What's Going On With Wave Life Sciences Stock On Tuesday?
What's Going On With Wave Life Sciences Stock On Tuesday?
Wave Life Sciences Ltd (NASDAQ:WVE) announced results from its Phase 1b/2a SELECT-HD trial of WVE-003, which is being developed as a potential disease-modifying therapeutic for Huntington's disease (HD).
Wave Life Sciences宣佈了WVE-003 Phase 1b/2a SELECT-HD試驗的結果，WVE-003正在開發作爲亨廷頓病（HD）潛在的疾病修飾治療藥物。
WVE-003 is designed to lower mutant huntingtin (mHTT) protein and preserve healthy, wild-type huntingtin (wtHTT) protein.
WVE-003旨在降低突變的亨廷頓蛋白（mHTT）並保留健康的野生型亨廷頓蛋白（wtHTT）。
In the multidose portion of the SELECT-HD study (n=23), participants received either every-eight-week (Q8W) intrathecal doses of 30 mg WVE-003 (n=16) or placebo (n=7), with 12 weeks of follow-up.
在SELECT-HD研究的多劑量部分（n=23）中，參與者接受每8周進行一次（Q8W）腰椎內注射30毫克WVE-003（n=16）或安慰劑（n=7），並進行12周的隨訪。
Key results are as follows:
關鍵結果如下：
- WVE-003 was generally safe and well-tolerated, with no Serious Adverse Events (SAEs) reported; ventricular volume was in line with natural history.
- Significant mHTT protein lowering was observed throughout the 28-week assessment period.
- At 24 weeks (8 weeks after the last dose), mean mHTT lowering in cerebrospinal fluid (CSF) was 46% versus placebo (p=0.0007).
- At 28 weeks (12 weeks after the last dose), mean mHTT lowering in CSF was 44% versus placebo (p=0.0002), which supports quarterly or less frequent dosing.
- wtHTT protein was preserved throughout the 28-week assessment period, validating allele-selective silencing. Additionally, statistically significant increases were observed in wtHTT protein versus placebo.
- wtHTT protein supports the health and function of neurons and is crucial for CSF flow in the ventricles.
- mHTT also has a detrimental effect on wtHTT at the protein level, further decreasing its function. Only selective lowering of mHTT can potentially relieve its negative impact on wtHTT protein function.
- Most WVE-003-treated participants had neurofilament light protein (NfL) levels that were in the range of placebo or had NfL levels that increased and returned to the range of placebo.
- At 24 weeks (the last MRI assessment), mHTT reduction was correlated with slowing of caudate atrophy (R=-0.50; p=0.047). Caudate atrophy is an imaging biomarker predictive of clinical outcomes, including clinically meaningful worsening of Total Motor Score (TMS).
- A slowing of decline was observed for TMS for WVE-003 versus placebo (4.25 mean difference at 24 weeks, p=not significant).
- WVE-003通常是安全和耐受的，沒有報告嚴重不良事件（SAEs）；心室容積與自然歷史相一致。
- 在整個28周的評估期內，觀察到明顯的mHTT蛋白降低。
- 在24周時（最後一次劑量後8周），腦脊液（CSF）中平均mHTT降低46％，而安慰劑組爲44％（p=0.0007）。
- 在28周時（最後一次劑量後12周），腦脊液中平均mHTT降低44％，而安慰劑組爲44％（p=0.0002），這支持季度或更少頻繁的給藥。
- wtHTT蛋白在整個28周評估期內得以維持，從而驗證了等位基因選擇性沉默。另外，與安慰劑相比，觀察到wtHTT蛋白的顯着增加。
- wtHTT蛋白支持神經元的健康和功能，並對腦室的CSF流動至關重要。
- mHTT也會在蛋白水平上對wtHTT產生不良影響，進一步降低其功能。只有選擇性降低mHTT才有可能減輕其對wtHTT蛋白功能的負面影響。
- 大多數接受WVE-003治療的參與者的神經絲輕鏈蛋白（NfL）水平與安慰劑相同，或者NfL水平有所增加並返回到安慰劑範圍內。
- 在24周時（最後一次MRI評估），mHTT的減少與caudate萎縮的減緩相關（R=-0.50；p=0.047）。Caudate萎縮是預測臨床結果的成像生物標誌物，包括臨床意義上的Total Motor Score（TMS）惡化。
- 與安慰劑相比，WVE-003觀察到TMS的下降速度放緩（24周時平均差異爲4.25，p=不顯著）。
Wave expects that its current cash and cash equivalents of $181 million will be sufficient to fund operations into the fourth quarter of 2025.
Wave預計其目前的現金及現金等價物爲1.81億美元，足以支持經營活動延續到2025年第四季度。
Price Action: WVE shares traded down 3.07% at $5.05 at last check Tuesday.
價格行動：WVE股票在最後一次交易中下跌了3.07％，報5.05美元。
Photo via Shutterstock
圖片來自shutterstock。
譯文內容由第三人軟體翻譯。