A Randomized, Double-blind, Placebo-controlled, Crossover Study of the Effects of Single Doses of AUT00201 in Patients With Myoclonus Epilepsy and Ataxia Due to Potassium (K+) Channel Mutation (MEAK)

A randomized, double-blind, placebo-controlled, crossover study to assess the safety, tolerability, and pharmacokinetics of single doses of AUT00201 at 100 mg or matching placebo in patients with myoclonus epilepsy and ataxia due to potassium channel mutation (MEAK).

开始日期2023-05-12

申办/合作机构

Autifony Therapeutics Ltd.

NCT04158453 / Completed临床1期

A Randomised, Double-blind, Placebo-controlled, Single and Repeated Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AUT00201 in Healthy Male and Female Volunteers

The study medicine is a potential future treatment for schizophrenia, an illness that affects the way that people think, feel or behave. It is not clear what causes schizophrenia, but it's been linked to chemical imbalance in the brain. It is hoped that the study medicine will activate specific sites in the brain to help correct that imbalance. Current treatments for schizophrenia don't work very well and can cause unpleasant side effects. It is hoped that the study medicine will work better, and have fewer side effects than existing medicines.
In this 2 part study (Part A: up to 40 healthy male subjects and up to 8 healthy female subjects, Part B: up to 32 healthy male subjects) the primary aim is to assess how safe the study medicine is in healthy men and women.
This study will be in 2 parts, as follows:
Part A will assess single doses of AUT00201 and Part B will assess multiple doses. Part A will be divided into 3 sub-parts: Part A1 will assess single ascending doses in healthy men, Part A2 will assess single ascending doses in healthy women, and Part A3 will assess the effect of food on the PK of AUT00201 in healthy men.
A pharmaceutical company, Autifony Therapeutics Limited, is funding the study. The study will take place at 1 centre in London.

开始日期2019-10-10

申办/合作机构

Autifony Therapeutics Ltd.

100 项与 AUT-00201 相关的临床结果

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100 项与 AUT-00201 相关的转化医学

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100 项与 AUT-00201 相关的专利（医药）

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项与 AUT-00201 相关的文献（医药）

2013-03-01·European Journal of Pharmaceutical Sciences2区 · 医学

Chemical synthesis, docking studies and biological effects of a pan peroxisome proliferator-activated receptor agonist and cyclooxygenase inhibitor

2区 · 医学

Article

作者: Marcelo M. Rabello ; Francisco de Assis Rocha Neves ; Flávia D.T. Uchôa ; Ivan Rocha Pitta ; Angelica A. Amato ; Maria do Carmo A. Lima ; Isabel Daufenback Machado ; Marcelo Z. Hernandes ; Paul Webb ; Suely L. Galdino ; José Roberto Santin ; Flora Aparecida Milton ; Sandra H.P. Farsky

The compound (5Z)-5-[(5-bromo-1H-indol-3-yl)methylene]-3-(4-chlorobenzyl)-thiazolidine-2,4-dione (LYSO-7) was synthesised in order to obtain a new type of anti-inflammatory drug, designed with hybrid features to inhibit cyclooxygenase (COX) and also to activate peroxisome proliferator-activated receptor (PPAR). Results obtained from docking (in silico) studies corroborated with experimental data, showing the potential affinity between the studied ligand and targets. The specificity of LYSO-7 for COX-enzymes was detected by the inhibition of COX-1 and COX-2 activities by 30% and 20%, respectively. In transactivation reporter gene assays LYSO-07 showed a pan partial agonist effect on the three PPAR subtypes (PPARγ, PPARα and PPARβ/δ). The agonist action on PPARγ was also observed by a pharmacological approach, as the reduction in the Escherichia coli lipopolysaccharide (LPS)-induced interleukin 1 beta (IL-1β) secretion and nitric oxide (NO) production by mouse neutrophils was blocked by GW9962, a specific PPARγ antagonist. Additionally, the in vivo effect was measured by reduced carrageenan-induced neutrophil influx into the subcutaneous tissue of mice. Taken together, these data show that LYSO-7 displays a potent in vivo anti-inflammatory effect during the innate acute response, which is dependent on its associated COX inhibitory activities and PPAR activation.

