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宣布了WVE-003 Phase 1b/2a SELECT-HD试验的结果，WVE-003正在开发作为亨廷顿病（HD）潜在的疾病修饰治疗药物。
WVE-003 is designed to lower mutant huntingtin (mHTT) protein and preserve healthy, wild-type huntingtin (wtHTT) protein.
WVE-003旨在降低突变的亨廷顿蛋白（mHTT）并保留健康的野生型亨廷顿蛋白（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研究的多剂量部分（n=23）中，参与者接受每8周进行一次（Q8W）腰椎内注射30毫克WVE-003（n=16）或安慰剂（n=7），并进行12周的随访。
Key results are as follows:
关键结果如下：
- 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，没有报告严重不良事件（SAEs）；心室容积与自然历史相一致。
- 在整个28周的评估期内，观察到明显的mHTT蛋白降低。
- 在24周时（最后一次剂量后8周），脑脊液（CSF）中平均mHTT降低46％，而安慰剂组为44％（p=0.0007）。
- 在28周时（最后一次剂量后12周），脑脊液中平均mHTT降低44％，而安慰剂组为44％（p=0.0002），这支持季度或更少频繁的给药。
- wtHTT蛋白在整个28周评估期内得以维持，从而验证了等位基因选择性沉默。另外，与安慰剂相比，观察到wtHTT蛋白的显着增加。
- wtHTT蛋白支持神经元的健康和功能，并对脑室的CSF流动至关重要。
- mHTT也会在蛋白水平上对wtHTT产生不良影响，进一步降低其功能。只有选择性降低mHTT才有可能减轻其对wtHTT蛋白功能的负面影响。
- 大多数接受WVE-003治疗的参与者的神经丝轻链蛋白（NfL）水平与安慰剂相同，或者NfL水平有所增加并返回到安慰剂范围内。
- 在24周时（最后一次MRI评估），mHTT的减少与caudate萎缩的减缓相关（R=-0.50；p=0.047）。Caudate萎缩是预测临床结果的成像生物标志物，包括临床意义上的Total Motor Score（TMS）恶化。
- 与安慰剂相比，WVE-003观察到TMS的下降速度放缓（24周时平均差异为4.25，p=不显著）。
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预计其目前的现金及现金等价物为1.81亿美元，足以支持经营活动延续到2025年第四季度。
Price Action: WVE shares traded down 3.07% at $5.05 at last check Tuesday.
价格行动：WVE股票在最后一次交易中下跌了3.07％，报5.05美元。
Photo via Shutterstock
图片来自shutterstock。