A Randomized Phase 2 Proof-of-Concept Study to Evaluate the Efficacy and Safety of Topical Bimiralisib Application in Participants Suffering From Actinic Keratosis on the Face and/or Scalp and/or Back of Hands Over a 2 and 4-week Treatment Period

The aim of the study is to evaluate efficacy and safety of bimiralisib gel treatment for treatment of actinic keratosis (AK) on the face and/or scalp and/or back of hands

开始日期2024-04-02

申办/合作机构

Torqur AG

NCT06243354 / Recruiting临床1/2期

An Open-label, Multi-center, Multi-cohort, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of HYP-2090PTSA in Patients With Advanced Solidt Tumors Harboring KRAS Mutation

This is a multicenter, open-label phase 1/2 study consisting of two parts: dose escalation phase and dose expansion phase. The objective of the dose escalation phase is to evaluate the safety, tolerability and pharmacokinetics of HYP-2090PTSA in patients with advanced solid tumors harboring KRAS mutation and to determine the RP2D. In the dose expansion phase, preliminary efficacy and safety at the RP2D will be further explored in patients with specific cancer harboring KRAS p.G12C mutation.

开始日期2024-02-04

申办/合作机构

四川汇宇制药股份有限公司

NCT05764395 / Suspended临床2期IIT

A Phase II Study to Evaluate the Safety and Efficacy of Rigosertib (ON 01910) Plus Pembrolizumab in Patients With Metastatic Melanoma Refractory to Immune Checkpoint Blockade

This phase II clinical trial tests how well rigosertib plus pembrolizumab workings in treating patients with melanoma which cannot be removed by surgery (unresectable) or that has spread from where it first started (primary site) to other places in the body (metastatic), and that has not responded to previous treatment with PD-1 or PD-L1 inhibitors (refractory). Rigosertib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and may change the immune system to make immunotherapy more effective. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving rigosertib in combination with pembrolizumab may be more effective in treating patients with unresectable metastatic melanoma that has not responded to previous treatment with PD-1 or PD-L1 inhibitors than giving either drug alone.

开始日期2023-05-09

申办/合作机构

The Vanderbilt-Ingram Cancer Center

100 项与 Class I PI3K 相关的临床结果

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100 项与 Class I PI3K 相关的转化医学

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0 项与 Class I PI3K 相关的专利（医药）

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152

项与 Class I PI3K 相关的文献（医药）

2024-12-11·Journal of Biomolecular Structure and Dynamics

Quantum biochemistry description of PI3Kα enzyme bound to selective inhibitors

Article

作者: Santos de Oliveira, Francisco Lucas ; Freire, Valder Nogueira ; Freitas de Sousa, Francisca Joseli ; Nunes Azevedo, Francisca Fernanda ; Zanatta, Geancarlo ; Vieira Carletti, Jaqueline

The PI3K class I is composed of four PI3K isoforms that serve as regulatory enzymes governing cellular metabolism, proliferation, and survival. The hyperactivation of PI3Kα is observed in various types of cancer and is linked to poor prognosis. Unfortunately, the development inhibitors selectively targeting one of the isoforms remains challenging, with only few agents in clinical use. The main difficulty arises from the high conservation among residues at the ATP-binding pocket across isoforms, which also serves as target pocket for inhibitors. In this work, molecular dynamics and quantum calculations were performed to investigate the molecular features guiding the binding of selective inhibitors, alpelisib and GDC-0326, into the ATP-binding pocket of PI3Kα. While molecular dynamics allowed crystallographic coordinates to relax, the interaction eergy between each amino acid residues and inhibitors was obtained by combining the Molecular Fractionation with Conjugated Caps scheme with Density Functional Theory calculations. In addition, the atomic charge of ligands in the bound and unbound (free) was calculated. Results indicated that the most relevant residues for the binding of alpelisib are Ile932, Glu859, Val851, Val850, Tyr836, Met922, Ile800, and Ile848, while the most important residues for the binding of GDC-0326 are Ile848, Ile800, Ile932, Gln859, Glu849, and Met922. In addition, residues Trp780, Ile800, Tyr836, Ile848, Gln859 Val850, Val851, Ile932 and Met922 are common hotspots for both inhibitors. Overall, the results from this work contribute to improving the understanding of the molecular mechanisms controlling selectivity and highlight important interactions to be considered during the rational design of new agents.Communicated by Ramaswamy H. Sarma.

