Wave Life Sciences (WVE) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic vision and innovation

• Focus on unlocking the potential of RNA medicines, leveraging proprietary chemistry and deep genetic insights for rapid translation from genetic insights to clinical candidates.

• In-house GMP manufacturing and strong IP position support innovation, scalability, and swift clinical advancement.

• Well-capitalized with $602 million in cash, funding operations into Q3 2028.

• Innovations in siRNA chemistry yield 10-15x improvement in AGO2 loading, enhancing potency and durability.

• Bifunctional oligonucleotide constructs enable simultaneous RNA editing and silencing, expanding therapeutic reach.

Clinical and preclinical program highlights

• WVE-007 (INHBE) for obesity shows durable fat loss and muscle preservation, with potential for once or twice-yearly dosing.

• INLIGHT studies demonstrate significant reductions in visceral and total fat, with lean mass preserved.

• Preclinical data support synergistic use of WVE-007 with GLP-1s and prevention of weight regain post-incretin therapy.

• RNA editing programs target Alpha-1 antitrypsin deficiency (AATD) and PNPLA3 for liver disease, with WVE-006 restoring wild-type AAT protein and WVE-008 targeting over nine million patients.

• Extrahepatic programs deliver to adipose, muscle, and CNS, with bifunctional oligonucleotides in development.

Near-term milestones and development plans

• Multiple obesity data readouts expected in 2024, including higher dose and longer-term results.

• Accelerating WVE-007 development with higher dose and longer follow-up data, and initiating Phase 2a multidose trial in 1H 2026.

• WVE-006 to deliver 400 mg multidose data in 1Q 2026 and 600 mg cohort data later in 2026.

• WVE-008 CTA filing expected in 2026, aiming to address PNPLA3-driven liver disease.

• NDA submission for WVE-N531 (DMD) and IND for WVE-003 (HD) planned for 2026.