Venetoclax

Strengthened balance sheet with successful completion of upsized $168.3 million initial public offering Announced FDA Fast Track designation for BH-30643 for the treatment of advanced EGFR C797S-positive NSCLC SAN DIEGO, Sept. 18, 2026 (GLOBE NEWSWIRE) -- BlossomHill Therapeutics, Inc. (Nasdaq: BLSM), a clinical-stage biopharmaceutical company applying an intentional, chemistry-based approach to design and develop innovative small molecule medicines for the treatment of cancer, today announced financial results for the second quarter 2026 and highlighted recent progress. “We’ve achieved meaningful progress across our pipeline, as well as our significant corporate milestones, since the beginning of the second quarter,” said Jean Cui, Ph.D., Founder, President and Chief Executive Officer of BlossomHill Therapeutics. “In April, we presented our first preclinical data from our pseudo-irreversible pan-KRAS inhibitor BH-501284, built on a novel chemical scaffold, at AACR where we highlighted the sustained target engagement leading to tumor regression at low dose levels. At ASCO in early June we presented the preliminary safety, PK and antitumor activities of BH-30643 in Phase 1 dose escalation of the SOLARA trial, along with the initial efficacy data in C797S-positive NSCLC. More recently we announced that BH-30643 received Fast Track designation, an important regulatory milestone that reflects the FDA's recognition of the potential for this molecule. We also presented encouraging safety data and early signs of anti-leukemic activity observed with BH-30236, our novel macrocyclic CLK inhibitor, both as a monotherapy and in combination with venetoclax, at EHA in the middle of June. We are now looking forward to our end-of-phase 1 meeting with the FDA later this year. With a strong balance sheet following our successful initial public offering in August, we believe we are well positioned to deliver important clinical and regulatory milestones over the coming quarters as we continue advancing our intentionally designed medicines that address significant unmet medical needs in cancer treatment.” Recent Business Highlights and Corporate Updates: Strengthened the balance sheet with approximately $168.3 million in gross proceeds from the initial public offering (IPO) in August 2026 Announced the U.S. Food and Drug Administration (FDA) granted Fast Track designation to BH-30643, a macrocyclic OMNI-EGFR™ inhibitor, for the treatment of adult patients with advanced or metastatic epidermal growth factor receptor (EGFR) C797S-positive non-small cell lung cancer (NSCLC) after prior treatment with a third-generation EGFR tyrosine kinase inhibitor (TKI) Presented preliminary results of BH-30643 from dose escalation and backfill cohorts in the ongoing Phase 1/2 SOLARA trial in advanced or metastatic EGFR-mutant NSCLC at the American Society of Clinical Oncology (ASCO) 2026 annual meeting, and additional follow up data at IASLC 2026 World Conference on Lung Cancer, which highlighted a 45% objective response rate and 88% disease