Lead Candidate

Our lead candidate NPT088 is a fusion protein combining GAIM with a human immunoglobulin Fc region. Each molecule of NPT088 displays two GAIM moieties. NPT088 is a New Molecular Entity (NME) and a first-in-class drug candidate.

     Ig-GAIM Fusion Candidate NPT088

GAIM

NPT088 binds with high affinity and specificity to aggregates of misfolded proteins, including those that form the canonical amyloid motif, which is implicated in the pathology of many serious diseases such as AD and PD. NPT088 recognizes the special conformation characteristic of amyloids, irrespective of the specific protein sequence. This enables GAIM to target all such misfolded pathological protein assemblies that lead to amyloid formation.

NPT088 disrupts all types of amyloid, including amyloid-beta and tau aggregates, the defining pathological features of Alzheimer’s disease, and alpha-synuclein aggregates, which are a key characteristic of Parkinson’s disease, and also aggregates of prion proteins, which characterize Creutzfeld-Jacob’s disease. In transgenic animal models of Alzheimer’s and Parkinson’s diseases, chronic treatment with NPT088 strongly reduces the accumulation of these aggregates, and this effect is associated with significant behavioral improvements of the animals.

                                       Click to expand preclinical development data

GAIM Treatment Reduces Pre-Existing Brain Deposits in AD Mouse Models

GAIM Treatment Reduces Pre-Existing Brain Deposits in AD Mouse Models

GAIM Treatment Reduces Pre-Existing Brain Deposits in PD Mouse Models

GAIM Treatment Reduces Pre-Existing Brain Deposits in PD Mouse Models

 

 

 

 

 

 

 

 

NPT088 has an excellent pharmacological profile with antibody-like duration and distribution in animals. It has shown no adverse effects in preclinical studies.