1992-01-01·Journal of Medicinal Chemistry1区 · 医学

Syntheses of 5-HT2 antagonist activity of bicyclic 1,2,4-triazol-3(2H)-one and 1,3,5-triazine-2,4(3H)-dione derivatives

1区 · 医学

Article

作者: Usui, Hiroyuki ; Watanabe, Yoshifumi ; Morishima, Yoshiyuki ; Kanao, Munefumi ; Shibano, Toshiro ; Yasuoka, Megumi ; Yoshiwara, Hirotaka ; Kobayashi, Shozo ; Tanaka, Tsuyoshi

A series of bicyclic 1,2,4-triazol-3(2H)-one and 1,3,5-triazine-2,4(3H)-dione derivatives with a 4-[bis(4-fluoro-phenyl)methylene]piperidine or 4-(4-fluorobenzoyl)piperidine group has been prepared and tested for 5-HT2 and alpha 1 receptor antagonist activity. Among the compounds prepared, 2-[2-[4-[bis(4-fluorophenyl)methylene]-piperidin-1-yl]ethyl]- 5,6,7,8-tetrahydro-1,2,4-triazolo[4,3-a]pyridin-3(2H)-one (7b) had the most potent 5-HT2 antagonist activity, which was greater than ritanserin (2), while 7b did not show alpha 1 antagonist activity in vivo. The central 5-HT2 receptor antagonism was approximately 1/30 that of 2 when tested for the ability to block head twitches induced by 5-hydroxytryptophan. Compound 21b, 3-[2-[4-(4-fluorobenzoyl)piperidin-1-yl]ethyl]-6,7,8,9-tetrahydro- 2H- pyrido[1,2-a]-1,3,5-triazine-2,4(3H)-dione also displayed potent 5-HT2 antagonist activity. The compound had moderate alpha 1 receptor antagonism, and the potency inhibiting head twitches was about one-third that of ketanserin (1). These results indicate that 5,6,7,8-tetrahydro-1,2,4-triazolo[4,3-a]pyrimidin-3(2H)-one and 6,7,8,9-tetrahydro-2H-pyrido-[1,2-a]-1,3,5-triazine-2,4(3H)-dione ring systems are useful components of 5-HT2 antagonists.

