Wave Life Sciences (WVE) Jefferies 2024 Global Healthcare Conference summary

Key program updates and clinical milestones

• Multi-dose data for the first allele-specific silencing therapy in Huntington's disease expected in Q2 2024, with potential market expansion to additional SNPs.

• Six-month dystrophin data from the N-531 program for DMD anticipated in Q3 2024, supporting possible expansion to 40% of DMD patients.

• Proof of mechanism data for WVE-006 in alpha-1 antitrypsin deficiency expected in 2024, with ongoing patient studies and potential to address all treatment goals.

• Inhibin E siRNA therapy for obesity, a novel genetic target, is on track for clinical trials in Q1 2025 after candidate declaration in Q1 2024.

• Additional AIMer RNA editing programs targeting both rare and prevalent diseases to be updated at R&D Day in fall 2024.

Platform and technology advancements

• Multimodal drug discovery platform enables antisense, RNAi, splicing, and RNA editing, supported by in-house GMP manufacturing and strong IP.

• Proprietary chemistry allows efficient, specific RNA editing without LNPs, achieving high editing rates and functional protein correction in preclinical models.

• Machine learning and the EditVerse platform facilitate translation of clinical genetic insights into new RNA medicine programs.

• RNAi platform improvements yield 30-fold better AGO2 loading, enabling more potent, durable silencing and potential for infrequent dosing.

Strategic collaborations and financial outlook

• Ongoing collaborations with GSK and Takeda provide access to genetic targets, with up to $2.8 billion in milestones and recent payments of $20 million and $12 million in Q1 2024.

• GSK partnership supports alpha-1 antitrypsin and siRNA programs, with additional target opportunities and substantial royalties.

• Anticipated significant cash flows in 2024 from milestones across existing and emerging programs.