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LeonaBio presents clinical strategy for ALS candidate brelgometon

Written by Heidi Cuthbert Published Jun 4, 2026, 11:50 AM

LeonaBio (NASDAQ:LONA) announced today that Chief Medical Officer Javier San Martin, MD, presented the company’s clinical strategy for its lead ALS candidate, brelgometon (ATH-1105), at the 5th Annual ALS Drug Development Summit in Boston.

The presentation highlighted the company’s approach to trial design, emphasizing the integration of specialized biomarkers and adaptive methodologies to accelerate the assessment of the drug’s therapeutic potential.

Brelgometon is an oral, brain-penetrant small molecule designed to act as a positive modulator of the hepatocyte growth factor (HGF) system.

By promoting neurotrophic signaling, the molecule is intended to address the underlying cellular mechanisms of neurodegeneration seen in amyotrophic lateral sclerosis (ALS).

According to LeonaBio, the development program has reached a critical juncture following the completion of initial clinical and preclinical evaluations.

Data from Phase 1 studies and preclinical models have provided sufficient support to advance the candidate into a randomized, double-blind, placebo-controlled Phase 2 study.

This proof-of-concept, dose-ranging trial is designed to establish safety and efficacy signals in patients, with the company currently targeting a start date in the second half of 2026.