Vorasidenib

盖伦奖（Prix Galien Award）由法国药物学家Roland Mehl于1969年创立，是生物医药与医疗技术领域重要奖项，被誉为“医药界的诺贝尔奖”，其抗癌类提名更是全球肿瘤药物创新的风向标。该奖项入围门槛明确：药品或生物技术需近5年内获美国药监局（FDA）批准上市，且能显著改善人类健康。 盖伦奖入围名单既是当下肿瘤治疗技术的“成绩单”，也是未来癌症治疗革命的“路线图”——正通过技术融合（如ADC+放疗）、资源整合（资本与研发协同）、临床范式迭代（精准治疗与联合方案），重塑从实验室到病床的全链条医疗生态。随着基因疗法、纳米机器人等颠覆性技术的介入，未来癌症治疗有望从“延长生命”迈向“功能性治愈”。 2025年美国盖伦奖（Prix Galien USA）已于8月11日公布最终入围名单，一批疗效卓越、能改善患者预后的抗癌“新武器”上榜。全球肿瘤医生网小编更新了上榜名单，不妨看看今年有哪些重磅抗癌新药/新技术上榜，以供癌友们参考。 2025年盖伦奖抗癌领域入围名单盘点，涵盖肺癌、胰腺癌、卵巢癌、脑瘤等十大癌种 抗癌入围名单速览 （一）西达基奥仑赛（CARVYKTI®） 药物简介 ① 药物名称 ：西达基奥仑赛（CARVYKTI®，cilta-cel）。 ② 作用靶点 ：BCMA。 ③ 适应证 ：复发或难治性多发性骨髓瘤。 ④ 研发公司 ：传奇生物、强生创新制药。 ③ 获批时间 ：2024年8月27日（中国）、2024年4月5日（美国）。 药物详情 西达基奥仑赛是一款靶向BCMA的CAR-T细胞疗法，具有多重里程碑意义：它是 首款获美国FDA批准的国产CAR-T疗法 ，也是 我国获批的第6款CAR-T产品，更是目前全球唯一获批用于多发性骨髓瘤二线治疗的BCMA靶向疗法 。 2024年4月5日，获美国FDA批准，用于治疗成人复发或难治性多发性骨髓瘤（R/RMM）；同年8月27日，中国国家药品监督管理局（NMPA）也批准了西达基奥仑赛，用于既往接受过1种免疫调节剂和1种蛋白酶抑制剂治疗的复发或难治性多发性骨髓瘤的治疗。 （二）替吉单抗（TECVAYLI®） 药物简介 ① 药物名称 ：替吉单抗（teclistamab，TECVAYLI®）。 ② 作用靶点 ：BCMA/CD3。 ③ 适应证 ：复发或难治性多发性骨髓瘤。 ④ 研发公司 ：杨森。 ③ 获批时间 ：2022年10月25日。 药物详情 替吉单抗（teclistamab，TECVAYLI®）是强生旗下杨森公司研发的一款BCMA/CD3双特异性抗体药物，2022年10月25日，获美国FDA批准，用于治疗成人复发或难治性多发性骨髓瘤，这些患者曾经接受过四种及以上治疗手段，包括蛋白酶体抑制剂、免疫调节药物和抗CD38单克隆抗体，并且疾病仍然出现进展。。 （三）替吉妥单抗（TALVEY®） 药物简介 ① 药物名称 ：替吉妥单抗（talquetamab，TALVEY®）。 ② 作用靶点 ：GPRC5D/CD3。 ③ 适应证 ：复发或难治性多发性骨髓瘤。 ④ 研发公司 ：杨森公司。 ③ 获批时间 ：2023年8月。 药物详情 替吉妥单抗（talquetamab，TALVEY®）是一种人源化免疫球蛋白G4（IgG4）双特异性抗体，通过与T细胞上的CD3和表达GPRC5D的细胞结合，阻止肿瘤生长并使已建立的肿瘤消退。 2023年8月，获美国FDA加速批准，用于复发或难治性多发性骨髓瘤成人患者的治疗，这些患者既往接受过至少4种疗法，包括蛋白酶体抑制剂、免疫调节剂和抗CD38单克隆抗体。 （四）瑞维美尼（Revuforj®） 药物简介 ① 药物名称 ：瑞维美尼（revumenib，Revuforj®）。 ② 作用靶点 ：menin蛋白。 ③ 适应证 ：急性白血病。 ④ 研发公司 ：Syndax Pharmaceuticals。 ③ 获批时间 ：2024年11月15日。 药物详情 瑞维美尼是一款Menin抑制剂，2024年11月15日获美国FDA批准，用于成人和1岁以上儿童患者KMT2A（赖氨酸甲基转移酶2A基因）易位的复发或难治性急性白血病的治疗。2025 年，FDA 又授予其补充新药申请（sNDA）优先审查资格，用于治疗复发或难治性 NPM1 突变急性髓系白血病，若能顺利获批，其适应症将进一步扩展。 （五）盐酸阿思尼布片（Scemblix®） 药物简介 ① 药物名称 ：盐酸阿思尼布片（Asciminib，Scemblix®，信倍立®）。 ② 适应证 ：Ph阳性慢性粒细胞白血病、Ph阳性慢性髓细胞白血病。 ③ 研发公司 ：诺华。 ④ 获批时间 ：2024年10月29日（美国）、2025年5月14日（中国）。 药物详情 阿思尼布（asciminib）是一种针对慢性髓细胞白血病（CML）的靶向治疗药物，2024年10月29日，获FDA批准，用于成人新诊断的费城染色体阳性慢性粒细胞白血病（Ph+CML）慢性期的治疗。2025年5月14日，该药在中国获批，用于成人新诊断的费城染色体阳性的慢性髓细胞白血病（Ph+CML）慢性期（CP）的治疗。 近日，更是传来好消息，根据国家医保局发布的《2025年国家基本医疗保险、生育保险和工伤保险药品目录及商业健康保险创新药品目录调整工作方案》，阿思尼布有望进入2025新版医保目录。 （六）泽妥珠单抗（BIZENGRI®） 药物简介 ① 药物名称 ：泽妥珠单抗（zenocutuzumab，BIZENGRI®）。 ② 作用靶点 ：HER2/HER3。 ③ 适应证 ：转移性非小细胞肺癌、胰腺腺癌。 ④ 研发公司 ：Merus。 ③ 获批时间 ：2024年12月4日。 药物详情 泽妥珠单抗（zenocutuzumab，BIZENGRI®）是由Merus公司开发的一款HER2/HER3双特异性抗体药物，可针对NRG1融合发挥治疗作用。 2024年12月4日，获美国FDA加速批准上市，用于治疗携带神经调节蛋白1（NRG1）基因融合的晚期、不可切除或转移性非小细胞肺癌（NSCLC）或胰腺腺癌成人患者，这些患者的癌症在之前的治疗中已经进展。它是 FDA批准的首个也是唯一一个专门针对这一患者群体的治疗药物 。 （七）Tarlatamab（IMDELLTRA®） 药物简介 ① 药物名称 ：Tarlatamab（IMDELLTRA®）。 ② 作用靶点 ：DLL3/CD3。 ③ 适应证 ：广泛期小细胞肺癌。 ④ 研发公司 ：安进公司。 ③ 获批时间 ：2024年5月16日。 药物详情 Tarlatamab是一种双特异性T细胞接合剂（BiTE）的免疫治疗方法，可与癌细胞上的DLL3（δ样配体3）、CD3结合，并将患者的T细胞，引导至表达DLL3的癌细胞，从而帮助T细胞识别并摧毁癌细胞。该药是 目前首个也是唯一一款获批用于治疗侵袭性肺癌的DLL3靶向双特异性T细胞接合疗法 。 其中，DLL3是一种抑制Notch信号传导的蛋白质，通常情况下位于正常细胞的内部，但在85%~94%的小细胞肺癌（SCLC）的细胞表面异常表达，使其成为治疗小细胞肺癌的潜在靶点。 2024年5月16日，Tarlatamab（IMDELLTRA®）获美国FDA批准，用于治疗在铂类化疗期间或之后疾病进展的广泛期小细胞肺癌（ES-SCLC）成年患者。 （八）索米妥昔单抗（ELAHERE®） 药物简介 ① 药物名称 ：索米妥昔单抗（ELAHERE®，HDM2002）。 ② 作用靶点 ：FRα（叶酸受体α分子）。 ③ 适应证 ：上皮性卵巢癌、输卵管癌或原发性腹膜癌（化疗耐药者）。 ④ 研发公司 ：华东医药、ImmunoGen。 ③ 获批时间 ：2024年3月22日。 药物详情 索米妥昔单抗是 全球首创的靶向叶酸受体α（FRα）阳性卵巢癌的抗体偶联药物（ADC） ，堪称“智能抗癌导弹”。由于叶酸受体α在76%-89%的上皮卵巢癌中高表达，该药具备明确的攻击靶点。其作用机制精准高效：药物进入肿瘤细胞后，连接子在特定条件下裂解，释放出强效细胞毒性药物美登木素生物碱DM4——如同释放“炸弹”，从细胞内部破坏肿瘤细胞，干扰其正常微管功能，进而诱导肿瘤细胞凋亡。相较于传统化疗，这种方式大幅提升了对肿瘤细胞的杀伤效果，同时减少了对正常细胞的损害。 2024年3月22日，获美国FDA全面批准，用于成人叶酸受体α（FRα）阳性、铂耐药的卵巢上皮、输卵管或原发性腹膜癌的治疗。 该药也是 首个且目前唯一获美国FDA批准的用于治疗此类恶性肿瘤的ADC药物，标志着铂耐药的复发性卵巢癌至此进入ADC治疗新时代 ！ （九）PLUVICTO® 药物简介 ① 药物名称 ：Pluvicto®（Lutetium Lu 177 vipivotide tetraxetan）。 ② 作用靶点 ：PSMA。 ③ 适应证 ：转移性去势抵抗性前列腺癌。 ④ 研发公司 ：诺华。 ③ 获批时间 ：2022年3月23日。 药物详情 Pluvicto®（Lutetium Lu 177 vipivotide tetraxetan）是 首款获美国FDA批准用于治疗转移性去势抵抗性前列腺癌（mCRPC）患者的靶向放射配体疗法 。 作为具有“自动追踪性”的放射性配体疗法药物，其分子中的靶向配体可与前列腺特异性膜抗原（PSMA）阳性的前列腺癌细胞结合——PSMA在超过80%的前列腺癌患者体内可检测到；结合后，药物会将放射性同位素镥-177携带至肿瘤细胞处，释放能量破坏靶细胞，抑制其复制能力并引发肿瘤细胞死亡。 该药于2022年3月23日获FDA批准上市，适应症为PSMA阳性、转移性去势抵抗性前列腺癌（mCRPC）患者。 （十）恩诺单抗（PADCEV®） 药物简介 ① 药物名称 ：恩诺单抗（Enfortumab Vedotin，PADCEV®）。 ② 作用靶点 ：Nectin-4。 ③ 适应证 ：尿路上皮癌。 ④ 研发公司 ：安斯泰来。 ③ 获批时间 ：2019年12月。 药物详情 恩诺单抗是一款靶向Nectin-4的ADC药物，2019年12月，或美国FDA加上批准，用于治疗局部晚期或转移性尿路上皮癌，并于2022年获得欧盟范围内的营销批准。它是 首个获批治疗尿路上皮癌的ADC药物，同时也是首个获批以Nectin-4为靶点的药物 。 （十一）沃拉西地尼（VORANIGO®） 药物简介 ① 药物名称 ：沃拉西地尼（VORANIGO®，vorasidenib）。 ② 作用靶点 ：IDH1/IDH2。 ③ 适应证 ：少突胶质细胞瘤、星形细胞瘤。 ④ 研发公司 ：施维雅（Servier）。 ③ 获批时间 ：2024年8月6日。 药物详情 沃拉西地尼（vorasidenib，VORANIGO®）是由施维雅（Servier）公司研发的一款口服IDH1和IDH2双重抑制剂，是 首个也是唯一一个获得FDA批准的针对2级IDH突变型胶质瘤的靶向治疗药物 。 2024年8月6日，该药获美国FDA批准上市，用于12岁及以上伴有易感IDH1或IDH2突变的2级少突胶质细胞瘤或星形细胞瘤儿童和成人患者，这些患者此前接受过包括活检、次全切或全切在内的手术。 小编寄语 从2025年盖伦奖入围名单可见，癌症治疗正迎来真正的“武器升级”：已从传统放化疗，迈向免疫、靶向、双抗、ADC等多疗法协同发力的新阶段。无论是“一次治疗、五年无复发”的新型CAR-T疗法，还是为罕见基因融合癌量身定制的靶向药，亦或是让晚期患者生存期翻倍的免疫组合疗法，都在不断重新定义癌症治疗的“生死线”。此外，还有多款抗癌新药与新技术正处于火热研发中。 不过需要提醒广大癌友，癌症具有高度突变、极具侵袭性的特点，目前仅靠单一疗法难以达到理想治疗效果。现阶段理想的抗癌方案，需以权威医院与专家的早期规范诊断为基础，在手术、放化疗等传统治疗前提下，结合患者自身状况、疾病特征、经济条件等，灵活联用CAR-T、TCR-T、TIL、NK、CAR-NK等免疫细胞疗法，以及癌症疫苗、靶向药物、硼中子俘获疗法、质子治疗等新药与新技术，从而有效预防癌症复发转移，尽可能延长患者生存期、提升生存质量。 参考资料 [1]Ahn M J,et al.Tarlatamab for patients with previously treated small-cell lung cancer[J]. New England Journal of Medicine, 2023, 389(22): 2063-2075. https://www.nejm.org/doi/full/10.1056/NEJMoa2307980 本文为全球肿瘤医生网原创，未经授权严禁转载