项与 AUT-00201 相关的新闻（医药）

2023-06-18

·药明康德

完全缓解率达91%的CAR-T疗法；使月度发作率下降95%的基因编辑疗法... | 一周盘点

▎药明康德内容团队编辑本期看点1. 用于治疗遗传性血管水肿（HAE）的在研体内CRISPR基因编辑疗法NTLA-2002早期临床数据亮眼，单次给药后使所有患者的月度发作率平均下降了95%。2. Mustang Bio公司公布了其靶向CD20的自体CAR-T细胞疗法MB-106的两项积极早期临床试验数据，滤泡性淋巴瘤患者高剂量组患者的总缓解率（ORR）达100%，完全缓解（CR）率达91%。3. 用于治疗癫痫的再生细胞疗法NRTX-1001疗效亮眼，一次给药后两名患者的癫痫发作活动持续减少超过90%，其中1名在接受治疗7个月后没有再发生任何癫痫发作事件。4. Chinook Therapeutics公司公布其用于治疗IgA肾病的抗APRIL单克隆抗体zigakibart（BION-1301）的积极长期数据，用药100周时5名患者的平均蛋白尿减少了72%。药明康德内容团队整理NTLA-2002：公布1期临床试验数据Intellia Therapeutics公司公布了其在研体内CRISPR基因编辑疗法NTLA-2002的1/2期临床试验的最新中期分析结果。NTLA-2002旨在使用CRISPR/Cas9技术对激肽释放酶B1（KLKB1）基因进行编辑，该基因负责编码激肽释放酶前体蛋白。通过让KLKB1失活，NTLA-2002可永久性降低激肽释放酶活性，以达到治愈HAE的目的。NTLA-2002包含靶向致病基因的指导RNA（gRNA），和编码Cas9酶的mRNA，它们共同开展精准编辑。截至2023年2月17日的数据，在最近一次随访中，单次给药NTLA-2002使所有患者（n=10）的HAE月度发作率平均下降了95%；在16周的主要观察期后，所有血浆激肽（kallikrein）水平降低超过60%以上的患者（n=9）仍然完全没有发作，最长的无发作间隔期已达13.0个月，并且还在持续；在所有在使用NTLA-2002后停止长期HAE预防治疗的患者（n=6）中，自停止治疗后还没有报告过HAE发作。此外，NTLA-2002在所有剂量水平上都有良好的耐受性。MB-106：公布两项1/2期临床试验的数据Mustang Bio公司公布了其靶向CD20的自体CAR-T细胞疗法MB-106在两项1/2期临床试验中的数据。在针对华氏巨球蛋白血症（WM）的临床试验中，MB-106继续表现出良好的安全性和有效性，ORR达83%（5/6）。此外，MB-106的作用持久，2名达CR的患者中有1名在22个月时仍处于CR状态。该研究中的所有患者均对BTK抑制剂耐药，在接受MB-106治疗后，没有患者开始新的抗WM治疗。在针对复发或难治性B细胞非霍奇金淋巴瘤（NHL）和慢性淋巴细胞白血病（CLL）的临床试验中，滤泡性淋巴瘤队列的结果显示，所有患者的ORR为95%（19/20），80%的患者达到了CR；较高剂量水平组的ORR为100%，CR率为91%，其中包括一名曾接受CD19靶向CAR-T细胞治疗的患者。在这20名患者中，有10名的CR状态保持了一年以上。NRTX-1001：公布1/2期临床试验的新数据Neurona Therapeutics公司公布了其候选再生细胞疗法NRTX-1001的1/2期临床试验的新数据。NRTX-1001是一种来源于人类多能干细胞的再生神经细胞治疗候选药物，用于治疗癫痫。NRTX-1001包括分泌抑制性神经递质γ-氨基丁酸（GABA）的中间神经元。作为一次性给药疗法，该药物能够提供局部长期GABA信号通路抑制，以重新平衡和修复引起癫痫以及神经系统其他疾病过度兴奋的神经网络。截至目前，已有两位患者接受了治疗，其分别有7年和9年的癫痫发作史，在使用NRTX-1001之前的六个月里，平均每月有32次和14次发作。在接受NRTX-1001治疗后，两名患者的癫痫发作活动在治疗后12个月和7个月时分别持续减少＞95%和＞90%，其中1名患者在接受治疗7个月后没有再发生任何癫痫发作事件，并且在认知测试中表现出记忆力的改善。此外，两名受试者都没有经历过严重的不良事件。Zigakibart：公布1/2期临床试验的新数据Chinook Therapeutics公司公布了其抗APRIL单克隆抗体zigakibart（BION-1301）治疗IgA肾病的1/2期试验的积极数据。结果显示，zigakibart的耐受性良好。在所有患者中，接受zigakibart治疗后12周时，其平均蛋白尿减少20%，在治疗24周时减少39%，在治疗52周时减少67%。当治疗时间进一步延长后，zigakibart依然带来了持续的临床益处，在治疗76周时，7名患者的蛋白尿平均减少了67%，在治疗100周时，5名患者的平均蛋白尿减少了72%。LX2006：公布1/2期临床试验首个队列的数据LEXEO Therapeutics公司公布了其基于腺相关病毒（AAV）的基因治疗候选药物LX2006的1/2期临床试验中首个队列的数据。LX2006旨在通过递送功能性共济蛋白基因来促进共济蛋白的表达，并恢复心肌细胞中的线粒体功能，从而治疗弗里德赖希共济失调心肌病。此次公布的结果显示，LX2006具有良好的耐受性，未观察到意外事件或毒性。根据数据安全监测委员会的建议，研究人员已开始在第二个队列的患者中进行给药。WU-CART-007：公布1/2期临床试验数据Wugen公司公布了其同种异体现货型CAR-T细胞疗法WU-CART-007用于治疗复发/难治性T细胞急性淋巴细胞白血病（ALL）或淋巴细胞淋巴瘤（LBL）的全球1/2期临床试验数据。WU-CART-007靶向CD7，并能够防止CAR-T细胞自相残杀，降低发生移植物抗宿主病（GvHD）的风险。