2024-01-01·Drug Discovery Today

Development of small-molecule inhibitors that target PI3Kβ

Review

作者: Yang, Haodong ; Cai, Lvtao ; Sheng, Rong ; Yu, Yanzhen ; Gu, Dongyan

Phosphatidylinositol-3 kinase (PI3K) β, a subtype of class I PI3Ks, has an essential role in PTEN-deficient tumors and links to thrombosis, male fertility, and Fragile X syndrome. PI3Kβ-specific targeting therapy could be an efficacious treatment for diseases highly dependent on PI3Kβ, while mitigating the severe toxicity of pan-PI3K inhibitors. Achieving selectivity can be accomplished through three primary strategies, namely, binding to the induced lipophilic pocket, targeting the unique amino acid residue of PI3Kβ, or using atropisomerism to lock conformation. In this review, we focus on advances in the development of these β-isoform-selective PI3K inhibitors, providing potential guidance for the further development of novel clinical candidates.

2023-12-01·Drug Development Research

Discovery of novel pyrido[3,2‐d]pyrimidine derivatives as selective and potent PI3Kδ inhibitors

Article

作者: Bai, Huanrong ; Yuan, Bo ; Zhang, San-Qi ; Sun, Jiajia ; Ma, Mengyan ; Lei, Hao ; Xin, Minhang

AbstractThe δ isoform of class I PI3K (PI3Kδ) has been shown as a promising target for the treatment of hematologic malignancies and immune diseases. Herein, a series of pyrido[3,2‐d]pyrimidine derivatives were designed, synthesized and evaluated for the preliminary bioactivity. Compared with idelalisib, compound S5 exhibited excellent enzyme activity against PI3Kδ (IC50 = 2.82 nM) and strong antiproliferation activity against SU‐DHL‐6 cells (IC50 = 0.035 μM). Besides, S5 inhibited the phosphorylation of Akt, which is downstream of PI3Kδ, in concentration‐dependent manner. In view of the significant improvement in potency of PI3Kδ and selectivity over other PI3K isoforms, Compound S5 deserved further investigation as a promising PI3Kδ inhibitor.

项与 Class I PI3K 相关的新闻（医药）

2023-10-02

·BioPharmaDive

Adlai Nortye IPO gives old Novartis cancer drug new life

Brief: Cancer drug developer Adlai Nortye on Thursday raised $57.5 million in an initial public offering, becoming the 16th biotechnology company to debut on a U.S. stock exchange this year, according to BioPharma Dive data. The company sold 2.5 million shares at $23 apiece — a smaller offering than it had pitched in early August. Nippon Kayaku, a licensing partner and Japan-based pharmaceutical manufacturer, also agreed to acquire $40 million in additional shares in a concurrent private financing deal, according to a regulatory filing. The offering will fund the development of a targeted cancer drug once owned by Novartis. That medicine, formerly known as buparlisib and now AN2025, is in late-stage trials for a form of head and neck cancer and in earlier testing against other solid tumors. Insight: Near the end of the last decade, Novartis had two similar drugs advancing through clinical development. Known as PI3 kinase inhibitors because of the enzyme they target, the drugs steadily moved through human testing. One, Piqray, has since reached market, winning regulatory approval in 2019 for certain breast cancer patients. It’s been modestly successful, generating $373 million in worldwide sales last year. In 2018, Novartis offloaded the other, buparlisib, to Adlai Nortye, then a privately held China-based startup . Novartis did so despite having studied buparlisib in dozens of trials and over 4,200 patients. Among them was a Phase 3 study in a common form of breast cancer that met its main goal, but revealed safety issues that “[did] not support its further development” in that setting, wrote investigators in a paper published in Lancet Oncology in December 2017. Novartis licensed buparlisib to Adlai Nortye for only $9.5 million upfront in a heavily backloaded deal, according to the biotech’s IPO filing. Novartis still stands to receive additional milestone payments and, if the drug is approved, sales royalties. Adlai Nortye has focused on buparlisib’s potential in different settings and in combination with other drugs. The furthest along is a late-stage study testing the drug and chemotherapy in people whose head and neck cancers have progressed after a commonly used immunotherapy. The company, which operates in China and the U.S. through subsidiaries owned by a Cayman Islands holding company, will report findings next year. It claims it could file for an accelerated approval afterwards if results are positive. The biotech is also evaluating buparlisib’s use in solid tumors with so-called PIK3CA mutations, the group for which Piqray is approved for use in breast cancer. The Food and Drug Administration has recently stepped up scrutiny of PI3K inhibitors due to safety concerns that emerged during testing in certain blood cancers. A panel of experts earlier this year voted to raise the approval standards for the medicines, at least when considering broader use in lymphomas and leukemias. Adlai Nortye’s stock offering, which the company initially set plans for in late July, follows positive signs for what has otherwise been a moribund IPO market this year. Earlier this month, RayzeBio and Neumora Therapeutics successfully pulled off lucrative IPOs amid a flurry of activity that also saw chip designer ARM and the grocery delivery company Instacart price offerings. The content comes from the Internet. If there is any infringement, please contact us for deletion.

