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New Era for Treatment
突破性胰腺癌药物标志着治疗新时代
Since last year, pharma companies have announced 24 final-stage trials for medicines targeting the disease.
自去年以来，制药公司已宣布进行 24 项针对该疾病的药物的最后阶段试验。
Pancreatic cancer medication daraxonrasib.
胰腺癌药物 daraxonrasib。Photographer: Jason LeCras for Bloomberg Businessweek
摄影师：Jason LeCras，《彭博商业周刊》
By Robert Langreth
August 27, 2026 at 2:12 AM GMT+8
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● Jay Williams, a former movie studio executive, started taking daraxonrasib and his energy came back, and blood tests suggest his tumors have begun to shrink.
前电影制片厂高管 Jay Williams 开始服用 daraxonrasib，他的精力恢复了，血液检查表明他的肿瘤已开始缩小。
● Revolution Medicines’ pill, daraxonrasib, secured US regulatory approval and is the first in a wave of targeted drugs that could reshape treatment for pancreatic cancer.
Revolution Medicines 的药物 daraxonrasib 获得了美国监管部门的批准，是有望重塑胰腺癌治疗的靶向药物浪潮中的第一个。
● Daraxonrasib works using a new “molecular glue” mechanism that cements the bad protein to another healthy protein to inactivate it, and it could ultimately reach more than $9 billion in annual sales.
Daraxonrasib 使用一种新的 "分子胶" 机制，将不良蛋白质与另一种健康蛋白质粘合在一起，使其失活，最终年销售额可能超过 90 亿美元。
Jay Williams, a former movie studio executive, was diagnosed with pancreatic cancer in 2023 when he was just 55. Despite surgery to remove a tumor and multiple rounds of chemotherapy, the disease progressed, eventually spreading to his liver. Toward the start of this year, his doctor bluntly told him he was starting to run out of treatment options.
前电影制片厂高管杰伊·威廉姆斯 (Jay Williams) 于 2023 年被诊断出患有胰腺癌，当时他年仅 55 岁。尽管进行了肿瘤切除手术和多轮化疗，但病情仍在恶化，最终扩散到肝脏。今年年初，他的医生直言不讳地告诉他，他的治疗方案已经开始耗尽。
But in early July, Williams qualified for special access to a powerful new drug from Revolution Medicines Inc. Within days after he started taking daraxonrasib, his energy started to come back and he was able to go on early morning walks with his wife for the first time in years. Blood tests suggest his tumors have begun to shrink, and side effects such as a skin rash have been much easier to tolerate than chemo. “It’s been life-changing,” says Williams, who lives in Santa Clarita, California.
但在 7 月初，威廉姆斯获得了从 Revolution Medicines Inc. 获得一种强效新药的特殊资格。在他开始服用 daraxonrasib 几天后，他的精力开始恢复，多年来第一次能够与妻子一起清晨散步。血液检查表明他的肿瘤已经开始缩小，而且皮疹等副作用比化疗更容易忍受。"这改变了我的生活，" 住在加利福尼亚州圣克拉丽塔的威廉姆斯说。
Revolution Medicines’ pill, which secured US regulatory approval today, is the first in a wave of targeted drugs that could reshape treatment for pancreatic cancer, long one of the hardest types of cancer to treat. In a final-stage trial whose results were first announced in April, the drug almost doubled the typical survival time for advanced patients whose cancer had stopped responding to chemo, to 13.2 months. When the results were presented at a big oncology conference in Chicago in May, attendees responded with a standing ovation.
