Wave Life Sciences .(US:WVE)2025-05-08 12:01Pipeline and Programs - Wave Life Sciences is developing WVE-007, a GalNAc-siRNA targeting INHBE for obesity treatment, addressing a market of approximately 175 million adults in the US and Europe[11, 14] - WVE-006, an RNA editing treatment for Alpha-1 antitrypsin deficiency (AATD), aims to address both liver and lung manifestations, targeting ~200,000 people in the US and Europe homozygous for the Z allele[37, 36] - WVE-N531, a splicing oligonucleotide for Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, showed 78% average dystrophin expression between 24 and 48 weeks in clinical trials, with 88% of boys above 5% dystrophin[52, 54, 56] - WVE-003, an allele-selective oligonucleotide for Huntington's Disease (HD), achieved up to a 46% reduction in mutant HTT protein in CSF[97, 101] Clinical and Regulatory Milestones - Clinical data for WVE-007 (INHBE) in obesity is expected in the second half of 2025[31] - Data from the complete 200 mg multidose and single dose cohorts of WVE-006 (AATD) is expected in 3Q 2025, with 400 mg single dose cohort data expected in the fall of 2025[43] - Wave Life Sciences plans to submit an NDA in 2026 to support accelerated approval of WVE-N531 with monthly dosing[53, 86] - An IND application for a potentially registrational Phase 2/3 study of WVE-003 in Huntington's Disease is expected to be submitted in the second half of 2025[104] Platform and Technology - Wave Life Sciences' RNA medicines platform (PRISM®) utilizes multiple modalities including RNA editing, RNAi, splicing, and allele-selective silencing[7] - The company is developing GalNAc-AIMer programs for liver disease (PNPLA3) targeting ~9 million patients, HeFH (LDLR) targeting ~900,000 patients with expansion to ~30 million, and HeFH (APOB) targeting ~70,000 patients[45]