control rate observed in patients with C797S resistance to prior TKIs, with or without concurrent T790M mutation Presented the first preclinical data from the pseudo-irreversible pan-KRAS inhibitor BH-501284, built on a novel chemical scaffold, at the American Association for Cancer Research (AACR) 2026 annual meeting Presented initial clinical data from the ongoing first-in-human Phase 1/1b trial of BH-30236, an orally bioavailable, macrocyclic CDC-like kinase (CLK) inhibitor, in relapsed or refractory acute myeloid leukemia (R/R AML) and higher-risk myelodysplastic syndromes (HR-MDS) at the European Hematology Association (EHA) 2026 Congress Expanded the Company’s Board of Directors with the appointments of Sheila Gujrathi, M.D., and John Schmid Anticipated Upcoming Milestones: BH-30643 Q4 2026: End of Phase 1 meeting regarding a recommended Phase 2 dose selection and a potential accelerated approval pathway in C797S resistance Q1 2027: First patient dosed in anticipated pivotal Phase 2 trial 1H 2027: Updated Phase 1 data, including C797S durability 2H 2027: Updated Phase 1 data on TKI-naive durability and initial chemo combo cohort data BH-501284 Q1 2027: Investigational New Drug submission BH-30236 1H 2027: Updated Phase 1 data on safety and anti-leukemic effect Second Quarter 2026 Financial Results Research and development (R&D) expenses for the second quarter of 2026 were $21.4 million, compared with $12.5 million for the same period in 2025. The increase was primarily due to greater clinical development expenses driven by the SOLARA trial, expenses to support IND-enabling studies for BH-501284, and greater costs related to personnel, facilities and other overhead. General and administrative (G&A) expenses for the second quarter of 2026 were $3.3 million, compared with $1.6 million for the same period in 2025. The increase was primarily due to greater legal expenses, personnel-related expenses and overhead. Net loss for the second quarter of 2026 was $23.8 million, or $(8.75) per basic and diluted share, compared with a net loss of $13.2 million, or $(5.51) per basic and diluted share for the same period in 2025. The increase in net loss was primarily attributable to increased operating expenses. Cash and cash equivalents totaled $95.4 million as of June 30, 2026. BlossomHill subsequently completed its IPO in August 2026 in which it sold 10,516,240 shares of its common stock, including partial exercise of the over-allotment option, for gross proceeds of $168.3 million. BlossomHill believes that its cash and cash equivalents as of June 30, 2026, together with the proceeds from its IPO, will be sufficient to fund its operations into the second quarter of 2028. About BH-30643 BH-30643 is an investigational, novel, orally bioavailable, non-covalent, macrocyclic, brain active, mutant-selective, OMNI-EGFR™ inhibitor for the treatment of EGFR -mutant NSCLC. BH-30643 was designed to overcome the limitations of currently approved EGFR inhibitors, which were discovered over a decade ago without the current, modern