MRT-2359

Interim clinical data from GFORCE-1 trial of NEK7-directed MGD MRT-8102 demonstrated profound CRP reductions in subjects with elevated CVD risk; readout of expanded GFORCE-1 trial in subjects with elevated CVD risk anticipated in H2 2026 Company expects to initiate multiple MRT-8102 Phase 2 studies, including in patients with elevated atherosclerotic risk in H2 2026, in patients with gout flares in Q4 2026/Q1 2027, and in patients with moderate to severe hidradenitis suppurativa in H1 2027 Presented positive interim Phase 1/2 clinical data of GSPT1-directed MGD MRT-2359 in combination with an AR inhibitor in mCRPC patients with AR mutations; initiation of Phase 2 study evaluating MRT-2359 in combination with apalutamide in mCRPC patients with AR mutations planned for Q3 2026 VAV1-directed MGD MRT-6160 advancing toward anticipated initiation by Novartis of multiple Phase 2 studies in immune-mediated diseases Preclinical data presented at AACR highlight a novel cyclin E1-directed MGD with superior selectivity and reduced off-target activity compared to CDK2 inhibitors; IND submission anticipated in H2 2026 Strong balance sheet with cash, cash equivalents, restricted cash, and marketable securities of $671 million, expected to support operations into 2029 BOSTON, May 07, 2026 (GLOBE NEWSWIRE) -- Monte Rosa Therapeutics, Inc. (Nasdaq: GLUE), a clinical-stage biotechnology company developing novel molecular glue degrader (MGD)-based medicines, today reported business highlights and financial results for the first quarter ended March 31, 2026. “We continue to make excellent progress advancing multiple programs through the clinic, with all three of our clinical-stage programs approaching Phase 2 trial initiations,” said Markus Warmuth, M.D., Chief Executive Officer of Monte Rosa Therapeutics. “Building on interim clinical data for our NEK7-directed MGD MRT-8102 demonstrating rapid, deep, and durable reductions in systemic inflammation, we expect to read out the GFORCE-1 study in subjects with elevated cardiovascular disease (CVD) risk this year, and to initiate three Phase 2 studies, starting in H2 2026, in diseases driven by the NLRP3/IL-1/IL-6 pathway. We also expect our collaborator Novartis to initiate multiple Phase 2 studies of our VAV1-directed MGD MRT-6160 in immune-mediated diseases this year. In addition, our oncology programs are also progressing rapidly, in particular with a Phase 2 study initiation of MRT-2359 in metastatic castration-resistant prostate cancer (mCRPC) patients with androgen receptor (AR) mutations planned for Q3 2026, following the encouraging Phase 1/2 data we presented at ASCO GU.” RECENT HIGHLIGHTS MRT-8102, NEK7-directed MGD for inflammatory diseases driven by the NLRP3 inflammasome, IL-1, and IL-6 In January, Monte Rosa announced positive interim data from an ongoing Phase 1 clinical study evaluating MRT-8102. In subjects with elevated CVD risk, after four weeks of MRT-8102 administration, CRP levels decreased by 85%, and 94% of study participants achieved CRP levels below 2 mg/L, a threshold associated with reduced CVD risk. The Company subsequently announced unblinded safety data from the single ascending dose / multiple ascending dose (SAD/MAD) cohorts and 3-month results from the ongoing long-term toxicology study in cynomolgus monkeys support a broad therapeutic index for MRT-8102. The ongoing GFORCE-1 study of MRT-8102 in subjects with elevated CVD risk is evaluating multiple dose levels to accelerate development in atherosclerotic cardiovascular