NX-5948

Fast track designation follows positive Phase 1 data presented at the 12th International Workshop on Waldenstrom’s Macroglobulinemia SAN FRANCISCO, Dec. 19, 2024 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with cancer and inflammatory diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for NX-5948, a highly selective degrader of Bruton’s tyrosine kinase (BTK), for the treatment of adult patients with relapsed or refractory Waldenstrom’s macroglobulinemia (WM) after at least two lines of therapy, including a BTK inhibitor. “Fast Track designation for NX-5948 is an important recognition of the unmet patient need in Waldenstrom’s macroglobulinemia, particularly in the growing number of patients whose cancer has progressed following BTK inhibitor therapy,” said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. “This designation follows encouraging safety and efficacy data from our ongoing Phase 1 clinical trial, demonstrating early promise of clinical benefit with potential for durable outcomes. We continue to enroll Waldenstrom’s macroglobulinemia patients in the ongoing Phase 1b expansion cohort and anticipate sharing additional clinical data in 2025.” In addition to the Fast Track designation announced today for Waldenstrom’s macroglobulinemia, NX-5948 previously received Fast Track designation in January 2024 for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) after at least two lines of therapy, including a BTK inhibitor and a B-cell lymphoma 2 (BCL2) inhibitor. In November 2024, the European Medicines Agency (EMA) granted NX-5948 PRIME designation for the treatment of adult patients with relapsed or refractory CLL/SLL after at least a BTK inhibitor and a BCL2 inhibitor. About Waldenstrom’s Macroglobulinemia WM is a rare, slow growing type of non-Hodgkin’s lymphoma that is characterized by the replacement of normal bone marrow cells by malignant lymphocytic cells that produce monoclonal IgM. This replacement leads to anemia, bleeding, and impaired immune function, while the elevated IgM levels may cause neurologic symptoms. The incidence of Waldenstrom’s macroglobulinemia ranges from 0.361,2 to 0.573 per 100,000 people in the United States or approximately 1,200 to 1,900 annually. With a median disease duration approaching 10 years,4 approximately 12,000 to 19,000 patients are living with Waldenstrom’s macroglobulinemia in the United States. Recommended first-line treatments including chemoimmunotherapy and BTK inhibitor therapy. There are no therapies approved to treat WM patients after BTKi. About Fast Track Designation The FDA’s Fast Track designation is intended to facilitate and expedite the development and review of drug candidates to treat serious conditions and fulfill an unmet medical need. To qualify, available clinical and non-clinical data need to demonstrate a therapeutic candidate’s potential to address this unmet medical need. A therapeutic candidate that receives Fast Track designation may be eligible for more frequent interactions with the FDA to discuss the candidate’s development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review. About PRIME Designation The PRIME initiative, launched by the EMA in 2016, offers early, proactive and enhanced support to developers of promising medicines to optimize development plans and accelerate evaluation so these medicines can reach patients faster. To be eligible for PRIME, medicines must target an unmet medical need and show potential benefit for patients based on early clinical data. About NX-5948 NX-5948 is an investigational, orally bioavailable, brain penetrant, small molecule degrader of BTK. NX-5948 is currently being evaluated in