Revolution Medicines 的药物今天获得美国监管机构的批准，是有望重塑胰腺癌治疗的靶向药物浪潮中的第一个，而胰腺癌长期以来一直是最难治疗的癌症类型之一。在 4 月份首次公布结果的最后阶段试验中，该药物几乎使癌症对化疗停止反应的晚期患者的典型生存时间延长了一倍，达到 13.2 个月。当五月在芝加哥举行的一次大型肿瘤学会议上公布研究结果时，与会者起立鼓掌。
Williams. Photographer: Jason LeCras for Bloomberg Businessweek
威廉姆斯. 摄影师:Jason LeCras, 《彭博商业周刊》
New drugs are badly needed. More than 67,000 Americans are diagnosed with pancreatic cancer each year, but only about 13% of patients survive longer than five years. Growing deep inside the body, the disease often doesn’t cause obvious symptoms until it’s advanced. Chemotherapy
typically works only for a few months once the cancer has spread widely. 急需新药。每年有超过 67,000 名美国人被诊断患有胰腺癌，但只有约 13% 的患者存活时间超过五年。这种疾病在身体深处生长，通常不会引起明显的症状，直到进展为止。一旦癌症广泛扩散，化疗通常只能持续几个月。
Pharmaceutical companies had few good new approaches for treating pancreatic cancer until recently. Now drugmaker interest is “just escalating exponentially,” says Eileen O’Reilly, an oncologist at Memorial Sloan Kettering Cancer Center in New York City and a top investigator on the big daraxonrasib trial. With all the promising drugs in testing, she predicts that a few years from now, newly diagnosed patients will likely have a choice of several regimens to treat their pancreatic cancer.
直到最近，制药公司还几乎没有治疗胰腺癌的良好新方法。纽约市纪念斯隆凯特琳癌症中心的肿瘤学家、大型 daraxonrasib 试验的高级研究员艾琳·奥莱利 (Eileen O’Reilly) 表示，现在制药商的兴趣 "呈指数级增长"。随着所有有前景的药物都在测试中，她预测几年后，新诊断的患者可能会选择多种治疗胰腺癌的方案。
Since the start of last year, drug companies have announced 24 final-stage trials of pancreatic cancer treatments – more than in the entire preceding five-year period, according to a Bloomberg Businessweek analysis of a government database. The global market for branded pancreatic cancer medicines could soar to about $12 billion in 2035, from less than $500 million today, according to estimates from Bloomberg Intelligence. Daraxonrasib alone could ultimately reach more than $9 billion in annual sales, say pharma industry analysts surveyed by Bloomberg.
根据《彭博商业周刊》对政府数据库的分析，自去年年初以来，制药公司已经宣布了 24 项胰腺癌治疗的最后阶段试验——比之前整个五年期的试验数量还要多。据 Bloomberg Intelligence 估计，到 2035 年，全球胰腺癌品牌药物市场可能会从目前的不到 5 亿美元飙升至约 120 亿美元。彭博社调查的制药行业分析师表示，仅 Daraxonrasib 的年销售额最终可能会超过 90 亿美元。
Behind the deluge are advances in drug-design technology that are for the first time allowing scientists to target the crucial molecular drivers behind the disease. Most cases of pancreatic cancer – as well as many other types – are caused by a gene mutation that’s proven almost impossible to target
with traditional pharmaceutical technology. The gene, called KRAS, governs the production of a protein that regulates cell growth. When the gene is mutated, the protein can get stuck in the “on” position, allowing cells to grow uncontrollably. Daraxonrasib, which will be sold under the brand name Rasonque, works using a newfangled “molecular glue” mechanism that cements the bad protein to another healthy protein to inactivate it. It wasn’t clear such an approach would be safe and practical until Revolution Medicines showed it could work. “What is exciting is we now have a targeted therapy that applies to the majority of pancreatic cancer patients,” says Vincent Chung, a medical oncologist at City of Hope medical center in Duarte, California, who treats Williams.
