Neurocrine doses first patients in Phase 2 schizophrenia trial of NBI-1117570

Neurocrine Biosciences has initiated and dosed the first patient(s) in a Phase 2 clinical study of NBI-1117570 in adults with schizophrenia. As a result, Nxera Pharma will receive milestone payments of US$22.5 million from Neurocrine pursuant to the agreement, and the milestone payments will be recognised as revenue in Q1 FY2026.

NBI-1117570 is an investigational, orally administered, dual muscarinic M1/M4 receptor agonist discovered using Nxera’s proprietary NxWave drug discovery platform. Neurocrine Biosciences acquired the rights to develop and commercialize NBI-1117570 from Nxera Pharma.

The Phase 2 study (NCT07288333) is a double-blind, placebo-controlled trial designed to evaluate the efficacy, safety and tolerability of NBI-1117570 in adult patients with schizophrenia requiring in-patient treatment. The study is expected to enroll approximately 120 patients. The primary endpoint is change from baseline in the Positive and Negative Syndrome Scale (PANSS) total score, and the key secondary endpoint is treatment-emergent adverse events (TEAEs).

Muscarinic receptors are fundamental to activating signaling pathways in the brain. There are five muscarinic acetylcholine receptors involved in neurotransmission, two of which are selectively targeted by NBI-1117570, M1 and M4, with M1 clinically validated as a potential drug target in cognition and M4 in psychosis.

Schizophrenia is a serious and complex syndrome with heterogeneous symptoms. The World Health Organization estimates that the disorder impacts approximately 24 million people worldwide. Annual associated costs for schizophrenia are estimated to be more than $150 billion in the United States.