LTX-002 / 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

Development Of LTX-002, A Novel ASO, For The Treatment Of ALS (ENCALS 2026) - "In vitro model systems further demonstrated the ability of LTX-002 to reduce multiple classes of sphingolipids. Conclusion These results demonstrate that LTX-002, a novel ASO, could be a viable therapeutic option for the treatment of ALS by reducing SPT enzyme activity and ameliorating sphingolipid dysregulation."

Amyotrophic Lateral Sclerosis • CNS Disorders

Clinical Development Of LTX-002, An ASO For The Treatment Of Sporadic And Familial Forms Of ALS (ENCALS 2026) - "Current therapies afford minimal benefits, with the exception of tofersen, an antisense oligonucleotide (ASO) directed against SOD1. Participants in the study will be eligible to enroll in an open-label extension study of LTX-002Conclusion LTX-002 is an ASO targeting SPTLC1 being developed for the treatment of ALS. This study will provide safety and tolerability data for LTX-002, and will provide exploratory clinical and biomarker data for further development."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders

NeurALS: A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis (clinicaltrials.gov) - P1/2 | N=56 | Recruiting | Sponsor: Leal Therapeutics, Inc

First-in-human • New P1/2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

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

First-in-human • Trial status • Amyotrophic Lateral Sclerosis

LTX-002-101: A Study to Investigate the Safety and Effects of LTX-002 Administered in the Spinal Canal of Participants with Amyotrophic Lateral Sclerosis (clinicaltrialsregister.eu) - P1/2 | N=24 | Not yet recruiting | Sponsor: Leal Therapeutics Inc. | N=10 ➔ 24

Enrollment change • First-in-human • Amyotrophic Lateral Sclerosis • CNS Disorders

LTX-002-101: A Study to Investigate the Safety and Effects of LTX-002 Administered in the Spinal Canal of Participants with Amyotrophic Lateral Sclerosis (clinicaltrialsregister.eu) - P1/2 | N=10 | Not yet recruiting | Sponsor: Leal Therapeutics Inc.

First-in-human • New P1/2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

Clinical development of LTX-002, an ASO for the treatment for ALS (ALS-MND 2025) - "Current therapies afford minimal benefits, with the exception of tofersen, an intrathecally-delivered antisense oligonucleotide (ASO) directed against SOD1. LTX-002 is an ASO targeting SPTLC1 being developed for the treatment of ALS. This study will provide safety and tolerability data for LTX-002, and will provide exploratory clinical and biomarker data for further development."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders

Development of LTX-002, a novel ASO for the treatment of ALS (ALS-MND 2025) - "In vitro model systems further demonstrated the ability of LTX-002 to reduce multiple classes of sphingolipids. These results demonstrate that LTX-002, a novel ASO, could be a viable therapeutic option for the treatment of ALS by reducing SPT enzyme activity and ameliorating sphingolipid dysregulation."

Amyotrophic Lateral Sclerosis • CNS Disorders

Clinical development of LTX-002, an ASO for the treatment for ALS (ALS-MND 2024) - "Currently, available therapies afford minimal benefits, with the exception of tofersen, an intrathecally-delivered antisense oligonucleotide (ASO) directed against the SOD1 gene. LTX-002 is an ASO targeting SPTLC1 that is being developed for the treatment of ALS. The nested SAD and MAD studies will provide safety and tolerability data for LTX-002 and will provide exploratory clinical and biomarker data for further development."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders • SOD1

Development of LTX-002, an ASO for the treatment of ALS (ALS-MND 2024) - "In vitro model systems further demonstrated the ability of LTX-002 to reduce multiple classes of sphingolipids. These results demonstrate that LTX-002, a novel ASO, could be a viable therapeutic option for the treatment of ALS by reducing SPT enzyme activity and ameliorating sphingolipid dysregulation."

Amyotrophic Lateral Sclerosis • CNS Disorders

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