VivoSeq
Metabolic & Endocrine

Congenital Adrenal Hyperplasia

27

Pipeline Programs↗

11

Active Trials↗

13

Companies Involved↗

Congenital Adrenal Hyperplasia is an emerging landscape with 27 programs across 13 companies. The most active players are Johnson & Johnson, Crinetics Pharmaceuticals Inc., Spruce Biosciences. 1 therapy is approved with 12 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

53/100

Pipeline Velocity

19/100

Mech. Concentration

29/100

Completion Rate

86/100

Attrition Rate

9/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

Johnson & Johnson logo

Johnson & Johnson

1 program

Phase 1C

Crinetics Pharmaceuticals Inc.

1 program

Phase 2S

Spruce Biosciences

1 program

Phase 2M

Millendo Therapeutics SAS

1 program

Phase 2Lundbeck logo

Lundbeck

1 program

Phase 2A

Adrenas Therapeutics Inc

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

96%

Small molecule

23 of 24 classified programs — the dominant modality here.

4%ALL OTHERSGene therapy14%

Small molecule leads; the ring shows all 24 classified programs, each share of the total. Modality classified for 96% of 28 pipeline programs.

Analysis

Trial Status

34

clinical trials

Completed18 · 53%
Recruiting7 · 21%
Active, not recruiting3 · 9%
Withdrawn2 · 6%
Unknown status2 · 6%
Terminated1 · 3%
Other1 · 3%

Clinical Trials

Trial Activity

34 trials

Congenital Adrenal Hyperplasia Once Daily Hydrocortisone Treatment
Recruiting·FFederico II University·Phase 4·NCT03760835·150 enrolled
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia
Recruiting·CCrinetics Pharmaceuticals Inc.·Phase 3·NCT07144163·150 enrolled
Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old
Recruiting·Neurocrine Biosciences logoNeurocrine Biosciences·Phase 2·NCT07187375·6 enrolled
A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia
Recruiting·Lundbeck logoLundbeck·Phase 1/2·NCT05669950·42 enrolled
Natural History Study of Patients With Excess Androgen
Recruiting·Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)·N/A·NCT00250159·3,000 enrolled
A Phase 2/3 Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Atumelnant Treatment in Pediatric Participants With Congenital Adrenal Hyperplasia Including a Long-Term Extension
Recruiting·CCrinetics Pharmaceuticals Inc.·Preclinical·NCT07159841·153 enrolled
An Open-label, Long-term Extension Study to Evaluate Safety and Efficacy of Atumelnant in Participants With Congenital Adrenal Hyperplasia (CALM2-CAH)
Recruiting·CCrinetics Pharmaceuticals Inc.·Phase 2·NCT06712823·200 enrolled
Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia
Active, not recruiting·Neurocrine Biosciences logoNeurocrine Biosciences·Phase 3·NCT04490915·182 enrolled
Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)
Active, not recruiting·Neurocrine Biosciences logoNeurocrine Biosciences·Phase 3·NCT04806451·103 enrolled
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
Active, not recruiting·AAdrenas Therapeutics Inc·Phase 1/2·NCT04783181·8 enrolled
Evaluation of a Healthcare Transition Protocol for Patients With Congenital Adrenal Hyperplasia
Not yet recruiting·UUniversity of Alabama at Birmingham·N/A·NCT07611786·40 enrolled
Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia
Completed·UUniversity of Minnesota·Phase 1·NCT03718234·11 enrolled
Research Study for Children With Salt Wasting Congenital Adrenal Hyperplasia
Completed·—·N/A·NCT00529841·7 enrolled
Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn)
Completed·CCrinetics Pharmaceuticals Inc.·Phase 2·NCT05907291·38 enrolled
Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia
Completed·NNeurocrine UK Limited·Phase 3·NCT05063994·55 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)