Neurocrine Biosciences FY Conference Summary Company Overview - **Company**: Neurocrine Biosciences (NasdaqGS:NBIX) - **Event**: 44th Annual J.P. Morgan Healthcare Conference - **Date**: January 12, 2026 - **Key Speaker**: Kyle Gano, CEO Core Themes 1. **Strong and Building Momentum** - Neurocrine has a workforce of 2,000 and a proven track record in discovering, developing, and commercializing medicines for patients [2][3] - The company has four FDA-approved medicines, including Ingrezza and Crinecerfont, which are marketed in the U.S. [3] - In 2025, Ingrezza is projected to generate $2.5-$2.55 billion in net sales, while Crinecerfont achieved $166 million in net sales in its first nine months [4] 2. **Strategic Balanced Diversification** - Transitioned from a single product company focused on small molecules to a diversified biopharmaceutical company [5][6] - Crinecerfont is expected to be a significant contributor to revenue diversification alongside Ingrezza [6] - The company is expanding its R&D efforts into new therapeutic areas, including peptides, antibodies, and gene therapies [7][8] Financial Performance - **Ingrezza**: Projected net sales of $2.5-$2.55 billion for 2025 [4] - **Crinecerfont**: $166 million in net sales for the first nine months of commercialization [4] R&D Pipeline - **Current Pipeline**: 12 programs in clinical development (8 in phase one, 2 in phase two, 2 in phase three) [35] - **Goals**: Targeting four new phase one programs and advancing two new phase two programs annually [11] - **Focus Areas**: Neurology, psychiatry, endocrinology, with a strong emphasis on validated biology [35] Key Products and Programs 1. **Crinecerfont**: - First approved treatment for classic congenital adrenal hyperplasia (CAH) [41] - Positive two-year data showing robust efficacy and safety, with an 80% retention rate in the open-label extension study [24][45] - 40% of patients experienced a 5% or greater weight loss during the two-year period [25] 2. **Neuropsychiatry Portfolio**: - Includes first-in-class AMPA PAM for major depressive disorder and Derecladine for schizophrenia [13][14] - Both programs are in phase three trials, with potential to change the standard of care [37] 3. **Emerging Endocrinology Pipeline**: - Focused on CRF biology, with programs targeting obesity and metabolic disorders [17][19] - New molecule MBIP2118 for obesity is set to be a once-weekly injectable with a differentiated product profile [19] Market Strategy - **Sales Force Expansion**: Aiming to deepen engagement with endocrinologists and reach patients not currently under specialized care [48] - **Reimbursement Success**: Patients have been able to secure commercial reimbursement within a week, exceeding initial expectations [43] Safety and Efficacy - Crinecerfont has shown a favorable safety profile with no new safety warnings and a low rate of adrenal insufficiency [49][50] - Long-term steroid reduction has led to improvements in weight loss and insulin resistance, critical for patients with CAH [50] Future Outlook - Anticipated data flow from various programs, including gene therapy for Friedreich's Ataxia and weight loss data from CRF2 peptide studies [38] - Continued focus on building a robust R&D engine to support future growth and innovation [36] Conclusion - Neurocrine is positioned for significant growth in 2026 and beyond, with a strong pipeline and strategic diversification efforts aimed at enhancing patient care and expanding market reach [9][10]