Healthcare Business

Is It Over for This Huntington's Disease Clinical Trial?

Wave Life Sciences Ltd. (NASDAQ: WVE) shares dropped sharply to start out the week after the company announced topline data from its ongoing midstage trial in Huntington’s disease.

Specifically, the results come from the firm’s Phase 1b/2a Precision-HD2 trial evaluating investigational therapy WVE-120102, designed to be the first allele-selective approach to treat Huntington’s disease.

In an analysis comparing all patients treated with multiple doses of WVE-120102 to placebo, a statistically significant reduction of 12.4% in mutant huntingtin (mHTT) protein was observed in cerebrospinal fluid. An analysis to assess a dose response across treatment groups suggested a statistically significant response in mHTT reduction at the highest doses tested.

While there was a statistically significant reduction in mHTT with WVE-120102 compared to the placebo in the topline analysis, there was no difference in total HTT compared to placebo, suggesting WVE-120102 may have a potentially differential effect on huntingtin as measured by the mHTT and the total HTT assays.

Overall, WVE-120102 was generally safe and well tolerated across all cohorts. These data support the addition of higher dose cohorts, and Wave expects to initiate a 32 mg cohort in January 2020.

Michael Panzara, M.D., MPH, chief medical officer of Wave Life Sciences, commented:

This topline analysis has given us the opportunity to evaluate early data from our ongoing dose finding study. The data demonstrate a reduction in mutant HTT and a safety and tolerability profile that supports exploration of higher doses of WVE-120102, with the goal of maximizing mutant HTT reduction and avoiding a negative impact on the healthy huntingtin protein. We plan to initiate the 32 mg cohort imminently and look forward to sharing data in the second half of 2020.

Shares of Wave Life Sciences were last seen down 49% at $8.06, in a 52-week range of $14.39 to $48.64. The consensus price target is $33.29.