IPO

2023-08-21

·PRNewswire

Inhalis Therapeutics SA Achieves Key Milestone with Patent Application Filing

LUGANO, Switzerland, Aug. 21, 2023 /PRNewswire/ -- Inhalis Therapeutics SA, a pioneering privately-held company specializing in advanced inhaled treatments for life-threatening diseases, proudly reveals a significant stride forward as it submits a new patent application on INHAL-101, its lead asset. This strategic move strengthens the company's positioning and enriches its intellectual property portfolio. Dr. Silvia Panigone, CEO of Inhalis Therapeutics, expressed her enthusiasm, stating, "The achievement of this pivotal milestone marks a moment of great pride for Inhalis. Our dedicated team has been steadfastly committed to developing INHAL-101, a unique small-molecule PI3K/mTOR/BRD4 tri-specific inhibitor. This ground-breaking formulation employs proprietary mSAS® supercritical fluid technology, demonstrating a compelling profile for addressing the complex challenges of Idiopathic Pulmonary Fibrosis (IPF). By selectively inhibiting the key IPF-driven pathogenic elements through a single inhalable medication, with excellent safety potential, INHAL-101 offers an opportunity to harness its full pharmacological power to be an efficacious and well-tolerated drug. This recent patent application strengthens our competitive positioning and provides additional value for Inhalis. We remain dedicated to our journey, and most importantly, to those people afflicted by IPF who stand to benefit from our unwavering commitment." About Inhalis Therapeutics SA Inhalis Therapeutics SA is committed to develop breakthrough inhaled treatments for life-threatening disorders. Solid science and industry-leading delivery technology are combined with an experienced management team to research and develop new inhaled treatments for Idiopathic Pulmonary Fibrosis and other diseases. The lead asset, INHAL-101, a ground-breaking solution for IPF, is poised to initiate clinical trials in Q4/2024. The company's pipeline also includes INHAL-102, tailored for lung cancer, and additional assets in late-preclinical proof-of-concept stages for oncology, fibrotic diseases and pulmonary infections. For further information, please contact: Silvia Panigone, PhD, EMBA Chief Executive Officer Inhalis Therapeutics SA Phone: +41 78 89 89 644 Email: [email protected] Logo - SOURCE Inhalis Therapeutics SA

