Wave Life Sciences (WVE) investor relations material

Strategic vision and platform progress

• Focus on programmable RNA medicines across multiple modalities, leveraging rapid translation from genetic insights to clinical data with 18-month cycle times for target-to-human studies.

• $600 million invested to advance the platform, with collaborations such as GSK supporting clinical progress and expansion.

• Clinical programs include INHBE for obesity (phase II-A), alpha-1 antitrypsin deficiency (AATD) with a path to accelerated approval, and new RNA editing targets like PNPLA3 for liver disease.

• Emphasis on integrating human genetics databases (e.g., UK Biobank) to validate and prioritize targets.

INHBE obesity program insights

• INHBE targets a genetically validated pathway distinct from incretins, aiming for fat loss without muscle loss and improved cardiometabolic outcomes.

• Preclinical and phase I data show potent, durable fat loss, increased lean mass, and up to 15% visceral fat reduction with a single dose.

• Phase I results included a 3.3% reduction in waist circumference and strong safety/tolerability profile, with no GI or liver side effects.

• Phase II-A study is enrolling patients with high BMI and comorbidities, aiming for >5% total body weight loss driven by fat reduction, with comprehensive biomarker analysis.

• Upcoming studies will assess INHBE in combination with incretins and as a maintenance therapy to prevent fat regain post-therapy.

Regulatory and competitive positioning

• FDA's regulatory threshold for obesity therapies is >5% total body weight loss, with increasing emphasis on fat loss over muscle loss.

• INHBE program is positioned to differentiate by delivering fat-driven weight loss and improved body composition.

• Strong strategic interest and support from industry partners, with recent entry of other large biotech companies into the INHBE space.