disease (ASCVD) with an anticipated readout in H2 2026. Monte Rosa expects to initiate multiple Phase 2 studies of MRT-8102 in indications with high unmet need and strong biologic rationale for targeting the NLRP3/IL-1/IL-6 pathway: A study (GFORCE-2) of MRT-8102 in patients with elevated atherosclerotic risk is expected to initiate in H2 2026 to evaluate the effect of MRT-8102 treatment for 12 weeks (plus open-label extension) on CRP levels, as well as effects on liver fat, liver inflammation, and obesity. A study of MRT-8102 in patients with gout flares is expected to initiate in Q4 2026 or Q1 2027. A study of MRT-8102 in patients with moderate to severe hidradenitis suppurativa is expected to initiate in H1 2027. MRT-6160, VAV1-directed MGD for immune-mediated conditions Advancement of MRT-6160 toward multiple Phase 2 studies in immune-mediated diseases is ongoing, in collaboration with Novartis. Results from the Phase 1, single ascending dose / multiple ascending dose (SAD/MAD) study in healthy volunteers (clinicaltrials.gov identifier NCT06597799) support a clear path into anticipated Phase 2 studies and broad potential applications in multiple immune-mediated diseases. Monte Rosa has a global exclusive development and commercialization license agreement with Novartis to advance VAV1-directed MGDs, including MRT-6160. Monte Rosa is eligible to receive up to $2.1 billion in development, regulatory, and sales milestones, beginning upon initiation of Phase 2 studies. Novartis is responsible for conducting and funding Phase 2 studies. Monte Rosa will co-fund any Phase 3 clinical development and will share 30% of any profits and losses associated with the manufacturing and commercialization of MRT-6160 in the U.S., and is also eligible for tiered royalties on ex-U.S. net sales. MRT-2359, GSPT1-directed MGD for metastatic CRPC In February, Monte Rosa presented additional interim data from an ongoing Phase 1/2 clinical study evaluating MRT-2359 in combination with enzalutamide in heavily pretreated patients with mCRPC at the ASCO Genitourinary Cancers Symposium (ASCO GU). MRT-2359 is an investigational, orally bioavailable, GSPT1-directed MGD. PSA responses in patients with AR mutations expanded to 5 of 5 patients, with a 100% disease control rate, including 2 patients with RECIST partial responses and 3 with stable disease, all showing reductions in size of target lesions. Across all 15 evaluable patients, the overall RECIST disease control rate was 67%, and 10 of 15 patients showed tumor size reductions of target lesions. The combination of MRT-2359 and enzalutamide was generally well-tolerated with primarily Grade 1-2 AEs. There were no treatment discontinuations due to AEs. The Company plans to initiate a Phase 2 study (MODeFIRe-1) in 2026 of up to 25 patients to efficiently assess the efficacy of MRT-2359 in combination with the second-generation AR inhibitor apalutamide in mCRPC patients with AR mutations, with potential to expand the study into additional patient subsets. In March, Monte Rosa announced it entered into a supply agreement with Johnson & Johnson to evaluate MRT-2359 in combination with ERLEADA® (apalutamide) for the treatment of patients with mCRPC with androgen receptor (AR) mutations in its planned Phase 2 study. Cyclin E1 (CCNE1)-directed MGD program for CCNE1-amplified solid tumors Monte Rosa presented preclinical data on the potential of its potent and highly selective cyclin E1 (CCNE1)-directed molecular glue degrader, MRT-55811, to treat CCNE1-amplified