Neurocrine Biosciences (NBIX) R&D Day 2025 summary

Pipeline overview and program updates

• Diversified pipeline spans neurology, psychiatry, endocrinology, immunology, and metabolic diseases, with over 35 active programs and multiple compounds in mid- to late-stage studies entering 2026.

• Osavampator (AMPA PAM) and direclidine (M4 agonist) anchor the late-stage neuropsychiatry portfolio, both in Phase 3 for major depressive disorder and schizophrenia, with topline data expected in 2027.

• Early- to mid-stage assets include muscarinic agonists (e.g., NBI-'570 in Phase 2 for schizophrenia, topline data in 2028), VMAT2 inhibitors, and CRF-based therapies for obesity and CAH.

• NBIP-'2118, a CRF2 agonist for obesity, is expected to enter Phase 1 in the first half of 2026, with preclinical data showing robust fat loss and muscle preservation.

• Robust early clinical pipeline with 24 new development candidates since 2022 and multiple gene therapy programs on track for FIH studies in 2026 and 2027.

Clinical trial data and development milestones

• Osavampator Phase 2 data showed significant MADRS reduction (7.5 points, p=0.0016) and strong tolerability; three Phase 3 trials are underway with data expected in 2027.

• Direclidine demonstrated significant efficacy and best-in-class safety in Phase 2 for schizophrenia, supporting advancement to Phase 3, with topline data expected in 2027.

• NBI-'570 Phase 2 study in schizophrenia recently initiated, with topline data expected in 2028.

• NBIP-'2118 to begin Phase 1 development in early 2026 for obesity.

R&D strategy and innovation priorities

• R&D transformation aims to deliver one new medicine every two years, with a steady output of at least four Phase 1 and two Phase 2 programs annually, focusing on validated mechanisms and multimodality innovation.

• Portfolio design emphasizes 75% validated targets, molecule design differentiation, and expansion of the CRF platform for metabolic diseases.

• Early-stage investment prioritized in neurology, with new modalities now representing ~60% of the portfolio.