MDNA-11

Ten (10) patients have achieved an objective response (5 confirmed) when treated with MDNA11 alone or in combination with KEYTRUDA® (pembrolizumab) – all responders had advanced and/or metastatic cancers with resistance to immune checkpoint inhibitors (ICI) or with historically low responses to ICI Combination of MDNA11 with KEYTRUDA in the dose escalation arm achieved an objective response rate (ORR) of 36% (5 of 14) in all tumor types enrolling in the Phase 2 expansion cohorts and an ORR of 31% (4 of 13) in cancers planned for the Phase 2 combination expansion cohort Patients from MDNA11 monotherapy dose expansion and dose escalation arms treated at ≥ 60 µg/kg achieved an ORR of 29.4% (5 of 17) in all tumor types enrolling in the Phase 2 expansion cohorts and an ORR of 40% (4 of 10) in cancers enrolling in the Phase 2 monotherapy expansion cohort Highest ORR of 50% achieved amongst MSI-H patients receiving MDNA11 monotherapy (2 of 4) and endometrial cancer patients receiving a combination of MDNA11 and KEYTRUDA (2 of 4) Monotherapy arm continues to demonstrate encouraging durability with a melanoma patient and a pancreatic cancer patient remaining tumor-free and off-treatment after entering the study 2 and 3 years ago, respectively Enrollment in the Phase 2 combination dose expansion arm is underway with the MDNA11 combination Recommended Dose for Expansion (cRDE) established at 90 µg/kg (every 2 weeks) together with KEYTRUDA (400 mg every 6 weeks) Tumor types being evaluated in the Phase 2 expansion cohorts include MSI-H/dMMR, TMB-H, virally-derived cancers (monotherapy), gynecological cancers (combination) and cutaneous melanoma with (combination) or without primary resistance to ICI (monotherapy and combination arms) Additional monotherapy and combination data from the ABILITY-1 study expected at future medical conferences throughout this calendar year TORONTO and HOUSTON, April 28, 2025 (GLOBE NEWSWIRE) -- Medicenna Therapeutics Corp. (“Medicenna” or the “Company”) (TSX: MDNA, OTCQX: MDNAF), a clinical-stage immunotherapy company focused on the development of Superkines targeting cancer and autoimmune diseases, today presents updated clinical data from the ongoing Phase 1/2 ABILITY-1 study evaluating MDNA11 in patients with advanced solid tumors at the 2025 Annual Meeting of the American Association for Cancer Research (AACR) in Chicago, Illinois. MDNA11, a long-acting ‘beta-enhanced not-alpha’ interleukin-2 (IL-2) super-agonist, is being evaluated as a monotherapy or in combination with Merck’s (known as MSD outside of the US and Canada) anti-PD-1 therapy, KEYTRUDA® (pembrolizumab). “We are encouraged by the anti-tumor activity and safety of MDNA11 in both the monotherapy and combination setting, including the 3 recent responders, particularly in patients who have shown resistance to checkpoint inhibitors or with tumor types with historically low responses to immunotherapies and addressing a vastly underserved cancer population,” commented Fahar Merchant, PhD, President and CEO of Medicenna. “Today’s efficacy and immunodynamic data have provided the foundation to commence the dose expansion portion for MDNA11 in combination with KEYTRUDA. In the monotherapy setting, we continue to see deep and durable single agent activity of MDNA11 with two patients remaining in complete remission and off therapy. Together, these data reinforce the best-in-class potential for MDNA11 as a potent and safe immunotherapy that is poised to transform the existing treatment paradigm for patients with difficult-to-treat tumors. We look forward to sharing additional clinical data from the ABILITY-1 study at future medical conferences this year.” Key findings from the on-going ABILITY-1 study (data cut-off as of April 15, 2025) include: Safety Profile MDNA11 continues to demonstrate an acceptable safety profile both as a single agent and in combination with KEYTRUDA. Over 90% of treatment-related adverse events (TRAEs) were Grade 1-2 and transient, with no dose limiting toxicities (DLTs) observed with MDNA11 at doses up to 120 µg/kg (Q2W) in monotherapy and in combination with KEYTRUDA (400 mg, Q6W). Immunodynamics Robust expansion of immune effector cells in monotherapy and combination with KEYTRUDA with notable increases in effector (DNAM), ‘stemness-like’ (TCF-1) and memory CD8+ T cells, which are critical for sustained anti-tumor responses.Robust, dose-dependent lymphocyte expansion in combination with KEYTRUDA, sustained with repeat MDNA11 dosing, and plateauing at the 90 µg/kg MDNA11 dose level. Clinical Activity of MDNA11 in Combination with KEYTRUDA during Dose Escalation Enrollment in the combination dose expansion portion of the ABILITY-1 study is underway having established the MDNA11 combination Recommended Dose for Expansion (cRDE) at 90 µg/kg administered every 2 weeks together with KEYTRUDA administered at 400 mg every 6 weeks. Encouraging anti-tumor activity continues to be observed with MDNA11 in combination with KEYTRUDA in patients enrolled in the dose