本期看点
1. Assembly Biosciences公司公布了用于治疗复发性生殖器疱疹的抗病毒疗法ABI-5366的积极早期临床研究数据，每周给药一次，患者的生殖器病变率下降94%。
2. 治疗帕金森病的变构小分子疗法GT-02287的早期临床结果积极，首批9名受试者已观察到运动障碍协会-统一帕金森病评定量表（MDS-UPDRS）的改善。
ABI-5366：公布1b期临床试验的中期数据
Assembly Biosciences公司公布了一种用于治疗复发性生殖器疱疹的长效解旋酶-引物酶抑制剂ABI-5366的1b期临床试验数据。该候选疗法通过靶向病毒解旋酶-引物酶复合体发挥作用。这是一种在HSV-1和HSV-2中都保守的重要病毒酶复合体，并且宿主细胞中没有类似的酶复合体。在短期临床研究中，HSV解旋酶-引物酶抑制剂显示出比核苷类似物更佳的疗效潜力。
此次公布的结果显示，在接受每周一次350 mg ABI-5366口服剂量的队列中，与安慰剂组相比，患者的HSV-2病毒脱落率、高病毒载量脱落率和生殖器病变率均出现具有统计学意义的显著下降，降幅分别达到94%、98%和94%。药代动力学特征支持ABI-5366采用每周甚至有望每月一次的给药方案。
GT-02287：公布1b期临床试验的初步数据
Gain Therapeutics公布了其用于治疗帕金森病的在研变构小分子GT-02287的1b期试验的初步数据。GT-02287是一种口服变构蛋白调节剂，具有可穿越血脑屏障的能力。该药物可恢复溶酶体蛋白酶葡萄糖脑苷脂酶（GCase）的功能，该酶由于GBA1基因突变（与帕金森病相关的最常见遗传异常）或其他与年龄相关的压力因素而发生错误折叠和受损。
在帕金森病的临床前模型中，GT-02287恢复了GCase酶功能，减少了聚集的α突触核蛋白、神经炎症和神经元死亡，提高了多巴胺水平，改善动物的运动功能和认知能力，可能具有减缓帕金森病进展的潜力。此外，GT-02287显著降低了血浆神经丝轻链（NfL）水平，这是一种神经退行性疾病的生物标志物。
此次公布的结果显示，GT-02287总体耐受性良好，未发生任何治疗伴发严重不良事件。首批9名受试者已观察到MDS-UPDRS的改善，其余受试者的90天随访仍在进行中。患者的血浆药物暴露水平处于预期的治疗范围内，且与此前1期健康受试者研究中的暴露水平相当。
Zeleciment basivarsen（DYNE-101）：公布1/2期临床试验的新数据
Dyne Therapeutics公司公布了在研抗体偶联反义寡核苷酸疗法zeleciment basivarsen，在治疗强直性肌营养不良1型（DM1）患者的1/2期临床试验ACHIEVE中的新数据。DYNE-101由与抗体片段（Fab）偶联的反义寡核苷酸组成，Fab与肌肉上高表达的转铁蛋白受体1（TfR1）结合。它旨在实现靶向肌肉组织递送，目的是减少细胞核中的有毒DMPK基因的RNA，允许正常mRNA处理和正常蛋白翻译，并有可能阻止或逆转疾病进展。DYNE-101已被欧洲药品管理局（EMA）和美国FDA授予孤儿药资格，用于治疗DM1。
此次公布的结果显示，zeleciment basivarsen为患者提供了广泛的功能改善。通过视频手部张开时间（vHOT）测试显示，患者手部肌强直相对于基线有显著且持续的改善。在多个功能终点上观察到相对于基线有意义且持续的改善，包括10米步行/跑步测试（10MWR）和5次坐站测试（5xSTS）的结果，以及用于衡量上肢功能的9孔钉测试（9HPT）结果的改善。定量肌力测试（QMT）显示上下肢多个肌群肌力均得到改善，测试项目包括握力、屈肘、伸肘、踝背屈、屈膝、伸膝。患者报告的强直性营养不良健康指数（MDHI）结果显示在认知障碍、睡眠障碍、疲劳、沟通、情绪问题、疼痛及活动能力等多个指标上，相对于基线有有意义且持续的改善。此外，患者和临床医生对整体功能的印象相对于基线有所改善。安全性方面，该药物安全性良好，未发现严重治疗伴发不良事件。
参考资料：
[1] Ovid announces positive topline results for the next-generation GABA-aminotransferase inhibitor, OV329, that demonstrate strong inhibitory activity and a potential best-in-category safety profile. Retrieved October 10, 2025, from https://investors.ovidrx.com/news/news-details/2025/Ovid-announces-positive-topline-results-for-the-next-generation-GABA-aminotransferase-inhibitor-OV329-that-demonstrate-strong-inhibitory-activity-and-a-potential-best-in-category-safety-profile/default.aspx
[2] Taysha Gene Therapies Presents New Supplemental Data Analysis from Part A of the REVEAL Phase 1/2 Trials for TSHA-102 in Rett Syndrome at the 54th CNS Annual Meeting. Retrieved October 10, 2025, from https://www.globenewswire.com/news-release/2025/10/09/3164079/0/en/Taysha-Gene-Therapies-Presents-New-Supplemental-Data-Analysis-from-Part-A-of-the-REVEAL-Phase-1-2-Trials-for-TSHA-102-in-Rett-Syndrome-at-the-54th-CNS-Annual-Meeting.html
[3] Complement Therapeutics Receives FDA IND Clearance to Advance CTx001 into Opti-GAIN, a Phase I/II Clinical Trial in Geographic Atrophy secondary to AMD. Retrieved October 10, 2025, from https://www.prnewswire.com/news-releases/complement-therapeutics-receives-fda-ind-clearance-to-advance-ctx001-into-opti-gain-a-phase-iii-clinical-trial-in-geographic-atrophy-secondary-to-amd-302577045.html
[4] Arvinas Presents Late Breaking, Positive Phase 1 Clinical Data for ARV-102, a PROTAC LRRK2 Degrader, at the 2025 International Congress of Parkinson’s Disease and Movement Disorders®. Retrieved October 10, 2025, from https://www.globenewswire.com/news-release/2025/10/05/3161349/0/en/Arvinas-Presents-Late-Breaking-Positive-Phase-1-Clinical-Data-for-ARV-102-a-PROTAC-LRRK2-Degrader-at-the-2025-International-Congress-of-Parkinson-s-Disease-and-Movement-Disorders.html
