LTX-001 / Leal Therapeutics - LARVOL DELTA

Leal Therapeutics…announced a second close of $30 million of its Series A financing (PRNewswire) - "The round includes new investor Eli Lilly and Company (Lilly), in addition to existing investors OrbiMed, Newpath Partners, Euclidean Capital, SV Health Investors' Dementia Discovery Fund (DDF), Chugai Venture Fund, Alexandria Venture Investments, and PhiFund. Proceeds from the second closing of Series A financing will be used to advance LTX-001 through an initial readout of its Phase 1b/2a clinical trial in schizophrenia patients, as well as progress LTX-002 through additional dosing cohorts in an ongoing Phase 1/2 clinical trial in amyotrophic lateral sclerosis (ALS)....Leal today also announces the initiation of the Phase 1b/2a trial (NCT07734493) in adults with schizophrenia, with initial data expected by year-end."

Financing • P1/2 data • Trial status • Amyotrophic Lateral Sclerosis • Schizophrenia

A Study of LTX-001 In Adult Participants With Acute Exacerbation of Schizophrenia (clinicaltrials.gov) - P1 | N=90 | Recruiting | Sponsor: Leal Therapeutics, Inc | Not yet recruiting ➔ Recruiting

Enrollment open • Monotherapy • CNS Disorders • Psychiatry • Schizophrenia

A Study of LTX-001 In Adult Participants With Acute Exacerbation of Schizophrenia (clinicaltrials.gov) - P1 | N=90 | Not yet recruiting | Sponsor: Leal Therapeutics, Inc

Monotherapy • New P1 trial • CNS Disorders • Psychiatry • Schizophrenia

Clinical Development Of LTX-001, A Small-Molecule GLS1 Inhibitor For The Treatment Of ALS (ENCALS 2026) - "Riluzole targets glutamate-associated toxicity through decreased glutamate release.LTX-001 is an investigational compound that inhibits glutaminase 1 (GLS1), a mitochondrial enzyme which converts glutamine to glutamate and ammonia, and consequently reduces glutamate levels in the CNS. Exploratory biomarker and efficacy data will also be collected, including: 1) Plasma glutamate and glutamine 2) Plasma levels of neurofilament light chain (NfL) 3) Selected clinical endpoints.Conclusion LTX-001 is a novel investigational oral small molecule GLS1 inhibitor. The proposed clinical trial is designed to provide safety and exploratory biomarker/efficacy data in people living with ALS."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders • Inflammation • NEFL • Plasma NfL

Development of LTX-001 a novel oral small molecule for the treatment of schizophrenia (APA 2026) - "These results demonstrate that LTX-001, a novel oral small molecule, was able to successfully achieve brain penetration in animal models and support its potential as a therapeutic option to treat schizophrenia by targeting the glutamatergic pathway and impacting both positive and negative symptoms of schizophrenia."

CNS Disorders • Inflammation • Psychiatry • Schizophrenia

Clinical development of LTX-001 a novel GLS1 inhibitor for the treatment of schizophrenia (APA 2026) - "Pharmacological agents known to induce acute psychosis in humans (e.g., ketamine) also increase brain glutamate regionally, as visualized by 1H-MRS (Javitt et al., 2018b). Safety, pharmacokinetic, and pharmacodynamic data from this FIH study support the further development of LTX-001 for the treatment of positive, negative and cognitive symptoms of individuals with schizophrenia."

Clinical • First-in-human • CNS Disorders • Mental Retardation • Psychiatry • Schizophrenia

Development of LTX-001, a Novel Oral Small Molecule for the Treatment of Schizophrenia (WFSBP 2025) - "In addition to treating positive symptoms of schizophrenia, modulation of the glutamatergic pathway has the potential to ameliorate negative and cognitive symptoms, while mitigating dopamine-related adverse effects. We have developed LTX-001, an investigational oral small molecule targeting glutaminase (GLS1), the rate-limiting enzyme for glutamate biosynthesis in the central nervous system (CNS), for the treatment of schizophrenia."

CNS Disorders • Psychiatry • Schizophrenia

Leal Therapeutics Announces $45 Million Financing to Progress Novel Therapies for Patients with Major Neurodegenerative and Neuropsychiatric Disorders (PRNewswire) - "The proceeds will be used to advance Leal's pipeline of antisense oligonucleotide (ASO) and small molecule therapeutics for patients with CNS disorders. Leal's lead programs are LTX-002, an ASO for patients with genetic or sporadic amyotrophic lateral sclerosis (ALS), and LTX-001, a small molecule for patients with schizophrenia. The company plans to submit Investigational New Drug (IND) applications for LTX-002 and LTX-001 by the end of 2024 and commence first-in-human clinical trials in early 2025."

Financing • IND • Amyotrophic Lateral Sclerosis • Schizophrenia