solid tumors at the American Association for Cancer Research (AACR) Annual Meeting 2026. MRT-55811 induced deep tumor regressions in CCNE1-amplified in vivo models of ovarian, breast, and gastric cancers, and demonstrated superior selectivity and reduced off-target activity compared to CDK2 inhibitors. Monte Rosa expects to submit an IND application for its CCNE1 MGD program in H2 2026. The Company expects to develop this molecule in ovarian cancer and other cancer types driven by CCNE1 amplification. CDK2-directed MGD program for ER+ breast cancer Monte Rosa continues to advance its CDK2-directed MGD program for the treatment of ER+ breast cancer toward clinical development. Corporate In January, Monte Rosa closed an upsized underwritten public offering. Gross proceeds, before deducting underwriting discounts and commissions and offering expenses, were $345 million. Monte Rosa continues to progress its collaboration with Novartis to develop novel degraders for immune-mediated diseases and its collaboration with Roche to discover and develop MGDs against targets in cancer and neurological diseases previously considered impossible to drug. ANTICIPATED UPCOMING MILESTONES AND DEVELOPMENT PRIORITIES Immunology and Inflammation programs Readout of MRT-8102 GFORCE-1 study in subjects with elevated CVD risk anticipated in H2 2026. Initiate multiple Phase 2 studies of MRT-8102, including in elevated atherosclerotic risk patients in H2 2026, in gout flare patients in Q4 2026/Q1 2027, and in hidradenitis suppurativa patients in H1 2027. Submit an IND application for a second-generation NEK7-directed MGD in H2 2026. Monte Rosa expects its collaborator, Novartis, to initiate multiple Phase 2 studies of VAV1-directed MGD MRT-6160 in immune-mediated diseases in 2026. Oncology programs Initiate the MODeFIRe-1 Phase 2 study of MRT-2359 in combination with apalutamide in mCRPC in Q3 2026. Submit an IND application for a cyclin E1-directed MGD in H2 2026. FIRST QUARTER 2026 FINANCIAL RESULTS Collaboration Revenue: Collaboration revenue for the first quarter of 2026 was $4.2 million, compared to $84.9 million for the first quarter of 2025. Collaboration revenue represents amounts earned from the Company’s collaboration and license agreements with Roche and Novartis. Research and Development (R&D) Expenses: R&D expenses for the first quarter of 2026 were $44.1 million, compared to $32.2 million for the first quarter of 2025. These increases were driven by increased spending during the quarter on our MRT-8102 program and on other development and discovery programs. General and Administrative (G&A) Expenses: G&A expenses for the first quarter of 2026 were $10.2 million, compared to $8.7 million for the first quarter of 2025. These increases, which include non-cash stock-based compensation, were driven by increased headcount and expenses in support of our growth and operations as a public company. Net Loss: Net loss for the first quarter of 2026 was $44.5 million, compared to $46.9 million net income for the first quarter of 2025. Cash Position and Financial Guidance: Cash, cash equivalents, restricted cash, and marketable securities as of March 31, 2026, were $671.2 million, compared to cash, cash equivalents, restricted cash, and marketable securities of $382.1 million as of December 31, 2025. The increase of $289.1 million was primarily due to net proceeds from the underwritten public offering in January, partially offset by operational use of cash. In January 2026, the Company closed an underwritten