LTX-002 / Leal Therapeutics - LARVOL DELTA

Clinical development of LTX-002, an ASO for the treatment for ALS (ALS-MND 2024) - "Currently, available therapies afford minimal benefits, with the exception of tofersen, an intrathecally-delivered antisense oligonucleotide (ASO) directed against the SOD1 gene. LTX-002 is an ASO targeting SPTLC1 that is being developed for the treatment of ALS. The nested SAD and MAD studies will provide safety and tolerability data for LTX-002 and will provide exploratory clinical and biomarker data for further development."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders • SOD1

Development of LTX-002, an ASO for the treatment of ALS (ALS-MND 2024) - "In vitro model systems further demonstrated the ability of LTX-002 to reduce multiple classes of sphingolipids. These results demonstrate that LTX-002, a novel ASO, could be a viable therapeutic option for the treatment of ALS by reducing SPT enzyme activity and ameliorating sphingolipid dysregulation."

Amyotrophic Lateral Sclerosis • CNS Disorders

Leal Therapeutics Announces $45 Million Financing to Progress Novel Therapies for Patients with Major Neurodegenerative and Neuropsychiatric Disorders (PRNewswire) - "The proceeds will be used to advance Leal's pipeline of antisense oligonucleotide (ASO) and small molecule therapeutics for patients with CNS disorders. Leal's lead programs are LTX-002, an ASO for patients with genetic or sporadic amyotrophic lateral sclerosis (ALS), and LTX-001, a small molecule for patients with schizophrenia. The company plans to submit Investigational New Drug (IND) applications for LTX-002 and LTX-001 by the end of 2024 and commence first-in-human clinical trials in early 2025."

Financing • IND • Amyotrophic Lateral Sclerosis • Schizophrenia