understanding of the structure and protein dynamics of mutant EGFRs. In preclinical studies, BH-30643 demonstrated potent inhibitory activity across diverse EGFR mutation categories – classical mutations, on-target resistance mutations such as C797S with or without T790M, atypical mutations and exon 20 insertions – while maintaining marked selectivity over wild-type EGFR. BH-30643 has received Fast Track designation and is being evaluated in SOLARA, a global Phase 1/2, first-in-human clinical trial spanning more than 40 sites in 10 countries. Ongoing dose expansion cohorts are enrolling in both TKI-pretreated and TKI-naive settings, including a C797S resistance cohort. For additional information on SOLARA, including a list of study sites and how to enroll, please visit clinicaltrials.gov ( NCT06706076 ). About BH-30236 BH-30236 is an investigational orally bioavailable, macrocyclic inhibitor of the CDC-like kinase (CLK) family. BH-30236 was intentionally designed to potently inhibit CLK, leading to modulation of aberrant alternative splicing in cancerous tissue, targeting the same aberrant splicing machinery that drives relapsed or refractory (R/R) acute myeloid leukemia (AML) and higher-risk myelodysplastic syndromes (HR-MDS) disease biology and that cancer cells exploit to develop resistance to venetoclax, FLT3 inhibitors and cytarabine. BH-30236 is being evaluated in a Phase 1/1b multicenter, open-label, first-in-human dose escalation and expansion trial in adults with R/R AML and HR-MDS. The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BH-30236 for the treatment of AML. For additional information on this trial, including a list of study sites and how to enroll, please visit clinicaltrials.gov ( NCT06501196 ). About BH-50 1284 BH-501284 is an investigational, orally bioavailable pan-KRAS inhibitor, which utilizes a novel Switch-II chemical scaffold to achieve prolonged, potent and selective inhibition of KRAS mutations. We believe this molecule, which uses a non-covalent scaffold, is unique in its potential to achieve tight and durable binding, a feature described as “pseudo-irreversible” binding. In preclinical studies, BH-501284 has achieved pseudo-irreversible binding characteristics with high binding affinity, while maintaining high selectivity for KRAS. About BlossomHill Therapeutics BlossomHill Therapeutics, Inc. is a clinical-stage biopharmaceutical company applying an intentional, chemistry-based approach to design and develop innovative small molecule medicines that address significant unmet medical needs in cancer treatment. Founded and led by industry veteran J. Jean Cui, Ph.D., with her proven track record in oncology drug design and development – including three FDA-approved drugs – BlossomHill Therapeutics applies cutting-edge science with a goal to address key oncogenic drivers and improve patient outcomes in difficult-to-treat cancers. The company’s lead clinical program is BH-30643, an investigational, non-covalent, macrocyclic, brain active, mutant-selective OMNI-EGFR TM inhibitor