Leucovorin Calcium

New evidence for established standard-of-care therapies, with updates for LORBRENA ® in ALK-positive metastatic non ‑ small cell lung cancer and a BRAFTOVI ® regimen in a type of BRAF-mutant colorectal cancer NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE: PFE) will present new data across its diverse, industry-leading Oncology pipeline and portfolio at the American Society of Clinical Oncology (ASCO) Annual Meeting, May 29 – June 2, 2026, in Chicago. Data from more than 40 company-sponsored, investigator-sponsored and collaborative research abstracts, including three late-breaking sessions and eight oral and rapid oral presentations, will be shared. These data highlight Pfizer’s leadership in establishing potential new standards of care across multiple cancer types and its next-generation pipeline of novel targets and combination strategies designed to extend impact into broader patient populations and earlier lines of therapy. “For people living with cancer and their families, every moment matters. We are moving with urgency to drive advances that have the potential to change standards of care – and striving to bring new, innovative options to patients in earlier lines of therapy,” said Jeff Legos, Chief Oncology Officer, Pfizer. “Our progress is evident in the data presented at ASCO this year, which span our portfolio of established therapies, as well as next-generation, early-stage clinical research from one of the industry’s largest oncology R&D programs. Together, these results reinforce our ability to advance breakthroughs that may redefine clinical practice and change the lives of people with cancer.” Key highlights of Pfizer’s presence at ASCO include: Sharing new evidence for standard-of-care therapies in certain types of biomarker-driven colorectal and lung cancers Seven-year update from the pivotal Phase 3 CROWN study further supports LORBRENA ® (lorlatinib) as a guideline-recommended first-line treatment for anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC). Late-breaking presentation of progression-free survival (PFS) and overall survival (OS) data from Cohort 3 of the BREAKWATER* trial evaluating BRAFTOVI ® (encorafenib) in combination with cetuximab and FOLFIRI (fluorouracil, leucovorin and irinotecan) as a first-line regimen for patients with BRAF V600E- mutant metastatic colorectal cancer (mCRC). These pivotal data further establish the benefit of this BRAFTOVI regimen following the U.S. Food and Drug Administration (FDA) full approval and the topline results announcement for objective response rate (ORR) from this Cohort earlier this year. Showcasing data on potential benefit of treatments in earlier lines of therapy for prostate and breast cancers Late-breaking presentation from the Phase 3 TALAPRO-3 study will highlight clinically meaningful radiographic progression-free survival (rPFS) benefit for TALZENNA ® (talazoparib) plus XTANDI ® ** (enzalutamide) in metastatic castration‑sensitive