a Phase 1 clinical trial in patients with relapsed or refractory B cell malignancies. Nurix has previously reported that NX-5948 is highly potent against a range of tumor cell lines that are resistant to current BTK inhibitor therapies, an important consideration in heavily pretreated CLL/SLL patient populations. Additional information on the ongoing clinical trial can be accessed at clinicaltrials.gov (NCT05131022). About Nurix Nurix Therapeutics is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative small molecules and antibody therapies based on the modulation of cellular protein levels as a novel treatment approach for cancer, inflammatory conditions, and other challenging diseases. Leveraging extensive expertise in E3 ligases together with proprietary DNA-encoded libraries, Nurix has built DELigase, an integrated discovery platform, to identify and advance novel drug candidates targeting E3 ligases, a broad class of enzymes that can modulate proteins within the cell. Nurix’s drug discovery approach is to either harness or inhibit the natural function of E3 ligases within the ubiquitin-proteasome system to selectively decrease or increase cellular protein levels. Nurix’s wholly owned, clinical stage pipeline includes targeted protein degraders of Bruton’s tyrosine kinase, a B-cell signaling protein, and inhibitors of Casitas B-lineage lymphoma proto-oncogene B, an E3 ligase that regulates activation of multiple immune cell types including T cells and NK cells. Nurix is headquartered in San Francisco, California. For additional information visit http://www.nurixtx.com. Forward-Looking Statements This press release contains statements that relate to future events and expectations and as such constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. When or if used in this press release, the words “anticipate,” “believe,” “could,” “estimate,” “expect,” “intend,” “may,” “outlook,” “plan,” “predict,” “should,” “will,” and similar expressions and their variants, as they relate to Nurix, may identify forward-looking statements. All statements that reflect Nurix’s expectations, assumptions or projections about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements regarding Nurix’s plans and strategies with respect to NX-5948, the potential advantages and therapeutic benefits of NX-5948, including its potential role in the treatment of B-cell malignancies, including Waldenstrom’s macroglobulinemia, the planned timing for the provision of updates from the NX-5948 clinical trial, and the potential benefits of Fast Track designation. Forward-looking statements reflect Nurix’s current beliefs, expectations, and assumptions. Although Nurix believes the expectations and assumptions reflected in such forward-looking statements are reasonable, Nurix can give no assurance that they will prove to be correct. Forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and changes in circumstances that are difficult to predict, which could cause Nurix’s actual activities and results to differ materially from those expressed in any forward-looking statement. Such risks and uncertainties include, but are not limited to: (i) the risks inherent in the drug development process, including the unexpected emergence of adverse events or other undesirable side effects during clinical development; (ii) uncertainties related to the timing and results of clinical trials; (iii) whether Nurix will be able to fund its research and development activities and achieve its research and development goals; (iv) the impact of economic and market conditions and global and regional events on Nurix’s business, clinical trials, financial condition, liquidity and results of operations; (v) whether Nurix will be able to protect intellectual property and (vi) other risks and uncertainties described under the heading “Risk Factors” in Nurix’s Quarterly Report on Form 10-Q for the fiscal period ended August 31, 2024, and other SEC filings. