Wave Life Sciences .(WVE) - 2025 Q1 - Quarterly Results

Executive Summary First Quarter 2025 Overview Wave Life Sciences reported Q1 2025 results, highlighting clinical progress and a cash runway into 2027 - Dosing completed in the first two cohorts of the INLIGHT trial for WVE-007 in obesity, with clinical data on track for 2H 2025[1] - Dosing is underway in the RestorAATion-2 trial for WVE-006 in AATD, with data expected in 3Q 2025 and Fall 2025[1] - Positive data from the FORWARD-53 trial for WVE-N531 in DMD showed significant functional improvement, with an NDA submission planned for 2026[1] - An IND submission is expected in 2H 2025 for the potentially registrational WVE-003 Phase 2/3 study in HD[1] - Cash and cash equivalents of $243.1 million as of March 31, 2025, provide a financial runway expected into 2027[1] CEO Commentary The CEO highlighted consistent execution, leadership in RNA medicines, and upcoming clinical data across key programs - Wave Life Sciences is on track to deliver multiple clinical datasets in 2025, demonstrating broad capabilities in RNA medicines[2] - WVE-007 in obesity has the potential to transform treatment with healthy weight loss and infrequent dosing[2] - WVE-N531 in DMD delivered the first-ever substantial improvements in muscle health with an exon skipping therapy[2] - WVE-003 in HD has industry-leading CSF mutant lowering and is the only program to demonstrate allele-selective knockdown in the clinic[2] Recent Business Highlights and Expected Milestones Obesity (WVE-007) WVE-007 shows preclinical potential for healthy weight loss, with clinical data from the INLIGHT trial expected in 2H 2025 - WVE-007 is a GalNAc-siRNA designed to silence INHBE mRNA, an obesity target with strong human genetics evidence[4] - Dosing has been completed in the first and second single dose cohorts of the INLIGHT clinical trial[4] - Preclinical data showed a single dose of WVE-007 led to weight loss on par with semaglutide but with no muscle loss[4] - Clinical data from INLIGHT, including safety, tolerability, and biomarkers, is expected in the second half of 2025[4] Alpha-1 Antitrypsin Deficiency (AATD) (WVE-006) WVE-006 demonstrated the first-ever clinical RNA editing in humans, with further data from the RestorAATion-2 trial expected in 2025 - WVE-006 is a subcutaneously delivered RNA editing oligonucleotide (AIMer) designed to address AATD-related lung and/or liver disease[4] - Multi-dosing is ongoing in the first cohort of RestorAATion-2 (200 mg) and dosing is underway in the second single dose cohort (400 mg)[4] - Proof-of-mechanism data showed mean circulating wild-type M-AAT protein reached 6.9 micromolar, with total AAT increasing to 10.8 micromolar[7] - Data from complete 200 mg multidose and single dose cohorts is expected in Q3 2025, with 400 mg single dose data expected in Fall 2025[7] Emerging Wholly Owned siRNA and RNA Editing Pipeline Wave is expanding its pipeline with new siRNA and RNA editing targets, with clinical development for additional programs planned for 2026 - Wave is advancing new targets to expand its pipeline of wholly owned programs in both rare and common diseases[7] - The wholly owned RNA editing pipeline includes programs for PNPLA3 (liver diseases) and LDLR/APOB (familial hypercholesterolemia)[7] - Preclinical data for AIMers in lung indications showed increased CFTR mRNA expression 3-fold and restored up to 50% of functional protein levels[7] - New preclinical data is expected in 2025, with initiation of clinical development for PNPLA3, LDLR, and APOB planned for 2026[7] Duchenne Muscular Dystrophy (DMD) (WVE-N531) Positive 48-week data for WVE-N531 showed significant functional and muscle health improvements, supporting a planned 2026 NDA submission - WVE-N531 is an exon skipping oligonucleotide for Duchenne muscular dystrophy amenable to exon 53 skipping[7] - Positive 48-week data from the FORWARD-53 trial included a statistically significant improvement of 3.8 seconds in Time-to-Rise vs natural history[7] - The trial demonstrated substantial improvements in muscle health, including a statistically significant reduction in fibrosis[10] - Dystrophin expression stabilized, averaging 7.8%, with 88% of boys above 5% average dystrophin[10] - The U.S. FDA confirmed the accelerated approval pathway using dystrophin expression as a surrogate endpoint remains open[10] - An NDA submission for accelerated approval of WVE-N531 with monthly dosing is planned for 