Pioneering neuro-metabolic therapies for high-need CNS disorders

  • Two lead programs: an ASO for sporadic and genetic ALS patients targeting lipid biology, and an oral small molecule for schizophrenia targeting glutamate biology
  • Lead programs on track for INDs this year, with clinical data in 2025
  • Additional pipeline programs in Alzheimer’s disease, epilepsy, and depression

Pipeline

Program

Target / pathway

Indication

Modality

Preclinical

DC

IND-Enabling

Clinical

LTX-001

GLS1 / Excessive CNS glutamate

Schizophrenia, BD, MDD, ALS

Small molecule

LTX-002

SPTLC1 / Excessive CNS sphingolipids

ALS

ASO

LTX-007

SPT / Excessive CNS sphingolipids

Alzheimer’s Disease, rare sphingolipidoses, obesity

Small molecule

LTX-003, LTX-004

Multiple

Alzheimer’s Disease

ASO

Pipeline

Program: LTX-001

Modality:  Small molecule

Target:  GLS1 / Excessive CNS glutamate

Indication: Schizophrenia, BD, MDD, ALS

Pre-clinical

DC

IND-enabling

Clinical

Program: LTX-002

Modality:  ASO

Target: SPTLC1 / Excessive CNS sphingolipids 

Indication: ALS

Pre-clinical

DC

IND-enabling

Clinical

Program: LTX-007

Modality:  Small molecule

Target: SPT / Excessive CNS sphingolipids 

Indication: Alzheimer’s Disease, rare sphingolipidoses, obesity

Pre-clinical

DC

IND-enabling

Clinical

Program: LTX-003, LTX-004

Modality:  ASO

Target:  Multiple

Indication: Alzheimer’s Disease

Pre-clinical

DC

IND-enabling

Clinical

Developing novel precision CNS therapeutics that target key pathogenic pathways