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. The statements in this press release speak only as of the date of this press release, even if subsequently made available by Nurix on its website or otherwise. Nurix disclaims any intention or obligation to update publicly any forward-looking statements, whether in response to new information, future events, or otherwise, except as required by applicable law. Contacts: Investors Jason Kantor, Ph.D. Nurix Therapeutics ir@nurixtx.com Elizabeth Wolffe, Ph.D. Wheelhouse Life Science Advisors lwolffe@wheelhouselsa.com Media Aljanae Reynolds Wheelhouse Life Science Advisors areynolds@wheelhouselsa.com ________________________________ ¹ Bibas M., Sarosiek S., Castillo J.J. Waldenström Macroglobulinemia - A State-of-the-Art Review: Part 1: Epidemiology, pathogenesis, clinicopathologic characteristics, differential diagnosis, risk stratification, and clinical problems. Mediterr J Hematol Infect Dis 2024, 16(1): e2024061. ² McMaster ML. The Epidemiology of Waldenström Macroglobulinemia. Semin Hematol. 2023 March; 60(2): 65–72. ³ Kyle Robert A, et al, 50 Year Incidence of Waldenström Macroglobulinemia in Olmsted County, Minnesota From 1961–2010: A Population-Based Study With Complete Case Capture and Hematopathologic Review. Mayo Clin Proc. 2018; 93(6): 739–746. ⁴ Gertz M.A., et.al., Waldenstrom Macroglobulinemia: 2023 update on diagnosis, risk stratification, and management. Am J Hematol. 2023;98(1):348-358.

▎药明康德内容团队编辑 本期看点 1. 用于治疗继发性噬血细胞性淋巴组织细胞增生症（sHLH）的单克隆抗体疗法ELA026在1b期研究中的表现亮眼，用药后第4周时的总缓解率（ORR）为100%（12/12）。 2. RNA干扰（RNAi）药物ARO-CFB用于治疗补体介导疾病，在一项1/2a期临床研究中使正常健康受试者中目标蛋白的水平平均降低90%，且持续时间超过3个月。 3. 6个月内使用1-2次血浆激肽释放酶单克隆抗体抑制剂navenibart在早期临床试验中使遗传性血管性水肿（HAE）患者的每月发作率降低了90%-95%。 药明康德内容团队整理 ELA026：公布1b期临床试验的新数据 Electra Therapeutics公司公布了其单克隆抗体疗法ELA026用于治疗继发性噬血细胞性淋巴组织细胞增生症的1b期研究结果。sHLH是一种罕见的、危及生命的高炎症性疾病，目前尚无批准的治疗方法。它可以由癌症、感染、自身免疫性疾病或免疫疗法引发。sHLH与严重的炎症反应有关，患者需要立即干预。如果不进行治疗，患者可能会出现多器官衰竭和死亡。sHLH与病程早期的高死亡率相关，使用可用疗法的恶性肿瘤相关HLH（mHLH）患者在两个月时的死亡率约为50%。ELA026靶向髓系细胞和T淋巴细胞表面的SIRP-α/β1/γ，髓系细胞和T淋巴细胞是诱发sHLH细胞因子风暴和高炎症反应的主要病理免疫细胞。以SIRP为靶点选择性地清除致病性免疫细胞具有在免疫学、炎症和癌症领域广泛应用的潜力。 此次公布的研究结果表明，对于mHLH患者，在接受ELA026作为一线治疗的患者中，第4周时的ORR为100%（12/12），出院率为100%(11/11)，2个月生存率为92%（11/12）。安全性方面，ELA026的耐受性良好，不良事件可控。 ARO-CFB：公布1/2a期临床试验的中期数据 Arrowhead Pharmaceuticals公司公布了其靶向补体因子B（CFB）的在研RNAi药物ARO-CFB用于治疗补体介导疾病的1/2a期临床研究的中期结果。ARO-CFB旨在减少肝脏中CFB的表达，CFB在放大补体替代通路活化过程中起着重要的调节作用。ARO-CFB正被开发为补体介导的肾脏疾病的潜在治疗药物，如免疫球蛋白A肾病（IgAN）。此外，ARO-CFB还有望用于涉及补体激活的非肾脏疾病的临床治疗。 截至2024年11月15日的数据，ARO-CFB在正常健康受试者中实现了剂量依赖性的循环CFB蛋白水平降低，平均达90%，且持续时间超过3个月。此外，无论是单次还是多次给药，ARO-CFB几乎完全抑制了替代通路的活性。截至目前，ARO-CFB总体耐受性良好，安全性数据支持进一步的临床开发。 Navenibart（STAR-0215）：公布1b/2期临床试验数据 Astria Therapeutics公司公布了其用于治疗遗传性血管性水肿的血浆激肽释放酶单抗抑制剂navenibart的1b/2期临床试验数据。在该研究中，16名患者被分入三个队列接受STAR-0215皮下给药：队列1的4名患者接受了1剂450 mg剂量；队列2的6名患者先接受了1剂600 mg剂量，并在3个月后又接受了1剂300 mg剂量；队列3的6名患者先接受了1剂600 mg剂量，并在1个月后又接受了1剂600 mg剂量。 此次公布的结果显示，在6个月内给药1次或2次navenibart，3个队列患者的平均每月发作率降低了90%-95%，该结果支持每年仅给药2次或4次。此外，使用navenibart后，中度或严重HAE发作减少了约95%-96%，需要使用急救药物的发作减少了91%-94%。队列2和队列3患者6个月内的无发作率为67%。安全性方面，navenibart总体耐受性良好，无严重不良事件，无停药现象。 IO-202：公布1b期临床试验扩展队列的新数据 Immune-Onc Therapeutics公司公布了其1b期扩展队列的最新数据，该队列评估了其抗LILRB4抗体IO-202联用阿扎胞苷（AZA）治疗慢性粒单核细胞白血病（CMML）患者的疗效和安全性。IO-202是一款潜在"first-in-class"的IgG1抗体，可特异性、高亲和力地与LILRB4结合，并通过抗体依赖性细胞毒性和抗体依赖性细胞吞噬作用消除LILRB4阳性细胞。因此，IO-202是一种在血癌、自身免疫性和炎症性疾病方面具有广阔潜力的靶向疗法。 此次公布的结果显示，根据国际工作组2023缓解标准，在接受IO-202与AZA联合治疗的18名疗效可评估的CMML患者中，ORR为66.7%，完全缓解（CR）率为50%。