Leal Therapeutics has dosed the first participant in NeurALS, a Phase 1/2 first-in-human, randomized, blinded, placebo-controlled trial (LTX-002-101; NCT07660614) evaluating LTX-002 in adults with genetic or sporadic amyotrophic lateral sclerosis.
LTX-002 is an investigational, intrathecally delivered antisense oligonucleotide that targets SPTLC1 and is designed to restore healthy sphingolipid levels in the central nervous system, according to the company.
The study will assess safety, tolerability and pharmacokinetics, with exploratory endpoints including cerebrospinal fluid SPTLC1 protein and sphingolipid levels to validate target engagement, plasma neurofilament light chain as a marker of disease progression, and clinical measures such as ALSFRS-R, pulmonary vital capacity and muscle strength.
The company describes LTX-002 as reducing excessive serine palmitoyltransferase activity that the company says leads to ceramide and sphingolipid buildup implicated in motor neuron toxicity in genetic and sporadic ALS, and the company claims SPT inhibition has been protective in preclinical models.
Leal Therapeutics is a clinical-stage biopharmaceutical company developing brain-penetrant neuro-metabolic therapies.
