Leal Therapeutics Secures $45 Million to Advance LTX-002 for ALS into Clinical Trials

• Leal Therapeutics has raised $45 million to advance its pipeline, including LTX-002 for amyotrophic lateral sclerosis (ALS), into clinical trials.
• LTX-002, an antisense oligonucleotide, aims to regulate fat metabolism by targeting SPLTC1 and reducing toxic sphingolipid accumulation in ALS patients.
• Clinical trials for LTX-002 are expected to begin early next year, following regulatory submissions by the end of the year.
• The funding round, led by Newpath Partners, will also support the advancement of LTX-001 for schizophrenia and expand Leal's early-stage drug pipeline.

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."