prostate cancer (mCSPC) patients with homologous recombination repair gene alterations, with effects consistent across subgroups and a strong OS trend. These data follow the announcement of topline results in March 2026 and support the potential of TALZENNA plus XTANDI to deliver benefit earlier in the disease course. Additional efficacy and safety outcomes by stratified subgroups from the Phase 3 HER2CLIMB-05 study investigating TUKYSA ® (tucatinib) in combination with trastuzumab and pertuzumab as first-line maintenance therapy for human epidermal growth factor receptor 2-positive (HER2+) metastatic breast cancer (MBC). These results support TUKYSA’s potential use as part of a chemotherapy-free, first-line maintenance strategy for HER2+ MBC. Advancing next-generation pipeline of novel mechanisms and differentiated combinations across solid tumors Updated Phase 2 data for PF‑08634404 (PF’4404), a novel bispecific antibody targeting PD-1 and VEGF, as monotherapy in first-line PD-L1-expressing NSCLC. PF’4404 is being developed as a potential backbone therapy across tumor types, including an ongoing Symbiotic-Lung-01 Phase 3 study in combination with chemotherapy in first-line NSCLC regardless of PD-L1 expression and an ongoing Symbiotic-GI-03 Phase 3 study in first-line mCRC. Updated results from a Phase 1 study of sigvotatug vedotin (SV), a novel integrin β6 (IB6)–directed ADC, in combination with pembrolizumab in NSCLC. These data support the ongoing SigVie-003 Phase 3 study of SV in combination with pembrolizumab in first-line NSCLC. An additional ongoing Phase 3 study, SigVie-002, is evaluating SV monotherapy in patients previously treated for advanced NSCLC. The first results from a Phase 2 study of neoadjuvant atirmociclib, a highly selective CDK4 inhibitor, in combination with letrozole versus letrozole alone in people with hormone receptor-positive (HR+), HER2- breast cancer. Atirmociclib is being developed as a potential first-in-class, next-generation cell cycle inhibitor backbone for HR+, HER2- breast cancer in the early adjuvant and first-line metastatic setting. Findings from a Phase 1b study of a brain-penetrant MEK inhibitor, PF-07799544 (polfurmetinib), plus a next-generation BRAF inhibitor, PF-07799933 (claturafenib), in advanced BRAF-mutant melanoma. Information on significant Pfizer and partner-sponsored abstracts, including date and time of presentation, follows in the chart below. A complete list of Pfizer and partner-sponsored abstracts and presentations is available here. LUNG CANCER Oral Presentation (Abstract 8502) May 29, 2026 1:00 PM-4:00 PM CDT Lorlatinib vs crizotinib as first-line treatment for advanced ALK+ non-small cell lung cancer: 7-year update from the phase 3 CROWN study Mok et al Rapid Oral Presentation (Abstract 8514) May 30, 2026 1:15 PM-2:45 PM CDT Updated results from a phase 2 trial of SSGJ-707 (PF-08634404), a PD-1/VEGF bispecific antibody, as monotherapy in patients with advanced non-small cell lung cancer (NSCLC) Wu et al Poster Presentation (Abstract 8522) May 31, 2026 9:00 AM-12:00 PM CDT Sigvotatug vedotin (SV), an investigational integrin beta-6 (IB6)–directed antibody-drug conjugate (ADC), plus pembrolizumab: updated results from phase 1 study (SGNB6A-001) Jaime et al Poster Presentation (Abstract 8609) May 31, 2026 9:00 AM-12:00 PM CDT Reduction in circulating tumor DNA (ctDNA) significantly correlates with radiographic response and tumor PD-L1 expression in a phase 1 study of PDL1V (PF-08046054) in patients with non-small cell lung cancer (NSCLC) Saleh et al COLORECTAL CANCER Oral Presentation (Abstract LBA3503) May 31, 2026 8:00 AM-11:00 AM CDT BREAKWATER: Progression-free survival analysis of first-line (1L) encorafenib + cetuximab (EC) + FOLFIRI in BRAF V600E-mutant metastatic colorectal cancer (mCRC) [LBA] Kopetz et al BREAST CANCER Oral Presentation (Abstract 1005) June 2, 2026 9:45 AM-12:45 PM CDT Efficacy and safety of tucatinib (TUC) vs placebo (PBO) combined with trastuzumab and pertuzumab (HP) as maintenance therapy for HER2+ metastatic breast cancer (MBC) by stratified randomized subgroups Hamilton et al Rapid Oral Presentation (Abstract LBA1018) May 31, 2026 11:30 AM-1:00 PM CDT Palbociclib plus Tamoxifen ± goserelin in women with hormone receptor (HR)-positive, HER2-negative advanced or metastatic breast cancer (BC): PATHWAY, an Asian international double-blind randomized phase 3 trial: Final Overall Survival (OS) analysis [LBA] Lu et al Poster Presentation (Abstract 518) June 1, 2026 1:30 PM-4:30 PM CDT Neoadjuvant atirmociclib plus letrozole versus letrozole alone in women with HR+/HER2− breast cancer: results from FOURLIGHT-2, a randomized phase 2 window of opportunity study Goel et al Poster Presentation (Abstract 1042) June 1, 2026 1:30 PM-4:30 PM CDT Tucatinib (TUC) combined with trastuzumab and pertuzumab (HP) as first-line (1L) maintenance therapy for HER2+ metastatic breast cancer (MBC): an in-depth safety analysis of HER2CLIMB-05 Dieras et al Poster Presentation (Abstract 1068) June 1, 2026 1:30 PM-4:30 PM CDT Long-term safety and adverse event (AE) management in patients with ER+/HER2− metastatic breast cancer (mBC) receiving prifestrastat, a first-in-class KAT6 inhibitor, in combination with fulvestrant Layman et al BLADDER/GENITOURINARY CANCERS Oral Presentation (Abstract LBA5007) May 30, 2026 3:00 PM-6:00 PM CDT TALAPRO-3: Talazoparib (TALA) + enzalutamide (ENZA) compared with placebo (PBO) + ENZA for the treatment of patients (pts) with metastatic castration-sensitive prostate cancer (mCSPC) harboring homologous recombination repair (HRR) gene alterations [LBA] Agarwal et al Oral Presentation (Abstract 4507) May 29, 2026 2:45 PM-5:45 PM CDT Enfortumab vedotin plus pembrolizumab (EV+P) vs chemotherapy for previously untreated locally advanced or metastatic urothelial carcinoma (la/mUC): 3.5-year follow-up and response analyses from the phase 3 EV-302 study Powles et al Oral Presentation (Abstract 4510) May 30, 2026 