LeonaBio (LONA) Goldman Sachs 45th Annual Global Healthcare Conference summary

Leadership and company overview

• Recent leadership changes include a new Chief Medical Officer joining weeks prior to the conference.

• The company focuses on neurodegeneration, targeting natural repair pathways for neuronal health.

Pipeline and mechanism of action

• Lead molecule Fosgonimeton targets the HGF neurotrophic signaling system, aiming to decrease inflammation and improve mitochondrial function in Alzheimer's disease.

• The approach is unique in focusing on neuron repair and protection, with robust preclinical and early clinical evidence.

• Fosgonimeton is designed to cross the blood-brain barrier, while ATH-1105, recently entered into the clinic, targets both CNS and periphery for ALS.

Clinical data and trial design

• The ACT trial in mild to moderate Alzheimer's showed cognitive improvement and positive biomarker trends, especially in patients not on cholinesterase inhibitors.

• LIFT-AD trial was amended to focus on patients not on cholinesterase inhibitors, with results expected in the fall and a primary analysis population of 315.

• Interim analysis of LIFT-AD passed futility and efficacy checks, leading to an increased sample size for robust statistical power.

• The primary endpoint is a Global Statistical Test combining cognition and daily living measures, requiring positive results in both.