2023-07-21

·SYNAPSE

全球新药进展早知道 07-21

药物研发进展 1.翰森制药引进的口服抗真菌新药枸橼酸艾瑞芬净片中国上市申请获受理 7月20日，中国国家药品监督管理局药品审评中心（CDE）官网最新公示，Scynexis公司和翰森制药联合申报了枸橼酸艾瑞芬净片的新药上市申请，并获得受理。公开资料显示，艾瑞芬净（ibrexafungerp，HS-10366）是一种新型的口服抗真菌药物，此前已获美国FDA批准。翰森制药负责该产品在中国的开发、监管批准和商业化。根据翰森制药早前公告介绍，艾瑞芬净是一款全新三贴类结构的糖原合成酶抑制剂，是一种全新作用机制的抗真菌类药物。该药物已被验证可以高效抑制糖原合成酶活性，并具有口服和注射两种剂型方便使用，有望在住院和门诊环境中得到广泛使用。目前，该药正开发用于治疗主要由念珠菌和曲霉菌引起的真菌感染。在体外和体内研究中，它表现出了广谱的抗真菌活性。2021年2月，翰森制药与Scynexis公司签订授权协议，翰森制药将负责艾瑞芬净在中国的开发、监管批准和商业化。2021年6月，艾瑞芬净首次获得FDA批准，用于治疗外阴阴道念珠菌病（VVC，也称为阴道酵母菌感染）。该批准基于两项3期临床研究的积极结果，试验表明，每日口服一次该药在VVC患者中表现出显著的疗效和良好的耐受性。治疗10天后，63.3%患者达到了“临床治愈”的主要终点，58.5%的患者达到了真菌学根除的次要终点，72.3%的患者获得了临床改善。根据中国药物临床试验登记与信息公示平台，此前艾瑞芬净已完成一项在中国成年外阴阴道念珠菌病患者中开展的多中心3期临床试验，目前还在开展一项针对念珠菌血症和/或侵袭性念珠菌病患者的3期临床试验。 2.杨森EGFR/MET双抗3期临床获积极结果，针对特定NSCLC一线治疗 7月20日，强生（Johnson & Johnson）旗下杨森（Janssen）宣布，其EGFR/MET双特异性抗体疗法Rybrevant（amivantamab-vmjw）联合化疗在新诊断EGFR外显子20插入突变的晚期或转移性非小细胞肺癌（NSCLC）患者中开展的3期PAPILLON研究取得积极结果，患者无进展生存期（PFS）出现具有统计学显著性和临床意义的改善。NSCLC大约占所有肺癌的80%~85%。其中最常见的驱动突变是表皮生长因子受体（EGFR）基因变异，EGFR是一种支持细胞生长和分裂的受体酪氨酸激酶。EGFR突变出现在40%~50%的亚洲患者中，所有接受EGFR酪氨酸激酶抑制剂治疗的转移性NSCLC患者的5年生存率低于20%。大约2%~3%的NSCLC患者携带EGFR外显子20插入突变，这些患者对普通EGFR抑制剂的应答不佳，预后尤其不良。Rybrevant是一款人源化EGFR/MET双特异性抗体。它具有多重抗癌作用机制，不但能够阻断EGFR和MET介导的信号传导，还可以引导免疫细胞靶向携带激活性和抗性EGFR/MET突变和扩增的肿瘤。此次取得积极结果的PAPILLON试验是一项随机、开放标签3期研究，目的是在新诊断EGFR外显子20插入突变的晚期或转移性NSCLC患者中，评估Rybrevant联合化疗与单独化疗相比的疗效和安全性。结果显示，PAPILLON研究达到其主要终点，与单独化疗相比，接受Rybrevant联合化疗的患者PFS出现具有统计学意义和临床意义的改善。联合疗法的安全性特征与各方案的安全性特征一致。杨森计划将这些结果提交给即将召开的科学大会进行展示。 3.降低疾病进展或死亡风险达55%！尼拉帕利3期研究在JAMA子刊发表 7月20日，再鼎医药宣布，JAMA Oncology发表了则乐（尼拉帕利）用于新诊断的中国晚期卵巢癌患者一线维持治疗的关键性3期临床研究PRIME的研究数据。数据显示，与安慰剂相比，尼拉帕利用于晚期卵巢癌一线维持治疗可显著延长无进展生存期（PFS），且使疾病进展或死亡风险降低了55%。