Leal Therapeutics Advances LTX-001 in Schizophrenia Trial

Worcester, Massachusetts, August 17, 2026

LTX-001 Enters Phase 1b/2a Schizophrenia Trial

LTX-001 is described by Leal as a first-in-class, brain-penetrant, orally administered GLS1 inhibitor being developed as a neuro-metabolic therapeutic. The candidate has already completed single ascending dose and multiple ascending dose Phase 1 studies, where the company reported a favorable safety and tolerability profile along with dose-dependent central nervous system target engagement. Leal has now initiated the Phase 1b/2a clinical trial, registered as NCT07734493, to assess the safety and efficacy of LTX-001 in adults with schizophrenia. The transition into a Phase 1b/2a study represents an important clinical development milestone because the program moves beyond initial Phase 1 evaluation toward a study designed to generate additional information on the candidate’s safety and potential therapeutic activity in patients with schizophrenia. The company expects an initial data readout from the study by the end of 2026.

$30 Million Financing Supports Clinical Pipeline

The $30 million Series A second closing provides funding to advance Leal’s clinical programs through upcoming development milestones. Eli Lilly’s participation adds a major pharmaceutical investor to the company’s existing investor syndicate and supports Leal’s efforts to advance its neuro-metabolic pipeline. The financing will specifically support LTX-001 through the initial readout of its Phase 1b/2a schizophrenia study while also funding additional dosing cohorts for LTX-002 in ALS. The company said the financing will also help advance its preclinical pipeline toward clinical development. For Leal, the investment provides additional resources to progress multiple CNS programs simultaneously while continuing to develop therapeutics based on the intersection between central nervous system biology and metabolic pathways. The combination of financing and clinical-trial initiation gives the company an important development runway as it approaches multiple clinical milestones.

LTX-002 Advances in ALS Development

Alongside LTX-001, Leal is advancing LTX-002, an intrathecally delivered antisense oligonucleotide designed to restore healthy sphingolipid levels in the central nervous system by targeting SPTLC1. The candidate is being investigated in NeurALS, a Phase 1/2 clinical trial identified as NCT07660614 in adults with ALS. Funding from the Series A extension will support additional dosing cohorts in the ongoing study, expanding the company’s clinical development activities beyond schizophrenia. Leal Therapeutics was launched in 2021 and is headquartered in Worcester, Massachusetts, with a focus on developing novel neuro-metabolic therapeutics for high-need CNS disorders. The company combines expertise in CNS therapeutic development with human genetics, functional genomics, and biomarker analysis. It also has capabilities for developing small-molecule and nucleic-acid therapeutics and is working on a next-generation CNS delivery platform using antibody-like shuttle technology. For cGxP.wire readers, the latest announcement is significant because it combines biopharmaceutical financing, clinical-trial initiation, CNS drug development, and innovative therapeutic approaches across schizophrenia and ALS. The initiation of the Phase 1b/2a study of LTX-001 marks the next stage in evaluating a novel neuro-metabolic approach in schizophrenia, while the continued development of LTX-002 broadens Leal’s clinical pipeline in neurological disease.

Source: Leal Therapeutics press relese