Wave Life Sciences Reports First Quarter 2025 Financial Results and Provides Business Update

Core Insights - Wave Life Sciences Ltd. has made significant progress in its clinical trials, including the completion of dosing in the INLIGHT trial for obesity and positive data from the FORWARD-53 trial for Duchenne muscular dystrophy (DMD) [1][2][3] - The company is on track to deliver multiple clinical datasets in 2025, which will showcase its capabilities in RNA medicines [2] - Financial results for Q1 2025 indicate a net loss of $46.9 million, with cash and cash equivalents of $243.1 million, expected to fund operations into 2027 [6][7][14] Obesity - WVE-007, a GalNAc-conjugated siRNA, aims to induce healthy weight loss by silencing INHBE mRNA, with dosing completed in the first two cohorts of the INLIGHT trial [3][4] - The trial assesses safety, tolerability, pharmacokinetics, and metabolic health, with clinical data expected in the second half of 2025 [3][4] - WVE-007 has shown potential for significant weight loss without muscle loss, and when combined with semaglutide, it doubled weight loss [4] Alpha-1 Antitrypsin Deficiency (AATD) - WVE-006 is being evaluated in the RestorAATion-2 trial, with ongoing dosing in both single and multiple cohorts [1][3] - Data from the 200 mg multidose and single dose cohorts are expected in Q3 2025, while data from the 400 mg cohort is anticipated in fall 2025 [1][4] - The trial aims to demonstrate the safety and efficacy of WVE-006 in individuals with the PiZZ mutation [4] Duchenne Muscular Dystrophy (DMD) - WVE-N531 has shown statistically significant improvements in muscle health and function in the FORWARD-53 trial, with a 3.8-second improvement in Time-to-Rise [7] - The drug has received Orphan Drug Designation and is expected to file for accelerated approval in 2026 [7] - All participants in the trial have opted to continue into the extension phase, receiving monthly doses [7] Huntington's Disease (HD) - WVE-003 is positioned as a first-in-class treatment for HD, demonstrating allele-selective reduction of mutant HTT protein while preserving wild-type HTT [7] - The company is preparing for a potentially registrational Phase 2/3 study, with an IND submission expected in the second half of 2025 [7][5] Financial Overview - Revenue for Q1 2025 was $9.2 million, down from $12.5 million in the same quarter of the previous year [6][14] - Research and development expenses increased to $40.6 million from $33.4 million year-over-year, while general and administrative expenses rose to $18.4 million from $13.5 million [6][14] - The net loss for the quarter was $46.9 million, compared to a loss of $31.6 million in the prior year [6][14]