LPX-TI641

BOSTON, Nov. 07, 2023 (GLOBE NEWSWIRE) -- LAPIX Therapeutics, Inc. (“LAPIX”), a biopharmaceutical company focused on developing novel, orally bioavailable immune system restoration therapies for autoimmune diseases, today announced that the first participants have been dosed in the Phase 1 study of the Company’s LPX-TI641therapy. LPX-TI641 is a novel, and proprietary first-in-class orally bioavailable small molecule designed as a T cell immunoglobulin and mucin domain-containing protein (Tim) 3/4 receptor agonist. The Tim family of receptors plays an important role in autoimmunity. LPX-TI641 restores the balance of regulatory immune cells (T- and B-regs) to autoreactive T-cells reestablishing immune tolerance. It is being developed for the treatment of Multiple Sclerosis (MS) and potentially other autoimmune diseases such as rheumatoid arthritis and lupus. “The dosing of the first participants is a major milestone in the clinical development of LPX-TI641,” said Anas M. Fathallah, Ph.D., Chief Executive Officer and co-founder of LAPIX. “We are excited to begin the clinical evaluation of our antigen-agnostic immune tolerance restoration therapy. We look forward to evaluating the therapeutic potential of LPX-TI641 and believe it will be a transformative and safer option for patients suffering from MS and other autoimmune diseases. The design of the Phase I study will allow us the flexibility to leverage the clinical data for other indications, enabling rapid pivots into other autoimmune diseases,” added Dr. Fathallah. The study is a randomized, double-blind, placebo-controlled study in healthy adult volunteers to evaluate the safety, tolerability, and pharmacokinetics of LPX-TI641 after single ascending oral doses. Exploratory pharmacodynamic and biomarker analysis will also be evaluated as part of this study. More information about the study can be found at NCT05853835 About LPX-TI641 LPX-TI641’s primary pharmacology is the upregulation of Foxp3+/CD4+ T-cells (T-regs) and of Tim1+/CD25+/CD19+ (B-regs) and inhibition/downregulation of Th17. The combined effects of LPX-TI641’s pharmacology is to allow the adaptive immune system to re-establish self-tolerance (T-reg/Th17 balance and B-regs) without affecting the innate immune system. The efficacy of LPX-TI641 versus standards of care has been evaluated in several animal models of MS and those models have shown favorable efficacy of LPX-TI641 in treatment escalation and treatment induction/maintenance paradigms mimicking current clinical management paradigms of MS without inducing neutropenia or lymphocytopenia suggesting better safety as well. It is currently under development for neuro-autoimmune indications such as MS, with the intent to expand into rheumatoid arthritis and lupus amongst other autoimmune conditions. About