Neurocrine Biosciences Starts Phase 1 Study of NBI-1117567 in Healthy Adults

Tokyo, Japan and Cambridge, UK, 9 May 2024 – Nxera Pharma ("the Company" or "Nxera"; TSE: 4565) has highlighted a significant development with its partner, Neurocrine Biosciences Inc. ("Neurocrine"; Nasdaq: NBIX). Neurocrine has commenced its Phase 1 first-in-human clinical trial to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of the investigational compound NBI-1117567 in healthy adult participants. This milestone was announced in a press release by Neurocrine.

NBI-1117567 is an investigational oral agent that targets muscarinic receptors, specifically preferring the M1 and M4 subtypes. This compound, discovered by Nxera, is believed to have the potential to address cognitive symptoms in patients suffering from various neurological and neuropsychiatric disorders. However, the current clinical milestone does not result in a milestone payment from Neurocrine to Nxera under their existing agreement from 2021. Payments are structured around the achievement of multiple, clearly defined development milestones within each program. Nxera will disclose any received milestone payments as per Tokyo Stock Exchange (TSE) reporting requirements.

Muscarinic receptors are a type of G protein-coupled receptor (GPCR) located in various tissues, including the brain, cardiovascular system, and gastrointestinal tract. The selective activation of M4 and M1 receptors in the brain is a validated method for treating cognitive and neuropsychological symptoms associated with neurological illnesses such as schizophrenia, Alzheimer’s disease-related dementia, and Parkinson’s disease. Previous attempts to develop selective M4 and M1 receptor-targeting drugs have faced challenges due to side effects from the activation of M2 and M3 receptors. Consequently, highly selective M4 or M1 agonists that avoid M2 and M3 activation are highly desirable and hold the potential to meet significant unmet medical needs.

In November 2021, Nxera Pharma and Neurocrine Biosciences entered into a collaboration and licensing agreement to develop novel muscarinic receptor agonists for treating schizophrenia, dementia, and other neuropsychiatric conditions. According to the agreement, Neurocrine has the development and commercialization rights for a range of novel clinical and preclinical subtype-selective muscarinic M4, M1, and dual M1/M4 receptor agonists discovered by Nxera. Neurocrine will handle the global development costs for these programs, except for M1 agonists being developed in Japan. Nxera retains the rights to develop M1 agonists in Japan for any indication, with Neurocrine holding co-development and profit-sharing options.

Under this agreement, Nxera could receive research and development funding, along with development, regulatory, and commercial milestone payments totaling up to $2.6 billion, plus additional product royalties, provided the agreement’s criteria are met.

Neurocrine Biosciences is a neuroscience-focused biopharmaceutical company committed to relieving suffering for individuals with limited treatment options. They are dedicated to discovering and developing transformative treatments for patients with neurological, neuroendocrine, and neuropsychiatric disorders. Neurocrine's diverse portfolio includes FDA-approved treatments and a robust pipeline featuring multiple compounds in advanced stages of clinical development.

Nxera Pharma, formerly known as Sosei Heptares, is a biopharmaceutical company driven by technology, committed to developing new specialty medicines to meet the needs of patients in Japan and globally. They are advancing a pipeline of over 30 active programs, ranging from discovery to late clinical stages, focusing on major unmet medical needs in areas such as neurology, gastrointestinal and immunological disorders, metabolic conditions, and rare diseases. Nxera employs more than 350 individuals across key locations in Tokyo, Osaka, London, Cambridge, Basel, and Seoul, and is listed on the Tokyo Stock Exchange.