LeonaBio (LONA) Jefferies 2024 Global Healthcare Conference summary

Company overview and scientific approach

• Focuses on neurodegeneration with a unique strategy to enhance natural nerve cell repair via the HGF system.

• Lead molecule, Fosgo, targets Alzheimer's by crossing the blood-brain barrier and enhancing HGF.

• Pipeline includes ATH-1105 for ALS, designed for peripheral action at neuromuscular junctions.

• All molecules are based on modulating the HGF pathway, with each tailored for specific indications.

Clinical development and trial updates

• LIFT, a late-stage trial for Fosgo in Alzheimer's, completed enrollment in December and will end this month.

• Data readout is expected in the fall after an 8-12 week data cleaning process.

• Interim analysis of the first 100 patients showed efficacy on composite cognition and function scores, supporting trial continuation.

• Baseline characteristics between the first 100 and next 200 patients are consistent, aiming for robust results.

Study design, endpoints, and regulatory engagement

• LIFT uses a composite GST score (ADAS-Cog11 + ADL) as the primary endpoint, with additional biomarker analysis (NfL, p-tau, GFAP, A-beta).

• FDA alignment was achieved on trial design and endpoints, including biomarker strategy.

• Success on GST and its components will inform the need for further pivotal studies; another trial is likely required for approval.

• Inclusion criteria target mild to moderate Alzheimer's with rapid expected decline, optimizing for measurable treatment effect.