← Pipeline
PreclinicalPreclinical programmePositive signal

Lead optimisation

CN045

Small-molecule OPC differentiation lead

Practical availability: Preclinical — not available as a clinical intervention

Compound / intervention
Small-molecule OPC differentiation lead
Mechanism
Small-molecule promotion of OPC differentiation
Development stage
Lead optimisation
Status
Peer-reviewed preclinical efficacy in human OPCs in vitro and mouse demyelination
Evidence level
Preclinical only • no proven clinical benefit in MS
Next substantive checkpoint

Medicinal-chemistry optimisation, PK/toxicology and selection of a clinical candidate

Peer-reviewed paperOpen source ↗Last checked: 16 Sep 2026

Related substantive developments

PreclinicalPreclinical programmePositive signal

Preclinical

CN045 promoted human OPC differentiation and remyelination in mice

An npj Drug Discovery paper described CN045, a CNS-penetrant lead that promoted OPC differentiation and myelin-like ensheathment in human cells in vitro and increased remyelination in a mouse demyelination model.

Practical availability: Preclinical — not available as a clinical intervention

What this means clinically

This is an interesting drug-development lead, but it remains preclinical. Pharmacokinetic optimisation, toxicology and then human testing are still required.

Source quality: High for a preclinical finding • peer-reviewed primary paper

Peer-reviewed papernpj Drug Discovery ↗