Leal Therapeutics Secures $30 Million Series A Extension and Initiates Phase 1b/2a Trial of LTX-001 in Schizophrenia
核心洞察
Leal Therapeutics (搜索) announced a $30 million second close of its Series A financing, adding Eli Lilly and Company as a new investor alongside existing backers including OrbiMed and Newpath Partners.
Proceeds will advance LTX-001 through an initial readout of its Phase 1b/2a trial in schizophrenia (搜索), with data expected by year-end, and progress LTX-002 through additional dosing cohorts in ALS.
Leal initiated the Phase 1b/2a trial (NCT07734493) of LTX-001, a first-in-class brain-penetrant oral GLS1 (搜索) inhibitor, in adults with schizophrenia (搜索).
Leal Therapeutics (搜索), Inc., a clinical-stage biopharmaceutical company developing first-in-class neuro-metabolic therapeutics, announced a second close of $30 million of its Series A financing, alongside the initiation of a Phase 1b/2a clinical trial of LTX-001 in adults with schizophrenia (搜索). The financing 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 the Series A financing will be used to advance LTX-001 through an initial readout of its Phase 1b/2a clinical trial in schizophrenia (搜索) patients, as well as to progress LTX-002 through additional dosing cohorts in an ongoing Phase 1/2 clinical trial in amyotrophic lateral sclerosis (搜索) (ALS).
LTX-001: A First-in-Class GLS1 Inhibitor for Schizophrenia
LTX-001 is a first-in-class, brain-penetrant oral GLS1 (搜索) inhibitor. The compound has completed single ascending dose (SAD) and multiple ascending dose (MAD) Phase 1 trials, demonstrating a favorable safety and tolerability profile as well as dose-dependent CNS target engagement. Leal announced the initiation of the Phase 1b/2a trial (NCT07734493) in adults with schizophrenia (搜索), with initial data expected by year-end.
LTX-002: An Antisense Oligonucleotide Approach to ALS
LTX-002 is an intrathecally delivered antisense oligonucleotide designed to restore healthy sphingolipid levels in the central nervous system (CNS) by targeting SPTLC1 (搜索). The therapy is being investigated in NeurALS, a Phase 1/2 trial (NCT07660614) in adults with ALS.
LTX-002 targets SPTLC1 (搜索), a core subunit of serine palmitoyltransferase (SPT). Excessive SPT activity results in the buildup of ceramides and sphingolipids, which are implicated in motor neuron toxicity and in both genetic and sporadic forms of ALS. By reducing SPT activity, LTX-002 is designed to restore healthy lipid balance in the CNS. SPT inhibition has been protective in preclinical models of ALS and related disorders.
The NeurALS trial (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 (搜索) 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.
Leadership Perspective
"We are very excited to partner with our top-tier syndicate of investors which now includes Lilly," said Asa Abeliovich, M.D., Ph.D., founder and chief executive officer of Leal. "This funding will support us through key clinical milestones for LTX-001 in schizophrenia (搜索), including initial data from the now ongoing Phase 1b/2a trial, and LTX-002 in ALS, while also advancing our preclinical pipeline towards clinical development."
Commenting on the ALS program, Dr. Abeliovich noted, "ALS patients have too few options, and recent science points clearly to dysregulated lipid metabolism as a driver of degeneration. Dosing our first patient with LTX-002 is the first real test of this approach in people and a meaningful milestone for both Leal and the ALS community."
About Leal Therapeutics
Leal Therapeutics (搜索) is a biotechnology company dedicated to developing novel neuro-metabolic therapeutics for patients with high-need central nervous system disorders. Launched in 2021 and headquartered in Worcester, Massachusetts, Leal's lead programs address the critical intersection between CNS and metabolic disorders. The company has particular expertise in CNS therapeutic development, as well as leveraging human genetics, functional genomics, and biomarker analyses. Leal has the technological capability to develop and produce small molecule and nucleic acid CNS therapeutics, and is also developing a next-generation CNS delivery platform utilizing antibody-like shuttle technology to enable the transport of peripherally administered therapeutics to the brain.