此次公布的结果显示，WU-CART-007在剂量水平（DL）2和DL3队列中的ORR达57%，并且耐受性良好，高级别的细胞因子释放综合征（CRS）和免疫相关不良事件的发生率较低。NX-2127、NX-5948：公布1期临床试验数据Nurix Therapeutics公司公布了旗下两款针对BTK的蛋白降解疗法在B细胞恶性肿瘤中的早期临床试验数据。结果显示，NX-2127治疗使患者的BTK和IKZF1出现预期内的快速且持续的降解。两名曾接受过4线治疗的弥漫性大B细胞淋巴瘤（DLBCL）患者在接受每日一次300 mg剂量的NX-2127后，1名患者在第8周时达到了CR，并维持到第24周，另一名患者在经历了疾病稳定后进展。此外，NX-2127的耐受性良好，安全性结果与先前披露的一致。NX-5948的1a期试验的临床生物标志物数据证明了该疗法可以使BTK快速、稳健和持续地降解，支持正在进行的B细胞恶性肿瘤研究。KT-333、KT-413：公布1a临床试验数据Kymera Therapeutics公布了旗下两款潜在“first-in-class”的蛋白降解小分子药物KT-333与KT-413的早期临床试验数据。KT-333可降解STAT3蛋白，此次公布的结果显示，KT-333在实体瘤以及周边和皮肤T细胞淋巴瘤患者中显示出剂量依赖性的STAT3降解，在外周血单核细胞（PBMC）中平均最大降解水平达88%，并有STAT3通路受抑制和外周血中炎症生物标记物下调的迹象。KT-413可降解IRAK4蛋白，此次公布的结果显示，KT-413在多种淋巴瘤患者中显示出剂量依赖性的IRAK4降解，在PBMC中的降解水平达70%。EO2463：公布1/2期临床试验的初步数据Enterome公司公布了其基于肿瘤肽的免疫疗法EO2463用于治疗惰性非霍奇金淋巴瘤的早期临床试验的积极数据。EO2463由4种模拟B细胞肿瘤相关抗原CD8、CD20、CD22和CD37（BAFF受体）中的特异性细胞毒性T细胞（CD268+T细胞）表位的肽以及辅助性的CD4肽组成，能够以高亲和力稳定地结合组织相容性复合体I（MHC I），从而激活抗肿瘤免疫反应。此次公布的数据显示，接受EO2463单药治疗的患者在6周后被观察到获得了有益的临床缓解；接受EO2463与来那度胺+利妥昔单抗联合治疗的可评估患者中，初步的CR率为50%。此外，EO2463作为单药治疗的耐受性良好，在联合治疗时也没有发现额外的安全信号。Barzolvolimab：公布1b期临床试验的新数据Celldex Therapeutics公司公布了其人源化的单克隆抗体barzolvolimab用于治疗抗组胺药难治的中到重度慢性自发性荨麻疹患者的1b期临床试验的新数据。Barzolvolimab能够高特异性地与维持肥大细胞功能和存活所必须的受体酪氨酸激酶KIT相结合，并能潜在地抑制其活性。此次公布的数据显示，血管性水肿患者有深度且持久的血管性水肿症状改善。单次3.0 mg/kg剂量的barzolvolimab耐受性良好，在抗组胺药难治的荨麻疹患者中表现出令人印象深刻的临床活性，CR率为56%，患者报告生活质量得到了临床意义上的显著改善。ALE.C04：IND申请获得FDA许可ALE.C04是Alentis Therapeutics公司开发的单克隆抗体，专门针对癌细胞上暴露的CLDN1而开发。这种研究性抗体旨在以两种方式治疗癌症：重塑细胞外基质，从而改善NK细胞和T细胞的通行，并通过效应器功能直接杀死肿瘤细胞。该候选疗法针对头颈部鳞状细胞癌的1/2期临床试验预计将于2023年下半年开展。Ziftomenib：公布1/2期临床试验的新数据Kura Oncology公司公布了其新型口服menin抑制剂ziftomenib用于治疗复发/难治性NPM1突变型急性髓系白血病（AML）的1/2期临床试验的新数据。Ziftomenib旨在靶向menin-KMT2A/MLL蛋白相互作用，此前已获得美国FDA授予的孤儿药资格，用于治疗AML。此次公布的结果显示，截至2023年4月12日的数据，接受600 mg剂量ziftomenib的20例NPM1突变型AML患者中有35%达到了CR，中位缓解持续时间为8.2个月。TP-3654：公布1/2期临床试验的初步数据Sumitomo Dainippon Pharma公司公布了其在研选择性口服PIM1激酶抑制剂TP-3654的早期临床数据。该疗法旨在治疗先前接受过JAK抑制剂治疗或不符合JAK抑制剂治疗的骨髓纤维化（MF）患者。TP-3654单药治疗复发/难治性MF患者的初步数据显示，治疗时间超过12周时，在10名可评估的患者中，7名患者的脾脏体积缩小，9名患者的总症状评分有改善。VRG50635：公布1期临床试验的初步数据Verge Genomics公司公布了其潜在“best-in-class”的PIKfyve小分子抑制剂用于治疗肌萎缩侧索硬化（ALS）的早期临床试验的初步数据。该公司基于人体组织和人类数据，通过AI赋能的技术平台CONVERGE发现内体/溶酶体功能的丢失是ALS的新致病机制，并发现PIKfyve是具有潜力的新治疗靶点。VRG50635是一款强力PIKfyve抑制剂，能够恢复ALS患者神经元的内体/溶酶体功能。临床前研究显示，VRG50635在ALS相关运动神经元退化模型中显示出效力。此次公布的结果显示，VRG50635在健康成人受试者中单次和重复给药后的安全性、耐受性和药代动力学特征均良好，支持推进到针对ALS患者的概念验证研究。LP-184：IND申请获得FDA许可LP-184是Lantern Pharma公司开发的新一代DNA损伤候选药物，旨在利用“合成致死”机制治疗多种晚期实体瘤以及脑和中枢神经系统癌症。在临床前研究中，LP-184可显著并快速缩小小鼠的胰腺癌异种移植瘤。在8周内，LP-184组的肿瘤与基线相比缩小了超过90%，而未接受治疗组肿瘤体积增长了11倍。