Leal Therapeutics Raises $30 Million in Series A Financing to Advance CNS Drug Programs

Lilly Joins Leal's Investor Group

Leal Therapeutics, a clinical-stage biopharmaceutical company developing therapies for central nervous system disorders, has raised another $30 million in its Series A financing round.

The second closing brings new investor Eli Lilly and Company into the company alongside its existing group of investors.

The investor group includes OrbiMed, Newpath Partners, Euclidean Capital, SV Health Investors' Dementia Discovery Fund, Chugai Venture Fund, Alexandria Venture Investments and PhiFund.

The new funding will be used mainly to advance two clinical programs, LTX-001 for schizophrenia and LTX-002 for amyotrophic lateral sclerosis, also known as ALS.

Funding Will Support Two Clinical Programs

Leal plans to use the financing to move LTX-001 toward its initial clinical readout in schizophrenia.

The company has also planned additional dosing cohorts for LTX-002, which is being evaluated in an ongoing Phase 1/2 study in adults with ALS.

The financing is expected to provide support through several upcoming clinical milestones while also allowing Leal to continue advancing its earlier-stage pipeline.

LTX-001 Enters Phase 1b/2a Study in Schizophrenia

LTX-001 is Leal's lead drug candidate for schizophrenia.

It is an oral, brain-penetrant inhibitor of glutaminase 1, or GLS1. The company describes it as a first-in-class approach designed to target the connection between brain biology and metabolism.

Before starting the current study, LTX-001 completed single ascending dose and multiple ascending dose Phase 1 trials.

According to Leal, those studies showed a favorable safety and tolerability profile along with dose-dependent target engagement in the central nervous system.

The company has now started a Phase 1b/2a clinical trial, registered as NCT07734493, in adults with schizophrenia.

Leal expects to report initial data from the study by the end of 2026.

What Leal Is Looking for With LTX-001

The Phase 1b/2a study is an important step because it moves LTX-001 into patients with schizophrenia after the earlier Phase 1 testing established initial safety and pharmacological activity.

The company will use the study to gather additional information about the treatment and its potential effects in people living with schizophrenia.

The upcoming readout is expected to provide the first clinical data from this stage of development.

LTX-002 Advances in ALS

The second major program supported by the financing is LTX-002.

Unlike LTX-001, LTX-002 is an antisense oligonucleotide delivered directly into the cerebrospinal fluid through intrathecal administration.

The treatment is designed to target SPTLC1, a gene involved in sphingolipid production.

Leal is developing LTX-002 with the goal of restoring healthier sphingolipid levels in the central nervous system.

The candidate is being evaluated in NeurALS, a Phase 1/2 clinical trial registered as NCT07660614 in adults with ALS.

The new funding will allow Leal to continue the study through additional dosing cohorts.

Why Leal Is Targeting Neuro-Metabolic Disorders

Leal focuses on what it describes as the intersection between neurological and metabolic biology.

The company's approach is based on the idea that metabolic processes can play an important role in diseases affecting the central nervous system.

Rather than focusing only on traditional neurological targets, Leal uses human genetics, functional genomics and biomarker analysis to identify potential therapeutic opportunities.

The company is applying this approach across both small-molecule and nucleic-acid-based medicines.

Lilly Adds to Leal's Investor Base

The participation of Eli Lilly is a notable addition to Leal's investor group.

Lilly joins a syndicate that already includes investors with experience in biotechnology, healthcare and life sciences.

For Leal, the financing provides additional resources to advance its clinical programs while continuing work on earlier-stage candidates.

Asa Abeliovich, M.D., Ph.D., founder and chief executive officer of Leal Therapeutics, said the financing will support important clinical milestones for both LTX-001 and LTX-002.

He also said the company plans to use the funding to continue moving its preclinical pipeline toward clinical development.

Leal Is Also Building a CNS Delivery Platform

Beyond its current drug candidates, Leal is working on a next-generation central nervous system delivery platform.

The technology uses an antibody-like shuttle approach that is intended to help therapeutics administered outside the brain reach the central nervous system.

This could potentially expand the range of medicines that can be delivered to the brain without requiring direct administration into the central nervous system.

The platform is part of Leal's broader strategy to develop new ways of treating CNS disorders.

About Leal Therapeutics

Leal Therapeutics was launched in 2021 and is headquartered in Worcester, Massachusetts.

The company develops neuro-metabolic therapies for patients with serious central nervous system disorders where treatment options remain limited.

Its team has expertise in CNS drug development, human genetics, functional genomics and biomarker analysis.

The company can develop and produce both small-molecule and nucleic acid-based CNS therapeutics.

With the latest $30 million Series A financing, Leal is focusing its near-term development efforts on the clinical progress of LTX-001 in schizophrenia and LTX-002 in ALS, while continuing to build its broader neuro-metabolic pipeline.