients with refractory MSS metastatic colorectal cancer without active liver metastases, estimated survival was 41% at two years and 33% at three years. These findings are particularly notable in cancers where conventional immunotherapy has historically offered limited benefit. The consistent and durable BOT+BAL activity in heavily pretreated cancers that are poorly immunogenic or resistant to prior immunotherapy, reinforced by deep pathologic responses in neoadjuvant studies, strengthens the rationale for moving treatment earlier in the course of disease. Before surgery, when the primary tumor and surrounding lymph nodes remain intact, BOT+BAL may have the greatest opportunity to generate lasting antitumor immunity, reduce recurrence and ultimately increase the number of patients who may be cured. The full presentation can be found on the publications section of the Agenus website at . BOT and BAL are in development and have not been approved by the U.S. Food and Drug Administration. Presentation details Abstract Title: Next Generation Immune Checkpoint Inhibition with Botensilimab Plus Balstilimab in Recurrent Ovarian Cancer: Results by Platinum Sensitivity Status and 3-Year Overall Survival Presenter: Rebecca L. Porter, MD; Dana-Farber Cancer Institute, Boston, MA, USA Poster Presentation number: 969 Session Title: Poster Rounds with Professors Session, Ovarian Cancer 03 Session Date/Time: Saturday, October 3, 2026 | 10:50am-10:55am EDT About the C-800-01 Study C-800-01 (NCT03860272) is a multicenter Phase 1b trial evaluating BOT with or without BAL in advanced solid tumors. The ovarian cancer cohort included patients with recurrent disease for whom no standard therapy was available or whose cancer had progressed after standard treatment. The efficacy analysis included 35 patients who had at least one tumor scan after starting therapy. The safety population included all 44 patients who received treatment. The data cutoff for this analysis was December 13, 2025. About Agenus Agenus is a leading immuno-oncology company targeting cancer with a comprehensive pipeline of immunological agents. The company was founded in 1994 with a mission to expand patient populations benefiting from cancer immunotherapy through combination approaches, using a broad repertoire of antibody therapeutics, adoptive cell therapies (through MiNK Therapeutics) and adjuvants. Agenus has robust end-to-end development capabilities, across commercial and clinical cGMP manufacturing facilities, research and discovery, and a global clinical operations footprint. Agenus is headquartered in Lexington, MA. For more information, visit or @agenus_bio. Information that may be important to investors will be routinely posted on our website and social media channels. Agenus’ Commitment to Patient Access Until marketing authorization is granted, BOT+BAL is accessible only through clinical trials and authorized early access mechanisms where permitted and available under each country’s regulatory framework. For eligible French patients treated in hospitals under AAC and meeting the predefined criteria, BOT+BAL is fully reimbursed by France’s national health system. Outside France, access may be available in select countries through paid named-patient programs, which may involve out-of-pocket costs to the patient or coverage under applicable national or private reimbursement arrangements. These programs are physician-driven and are not promotional. About Botensilimab (BOT) Botensilimab (BOT) is a human multifunctional, Fc-enhanced anti-CTLA-4 antibody designed to boost both innate and adaptive anti-tumor immune responses. Its novel design leverages mechanisms of action to extend immunotherapy benefits to “cold” tumors which generally respond poorly to standard of care or are refractory to conventional PD-1/CTLA-4 therapies and investigational therapies. BOT augments immune responses across a wide range of tumor types by priming and activating T cells, reducing intratumoral regulatory T cells, activating myeloid cells and inducing durable memory responses. Approximately 1,500 patients have been treated with BOT and balstilimab (BAL) in phase 1 and phase 2 clinical trials. The combination with Agenus’ investigational PD-1 antibody, BAL, has shown clinical responses across more than nine cancers. For more information about BOT trials, visit . About Balstilimab (BAL) Balstilimab is a novel, fully human monoclonal immunoglobulin G4 (IgG4) designed to block PD-1 (programmed cell death protein 1) from interacting with its ligands PD-L1 and PD-L2. It has been evaluated in more than 900 patients to date and has demonstrated clinical activity and a favorable tolerability pro several tumor types. Forward-Looking Statements This press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws, including statements regarding its botensilimab and balstilimab programs, expected regulatory timelines and filings, and any other statements containing the words "may," "believes," "expects," "anticipates," "hopes," "intends," "plans," "forecasts," "estimates," "will," “establish,” “potential,” “superiority,” “best in class,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially. These risks and uncertainties include, among others, the factors described under the Risk Factors section of our most recent Annual Report on Form 10-K for 2025, and subsequent Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commission. Agenus cautions investors not to place considerable reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this press release, and Agenus undertakes no obligation to update or revise the statements, other than to the extent required by law. All forward-looking statements are expressly qualified in their entirety by this cautionary statement. Contacts Investors 917-362-1370 | investor@agenusbio.com Media 781-674-4422 | communications@agenusbio.com