Shares of Wave Life Sciences (NASDAQ: WVE) are down 29% at 1:29 p.m. EDT after the company announced that WVE-120102 and WVE-120101, two of its drugs to treat Huntington’s disease, failed to show an effect in early stage clinical trials.
Huntington’s disease is caused by different mutations in the huntingtin protein. WVE-120101 and WVE-120102 are designed to reduce the expression of mutant huntingtin protein (mHTT) for patients with the SNP1 and SNP2 mutations, respectively.
The drugs appear to be working, but unfortunately the reduction on mHTT wasn’t clinically meaningful. Results from the clinical trial testing WVE-120102 showed the highest dose of the drug reduced mHTT by 9.9% in the cerebrospinal fluid compared to a 0.8% decrease for placebo. In a separate clinical trial of WVE-120101, which wasn’t as far along, the highest dose reduced mHTT by 11.6% compared to 10% for placebo.