public equity offering of $345.0 million aggregate gross proceeds. Aggregate net proceeds from the offering after deducting underwriting discounts and commissions and offering expenses were $323.8 million. Based on current cash, cash equivalents, restricted cash, and marketable securities, together with the proceeds from the January 2026 offering, the Company expects its cash and cash equivalents to be sufficient to fund planned operations and capital expenditures into 2029. About Monte Rosa Monte Rosa Therapeutics is a clinical-stage biotechnology company developing highly selective molecular glue degrader (MGD) medicines for patients living with serious diseases. MGDs are small molecule protein degraders that have the potential to treat many diseases that other modalities, including other degraders, cannot. Monte Rosa’s QuEEN™ (Quantitative and Engineered Elimination of Neosubstrates) discovery engine combines AI-guided chemistry, diverse chemical libraries, structural biology, and proteomics to rationally design MGDs with unprecedented selectivity. Monte Rosa has developed the industry’s leading pipeline of first-in-class and only-in-class MGDs, spanning autoimmune and inflammatory diseases, oncology, and beyond, with three programs in the clinic. Monte Rosa has ongoing collaborations with leading pharmaceutical companies in the areas of immunology, oncology, and neurology. For more information, visit . Forward-Looking Statements This communication includes express and implied “forward-looking statements,” including forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that are not historical facts and in some cases, can be identified by terms such as “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “objective,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue,” “ongoing,” or the negative of these terms, or other comparable terminology intended to identify statements about the future. Forward-looking statements contained herein include, but are not limited to, statements about our ability to grow our product pipeline, our ability to successfully complete research and further development and commercialization of our drug candidates in current or future indications, including the timing and results of our clinical trials and our ability to conduct and complete clinical trials, statements regarding our progress and speed of development of only-in-class and first-in-class molecular glue degrader therapeutics, statements around the Company’s QuEEN™ discovery engine and the broad potential applications of the platform and the Company’s ability to create long-term value through focused pipeline execution and strategic collaborations, as well as to expand the targetable protein space for MGD drug discovery, statements about the Company’s view of its potential to rationally design MGDs with unprecedented selectivity, statements about the advancement and timeline of our preclinical and clinical programs, pipeline and the various products therein, including (i) the ongoing development of our VAV1-directed degrader, referred to as MRT-6160, statements related to its clear path into anticipated Phase 2 studies in collaboration with Novartis and our expectations regarding the broad potential applications in multiple immune-mediated diseases, as well as our expectation that our collaborator, Novartis, will initiate multiple Phase 2 studies of VAV1-directed MGD MRT-6160 in immune-mediated diseases in 2026, (ii) the ongoing development and progress of our NEK7-directed MGD, referred