Neurocrine Biosciences has initiated a Phase 1 first-in-human clinical study to evaluate NBIP-01435, a long-acting corticotropin-releasing factor type 1 receptor antagonist for the potential treatment of congenital adrenal hyperplasia (CAH). The study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of the investigational compound in healthy adult participants.
NBIP-01435 is administered as a subcutaneous injection and represents the first investigational peptide from Neurocrine's biologics pipeline to advance to clinical trials. "This is the first investigational peptide from our biologics pipeline to proceed to the clinic, and we look forward to seeing the potential of these molecules and how they can complement our robust small molecule portfolio," said Sanjay Keswani, M.D., Chief Medical Officer at Neurocrine Biosciences.
Building on CAH Treatment Leadership
The development of NBIP-01435 builds on Neurocrine's established position in CAH treatment. In December 2024, the company received FDA approval for crinecerfont, an oral CRF1 antagonist that marked the first new treatment for CAH in 70 years. "As a leader in CAH, Neurocrine is dedicated to expanding treatment options for patients with congenital adrenal hyperplasia," Keswani noted.
CAH is a rare genetic condition characterized by an enzyme deficiency that disrupts the production of adrenal steroid hormones, including cortisol, aldosterone and adrenal androgens. The cortisol deficiency, involving an essential hormone for life, leads to excess adrenal androgen production, which has traditionally been managed with supraphysiologic doses of glucocorticoids.
Mechanism and Therapeutic Potential
NBIP-01435 functions as a long-acting corticotropin-releasing factor type 1 receptor (CRF1) antagonist. CRF1 antagonism has demonstrated the ability to improve androgen control while allowing for lower, more physiological glucocorticoid dosing regimens in CAH patients. This mechanism offers potential advantages over current treatment approaches that rely on high-dose glucocorticoid therapy.
The compound emerged from a research collaboration established in April 2021 between Neurocrine and Sentia Medical Sciences, Inc., focused on discovering novel peptide CRF receptor antagonists. Neurocrine holds exclusive rights to develop and commercialize NBIP-01435 and other compounds from this partnership.
Company Portfolio and Focus
Neurocrine Biosciences maintains a diverse portfolio of FDA-approved treatments spanning multiple therapeutic areas, including tardive dyskinesia, chorea associated with Huntington's disease, classic congenital adrenal hyperplasia, endometriosis and uterine fibroids. The company's pipeline includes multiple compounds in mid- to late-phase clinical development across neurological, neuroendocrine and neuropsychiatric disorders.
The advancement of NBIP-01435 to clinical trials represents a significant milestone for Neurocrine's biologics pipeline, complementing their established small molecule portfolio. The Phase 1 study will provide initial data on the safety and pharmacological profile of this long-acting peptide approach to CAH treatment.