8:00 AM-9:30 AM CDT Health-related quality of life (HRQoL) with neoadjuvant and adjuvant (neoadj-adj) enfortumab vedotin (EV) plus pembrolizumab (pembro) in participants (pts) with muscle-invasive bladder cancer (MIBC) who are cisplatin ineligible: phase 3 KEYNOTE-905 study [MSD led] O’Donnell et al Oral Presentation (Abstract 5006) May 30, 2026 3:00 PM-6:00 PM CDT Final results from ZZFIRST: a randomized phase 2 trial of enzalutamide (EZ) and talazoparib (TALA) in metastatic hormone-naïve prostate cancer (mHNPC) Mateo et al Poster Presentation (Abstract 4613) May 31, 2026 9:00 AM-12:00 PM CDT Neoadjuvant and adjuvant (neoadj-adj) enfortumab vedotin (EV) plus pembrolizumab (pembro) in participants with cisplatin-ineligible muscle-invasive bladder cancer (MIBC): An analysis of clinically relevant subgroups in KEYNOTE-905 [MSD led] Adra et al Poster Presentation (Abstract 5043) May 31, 2026 9:00 AM-12:00 PM CDT Impact of baseline demographics on therapy management in patients (pts) with metastatic castration-resistant prostate cancer (mCRPC) treated with talazoparib (TALA) + enzalutamide (ENZA) in the TALAPRO-2 study: extended follow-up De Giorgi et al MELANOMA Rapid Oral Presentation (Abstract 9512) May 30, 2026 4:30 PM-6:00 PM CDT Phase 1b study of a brain-penetrant MEK inhibitor, PF-07799544, plus next-generation BRAF dimer inhibitor, PF-07799933, in advanced BRAF-mutant melanoma Chen et al MULTIPLE MYELOMA Oral Presentation (Abstract 7500) May 29, 2026 2:45 PM-5:45 PM CDT Safety and efficacy of elranatamab as early intervention in patients with high-risk smoldering myeloma: First results from the phase 2 ERASMM (EMN34) study Touzeau et al OTHER GYNECOLOGICAL CANCERS Poster Presentation (Abstract 5627) June 1, 2026 9:00 AM-12:00 PM CDT A phase 2 study evaluating SSGJ-707 (PF-08634404), a PD-1/VEGF bispecific antibody, + chemotherapy (chemo) in patients (pts) with first-line (1L) advanced/recurrent endometrial cancer (EC) Zhou et al Pfizer is continuing its commitment to help non-scientists understand the latest findings with the development of abstract plain language summaries (APLS) for company-sponsored research being presented at ASCO, which are written in non-technical language. Those interested in learning more can visit www.Pfizer.com/apls to access the summaries. *The BREAKWATER trial was conducted with support from ONO Pharmaceutical, Merck KGaA, Darmstadt, Germany and Eli Lilly and Company. Pierre Fabre Laboratories also has exclusive rights to commercialize the product in all other countries, including Europe and Asia (excluding Japan and South Korea). **XTANDI® is jointly developed and commercialized by Pfizer and Astellas in the United States. Prescribing Information for Pfizer Medicines Please see full Prescribing Information for LORBRENA® (lorlatinib). Please see full Prescribing Information for BRAFTOVI® (encorafenib). Please see full Prescribing Information for TALZENNA® (talazoparib). Please see full Prescribing Information for