Wave Life Sciences (WVE) Jefferies Global Healthcare Conference 2026 summary

Strategic objectives and resource allocation

• Entered the year with clear objectives and disciplined resource allocation, supported by substantial cash reserves.

• Focused on advancing three core programs: INHBE for obesity, alpha-1 antitrypsin deficiency (AATD), and PNPLA3 for liver disease.

INHBE program for obesity and cardiometabolic indications

• INHBE program includes monotherapy, combination, and maintenance studies, all set to initiate in succession this year.

• Phase II-A monotherapy study will enroll high BMI patients with and without diabetes, focusing on fat loss and muscle preservation.

• Preclinical and clinical data show significant reductions in visceral and subcutaneous fat, with preservation of lean mass.

• Differentiation from other siRNAs is based on unique chemistry, potency, and durability, with once or twice yearly dosing anticipated.

• Regulatory focus is shifting from weight loss alone to body composition, with ongoing engagement with agencies on new endpoints like VMR.

Alpha-1 antitrypsin deficiency (AATD) program

• Recent data updates show dose response and dynamic response, with substantial reduction in harmful Z protein and increase in beneficial M protein.

• Regulatory feedback expected mid-year, aiming for accelerated approval based on biomarker and dynamic response data.

• RNA editing approach offers a non-permanent, redosable, and safer alternative to DNA editing and protein replacement therapies.

• Frontline use case for ZZ mutation patients is emphasized, leveraging endogenous protein production during acute events.