[5] Herantis Pharma presents positive topline data for HER-096 in Phase 1b trial for people living with Parkinson’s disease. Retrieved October 10, 2025, from https://www.globenewswire.com/news-release/2025/10/07/3162855/0/en/Herantis-Pharma-presents-positive-topline-data-for-HER-096-in-Phase-1b-trial-for-people-living-with-Parkinson-s-disease.html
[6] Adicet Bio Announces Positive Preliminary Data from ADI-001 Phase 1 Study in Patients with Lupus Nephritis (LN) and Systemic Lupus Erythematosus (SLE). Retrieved October 10, 2025 from https://www.businesswire.com/news/home/20251006091236/en/Adicet-Bio-Announces-Positive-Preliminary-Data-from-ADI-001-Phase-1-Study-in-Patients-with-Lupus-Nephritis-LN-and-Systemic-Lupus-Erythematosus-SLE
[7] BlueRock Therapeutics reports positive 36-month results from Phase I trial of bemdaneprocel for treating Parkinson’s disease. Retrieved October 10, 2025 from https://www.bluerocktx.com/bluerock-therapeutics-reports-positive-36-month-results-from-phase-i-trial-of-bemdaneprocel-for-treating-parkinsons-disease/
[8] Gain Therapeutics Presents Initial Data from Phase 1b Clinical Study of GT-02287 in Parkinson’s Patients at International Congress of Parkinson’s Disease and Movement Disorders®. Retrieved October 10, 2025 from https://gaintherapeutics.com/newsroom/press-releases/press-releases-2025/gain-therapeutics-presents-initial-data-from-phase-1b-clinical-study-of-gt-02287-in-parkinsons-patients-at-international-congress-of-parkinsons-disease-and-movement-disorders/
[9] Spur Therapeutics Presents Updated Phase 1/2 Data on Its Gene Therapy Candidate in Gaucher Disease at ESGCT 32nd Annual Congress. Retrieved October 10, 2025 from https://www.globenewswire.com/news-release/2025/10/07/3162226/0/en/Spur-Therapeutics-Presents-Updated-Phase-1-2-Data-on-Its-Gene-Therapy-Candidate-in-Gaucher-Disease-at-ESGCT-32nd-Annual-Congress.html
[10] Dyne Therapeutics Announces Additional One-Year Clinical Data Demonstrating Functional Improvement from Phase 1/2 ACHIEVE Trial of Zeleciment Basivarsen (DYNE-101) for Myotonic Dystrophy Type 1 (DM1). Retrieved October 10, 2025 from https://www.globenewswire.com/news-release/2025/10/06/3162171/0/en/Dyne-Therapeutics-Announces-Additional-One-Year-Clinical-Data-Demonstrating-Functional-Improvement-from-Phase-1-2-ACHIEVE-Trial-of-Zeleciment-Basivarsen-DYNE-101-for-Myotonic-Dystr.html
[11] Cabaletta Bio Presents First Rese-cel Data with No Preconditioning Demonstrating Biologic Activity and Early Clinical Responses at the 2025 ESGCT Annual Congress. Retrieved October 10, 2025 from https://www.cabalettabio.com/investors/news-events/press-releases/detail/135/cabaletta-bio-presents-first-rese-cel-data-with-no
[12] AskBio Presents Interim Safety Results from Phase 1/Phase 2 LION-CS101 Clinical Trial of AB-1003 in Participants with Limb-Girdle Muscular Dystrophy 2I/R9. Retrieved October 10, 2025 from https://www.globenewswire.com/news-release/2025/10/10/3164826/0/en/AskBio-Presents-Interim-Safety-Results-from-Phase-1-Phase-2-LION-CS101-Clinical-Trial-of-AB-1003-in-Participants-with-Limb-Girdle-Muscular-Dystrophy-2I-R9.html
[13] Assembly Biosciences Presents Interim Phase 1b Data for HSV Helicase-Primase Inhibitor Candidate ABI-5366 at the 38th Congress of the International Union Against Sexually Transmitted Infections (IUSTI)- Europe. Retrieved October 10, 2025 from https://www.globenewswire.com/news-release/2025/10/10/3164831/16259/en/Assembly-Biosciences-Presents-Interim-Phase-1b-Data-for-HSV-Helicase-Primase-Inhibitor-Candidate-ABI-5366-at-the-38th-Congress-of-the-International-Union-Against-Sexually-Transmitt.html
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