LAPIX Therapeutics LAPIX Therapeutics Inc. is a Boston, MA-based company in the biopharma sector, focused on developing novel, orally bioavailable immune system restoration therapies, including immune tolerance restoration therapy for autoimmune diseases, immune tolerance induction therapies for gene therapy, and immune function restoration therapies for oncology. To learn more, visit or follow us on LinkedIn and X. Contact: Anas M. Fathallah, Ph.D. CEO and Co-Founder info@lapixtherapeutics.com

▎药明康德内容团队编辑本期看点1. 新一代高选择性CDK9小分子抑制剂SLS009用于复发/难治性（r/r）淋巴瘤患者的临床研究结果积极，96%的患者在最后一次评估时存活，获得临床缓解的患者的肿瘤负荷减少达62%。2. 一种基于人类巨细胞病毒（HCMV）的在研皮下注射人类免疫缺陷病毒（HIV）T细胞疫苗VIR-1388的1期临床试验于近期完成了首例患者给药。3. 新型吸入式基因疗法KB408的IND申请获FDA批准，用于治疗α1-抗胰蛋白酶缺乏症（AATD）。药明康德内容团队整理SLS009（GFH009）：公布1期临床试验数据 SELLAS Life Sciences公布了其获独家授权的新一代高选择性CDK9小分子抑制剂SLS009在1期临床试验的r/r淋巴瘤患者队列中的积极顶线结果。临床前实验数据显示，SLS009对CDK9蛋白的选择性抑制效力超过其它CDK亚型100倍以上。此次试验共有52例r/r淋巴瘤患者入组，96%的患者在最后一次评估时存活，表明生存获益显著。在34名可评估的患者中，有5人（14.7%）获得了临床缓解，肿瘤负荷减少达62%，另有7名（20.6%）患者实现疾病稳定（SD），总体疾病控制率为35.3%。其中，外周T细胞淋巴瘤患者的临床缓解率为36.4%（4/11）。在每周给药一次的治疗方案中，100%的患者观察到生物标志物MCL1和/或MYC呈剂量依赖性下降。安全性方面，在任何剂量水平下均未出现脱靶安全性问题。VIR-1388：1期临床试验完成首例患者给药 VIR-1388是Vir Biotechnology公司开发的一种基于HCMV的在研皮下注射HIV T细胞疫苗。与之前的在研HIV疫苗不同，VIR-1388旨在诱导大量T细胞识别HIV蛋白，从而预防HIV感染。HCMV载体是一种弱化版的病毒，旨在将HIV疫苗成分传递给免疫系统，而不会导致受试者患病。几个世纪以来，HCMV一直存在于全球大部分人口中。大多数HCMV感染者没有任何症状，也不知道自己感染了这种病毒。HCMV在人体内终生可被检测到，这表明它们有可能输送并安全地帮助人体长期保留艾滋病疫苗成分，从而有望克服在使用寿命较短的疫苗载体时观察到的免疫反应下降问题。目前，VIR-1388正处于1期临床试验中接受安全性、反应原性和免疫原性的评估。KB408：IND申请获得FDA许可 KB408是Krystal Biotech公司以存在复制缺陷、失去了整合基因组能力的1型单纯疱疹病毒（HSV-1）作为载体开发的新型吸入（雾化）制剂，旨在递送编码人α-1抗胰蛋白酶的SERPINA1转基因的两个全长拷贝，用于治疗AATD。近期，KB408的IND申请获得了FDA的许可，该公司计划在2024年第1季度开展1期临床试验。此前，FDA曾授予KB408孤儿药资格，用于治疗AATD。RGLS8429：公布1b期临床试验数据Regulus Therapeutics公司公布了其开发的新型下一代寡核苷酸疗法RGLS8429用于治疗常染色体显性多囊肾病（ADPKD）的1b期临床试验数据。常染色体显性多囊肾病是由PKD1或PKD2基因突变引起的，也是最常见的人类单基因疾病之一，同时是终末期肾病的主要原因。RGLS8429旨在抑制miR-17并优先靶向肾脏，在临床前研究中已被证明能够明显改善肾脏功能、大小和其他疾病严重程度指标，并具有优越的药理学特征。此前，该公司已公布1期单次递增剂量临床试验的结果，结果显示，RGLS8429具有良好的安全性、耐受性和药代动力学（PK）特征。此次公布的结果显示，患者对RGLS8429的耐受性良好，未发现任何安全性问题。经RGLS8429治疗后，与疾病相关的尿液生物标志物尿多囊蛋白1（PC1）有统计学意义的显著增加，并且尿多囊蛋白（PC1和PC2）在ADPKD患者中显示出预期的药代动力学/药效学（PK/PD）关联性。Briquilimab：公布1b期临床试验的新数据Jasper Therapeutics公布了其c-KIT（CD117）单克隆抗体briquilimab作为治疗范可尼贫血（Fanconi Anemia）预处理剂的的积极早期临床结果。结果显示，所有接受briquilimab治疗的患者（3例）均实现了完全供体植入和全血细胞计数恢复。此外，briquilimab的耐受性良好，无任何并发症。PLG0206：公布1b期临床试验的新数据Peptilogics公司公布了其在研广谱、抗菌和抗生物膜肽治疗药物PLG0206用于治疗假体周围感染（PJI）的1b期临床试验的新数据。PLG0206通过独特的作用机制靶向并破坏细菌膜，可直接作用于难以被常规抗生素清除的细菌病原体。在体外和非临床动物研究中，PLG0206表现出卓越的广谱、高效杀菌活性，对包括世界卫生组织和疾病预防控制中心认定的具有重要、紧急或高优先级的各种病原体均有效，无论其耐药性表型如何。PLG0206已被美国FDA授予用于治疗PJI的孤儿药资格，并获得了快速通道资格。此次公布的结果显示，PLG0206在PJI患者中的应用显示出良好的安全性和有效性迹象，高、低剂量组患者均未报告与治疗相关的严重不良事件。此外，接受PLG0206的患者在清创术后、抗生素和植入物保留（DAIR）手术后180天时的感染复发率为7%（1/14），与此前其它研究中报告的类似群体在术后180天时的感染复发率（45%）相比显著下降（p=0.029）。GM-1020：完成1期临床试验Gilgamesh Pharmaceuticals公司宣布其新型非竞争性小分子NMDA通道阻滞剂GM-1020完成了1期单剂量递增和多剂量递增试验。