WAVE Life Sciences Continues Development of Two Drug Therapies for Huntington’s

WAVE Life Sciences’ products WVE-120101 (SNP-1) and WVE-120102 (SNP-2) will enter clinical trials in mid-2017 as part of the effort to develop new treatments for patients with Huntington’s disease.

“2017 will be an important year for WAVE as we transition our two lead candidates in Huntington’s disease … into clinical trials,” Paul Bolno, MD, and WAVE Life Sciences’ president and CEO, said in a press release. “Furthermore, we plan to select three additional proprietary candidates this year which will keep us on track to initiate six development programs by the end of 2018, each with the potential to make a meaningful impact to patients with rare genetic diseases,” he said.

Both WVE-120101 and WVE-120102 act by selectively binding the mRNA (a copy of a portion of DNA containing the necessary information for the production of a given protein) for mutant huntingtin (mHTT, the protein affected in Huntington’s disease). This allows the investigational drugs to block the expression of the mutant protein.

Importantly, treatment with these drugs allows the selective silencing of mHTT while sparing the healthy HTT version to function normally, which means the corresponding functional protein is expressed.

Each drug targets distinct patient populations, with different genetic mutations. Together, the drugs in development possibly could help treat more than two-thirds of the Huntington’s disease population.

In January, the company announced going forward with a plan to file a Clinical Trial Application (CTA) in Europe in the first half of 2017. The trial design has been updated, and WAVE will conduct two multi-ascending-dose (MAD) studies, and not first go through with a single-ascending-dose phase. MAD studies usually are used to assess the drug’s behavior and determine the maximum tolerated multiple dose.

After informed discussion with the U.S. Food and Drug Administration (FDA), WAVE plans to refile an Investigational New Drug (IND) Application for WVE-120101 and to file a second one for WVE-120102, as potential treatments for patients with Huntington’s disease.

In July 2016, the FDA granted orphan drug designation to WVE-120101, to accelerate its development as a treatment for certain patients with the neurodegenerative condition.