XTANDI® (enzalutamide). Please see full Prescribing Information for TUKYSA® (tucatinib). About Pfizer Oncology At Pfizer Oncology, we are at the forefront of a new era in cancer care. Our industry-leading portfolio and extensive pipeline includes three core mechanisms of action to attack cancer from multiple angles, including small molecules, antibody-drug conjugates (ADCs), and multispecific antibodies, including immune-oncology biologics. We are focused on delivering transformative therapies in some of the world’s most common cancers, including breast cancer, gastrointestinal cancer, genitourinary cancer, hematologic malignancies, and lung cancers. Driven by science, we are committed to accelerating breakthroughs to help people with cancer live better and longer lives. About Pfizer: Breakthroughs That Change Patients’ Lives At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For 175 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.pfizer.com. In addition, to learn more, please visit us on www.pfizer.com and follow us on X at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer. Disclosure Notice The information contained in this release is as of April 21, 2026. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments. This release contains forward-looking information about Pfizer Oncology and Pfizer’s oncology portfolio of marketed and investigational therapies, including their potential benefits; expectations for our product pipeline, in-line products and product candidates, including anticipated regulatory submissions, data read-outs, study starts, approvals, launches, clinical trial results and other developing data; the development or commercial potential of our product pipeline, in-line products, product candidates and additional indications or combinations, including expected clinical trial protocols, the potential and timing for the initiation and progress of clinical trials and data read-outs from trials; the timing and potential for the submission of applications for and receipt of regulatory approvals; the timing and potential for product launches and commercialization; expected breakthrough, best- or first-in-class or blockbuster status or expected market entry of our medicines; potential patients reached; the regulatory landscape; the competitive landscape; and other statements about our business, operations and financial results that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risk and uncertainties include, among other things, uncertainties regarding the commercial success of Pfizer’s oncology portfolio; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with interim and preliminary data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any drug applications, biologics license applications and/or emergency use authorization applications may be filed in any jurisdictions for any potential indication for Pfizer’s product candidates; whether and when any such applications that may be filed for any of Pfizer’s product