A phase 2 clinical trial for the treatment of schizophrenia has been accepted by the US Food and Drug Administration (FDA) and a study can now go ahead.
Neurocrine Biosciences received the news and informed its partner Sosei Group Corporation. The approval of the study of NBI-1117568 has triggered a $30 million payment to Sosei.
The drug is an oral, selective muscarinic M4 receptor agonist in development for the treatment of schizophrenia and other neuropsychiatric disorders.
As a selective M4 orthosteric agonist, NBI-1117568 offers the potential to deliver therapeutic effects without the need of combination therapy to minimize side effects, as required with non-selective muscarinic agonists.
At the same time, it avoids the requirement for cooperativity with acetylcholine (ACh) when compared to positive allosteric modulators. Clinical studies completed to date have shown NBI-1117568 to be generally well tolerated.
Chris Cargill, president and CEO of Sosei Heptares, said: “We are delighted with the progress of NBI-1117568 and of our collaboration with Neurocrine across our portfolio of novel selective M4 and M1 agonists. Muscarinic receptors are important drug targets in psychosis and cognitive disorders and through the application of our StaR technology platform and expertise in GPCR-focused structure-based drug design, we have discovered agonists selective to M4 and M1.
“These novel drug candidates have been designed to deliver improved therapeutic effects while avoiding the unwanted activation of M2 and M3 and its associated side effects. Our selective muscarinic agonist approach is supported by significant scientific and clinical evidence reinforcing its potential to address major unmet needs in neurological and psychiatric diseases and we are excited to be advancing developments in this area with Neurocrine.”
NBI-1117568 is the most advanced candidate from a broad portfolio of clinical and preclinical subtype-selective muscarinic M4, M1 and dual M1/M4 receptor agonists discovered by Sosei Heptares and in development under the 2021 collaboration with Neurocrine for the treatment of major neurological disorders.
Upon successful completion of pre-clinical studies, U.S.-based Neurocrine anticipates initiating phase 1 studies for a dual M1/M4 and selective M1 agonist. Advancing additional compounds into clinical studies would trigger further milestone payments from Neurocrine to Japan-based Sosei Heptares.
Eiry Roberts, chief medical officer of Neurocrine, said: “We are excited to advance the lead asset in our strategic collaboration, NBI-1117568, into a phase 2 study for the treatment of schizophrenia this year and look forward to our continued partnership with the Sosei Heptares team.”