Korro Bio (KRRO) Piper Sandler 36th Annual Healthcare Conference summary

Clinical program updates

• Regulatory clearance obtained in Australia, with first site activated and up to four sites planned in the country; global expansion anticipated for later study phases.

• Study design includes a single ascending dose (SAD) phase in healthy volunteers and ZZ genotype patients, followed by a multiple ascending dose (MAD) open-label phase.

• Up to six dose cohorts may be explored in the SAD, with fewer in ZZ patients; total study enrollment is 64 participants.

• SAD data readout expected in the second half of 2025, with MAD completion targeted for 2026; separate disclosures planned for each phase.

• Primary endpoints include safety, pharmacokinetics, and initial pharmacodynamics, focusing on AAT protein levels and ratios.

Technology and product differentiation

• Lipid nanoparticle (LNP) delivery was chosen for its ability to achieve therapeutically relevant protein levels and rapid action, with Genevant technology in-licensed.

• Preclinical studies showed over 50% editing and high protein levels, with a favorable safety window at higher doses compared to competitors.

• LNP enables higher dosing and early efficacy in both lung and liver, with lifecycle management plans to revisit delivery modalities as needed.

• Monthly intravenous dosing is targeted, aiming for normal protein levels and improved patient convenience over current weekly therapies.

• Assays for protein measurement will use mass spectrometry, with details to be disclosed alongside study data.

Market opportunity and strategic outlook

• Initial target population is ZZ homozygous patients, with potential expansion to heterozygous groups (MZ, SZ) in future lifecycle management.

• U.S. market includes 10,000 patients currently treated, with up to 35,000 diagnosed and a broader pool of 80,000–100,000 potentially addressable.

• 2025–2026 expected to be pivotal, with global phase 1/2 study progress, additional candidate visibility, and partnership developments.

• Company maintains a strategy to demonstrate efficacy in humans, create de novo proteins, and expand beyond liver indications.

• Additional assets and collaborations, including with Novo for cardiometabolic diseases, are in development.