to as MRT-8102, including our expectations regarding anticipated readout of data of the GFORCE-1 study in subjects with elevated CVD risk in H2 2026, as well as our plans to target initiation of multiple Phase 2 studies of MRT-8102, including in elevated atherosclerotic risk patients in H2 2026, in gout flare patients in Q4 2026/Q1 2027, and in hidradenitis suppurativa patients in H1 2027, (iii) the ongoing development of a second-generation NEK7-directed MGD and our statements around targeted IND submission in H2 2026, (iv) our ongoing clinical development of MRT-2359, statements relating to our plans to target initiation of the MODeFIRe-1 Phase 2 study of MRT-2359 in combination with the second-generation androgen receptor inhibitor apalutamide in mCRPC in Q3 2026 and the potential to expand the study into additional patient subsets, (v) statements around the progress of both our CDK2 and cyclin E1-directed MGD programs, including statements around the targeted timing of submission of an IND application in H2 2026 for a cyclin E1-directed MGD, and statements regarding the expected potential clinical benefit of MRT-55811 in CCNE1-amplified solid tumors, as well as statements related to the expected potential clinical benefit of any of our candidates, advancement and application of our platform, statements around our ability to capitalize on and potential benefits resulting from our research and translational insights, including announcements related to preclinical programs, as well as our ability to optimize collaborations with industry partners on our development programs, including our collaborations with Novartis and Roche, statements about obligations under our collaboration agreements, expectations around the receipt of any payments under such agreements and the future studies, development and commercialization of various products, statements regarding regulatory filings for our development programs, including the planned timing of such regulatory filings, such as IND applications, and potential review by regulatory authorities, our use of capital, expenses and other financial results in the future, availability of funding for existing programs through multiple anticipated Phase 2 study initiations and clinical data readouts, ability to fund operations and capital expenditures into 2029, as well as our expectations of success for our programs, strength of collaboration relationships and the strength of our financial position, among others. By their nature, these statements are subject to numerous risks and uncertainties, including those risks and uncertainties set forth in our most recent Annual Report on Form 10-K for the year ended December 31, 2025, filed with the U.S. Securities and Exchange Commission on March 17, 2026, and any subsequent filings, that could cause actual results, performance or achievement to differ materially and adversely from those anticipated or implied in the statements. You should not rely upon forward-looking statements as predictions of future events. Although our management believes that the expectations reflected in our statements are reasonable, we cannot guarantee that the future results, performance, or events and circumstances described in the forward-looking statements will be achieved or occur. Recipients are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date such statements are made and should not be construed as statements of fact. We undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, any future presentations, or otherwise, except