Wave Life Sciences (WVE) investor relations material

Strategic progress and innovation

• Focused on advancing a differentiated siRNA platform, with INHBE as a lead program targeting obesity by reducing body fat and preserving lean mass with infrequent dosing.

• Leadership in RNA editing, particularly for alpha-1 antitrypsin deficiency (AATD), aiming for less frequent dosing and holistic treatment of lung and liver disease.

• PNPLA3 program is on track to enter clinical trials for genetic liver disease later this year.

• Continued investment in both RNA editing and siRNA platforms, maintaining momentum into 2026.

Clinical data highlights and competitive landscape

• Recent ATS conference showcased late-breaking data on RNA editing for AATD, demonstrating meaningful changes in patient outcomes and strong enthusiasm from the clinical community.

• Differentiation from competitors by addressing the genetic root cause of AATD, with data showing significant reduction in toxic liver aggregates and improved protein levels.

• RNA editing approach achieves high specificity, avoiding off-target edits and permanent genetic changes, which is a key safety advantage over DNA editing.

• Monthly dosing for alpha-1 antitrypsin therapy is now considered feasible, with ongoing studies to optimize dosing intervals.

Regulatory and development pathways

• Biomarker-driven accelerated approval is being pursued for both alpha-1 antitrypsin and DMD programs, with regulatory feedback expected mid-year.

• CT densitometry and non-invasive liver function tests are being considered as endpoints for registrational studies.

• For INHBE, high BMI phase IIa studies are starting, focusing on achieving regulatory thresholds for weight loss and exploring additional cardiometabolic endpoints.

• DMD program continues to monitor the evolving regulatory landscape and maintains strong clinical data on muscle exposure and function.