尼拉帕利是一种每日一次的口服聚（ADP-核糖）聚合酶（PARP）抑制剂，可单药用于晚期卵巢癌治疗。再鼎医药与Tesaro公司（后被葛兰素史克收购）达成了授权与合作协议，负责该产品（再鼎医药独立生产）在中国内地、香港和澳门地区的开发和商业化工作。PRIME研究是一项随机、双盲、安慰剂对照的3期临床研究。该试验在中国29家医院开展，评估了384名新诊断的中国晚期卵巢癌患者在一线含铂化疗达到完全缓解或部分缓解后，接受尼拉帕利一线维持治疗的有效性和安全性。主要研究终点为意向治疗（ITT）人群中通过独立盲法中心评估的PFS。该研究主要疗效和安全性结果如下。与安慰剂相比，尼拉帕利维持治疗显著延长了PFS，且使疾病进展或死亡风险降低了55%，无论术后病灶残留状态和生物标记物状态如何。ITT人群中，尼拉帕利组中位PFS为24.8个月（vs 8.3个月）。数据截止时，ITT人群总生存期（OS）数据尚未成熟。个体化起始剂量的维持治疗安全可耐受。两组受试者≥3级治疗期间出现的不良事件（TEAE）发生率分别为54.5%和17.8%，严重不良事件（SAE）发生率分别为18.8%和8.5%，因不良事件而终止治疗的患者比例相似（6.7% vs 5.4%）。 4.一线治疗宫颈癌，默沙东宣布其Keytruda组合疗法显著延长患者PFS 7月20日，默沙东（MSD）公司宣布其抗PD-1疗法Keytruda（pembrolizumab）联合外照射放疗（EBRT）加同步化疗，随后再进行近距离放疗（brachytherapy，也称为同步放化疗），达到3期试验的主要终点之一，即此联合疗法可显著改善新确诊的高危局部晚期宫颈癌患者的无进展生存期（PFS）。宫颈癌是全球女性第四常见确诊的癌症，2020年全球有超过60.4万名女性被诊断患有宫颈癌，约34.2万名女性因该疾病而死亡。所有女性都有患宫颈癌的风险，但最常确诊于35-44岁女性患者。中国是全球宫颈癌患者数最多的国家之一，宫颈癌新发病例约10.6万例，每年死亡病例约4.8万例。虽然筛查和预防方法已降低了宫颈癌发病率，但这种疾病仍然影响着世界各地的许多女性。Keytruda是默沙东公司开发的重磅抗PD-1疗法，它通过阻断PD-1与其配体PD-L1和PD-L2的相互作用，激活T淋巴细胞，增强人体免疫系统发现和消灭癌细胞的能力。Keytruda在宫颈癌方面获美国FDA批准两项适应症：Keytruda联合化疗，加或不加贝伐珠单抗（bevacizumab），用于治疗持续性、复发性或转移性宫颈癌患者，患者经FDA批准的检测确定肿瘤表达PD-L1（CPS≥1）。Keytruda亦获批准作为单药，用于治疗化疗期间或化疗后疾病产生进展的复发性或转移性宫颈癌患者，患者肿瘤经FDA批准的检测确定表达PD-L1（CPS≥1）。这次所公布的KEYNOTE-A18试验是一项随机双盲的3期临床试验。由独立数据监测委员会进行的预定中期分析显示，与单独同步放化疗相比，Keytruda联合同步放化疗使得患者的PFS出现具统计学和临床意义的改善。与单独同步放化疗相比，Keytruda联合同步放化疗在OS方面也观察到了有利的趋势。 5.必贝特医药PI3K/HDAC双靶点抑制剂双利司他拟纳入优先审评 7月19日，中国国家药品监督管理局药品审评中心（CDE）官网公示，必贝特医药申请的注射用BEBT-908（注射用双利司他）拟纳入优先审评，拟定适应症为：既往接受过至少两种系统治疗的复发或难治弥漫性大B细胞淋巴瘤（r/r DLBCL）成人患者。公开资料显示，BEBT-908是一款具有全新化学结构的PI3K/HDAC双靶点抗肿瘤1类新药，此前针对r/r DLBCL适应症已被CDE纳入突破性治疗品种。PI3K是磷脂酰肌醇-3-激酶。PI3K/AKT/mTOR是细胞内重要的信号通路之一，在调节细胞生长、运动、存活、代谢和血管生成过程中起着重要作用。