WAVE Life Sciences expects to report data from both its ongoing PRECISION-HD phase 1b/2a trials, and available data from the related OLE studies this spring. The analysis will include biomarker and safety data from all dosing cohorts up to and including the 32mg cohort for PRECISION-HD2 and completed dosing cohorts for PRECISION-HD1 (including all participants from the 16mg cohort). These data are expected to support a decision regarding a potential phase 3 study.
Participants from the phase 1b/2a trials have been enrolling into the OLE studies since 2020, and the vast majority of eligible patients have been enrolled. In December 2020, WAVE submitted a clinical trial application for WVE-003 (SNP3). This is WAVE’s first HD candidate to use its novel PN backbone chemistry modifications which, according to preclinical data, increase the potency, exposure and durability of a compound. WVE-003 is also designed to selectively target the mutant huntingtin mRNA transcript while leaving the wild-type protein relatively intact.
The protocol was also submitted to EHDN, which endorsed it in December 2020. WAVE expects to start dosing HD participants with SNP3 this year, once regulatory and ethical approvals are in place.