Leal Therapeutics secures $30 million in Series A financing to advance LTX-001 in schizophrenia and LTX-002 in an ongoing ALS Phase 1/2 trial.
Written By: Samiksha Jadhav, BPharm
Reviewed By: Pharmacally Editorial Team
Leal Therapeutics has secured an additional $30 million in a second closing of its Series A financing, bringing total proceeds to advance LTX-001 into its first clinical efficacy readout in schizophrenia and expand development of LTX-002 in amyotrophic lateral sclerosis (ALS). The extension brings Eli Lilly and Company into the investor syndicate alongside OrbiMed, Newpath Partners, Euclidean Capital, SV Health Investors’ Dementia Discovery Fund, Chugai Venture Fund, Alexandria Venture Investments, and PhiFund.
LTX-001 Enters Phase 1b/2a Schizophrenia Trial
The company has initiated a Phase 1b/2a study of LTX-001 (NCT07734493) in adults experiencing an acute exacerbation of schizophrenia, with initial data expected by the end of 2026. The trial follows single ascending dose (SAD) and multiple ascending dose (MAD) Phase 1 studies that demonstrated favorable safety, tolerability, and dose-dependent central nervous system (CNS) target engagement.
Approximately 90 participants will receive oral dosing regimens or placebo, with safety, tolerability, and early efficacy serving as primary evaluation metrics. LTX-001 is a brain-penetrant oral small-molecule inhibitor of glutaminase 1 (GLS1)—the enzyme responsible for converting glutamine to glutamate. Excessive glutamatergic activity drives neuronal dysfunction, inflammation, oxidative stress, and mitochondrial injury. By inhibiting GLS1, LTX-001 aims to reduce excess glutamate production and restore CNS metabolic balance.
Unlike standard schizophrenia treatments targeting dopaminergic or muscarinic pathways—which can be constrained by metabolic, sedative, or motor side effects—LTX-001 offers a distinct biological mechanism with potential utility across broader symptom domains.
LTX-002 Expands Phase 1/2 Development in ALS
Proceeds will also fund additional dosing cohorts for LTX-002 in NeurALS (NCT07660614), an ongoing Phase 1/2 study in adults with ALS. LTX-002 is an intrathecally administered antisense oligonucleotide (ASO) targeting SPTLC1, a key subunit of serine palmitoyltransferase involved in sphingolipid biosynthesis. The program aims to normalize sphingolipid levels within the CNS by dampening pathway hyperactivity.
The randomized, placebo-controlled NeurALS study (estimated enrollment: 56 participants) is evaluating safety, tolerability, pharmacokinetics, pharmacodynamics, and biomarkers—including SPTLC1, sphingolipids, neurofilament light chain (NfL), and clinical measures of motor, respiratory, and bulbar function.
“This funding will support us through key clinical milestones for LTX-001 in schizophrenia, including initial data from the now ongoing Phase 1b/2a trial, and LTX-002 in ALS,” said Asa Abeliovich, M.D., Ph.D., founder and chief executive officer of Leal Therapeutics.
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About the Writer
Samiksha Vikram Jadhav (LinkedIn) is a B. Pharm graduate with a strong academic foundation in pharmaceutical sciences, pharmacology, and drug development. She specializes in pharma market research, with a focused interest in mergers and acquisitions, strategic partnerships, and global pharma and biotech deals. Her work centers on analyzing industry transactions, market positioning, and business strategies, translating complex developments into clear, accurate, and insightful scientific and commercial reporting.