研究发现，PI3K信号通路的过度活跃与肿瘤的进展、肿瘤微血管密度的增加、癌细胞的趋化性和侵袭性增强有显著的相关性。HDAC（组蛋白去乙酰化酶）控制着细胞的存活、增殖、血管生成、炎症和免疫，在细胞信号传导中起着重要作用。由于HADC在基因表达中的基本作用以及对组蛋白和非组蛋白的不同影响，它已被认为是治疗淋巴瘤等恶性肿瘤的理想靶点。根据必贝特医药官网资料，BEBT-908是一种双靶点PI3K和HDAC抑制剂，可选择性抑制肿瘤赖以生存的核心靶点，协同性破坏肿瘤信号通路，从而有效抑制肿瘤细胞生长。同时，该产品还能增强小鼠结直肠癌中的抗PD-1抗体的免疫治疗效果。根据CDE公示，此次BEBT-908拟纳入优先审评，针对适应症为r/r DLBCL。DLBCL是一种侵袭性强、生长迅速的非霍奇金淋巴瘤（NHL），是常见的NHL类型。DLBCL患者通常接受化疗免疫疗法为基础的治疗方案。对于复发/难治性患者，近期出现了几种包括T细胞介导治疗在内的靶向疗法。然而，单药和现货型治疗选择有限。公开资料显示，此前BEBT-908已完成治疗r/r DLBCL的2a期临床研究，初步获得了安全有效性数据。 6. argenx重磅疗法迎新适应症临床积极进展近日，argenx公司宣布，其Vyvgart Hytrulo皮下注射液用于治疗慢性炎症性脱髓鞘性多发神经根神经病（CIDP）成人患者的2期临床试验取得了积极的初步结果。该研究达到了主要终点，与安慰剂相比，Vyvgart Hytrulo使患者的复发风险降低了61%（p=0.000039）。CIDP是一种罕见而严重的外周神经系统自身免疫性疾病。尽管该疾病的病理生理学尚未得到证实，但越来越多的证据表明，IgG抗体在周围神经损伤中起着关键作用。CIDP患者会感到疲倦、肌肉无力、手脚失去知觉，这些症状可能会随着时间的推移而加重，也可能时好时坏。这些症状会严重影响患者的日常生活能力。如果不进行治疗，约三分之一的CIDP患者将需要坐轮椅。Vyvgart Hytrulo是efgartigimod alfa和重组人透明质酸酶PH20的皮下注射产品组合。Efgartigimod是一款靶向Fc受体（FcRn）的“first-in-class”疗法。Efgartigimod可减少致病性IgG抗体，阻断IgG再循环过程。FcRn受体的作用是防止IgG的降解，因此通过防止IgG与FcRn的结合，能够导致介导自身免疫性疾病的IgG抗体更快耗竭，从而减轻疾病症状。重组人透明质酸酶PH20由Halozyme Therapeutics公司开发，它可以降解体内的透明质酸，以帮助皮下注射药物的渗透和吸收，为患者提供额外的治疗选择。2023年6月，美国FDA已经批准Vyvgart Hytrulo皮下注射液上市，用于治疗抗乙酰胆碱受体（AChR）抗体阳性的成人全身性重症肌无力（gMG）患者。新闻稿指出，这是FDA批准治疗这一患者群体的首款皮下注射疗法。 7.阿尔茨海默病抗体疗法试验结果积极，显著降低大脑淀粉样蛋白水平近日，Acumen Pharmaceuticals公布了靶向毒性可溶性β淀粉样蛋白寡聚体（AβOs）的单克隆抗体ACU193的首个人体临床试验结果。结果显示，试验达到主要和次要目标，证明了ACU193的作用机制和安全性。在早期阿尔茨海默病（AD）患者中，ACU193剂量依赖性迅速降低患者的淀粉样蛋白斑块水平。阿尔茨海默病是老年人中最常见的神经退行性疾病。在患者症状开始前20年，其脑部便出现复杂的变化，最终造成脑细胞死亡或连接丧失。淀粉样蛋白沉积是患者大脑的标志性特征，靶向淀粉样蛋白是阿尔茨海默病新药开发的重要方向之一。随着近年来两款抗淀粉样蛋白抗体疗法获得FDA的批准，这一研发策略也受到广泛关注。ACU193是一种针对可溶性AβOs开发的选择性人源化单克隆抗体。