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Neurocrine Biosciences (NBIX) Receives Breakthrough Therapy Designation from U.S. Food and Drug Administration for Crinecerfont in Congenital Adrenal Hyperplasia

December 5, 2023 4:02 PM EST

Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced it received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for crinecerfont in congenital adrenal hyperplasia. The Company also provided updates on its R&D portfolio and strategy at its Analyst Day, held in New York.

"We are very pleased that the FDA granted Breakthrough Therapy designation for crinecerfont, thus recognizing both the seriousness of congenital adrenal hyperplasia and the significant unmet need currently faced by patients and families living with this condition," said Eiry W. Roberts, Chief Medical Officer, Neurocrine Biosciences. "The outstanding safety and efficacy results from the Phase 3 CAHtalyst studies in pediatric and adult patients suggest that crinecerfont has the potential to represent a substantial improvement over current standard of care in CAH by controlling androgen levels and allowing for reduced steroid doses. We remain on track to submit the new drug application in 2024."

2023 Analyst Day R&D Portfolio and Strategy Updates

At today's Analyst Day, Neurocrine Biosciences provided an update on its R&D portfolio and strategy, including the Company's focus on building breadth and depth across therapeutic areas and modalities with key focus areas in VMAT2 inhibition, CRF antagonism, muscarinic agonism to antagonism, and gene therapy.

With a diversified early-stage portfolio spanning a range of modalities including small molecules and biologics, and a growing pre-clinical and development candidate portfolio, Neurocrine is uniquely positioned to advance a steady flow of innovative clinical candidates to patients across its neuroscience-focused therapeutic areas of interest.

The Company remains on-track to advance two gene therapies into the clinic in 2025, and anticipates at least 20 development candidates by 2027. Other key portfolio and pipeline updates include:

Valbenazine

  • Ongoing Phase 3 study of valbenazine in adjunctive treatment of schizophrenia (ATS) will inform potential advancement of NBI-'890, a next-generation, long-acting VMAT2 inhibitor, which we anticipate will enter the clinic in 2024
  • No additional Phase 3 studies of valbenazine in ATS planned

NBI-'770 (Oral, NMDA NR2B Negative Allosteric Modulator)

  • Represents a potential first-in-class opportunity on a clinically validated target
  • Program entering Phase 2 for treatment of major depressive disorder

Muscarinic Compounds

  • Validated and differentiated mechanisms of action
  • Five programs represent largest number of muscarinic assets in human studies, including lead molecule, NBI-'568, in Phase 2 study for the treatment of schizophrenia
  • 2024 portfolio will include four Phase 1 programs:
    • NBI-'570 (dual M1/M4 agonist)
    • NBI-'567 (M1-preferring agonist)
    • NBI-'569 (M4-preferring agonist)
    • NBI-'986 (M4 antagonist, internally discovered at Neurocrine Biosciences)

Phase 2 Top-Line Clinical Data Expected in 2024

  • NBI-'568 (M4 agonist) for the treatment of schizophrenia
  • Luvadaxistat (DAAO inhibitor) for cognitive impairment associated with schizophrenia
  • NBI-'845 (AMPA potentiator) for inadequate response to treatment in major depressive disorder
  • Efmody (long-acting glucocorticoid) for adrenal insufficiency
  • Efmody for congenital adrenal hyperplasia


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