近期，该疗法的IND申请已获得FDA的许可，将在今年夏天启动首次人体试验。大家都在看▲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，填写业务需求信息参考资料（可上下滑动查看）[1] NS Pharma Announces FDA Clearance to Initiate a Phase I/II Study for NS-050/NCNP-03, an Exon 50 Skipping Candidate for the Treatment of Duchenne Muscular Dystrophy. Retrieved June 16, 2023, from https://www.prnewswire.com/news-releases/ns-pharma-announces-fda-clearance-to-initiate-a-phase-iii-study-for-ns-050ncnp-03-an-exon-50-skipping-candidate-for-the-treatment-of-duchenne-muscular-dystrophy-301851575.html[2] Wugen Presents Initial Data from First-in-Human Phase 1/2 Trial of WU-CART-007 at the European Hematology Association (EHA) 2023 Congress. Retrieved June 16, 2023, from https://www.businesswire.com/news/home/20230609005019/en/[3] Kymera Therapeutics’ STAT3 Degrader KT-333 and IRAKIMiD Degrader KT-413 Demonstrate Desired Target Knockdown and Safety with Continued Dose Escalation in Ongoing Phase I Clinical Trials. Retrieved June 14, 2023 from https://investors.kymeratx.com/news-releases/news-release-details/kymera-therapeutics-stat3-degrader-kt-333-and-irakimid-degrader[4] Sumitomo Pharma Oncology, Inc. Presents Updated Preliminary Data from Phase 1/2 Clinical Study Evaluating Investigational Agent TP-3654 in Patients with Myelofibrosis at European Hematology Association 2023 Hybrid Congress. Retrieved June 14, 2023 from https://www.prnewswire.com/news-releases/sumitomo-pharma-oncology-inc-presents-updated-preliminary-data-from-phase-12-clinical-study-evaluating-investigational-agent-tp-3654-in-patients-with-myelofibrosis-at-european-hematology-association-2023-hybrid-congress-301847453.html[5] Intellia Therapeutics Announces New Positive Clinical Data from Phase 1 Study of NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema (HAE) . Retrieved June 14, 2023 from https://www.globenewswire.com/news-release/2023/06/11/2685902/0/en/Intellia-Therapeutics-Announces-New-Positive-Clinical-Data-from-Phase-1-Study-of-NTLA-2002-an-Investigational-In-Vivo-CRISPR-Genome-Editing-Treatment-for-Hereditary-Angioedema-HAE.html[6] Lantern Pharma Receives FDA Clearance of IND Application for Drug Candidate LP-184 in Solid Tumors. Retrieved June 14, 2023 from https://www.businesswire.com/news/home/20230612954913/en[7] MARKER THERAPEUTICS ANNOUNCES FIRST LYMPHOMA PATIENT TREATED WITH MT-601 IN PHASE 1 CLINICAL TRIAL. Retrieved June 14, 2023 from