candidates may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether any such product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of Pfizer’s products or product candidates, including development of products or therapies by other companies; manufacturing capabilities or capacity; risks and uncertainties related to issued or future executive orders or other new, or changes in, laws or regulations; uncertainties regarding the impact of COVID-19 on Pfizer’s business, operations and financial results; and competitive developments. A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2025 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com.

民为生物启动了MWN105（FGF21/GLP-1/GIP Fc融合蛋白）在中国的I期临床试验 2026年4月20日，该药物用于治疗2型糖尿病、肥胖和超重，试验登记编号为CTR20261331。试验名称为：评估MWN105注射液在中国非糖尿病超重或肥胖参与者中多次皮下给药的安全性、耐受性、药代动力学和药效学的随机、双盲、安慰剂对照I期临床试验。  本项临床试验计划在中国进行，目标入组人数为24人。试验药物为MWN105。本试验的主要终点为治疗期间所有自发报告和观察到的不良事件（AE）及严重不良事件（SAE）。 复宏汉霖启动了西妥昔单抗生物类似物（EGFR单抗）的中国I期临床试验 2026年4月20日，（试验编号：CTR20261471），适应症为结直肠癌。该项试验题为“一项评估HLX05-N与爱必妥®（美国/欧盟来源）在转移性结直肠癌受试者中药代动力学特征、有效性、安全性及免疫原性的多中心、随机、双盲、平行对照I期临床研究”，计划共入组387例受试者，试验地区为中国。  本研究试验组使用药品包括：亚叶酸、西妥昔单抗、氟尿嘧啶和奥沙利铂。 对照组所用药品为西妥昔单抗。本项临床研究的主要终点包括：给药前至给药后7天的血清药物浓度-时间曲线下面积（AUC0-7d），以及第3次给药后稳态给药间隔内的血清药物浓度-时间曲线下面积（AUCss）。 康哲药业启动了一项有关CMS-D001（TYK2抑制剂）的中国境内Ⅰ期临床试验 2026年4月20日，（试验编号：CTR20261530）。试验标题为“在健康参与者中评价CMS-D001与伊曲康唑或利福平药物-药物相互作用（DDI）的单中心、开放、自身对照的临床研究”，计划招募32名健康志愿者，试验将在中国地区进行。  本项研究的试验组药品包括：利福平、CMS-D001及伊曲康唑。 主要研究终点为CMS-D001的关键药代动力学参数，包括：峰浓度（Cmax）；从给药后0时至最后一个可定量时间点的药物浓度-时间曲线下面积（AUC0-last）；从给药后0时至无穷大时间点的药物浓度-时间曲线下面积（AUC0-inf）。 海思科公司的HSK55879（激动剂，具体靶点信息尚未公布）正式启动一项关于肥胖和超重人群的中国I期临床试验 2026年4月20日，（试验编号：CTR20261409）。该试验的研究标题为“HSK55879片在超重/肥胖试验参与者中的多次给药安全性、耐受性、药代动力学和药效动力学影响的I期临床研究”。 研究地点选定在中国，预计目标入组人数为44人。 该试验组药物为HSK55879。主要研究内容包括多次给药后对超重或肥胖受试者的安全性、耐受性以及药代动力学和药效动力学特征进行评估。  主要终点指标涵盖：不良事件及严重不良事件监测、体格检查、生命体征评估、12导联心电图检查、实验室分析（包括血常规、血生化、凝血功能、尿常规和甲状腺功能三项），以全面评估药物的临床安全性。 康诺亚公司启动了ocankitug（TSLP单克隆抗体）针对哮喘的中国I期临床试验 2026年4月20日，试验编号为CTR20261433。试验标题为：“比较健康成年男性参与者使用CM326注射液不同给药装置单次皮下注射的随机、开放、平行对照的药代动力学、安全性和免疫原性的I期临床研究”。 本试验将在中国地区开展，计划入组目标人数为192人。 本次临床试验的试验组和对照组均使用ocankitug作为研究药物。 主要终点指标为CM326的药代动力学参数：Cmax（最大血药浓度）和AUC0-∞（曲线下面积）。 Eli Lilly正式启动一项国际多中心、Ⅲ期临床试验 2026年4月20日（试验编号：CTR20261445），旨在针对卵巢癌、输卵管癌以及腹膜癌患者群体，评估创新药物Sofetabart Mipitecan（LY4170156）的治疗效果。本研究名为“FRAmework-01”，共分两部分： 第一部分针对铂耐药卵巢癌患者，比较Sofetabart Mipitecan与传统化疗或索米妥昔单抗的疗效； 第二部分针对铂敏感卵巢癌患者，评价Sofetabart Mipitecan联合贝伐珠单抗与含铂化疗联合贝伐珠单抗的疗效差异。 该项目在国际多中心开展，计划入组患者共1080例。 试验组药物：Sofetabart Mipitecan。 对照组药物：吉西他滨、紫杉醇、聚乙二醇多柔比星脂质体。主要终点为无进展生存期（PFS），由研究者根据RECIST v1.1标准进行评估。 三生制药启动了SSS67（ACVR2A/ACVR2B双特异性抗体）注射液的中国I期临床试验 2026年4月20日，（试验编号：CTR20261489），旨在评估其在健康及超重/肥胖受试者中的安全性、耐受性、药代动力学及药效学特征。本研究为随机、双盲、安慰剂对照设计，计划在中国地区招募52例受试者。  本次临床试验的研究药物为SSS67。 主要研究终点包括不良事件与不良反应的发生情况，实验室检查（血常规、尿常规、血生化、内分泌指标、胸片/肺部CT等），生命体征，12导联心电图（ECG）以及体格检查结果。 圣因生物启动了SGB-3908（AGT，siRNA疗法）针对高血压的中国I期临床试验 2026年4月20日，试验编号为CTR20261398。该研究旨在评估IBI3016在中国轻至中度高血压患者中单次皮下给药的安全性、耐受性、药代动力学特征及药效学特征。试验计划在中国地区进行，目标入组人数为30人。  本次临床研究的试验组药物为SGB-3908。 该试验的主要终点为安全性评估指标，包括不良事件发生情况、体格检查、生命体征、实验室检查及12导联心电图等。 