Leal Therapeutics Doses First Patient in NeurALS Phase 1/2 Trial of LTX-002 for Amyotrophic Lateral Sclerosis (ALS)

LTX-002 is a first-in-class antisense oligonucleotide targeting SPTLC1, designed to restore lipid metabolism in the CNS, a novel approach with the potential to address both sporadic and genetic forms of ALS

WORCESTER, Mass., June 23, 2026 /PRNewswire/ -- Leal Therapeutics, Inc. (Leal), a clinical-stage biopharmaceutical company developing first-in-class neuro-metabolic therapeutics, today announced that the first participant has been dosed in NeurALS, a Phase 1/2 trial of LTX-002 in adults with amyotrophic lateral sclerosis (ALS). LTX-002 is an investigational, intrathecally delivered antisense oligonucleotide designed to restore healthy sphingolipid levels in the central nervous system (CNS) by targeting SPTLC1.

A lipid-metabolism approach to ALS

LTX-002 targets SPTLC1, a core subunit of serine palmitoyltransferase (SPT). Excessive SPT activity results in the buildup of ceramides and sphingolipids, which are implicated in motor neuron toxicity and in both genetic and sporadic forms of ALS. By reducing SPT activity, LTX-002 is designed to restore healthy lipid balance in the CNS. SPT inhibition has been protective in preclinical models of ALS and related disorders.

About the NeurALS trial

NeurALS (LTX-002-101; NCT07660614) is a first-in-human, randomized, blinded, placebo-controlled study in adults with genetic or sporadic ALS, evaluating the safety, tolerability, and pharmacokinetics of LTX-002. Exploratory endpoints include CSF SPTLC1 protein and sphingolipid levels (validating target engagement), plasma neurofilament light chain (NfL; a marker of disease progression), and clinical measures including the ALSFRS-R rating scale, pulmonary vital capacity, and muscle strength.

"ALS patients have too few options, and recent science points clearly to dysregulated lipid metabolism as a driver of degeneration," said Dr. Asa Abeliovich, CEO and Founder of Leal. "Dosing our first patient with LTX-002 is the first real test of this approach in people and a meaningful milestone for both Leal and the ALS community."

About Leal Therapeutics

Leal Therapeutics is a clinical-stage biopharmaceutical company developing first-in-class, brain-penetrant therapies for high-need CNS and metabolic disorders. Leal integrates human genetics, human biomarker data, human physiology, and preclinical model data to identify validated disease targets and develops brain-penetrating therapies to maximize target engagement. Learn more at lealtx.com.

Media Contact: Tehya Frank | [email protected] | 347-640-1334

SOURCE Leal Therapeutics