在LILRB4高表达的CMML患者中，ORR为100%，CR率为83.3%。值得注意的是，过往获批疗法的CR率仅为7-17%。 ISB 2001：公布1期临床试验的新数据 Ichnos Glenmark Innovation公司公布了其三特异性抗体ISB 2001用于治疗复发/难治性多发性骨髓瘤（RRMM）的1期研究的新数据。ISB 2001同时靶向MM细胞上的BCMA和CD38，以及T细胞上的CD3，旨在增加对MM细胞的特异性结合，同时最大限度地减少脱靶效应。 截至2024年10月1日的数据，20名可评估疗效患者的ORR为75%（5/20），CR/严格的CR（sCR）率为20%。在接受活性剂量（0.05 mg/kg和更高剂量）治疗的18例患者中，ORR为83%，CR/sCR率为22%。安全性方面，在每周皮下注射剂量为1.2 mg/kg时未观察到剂量限制性毒性（DLT），大多数是轻度的细胞因子释放综合征（CRS），未报告神经毒性，也没有患者因不良事件而停药。 NX-5948：公布1a/1b期临床试验的新数据 Nurix Therapeutics公司公布了其布鲁顿酪氨酸激酶（BTK）降解剂NX-5948用于治疗复发或难治性慢性淋巴细胞白血病或小淋巴细胞淋巴瘤（CLL/SLL）的1a/1b期临床试验的新数据。NX-5948是一种口服、可穿越血脑屏障的小分子BTK蛋白降解剂。 截至2024年10月10日的数据，首次疾病评估时（治疗后第8周）49名疗效可评估的CLL/SLL患者的客观缓解率为75.5%，该数据在至少进行两次疾病评估（治疗后第16周时）的患者中增加至84.2%。无论患者既往治疗、基线突变、高危分子特征或中枢神经系统（CNS）受累如何，均观察到缓解。13名患者的缓解持续时间超过6个月，5名患者在治疗一年后仍在接受治疗并保持缓解。此外，NX-5948在所有评估的剂量中均具有良好的耐受性。 Enzomenib（DSP-5336）：公布1/2期临床试验的新数据 Sumitomo Pharma America公司公布了其在研口服小分子enzomenib用于治疗复发/难治性急性白血病的1/2期临床试验的新数据。Enzomenib可抑制menin与MLL蛋白的相互作用，并能降低白血病相关基因HOXA9和MEIS1的表达，增加带有MLL重排和NPM1突变的人类急性白血病细胞系中分化基因CD11b的表达。 此次公布的结果显示，在23例KMT2A重排患者中，客观缓解率为65.2%，达到CR加部分血液学恢复的完全缓解（CRh）的患者比例为30.4%。在接受300 mg每日两次（BID）治疗的15名KMT2A重排患者中，客观缓解率为73.3%，达到CR加CRh的比例为40%。在接受200 mg BID或300 mg BID治疗的17名NPM1突变患者中，客观缓解率为58.8%，达到CR加CRh的比例为47.1%。Enzomenib的耐受性良好，药物相关不良事件的总体发生率较低，没有报告DLT。10.7%的患者患上了分化综合征，但未导致患者死亡，也不需要永久停用enzomenib。 Evorpacept：公布1b/2期联合治疗试验数据 ALX Oncology公司公布了其CD47抑制剂evorpacept联用HER2靶向双特异性抗体zanidatamab治疗此前接受过大量治疗的HER2阳性和HER2低表达转移性乳腺癌患者的1b/2期临床试验数据。Evorpacept是一种下一代CD47阻断疗法，它将高亲和性CD47结合结构域与经特殊处理的Fc结构域相结合，因此无法激活Fc受体，有望降低CD47靶向抗体因激活Fc受体可能产生的毒副作用。此外，与其它抗CD47抗体相比，evorpacept的分子量更小，更容易渗透到实体瘤中，从而提高疗效。 此次公布的结果显示，evorpacept联用zanidatamab对经中央实验室评估确认为HER2阳性的患者（n=9）显示出最强的抗肿瘤活性。这些患者经确认的客观缓解率为55.6%，中位无进展生存期（PFS）为7.4个月。总体而言，队列1（HER2阳性的转移性乳腺癌患者）患者（n=21）的经确认的客观缓解率为33.3%，中位PFS为3.6个月，中位缓解持续时间（DOR）尚未达到。在队列2（HER2低表达的转移性乳腺癌患者）的患者（n=15）中也观察到了一定的缓解，经确认的客观缓解率为20.0%，中位PFS为1.9个月，中位DOR为5.5个月。 MDNA11：公布1/2期临床试验的新数据 Medicenna Therapeutics公司公布了正在进行的1/2期ABILITY-1研究的最新临床数据，该研究旨在评估其下一代长效IL-2超级激动剂MDNA11单药或联用PD-1抑制剂帕博利珠单抗治疗晚期实体瘤的疗效和安全性。MDNA11是一种通过静脉注射给药的长效“增强β而非α”的IL-2超级细胞因子（IL-2 Superkine），它能够优先激活负责杀伤癌细胞的免疫效应细胞（CD8阳性T细胞和自然杀伤细胞，即NK细胞），同时最小化或不刺激具有免疫抑制作用的调节性T细胞（Tregs）。 此次公布的结果显示，MDNA11单药治疗和联用帕博利珠单抗的疾病控制率（DCR）分别为55%（1例CR、4例部分缓解和6例疾病稳定）和78%（1例CR、1例部分缓解和5例疾病稳定）。1名70岁的晚期化疗难治性肛门癌患者在接受MDNA11联用帕博利珠单抗治疗时，在8周内达到了CR。单药治疗组中两名免疫检查点抑制剂耐药患者的所有肿瘤完全消失，1名获得CR的黑色素瘤患者在第63周时依然没有复发，1名微卫星不稳定性高（MSI-H）的胰腺癌患者在肝脏中实现了靶病灶和非靶转移灶的完全消除，在研究期间实现了20个月的持久缓解，并已持续停止抗癌治疗达11个月。 VGA039：公布1期临床试验的中期数据 Star Therapeutics公司公布了其在研单克隆抗体VGA039用于治疗血管性血友病（Von Willebrand Disease，VWD）的1期单剂量递增（SAD）研究的中期数据。VGA039靶向蛋白质S，旨在恢复血液凝固的平衡，有望作为一种适用于多种出血性疾病的通用止血疗法。 此次公布的数据显示，在3名高年出血率（ABR）VWD患者中，皮下注射VGA039与ABR的显著降低相关，单次注射使患者的ABR降低了75%-88%，且治疗浓度保持了约4周。该降幅与已获批准的VWD预防疗法在临床试验中观察到的降幅一致，但这些疗法需要每周进行多次静脉注射。在安全性可评估的7名患者中，VGA039安全且耐受性良好，未观察到注射部位反应。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料（可上下滑动查看） [1] Nurix Therapeutics Presents New Positive Data from Phase 1a/1b Clinical Trial of NX-5948 in Chronic Lymphocytic Leukemia at the 66th American Society of Hematology Annual Meeting. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/09/2994129/0/en/Nurix-Therapeutics-Presents-New-Positive-Data-from-Phase-1a-1b-Clinical-Trial-of-NX-5948-in-Chronic-Lymphocytic-Leukemia-at-the-66th-American-Society-of-Hematology-Annual-Meeting.html [2] Ichnos Glenmark Innovation (IGI) Presents First Clinical Data from Phase 1 Study of Trispecific TREAT™ Antibody, ISB 2001, Showing High Overall Response Rate (ORR) with Durable Responses and Favorable Safety Profile in Patients with Heavily Pretreated Multiple Myeloma. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/10/2994146/0/en/Ichnos-Glenmark-Innovation-IGI-Presents-First-Clinical-Data-from-Phase-1-Study-of-Trispecific-TREAT-Antibody-ISB-2001-Showing-High-Overall-Response-Rate-ORR-with-Durable-Responses-.html [3] Immune-Onc Therapeutics Presents Updated Data from Phase 1b Study of IO-202 Highlighting Promising Efficacy and Safety Data in CMML Patients at 2024 American Society of Hematology (ASH) Annual Meeting. Retrieved December 12, 2024, from https://www.immune-onc.com/pr-posts/immune-onc-therapeutics-presents-updated-data-from-phase-1b-study-of-io-202-highlighting-promising-efficacy-and-safety-data-in-cmml-patients-at-2024-american-society-of-hematology-ash-annual-meeting [4] Electra Therapeutics Presents Positive Clinical Results from Completed Phase 1b Study of ELA026 in Secondary Hemophagocytic Lymphohistiocytosis (sHLH) in an Oral Session at ASH 2024. Retrieved December 12, 2024, from https://electra-therapeutics.com/electra-therapeutics-presents-positive-clinical-results-from-completed-phase-1b-study-of-ela026-in-secondary-hemophagocytic-lymphohistiocytosis-shlh-in-an-oral-session-at-ash-2024/ [5] Star Therapeutics Presents Interim Clinical Data for VGA039 in Patients with Von Willebrand Disease (VWD) at ASH 2024. Retrieved December 12, 2024, from https://star-therapeutics.com/star-therapeutics-presents-interim-clinical-data-for-vga039-in-patients-with-von-willebrand-disease-vwd-at-ash-2024/ [6] Sumitomo Pharma America Presents New Data on Nuvisertib and Enzomenib at the 2024 American Society of Hematology Annual Meeting. Retrieved December 12, 2024, from https://www.prnewswire.com/news-releases/sumitomo-pharma-america-presents-new-data-on-nuvisertib-and-enzomenib-at-the-2024-american-society-of-hematology-annual-meeting-302326146.html [7] Arvinas and Pfizer Announce Initial Phase 1b Data from the TACTIVE-U Sub-Study of Vepdegestrant in Combination with Abemaciclib at 2024 San Antonio Breast Cancer Symposium. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/10/2994631/0/en/index.html [8] ALX Oncology Announces New Data Demonstrating Evorpacept in Combination with Zanidatamab Generates Promising Antitumor Activity in Advanced Breast Cancer. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/10/2994624/0/en/index.html [9] Arrowhead Pharmaceuticals Presents Interim Clinical Data on ARO-CFB for the Treatment of Complement Mediated Diseases. Retrieved December 12, 2024, from https://arrowheadpharma.com/news-press/arrowhead-pharmaceuticals-presents-interim-clinical-data-on-aro-cfb-for-the-treatment-of-complement-mediated-diseases/ [10] Astria Therapeutics Announces Positive Final Results from Target Enrollment in the ALPHA-STAR Phase 1b/2 Trial of Navenibart for HAE. Retrieved December 12, 2024, from https://ir.astriatx.com/news-releases/news-release-details/astria-therapeutics-announces-positive-final-results-target [11] C4 Therapeutics Presents Cemsidomide Phase 1 Data at the American Society for Hematology (ASH) Annual Meeting that Demonstrated Potential to Become Best-in-Class IKZF1/3 Degrader. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/08/2993401/0/en/C4-Therapeutics-Presents-Cemsidomide-Phase-1-Data-at-the-American-Society-for-Hematology-ASH-Annual-Meeting-that-Demonstrated-Potential-to-Become-Best-in-Class-IKZF1-3-Degrader.html [12] Medicenna Provides Clinical Update and Announces First Complete Responder in MDNA11 in Combination with KEYTRUDA® (pembrolizumab) Combination Dose Escalation Arm of the ABILITY-1 Study. Retrieved December 12, 2024, from https://ir.medicenna.com/news-releases/news-release-details/medicenna-provides-clinical-update-and-announces-first-complete [13] Disc Medicine Presents Positive Updated Results from Phase 1b Trial in Patients with Myelofibrosis (MF) and Anemia in an Oral Presentation at the 66th American Society of Hematology (ASH) Annual Meeting. