Wave Life Sciences begins Phase Ib/IIa trials of two Huntington’s disease drugs


Wave Life Sciences has begun new Phase Ib/IIa clinical trials (PRECISION-HD1 and PRECISION-HD2) of WVE-120101 and WVE-120102 to treat patients suffering with Huntington’s disease (HD).

WVE-120101 is an investigational stereopure antisense oligonucleotide currently being developed to target the mHTT mRNA transcript of single nucleotide polymorphism (SNP) rs362307 (SNP1), while WVE-120102 belongs to the same category and is designed to target SNP rs362331 (SNP2).

The PRECISION-HD1 trial will assess the safety and tolerability of single and multiple doses of WVE-120101 and PRECISION-HD2 will investigate those of WVE-120102.

Both multi-centre, randomised, double-blind, placebo-controlled trials will enrol a total of around 100 adults with early manifest HD harbouring a SNP at the rs362307 or the rs362331 location.

"Wave’s PRECISION-HD programme is the first to target the underlying cause of Huntington’s disease with an allele-specific approach."

Wave Life Sciences neurology franchise lead Michael Panzara said: “Wave’s PRECISION-HD programme is the first to target the underlying cause of Huntington’s disease with an allele-specific approach.

“More importantly, these investigational compounds have the potential to address a critical unmet need for the HD patient community where no disease-modifying treatments are currently approved.”

To be initially conducted across Canada and later in Europe and the US, the trials are part of the firm’s PRECISION-HD programme.

The trials will measure exploratory objectives such as the impact of each compound on the toxic mutant protein reported to cause loss of brain cells in HD, and also potential clinical effects and impact on brain atrophy using magnetic resonance imaging (MRI).