https://ir.markertherapeutics.com/news-releases/news-release-details/marker-therapeutics-announces-first-lymphoma-patient-treated-mt[8] Mustang Bio Announces Updated Results from Waldenstrom Macroglobulinemia Cohort of Ongoing Phase 1/2 Clinical Trial of MB-106, CD20-Targeted Autologous CAR T Therapy. Retrieved June 14, 2023 from https://ir.mustangbio.com/news-events/press-releases/detail/160/mustang-bio-announces-updated-results-from-waldenstrom[9] Mustang Bio Announces Final Results from Follicular Lymphoma Cohort of Single-Institution Phase 1/2 Clinical Trial of MB-106, CD20-Targeted Autologous CAR T Therapy. Retrieved June 16, 2023 from https://www.globenewswire.com/news-release/2023/06/15/2689364/0/en/Mustang-Bio-Announces-Final-Results-from-Follicular-Lymphoma-Cohort-of-Single-Institution-Phase-1-2-Clinical-Trial-of-MB-106-CD20-Targeted-Autologous-CAR-T-Therapy.html[10] CHINOOK THERAPEUTICS TO PRESENT UPDATED DATA FROM ZIGAKIBART (BION-1301) PHASE 1/2 TRIAL IN PATIENTS WITH IGA NEPHROPATHY (IGAN) AT THE 60TH EUROPEAN RENAL ASSOCIATION (ERA) CONGRESS. Retrieved June 16, 2023 from https://investors.chinooktx.com/news-releases/news-release-details/chinook-therapeutics-present-updated-data-zigakibart-bion-1301[11] KURA ONCOLOGY PRESENTS LATE-BREAKING CLINICAL DATA FOR MENIN INHIBITOR ZIFTOMENIB AT 2023 EUROPEAN HEMATOLOGY ASSOCIATION (EHA) CONGRESS Retrieved June 16, 2023 from https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-presents-late-breaking-clinical-data-menin[12] Celldex Therapeutics Presents Positive Data from Barzolvolimab Chronic Urticaria Program at EAACI 2023. Retrieved June 16, 2023 from https://ir.celldex.com/news-releases/news-release-details/celldex-therapeutics-presents-positive-data-barzolvolimab[13] Can-Fite to Submit IND for the Treatment of Pancreatic Cancer Patients with Namodenoson. Retrieved June 17, 2023 from https://www.businesswire.com/news/home/20230613143878/en[14] Ventyx Biosciences Announces Initiation of Dosing in a Phase 1 Trial of VTX3232, a Novel CNS-Penetrant NLRP3 Inhibitor. Retrieved June 17, 2023 from https://www.globenewswire.com/news-release/2023/06/14/2687919/0/en/Ventyx-Biosciences-Announces-Initiation-of-Dosing-in-a-Phase-1-Trial-of-VTX3232-a-Novel-CNS-Penetrant-NLRP3-Inhibitor.html[15] Nurix Therapeutics Presents Data from Studies of Its Targeted Protein Degraders in B Cell Malignancies and Initiates Expansion of NX-2127 Phase 1b Trial in Diffuse Large B Cell Lymphoma and Mantle Cell Lymphoma Indications. Retrieved June 17, 2023 from