ONESTX-01 / ONESTX - LARVOL DELTA

EFFICACY OF STX64 LINKED TO STEROID SULFATASE INHIBITION IN THE R6/1 MODEL OF HUNTINGTON DISEASE (ADPD 2026) - "STX64 produced behavioral benefits in R6/1 mice consistent with clear inhibition of brain STS. These results support STX64 as a promising therapeutic candidate for Huntington's disease."

Clinical • Cognitive Disorders • Genetic Disorders • Huntington's Disease • Movement Disorders

STX64, A STEROID SULFATASE INHIBITOR, AS A NOVEL DISEASE-MODIFYING CANDIDATE FOR ALZHEIMER'S DISEASE THROUGH CHOLINERGIC MODULATION (ADPD 2026) - "STX64 enhances acetylcholine tone independently of AChE inhibition, rescues cognition in AD-relevant rodent models, and preserves cholinergic neurons in APP/PS1 mice. Cross-species convergence, together with validated biomarkers (brain STS inhibition, plasma steroid ratios), strongly supports the ongoing Phase IIa clinical trial in Alzheimer's disease."

Alzheimer's Disease • CNS Disorders • Cognitive Disorders • CHAT

A STEROID SULFATASE INHIBITOR, ONESTX -01, AS A NOVEL TREATMENT FOR NEURODEGENERATIVE DISEASES: EFFECT IN PROGRESSION IN HUNTINGTON DISEASE MODELS (ADPD 2025) - "Aims: ONESTX-01 (formerly Irosustat) is a small molecule that blocks steroid sulfatase (STS), increasing the ratio of sulfated to free steroids in both animals and humans. Preliminary data suggest ONESTX -1 treatment could be a new approach to reversing or alleviating key symptoms in HD patients. The beneficial effect of ONESTX -01 in proteinopathies may be conserved accross evolutionary distant animal models."

Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Genetic Disorders • Huntington's Disease • Inflammation • Movement Disorders • Proteinopathy