Pipeline

The Alectos pipeline is focused on creating new treatments for patients living with chronic neurodegenerative diseases such as Parkinson’s and Alzheimer’s diseases, as well as those with acute neurological disorders such as ischemic stroke and traumatic brain injury. Our preclinical programs aim to identify and develop novel small molecules as potential disease-modifying therapies.

Glucocerebrosidase 2 (GBA2) inhibition has emerged as a novel therapeutic strategy to enhance lysosomal function in neurodegenerative diseases. In the Alectos GBA2 program, we have identified molecules that possess drug-like properties including good potency, selectivity, pharmacokinetics, and CNS penetrance. Our lead compound, AL01811, is a selective GBA2 inhibitor with first-in-class potential as an oral disease-modifying treatment for patients with Parkinson’s disease. We are also exploring the therapeutic potential of GBA2 inhibitors in Niemann-Pick type C and Batten diseases.

Inhibition of O-GlcNAcase (OGA) is a promising approach for the treatment of neurodegenerative diseases, including Alzheimer’s and Parkinson’s disease, as well as acute conditions including ischemic stroke and traumatic brain injury.  Alectos previously partnered with Merck to advance the first clinical OGA inhibitor and OGA PET ligand to phase 1 studies, where they were found to be safe and well-tolerated.  Alectos has repatriated this program and we are focused on advancing a novel OGA development candidate, enabled by our OGA PET ligand to measure target engagement in the clinic.  The Alectos OGA program builds on our deep knowledge and scientific leadership in this area.  We welcome partnership inquiries regarding our OGA inhibitor program and our advanced preclinical development candidates.