https://www.globenewswire.com/news-release/2023/06/14/2687921/0/en/Nurix-Therapeutics-Presents-Data-from-Studies-of-Its-Targeted-Protein-Degraders-in-B-Cell-Malignancies-and-Initiates-Expansion-of-NX-2127-Phase-1b-Trial-in-Diffuse-Large-B-Cell-Lym.html[16] Alentis Therapeutics Receives IND Clearance from FDA for ALE.C04 for the Treatment of CLDN1+ Tumors. Retrieved June 17, 2023 from https://www.businesswire.com/news/home/20230614534402/en[17] Enterome’s new OncoMimics™ immunotherapy, EO2463, demonstrates early efficacy and favorable safety in Phase 1/2 trial for indolent non-Hodgkin lymphoma. Retrieved June 17, 2023 from https://www.enterome.com/news-events/enteromes-new-oncomimics-immunotherapy-eo2463-demonstrates-early-efficacy-and-favorable-safety-in-phase-1-2-trial-for-indolent-non-hodgkin-lymphoma/[18] EyeBio Announces First Patients Dosed in Phase 1b/2 Trial of Restoret™ in Neovascular Age Related Macular Degeneration and Diabetic Macular Edema. Retrieved June 17, 2023 from https://www.businesswire.com/news/home/20230614951729/en[19] Autifony Therapeutics announces commencement of a US Phase Ib study of AUT00201, a novel Kv3 modulator, in Progressive Myoclonic Epilepsy-7 (EPM7) . Retrieved June 17, 2023 from http://www.businesswire.com/news/home/20230613182220/en[20] LEXEO THERAPEUTICS ANNOUNCES COMPLETION OF FIRST COHORT AND DOSING IN SECOND COHORT IN SUNRISE-FA, A PHASE 1/2 CLINICAL TRIAL OF LX2006 FOR THE TREATMENT OF FRIEDREICH’S ATAXIA CARDIOMYOPATHY. Retrieved June 17, 2023 from https://www.lexeotx.com/post/lexeo-therapeutics-announces-completion-of-first-cohort-and-dosing-in-second-cohort-in-sunrise-fa-a-phase-1-2-clinical-trial-of-lx2006-for-the-treatment-of-friedreichs-ataxia-cardiomyopathy/[21] Verge Genomics Announces Positive Safety and Tolerability Data from the Phase 1 Clinical Trial of VRG50635, a Potential Best-in-Class Therapeutic for All Forms of ALS. Retrieved June 17, 2023 from https://www.businesswire.com/news/home/20230615018038/en[22] Neurona Therapeutics Presents One-Year Data on the First Patient Treated with NRTX-1001 Cell Therapy in an Ongoing Phase I/II Trial for Drug-resistant Focal Epilepsy. Retrieved June 17, 2023 from https://www.globenewswire.com/news-release/2023/06/15/2689142/0/en/Neurona-Therapeutics-Presents-One-Year-Data-on-the-First-Patient-Treated-with-NRTX-1001-Cell-Therapy-in-an-Ongoing-Phase-I-II-Trial-for-Drug-resistant-Focal-Epilepsy.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

