Asciminib Hydrochloride

▎Armstrong 2025年12月8日，Terns Pharmaceuticals更新了TERN-701治疗CML的最新临床数据。 从11月3日首次披露TERN-701治疗CML初步临床数据以来，Terns Pharmaceuticals的股价已经涨了5倍，市值如今达到36亿美元。 TERN-701为一款高选择性的BCR-ABL变构抑制剂，对于ABL1、ABL2的选择性超过10000倍，比其他活性位点TKI的选择性高450倍。 CML目前标准治疗包括活性位点TKI和变构TKI如Asciminib，其中Asciminib占据主要市场份额，预计销售峰值超过30亿美元。 不过，CML领域仍然存在严重未满足临床需求，Asciminib治疗仍有很多患者无法达到MMR（主要分子学反应）。 TERN-701具有更高选择性、更好的体内靶点覆盖和更宽的治疗窗，有望成为BIC。 TERN-701治疗24周，获得历史最佳MMR数据，且对于Asciminib及其他TKI治疗后的患者仍然有60-70%的MMR。 320mg以上剂量组达到75%的MMR。 BIC地位已经非常清晰。 Terns Pharmaceuticals将尽快启动三期临床。 此次ASH公布数据比11月初披露的增加了6例，其中4例达到MMR。 在获得突破性疗效的同时，TERN-701仍然保持了极佳的安全性数据。 总结 今年10月，Terns Pharmaceuticals小分子GLP-1不达预期，已经终止开发，市值跌到6亿美元。仅仅一个月之后，新一代BCR-ABL变构抑制剂公布突破性临床数据，并有望快速推进到三期临床，这一突破性临床数据助推Terns的市值从6亿美元快速攀升至36亿美元。 Armstrong技术全梳理系列 GPRC5D靶点全梳理； CD40靶点全梳理； CD47靶点全梳理； 补体靶向药物技术全梳理； 补体药物：眼科治疗的重要方向； Claudin 6靶点全梳理； Claudin 18.2靶点全梳理； 靶点冷暖，行业自知； 中国大分子新药研发格局； 被炮轰的“me too”； 佐剂百年史； 胰岛素百年传奇； CUSBEA：风雨四十载； 中国新药研发的焦虑； 中国生物医药企业的研发竞争； 中国双抗竞争格局； 中国ADC竞争格局； 中国双抗技术全梳理； 中国ADC技术全梳理； Ambrx技术全梳理； Vir Biotech技术全梳理； Immune-Onc技术全梳理； 亘喜生物技术全梳理； 康哲药业技术全梳理； 科济药业技术全梳理； 恺佧生物技术全梳理； 同宜医药技术全梳理； 百奥赛图技术全梳理； 腾盛博药技术全梳理； 创胜集团技术全梳理； 永泰生物技术全梳理； 中国抗体技术全梳理； 德琪医药技术全梳理； 德琪医药技术全梳理2.0； 和铂医药技术全梳理； 荣昌生物技术全梳理； 再鼎医药技术全梳理； 药明生物技术全梳理； 恒瑞医药技术全梳理； 豪森药业技术全梳理； 正大天晴技术全梳理； 吉凯基因技术全梳理； 基石药业技术全梳理； 百济神州技术全梳理； 百济神州技术全梳理第2版； 信达生物技术全梳理； 信达生物技术全梳理第2版； 中山康方技术全梳理； 复宏汉霖技术全梳理； 先声药业技术全梳理； 君实生物技术全梳理； 嘉和生物技术全梳理； 志道生物技术全梳理； 道尔生物技术全梳理； 尚健生物技术全梳理； 康宁杰瑞技术全梳理； 科望医药技术全梳理； 岸迈生物技术全梳理； 礼进生物技术全梳理； 康桥资本技术全梳理； 余国良的抗体药布局； 荃信生物技术全梳理； 安源医药技术全梳理； 三生国健技术全梳理； 仁会生物技术全梳理； 乐普生物技术全梳理； 同润生物技术全梳理； 宜明昂科技术全梳理； 派格生物技术全梳理； 迈威生物技术全梳理； Momenta技术全梳理； NGM技术全梳理； 普米斯生物技术全梳理； 普米斯生物技术全梳理2.0； 三叶草生物技术全梳理； 贝达药业抗体药全梳理； 泽璟制药抗体药全梳理； 恒瑞医药抗体药全梳理； 齐鲁制药抗体药全梳理； 石药集团抗体药全梳理； 豪森药业抗体药全梳理； 华海药业抗体药全梳理； 科伦药业抗体药全梳理； 百奥泰技术全梳理； 凡恩世技术全梳理。

ORLANDO, Fla. — In October, Terns Pharmaceuticals made an announcement that seemed to run counter to every popular sentiment in biotech: It got out of obesity to focus on cancer. The 9.4% stock drop that day showed investors’ disappointment. But in the months since, the company’s decision has been more than proven to be the right one. Soon after dropping the obesity program, it released data for its rare leukemia treatment that has more than tripled the company’s value. And it believes it may be able to compete with an emerging Novartis blockbuster. On Monday, Terns announced updated data from the leukemia program, called TERN-701, showing that 64% of patients in its trial hit a benchmark known as major molecular response after earlier drugs stopped working. The data were an update from the release in November, and from the American Society of Hematology’s annual meeting, that helped drive up the stock. In an interview with Endpoints News at ASH, Terns CEO Amy Burroughs said that trying to move forward in obesity would have required a partnership, and the company was more confident — and had better data — in chronic myeloid leukemia. “It was easier for us that it wasn’t gray. It just clearly was not a best-in-class type of asset,” Burroughs said. “We’d already planned, from an operational standpoint, to move people into oncology, and so it wasn’t very disruptive to the company. It really was just a loss of potential.” There