$12.5 million in second quarter PYRUKYND® (mitapivat) net revenues; ended second quarter with $1.3 billion dollars in cash, cash equivalents and marketable securitiesPYRUKYND sNDA for thalassemia under active review, with FDA PDUFA goal date of September 7, 2025Topline results from RISE UP Phase 3 trial of mitapivat in sickle cell disease on track by year-end with potential U.S. commercial launch in 2026Dosed first patient in the tebapivat Phase 2 sickle cell disease trial and received IND clearance for AG-236 CAMBRIDGE, Mass., July 31, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced financial results and updates for the second quarter ended June 30, 2025. “With fewer than 40 days to our PDUFA goal date, our commercial team is prepared for the potential U.S. approval of PYRUKYND for thalassemia,” said Brian Goff, Chief Executive Officer, Agios. “In the second quarter, we made progress advancing our early- and mid-stage pipeline and remain on track to deliver topline results of the RISE UP Phase 3 trial for PYRUKYND in sickle cell disease by the end of the year. Collectively, our progress reflects our continued focus on delivering innovative medicines with the potential to transform the lives of those affected by rare diseases and deliver long-term shareholder value.” Second Quarter 2025 and Recent Corporate Highlights Commercial Performance – PYRUKYND® (mitapivat) Generated $12.5 million in net revenue for the second quarter of 2025, compared to $8.6 million in the second quarter of 2024. 248 unique patients completed prescription enrollment forms, representing an increase of 6 percent over the first quarter of 2025.142 patients are on PYRUKYND therapy, inclusive of new starts and continued therapy, representing an increase of 4 percent over the first quarter of 2025. Entered into a distribution agreement with Avanzanite Bioscience B.V., a rapidly growing European specialty pharmaceutical company focused on rare diseases, to distribute and commercialize PYRUKYND across the European Economic Area, the United Kingdom and Switzerland. R&D Highlights PYRUKYND (mitapivat) Thalassemia – Launch preparations underway ahead of U.S. PDUFA goal date of September 7, 2025. The sNDA for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia remains under active review by the U.S. Food and Drug Administration (FDA).Other regulatory applications remain under review by health authorities in Saudi Arabia, United Arab Emirates, and the European Union. Sickle Cell Disease – Topline results from RISE UP Phase 3 trial of mitapivat in sickle cell disease on track by year-end with potential U.S. commercial launch in 2026. Tebapivat Sickle Cell Disease – Dosed the first patient in the Phase 2 trial investigating