for the treatment of EGFR-mutant non-small cell lung cancer (NSCLC), which has received Fast Track designation for the C797S resistance population after 3rd generation EGFR TKI treatment. The company is also conducting clinical development of BH-30236, an investigational macrocyclic CDC-like kinase (CLK) inhibitor initially being studied in a clinical trial for the treatment of relapsed or refractory acute myeloid leukemia (R/R AML) and higher-risk myelodysplastic syndromes (HR-MDS). The company’s pipeline also includes BH-501284, a preclinical, non-covalent, selective, pan-KRAS Switch-II inhibitor for potential future development in diverse KRAS-mutant tumors. BlossomHill Therapeutics is headquartered in San Diego, California. For more information, visit bhtherapeutics.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, as amended, and other federal securities laws, including, without limitation, statements regarding: the therapeutic potential, clinical benefits, safety and potential competitive differentiation of the company's product candidates, including BH-30643, BH-30236 and BH-501284; the anticipated benefits of regulatory designations received, or that may be received, by the company's product candidates; the design, enrollment, timing, progress and results of the company's clinical trials and preclinical studies; the company's planned regulatory interactions and submissions; anticipated program milestones, including the timing of program and data updates; the period over which the company estimates its existing cash and cash equivalents, together with the net proceeds from its initial public offering, will be sufficient to fund its current operating plan; statements by the company's management; and the company's development plans and continued advancement of its pipeline. The words "anticipate," "believe," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "upcoming," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially, including, without limitation: the company's limited operating history, history of significant losses and the early stage of development of its product candidates; the risk that preliminary and interim clinical data are subject to further analysis and may not be predictive of, may be inconsistent with, or may be more favorable than, data generated as clinical trials continue or data from future clinical trials; uncertainties inherent in the initiation, timing, design and enrollment of clinical trials, and the availability and timing of data from ongoing and future trials; the company's ability to successfully demonstrate the safety and efficacy of its product candidates and to obtain and maintain regulatory approvals; the timing and outcome of planned interactions with, and submissions to, the FDA and other regulatory authorities, including whether an accelerated approval pathway will be available to the company; the risk that regulatory designations, including Fast Track and orphan drug designation, may not result in a faster development, review or approval process, may not