Leal Therapeutics Raises $30M for CNS Drug Development

Leal Therapeutics has successfully closed an additional $30 million tranche of its Series A financing, bolstering its efforts to bring novel neuro-metabolic treatments to market. This significant capital infusion, which notably includes new backing from pharmaceutical giant Eli Lilly and Company, alongside a robust syndicate of existing investors, will propel two key clinical programs forward.

The newly acquired funds are earmarked to advance LTX-001, a promising candidate for schizophrenia, through an initial readout of its Phase 1b/2a clinical trial. Simultaneously, the financing will support the progression of LTX-002, an investigational therapy for amyotrophic lateral sclerosis (ALS), through further dosing cohorts in its ongoing Phase 1/2 study. The company, headquartered in Worcester, Massachusetts, is focused on developing first-in-class therapeutics for high-need central nervous system disorders.

LTX-001, a brain-penetrant oral inhibitor targeting GLS1, has already demonstrated a favorable safety and tolerability profile in earlier Phase 1 studies, including dose-dependent central nervous system engagement. The initiation of the Phase 1b/2a trial in adults diagnosed with schizophrenia marks a critical step, with initial data anticipated by the close of the year. This trial, identified by clinicaltrials.gov identifier NCT07734493, aims to assess both the safety and efficacy of LTX-001 in this patient population.

Complementing the schizophrenia program, LTX-002 is an antisense oligonucleotide delivered intrathecally. Its mechanism of action is designed to normalize sphingolipid levels within the central nervous system by targeting SPTLC1. This therapy is currently being evaluated in the NeurALS Phase 1/2 trial (NCT07660614) for adults suffering from ALS, a progressive neurodegenerative disease with limited treatment options. The market for neurodegenerative disease treatments is substantial and growing, driven by an aging global population and increased understanding of disease mechanisms.

The expanded investor base, now featuring Eli Lilly and Company, underscores the potential perceived in Leal's therapeutic approach. The round also saw continued commitment from prominent venture capital firms including OrbiMed, Newpath Partners, Euclidean Capital, SV Health Investors' Dementia Discovery Fund (DDF), Chugai Venture Fund, Alexandria Venture Investments, and PhiFund. This diverse group of investors provides not only capital but also strategic expertise in drug development and commercialization.

Dr. Asa Abeliovich, founder and CEO of Leal Therapeutics, expressed enthusiasm for the expanded investor syndicate, highlighting the crucial role this funding plays. "This capital infusion will empower us to achieve significant clinical milestones for both LTX-001 in schizophrenia and LTX-002 in ALS," stated Dr. Abeliovich. "It also enables us to advance our preclinical pipeline, paving the way for future clinical development and reinforcing our mission to address unmet needs in CNS disorders." The company's strategy leverages human genetics and functional genomics, alongside a proprietary CNS delivery platform, to develop both small molecule and nucleic acid therapeutics.