tebapivat in sickle cell disease. The trial is enrolling across three dose cohorts (2.5mg, 5mg, 7.5mg) and placebo and the primary endpoint will measure hemoglobin response, defined as a ≥1g/dL increase in hemoglobin concentration from week 10 to week 12, compared to baseline. Lower-risk Myelodysplastic Syndromes (LR-MDS) – Continue to progress patient enrollment in the Phase 2b trial for tebapivat in LR-MDS with target enrollment completion by the end of 2025. Early Pipeline Investigational New Drug (IND) clearance received for AG-236, an siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera (PV). Presented new data on mitapivat and tebapivat at the 30th European Hematology Association Congress. A total of 14 presentations and publications, led by Agios and external collaborators, were shared, covering sickle cell disease, thalassemia, PK deficiency, and MDS. Second Quarter 2025 Financial Results For the quarter ended June 30, 2025, net loss was $112.0 million dollars, compared to a net loss of $96.1 million dollars for the second quarter ended June 30, 2024. Net product revenue from sales of PYRUKYND for the second quarter of 2025 was $12.5 million, compared to $8.6 million for the second quarter of 2024.Cost of sales for the second quarter of 2025 was $1.7 million.Research and Development (R&D) Expenses were $91.9 million for the second quarter of 2025, compared to $77.4 million for the second quarter of 2024. The year-over-year increase was primarily attributed to a $10.0 million regulatory milestone payment to Alnylam associated with our agreement to develop and commercialize AG-236, an siRNA targeting TMPRSS6, intended for the treatment of polycythemia vera.Selling, General and Administrative (SG&A) Expenses were $45.9 million for the second quarter of 2025 compared to $35.5 million for the second quarter of 2024. The year-over-year increase was primarily attributable to an increase in commercial-related activities, including headcount, as the company prepares for the potential approval of PYRUKYND in thalassemia.Cash, cash equivalents and marketable securities as of June 30, 2025, were $1.3 billion compared to $1.5 billion as of December 31, 2024. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND launches in thalassemia and sickle cell disease, advance existing programs, and opportunistically expand its pipeline through both internally and externally discovered assets. Conference Call Information Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s second quarter 2025 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company's website (www.agios.com) under the “Events & Presentations” tab. A replay