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.

Read the community letter

Read the press release

Categories: NewsResearch


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