Leal Therapeutics has successfully raised $45 million to propel its drug development efforts, with a primary focus on advancing LTX-002, a novel therapy for amyotrophic lateral sclerosis (ALS), into clinical trials. This funding round, following a previous $39 million investment, will support the progression of LTX-002 and LTX-001 (for schizophrenia) into first-in-human studies, anticipated to commence early next year.
Targeting Sphingolipid Metabolism in ALS
LTX-002 is an antisense oligonucleotide designed to modulate fat metabolism, which is often dysfunctional in ALS and other neurological conditions. Specifically, LTX-002 binds to RNA derived from the SPLTC1 gene, thereby reducing the production of serine palmitoyltransferase (SPT). SPT is a crucial enzyme involved in the synthesis of sphingolipids, a class of lipids that, when produced in excess, can contribute to cell death.
Mutations in SPLTC1 have been identified in ALS patients, leading to the production of toxic sphingolipids. By reducing functional SPT, LTX-002 aims to decrease the accumulation of these toxic sphingolipids, potentially slowing disease progression in ALS patients.
Leal's Broader Pipeline and Investment
Founded in 2021, Leal Therapeutics is dedicated to developing precision treatments that target specific pathways in the brain and nervous system to address complex neurological and mental health disorders. The recent investment round was spearheaded by Newpath Partners, with participation from existing investors such as OrbiMed, Euclidean Capital, and PhiFund, as well as new investors including Chugai Venture Fund and Alexandria Venture Investments.
"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," said Asa Abeliovich, MD, PhD, founder and CEO of Leal.
Thomas Cahill, MD, PhD, founder of Newpath Partners, emphasized the importance of supporting Leal's work in addressing neurological disorders with significant unmet medical needs, stating, "We are excited to support Leal as they progress their lead programs in ALS and schizophrenia...towards the clinic."
