Pegozafermin

iStock, Volodymyr Kryshtal After last year’s ‘stampede’ for FGF21 assets, the focus for the metabolic dysfunction-associated steatohepatitis space has shifted toward differentiated approaches, such as THR-β agonists and combination treatments, that seek to mirror the commercial success of Madrigal’s Rezdiffra. Before Madrigal’s Rezdiffra, the quest for a cure for metabolic dysfunction-associated steatohepatitis was a veritable graveyard for drug developers. However, the 2024 approval of that first drug for the inflammatory liver disease demonstrated to the pharma industry that the feat was possible. Not only that, but Madrigal proved it could be lucrative. In November 2025, the company announced in its third-quarter financial update that Rezdiffra had generated revenues of $287 million, just six quarters after its launch. Altogether, the product has amassed $817 million in revenue for its maker. This commercial success has not gone unnoticed, with three Big Pharma acquisitions in the MASH space in 2025, all worth billions of dollars. GSK began the rush in May by acquiring the investigational FGF21 analog efimosfermin alfa from Boston Pharmaceuticals for $1.2 billion upfront. Then, in September, Roche snapped up 89bio and another FGF21 analog pegozafermin for around $3.5 billion. Novo Nordisk followed weeks later with a $5.2 billion deal for Akero Therapeutics . The spate of acquisitions—all focused on FGF21 analogs—was centered on the success of Akero Therapeutics’ efruxifermin, which, in January 2025, showed positive long-term data in a mid-stage trial. “I think that really set off the stampede because there were very few FGF21 assets in development,” Edward Nash, managing director at Canaccord Genuity, told BioSpace . “This caused a big pharma reaction of, ‘Wait a minute, we need one of those too because we have a metabolic franchise and we need to be able to compete, especially given the data seen to date in the FGF21 space.’” This, alongside the approval of Novo Nordisk’s GLP-1 weight-loss blockbuster Wegovy for MASH, has led to the market opening up, and now, more companies want in, Nash said. All of this activity means the overall MASH landscape has changed significantly in the past year . With the key FGF21 developers off the market, there is an increased focus on the other assets in the field’s late-stage pipeline. Here are four candidates, each with differentiated mechanisms of action, that Big Pharma companies could have in their sights in 2026. Policy FDA Approves Madrigal’s Rezdiffra as First MASH Therapy Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) is the first-ever approved therapy for metabolic dysfunction-associated steatohepatitis—a decision experts say could signal a sea change in treatment of the disease. March 14, 2024 · 6 min read · Jill Neimark Inventiva Homes In On Potential Next Oral MASH Drug Inventiva’s upcoming Phase III readout for pan-PPAR agonist lanifibranor is “the biggest event in the MASH space” this year, according to Nash. “It’s been a long trial, and it’s been long-awaited by the Street,” he said. “Depending on the data, [Inventiva] could definitely become another takeover candidate.” The French biotech is now solely focused on lanifibranor after halving its workforce and stopping all preclinical research not related to the drug to help fund its development in February 2025. This singular focus will help push the drug through its Phase III trial, with Inventiva poised to publish topline results from the NATiV3 trial in the second half of 2026. With these data expected, “lanifibranor could potentially be the next oral therapy approved for the treatment of patients with MASH,” CEO Frederic Cren said in a statement in April 2025. Lanifibranor is an oral small molecule that activates three peroxisome proliferator-activated receptor (PPAR) isoforms. Through the activation of these isoforms, Inventiva aims to address the key drivers of the disease, including inducing anti-fibrotic, anti-inflammatory and beneficial metabolic changes. In the NATIVE Phase IIb trial , reported in June 2020, lanifibranor achieved statistically significant MASH resolution and fibrosis improvement, with 42% of patients who received a 1200 mg dose seeing fibrosis improvement vs. 24% of placebo comparators after 24 weeks. The path to this year’s Phase III readout has not been straightforward for Inventiva. In February 2024 , the company voluntarily suspended screening and enrollment of patients in NATiV3 after a patient exhibited severely elevated aminotransferase