VivoSeq
Rare Disease

Phenylketonuria

22

Pipeline Programs↗

16

Active Trials↗

14

Companies Involved↗

Phenylketonuria is an emerging landscape with 22 programs across 14 companies. The most active players are Changchun GeneScience Pharmaceutical Co., Ltd., Agios Pharmaceuticals, Inc., Gritgen Therapeutics Co., Ltd.. 2 therapies are approved with 4 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

51/100

Pipeline Velocity

25/100

Mech. Concentration

33/100

Completion Rate

77/100

Attrition Rate

15/100

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.

Changchun GeneScience Pharmaceutical Co., Ltd. logo

Changchun GeneScience Pharmaceutical Co., Ltd.

2 programs

Phase 1A

Agios Pharmaceuticals, Inc.

1 program

Phase 1G

Gritgen Therapeutics Co., Ltd.

1 program

Phase 1R

Rubius Therapeutics

1 program

Phase 1O

Orphanetics Pharma Entwicklungs GmbH

1 program

Phase 1Moderna logo

Moderna

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

91%

Small molecule

21 of 23 classified programs — the dominant modality here.

9%ALL OTHERSProtein / enzyme14%RNA therapy14%

Small molecule leads; the ring shows all 23 classified programs, each share of the total. Modality classified for 83% of 30 pipeline programs.

Analysis

Trial Status

47

clinical trials

Completed24 · 51%
Recruiting8 · 17%
Active, not recruiting6 · 13%
Terminated5 · 11%
Withdrawn2 · 4%
Not yet recruiting2 · 4%

Clinical Trials

Trial Activity

47 trials

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Recruiting·PPTC Therapeutics·Phase 3·NCT06302348·56 enrolled
A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria
Recruiting·Otsuka logoOtsuka·Phase 2·NCT06637514·10 enrolled
Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
Recruiting·EEmory University·N/A·NCT04404530·45 enrolled
Eating Disorders in Patients With Phenylketonuria
Recruiting·CCentral Hospital, Nancy, France·N/A·NCT07698743·70 enrolled
A Phase 3, Double-Blind, Randomized, Two-Period, Multicenter, Placebo-Controlled, Efficacy and Safety Study of JNT-517 for the Treatment of Participants With Phenylketonuria
Recruiting·Otsuka logoOtsuka·Phase 3·NCT06971731·120 enrolled
A Single-center, Randomized, Double-blind, Placebo-controlled Phase I Clinical Trial in Healthy Adult Participants in China to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single Ascending Oral Doses of GenSci144
Recruiting·Changchun GeneScience Pharmaceutical Co., Ltd. logoChangchun GeneScience Pharmaceutical Co., Ltd.·Phase 1·NCT07685210·48 enrolled
Announcement of Rare Metabolic Diseases as Part of Systematic New-born Screening: the Experience of Phenylketonuria.
Recruiting·AAssistance Publique - Hôpitaux de Paris·N/A·NCT06289348·80 enrolled
A Phase 1b, Open-label, Multicenter, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of AG-181 in Subjects With Phenylketonuria
Recruiting·AAgios Pharmaceuticals, Inc.·Phase 1·NCT07241234·20 enrolled
Effect of L-carnitine Supplementation on Phenylalanine and Brain-derived Neurotrophic Factor Levels in Infants and Children With Phenylketonuria
Active, not recruiting·MMansoura University·Phase 4·NCT06901323·176 enrolled
A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria
Active, not recruiting·Sanofi logoSanofi·Phase 1/2·NCT05972629·32 enrolled
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
Active, not recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Phase 3·NCT05270837·55 enrolled
A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
Active, not recruiting·PPTC Therapeutics·Phase 3·NCT05166161·200 enrolled
SHINE-PKU: Pegvaliase in Adult PKU Long-Term
Active, not recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Phase 3·NCT05700004·244 enrolled
PEGVALIASE-LT: Pegvaliase Long-Term in PKU
Active, not recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Phase 3·NCT04619056·168 enrolled
To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria
Not yet recruiting·GGritgen Therapeutics Co., Ltd.·Phase 1·NCT07318909·9 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)