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/09/2993429/0/en/Disc-Medicine-Presents-Positive-Updated-Results-from-Phase-1b-Trial-in-Patients-with-Myelofibrosis-MF-and-Anemia-in-an-Oral-Presentation-at-the-66th-American-Society-of-Hematology-.html [14] Rigel Highlights Initial Data from Ongoing Phase 1b Study Evaluating R289 in LR-MDS at the 66th ASH Annual Meeting and Exposition. Retrieved December 12, 2024, from https://www.prnewswire.com/news-releases/rigel-highlights-initial-data-from-ongoing-phase-1b-study-evaluating-r289-in-lr-mds-at-the-66th-ash-annual-meeting-and-exposition-302325895.html [15] OnKure Announces Encouraging Preliminary Safety, Tolerability, and Pharmacokinetic (PK) Data from its First-In-Human PIKture-01 Trial of OKI-219. Retrieved December 12, 2024, from https://investors.onkuretherapeutics.com/news-releases/news-release-details/onkure-announces-encouraging-preliminary-safety-tolerability-and [16] Prelude Therapeutics Presents Preliminary Results of Phase 1 Dose-escalation Study of PRT2527 as Monotherapy and in Combination with Zanubrutinib in Patients with Relapsed/Refractory Lymphoid Malignancies. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/11/2995306/0/en/Prelude-Therapeutics-Presents-Preliminary-Results-of-Phase-1-Dose-escalation-Study-of-PRT2527-as-Monotherapy-and-in-Combination-with-Zanubrutinib-in-Patients-with-Relapsed-Refracto.html [17] Enterome’s Immunotherapy EO2463 Shows Early Clinical Response in Newly Diagnosed Follicular Lymphoma Suggesting a Potential Alternative to ‘Watchful Waiting’. Retrieved December 12, 2024, from https://www.enterome.com/news-events/enteromes-immunotherapy-eo2463-shows-early-clinical-response-in-newly-diagnosed-follicular-lymphoma-suggesting-a-potential-alternative-to-watchful-waiting/ [18] CatalYm’s Visugromab Phase 1/2a Data Demonstrating Clinical Proof-of-Concept for Reversing Resistance to Checkpoint Inhibitors through GDF-15 Neutralization Published in Nature. Retrieved December 12, 2024, from https://www.catalym.com/catalyms-visugromab-phase-1-2a-data-demonstrating-clinical-proof-of-concept-for-reversing-resistance-to-checkpoint-inhibitors-through-gdf-15-neutralization-published-in-nature/ [19] Opna Bio Announces Interim Data from Phase 1 Combination Study of OPN-2853 with Ruxolitinib in Patients with Advanced Myelofibrosis. Retrieved December 12, 2024, from https://www.opnabio.com/opna-bio-announces-interim-data-from-phase-1-combination-study-of-opn-2853-with-ruxolitinib-in-patients-with-advanced-myelofibrosis/ [20] Recursion Reports Interim Phase 1 Clinical Data for REC-617 Monotherapy, a Potential Best-in-Class CDK7 Inhibitor, With Encouraging Patient Response and Favorable Tolerability. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/09/2994128/0/en/Recursion-Reports-Interim-Phase-1-Clinical-Data-for-REC-617-Monotherapy-a-Potential-Best-in-Class-CDK7-Inhibitor-With-Encouraging-Patient-Response-and-Favorable-Tolerability.html [21] Galapagos Announces Encouraging New Results from Ongoing Phase 1/2 Study of CD19 CAR T-Cell Therapy, GLPG5101, in Patients with Relapsed/Refractory Non-Hodgkin Lymphoma. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/07/2993348/0/en/Galapagos-Announces-Encouraging-New-Results-from-Ongoing-Phase-1-2-Study-of-CD19-CAR-T-Cell-Therapy-GLPG5101-in-Patients-with-Relapsed-Refractory-Non-Hodgkin-Lymphoma.html [22] Poseida Therapeutics Highlights Positive Interim Phase 1 Results for P-BCMA-ALLO1 and Preclinical Data for Dual CAR-T P-CD19CD20-ALLO1 at the 66th American Society of Hematology (ASH) Annual Meeting. Retrieved December 12, 2024, from https://www.prnewswire.com/news-releases/poseida-therapeutics-highlights-positive-interim-phase-1-results-for-p-bcma-allo1-and-preclinical-data-for-dual-car-t-p-cd19cd20-allo1-at-the-66th-american-society-of-hematology-ash-annual-meeting-302325606.html [23] Lyell Presents Positive Initial Clinical Data from the Phase 1-2 Clinical Trial of IMPT-314 for the Treatment of B-cell Lymphoma at the 2024 ASH Annual Meeting. