Today, Wave Life Sciences announced positive results from SELECT-HD, our Phase 1b/2a placebo-controlled trial evaluating the investigational therapy WVE-003. These results demonstrate that WVE-003 selectively lowers toxic, mutant huntingtin (mHTT) protein and preserves healthy, wild-type huntingtin (wtHTT) protein for individuals with Huntington’s disease.
Trial Results
In the analysis, it was compared participants treated with three 30 mg intrathecal doses of WVE-003
every 8 weeks to participants who received placebo and saw the following:
- Significant, durable mHTT lowering of up to 46%.
- Preservation of healthy, wtHTT.
- WVE-003 was generally safe and well-tolerated.
- Statistically significant correlation of mHTT lowering with slowing of caudate loss, which
is an imaging biomarker that is predictive of clinical outcomes. - While this study was not powered for clinical outcomes, slowing of decline was
observed for Total Motor Score (TMS).
What happens next?
Based on the successful SELECT-HD study, next steps for WVE-003 will be informed by meetings
with regulators to discuss the development path for WVE-003, which will include a potential for
accelerated approval as well as an open-label extension (OLE) study for SELECT-HD.
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