Wave Life Sciences (WVE) Corporate presentation summary

Platform and technology

• PRISM platform enables multi-modal RNA medicines, including RNA editing, RNAi, splicing, and allele-selective silencing, using best-in-class, clinically validated oligonucleotide chemistry with proprietary modifications.

• Deep genetic insights and machine learning models, leveraging large human genetic databases, drive target selection and therapeutic design.

• Foundational advances in nucleic acid chemistry expand platform capabilities, with multiple peer-reviewed publications supporting technology differentiation.

Pipeline and clinical programs

• Robust pipeline includes first-in-class RNA editing (WVE-006 for AATD), RNAi (WVE-007 for obesity), splicing (WVE-N531 for DMD), and allele-selective silencing (WVE-003 for HD).

• WVE-006 demonstrated restoration of wild-type AAT protein and reduction of liver pathology in preclinical AATD models; proof-of-mechanism data expected 4Q 2024.

• WVE-007 (INHBE siRNA) shows potent, durable weight loss in preclinical models, with clinical trial initiation planned for 1Q 2025.

• WVE-N531 for DMD achieved high exon skipping and muscle concentrations in clinical data; potentially registrational Phase 2 data expected 3Q 2024.

• WVE-003 for HD achieved up to 46% allele-selective mHTT reduction with preservation of wild-type HTT and correlated with slowing of caudate atrophy.

Strategic collaborations and business development

• Strategic partnership with GSK includes $170M upfront, up to $3.3B in milestones, and tiered royalties; collaboration covers WVE-006 and up to eight additional programs.

• Takeda holds a 50:50 option on certain programs, with a decision expected by year-end 2024.

• Recent milestones include a $20M payment for first patient dosing and $12M for GSK's selection of two programs.