Press release
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Exhibit 99.1 WAVE LIFE SCIENCES Wave Life Sciences Provides Update on Phase 1b / 2a PRECISION - HD Trials mHTT results from PRECISION - HD trials do not support further development of WVE - 120102 and WVE - 120101 No observed change in wtHTT and NfL during trials Advancing Phase 1b / 2a trial for WVE - 003 ( SNP3 ) program in HD ; WVE - 003 uses new PN backbone chemistry modifications , which demonstrate improved preclinical pharmacology Wave to host investor conference call and webcast today at 4:30 p.m. ET CAMBRIDGE , Mass . , March 29 , 2021 - Wave Life Sciences Ltd. ( Nasdaq : WVE ) , a clinical - stage genetic medicines company committed to delivering life - changing treatments for people battling devastating diseases , today announced data from the Phase 1b / 2a PRECISION - HD2 and PRECISION - HD1 trials evaluating investigational treatments WVE - 120102 and WVE - 120101 , respectively , in Huntington's disease ( HD ) . In the PRECISION - HD2 core trial , results from all participants ( n = 88 ) showed no statistically significant change in mutant huntingtin protein ( mHTT ) versus placebo after single or multiple doses of WVE - 120102 up to and including 32 mg monthly . There was no evidence of a dose response across the dose levels tested . In the PRECISION - HD2 open label extension ( OLE ) trial , results from all participants ( n = 28 ) showed modest reductions in mHTT after a mean of 8.1 monthly doses ( range : 1-17 ) , but effects were inconsistent over the course of the trial . While there was a correlation between WVE - 120102 cerebrospinal fluid ( CSF ) concentrations and mHTT lowering , pharmacokinetic ( PK ) modeling suggests that additional dose escalation is unlikely to achieve drug concentrations needed for robust mHTT knockdown . Given the lack of consistent and significant reductions in mHTT and the PK modeling projections , Wave will stop clinical development of WVE - 120102 . Trial participants will have a final follow - up visit but receive no further doses . Results in all participants through 16 mg ( n = 51 ) from the PRECISION - HD1 core trial are similar to those in PRECISION - HD2 at those dose levels . Given these and results from Wave's previous clinical trials , as well as current understanding of the limitations of the company's first - generation candidates , Wave will also stop clinical development of WVE - 120101 . Dosing in the 32 mg cohort of the PRECISION - HD1 core trial is complete , and core and OLE trial participants will have a final follow - up visit but receive no further doses . Wave expects to complete analysis of the 32 mg cohort in the second quarter of 2021 . " We are disappointed not to have better news to share with the HD community and would like to extend our gratitude to the trial participants and their families for their commitment to these programs , " said Michael Panzara , MD , MPH , Chief Medical Officer and Head of Therapeutics Discovery and Development at Wave Life Sciences . " However , we have learned an enormous amount from these trials and are encouraged by data that suggest our SNP targeting approach may achieve allele - selectivity . We look forward to continuing our collaboration with our research partners to advance our first HD clinical candidate that incorporates our next - generation PN backbone chemistry , WVE - 003 , and remain committed to the science of HD and , in particular , selective mutant huntingtin lowering . "