increase the likelihood of regulatory approval, and may be withdrawn; competition from third parties that are developing products for similar indications; the prior success of the company’s management team may not be indicative of future success; the company's reliance on third parties, including contract research organizations and contract manufacturing organizations; the company's ability to obtain, maintain and protect its intellectual property; and the company's need for additional financing and its estimates regarding operating expenses and capital requirements. These and other risks are described in greater detail under the heading "Risk Factors" in the company's filings with the Securities and Exchange Commission (the “SEC”), its Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, which will be filed with the SEC later today, as well as in the company's subsequent filings with the SEC. Any forward-looking statements represent the company's views only as of the date of this press release, and the company expressly disclaims any obligation to update any forward-looking statements, except as required by law. Company Contact: Michael Moore, BlossomHill Therapeutics michael.moore@bhtherapeutics.com Media: Ashlea Kosikowski, 1AB ashlea@1abmedia.com BLOSSOMHILL THERAPEUTICS, INC. UNAUDITED CONDENSED BALANCE SHEETS (in thousands, except share and par value data) June 30, 2026 December 31, 2025 (unaudited) Assets Current Assets: Cash and cash equivalents $ 95,376 $ 136,682 Prepaid expenses and other current assets 5,592 2,144 Total current assets 100,968 138,826 Property and equipment, net 1,489 1,726 Operating lease right-of-use assets 19,197 19,921 Other assets 2,477 2,495 Total assets $ 124,131 $ 162,968 Liabilities, convertible preferred stock and stockholders' deficit Current liabilities: Accounts payable $ 3,930 $ 2,314 Accrued expenses 9,194 8,431 Accrued compensation 3,539 4,730 Other current liabilities 809 - Operating lease liability, current 1,149 5 Total current liabilities 18,621 15,480 Operating lease liability, non-current 22,222 22,393 Other long-term liabilities 1,245 - Total liabilities 42,088 37,873 Convertible preferred stock, $0.0001 par value; 85,552,612 shares authorized at June 30, 2026 and December 31, 2025; 18,258,960 shares issued and outstanding at June 30, 2026 and December 31, 2025; $257,153 aggregate liquidation preference at June 30, 2026 and December 31, 2025 256,823 256,823 Stockholders' deficit: Common stock, $0.0001 par value; 110,000,000 shares authorized at June 30, 2026 and December 31, 2025; 2,767,848 and 2,548,659 shares issued and outstanding as of June 30, 2026 and December 31, 2025, respectively 1 1 Additional paid-in capital 5,014 3,300 Accumulated deficit (179,795) (135,029) Total stockholders' deficit (174,780) (131,728) Total liabilities, convertible preferred stock and stockholders’ deficit $ 124,131 $ 162,968 BLOSSOMHILL THERAPEUTICS, INC. UNAUDITED CONDENSED STATEMENTS OF OPERATIONS (in thousands, except share and per share data) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 