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.
周一，Denali Therapeutics Inc.（纳斯达克：DNLI）公布了对DNL343在肌萎缩侧索硬化症的2/3期HEALEY ALS平台试验G方案分析的初步结果。
Compared to placebo, the study did not meet the primary endpoint of efficacy in slowing disease progression.
与安慰剂相比，该研究未能达到减缓疾病进展的主要疗效终点。
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.
主要终点是评估疾病严重程度随时间变化的情况，测量工具为ALS功能评分量表修订版（ALSFRS-R）以及生存率通过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.
关键的次要终点，测量肌肉力量和呼吸功能，在第24周时也在活性组和安慰剂组之间没有统计学差异。
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名随机接受DNL343治疗的参与者与139名随机接受安慰剂的参与者进行了比较，后者的方案为(n=63)或来自同时入组方案的共享(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.
总体而言，DNL343被发现是安全且耐受良好的。预计在2025年晚些时候进行进一步分析，包括神经丝轻链（NfL）和其他液体生物标志物、预先指定的亚组数据以及活性治疗扩展期的扩展发现。
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·布莱尔指出，虽然此更新令患者和投资者感到失望，但并不是完全意外。这是由于治疗时间短和疾病复杂性历史上临床试验面临的挑战。
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方面面临类似情况，该药物在六个月内未能显示出ALSFRS-R评分的显著改善。然而，它最终基于NfL水平降低和早期治疗52周的好处指示获得了加速批准。
Analyst Sarah Schram believes it's too soon to abandon the program and is awaiting a more detailed analysis, expected in late 2025.
分析师Sarah Schram认为现在放弃该项目为时尚早，并在等待预计在2025年底发布的更详细分析。
Price Action: DNLI stock is up 7.29% at $21.27 at the last check on Tuesday.
价格动作：DNLI股票在周二最后检查时上涨了7.29%，报21.27美元。
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