levels. Now, Inventiva expects the results from NATiV3, if positive, to form the basis for a submission for regulatory approval. Viking Still Up For Grabs Viking Therapeutics has long attracted significant interest from Big Pharma. With pipeline assets that target both obesity and MASH, analysts have marked the biotech as one likely to draw suitors. For MASH, the company is developing VK2809, an oral thyroid hormone receptor-beta (THR-β) agonist. In the Phase IIb VOYAGE trial , VK2809 demonstrated a 37%-55% mean reduction in liver fat, with up to 75% of patients reaching MASH resolution without fibrosis worsening, according to data released in November 2024. In addition to these outcomes, the drug also demonstrated lipid-lowering effects and minimal side effects, Brian Lian, CEO of Viking, told BioSpace in an email. The overall body of evidence for VK2809 makes it “highly competitive” against both marketed products and drug candidates for MASH, he added. While Lian has previously stated that Viking would be open to partnering on the asset and confirmed that the company is still exploring partnering opportunities—he previously said that having a partner is not mandatory in order to bring the product to the market. Earnings MASH, Metsera Deals Send Analysts Marauding to Viking Viking Therapeutics CEO Brian Lian is watching the growth of interest in MASH and obesity but prepared to go it alone. October 23, 2025 · 6 min read · Annalee Armstrong Altimmune Open to Partnering Pemvidutide But Could Go It Alone Altimmune’s pemvidutide stands apart from others in the MASH pipeline due to its ability to preserve lean muscle mass, as well as ensuring MASH resolution and weight loss, a company spokesperson told BioSpace in an email. Pemvidutide is a weekly injectable GLP-1/glucagon dual receptor agonist that last month received the FDA’s Breakthrough Therapy Designation for MASH. This follows the November release of Phase IIb data from Altimmune’s IMPACT study, which showed that 52% of patients achieved MASH resolution without worsening of fibrosis, as well as improvements to liver fat content and weight loss. Altimmune is in a similar position to Viking, as both companies have the data to progress their drugs into Phase III, but may not begin those trials until they can find a partner, Nash said. “These two companies are all in on obesity, and that treatment area is already a big enough nut to crack,” he added. “To try to do that and run a MASH trial at the same time, I think you’re going to get a lot more bang for your buck if your molecule is differentiated in obesity.” The Altimmune spokesperson countered this, however, saying that independently bringing the asset forward in MASH is a “legitimate path forward.” However, they also noted that Altimmune remains open to any opportunity “that adds value, including partnerships.” Sagimet Looks to Combo FASN Inhibitor For Optimum Effect Like pemvidutide, Sagimet Biosciences’ denifanstat, an oral, once-daily fatty acid synthase (FASN) inhibitor that blocks the production of new fat in the liver, holds FDA Breakthrough Therapy Designation in MASH. Unlike Altimmune, however, Sagimet is not considering pursuing a Phase III trial alone, according to Nash. Instead, the California-based company is looking to cement its drug as a validated part of a combination therapy. In line with this, Sagimet released data in December from a Phase I trial that featured denifanstat paired with Rezdiffra, the current market leader. The trial showed that the combination was well-tolerated, and Sagimet plans to advance denifanstat into Phase II efficacy trials for MASH patients with F4 fibrosis. The combined treatment could boost fibrosis reduction more than Rezdiffra alone, Nash said. This type of combination approach is likely to be the future of MASH treatments because the disease is multifactorial, he added, with the main two targets being liver fat and fibrosis. So far, the marketed drugs— Rezdiffra and Wegovy—and the investigational pipeline have not proven outstanding at hitting both of these targets, making a combination treatment approach logical, Nash said. It seems the same could be true for the companies themselves, with partnering and M&A the likeliest outcome for the smaller players in the MASH landscape. “It’s hard to be successful as a standalone company,” Nash said. “There’s usually only one company when that happens, like Madrigal. I think we’re going to have one, maybe two biotechs, that can do it—after that, it’s going to be Big Pharma taking them in and developing them.”