这种新一代疗法旨在改变抑郁症、双相情感障碍、强迫症和其他使人衰弱的精神疾病的治疗格局。这种开创性的疗法是在现有氯胺酮疗法的基础上发展起来的，有望成为一种方便、耐受性良好的家庭治疗选择。此次公布的结果显示，该候选疗法的口服剂量耐受性良好，未发现严重不良反应，并通过脑电图确认了靶点作用。LP-284：IND申请获得FDA许可Lantern Pharma近日宣布，美国FDA已批准其在研新型小分子候选疗法LP-284的IND申请。LP-284是Lantern利用其专有的人工智能和机器学习平台RADR加速和重点开发的具有合成致死作用机制的小分子疗法，可优先对DNA损伤修复通路中携带突变的癌细胞造成损伤。该疗法拟用于治疗复发或难治性非霍奇金淋巴瘤（NHL），包括套细胞淋巴瘤（MCL）、双重打击淋巴瘤（DHL），以及其他高级别B细胞淋巴瘤（HGBL）。Lantern预计将于2023年第4季度开始招募LP-284首次人体1期试验的患者。LPX-TI641：IND申请获得FDA许可LPX-TI641是LAPIX Therapeutics公司开发的一种在研口服生物可利用的小分子T细胞免疫球蛋白和含粘蛋白结构域蛋白（TIM）3/4受体激动剂，正在开发用于治疗神经-自身免疫适应症（如多发性硬化），并打算扩展到类风湿性关节炎和其他自身免疫性疾病。TIM家族受体在自身免疫中发挥着重要作用。LPX-TI641的主要药理作用是上调Foxp3+/DC4+ T细胞（T-regs）和Tim1+/CD25+/CD19+（调节性B细胞，B-regs），以及抑制/下调Th17。LPX-TI64的药理学综合作用是在不影响先天性免疫系统的情况下，让适应性免疫系统重建自我耐受性（Treg/Th17平衡和B-regs）。研究人员在多个多发性硬化动物模型中评估了LPX-TI641与标准疗法的疗效，这些模型显示，LPX-TI641在诱导/维持治疗上具有良好的疗效，模拟了目前多发性硬化的临床治疗模式，且不会诱发中性粒细胞减少症或淋巴细胞减少症。LPX-TI641用于治疗多发性硬化的IND申请已获得FDA批准，预计将于2023年第4季度开始给药。CF-370：向FDA提交IND申请CF-370是ContraFect公司开发的一种工程化的溶素候选疗法，对铜绿假单胞菌具有强活性。该公司官网资料显示，CF-370是首个能穿透铜绿假单胞菌外膜并在人体血清中发挥强效活性的溶素。此外，CF-370还表现出了溶素类药物的体外特征，包括快速而有效的杀菌活性、与多种标准治疗药物的协同作用以及在临床前研究中能够摧毁生物膜。在兔肺炎模型中，CF-370具有良好的耐受性，并能提高动物的存活率。接受CF-370治疗动物的存活率为100%，而接受对照药物治疗动物的存活率仅为40%。此外，CF-370与美罗培南具有协同作用，与单独使用这两种药物相比，模型动物的平均肺部细菌计数显著减少。ContraFect公司已于近期向FDA递交了CF-370的IND申请，拟用于治疗医院获得性细菌性肺炎和呼吸机相关性细菌性肺炎。大家都在看药明康德为全球生物医药行业提供一体化、端到端的新药研发和生产服务，服务范围涵盖化学药研发和生产、生物学研究、临床前测试和临床试验研发、细胞及基因疗法研发、测试和生产等领域。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲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] Harpoon Therapeutics Announces First Patients with Small Cell Lung Cancer Dosed in HPN328 Combination Cohort. 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Retrieved September 21, 2023, from https://www.prnewswire.com/news-releases/gilgamesh-pharmaceuticals-successfully-completes-phase-1-sadmad-clinical-trials-of-gm-1020-301934271.html[17] Via Nova Therapeutics Announces FDA clearance of Investigational New Drug (IND) application for VNT-101, a novel direct-acting antiviral against Influenza A virus. Retrieved September 21, 2023, from https://www.prnewswire.com/news-releases/via-nova-therapeutics-announces-fda-clearance-of-investigational-new-drug-ind-application-for-vnt-101-a-novel-direct-acting-antiviral-against-influenza-a-virus-301934192.html[18] SELLAS Announces Positive Topline Data in Lymphoma Cohort from SLS009 Phase 1 Dose-Escalation Trial, Supporting Advancement to Phase 2 Clinical Study; Primary and Secondary Endpoints Met. 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Retrieved September 22, 2023, from https://www.globenewswire.com/news-release/2023/9/21/2747316/0/en/Krystal-Biotech-Announces-FDA-Clearance-of-Investigational-New-Drug-Application-for-KB408-for-the-Treatment-of-Type-1-Alpha-1-Antitrypsin-Deficiency.html 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