escalation portion of the study: 5 objective responses (1 CR and 4 PRs) among heavily pre-treated patients, including two previously reported objective responses in historically low immunotherapy responders: 70-year-old male with anal squamous cell carcinoma (SCC) who previously progressed on two prior lines of chemotherapy achieved a CR on the first study evaluable imaging scan (week 8); patient continues on combination treatment following a confirmed CR.52-year-old female with TMB-H/MSS colorectal cancer who previously progressed on two prior lines of chemotherapy achieved a confirmed PR. While off-study for 4 months, the patient continues to show deepening of the tumor response in the absence of any treatment. Objective response rate (ORR) of 31% (4 of 13) in patients with cancers planned for the Phase 2 combination expansion cohort, including cutaneous melanoma, MSI-H/dMMR, and TMB-H tumors, and an ORR of 36% (5 of 14) when including the virally-derived cancers.Stable disease (SD) in 3 patients for a disease control rate (DCR = CR+PR+SD) of 57% (8/14). Monotherapy Tumor Response in Immune Checkpoint Inhibitor-Resistant Patients MDNA11 continues to demonstrate encouraging deep and durable single-agent anti-tumor activity among patients who progressed on prior immune checkpoint inhibitor (ICI) therapy: ORR of 40% in the monotherapy dose escalation (treated at ≥ 60 μg/kg) and expansion arm (1 confirmed CR, 1 confirmed PR and 2 unconfirmed PRs) among 10 patients with tumor types currently enrolling in the Phase 2 monotherapy expansion cohort. All patients had previously failed ICI therapy and had advanced and/or metastatic cutaneous melanoma with secondary resistance to ICI or MSI-H/dMMR tumors. Note that the non-melanoma skin cancer cohort is not recruiting as it was replaced with a new cohort of virally-derived cancers subsequent to a protocol amendment. The ORR is 29.4% from a total of 17 patients when including patients with cancers planned for Phase 2 expansion cohort and patients with primary ICI resistant melanoma who received at least 60 µg/kg of MDNA11 (Q2W).Overall, objective responses in ICI-resistant patients include 1 CR and 4 PRs 2 PRs among 4 MSI-H patients (ORR of 50%) with both responders having metastatic pancreatic ductal adenocarcinoma (PDAC).1 CR and 1 PR among 6 patients with ICI secondary resistant melanoma (ORR of 33.3%).1 PR among patients with ICI primary resistant melanoma. Complete resolution of all target and non-target lesions in two patients with on-going remission following end-of-treatment: Cutaneous melanoma patient achieved a durable response exceeding 12 months during the study with complete resolution of target and non-target metastatic lesions, and continues to remain off anti-cancer therapy for more than 6 weeks.Pancreatic cancer patient (MSI-H) achieved a durable response for 20 months during the study with complete resolution of target and non-target metastatic lesions in the liver including a new lesion treated with MDNA11 following a single cycle of radiation, and continues to remain off anti-cancer therapy for more than 12 months. SD in 6 patients for a disease control rate (DCR = CR+PR+SD) of 65% including 2 with duration > 6 months, yielding a clinical benefit rate of 41% (7/17). A copy of the poster and related slide deck has been posted on the “Scientific Presentations” page of Medicenna’s website. KEYTRUDA® is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA. About MDNA11 MDNA11 is an intravenously administered, long-acting, ‘beta-enhanced not-alpha’ IL-2 Superkine specifically engineered to overcome the shortcomings of aldesleukin and other next generation IL-2 variants by preferentially activating immune effector cells (CD8+ T and NK cells) responsible for killing cancer cells, with minimal or no stimulation of immunosuppressive Tregs. These unique proprietary features of the IL-2 Superkine have been achieved by incorporating seven specific mutations and genetically fusing it to a recombinant human albumin scaffold to improve the pharmacokinetic (PK) profile and pharmacological activity of MDNA11 due to albumin’s natural propensity to accumulate in highly vascularized sites, in particular tumor and tumor draining lymph nodes. MDNA11 is currently being evaluated in the Phase 1/2 ABILITY-1 study as both monotherapy and in combination with KEYTRUDA®. About the ABILITY-1 Study The ABILITY-1 study (NCT05086692) is a global, multi-center, open-label study that assesses the safety, tolerability, pharmacokinetics, pharmacodynamics and anti-tumor activity of MDNA11 as monotherapy or in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab). In the combination dose escalation portion of the Phase 2 study, patients have been enrolled and administered ascending doses of MDNA11 intravenously in combination with KEYTRUDA. This portion of the study includes patients with a wide range of solid tumors with the potential for susceptibility to immune modulating therapeutics. The combination Recommended Dose for Expansion (cRDE) has been established and the study has commenced combination dose expansion. About Medicenna Therapeutics Medicenna is a clinical-stage immunotherapy company focused on developing novel, highly selective versions of IL-2, IL-4 and IL-13 Superkines and first-in-class Empowered Superkines. Medicenna’s long-acting IL-2 Superkine, MDNA11, is a next-generation IL-2 with superior affinity toward CD122 (IL-2 receptor beta) and no CD25 (IL-2 receptor alpha) binding, thereby preferentially stimulating cancer-killing effector T cells and NK cells. MDNA11 is being evaluated in the Phase 1/2 ABILITY-1 Study (NCT05086692) as a monotherapy and in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab). Medicenna’s IL-4 Empowered Superkine, bizaxofusp (formerly MDNA55), has been studied in 5 clinical trials enrolling over 130 patients, including a Phase 2b trial for recurrent GBM, the most common and uniformly fatal form of brain cancer. Bizaxofusp has obtained FastTrack and Orphan Drug status from the FDA and FDA/EMA, respectively. Medicenna’s early-stage high-affinity IL-2β biased IL-2/IL-15 Super-antagonists, from its MDNA209 platform, are being evaluated as potential therapies for autoimmune and graft-versus host diseases. Medicenna’s early-stage BiSKITs™ (Bifunctional SuperKine ImmunoTherapies) and the T-MASK™ (Targeted Metalloprotease Activated SuperKine) programs are designed to enhance the ability of Superkines to treat immunologically “cold” tumors. For more information, please visit www.medicenna.com, and follow us on Twitter and LinkedIn. Forward-Looking Statements This news release may contain forward-looking statements within the meaning of applicable securities laws. Forward-looking statements include, but are not limited to, express or implied statements regarding the future operations of the Company, estimates, plans, strategic ambitions, partnership activities and opportunities, objectives, expectations, opinions, forecasts, projections, guidance, outlook or other statements that are not historical facts, such as statements on the therapeutic treatment potential and safety profile of MDNA11 (both as monotherapy and in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® [pembrolizumab]) and the timing and/or release of any additional clinical updates. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage pre-clinical or clinical studies may not be indicative of full results or results from later stage or larger scale clinical studies and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented. Forward-looking statements are often identified by terms such as “will”, “may”, “should”, “anticipate”, “expect”, “believe”, “seek”, “potentially” and similar expressions. and are subject to risks and uncertainties. Forward-looking statements are based on a number of assumptions believed by the Company to be reasonable at the date of this news release. Although the Company believes that the expectations reflected in such forward-looking statements are reasonable, there can be no assurance that such statements will prove to be accurate. These statements are subject to certain risks and uncertainties and may be based on assumptions that could cause actual results and future events to differ materially from those anticipated or implied in such statements. Important factors that could cause actual results to differ materially from the Company’s expectations include the risks detailed in the latest annual information form of the Company and in other filings made by the Company with the applicable securities regulators from time to time in Canada. The reader is cautioned that assumptions used in the preparation of any forward-looking information may prove to be incorrect. Events or circumstances may cause actual results to differ materially from those predicted, as a result of numerous known and unknown risks, uncertainties, and other factors, many of which are beyond the control of the Company. The reader is cautioned not to place undue reliance on any forward-looking information. Such information, although considered reasonable by management, may prove to be incorrect and actual results may differ materially from those anticipated or implied in forward-looking statements. Forward-looking statements contained in this news release are expressly qualified by this cautionary statement. The forward-looking statements contained in this news release are made as of the date hereof and except as required by law, we do not intend and do not assume any obligation to update or revise publicly any of the included forward-looking statements. This news release contains hyperlinks to information that is not deemed to be incorporated by reference in this new release. Investor and Company Contact: Christina CameronInvestor Relationsir@medicenna.com(647) 953-0673

▎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. 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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. 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