2026[10] Huntington's Disease (HD) (WVE-003) WVE-003 demonstrated allele-selective mHTT reduction correlated with slowing caudate atrophy, with an IND for a Phase 2/3 study expected in 2H 2025 - WVE-003 is a first-in-class, allele-selective oligonucleotide for the treatment of Huntington's disease[10] - SELECT-HD trial data showed the first-ever allele-selective reduction in CSF mHTT protein and a significant correlation with slowing of caudate atrophy[10] - Wave has received supportive initial feedback from the FDA regarding a potential pathway to accelerated approval[10] - An IND application for a potentially registrational Phase 2/3 study of WVE-003 in HD is expected in the second half of 2025[10] Financial Highlights First Quarter 2025 Financial Performance The company reported a net loss of $46.9 million for Q1 2025, with a cash runway projected into 2027 First Quarter 2025 Financial Summary (in thousands) | Metric | Q1 2025 | Q1 2024 | Change (YoY) | | :------------------------ | :------ | :------ | :----------- | | Revenue | $9,175 | $12,538 | -26.8% | | Research and development | $40,622 | $33,447 | +21.4% | | General and administrative| $18,357 | $13,549 | +35.5% | | Total operating expenses | $58,979 | $46,996 | +25.5% | | Loss from operations | $(49,804)| $(34,458)| -44.5% | | Net loss | $(46,878)| $(31,558)| -48.5% | | Net loss per share (basic & diluted) | $(0.29) | $(0.24) | -20.8% | - Cash and cash equivalents were $243.1 million as of March 31, 2025, which is expected to fund operations into 2027[10][18] Company Information Investor Conference Call and Webcast Wave Life Sciences hosted an investor call on May 8, 2025, to review its Q1 2025 financial results and pipeline updates - An investor conference call and webcast was held on May 8, 2025, at 8:30 a.m. ET[11] - The purpose was to review first quarter 2025 financial results and pipeline updates[11] About Wave Life Sciences Wave Life Sciences is a clinical-stage biotechnology company developing RNA medicines using its proprietary PRISM® platform - Wave Life Sciences is a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines[12] - The company's PRISM® platform combines multiple modalities, chemistry innovation, and deep insights in human genetics[12] - Wave's diversified pipeline includes clinical programs in AATD, DMD, HD, and Obesity[12] Forward-Looking Statements This report contains forward-looking statements regarding future goals and financial outlook, which are subject to various risks and uncertainties - The press release contains forward-looking statements concerning goals, clinical trial data, regulatory submissions, and financial runway[13] - Actual results may differ materially due to factors such as financing ability, clinical trial outcomes, regulatory actions, and market competition[15] Contact Information Contact information is provided for investor relations and media inquiries - Contact for investors: InvestorRelations@wavelifesci.com[16] - Contact for media: MediaRelations@wavelifesci.com[16] Unaudited Consolidated Financial Statements Consolidated Balance Sheets Total assets decreased to $288.3 million as of March 31, 2025, primarily due to a reduction in cash and cash equivalents Consolidated Balance Sheet Highlights (in thousands) | Metric | March 31, 2025 | December 31, 2024 | Change | | :-------------------------- | :------------- | :---------------- | :----- | | Cash and cash equivalents | $243,075 | $302,078 | $(59,003) | | Total current assets | $257,976 | $320,394 | $(62,418) | | Total assets | $288,343 | $352,207 | $(63,864) | | Total current liabilities | $87,367 | $110,953 | $(23,586) | | Total liabilities | $108,666 | $134,818 | $(26,152) | | Total shareholders' equity | $171,803 | $209,515 | $(37,712) | Consolidated Statements of Operations and Comprehensive Loss The company reported a net loss of $46.9 million for Q1 2025, an increase from $31.6 million in the prior year quarter Consolidated Statements of Operations Highlights (in thousands) | Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :---------------------------------- | :-------------------------------- | :-------------------------------- | | Revenue | $9,175 | $12,538 | | Research and development | $40,622 | $33,447 | | General and administrative | $18,357 | $13,549 | | Total operating expenses | $58,979 | $46,996 | | Loss from operations | $(49,804) | $(34,458) | | Net loss | $(46,878) | $(31,558) | | Net loss per share (basic and diluted) | $(0.29) | $(0.24) |