of the webcast will be available on the company’s website approximately two hours after the event. About Agios: Fueled by Connections to Transform Rare Diseases At Agios, our vision is to redefine the future of rare disease treatment. Fueled by connections, we build trusted partnerships with communities – collaborating to develop and deliver innovative medicines that have the potential to transform lives. With a foundation in hematology, we combine biological expertise with real-world insights to advance a growing pipeline of rare disease medicines that reflect the priorities of the people we serve. Agios is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts. To learn more, visit www.agios.com and follow us on LinkedIn and X. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of PYRUKYND® (mitapivat), tebapivat, AG-236 and AG-181; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, tebapivat, AG-236 and AG-181; Agios’ use of proceeds from the transaction with Royalty Pharma; potential U.S. net sales of vorasidenib and potential future royalty payments; Agios’ strategic vision and goals, including its key milestones for 2025; and the potential benefits of Agios’ strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Consolidated Balance Sheet Data(in thousands)(Unaudited) June 30, 2025 December 31, 2024Cash, cash equivalents, and marketable securities $1,339,404 $1,532,031Accounts receivable, net 4,986 4,109Inventory 30,848 27,616Total assets 1,471,237 1,663,199Stockholders' equity 1,369,555 1,540,956 Consolidated Statements of Operations Data(in thousands, except share and per share data)(Unaudited) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 2025 2024 Revenues: Product revenue, net$12,455 $8,615 $21,181 $16,804 Total revenue 12,455 8,615 21,181 16,804 Operating expenses: Cost of sales$1,702 $1,495 $2,787 $2,122 Research and development 91,940 77,401 164,683 146,021 Selling, general and administrative 45,869 35,536 87,396 66,550 Total operating expenses 139,511 114,432 254,866 214,693 Loss from operations (127,056) (105,817) (233,685) (197,889)Interest income, net 14,513 8,120 30,600 17,009 Other income, net 523 1,579 1,776 3,213 Net loss$(112,020) $(96,118) $(201,309) $(177,667)Net loss per share - basic and diluted$(1.93) $(1.69) $(3.49) $(3.14)Weighted-average number of common shares used in computing net loss per share – basic and diluted 57,932,576 56,802,546 57,697,193 56,593,011 Contacts: Investor ContactMorgan Sanford, VP, Investor Relations Agios PharmaceuticalsMorgan.Sanford@agios.com Media ContactEamonn Nolan, Senior Director, Corporate CommunicationsAgios PharmaceuticalsEamonn.Nolan@agios.com