Wave Life Sciences Ltd. Q4 FY2024 earnings call

• Progress on WVE-007 for obesity: Enrollment completed in first cohort of Phase 1 trial, expecting initial data in second half of 2025 on safety, tolerability, and biomarkers.
• WVE-006 for AATD: Completed cohorts in healthy volunteers, multi-dosing underway in 200 mg cohort, initiated 400 mg single dose cohort, expecting data from full first cohort and 400 mg cohort in 2025.
• DMD program: Collected all muscle biopsies, on track to deliver 48-week data by end of March, with upcoming data including dystrophin, functional measures, safety, and tolerability.
• WVE-003 for HD: Engaged with FCA for supportive feedback, preparation ongoing for phase 2/3 study using caudate atrophy as primary endpoint, on track to submit IND in second half of 2025.
• Emerging RNA editing pipeline: Advancing programs with support from human genetics, leveraging best-in-class oligonucleotide chemistry and GalNAc conjugation.

Revenue for the fourth quarter of 2024 was $83.7 million, and for the full year 2024 was $108.3 million, compared to $29.1 million and $113.3 million in the prior year quarter and year respectively. Research and Development expenses in the fourth quarter of 2024 were $44.6 million, and for the full year 2024 were $159.7 million. G&A expenses for the fourth quarter of 2024 were $16.1 million, and for the full year 2024 were $59 million. Net income for the fourth quarter of 2024 was $29 million, while the full year net loss was $96.7 million. Cash and cash equivalents ended 2024 at $302.1 million, up from $200.4 million in 2023.

2025 is expected to have multiple value accretive inflection points with milestones across all four clinical programs and data from the growing RNA editing pipeline. Current cash and cash equivalents are expected to fund operations into 2027.

Discussions include general risks associated with forward-looking statements, including factors that could cause actual results to differ from those in forward-looking statements, as detailed in the press release and SEC filings.

Q: What will a late-stage clinical study for 007 as maintenance therapy look like?

A: Planning to generate data on single dose and maintenance, designing studies to support registration and reimbursement, considering mono therapy and off-ramp to GLP-1.

Q: Details on multiple dose cohorts in AATD and Maat threshold?

A: Multiple dose cohorts have 8 patients with repeat dosing, expecting data in 2025, highlighting M-protein as key for editing efficiency, with therapeutic relevance at 200 mg dose.