Operating expenses: Research and development $ 21,355 $ 12,530 $ 41,256 $ 22,102 General and administrative 3,315 1,573 5,521 3,455 Total operating expenses 24,670 14,103 46,777 25,557 Loss from operations (24,670 ) (14,103 ) (46,777 ) (25,557 ) Other income (expense), net: Interest income, net 915 859 2,017 1,835 Other expense, net (3 ) - (6 ) - Total other income, net 912 859 2,011 1,835 Net loss $ (23,758 ) $ (13,244 ) $ (44,766 ) $ (23,722 ) Net loss per share, basic and diluted $ (8.75 ) $ (5.51 ) $ (16.91 ) $ (9.90 ) Weighted average common shares outstanding, basic and diluted 2,713,696 2,405,140 2,646,721 2,395,789

CD47/SIRPα作为肿瘤免疫领域极具潜力的先天免疫通路，凭借激活巨噬细胞吞噬功能、逆转肿瘤免疫抑制微环境的独特机制，成为全球创新药赛道的核心布局方向。相较于T细胞免疫治疗的耗竭、实体瘤浸润不足等瓶颈，该通路依托髓系免疫激活机制，可有效实现“冷肿瘤转热”，适配多类实体瘤与血液瘤治疗场景，同时拓展自身免疫疾病治疗新空间，临床价值与商业化潜力突出。但赛道发展长期受限于核心技术痛点：CD47广泛表达于人体正常血细胞，早期单抗、初代融合蛋白等技术路线，普遍存在肿瘤靶向特异性不足、血液毒性偏高、疗效与安全性难以平衡的问题，大量早期项目因设计缺陷迭代出局，导致全球至今暂无获批药物，赛道处于“有靶点、无成药最优解”的竞争窗口期。 重回立项视野，原因在分子设计已迭代到第三代：谁能把吞噬信号精确限制在病变细胞、保住安全窗口，谁就拿到下一轮船票。 01○✦ CD47 的问题从来不在生物学，在分子设计 CD47 高表达于肿瘤细胞表面，与巨噬细胞上的 SIRPα 结合后释放“别吃我”信号。阻断这一信号，即可恢复巨噬细胞的清除能力。 CD47 在红细胞、血小板表面也广泛存在：与红细胞结合过强，到达肿瘤的有效浓度就不足；加大剂量，又带来难以耐受的血液学毒性。项目成败由分子设计而非生物学决定。 差距可量化：2014 年《Cancer Research》研究显示，野生型 SIRPα-Fc 亲和力处纳摩尔级、对人红细胞结合极弱，改为高亲和力突变体后结合显著增强。“亲和力越强越好”这条直觉，在 CD47 上失效。 赛道分化为：第一代是抗 CD47 单抗直接阻断，绕不开红细胞结合；第二代转向 SIRPα 侧，用融合蛋白做“诱饵受体”绕开毒性；第三代做成双抗或多重分子，把 CD47 通路与 PD-L1、VEGF 等绑定于同一分子。 机制层面还有一块新拼图。2025 年 10 月发表于《Science Immunology》的研究发现，SIRPα 除与 CD47 结合外，还与巨噬细胞表面的 CD18 顺式作用，形成“双锁”抑制；同时阻断顺式与反式两条作用通路才能最大化吞噬作用与抗癌效应。设计思路正从“关掉一个信号”转向“重新编排吞噬程序”。 02○✦ 赛道被重新定义的三个信号 第一个信号在交易结构。2025 年 6 月 30 日，复宏汉霖与汉康生技控股子公司 FBD Biologics Limited 签署许可协议：1000 万美元首付款、共计 1.92 亿美元潜在里程碑付款及合作区域销售分成，获 HCB101 在中国（除台湾地区）、东南亚特定国家及中东和北非地区（MENA）特定国家的开发、生产及商业化独家许可，另获日本市场优先谈判权与一到两位数百分比计的特许权使用费。买方看中的不是更晚的阶段，而是 HCB101 在早期临床中的低红细胞结合特性——市场愿意为分子设计上的安全性验证付费。 第二个信号，是靶点正被放进更大框架里重新命名。MCE（Myeloid Cell Engager，髓系细胞衔接器）代表行业思路从“找靶点”切换到“调用免疫系统”。CAR-T 与 TCE 暴露了 T 细胞路径的边界：持续强激活易耗竭，实体瘤中又受浸润不足制约。MCE 换了兵种：动员巨噬细胞、单核细胞，重新打开被抑制的生理清除程序，释放 CXCL9、CXCL10 等趋化因子推动“冷肿瘤”转“热肿瘤”。这也是它被称为“原位免疫激活”一环的原因。 2024 年 7 月，诺华以 1.5 亿美元预付款（含 2500 万美元股权投资）及最高 28.5 亿美元里程碑款（合计最高 30 亿美元）引进 Dren Bio 多款髓系双抗；2025 年 3 月，赛诺菲以 6 亿美元首付款、最高 13 亿美元的开发与上市里程碑款收购髓系衔接器 DR-0201，于当年二季度完成交割；2025 年 11 月，GSK 与泽安生物（LTZ Therapeutics）以 5000 万美元预付款及后续里程碑、分级特许权使用费，共同开发最多 4 款首创性（First-in-Class）MCE 候选药物，聚焦血液瘤与实体瘤。三笔交易的共同点在于：巨头在规则尚未定型时就开始下注。 第三个信号，自身免疫正在成为这条通路的第二战场。部分自免疾病与异常 B 细胞相关，深度清除致病 B 细胞有望实现“免疫重置”，CAR-T 已展示这种可能性，但需要采集、制备、回输。MCE 的机会在于：用标准化生物药的形态，把更深层次的 B 细胞清除与更温和的体验放进同一方案。 03○✦ 竞争格局：谁站在第几代 截至 2026 年 9 月，全球尚无靶向 CD47/SIRPα 的药物获批：已卡位，却无赢家。 抗体路线。莱法利单抗（AK117，康方生物）经表位修饰后不诱导红细胞凝集；2026 年 EHA 年会公布其联合阿扎胞苷+维奈克拉一线治疗 AML 的 II 期随机双盲安慰剂对照数据（数据截至 2025 年 11 月）：试验组 ORR 80.0% vs 对照组 66.7%，9 个月 OS 率 78.7% vs 43.1%，中位 EFS 9.1 个月 vs 6.9 个月，HR 0.46。 SIRPα-Fc 融合蛋白路线。以 SIRPα 作“诱饵受体”，绕开抗体直接结合红细胞的风险，代表分子包括宜明昂科 IMM01、汉康生技 HCB101、ALX Oncology 的 Evorpacept。 双抗/多抗路线。把 CD47 通路与第二靶点（CD20、PD-L1 等）绑定于同一分子，代表分子为宜明昂科 IMM0306（CD47×CD20）、科望医药 ES019（PD-L1/SIRPα）、泽安生物 LTZ-301（靶向 CD79b）、Dren Bio DR-0201（靶向 CD20），均从髓系受体激活切入。 全球进度最快的几个资产里，中国企业占比明显，技术路线也不集中。 