注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需要得到授权。 昔日的肿瘤药王者罗氏，正逐步走出“青黄不接”的低谷。最新财报显示，公司全年营收增长7%，制药业务劲增9%，这不仅是一份稳健的成绩单，更勾勒出一幅重整旗鼓的战略图景。六大核心产品——Phesgo、Xolair、Ocrevus、Hemlibra、Vabysmo与Polivy齐齐发力，既填补了老药专利到期留下的缺口，也撑起了制药业务的持续增长曲线。 在肿瘤主阵地，Phesgo以48%的惊人增速证明HER2防线依旧牢靠；在血液瘤板块，Polivy攻势凌厉，复兴势头不减。罗氏CEO多次强调公司不会陷入专利悬崖困境，原因是2030年前将推出19款新药，其中16款有望成为“重磅炸弹”，9款峰值销售将突破30亿美元。乳腺癌领域的新王牌giredestrant被寄予厚望；而在炙手可热的减重赛道，罗氏更喊出要做全球“第三极”的豪言。当百亿美元资本砸向肥胖、阿尔茨海默病等慢病领域，一场关乎未来十年的战略博弈已然开启——胜，则版图永固；败，则新的悬崖或已在眼前。罗氏的案例告诉我们，在创新药行业的至暗时刻，唯有保持定力、跨出舒适区，才能在不确定中觅得新增长。毕竟，这里从来没有永远的王者，只有持续进化的生存者。 01 乳腺癌：新王牌登场 2025年，罗氏制药业务收入达476.69亿瑞士法郎（约576.27亿美元），同比增长9%。神经科学、免疫与眼科板块增速更亮眼，分别为11%、12%和10%。但作为传统强项的肿瘤领域依旧举足轻重——连续八季度增长，全年贡献239.38亿瑞士法郎，同比增长6%。 Phesgo、Xolair、Ocrevus、Hemlibra、Vabysmo五款重磅产品，加上快速崛起的Polivy，成为业绩主引擎，2025年合计新增销售36亿瑞士法郎，完全抵消了Avastin、Herceptin等专利到期造成的7亿瑞士法郎下滑。其中Phesgo表现最为抢眼，增幅高达48%。这款将帕妥珠单抗与曲妥珠单抗合二为一的皮下制剂，凭借给药便利优势在全球加速普及。财报披露其全球转化率已达54%，正向60%的目标迈进，意味着增长势头有望延续。 从曲妥珠单抗到帕妥珠单抗、恩美曲妥珠单抗，再到Phesgo，HER2靶点始终是罗氏命脉所在。公司预计HER2产品线2026年峰值约90亿瑞士法郎，2030年后平稳过渡至约40亿尾部收入，主要由Phesgo（30亿）与Kadcyla（10亿）支撑。 防守之余，罗氏积极拓展ER+（激素受体阳性）乳腺癌市场。该亚型占乳腺癌病例约70%，且多达三分之一早期患者在辅助内分泌治疗期间或之后会复发，部分因耐受性问题被迫停药，这为更优方案创造需求。罗氏口服SERD药物Giredestrant被视作新一线疗法的有力候选，官方峰值指引“超30亿美元”，但管理层预期更高。CEO Teresa Graham在电话会上反复强调其辅助用药潜力。 信心源自两项关键III期成功：晚期二线的evERA研究，以及2025年底公布阳性结果的早期辅助lidERA研究——后者首次证实口服SERD在早期乳腺癌复发风险上显著优于标准内分泌治疗，覆盖约55%辅助治疗人群。Graham直言，看过lidERA结果便会相信它将改写标准疗法。在200-300亿美元的ER+市场中，辅助治疗占三分之二，Giredestrant志在成为新基石。但挑战同样严峻：礼来、阿斯利康等强敌环伺，且医生接受度需时间培育；lidERA对照组未纳入当前主流“AI+CDK4/6抑制剂”方案，说服力有待验证。Giredestrant能否撼动格局，成为罗氏新王牌，仍待临床与市场的双重考验。 02 血液瘤：王者归来 血液瘤曾是罗氏的天下，但利妥昔单抗专利到期后一度退潮。所幸血友病双抗Emicizumab与CD79b ADC Polivy的崛起止住了颓势，“血液瘤复兴”在2025年继续兑现。 全年血液学产品线销售额86亿瑞士法郎，同比增长15%，为制药业务增速冠军，Polivy则是复兴核心。