Acumen认为，相比于Aβ淀粉样蛋白单体和淀粉样蛋白斑块，AβOs是毒性和致病性最强的一种形式。可溶性的AβOs已被观察到能与神经元结合，抑制突触功能并诱导神经变性，是一种强效的神经毒素。ACU193能够结合AβOs，由于其独特的结合特性，ACU193有可能在产生淀粉样蛋白相关成像异常（ARIA，一种抗体疗法产生的常见主要副作用）风险较低的情况下提供治疗益处，因为ACU193可以在不直接靶向淀粉样斑块的情况下阻断AβOs的毒性作用。在这项随机双盲，含安慰剂对照的1期临床试验中，早期AD患者接受不同剂量的ACU193或安慰剂的治疗。试验结果显示，ACU193表现出良好的耐受性和安全性特征，达到试验的主要目标。最常见的治疗后出现不良事件为ARIA，总体来说10.4%的患者出现ARIA-E（意味着大脑某个或多个区域因为ARIA出现暂时性肿胀）。出现症状的AIRA-E比例为2.1%。 8.星济生物单域抗体-Fc融合蛋白获批临床 7月20日，中国国家药监局药品审评中心（CDE）官网公示，星济生物1类新药注射用XJ101获批临床，拟定适应症为：金黄色葡萄球菌血流感染，包括甲氧西林敏感金黄色葡萄球菌（MSSA）和耐甲氧西林金黄色葡萄球菌（MRSA）引起的菌血症、败血症和脓毒症等。根据星济生物新闻稿，注射用XJ101为重组人源化单域抗体-Fc融合蛋白。星济生物于2020年6月由复星医药旗下复健新药基金、重庆药友制药携手安毛毛博士及李博华博士共同投资创建，是一家专注于抗感染大分子药物（抗体药物、长效融合蛋白药物等）研究与开发的公司。血流感染（BSI）是指病原微生物进入机体血流，并释放毒素和代谢产物，从而引起的全身播散性感染，是临床上严重的感染类型之一。金黄色葡萄球菌是导致血流感染常见的革兰阳性细菌。据公开数据报道，全球每年与金黄色葡萄球菌血流感染相关性死亡超70万例，65岁以上患者死亡率高达30%以上，造成了巨大的疾病负担。耐甲氧西林金黄色葡萄球菌（MRSA）是在全球范围内流行的“超级”耐药菌，对几乎所有的β-内酰胺类抗生素均耐药，同时还可能对其他多种类抗生素表现出交叉耐药。MRSA感染导致的临床治疗失败率显著高于甲氧西林敏感的金黄色葡萄球菌（MSSA）。然而，目前临床上只有少数抗菌药物可以用于MRSA血流感染。万古霉素是治疗MRSA血流感染的一线药物，但是临床治疗失败率较高，临床上急需更加有效的药物用于治疗金黄色葡萄球菌导致的血流感染。世界卫生组织发布迫切需要新型抗生素的细菌清单，将MRSA定义为急需开发新型药物的耐药病原体。行业资讯普米斯生物与BioNTech公司达成许可协议 7月20日，普米斯生物宣布与BioNTech公司达成战略研究合作、项目引进选择以及全球许可协议。根据协议条款，BioNTech公司将获得普米斯生物用于治疗恶性肿瘤的一款临床前研究阶段双特异性抗体和一款临床研究阶段单克隆抗体的全球独家选择权。相应地，普米斯生物将获得BioNTech公司提供的预付款，并在BioNTech公司选定临床前研究阶段双特异性抗体后，获得临床研究、注册和商业里程碑付款以及分级销售提成。BioNTech是一家致力于开创癌症及其他重大疾病创新疗法的新一代免疫疗法公司。普米斯生物则聚焦恶性肿瘤及自身免疫疾病，致力于1类创新生物药的研发及产业化。公开资料显示，普米斯生物正在推进超过20个1类生物新药项目，且多个新药项目处于临床研究阶段，针对靶点包括PD-L1 + TGFβ、PD-L1 + VEGF、TIGIT、TIGIT + PVRIG、4-1BB + PD-L1、CD73、EGFR + cMET等。普米斯生物联合创始人、董事长兼首席执行官刘晓林先生表示，普米斯生物一直致力于利用创新技术解决未被满足的临床需求，与创新疗法领域公司BioNTech的合作将极大地推进他们的使命达成。他们希望通过这次合作，集合双方优势，共同研究出具有广阔治疗潜力的创新疗法，惠及全球广大患者。