Denali Therapeutics' Amyotrophic Lateral Sclerosis Misses Pivotal Trial, But Analyst Says It Was Expected
Denali Therapeutics' Amyotrophic Lateral Sclerosis Misses Pivotal Trial, But Analyst Says It Was Expected
On Monday, Denali Therapeutics Inc. (NASDAQ:DNLI) revealed topline results from an analysis of Regimen G of the Phase 2/3 HEALEY ALS Platform Trial evaluating DNL343 for amyotrophic lateral sclerosis.
週一，Denali Therapeutics Inc.（納斯達克：DNLI）公佈了對DNL343在肌萎縮側索硬化症的2/3期HEALEY ALS平台試驗G方案分析的初步結果。
Compared to placebo, the study did not meet the primary endpoint of efficacy in slowing disease progression.
與安慰劑相比，該研究未能達到減緩疾病進展的主要療效終點。
The primary endpoint was evaluated as a change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) and survival through week 24.
主要終點是評估疾病嚴重程度隨時間變化的情況，測量工具爲ALS功能評分量表修訂版（ALSFRS-R）以及生存率通過24周。
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Key secondary endpoints, measuring muscle strength and respiratory function, were also not statistically different between the active and placebo groups at week 24.
關鍵的次要終點，測量肌肉力量和呼吸功能，在第24周時也在活性組和安慰劑組之間沒有統計學差異。
For the primary analysis, 186 participants who were randomized to receive DNL343 treatment were compared to 139 participants randomized to receive placebo in this regimen (n=63) or shared from a concurrently enrolling regimen (n=76).
在主要分析中，186名隨機接受DNL343治療的參與者與139名隨機接受安慰劑的參與者進行了比較，後者的方案爲(n=63)或來自同時入組方案的共享(n=76)。
Overall, DNL343 was found to be safe and well-tolerated. Further analyses are anticipated later in 2025, including neurofilament light (NfL) and other fluid biomarkers, data from pre-specified subgroups, and extended findings from the active treatment extension period.
總體而言，DNL343被發現是安全且耐受良好的。預計在2025年晚些時候進行進一步分析，包括神經絲輕鏈（NfL）和其他液體生物標誌物、預先指定的亞組數據以及活性治療擴展期的擴展發現。
William Blair noted that while the update is disappointing for patients and investors, it isn't entirely unexpected. This is due to the short treatment period and the complexity of the disease, which has a history of clinical trial challenges.
威廉·布萊爾指出，雖然此更新令患者和投資者感到失望，但並不是完全意外。這是由於治療時間短和疾病複雜性歷史上臨牀試驗面臨的挑戰。
Biogen Inc. (NASDAQ:BIIB) and Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) faced a similar situation with Qalsody, which failed to show significant improvement in ALSFRS-R scores over six months. However, it eventually received accelerated approval based on reduced NfL levels and indications of benefit with early treatment over 52 weeks.
生物科技公司（NASDAQ:BIIB）和Ionis Pharmaceuticals, Inc.（NASDAQ:IONS）在Qalsody方面面臨類似情況，該藥物在六個月內未能顯示出ALSFRS-R評分的顯著改善。然而，它最終基於NfL水平降低和早期治療52周的好處指示獲得了加速批准。
Analyst Sarah Schram believes it's too soon to abandon the program and is awaiting a more detailed analysis, expected in late 2025.
分析師Sarah Schram認爲現在放棄該項目爲時尚早，並在等待預計在2025年底發佈的更詳細分析。
Price Action: DNLI stock is up 7.29% at $21.27 at the last check on Tuesday.
價格動作：DNLI股票在週二最後檢查時上漲了7.29%，報21.27美元。
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