Neurocrine Biosciences Announces Initiation of Phase 1 Clinical Study Evaluating NBIP-01435, a Long-Acting Corticotropin-Releasing Factor Type 1 Receptor Antagonist

Rhea-AI Summary

Neurocrine Biosciences (Nasdaq: NBIX) has initiated a Phase 1 first-in-human clinical study for NBIP-01435, a long-acting corticotropin-releasing factor type 1 receptor (CRF1) antagonist. The study will evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity in healthy adult participants.

NBIP-01435 is administered via subcutaneous injection and is being developed for the treatment of congenital adrenal hyperplasia (CAH), a rare genetic condition affecting adrenal steroid hormone production. This represents the company's first investigational peptide from its biologics pipeline to enter clinical trials.

The development builds on Neurocrine's existing presence in CAH treatment, following the FDA approval of crinecerfont in December 2024, which was the first new CAH treatment in 70 years. NBIP-01435 originated from a research collaboration with Sentia Medical Sciences, Inc., initiated in April 2021, for which Neurocrine holds exclusive development and commercialization rights.

Positive

• First investigational peptide from Neurocrine's biologics pipeline to enter clinical trials
• Builds on recent FDA approval of crinecerfont for CAH treatment in December 2024
• Exclusive rights to develop and commercialize the compound
• Potential to improve androgen control with lower glucocorticoid dosing

Negative

• Early-stage Phase 1 trial with no efficacy data yet available
• Will require extensive clinical development before potential commercialization

Insights

Biotechnology R&D Expert

Neurocrine strengthens its CAH franchise with an injectable long-acting therapy entering clinical testing, complementing its recently approved oral treatment.

Neurocrine's advancement of NBIP-01435 into Phase 1 represents a strategic expansion of their congenital adrenal hyperplasia (CAH) franchise. This milestone is particularly significant as it marks the first biologics pipeline candidate from Neurocrine to reach clinical development, diversifying their portfolio beyond small molecules.

The scientific approach here is noteworthy - NBIP-01435 targets the same corticotropin-releasing factor type 1 receptor (CRF1) as their recently approved oral drug crinecerfont, but offers a different administration route (subcutaneous injection) and potentially longer duration of action. This creates a complementary product strategy that could address different patient needs within the CAH population.

Mechanistically, CRF1 antagonism helps normalize adrenal androgen levels while potentially reducing the need for high-dose glucocorticoids that have significant side effects. The company's investment in a long-acting formulation suggests they've identified clinical advantages to sustained receptor blockade that weren't achievable with their oral therapy.

The timing is particularly strategic, coming just six months after FDA approval of crinecerfont - the first new CAH treatment in 70 years. This demonstrates Neurocrine's commitment to building a comprehensive CAH franchise rather than simply marketing a single product. The external collaboration with Sentia Medical Sciences that yielded this compound highlights Neurocrine's pragmatic approach to pipeline building through targeted partnerships.

For a rare genetic condition like CAH, having multiple treatment modalities could significantly strengthen Neurocrine's market position and provide more personalized treatment options for patients with varying disease presentations and preferences.

SAN DIEGO, June 30, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the initiation of a Phase 1 first-in-human clinical study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of investigational compound NBIP-01435 in healthy adult participants. NBIP-01435 is a long-acting corticotropin-releasing factor type 1 receptor antagonist administered as a subcutaneous injection for the potential treatment of congenital adrenal hyperplasia (CAH).

"As a leader in CAH, Neurocrine is dedicated to expanding treatment options for patients with congenital adrenal hyperplasia," said Sanjay Keswani, M.D., Chief Medical Officer, Neurocrine Biosciences. "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."

CAH is a rare genetic condition that results in an enzyme deficiency that alters the production of adrenal steroid hormones, such as cortisol, aldosterone and adrenal androgens. The deficiency of cortisol, an essential hormone for life, results in the production of excess adrenal androgens, which has typically been treated with supraphysiologic doses of glucocorticoids.

NBIP-01435 is an investigational long-acting corticotropin-releasing factor type 1 receptor (CRF₁) antagonist. CRF₁ antagonism has been shown to improve androgen control and allow for lower, more physiological glucocorticoid dosing regimens for patients with CAH. In December 2024, Neurocrine received FDA approval for crinecerfont, an oral CRF₁ antagonist, the first new treatment in 70 years for CAH.  

In April 2021, Neurocrine entered into a research collaboration with Sentia Medical Sciences, Inc., for the discovery of novel peptide CRF receptor antagonists, including NBIP-01435, for which Neurocrine has exclusive rights to develop and commercialize.

About Neurocrine Biosciences, Inc.  
Neurocrine Biosciences is a leading neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, neuroendocrine and neuropsychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, classic congenital adrenal hyperplasia, endometriosis* and uterine fibroids,* as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders because you deserve brave science. For more information, visit neurocrine.com, and follow the company on LinkedIn, X and Facebook. (*in collaboration with AbbVie)

NEUROCRINE, the NEUROCRINE BIOSCIENCES Logo and YOU DESERVE BRAVE SCIENCE are registered trademarks of Neurocrine Biosciences, Inc.

Forward-Looking Statements
In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements regarding the efficacy and therapeutic potential of NBIP-01435. Factors that could cause actual results to differ materially from those stated or implied in the forward-looking statements include, but are not limited to, the following: risks that clinical development activities may not be initiated or completed on time or at all, or may be delayed for regulatory, manufacturing or other reasons, may not be successful or replicate previous clinical trial results, may fail to demonstrate that our product candidates are safe and effective, or may not be predictive of real-world results or of results in subsequent clinical trials; risks that regulatory submissions for our product candidates may not occur or be submitted in a timely manner; our future financial and operating performance; risks associated with our dependence on third parties for development, manufacturing and commercialization activities for our products and product candidates and our ability to manage these third parties; risks that the FDA or other regulatory authorities may make adverse decisions regarding our products or product candidates; risks that the potential benefits of the agreements with our collaboration partners may never be realized; risks that our products and/or our product candidates may be precluded from commercialization by the proprietary or regulatory rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; risks associated with U.S. federal or state legislative or regulatory and/or policy efforts which may result in, among other things, an adverse impact on our revenues or potential revenue; risks associated with potential generic entrants for our products; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended March 31, 2025. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof other than required by law.

© 2025 Neurocrine Biosciences. Inc. All Rights Reserved.

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SOURCE Neurocrine Biosciences, Inc.

FAQ

What is NBIX's new Phase 1 clinical trial investigating?

The trial is investigating NBIP-01435, a long-acting CRF1 receptor antagonist administered via subcutaneous injection for the potential treatment of congenital adrenal hyperplasia (CAH).

What will Neurocrine's Phase 1 trial of NBIP-01435 evaluate?

The trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of NBIP-01435 in healthy adult participants.

How does NBIP-01435 fit into Neurocrine's CAH treatment portfolio?

NBIP-01435 is Neurocrine's first investigational peptide from their biologics pipeline, complementing their recently FDA-approved oral treatment crinecerfont for CAH.

When did Neurocrine receive FDA approval for crinecerfont?

Neurocrine received FDA approval for crinecerfont in December 2024, marking the first new CAH treatment in 70 years.

What is the origin of NBIX's NBIP-01435 compound?

NBIP-01435 originated from a research collaboration with Sentia Medical Sciences initiated in April 2021, with Neurocrine holding exclusive development and commercialization rights.