Neurocrine Biosciences (NBIX) Annual Novel Mechanisms in Neuropsychiatry Summit 2024 summary

Strategic overview and pipeline focus

• Positioned for a potential launch of crinecerfont for CAH in early 2025, following strong Phase III data and FDA breakthrough designation.

• Ingrezza continues to drive revenue growth, with Q2 revenue of $580 million and raised annual guidance to $2.25–$2.3 billion.

• Emphasis on revenue growth, diversification, and a regenerative pipeline spanning preclinical to commercial stages.

• Advancing multiple Phase III programs in psychiatry, including NBI-1065845 for major depressive disorder and NBI-568 for schizophrenia.

• Early-stage R&D transformation underway, expanding into multi-modality programs and gene therapies, with two gene therapy programs entering the clinic next year.

Business development and innovation approach

• Shifting balance toward internally discovered programs while remaining open to select external opportunities.

• Strong financial position enables consideration of later-stage and commercial assets for portfolio expansion.

• Focused on acquiring differentiated modalities and technologies that can change standards of care in CNS diseases.

• Recent business development efforts prioritize tools and technologies to accelerate internal research and proof-of-concept milestones.

Clinical data highlights and investor perspectives

• NBI-568 Phase II trial in schizophrenia met its primary endpoint at 20 mg, showing robust efficacy and a clean safety profile.

• Effect size of 0.61 and over 18-point total PANSS improvement at 20 mg, with once-daily dosing and no titration required.

• No clear dose response observed, but 20 mg consistently outperformed other doses; Phase III will simplify design and increase sample size.

• External KOL feedback has been strongly positive, and safety/tolerability is seen as a key differentiator.

• Plans to expand NBI-568 into additional indications beyond schizophrenia, leveraging its safety profile for broader populations.