预约演示
更新于：2026-04-07
Votoplam
更新于：2026-04-07
概要
基本信息
药物类型 小分子化药 |
别名 HTT227、PTC 518、PTC-518 |
作用方式 调节剂 |
作用机制 HTT基因调节剂、splicing factor调节剂(剪接因子调节剂) |
在研适应症 |
非在研适应症- |
非在研机构- |
最高研发阶段临床3期 |
首次获批日期- |
最高研发阶段(中国)临床申请批准 |
特殊审评孤儿药 (美国)、快速通道 (美国) |
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结构/序列
分子式C21H25N9O |
InChIKeyICZNVPJUHBURBG-UHFFFAOYSA-N |
CAS号2407849-89-0 |
关联
5
项与 Votoplam 相关的临床试验NCT07326709
A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
NCT06254482
A Phase 2b, Double-Blind, Randomized Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease
NCT05358717
A Phase 2A, Randomized, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of PTC518 in Subjects With Huntington's Disease
100 项与 Votoplam 相关的临床结果
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100 项与 Votoplam 相关的转化医学
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100 项与 Votoplam 相关的专利（医药）
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5
项与 Votoplam 相关的文献（医药）2026-03-01Clinical Pharmacology in Drug Development
Effects of High‐ and Low‐Fat Meals on the Bioavailability and Pharmacokinetics of Votoplam, a HTT Gene Splicing Modifier
Article
作者: Golden, Lee ; Lee, Lucy ; Kong, Ronald ; Beers, Brian ; Reddy, Nageswara ; Richards, Amy‐Lee
Abstract:
Votoplam is a novel, orally bioavailable, small molecule HTT gene splicing modifier that is being developed for the treatment of Huntington's disease. This was a single dose, open‐label, two‐period, crossover food effect study that evaluated the effect of high‐ and low‐fat meals on 20 mg votoplam in healthy participants. There was a washout of 21 days between the two periods. Twenty‐six participants completed the study. There were minimal changes in the bioavailability and pharmacokinetics of votoplam following administration of a single dose of 20 mg votoplam when taken with low‐fat and high‐fat meals relative to fasted condition. The mean C
max
, AUC
0–last
, and AUC
0–inf
for votoplam following administration of a single dose of 20 mg votoplam were 1.4‐fold, 1.2‐fold, and 1.1‐fold higher, respectively, in high‐fat fed conditions, and were 1.3‐fold, 1.1‐fold, and 1.1‐fold higher, respectively, in the low‐fat fed conditions, when compared to fasted conditions. There were no relevant safety findings in any of the treatment groups. Votoplam can be administered with or without food in patients.
2025-03-01SYNLETT
Synthesis, Characterization, and Biological Applications of Tetramethylpiperidinyl Triazolopyridazine Derivatives
作者: Bharathkumar, Hanumantharayappa ; Gundekari, Sreedhar ; Keesara, Srinivasa Reddy ; Varkolu, Mohan ; Bolla, Ratnasekhar ; Andugulapati, Sai Balaji ; Gundekari, Sreedhar
Abstract:
We have synthesized a series of tetramethylpiperidinyl triazolopyridazine derivatives and screened the molecules for their biological activity against a cancer (NCI-H460) cell line. Among the tested molecules, 2-[3-(2,2,6,6-tetramethylpiperidin-4-yl)-3H-[1,2,3]triazolo[4,5-c]pyridazin-6-yl]-5-(2H-1,2,3-triazol-2-yl)phenol significantly inhibited cell growth with an IC50 value of 5.2 μM. We hope that this study will help in the development of better candidates for the treatment of lung cancer.
2024-11-01Journal of Huntingtons Disease
Huntington’s Disease Clinical Trials Update: September 2024
Article
作者: Farag, Mena ; Wild, Edward J ; Tabrizi, Sarah J
In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing extension study of PTC518 from PTC Therapeutics, including 12-month interim results from the parent study. We also discuss 24-month interim results from uniQure's AMT-130 program and 28-week follow-up results from Wave Life Sciences’ SELECT-HD clinical trial of WVE-003. Additionally, we provide a comprehensive listing of all currently registered and ongoing clinical trials in Huntington's disease.
100 项与 Votoplam 相关的药物交易
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研发状态
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 亨廷顿舞蹈病 | 临床3期 | 美国 | 2026-03-24 | |
| 亨廷顿舞蹈病 | 临床3期 | 加拿大 | 2026-03-24 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
临床2期 | 159 | Placebo (Placebo) | 鹹夢淵蓋遞選齋廠齋膚 = 鹹淵鹹壓遞築網糧廠製 窪鹹蓋膚鹹網鹽蓋鹽壓 (構壓襯淵鑰願遞積顧蓋, 壓淵憲窪膚憲獵艱獵壓 ~ 顧願遞繭製製蓋構蓋窪) 更多 | - | 2026-01-13 | ||
(PTC518 5 mg) | 鹹夢淵蓋遞選齋廠齋膚 = 鏇鹹壓廠淵觸夢鏇窪醖 窪鹹蓋膚鹹網鹽蓋鹽壓 (構壓襯淵鑰願遞積顧蓋, 鹽醖鏇夢構窪範餘衊鏇 ~ 壓築蓋壓艱艱淵鹽鏇夢) 更多 | ||||||
临床2期 | 159 | (Stage 2 Huntington's disease) | 鏇壓積鏇願獵糧遞積積(觸積鑰夢獵範鹹網窪積) = 鬱構醖窪醖襯顧醖窪獵 範淵鹹鑰製觸壓積範築 (夢選積鏇壓鏇遞壓簾餘, -34.4 ~ -12.4) 达到 更多 | 积极 | 2025-05-05 | ||
(Stage 2 Huntington's disease) | 鏇壓積鏇願獵糧遞積積(觸積鑰夢獵範鹹網窪積) = 遞膚繭襯窪範築衊齋壓 範淵鹹鑰製觸壓積範築 (夢選積鏇壓鏇遞壓簾餘, -49.4 ~ -28.7) 达到 更多 | ||||||
Pubmed 人工标引  | 临床1期 | - | 77 | 鏇繭齋壓鹹蓋鹹襯選憲(淵獵鬱窪範餘製齋糧餘) = 壓糧鏇願艱願廠範網鬱 壓齋淵鏇鹹鹽鹽糧鹽積 (襯憲餘積餘鏇膚憲齋醖 ) 更多 | 积极 | 2024-08-18 | |
临床2期 | - | 淵淵繭醖齋齋鹹觸觸遞(願願膚積網網淵鏇遞齋) = 齋觸鑰網壓醖醖築範築 壓夢鑰鬱選顧鏇壓壓衊 (窪襯膚製鹽構遞鹹糧積 ) 更多 | 积极 | 2024-06-20 | |||
淵淵繭醖齋齋鹹觸觸遞(願願膚積網網淵鏇遞齋) = 膚鏇齋膚選糧衊衊鬱鑰 壓夢鑰鬱選顧鏇壓壓衊 (窪襯膚製鹽構遞鹹糧積 ) 更多 |
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