2021年关键III期研究证实，Polivy联合R-CHP方案一线治疗弥漫大B细胞淋巴瘤（DLBCL），可降低27%的疾病进展、复发或死亡风险。2023年获FDA批准，成为20年来首个DLBCL一线新方案。此后快速放量，2023年销售翻倍破10亿美元，2025年达14.7亿瑞士法郎，同比增长38%，并在美国中高危患者中成为最常用一线方案，仅此适应症销售已破10亿瑞士法郎。目前一线市占率约36%，治疗患者超6万，距公司设定的65%目标仍有增长空间。 双抗布局亦见成效：CD20/CD3双抗Columvi在三线及以上DLBCL快速渗透，2025年销售2.88亿瑞士法郎，同比增75%；Lunsumio在滤泡性淋巴瘤表现亮眼，皮下制剂获批后销售同比增68%至1.14亿瑞士法郎。但Columvi二线上市申请遭FDA拒绝，峰值预期由10亿降至数亿。整体看，这些产品构筑了血液瘤多层次、差异化火力网，助罗氏完成从单抗到ADC与双抗时代的引擎切换。 03 百亿豪赌慢病“重磅炸弹”  经历专利悬崖冲击，罗氏决心拓展边界，不再单押肿瘤。2025年研发日上，公司提出下个十年将主攻阿尔茨海默症、高血压、肥胖等大病种。行动胜于言辞——2025年以来，罗氏达成逾20笔交易，总金额超200亿美元，多数集中于代谢与自免领域，这是其史上首次在肿瘤之外投入更多资源。 减重是重中之重，目标直指全球前三。2023年底27亿美元收购Carmot，拿下GLP-1/GIP双激动剂CT-388与口服GLP-1激动剂CT-996；2025年3月与Zealand Pharma达成53亿美元合作（首付16.5亿），引进长效胰淀素类似物Petrelintide；9月再以35亿美元收购89bio，获得FGF21类似物Pegozafermin，进军MASH。罗氏认为其组合能满足减重患者多元需求，这是称雄底气所在。 按计划，2030年前将推出19款新药，17款具重磅潜力，10款峰值破30亿美元，多数来自慢病领域。仅减重板块就有望贡献两个30亿美元级产品组合：GLP-1药物组合（CT-388、CT-868、CT-996）与Petrelintide。CT-388已启动III期研究，II期数据显示48周 placebo校正体重下降22.5%，且无平台期，与Petrelintide联用潜力可观。  此外，Pegozafermin的MASH适应症进入III期，预计2027年读出；从Alnylam引进的siRNA疗法Zilebesiran瞄准高血压，III期临床有望实现半年一针；TL1A单抗Afimkibart在IBD与MASH双线推进。这些都锚定30亿美元峰值。CEO谈BD策略时底气十足：因暂无迫近的专利悬崖，“不必为买而买”，但会紧盯中国市场动向。未来数年将是这批重磅炸弹的密集验证期，豪赌成败留待时间作答。 结语 创新药疆场从无永恒王者，唯有持续进化。罗氏从肿瘤霸主蜕变为多赛道玩家，本质是一场巨头的自我革新。宏伟蓝图仍需科学与市场的双重检验：Giredestrant能否重塑乳腺癌格局？重金布局的GLP-1军团能否在诺和诺德与礼来的夹击中突围？答案将决定这幅新画卷是空中楼阁，还是可持永续的江山宏图。 氨基观察-创新药组原创出品  作者 | 武月 END 本文为原创文章，转载请留言获取授权 近期热门资源获取 中国临床试验趋势与国际多中心临床展望-202505 2024年医药企业综合实力排行榜-202505 中国带状疱疹疫苗行业分析报告-202505 2023H2-2024H1中国药品分析报告-202504 2024年中国1类新药靶点白皮书-202503 中国AI医疗健康企业创新发展百强榜单-202502 2024年FDA批准上市的新药分析报告-202501 小分子化药白皮书（上）-202501 2024年中国医疗健康投融资全景洞察报告-202501 2024年医保谈判及市场分析报告-202501 近期更多摩熵咨询热门报告，识别下方二维码领取 联系我们，体验摩熵医药更多专业服务 会议 合作 园区 服务 数据库 咨询 定制 服务 媒体 合作 点击阅读原文，申请摩熵医药企业版免费试用！