as required by applicable law. Certain information contained in these materials and any statements made orally during any presentation of these materials that relate to the materials or are based on studies, publications, surveys and other data obtained from third-party sources and our own internal estimates and research. While we believe these third-party studies, publications, surveys and other data to be reliable as of the date of these materials, we have not independently verified, and make no representations as to the adequacy, fairness, accuracy or completeness of, any information obtained from third-party sources. In addition, no independent source has evaluated the reasonableness or accuracy of our internal estimates or research and no reliance should be made on any information or statements made in these materials relating to or based on such internal estimates and research. Condensed Consolidated Balance Sheets (in thousands, except share amounts) (unaudited) March 31, December 31, 2026 2025 Assets Current assets: Cash and cash equivalents $ 159,859 $ 129,883 Marketable securities 506,361 247,221 Collaboration receivable ― 7,000 Other receivables 4,609 4,600 Prepaid expenses and other current assets 6,718 4,481 Total current assets 677,547 393,185 Property and equipment, net 28,409 25,986 Operating lease right-of-use assets 24,693 24,386 Restricted cash 4,947 4,954 Other long-term assets 832 148 Total assets $ 736,428 $ 448,659 Liabilities and stockholders’ equity Current liabilities: Accounts payable $ 7,900 $ 3,550 Accrued expenses and other current liabilities 25,594 26,694 Current deferred revenue 33,059 29,571 Current portion of operating lease liability 4,483 4,397 Total current liabilities 71,036 64,212 Deferred revenue, net of current 103,635 111,332 Defined benefit plan liability 5,260 5,265 Operating lease liability 34,581 34,794 Total liabilities 214,512 215,603 Stockholders’ equity Common stock, $0.0001 par value; 500,000,000 shares authorized, 84,321,705 and 65,543,723 shares issued and outstanding as of March 31, 2026 and December 31, 2025, respectively 8 7 Additional paid-in capital 1,048,371 714,090 Accumulated other comprehensive loss (4,746 ) (3,827 ) Accumulated deficit (521,717 ) (477,214 ) Total stockholders’ equity 521,916 233,056 Total liabilities and stockholders’ equity $ 736,428 $ 448,659 Condensed Consolidated Statement of Operations (in thousands) (unaudited) Three months ended March 31, 2026 2025 Collaboration revenue $ 4,210 $ 84,929 Operating expenses: Research and development 44,069 $ 32,190 General and administrative 10,175 8,703 Total operating expenses 54,244 40,893 (Loss) income from operations (50,034 ) 44,036 Other income: Interest income 5,591 3,439 Foreign currency exchange (loss) gain (8 ) 173 Gain on disposal of property and equipment ― 59 Total other income 5,583 3,671 Net (loss) income before income taxes $ (44,451 ) $ 47,707 Income tax provision (52 ) (822 ) Net (loss) income $ (44,503 ) $ 46,885 Investors Andrew Funderburk ir@monterosatx.com Media Cory Tromblee, Scient PR media@monterosatx.com