药物泛滥的背后是药物设计技术的进步，这些技术首次使科学家能够瞄准疾病背后的关键分子驱动因素。大多数胰腺癌以及许多其他类型的癌症都是由基因突变引起的，事实证明传统制药技术几乎不可能靶向这种基因突变。该基因称为 KRAS，控制着调节细胞生长的蛋白质的产生。当基因突变时，蛋白质会卡在 "开启" 位置，使细胞不受控制地生长。Daraxonrasib 将以 Rasonque 品牌销售，其工作原理是使用一种新型 "分子胶" 机制，将不良蛋白质粘合到另一种健康蛋白质上，使其失活。在革命药物公司证明这种方法可行之前，尚不清楚这种方法是否安全实用。"令人兴奋的是，我们现在有了一种适用于大多数胰腺癌患者的靶向治疗，" 加利福尼亚州杜阿尔特市希望之城医疗中心的肿瘤内科医生文森特·钟（Vincent Chung）说，他负责治疗威廉姆斯。
“This is probably one of the greatest unmet needs in oncology,” says Incyte Corp. Chief Executive Officer Bill Meury, whose company in April started a final-stage trial of a drug that could be used to treat about 40% of pancreatic cancer patients with a particular type of KRAS mutation. The same month, Astellas Pharma Inc. dosed the first pancreatic cancer patient in a final-stage trial of its drug setidegrasib targeting the same subset of patients. Meanwhile, Erasca Inc. is planning final-stage trials next year for its daraxonrasib-style drug ERAS-O015, a compound it says is more potent than Revolution Medicines’ pill. A dark horse in the race is Immuneering Corp., whose drug blocks a related protein called MEK that
promotes the growth of tumors.
Incyte Corp. 首席执行官 Bill Meury 表示："这可能是肿瘤学领域最大的未满足需求之一。" 该公司于 4 月份开始了一种药物的最后阶段试验，该药物可用于治疗约 40% 具有特定类型 KRAS 突变的胰腺癌患者。同月，安斯泰来制药公司 (Astellas Pharma Inc.) 在针对同一患者群体的药物 setidegrasib 的最后阶段试验中，对第一位胰腺癌患者进行了给药。与此同时，Erasca Inc. 计划明年对其 daraxonrasib 类药物 ERAS-OO15 进行最后阶段试验，据称该化合物比 Revolution Medicines 的药丸更有效。这场竞赛中的一匹黑马是 Immuneering Corp.，该公司的药物可以阻断一种名为 MEK 的相关蛋白质，这种蛋白质可以促进肿瘤的生长。
So far, Revolution Medicines is in the lead. Even before the Food and Drug Administration had completed its review of daraxonrasib for previously treated pancreatic cancer, the drugmaker provided the treatment at no cost to more than 2,000 US patients, including Williams, through a special expanded-access program for people who didn’t qualify for ongoing trials. The company has begun additional final-stage trials that could expand use of the drug to patients with earlier stages of pancreatic cancer as well as advanced lung cancer. “We are launch-ready. This is something we have been preparing for a long time. Our engines are revving,” says Revolution Medicines CEO Mark Goldsmith. Until recently, he says, “pretty much everyone viewed pancreatic cancer as a place drugs go to die.”
到目前为止，Revolution Medicines 处于领先地位。甚至在美国食品和药物管理局完成对 daraxonrasib 用于既往治疗的胰腺癌的审查之前，这家制药商就通过一项针对不符合正在进行的试验资格的患者的特殊扩大访问计划，免费为包括 Williams 在内的 2,000 多名美国患者提供了治疗。该公司已经开始了额外的最终阶段试验，可以将该药物的使用范围扩大到早期胰腺癌和晚期肺癌患者。"我们已经做好上市准备。这是我们已经准备了很长时间的事情。我们的引擎正在加速运转，"Revolution Medicines 首席执行官马克·戈德史密斯 (Mark Goldsmith) 说道。他说，直到最近，“几乎每个人都认为胰腺癌是药物致死的地方。”
Revolution Medicines reported drew takeover interest from several large pharma players early this year, before its shares surged when positive results from the latest daraxonrasib trial were first announced.With a current market capitalization of around $45 billion, the Redwood City, California-based company’s valuation is too rich for all but a handful
of potential buyers, especially factoring in the type of lofty premium that’s typical in biotech takeovers.
