Clinical Trials

Date: 2017-07-17

Type of information: Initiation of the trial

phase: 1-2

Announcement: initiation of the trial

Company: Wave LIfe Sciences (USA - MA)

Product: WVE-120102

Action mechanism:

antisense oligonucleotide. WVE-120102 is an investigational stereopure antisense oligonucleotide designed to selectively target the mHTT mRNA transcript of SNP rs362331 (SNP2), respectively. This is one of the most common SNPs associated with the mutant allele, which is believed to encompass approximately two-thirds of the HD patient population. In vitro studies in patient-derived cell lines have shown that WVE-120102 selectively reduce levels of mHTT mRNA and protein, while leaving wtHTT mRNA and protein largely intact.

Disease: Huntington's disease

Therapeutic area: Rare diseases - Genetic diseases - Neurodegenerative diseases

Country: Canada, Poland

Trial details:

PRECISION-HD1 and PRECISION-HD2 are Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled studies that will primarily evaluate the safety and tolerability of single and multiple doses of WVE-120101 and WVE-120102, respectively, administered intrathecally in HD patients. Additional exploratory objectives include assessing the impact that each compound has on the toxic mutant protein known to cause loss of brain cells in HD, as well as evaluating potential clinical effects and impact on brain atrophy as measured by magnetic resonance imaging (MRI).
(NCT03225846)

Latest news:

• On July 17, 2017, Wave Life Sciences Ltd. (NASDAQ:WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced the initiation of the Company’s PRECISION-HD program, which includes PRECISION-HD1 and PRECISION-HD2, the Company’s two Phase 1b/2a clinical trials evaluating WVE-120101 and WVE-120102, respectively, for patients with Huntington’s disease (HD).
Both PRECISION-HD trials will follow the same protocol, and each will target a single nucleotide polymorphism, or “SNP,” that marks a separate and distinct location on the mutant huntingtin (HTT) gene transcript. Wave intends to enroll approximately 50 patients globally in each of the two studies through multiple sites, in Canada initially, with Europe and the United States to follow. The PRECISION-HD trials for WVE-120101 and WVE-120102 will include adult patients with early manifest HD who carry a SNP at the rs362307 (“SNP1”) or the rs362331 (“SNP2”) location, respectively. Potential HD patients for the PRECISION-HD program will be pre-screened for the presence of SNP1 or SNP2, and directed to the appropriate study upon qualifying for entry. Approximately two-thirds of all HD patients are expected to carry either SNP1, SNP2, or both, in association with the HD gene.