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/10/2994158/0/en/Lyell-Presents-Positive-Initial-Clinical-Data-from-the-Phase-1-2-Clinical-Trial-of-IMPT-314-for-the-Treatment-of-B-cell-Lymphoma-at-the-2024-ASH-Annual-Meeting.html [24] Synthekine Presents Positive Initial Results from Phase 1 Clinical Trial of CD19 CAR-T (SYNCAR-001) and Orthogonal IL-2 (STK-009) Combination Therapy for Treatment of Hematologic Malignancies at ASH 2024 Annual Meeting. Retrieved December 12, 2024, from https://www.synthekine.com/news/synthekine-presents-positive-initial-results-from-phase-1-clinical-trial-of-cd19-car-t-syncar-001-and-orthogonal-il-2-stk-009-combination-therapy-for-treatment-of-hematologic-malignancies-at-ash-2/ [25] AAVantgarde presents safety data of the LUCE-1 study of AAVB-081 for RP related to Usher 1B. Retrieved December 12, 2024, from https://www.aavantgarde.com/en/news/aavantgarde-presents-safety-data-of-the-luce-1-study-of-aavb-081-for-rp-related-to-usher-1b/ [26] Neurona Therapeutics Presents Positive Clinical Update: New Data from NRTX-1001 Cell Therapy Trial in Drug-resistant Epilepsy Announced at 2024 Annual Meeting of the American Epilepsy Society. Retrieved December 12, 2024, from https://www.neuronatherapeutics.com/news/press-releases/120624/ [27] IN8bio Reports Continued Progression-Free Survival in Phase 1 Investigator-Sponsored Trial of INB-100 Allogeneic Gamma-Delta T Cells for Leukemias at the 2024 American Society of Hematology Annual Meeting. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/10/2994365/0/en/IN8bio-Reports-Continued-Progression-Free-Survival-in-Phase-1-Investigator-Sponsored-Trial-of-INB-100-Allogeneic-Gamma-Delta-T-Cells-for-Leukemias-at-the-2024-American-Society-of-H.html [28] Sonnet BioTherapeutics Announces Topline Safety Data Following Successful Completion of SON-1010 Monotherapy Dose Escalation in Phase 1 SB101 Trial. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/09/2993772/0/en/Sonnet-BioTherapeutics-Announces-Topline-Safety-Data-Following-Successful-Completion-of-SON-1010-Monotherapy-Dose-Escalation-in-Phase-1-SB101-Trial.html [29] CRISPR Therapeutics Presents Data at the 2024 American Society of Hematology (ASH) Annual Meeting. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/09/2993974/0/en/CRISPR-Therapeutics-Presents-Data-at-the-2024-American-Society-of-Hematology-ASH-Annual-Meeting.html [30] Monte Rosa Therapeutics Provides Development Progress Update for Ongoing MRT-2359 Phase 1/2 Study in Patients with MYC-driven Solid Tumors. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/05/2992220/0/en/Monte-Rosa-Therapeutics-Provides-Development-Progress-Update-for-Ongoing-MRT-2359-Phase-1-2-Study-in-Patients-with-MYC-driven-Solid-Tumors.html [31] Olema Oncology Announces FDA Clearance of Investigational New Drug Application for OP-3136, a Potent KAT6 Inhibitor. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/09/2993612/0/en/Olema-Oncology-Announces-FDA-Clearance-of-Investigational-New-Drug-Application-for-OP-3136-a-Potent-KAT6-Inhibitor.html [32] Theratechnologies Announces Preliminary Tolerability and Efficacy Data from Phase 1b, Dose-Ranging Trial of Sudocetaxel Zendusortide in Patients with Advanced Ovarian Cancer. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/09/2993661/0/en/Theratechnologies-Announces-Preliminary-Tolerability-and-Efficacy-Data-from-Phase-1b-Dose-Ranging-Trial-of-Sudocetaxel-Zendusortide-in-Patients-with-Advanced-Ovarian-Cancer.html [33] Artelo Biosciences Announces Presentation of Phase 1 Data with ART27.13 in Cancer-Related Anorexia. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/09/2993818/0/en/Artelo-Biosciences-Announces-Presentation-of-Phase-1-Data-with-ART27-13-in-Cancer-Related-Anorexia.html [34] Silence Therapeutics Presents Promising Phase 1 Data in Polycythemia Vera Patients at the American Society of Hematology (ASH) Annual Meeting. Retrieved December 12, 2024, from https://silence-therapeutics.com/investors/press-releases/press-releases-details/2024/Silence-Therapeutics-Presents-Promising-Phase-1-Data-in-Polycythemia-Vera-Patients-at-the-American-Society-of-Hematology-ASH-Annual-Meeting/default.aspx [35] AC Immune Reports Interim Safety Data from Phase 1b/2 ABATE Trial of ACI-24.060 in Down syndrome. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/10/2994352/0/en/AC-Immune-Reports-Interim-Safety-Data-from-Phase-1b-2-ABATE-Trial-of-ACI-24-060-in-Down-syndrome.html [36] Affimed Reports Promising Phase 1 Efficacy and Safety Data for AFM28 in Relapsed/Refractory Acute Myeloid Leukemia (R/R AML). Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/09/2994033/0/en/Affimed-Reports-Promising-Phase-1-Efficacy-and-Safety-Data-for-AFM28-in-Relapsed-Refractory-Acute-Myeloid-Leukemia-R-R-AML.html [37] PS3-08: Interim Overall Survival of Patients with Locally Advanced or Metastatic Triple-Negative Breast Cancer treated with First Line PM8002/BNT327 in Combination with Nab-Paclitaxel in Phase Ib/II Study. Retrieved December 10, 2024, from https://sabcs.org/Portals/0/Documents/Formatted_Abstracts%2011-27%20Without%20Embargoed.pdf?ver=qjVzk5LPNjOteEIvNEYJFw%3d%3d [38] Olema Oncology Presents Updated Clinical Results for Palazestrant in Combination with Ribociclib at the San Antonio Breast Cancer Symposium. Retrieved December 12, 2024, from https://ir.olema.com/news-releases/news-release-details/olema-oncology-presents-updated-clinical-results-palazestrant [39] Astria Therapeutics Announces FDA Clearance of IND Application for STAR-0310, a Monoclonal Antibody OX40 Antagonist for the Treatment of Atopic Dermatitis. Retrieved December 12, 2024, from https://ir.astriatx.com/news-releases/news-release-details/astria-therapeutics-announces-fda-clearance-ind-application-0 [40] Aptose Clinical Data Featured in Poster Presentation at the 2024 ASH Annual Meeting Support Tuspetinib Triple Drug Therapy for Newly Diagnosed AML. Retrieved December 12, 2024, from https://www.aptose.com/news-media/press-releases/detail/304/aptose-clinical-data-featured-in-poster-presentation-at-the [41] Biomea Fusion Announces Preliminary Data from Ongoing COVALENT-103 Study of Investigational Covalent FLT3 Inhibitor BMF-500 in Relapsed or Refractory Acute Leukemia. Retrieved December 12, 2024, from https://investors.biomeafusion.com/news-releases/news-release-details/biomea-fusion-announces-preliminary-data-ongoing-covalent-103 [42] NextCure Announces Acceptance of IND Application for LNCB74. Retrieved December 12, 2024, from https://www.globenewswire.com/news-release/2024/12/10/2994522/0/en/NextCure-Announces-Acceptance-of-IND-Application-for-LNCB74.html [43] PureTech Founded Entity Seaport Therapeutics Presents Additional Data from Phase 1 Study of SPT-300 at ACNP Annual Meeting 2024. Retrieved December 12, 2024, from https://news.puretechhealth.com/news-releases/news-release-details/puretech-founded-entity-seaport-therapeutics-presents-additional 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新