恒瑞医药启动一项针对B细胞血癌的中国境内一期临床试验 2026年4月20日（试验编号：CTR20261435，药物：HRS-3005）。本试验为开放、多中心临床研究，旨在评价HRS-3005在B细胞恶性肿瘤患者中的安全性、耐受性、药代动力学以及初步疗效。 计划入组患者总数为190人，试验地区为中国。本研究的药物试验组为HRS-3005。主要研究终点包括：最大耐受剂量（MTD）、推荐二期剂量（RP2D）、不良事件（AE）发生率及严重程度（基于NCI-CTCAE 6.0标准）、以及异常实验室指标的评估。 海湃泰克HEP-50768（MRGPRX4拮抗剂）在中国启动了一项针对慢性肾病和胆汁淤积的I期临床试验 2026年4月20日，（试验编号：CTR20261473）。本次研究是一项随机、单剂量与多剂量递增给药的I期研究，旨在评估HEP-50768在健康受试者、胆汁淤积性瘙痒受试者和慢性肾病相关性瘙痒受试者中的安全性、耐受性及药代动力学特征。 计划在全国多个中心开展，目标入组人数为96例。本次临床试验的研究药物为HEP-50768。 主要研究终点包括：不良事件发生例数、发生次数和发生率，以及体格检查、超声检查、生命体征、体重、12导联心电图和实验室检查等各项安全性指标的变化值。 阿斯利康IL-33单抗第3项COPD III期研究成功  4月20日，阿斯利康宣布潜在首创IL-33单抗Tozorakimab治疗慢性阻塞性肺病（COPD）的III期MIRANDA研究取得了高水平积极结果。 该研究招募了在接受标准吸入治疗期间仍出现中度至重度加重的COPD患者，评估了Tozorakimab（300mg，每2周1次）的有效性和安全性。结果显示，已戒烟/未戒烟人群，血嗜酸性粒细胞（EOS）水平不同人群以及肺功能严重程度不同人群的中重度COPD急性加重率均达到了具有统计学意义和临床意义的降低效果。 此外，Tozorakimab耐受性良好，安全性与既往研究一致。为评估Tozorakimab在COPD人群中的疗效和安全性，阿斯利康共开展了4项全球注册性III期研究，其它三项为OBERON、TITANIA和PROSPERO，其中前两项已达到主要终点，最后一项预计将在今年上半年读出数据。  诺和诺德同类首创新药III期临床成功，针对镰状细胞病  4月20日，诺和诺德宣布其针对成人和青少年镰状细胞病（SCD）的研究性药物etavopivat在HIBISCUS关键3期临床试验中取得了积极的顶线结果。 作为首个达到双重主要终点的同类首创（first-in-class）新药，Etavopivat是一种每日口服的小分子别构激活剂，专门针对红细胞中的PKR激酶发挥作用。 在针对385名12岁及以上SCD患者的52周双盲试验中，在接受标准护理的基础上，与安慰剂相比，接受etavopivat治疗的患者血管闭塞性危象（VOC）年化发生率显著降低了27%。首次发生VOC的时间被大幅延长，中位时间达到了38.4周，而安慰剂组仅为20.9周。 在血红蛋白（Hb）响应方面，etavopivat表现出优效性：在治疗24周后，48.7%的患者血红蛋白增加超过1g/dL，远高于安慰剂组的7.2%。作为探索性分析的一部分，该药物还显著降低了患者的输血风险。 玛贝兰妥单抗联合方案获批，治疗二线及以上多发性骨髓瘤成年患者 4月20日，葛兰素史克宣布国家药监局批准注射用玛贝兰妥单抗（商品名：倍乐锐）联合硼替佐米和地塞米松，用于既往接受过至少一线治疗的多发性骨髓瘤成年患者，该申请已被纳入优先审评及突破性治疗药物程序。 多发性骨髓瘤为全球第三大常见血液肿瘤，中国年新增病例约3万例且发病率三十年间已翻倍。 倍乐锐是目前唯一获批用于该适应症的抗BCMA抗体偶联药物，可实现30分钟门诊输注，满足患者多样化治疗需求。该药已在美国获批用于至少二线治疗患者，并在欧盟、日本等全球市场获得超15项监管批准。 8.5亿美元！天境生物CD38抗体大中华区权益授权给Biogen 4月20日，天境生物宣布与Biogen达成合作协议，将CD38抗体菲泽妥单抗的大中华区权益授权给Biogen，后者支付1亿美元预付款，7.5亿美元里程碑金额，以及中个位数至低两位数比例的销售分成。 根据协议，Biogen将继续主导菲泽妥单抗免疫领域适应症的开发，并将在大中华区主导该产品的生产和商业化。 天境生物已于2024年12月向NMPA成功递交菲泽妥单抗治疗多发性骨髓瘤的上市申请，目前正在审评中。该适应症获批后，Biogen将主导商业化工作，天境生物将作为生产方，依托其位于杭州的GMP生产基地，负责产品的商业化供药。 特朗普签署行政令，加速致幻药物的获取 特朗普总统签署行政令要求FDA降低致幻药物治疗严重精神疾病的监管门槛，包括将国家优先审评券扩展至获得突破性疗法认定的致幻药物以缩短审批时间，并通过ARPA-H拨款5000万美元支持相关研究。 该命令在白宫活动中宣布，播客主持人Joe Rogan出席，他曾向特朗普推荐伊博格碱治疗PTSD。伊博格碱目前在美国属一级管制物质，白宫同时呼吁FDA与DEA依据《2017年尝试权法案》为符合条件的患者建立获取研究性致幻药物的途径。 Gilgamesh Pharma正开发其衍生物GM-3009以降低心脏副作用，德州去年也启动相关研究联盟加速临床试验。出席活动的退役海豹突击队员Marcus Luttrell表示，伊博格碱和5-MeO-DMT改变了其因战伤导致的疼痛、创伤和阿片类药物依赖，卫生部长Robert F Kennedy Jr亦长期支持致幻剂研究，曾指责FDA压制此类疗法。 FDA庆祝在结束动物实验方面取得进展，但专家警告仍有很长的路要走 FDA宣布完成减少动物实验路线图的年度目标，包括建立新替代方法数据库、推出AI肝脏影像分析工具及限制单抗灵长类动物测试的指南草案，计划2030年使动物实验成为例外而非常态。 但专家指出全面替代仍需漫长过程，NABR强调动物模型与新方法均为人类疾病替代物，成功率与人体试验相当，仅9.6%人体试验药物最终获批；Charles River首席科学官认为过渡应是渐进式而非革命性。 Vanda Pharmaceuticals批评指南缺乏明确替代案例和科学引用，要求撤回并制定更强科学基础的版本。FDA新草案允许提交未经验证的新方法数据被视为重要推进，但患者安全仍是最终指导原则。 勃林格殷格翰在伦敦设立人工智能中心 勃林格殷格翰4月20日在伦敦国王十字区开设新的人工智能与机器学习中心，作为其10年1.5亿英镑AI研发投资的一部分，旨在通过基础AI技术深入理解疾病生物学机制、加速靶向药物开发以改善患者预后。 该中心将与公司现有美国、奥地利、德国AI站点协同，预计2027年底前配备50名专家，充分利用英国数据资源及学术、生物科技人才。 此举与伦敦癌症中心10亿美元扩张计划相呼应，助力伦敦打造全球生命科学枢纽，也为此前因MSD取消10亿英镑扩张计划而受挫的英国生命科学领域注入信心。英国科学大臣表示，AI正以前所未有的方式推动生命科学发现。 生物医药行业持续创新，未来可期!  注:本文整理自行业公开信息，仅供参考！