据报道，Revolution Medicines 今年年初吸引了几家大型制药公司的收购兴趣，随后最新 daraxonrasib 试验的积极结果首次公布后，该公司股价飙升。这家总部位于加利福尼亚州雷德伍德城的公司目前的市值约为 450 亿美元，除了少数潜在买家外，其估值对于所有人来说都太高了，尤其是考虑到生物技术收购中常见的高额溢价。
Revolution Medicines, founded in 2014, has retained most rights to daraxonrasib and several other drugs in its portfolio that target RAS (of which KRAS is a subset), outside of certain Asian markets, meaning it will also get to keep the sales revenue as the medicines are approved. In late 2023, when some investors were still skeptical about daraxonrasib’s potential, the company did an unusual all-stock transaction to buy a struggling biotech company called EQRx Inc., to gain access to its $1.1 billion pile of cash. That money helped it complete costly Phase III trials on its own, Goldsmith says.
Revolution Medicines 成立于 2014 年，在某些亚洲市场之外，保留了 daraxonrasib 和其产品组合中其他几种针对 RAS（其中 KRAS 是其中一个子集）的药物的大部分权利，这意味着在这些药物获得批准后，该公司还将保留销售收入。2023 年末，当一些投资者仍对 daraxonrasib 的潜力持怀疑态度时，该公司进行了一次不同寻常的全股票交易，收购了一家陷入困境的生物技术公司 EQRx Inc.，以获得其 11 亿美元的现金。戈德史密斯说，这笔钱帮助它自行完成了昂贵的第三阶段试验。
Unlike some targeted cancer drugs that work for only a narrow subset of patients, daraxonrasib is effective against all forms of the RAS protein. That gives it potential to treat the vast majority of pancreatic cancer cases, as well as other tumors such as lung cancer, another disease often linked to the mutated gene. Because patients eventually develop resistance to the drug, researchers are racing to find combination treatments that have longer-lasting effects.
与某些仅对一小部分患者有效的靶向癌症药物不同，daraxonrasib 对所有形式的 RAS 蛋白均有效。这使得它有可能治疗绝大多数胰腺癌病例以及其他肿瘤，例如肺癌，另一种通常与突变基因有关的疾病。由于患者最终会对药物产生耐药性，因此研究人员正在竞相寻找具有更持久效果的联合治疗方法。
In July, Revolution Medicines released data from an early trial showing that combining daraxonrasib with zoldonrasib, another targeted pill it developed, shrunk tumors in half of patients, better than daraxonrasib can do alone. A final-stage trial of this combo has been initiated. In June, Tango Therapeutics Inc. reported that tumors shrank in 11 of 12 pancreatic cancer patients who received daraxonrasib in combination with its own drug vopimetostat, which targets a different growth-promoting gene present in a subset of pancreatic cancer patients. Tango says it’s planning a final-stage trial.
今年 7 月，Revolution Medicines 发布的一项早期试验数据显示，将 daraxonrasib 与其开发的另一种靶向药物 zoldonrasib 结合使用，可以缩小一半患者的肿瘤，效果比单独使用 daraxonrasib 效果更好。该组合的最后阶段试验已经启动。6 月，Tango Therapeutics Inc. 报告称，接受 daraxonrasib 联合自有药物 vopimetostat 治疗的 12 名胰腺癌患者中，有 11 名患者的肿瘤缩小了，该药物针对的是胰腺癌患者亚群中存在的一种不同的生长促进基因。Tango 表示正在计划进行最后阶段的试验。
Williams, the former film executive, knows daraxonrasib is unlikely to be a cure for him. But he’s optimistic the drug could buy him enough time for even more effective regimens to be developed. “They are making so much progress now in pancreatic cancer,” he says. “Who knows what can happen in the next 12 months to 18 months?”
前电影高管威廉姆斯知道达拉克松拉西布不太可能治愈他。但他乐观地认为这种药物可以为他赢得足够的时间来开发更有效的治疗方案。"他们现在在胰腺癌方面取得了很大进展，" 他说。“谁知道未来 12 个月到 18 个月会发生什么？”
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