Alnylam's Closely Watched Phase 3 Heart Disease Trial Of Vutrisiran Hits Primary Endpoint, Shares Surge
Alnylam's Closely Watched Phase 3 Heart Disease Trial Of Vutrisiran Hits Primary Endpoint, Shares Surge
Monday, Alnylam Pharmaceuticals Inc (NASDAQ:ALNY) revealed topline results from its HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for ATTR amyloidosis with cardiomyopathy (ATTR-CM).
週一，Alnylam Pharmaceuticals Inc（NASDAQ:ALNY）宣佈了其vutrisiran的HELIOS-B 3期研究的頂線結果，該藥正在開發用於帶有心肌病（ATTR-CM）的ATTR澱粉樣變性的RNAi治療。
ATTR-CM is a type of heart muscle disease that occurs when amyloid fibrils – clumps of misfolded proteins – are deposited into heart tissue and cause it to stiffen.
ATTR-CM是一種心肌疾病，當澱粉樣纖維（錯誤摺疊蛋白的堆積物）沉積到心臟組織中並導致其變硬時，會出現該疾病。
Also Read: Analysts Express Confidence in Alnylam's Hypertension Drug Despite Mixed Trial Data.
還閱讀：儘管臨床試驗數據混合，分析師們對Alnylam的高血壓藥表達信心。
The study met the primary endpoint, demonstrating a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular (CV) events during the double-blind period in both the overall population (HR 0.718, p-value 0.0118; n=654) and in the monotherapy population (patients not receiving tafamidis at baseline; HR 0.672, p-value 0.0162; n=395).
該研究在雙盲期內，在總體人口（HR 0.718，P值0.0118；n = 654）和單一治療人群（在基線未接受tafamidis的患者；HR 0.672，P值0.0162；n = 395）中展示了減少全因死亡率和複發性心血管（CV）事件複合的主要終點的統計顯着性。
The study also demonstrated statistically significant improvements across all secondary endpoints in both the overall and monotherapy populations.
該研究還展示了在總體和單一治療人群中所有次要終點的統計顯着性改善。
This includes key measures of disease progression: 6-minute walk test (6-MWT), Kansas City Cardiomyopathy Questionnaire (KCCQ), and New York Heart Association (NYHA) Class at Month 30 (p<0.025 for all).
包括疾病進展的關鍵指標：30個月時的6分鐘步行測試（6-MWT），堪薩斯城心肌病問卷（KCCQ）和紐約心臟協會（NYHA）分級（對所有p值小於0.025）。
Vutrisiran treatment also reduced all-cause mortality in the overall population (HR 0.645, p<0.025) and the monotherapy population (HR 0.655, p<0.05) up to Month 42.
Vutrisiran治療還在總體人群中（HR 0.645，p值小於0.025）和單一治療人群中（HR 0.655，p值小於0.05）減少了全因死亡率，直至42個月。
In addition, vutrisiran demonstrated consistent effects on the primary composite endpoint and all secondary endpoints across all key subgroups, including baseline tafamidis use, ATTR disease type, and measures of disease severity.
此外，vutrisiran在所有關鍵亞組中，包括基線tafamidis使用、ATTR疾病類型和疾病嚴重程度指標上，對主要複合終點和所有次要終點顯示了一致的效果。
In the HELIOS-B study, vutrisiran demonstrated encouraging safety and tolerability, consistent with its established profile. Rates of adverse events (AEs), serious AEs, and AEs leading to study drug discontinuation were similar between the vutrisiran and placebo arms.
在HELIOS-B研究中，vutrisiran表現出令人鼓舞的安全性和耐受性，與其已知的藥物特性一致。研究藥物不良事件（AEs）、嚴重AEs和引起研究藥物停用的AE發生率在vutrisiran和安慰劑組之間相似。
The company plans to proceed with global regulatory submissions starting later this year, including filing a supplemental New Drug Application with the FDA using a Priority Review Voucher.
該公司計劃從今年晚些時候開始進行全球監管提交，包括使用優先審查憑證向FDA提交補充新藥申請。
Price Action: ALNY shares are up 29.2% at $214.15 at last check Monday.
截至上週一，ALNY股票價格漲幅達29.2%，報214.15美元/股。
Illustration of Phrama lab worker created with MidJourney.
藥廠實驗室工作者插圖，由MidJourney創建。