Pipeline

Asceneuron is developing new chemical entities designed to halt abnormal protein aggregation in the brain, which is a unifying feature of all neurodegenerative diseases.

Asceneuron’s clinical-stage orally administered small molecules are fully brain penetrant in human beings and have demonstrated preclinical potential to improve both tau and ⍺-synuclein pathology. By treating both pathologies, Asceneuron aims to address the high unmet medical need for a safe and effective oral disease modifying therapy in both Alzheimer’s and Parkinson’s disease.

About ASN90

ASN90 (formerly labelled as ASN120290) is an in-house developed OGA inhibitor that has been licensed to Ferrer for the treatment of an orphan tau-related disease with very high unmet medical need, progressive supranuclear palsy (PSP). ASN90 has received orphan drug designations from the US FDA and the European EMA for the treatment of PSP. The molecule has completed three clinical studies in healthy volunteers including a randomized, double-blind, placebo-controlled phase I study to assess the safety and tolerability of single and multiple doses in healthy young and elderly volunteers and a human positron emission tomography (PET) CNS target engagement study. Asceneuron is exploring further potential indications for ASN90.

About ASN51

Asceneuron’s next generation OGA inhibitor ASN51 has been awarded USD 2.2 million from the Alzheimer’s Drug Discovery Foundation for a first in human Phase I study. ASN51 has completed three Phase I clinical studies in healthy volunteers and has defined single and multiple dose target engagement using O-GlcNAcase PET and a pharmacodynamic peripheral blood mononuclear cell response assay to quantify protein O-GlcNAcylation. Data support daily dosing of ASN51 and Alzheimer’s disease as a first indication with planned Phase 2 entry in the second half of 2024.