04○✦ CD47 赛道上的主要玩家 三条技术路线上都有企业把分子推进临床。以下按公开信息梳理部分代表性企业，不作排名。 宜明昂科 国内 SIRPα-Fc 融合蛋白路线的代表企业。IMM01（替达派西普）为中国首个进入临床阶段的 SIRPα-Fc 融合蛋白，通过双重作用机制激活巨噬细胞：既阻断 CD47/SIRPα“别吃我”信号，也经 Fc 段传递“吃我”信号；联合阿扎胞苷一线治疗 CMML 的 III 期注册临床已于 2026 年 6 月完成 173 例全部入组（公司于 7 月 16 日宣布），MDS、复发难治经典霍奇金淋巴瘤等适应症的 II 期数据已完成数据收集。公司同时向双抗与非肿瘤延展，自身免疫、肺动脉高压、减重增肌等管线已进入临床或 IND 阶段。 康方生物 其 CD47 单抗莱法利单抗（AK117）经表位修饰后不诱导红细胞凝集，无需预激即可全剂量爬坡。2026 年 EHA 年会公布其联合阿扎胞苷+维奈克拉一线治疗 AML 的 II 期随机双盲安慰剂对照数据（数据截至 2025 年 11 月）：试验组 ORR 80.0% vs 对照组 66.7%，9 个月 OS 率 78.7% vs 43.1%，中位 EFS 9.1 个月 vs 6.9 个月，HR 0.46。其联合依沃西一线治疗 PD-L1 阳性头颈鳞癌的 III 期已于 2024 年 10 月完成首例入组，是全球首个进入实体瘤注册性 III 期的 CD47 单抗，同法联合依沃西一线治疗胰腺癌的 III 期也在入组中；获 FDA 孤儿药资格认定（AML）。 汉康生技 自有 FBDB™（Fc-Based Designer Biologics）平台设计的 SIRPα-IgG4 Fc 融合蛋白 HCB101，经 AI 辅助结构建模，保留对肿瘤细胞 CD47 的结合能力，同时维持对红细胞 CD47 的低亲和力。单药 I 期剂量爬坡至 36 mg/kg 未确定最大耐受剂量，67 例受治者中不良事件多为 1—2 级、出现 1 例 3 级贫血，8 mg/kg 以上呈现持续的 ≥90% CD47 受体占有率；二线胃癌联合 ramucirumab 与 paclitaxel，2026 年 1 月 ASCO GI 公布的数据显示：13 例可评估患者中 5.12 mg/kg 与 8 mg/kg 剂量组 ORR 均达 100%，中剂量组（5.12—8 mg/kg）共 10 例中 8 例达部分缓解，最大肿瘤退缩 78.2%；2 月获 FDA 胃癌孤儿药资格。其与复宏汉霖的许可合作，使分子同时在中国（除台湾地区）、东南亚与中东和北非多地区推进。 复宏汉霖 2025 年 6 月自 FBD Biologics Limited 引进 HLX701（重组人 SIRPα-IgG4 Fc 融合蛋白），获中国（除台湾地区）、东南亚特定国家及中东和北非地区（MENA）特定国家的开发、生产及商业化独家许可。该分子在临床前与早期临床中选择性结合肿瘤细胞 CD47，对红细胞亲和力较低、不引起红细胞凝集。2026 年 1 月获 NMPA 批准开展联合西妥昔单抗和化疗治疗晚期 RAS/BRAF 野生型结直肠癌的 Ib/II 期临床，并于 2026 年 3 月 26 日完成首例患者给药。 泽安生物医药 基于自研 U-MCE™ 平台开发髓系细胞衔接器，核心品种 LTZ-301 为靶向 CD79b 的双特异性抗体，引导巨噬细胞与单核细胞清除 CD79b 阳性 B 细胞，正在美国多家临床中心开展 I 期研究（复发或难治非霍奇金淋巴瘤），第二款候选药物 LTZ-232（EpCAM × 髓系受体）已于 2026 年 7 月 7 日获 FDA 新药临床试验申请（IND）批准，拟用于晚期转移性结直肠癌（mCRC）及其他 EpCAM 高表达实体瘤，计划 2026 年四季度启动 I 期临床。2026 年 5 月完成 3800 万美元融资（高瓴创投领投），自 2022 年成立以来累计融资约 1.3 亿美元，近 10 个月内先后与礼来、GSK 达成合作。 科望医药 基于自有 BiME® 平台开发双特异性巨噬细胞衔接器，ES019 为 PD-L1/SIRPα 双特异性抗体（临床前阶段），从髓系受体激活切入，与传统抗体路线形成差异。 Dren Bio 围绕髓系细胞衔接器建立了多条管线，核心分子 DR-0201 为靶向 CD20 的双特异性 MCE，已进入自身免疫相关适应症的 I 期临床；公司的靶向髓系衔接器与吞噬平台已与多家国际药企达成授权合作。 ALX Oncology 其 SIRPα-Fc 融合蛋白 Evorpacept（ALX148）是该路线上全球进度较快的分子之一，在 HER2 阳性胃癌上开展的 II 期 ASPEN-06 随机试验中，全人群（n=127）ORR 41.3% vs 对照组 26.6%，确证 HER2 阳性人群（n=96）ORR 48.9% vs 24.5%、中位 PFS 7.5 vs 6.7 个月（HR 0.64）（该试验是首个在随机试验中显示出实质肿瘤缓解的 CD47 抑制剂）；2026 年 1 月 8 日，其 ASPEN-09-Breast 二期试验在经 T-DXd 治疗后进展的 HER2 阳性转移性乳腺癌中完成首例患者给药（入组 80 例，预计 2026 年三季度公布中期数据）。 这份名单完整呈现了一个靶点在十年里能分化出多少种分子设计选项：同一条信号通路，既可以被单抗直接阻断，也可以被诱饵受体绕开，还可以被重组进双抗与衔接器。 05○✦ 这套设计逻辑的立足点 这套逻辑已跳出单一公司的范围。两年间，围绕这条通路的交易从单分子拓展到平台化组合交易：买方不再只为一个分子下注，而是为“能不能稳定地造出一批同类分子”付费。 Dren Bio 在自身管线之外建立了一套能被反复调用的髓系衔接器平台：2024 年 7 月诺华以 1.5 亿美元预付款（含 2500 万美元股权投资）及最高 28.5 亿美元里程碑款引进其多款髓系双抗；2025 年 3 月赛诺菲又以 6 亿美元首付款、最高 13 亿美元开发与上市里程碑款收购 DR-0201，于当年二季度完成交割。两笔交易全部围绕同一套平台展开，而非某一个适应症。 泽安生物医药则以自研 U-MCE™ 平台连接两家跨国药企：2025 年以来，公司先后与礼来（2025 年 7 月）、GSK（2025 年 11 月）就髓系细胞衔接器（MCE）疗法达成合作，其中与 GSK 一项以 5000 万美元预付款及后续里程碑、分级特许权使用费的形式，共同开发最多 4 款首创性（First-in-Class）MCE 候选药物；同一平台上的 LTZ-301（靶向 CD79b 的双特异性抗体，处 I 期研究）则由公司自行推进。与此同时，复宏汉霖选择以许可引进的方式接入这条路径，持有 HLX701 在中国（除台湾地区）、东南亚与中东和北非多地区的开发、生产及商业化权益。买方愿意为早期资产付预付款，看重的是平台的重复产出能力。 这也让项目的评估标准发生了变化，首付款的高低要结合合作方的情况和项目所处的开发阶段来决定。首付款对应已验证的确定性，里程碑对应未来的想象空间。当一个靶点的天花板由分子设计而非靶点本身决定时，立项的重心就从“选什么靶点”前移到了“用什么结构去做”。能被验证的分子设计数据，比故事更能换成现金。 ---完---