are about 17,000 new CML diagnoses a year in G7 countries, and Novartis’ drug Scemblix — the product Terns believes it can compete with — is on pace to reach $1 billion in full-year sales for the first time in 2025. Success in CML patients is not measured the same way as most other oncology studies, because the disease is a slow-progressing cancer and patients can live with it for years, trial investigator David Andorsky told Endpoints. Rather than using progression-free survival or overall survival, clinicians use the major molecular response, or MMR, metric to track deep responses over long periods of time. Patients achieve a major response when the ratio of a genetically mutated protein, compared to all healthy cells, falls below 0.1%. In CML, the mutation is caused by an abnormality between chromosomes 9 and 22, promoting the overproduction of white blood cells. The 38 evaluable patients in the trial had at least two prior treatments: 28 entered who had cancer that had worsened already, while 10 were still in a major response. Of the patients whose cancer had resurged, 64% reached a major response within 24 weeks of starting TERN-701. All 10 who started the trial with a major response kept it, and in some cases their response got even deeper. That appears to be stronger than Novartis’ results at the same time point: In 2020, Scemblix had a 25.5% major response rate at 24 weeks, though the trial was larger and placebo-controlled. The study eventually ran for 96 weeks and hit a rate of 37.6%. The last two decades have revolutionized care for CML. Before 2001, the only option for patients was a bone marrow transplant, Andorsky said. That changed with the approval of Novartis’ Gleevec, which shifted treatment to one or two pills per day — and much longer prognoses. “Within five years, patients were gonna die from it, versus ‘Take this drug every day, and you’ll live a normal life,’” Andorsky said of Gleevec. “That was really paradigm-shifting.” Novartis has a substantial head start — Scemblix was first approved in 2021. And Terns wants to try and compete solo: Burroughs said she believes Terns can take the drug to market itself and that it’s “unlikely” the company seeks a partner. Terns is now deciding between one of two doses to take into pivotal trials, the first of which will likely start by the end of 2026 or early 2027. The company is also still considering whether to test TERN-701 directly against Scemblix in its studies. For second-line-plus patients, Burroughs said the company thinks it can conduct a study similar to the one Novartis ran for Scemblix, enrolling about 250 patients and comparing TERN-701 to second-generation TKI inhibitors. But Burroughs was non-committal about a head-to-head study against Scemblix in the first-line setting. “We definitely want to get that data. We could do that in a pivotal trial, we could do that in other ways,” Burroughs said. The company will likely raise more money to fund its studies. Terns has about $300 million in cash that should last it into 2028, but that’s not enough for Terns to support multiple studies and a commercial launch. “We’re likely to raise money sometime before we start pivotals,” she said.