关注“新药研发质量指南”公众号，查看更多精彩内容！ 正文如下：分子胶成新药研发下一个黄金赛道 今年初，艾伯维宣布以超16亿美元与生物技术公司Neomorph达成合作，就肿瘤和免疫学的多个靶点开发新型分子胶蛋白降解剂。这笔交易，让分子胶再次站在全球的聚光灯下。 此前，分子胶的布局主要集中在IKZF1/3靶点，适应证为多发性骨髓瘤（MM）、非霍奇金淋巴瘤（NHL）等血液肿瘤。目前，全球药企正在不断拓展分子胶的应用范围，新发现GSPT1、RBM3、PRMT5等靶点，覆盖实体瘤、自身免疫性疾病等领域。 凭借独特的作用机制和广阔的应用前景，分子胶逐渐成为跨国药企（MNC）必争的下一个黄金赛道，诺诚健华、恒瑞医药、正大天晴等国内药企也进入了这一赛道。 跨国药企纷纷布局 分子胶降解剂临床应用广泛，且覆盖众多疾病领域，市场潜力巨大。据不完全统计，2023—2024年，分子胶领域已产生13笔交易，总交易金额超过180亿美元，合作方不乏默沙东、罗氏、百时美施贵宝（BMS）、武田等MNC巨头（详见表）。 2019年，BMS豪掷740亿美元收购美国著名生物技术公司新基，囊括了分子胶降解剂来那度胺、泊马度胺等产品管线。根据西南证券研报数据，来那度胺2005—2023年累计销售额高达近900亿美元；泊马度胺的年销售额保持在30亿美元左右。尽管目前已获美国食品药品管理局（FDA）批准上市的分子胶降解剂仅有寥寥几款，但这两款度胺类药物强劲的销售表现，充分验证了分子胶赛道是MNC的必争之地。 从更深层次看，分子胶赛道的爆发离不开其独特的作用机制和治疗优势。与靶向蛋白降解嵌合体（PROTAC）一样，分子胶也属于蛋白降解剂的技术路线之一。尽管两者作用机制类似，但结构和药学性质存在较大差异。 PROTAC在构造上与抗体偶联药物（ADC）有一定的相似之处，也可分为3部分，即POI配体连接靶蛋白，通过linker与E3连接酶配体连接，通过诱导E3连接酶和靶蛋白连接，泛素化靶蛋白进而将靶蛋白降解。不过，由于PROTAC分子量较大，难以实现口服给药，存在较大的技术挑战。而分子胶是通过改变E3连接酶和目标蛋白之间的接触面，从而促进二者结合，进而导致蛋白降解，其具有分子量更小、化学结构更简单、空间干扰少、成药性更好等特点，且能够作用于没有小分子结合口袋的不可成药靶点，比如IKZF1/3。此外，由于分子胶分子量更小，具备更好的生物利用度，相较PROTAC更容易穿透血脑屏障，有利于治疗存在巨大未被满足临床需求的中枢神经系统疾病。 此外，分子胶设计难度大、技术壁垒高，行业正处于潜力无限的蓝海阶段。目前，全球已进入临床试验阶段的分子胶管线较少，布局企业多以MNC与Biotech为主。MNC中布局最早且研发进度最快的是BMS，其次是勃林格殷格翰、安进、卫材、默沙东等。 BMS已有多款分子胶管线处于临床后期阶段。其中，靶向IRAK1/3的Iberdomide、Mezigdomide和Golcadomide处于Ⅲ期临床试验阶段；靶向GSPT1的CC-90009正处于Ⅰ期临床试验阶段。 Iberdomide是新一代口服Cereblon（CRBN）E3连接酶调节剂，在体外可诱导转录因子Aiolos和Ikaros的降解，从而抑制骨髓瘤细胞的生长，目前正在开展对比来那度胺治疗新诊断的MM患者的Ⅲ期临床试验。 Mezigdomide也是新一代口服CRBN E3连接酶调节剂，其特点在于可克服骨髓瘤细胞系中的来那度胺和泊马度胺耐药性，并诱导强效免疫刺激活性。Ⅰ/Ⅱ期临床试验的初步结果显示，Mezigdomide联合硼替佐米和地塞米松治疗既往接受过2~4种治疗（包括来那度胺）的复发或难治性MM患者，具有良好的疗效和安全性。 Golcadomide是一种针对NHL的口服CRBN E3连接酶调节剂，该药针对NHL的疾病特点做了优化，使其主要分布在淋巴器官。目前，Golcadomide正在开展针对多种类型B细胞淋巴瘤的Ⅲ期临床试验，其Ⅰ/Ⅱ期临床试验结果显示，该药针对B细胞淋巴瘤患者的客观缓解率达到91.1%。 卫材研发了全新的芳基磺酰胺分子胶降解剂E7820，正在开展用于治疗复发或难治性急性髓系白血病（AML）、骨髓增生异常综合征（MDS）或单核细胞白血病（CMML）患者的Ⅱ期临床试验。 Monte Rosa Therapeutics的MRT-2359、安进的AMG 193、C4 Therapeutics的Cemsidomide也均进入Ⅰ/Ⅱ期临床试验阶段。其中，MRT-2359是用于治疗实体瘤的类IMiD的GSPT1降解剂，针对适应证包括非小细胞肺癌、小细胞肺癌、任何原发部位的高级别神经内分泌癌等。AMG 193是一款MTA协同的PRMT5抑制剂，正在开展针对MTAP缺失型实体瘤患者的Ⅰ/Ⅱ期临床试验，初步展现出良好的有效性和安全性。Cemsidomide的Ⅰ期临床试验结果显示，联合地塞米松治疗MM、单药治疗NHL，均展现出良好的疗效和安全性。 国内药企加速突围 据不完全统计，我国涉及分子胶领域的企业覆盖上市巨头和新锐Biotech，包括诺诚健华、恒瑞医药、正大天晴、万春医药、标新生物、康朴生物、格博生物、卫本药业、嘉越医药、医诺康、优济普世等。 诺诚健华基于其分子胶平台研发的高效选择性IKZF1/3降解剂ICP-490，正在开展单一疗法或与其他疗法联用治疗MM、NHL等血液肿瘤的临床试验。相比前几代免疫调节剂，ICP-490表现出优异的抗肿瘤活性，可以克服来那度胺的耐药性。此外，该药物通过增强抗体依赖的细胞毒性作用（ADCC）活性，在临床前研究中与CD38单抗Darzalex展现出了协同作用，为临床联合治疗提供了科学依据。 恒瑞医药的HRS-3738（靶点CRBN-E3连接酶）正在开展单药/联合（含地塞米松方案）治疗MM和NHL的Ⅰ期临床试验。公开资料显示，HRS-3738在来那度胺、泊马度胺等类似结构基础上增加了侧链，增大与CRBN的结合表面以增加亲和力，同时较来那度胺、泊马度胺对IKZF1/IKZF3在降解速率和最大降解幅度上也大幅改善，有望克服因CRBN表达下调引起的来那度胺耐药性。 已进入临床试验阶段的国产分子胶管线还包括正大天晴的TQB3820、标新生物的GT929和GT919、康朴生物的KPG-818和KPG-121、格博生物的GLB-001和GLB-002、分迪药业的FD-001、卫本药业的WBC100等。其中，标新生物的GT919和GT929均已获得中美I ND批准，并在复发/难治血液瘤适应证中观察到了疗效。格博生物的GLB-001是CK1α分子胶蛋白降解剂，正在开展治疗髓系恶性肿瘤的Ⅰ期临床试验，GLB-002针对的适应证为复发或难治性NHL。分迪药业的FD-001与BMS的CC-90009等单独降解GSPT1的分子胶不同，除主要降解GSPT1，后续研究证明其也降解I KZF1/3，双靶点的降解结合了对肿瘤细胞的有效杀灭和对免疫系统的调节作用，可产生更好的疗效和更少的潜在毒副作用，该药物拟开发的适应证包括AML、MM和NHL等。 值得一提的是，万春医药子公司Seed Therapeutics在去年8月与卫材达成了一项15亿美元的分子胶降解剂协议，旨在开发针对多种未公开的神经退行性疾病和肿瘤学靶点的新型分子胶降解剂。Seed Therapeutics是一家专注于利用分子胶技术攻克“不可成药”靶点的创新公司，计划在今年推进潜在“Best-in-class”口服RBM39降解剂至Ⅰ期临床试验，用于治疗神经母细胞瘤、肝癌等。 分子胶作为蛋白降解剂领域的一颗新星，其广阔的应用前景和巨大的市场潜力不容小觑。分子胶赛道的兴起，为我国药企提供了与国际巨头同台竞技的舞台。由于分子胶赛道技术壁垒高，竞争相对较小，且正从肿瘤向更广阔的疾病领域渗透，为具备快速跟进能力的国内药企提供了弯道超车的战略窗口。未来，随着临床试验数据不断涌现，一些具有潜力的国产分子胶管线有望脱颖而出，为我国药企在全球市场赢得更多的话语权。 来源/ 中国医药报 文/ 博腾制药股份有限公司 邱浩洋 陈浩 新媒体编辑：李佳欢     统筹策划：何红梅 ------< END >------来源：中国医药报  免责声明：本文仅作信息分享，登载该文章目的为更广泛的传递行业信息，不代表赞同其观点或对其真实性负责。文章版权归原作者及原出处所有，文章内容仅供参考。本网拥有对此声明的最终解释权，若无意侵犯版权，请联系小编（32361149@qq.com）删除。  