Leal raises $45M to advance LTX-002 to clinical trials for ALS
Trials for lead candidates expected to start early next year
Leal Therapeutics has raised $45 million to continue advancing its pipeline of drugs for diseases of the brain and spinal cord, including a promising new therapy for amyotrophic lateral sclerosis (ALS).
The financing round, which follows an earlier $39 million investment, will help Leal move its two lead assets — LTX-002 for ALS and LTX-001 for schizophrenia — into clinical trials, while continuing to expand its pipeline and advancing early-stage drugs into preclinical studies.
The company expects both lead candidates to enter first-in-human clinical trials early next year, following regulatory submissions by year’s end.
“This investment allows us to progress our lead programs in ALS and schizophrenia towards the clinic while also continuing our earlier-stage pipeline and platform development,” Asa Abeliovich, MD, PhD, founder and CEO of Leal, said in a company press release.
Leal’s lead candidate for ALS, LTX-002, is designed to regulate the metabolism of fats, which is dysfunctional in ALS and other neurological conditions. It belongs to a class of therapies called antisense oligonucleotides, which are small RNA or DNA molecules that alter protein production.
Supporting clinical trials for ALS
LTX-002 binds to an RNA molecule made from the SPLTC1 gene that’s used to produce part of a protein called serine palmitoyltransferase, or SPT. This protein is critical for the production of a class of lipids called sphingolipids, which help to coordinate a variety of biological processes but can also contribute to cell death when produced in excess.
Mutations in SPLTC1, which result in the production of toxic forms of sphingolipids, have been described in people with ALS, including in a new type of juvenile ALS.
By reducing the amount of functional SPT available to produce sphingolipids, the experimental therapy is expected to lower the accumulation of toxic sphingolipids in patients and potentially slow disease progression.
Leal, founded in 2021, aims to develop precision treatments targeting specific brain and nervous system pathways to address complex neurological and mental health disorders.
The recent investment round was led by Newpath Partners. It included existing investors OrbiMed, Euclidean Capital and PhiFund, as well as new investors Chugai Venture Fund and Alexandria Venture Investments.
“We are excited to support Leal as they progress their lead programs in ALS and schizophrenia, two [neurological] disorders with major unmet medical needs, towards the clinic,” said Thomas Cahill, MD, PhD, founder of Newpath Partners, who joined Leal’s board.
Abeliovich said the company’s investors “share our vision for better therapeutics for people with neurodegenerative or neuropsychiatric disorders.”
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