Pipeline

Our novel pipeline of therapies for neurological and psychiatric disease is based on decades of research focused on cognitive neuroscience and the neurobiology of the aging brain.

Research at Johns Hopkins and leading research centers around the world have demonstrated that overactivity in key brain circuits contributes to cognitive impairment and drives neurodegeneration if not controlled.

Hippocampal overactivity is a shared driver of memory loss and psychosis. This pathological state appears across several conditions in adult and elderly patients, including Parkinson’s disease psychosis (PDP), mild cognitive impairment (MCI) in Alzheimer’s disease, cognitive impairment associated with schizophrenia (CIAS), and even elderly individuals with subjective cognitive decline (SCD). This excess hippocampal activity degrades the accuracy of memory recall, and can blur the line between what a person remembers and what they imagine. It is both a biomarker and a driver of cognitive impairment and psychosis.

AgeneBio is advancing two programs against this pathological state.

The first is the company’s portfolio of α5 GABA-A receptor positive allosteric modulators (PAMs), selective over α1 GABA-A receptors. It is the largest and most advanced portfolio of its kind with several differentiated molecular scaffolds. It is led by AGB-501, a first-in-class, Phase 2-ready compound, with follow-on molecules designed for even greater selectivity.

The second, AGB-101, is an SV2A modulator being developed through the 505(b)(2) regulatory pathway. AGB-101 is being evaluated for the treatment of hippocampal overactivity and for slowing the progression to Alzheimer’s dementia in MCI due to AD.

Selective α5 GABAA PAM program

AGB-501 Discovery through Phase 2-ready
AGB-4203 Discovery to IND-enabling
Follow-on compounds Discovery

Hippocampal overactivity: AGB-101

AGB-101 (MCI due to AD) Phase 2b (completed)

These investigational compounds are not approved by the FDA. The safety and efficacy have not been established.