LeonaBio Unveils Phase 2 Plans for Brelgometon (ATH-1105) in ALS, Highlighting Biomarker-Driven Trial Design at Summit
核心洞察
LeonaBio (搜索) presented its Phase 2 clinical development strategy for brelgometon (搜索) (ATH-1105), an oral brain-penetrant HGF (搜索) system modulator, at the 5th Annual ALS Drug Development Summit in Boston.
The planned randomized, double-blind, placebo-controlled Phase 2 proof-of-concept study is targeted for initiation in the second half of 2026 and will integrate neurofilament light chain (搜索) as a validated biomarker.
Phase 1 data in 80 healthy volunteers demonstrated favorable safety, tolerability, dose-proportional pharmacokinetics, and central nervous system penetration, supporting advancement into ALS patients.
LeonaBio (搜索), Inc. (NASDAQ: LONA) has outlined the next stage of clinical development for its investigational therapy brelgometon (搜索) (ATH-1105) in amyotrophic lateral sclerosis (搜索) (ALS). Javier San Martin, MD, Chief Medical Officer of LeonaBio, delivered a presentation titled "Advancing ATH-1105 in ALS: Strategic Trial Design, Biomarker Integration & Adaptive Methodologies" at the 5th Annual ALS Drug Development Summit held June 2–4, 2026 in Boston, Massachusetts.
The presentation detailed the company's plans to advance brelgometon (搜索) into a Phase 2 proof-of-concept and dose-ranging study, with initiation targeted for the second half of 2026.
Mechanism of Action and Preclinical Rationale
Brelgometon (搜索) is a novel, orally available, brain-penetrant, next-generation small molecule designed to positively modulate the neurotrophic HGF (搜索) (hepatocyte growth factor) system. The drug candidate is being developed for the potential treatment of neurodegenerative diseases, including ALS, Alzheimer's disease, and Parkinson's disease, with ALS as the current lead indication.
The compound's potential is supported by a body of preclinical evidence demonstrating statistically significant improvements in nerve and motor function, biomarkers of inflammation and neurodegeneration, and survival in various models of ALS.
Phase 1 Clinical Data
LeonaBio (搜索) completed the first-in-human Phase 1 double-blind, placebo-controlled clinical trial of brelgometon (搜索) (NCT06432647) in November 2024. The study enrolled 80 healthy volunteers to evaluate single and multiple oral ascending doses. Results demonstrated a favorable safety and tolerability profile, dose-proportional pharmacokinetics, and confirmed central nervous system penetration.
Phase 2 Trial Design
The planned Phase 2 proof-of-concept and dose-ranging study will be a randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy, safety, and pharmacodynamic activity of brelgometon (搜索) in people with ALS.
Dr. San Martin emphasized the integration of biomarkers into the trial framework: "Our Phase 1 and preclinical data provide a compelling rationale for advancing brelgometon (搜索) into Phase 2. Our Phase 2 design reflects both the translational science and the realities of ALS by integrating validated biomarkers such as neurofilament light chain (搜索) alongside clinically meaningful endpoints."
He further underscored the differentiation of the program: "We believe brelgometon (搜索) represents a meaningful opportunity to potentially help address the significant unmet need in ALS, with a neuroprotective profile and translational rationale that distinguish it within the ALS development landscape."
ALS Disease Context
ALS is a progressive, fatal neurodegenerative disease characterized by the degeneration of motor neurons in the brain and spinal cord, leading to loss of voluntary muscle control. Patients typically experience progressive muscle weakness, paralysis, and respiratory failure, with a median survival of two to five years from symptom onset. The disease remains one of high unmet medical need, with limited therapeutic options that modestly slow disease progression. Both sporadic and familial forms of ALS present significant challenges for drug development given the heterogeneity of disease presentation and course.