VivoSeq
Metabolic & Endocrine

Turner Syndrome

20

Pipeline Programs↗

9

Active Trials↗

12

Companies Involved↗

Turner syndrome is an emerging landscape with 20 programs across 12 companies. The most active players are Ascendis Pharma Endocrinology Division A/S, Pfizer, Par Pharmaceutical, Inc.. 4 therapies are approved with 3 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

48/100

Pipeline Velocity

15/100

Mech. Concentration

50/100

Completion Rate

100/100

Attrition Rate

0/100

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.

A

Ascendis Pharma Endocrinology Division A/S

1 program

Phase 2Pfizer logo

Pfizer

1 program

Phase 2P

Par Pharmaceutical, Inc.

1 program

Phase 2Bayer logo

Bayer

1 program

Phase 2BioMarin Pharmaceutical logo

BioMarin Pharmaceutical

1 program

Phase 2Changchun GeneScience Pharmaceutical Co., Ltd. logo

Changchun GeneScience Pharmaceutical Co., Ltd.

2 programs

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

95%

Small molecule

20 of 21 classified programs — the dominant modality here.

5%ALL OTHERSPeptide15%

Small molecule leads; the ring shows all 21 classified programs, each share of the total. Modality classified for 100% of 23 pipeline programs.

Analysis

Trial Status

30

clinical trials

Completed20 · 67%
Recruiting7 · 23%
Unknown status1 · 3%
Not yet recruiting1 · 3%
Active, not recruiting1 · 3%

Clinical Trials

Trial Activity

30 trials

Bleeding Patterns in Sequential and Continuous Progesterone Supplementation in Adolescents With Turner Syndrome
Recruiting·—·Phase 4·NCT06834594·40 enrolled
Long Term Effects of Oral Versus Transdermal Estrogen Replacement Therapy in Turner Syndrome
Recruiting·—·Phase 4·NCT06570460·50 enrolled
Vosoritide for Short Stature in Turner Syndrome
Recruiting·RRoopa Kanakatti Shankar, MBBS, MS·Phase 2·NCT05849389·20 enrolled
Lymphedema, Low-grade Inflammation and the Vasculature in Turner Syndrome
Recruiting·UUniversity of Aarhus·N/A·NCT06325618·150 enrolled
Determining Dose Equivalence Between Oral and Transdermal Estrogen Treatment in Women With Turner Syndrome
Recruiting·—·Phase 4·NCT06544473·50 enrolled
A Pivotal, Parallel-Arm, Phase 3, Open-Label, Active-controlled, Global, Multicenter, Randomized Basket Trial Investigating the Efficacy and Safety of Once-weekly Lonapegsomatropin Compared to Daily Somatropin in Prepubertal Children and Adolescents With Growth Failure or Short Stature Due to Growth Hormone Sufficient Disorders - Turner Syndrome, SHOX Deficiency, Small for Gestational Age, and Idiopathic Short Stature
Recruiting·AAscendis Pharma A/S·Phase 3·NCT07221851·186 enrolled
Gonadal Tissue Freezing for Fertility Preservation in Individuals at Risk for Ovarian Dysfunction, Premature Ovarian Insufficiency and Clinically Indicated Gonadectomy
Recruiting·Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)·N/A·NCT04948658·200 enrolled
A Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner Syndrome
Active, not recruiting·AAscendis Pharma Endocrinology Division A/S·Phase 2·NCT05690386·48 enrolled
A Study Comparing Different Treatment Approaches for the Initiation of Puberty in Girls With Turner Syndrome Using a TRIFECTA-DARED Approach for Rare Diseases
Not yet recruiting·UUniversiti Kebangsaan Malaysia Medical Centre·Phase 2·NCT07041814·24 enrolled
Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years
Completed·Merck & Co. logoMerck & Co.·Phase 4·NCT01066052·115 enrolled
Growth Hormone Treatment of Women With Turner Syndrome
Completed·—·N/A·NCT00420654·24 enrolled
Estrogen Dosing in Turner Syndrome: Pharmacology and Metabolism
Completed·NNemours Children's Clinic·Phase 4·NCT00837616·41 enrolled
Estrogen Treatment (Oral vs. Patches) in Turner Syndrome
Completed·NNemours Children's Clinic·Phase 3·NCT00140998·16 enrolled
D3-GHR Polymorphism and Turner Syndrome
Completed·UUniversity Hospital Tuebingen·N/A·NCT00443144
Use of Somatropin in Turner Syndrome
Completed·Novo Nordisk logoNovo Nordisk·Phase 3·NCT01518036·57 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)