近期制药课程推荐  地点 点击下方 ↓ 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【QP专题】第二期质量负责人/质量受权人高级研修班 制剂国际化（美国、欧盟）注册申报和GMP迎检准备专题 化药工艺开发CQA及CPP评估实操演练专题 实验动物管理与药物安全性评价策略专题 第三期：新酶设计及酶技术应用专题 【直播】细胞治疗产品生产工艺优化及合规制备与质控要点专题 生物发酵与人体健康高峰论坛暨发酵技术优化与功能发酵制品生产应用研讨会 【实操演练】群体药动学-药效学基础（NONMEM软件）专题  【上海市】第二期医药化工中试放大关键技术专题 +有回放 化药新药药学注册申报要求与核查、检验及临床变更【3天】 ADC药物工艺开发、放大及工艺验证 干细胞及免疫细胞研发要点及注册申报策略 【杭州】药物晶型和制剂研究案例分析专题 ADC药物IND申报要点与非临床设计及研究策略案例分析专题 研发QA技能全方位提升研修班 医学稽查技能及实践专题 药物非临床安全性评价质量保证（QAU）专题 通用能源系统节能技术与管理专题 细胞治疗IIT临床研究政策落地实施及其开展的全流程解析专题 廖明阳 --新药非临床与临床桥接及临床药理专题 重组蛋白生物药发酵工艺专题 如何科学高效建立药物质量控制的分析方法及典型案例 药品申报资料CTD准备全流程及电子递交实操演练 药物非临床安全性评价项目负责人 || SD专题 化学原料药选题立项与合成工艺技术开发 6月：药品研发质量体系专题 2024精细化工高纯化学品分离提纯精制技术应用与装备开发论坛 生物分析非临床药代动力学评价 清洁方法设计开发及验证实施要点与实例分析 稳定细胞株构建和高效上游工艺开发与放大策略 最新分析方法开发与验证及其生命周期管理要点难点解析 小核酸药物分子的设计与开发专题 新药开发中的非临床药理毒理研究策略 临床试验设计与样本量计算关键技术专题 生物制品原液GMP管理和原液工艺验证/持续性工艺验证 原料药药学审评与现场检查中常见问题分析 FDA最新检查趋势、典型缺陷及整改与迎检全流程模拟演练【南京市 】 轻工业核心酶的分子设计、智造与应用技术交流研讨会 化学药品注册报批的药学资料编制（原料药与制剂） 全生命周期的分析方法开发及杂质研究技术要点及案例分析专题-杭州市 基于化学仿制药案例的药物分析人员关键技能提 临床期间生物制品药学变更策略与落地执行 发酵原料药的制备工艺与固定化酶催化工程研究专题 合成生物发酵工程关键工艺技术理论和实践综合提高班【杭州】 2024年药品出海东南亚、TGA及WHO全流程指导专题【回放火爆】 稳定细胞株构建与上游工艺技术及实现案例分析专题 杂质控制策略与杂质分析方法开发专题 药厂工程及设备的项目建设、运行管理及GMP相关系统管理 【第二轮通知】新食品原料合成生物技术创新、申报注册及安全评估专题研讨会 AI颠覆合成化学之路——人工智能在化学合成及生物合成中创新应用技术论坛 新药的供试品管理、生物样本分析、毒代动力学研究专题 发酵工艺设计优化：从小试到中试放大关键技术专题研讨班（杭州） 药品出海东南亚药政法规全梳理及GMP认证准备专题（南京） 实验室到应用场的‘路’与‘桥’--医药化工中试平台建设探索创新及工艺优化专题（杭州） 欧盟、东盟、印度药品注册监管模式暨研发质量体系、技术转移专题（上海） 结晶技术创新与过程强化研讨班（上海） 药品出海新兴市场注册策略全流程指导（巴基斯坦、俄罗斯、哈萨克斯坦及巴西地区）直播有回放 如何构建研发管理体系中的QA（直播有回放） 基于AI的蛋白质结构解析与设计（小鹅通平台） 2025跨年盛会 || 大健康产业技术创新论坛 连续流动化学工艺开发及绿色工艺新技术装备应用论坛--济南 药品出海-港澳及东南亚各国药品注册管理专题（有回放） 2023年课题回放： 1.生物制药下游纯化工艺开发、放大及工艺验证和应用实战经验分享 2.“ICH M10《生物样品分析方法验证》解读与实践高级研修班” 3.mRNA新药研发、生产技术及质控 4.药品冻干工艺技术的确认和验证以及符合性审计技术要求详解 5.GLP实验室质量体系建设专题 6.药品污染控制策略（CCS）建立与执行策略详解 7.最新中美生物制品原液制备GMP管理及FDA的CGMP符合性7356.002标准检查要点 8.临床试验数据管理实操 9.干细胞与免疫细胞治疗产品非临床评价策略和考虑要点专题 10.细胞治疗注册临床与IIT研究全流程案例介绍及临床科研新方向解析 11.新药研发项目经理十项核心技能进阶 12.2023新版GMP指南全方位解析-丁恩峰 13.药品技术转移、工艺验证和清洁验证最新要求与合规操作专题 14.2023第五届化学实验室安全与智能化专题 15.药如何构建研发管理体系中的QA组织 16.重组蛋白生物药发酵工艺：从原核到真核，从小试到中试放大专题 17.2023特医食品生产工艺、原料筛选与现场动态核查要点及特定全营养配方设计指导专场||5月26日青岛 参观考察特医企业 18.第八届精细化工废水、废气、危废处理新技术、新成果、新设备应用发布推广年会暨三废项目对接研讨会 19.第七届药食同源健康产业论坛暨药食同源功效研究与产品开发创新研讨会 20.冻干产品配方研发及工艺流程 23.【精讲】mRNA疫苗开发及IND申请 24.【尚飞】高活性产品共线的风险挑战及关键要点案例分析专题 25..新药研发项目管理全流程实操演练 26.药品质量可控性原理指导下的分析方法验证和质量标准制定策略专题【夏振华】 27.【3天】化药注册法规要求与IND到NDA注册策略及申报资料撰写演练案例分析 28.GLP实验室质量体系建设及认证检查全流程专题 29.原料药工艺研究关键点及研究策略专题 30.【李宏业】新版GMP指南全面解读和重点内容解析专题 31.2023第二届发酵技术高峰论坛暨过程控制、工艺优化及产物分离纯化技术/济南市 32.【相约成都】冻干工艺技术的确认和验证专题 //7.20-22日 33.【细胞治疗产品】从IND到BLA全流程梳理和各申报阶段细节分析 34.【制药企业】计算机化系统生命周期良好管理及实施专题 35.【化药分析】高效的化药分析方法开发全流程实例分析专题 36.【南京市】细胞治疗产品质控体系建立、实施与分析方法开发及验证专题 37.药物非临床安全性评价专题负责人（SD）高级研修班//贺博士主讲 38.【ADC药物】IND申报全流程与案例分析专题 39.【上海市】基因毒性杂质评估策略、分析方法开发及API中美欧注册实操 40.重组蛋白药物下游纯化工艺开发与放大策略及工艺表征研究专题 41.【上海】基于OECD-GLP：质量保证QA专题互动问答一对一指导高级研讨会 42.实验室化学品安全管理事故预防与应急安全处置实操班 43.2023细胞治疗产品注册临床与IIT研究新政策落地介绍及全流程解析专题 44.原料药国际注册DMF、ASMF和CEP文件编写最新要求和案例解析 45.夏振华-药品质量可控性原理指导下的分析方法验证和质量标准制定策略专题 46.【广州】微生物检测技能实操//9.7-9日 47.临床试验数据管理专题分享--王正华主讲 48.CHO细胞培养过程质量调控专题 49.【成都市】新药研发项目经理十项核心技能进阶（4800元/3人参会） 50.【企业观摩】药物晶型研究案例分析专题21日下午到企业参观学习 51.GMP检查法规全梳理、迎检准备 52.贺博士 || 药物非临床安全性评价项目负责人专题 53.9大模块 ||  研发质量体系构建 54.生物冻干制剂开发：处方研究与工艺流程专题 55.医药工厂节能降耗管理体系建立及实际改善方案专题 56.临床试验质量管理体系建设和现场核查专题 57.【线上】研发中各阶段原料药工艺开发案例分析专题 58.非临床药效学评价整体策略//创新药药效的试验设计及动物模型专题 59.【北京市】新药研发项目经理十项核心技能进阶（线下无回放） 60.【推荐】新药临床前研究的开展及质量把控专题（延期的） 61.【直播】MAH质量体系合规性重点详解 62.2023高级酶工程与酶技术应用大会 63.【医学监察】的高效管理与实施要点案例分析及实操 64.【直播】注册经理能力全面提升实战 65.【上海】临床前药代动力学研究策略专题 66.细胞治疗产品技术转移、工艺变更和工艺验证专题 67质量负责人/质量受权人高级研修班 68.【精讲】研发质量管理体系//高老师，刘老师 69.蛋白纯化工艺开发及放大要点与工艺验证和保存稳定性策略与应用高级研修班 70.2023高活性产品共线的风险挑战及关键要点案例分析专题培训 71.2023加强MAH生产监管及现场检查应对策略专题/南京 72.【精讲】抗体类药物制剂开发全流程 73.B证许可持有人 MAH 重点实操流程及210项省局现场检查缺陷项解读 74.2023包材供应商质量管控及关联审评策略专题 75.定量药理模型构建与质控及在新药研发中应用案例分析 76.最新生物制品原液制备GMP管理及现场检查要点解析和原液工艺验证专题 77.【上海】小核酸药物研发要点及注册申报策略专题 78.重组蛋白生物药发酵工艺开发设计、放大策略专题 79.【线上】欧盟原料药注册法规和GMP检查细节专题培训 80.如何做好新药研发项目经理？？ 主办方联系人：左老师  15652390923 微信同号 组团报名可优惠，各年度回放视频可询价购买~~  ~~ 回放都可点开查看内容，课程精选 ~~  点击“阅读原文”填写参会信息