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Crinecerfont

Phase 3

Congenital Adrenal Hyperplasia | Small molecule | Rare Disease |Neurocrine Biosciences, Inc.|Last Updated: May 11, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials4
Total Enrollment311
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04806451Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)PHASE3 ACTIVE NOT_RECRUITING 103Jun 25, 2021Aug 1, 2027Feb 5, 202546 United States, Belgium +8
NCT04490915Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal HyperplasiaPHASE3 ACTIVE NOT_RECRUITING 182Dec 16, 2020Aug 1, 2027May 11, 202670 United States, Austria +16
NCT07536269Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years OldPHASE2 NOT YET_RECRUITING 20Apr 1, 2026Mar 1, 2028Apr 17, 2026 -
NCT07187375Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years OldPHASE2 RECRUITING 6Sep 30, 2025Oct 4, 2029Mar 20, 20263 Germany
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Study Endpoints
Primary Endpoints
Change From Baseline in Serum Androstenedione at Week 4
Baseline, Week 4

Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.

Percent Change From Baseline in Glucocorticoid Daily Dose at Week 24
Baseline, Week 24

Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.

Number of Participants With Treatment-emergent Adverse Events (TEAE)
Day 1 up to 28 weeks
Plasma Concentration of Crinecerfont
Days 7 and 15
Secondary Endpoints
Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4
Baseline, Week 4
Percent Change From Baseline in Glucocorticoid Daily Dose at Week 28
Baseline, Week 28
Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 28
Week 28
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
CrinecerfontEXPERIMENTALCrinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks.
PlaceboPLACEBO_COMPARATORPlacebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.
Interventions
NameTypeDescription
CrinecerfontDRUGCRF type 1 receptor antagonist
PlaceboDRUGNon-active dosage form
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Eligibility Criteria
Age Range2 Years — 17 Years
SexALL
Healthy VolunteersNo
Study Sites46

Inclusion Criteria: * Be willing and able to adhere to the study procedures, including all requirements at the study center, and return for the follow-up visit. * Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency. * Be on a stable steroid regimen. * Have elevated ...

Countries:United StatesBelgiumCanadaFranceGermanyGreeceItalyPolandSpainUnited KingdomAustriaBulgariaCzechiaIsraelNetherlandsPortugalSerbiaSweden
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07187375primaryCompletionDate: changed
LOWMay 26, 2026NCT07536269primaryCompletionDate: changed
LOWMay 26, 2026NCT04490915primaryCompletionDate: changed
LOWMay 26, 2026NCT04806451primaryCompletionDate: changed
LOWMay 24, 2026NCT07187375studyFirstPostDate: changed
LOWMay 24, 2026NCT07536269studyFirstPostDate: changed
LOWMay 24, 2026NCT04806451studyFirstPostDate: changed
LOWMay 24, 2026NCT04490915studyFirstPostDate: changed
LOWMay 21, 2026NCT07536269NEW_TRIAL: changed
LOWMay 21, 2026NCT07536269NEW_TRIAL: changed