细胞疗法基因疗法免疫疗法临床结果临床1期

2023-06-13

·Business Wire

Autifony Therapeutics announces commencement of a US Phase Ib study of AUT00201, a novel Kv3 modulator, in Progressive Myoclonic Epilepsy-7 (EPM7)

STEVENAGE, England--( BUSINESS WIRE )--Autifony Therapeutics Limited (“Autifony”), which is pioneering the development of novel pharmaceutical treatments for rare CNS disorders and other serious brain diseases, today announced that a Phase Ib trial of AUT00201, a potent and selective modulator of Kv3 ion channels, is enrolling patients with Myoclonus Epilepsy and Ataxia due to Potassium (K+) Channel Mutation or MEAK (also known as EPM7) at the Hospital of the University of Pennsylvania. Epilepsy is a brain disorder that causes recurring, unprovoked seizures. It is the fourth most common neurological disorder worldwide. Patients with EPM7 have a very rare form of epilepsy, caused by reduced function of Kv3.1 ion channels, which gives rise to myoclonic seizures (brief shock-like jerks of a muscle or a group of muscles), action-induced myoclonus and a progressive ataxia (impairing co-ordination, balance and speech). EPM7 symptoms typically emerge between the ages of 3 and 15, with progressively severe myoclonus, ataxia, and occasional tonic-clonic seizures. Patients are typically wheel-chair bound by the time they are in their late teens. Current anti-seizure drugs can help to reduce the incidence of tonic-clonic seizures, but these drugs do not improve the myoclonus or ataxia. AUT00201 is a potent positive modulator of Kv3.1 ion channels. In a genetic mouse model of EPM7 conducted by Dr. Ethan Goldberg at the Children’s Hospital of Philadelphia, AUT00201 completely reversed the seizure and ataxia phenotype. The Phase Ib study in patients with EPM7 is a randomized, double-blind, placebo-controlled, crossover study in 8-10 patients aged 18 or over in which effects both on clinical symptoms and biomarkers related to dysfunction of Kv3.1 channels are evaluated. Separately, the US FDA has designated AUT00201 for treatment of EPM7 as a drug for a ‘rare pediatric disease’ as defined in section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act. The Office of Orphan Products Development has also granted orphan drug designation for AUT00201 for the treatment of EPM7. Dr. Charles Large, Chief Executive Officer of Autifony Therapeutics, commented: “This is a unique opportunity for precision medicine, in which we aim to reverse the impact of a genetic mutation that causes a highly debilitating disorder. We are hopeful that this will offer benefit to patients with EPM7 where there are currently no effective treatments, and might one day also bring benefits to patients suffering from other forms of myoclonic epilepsy, which we will be looking to investigate in future.” Notes to Editors About Autifony Therapeutics Ltd Autifony Therapeutics is an independent UK-based biotechnology company formed in 2011 as a spin-out from GSK, which retains equity in the company. The Company is focused on the development of high value, novel medicines to treat serious diseases of the central nervous system. It is funded by SV Health Investors, IP Group, Pfizer Ventures, International Biotechnology Trust PLC, and UCL Business plc. For more information, please visit www.autifony.com . About AUT00201 AUT00201 is a novel, orally active small molecule designed to selectively modulate Kv3 potassium channels. Safety and toxicology studies support the clinical development of AUT00201.

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适应症	最高研发状态	国家/地区	公司	日期
共济失调	临床1期	美国	Autifony Therapeutics Ltd.	2023-05-12
进行性肌阵挛性癫痫	临床1期	美国	Autifony Therapeutics Ltd.	2023-05-12
精神分裂症	临床1期	英国	Autifony Therapeutics Ltd.	2019-10-10