VivoSeq
Metabolic & Endocrine

Prader-Willi Syndrome

42

Pipeline Programs↗

12

Active Trials↗

28

Companies Involved↗

Prader-Willi syndrome is a moderately active landscape with 42 programs across 28 companies. The most active players are Rhythm Pharmaceuticals, ConSynance Therapeutics, Aardvark Therapeutics, Inc.. 5 therapies are approved with 13 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

58/100

Pipeline Velocity

19/100

Mech. Concentration

13/100

Completion Rate

69/100

Attrition Rate

24/100

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.

Rhythm Pharmaceuticals logo

Rhythm Pharmaceuticals

1 program

Phase 2C

ConSynance Therapeutics

1 program

Phase 2A

Aardvark Therapeutics, Inc.

1 program

Phase 2G

Gedeon Richter Plc.

1 program

Phase 2Ferring logo

Ferring

1 program

Phase 2B

Bright Minds Biosciences Pty Ltd

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

89%

Small molecule

41 of 46 classified programs — the dominant modality here.

11%ALL OTHERSPeptide511%

Small molecule leads; the ring shows all 46 classified programs, each share of the total. Modality classified for 100% of 46 pipeline programs.

Analysis

Trial Status

59

clinical trials

Completed31 · 53%
Terminated10 · 17%
Active, not recruiting4 · 7%
Withdrawn4 · 7%
Enrolling by invitation3 · 5%
Recruiting3 · 5%
Other4 · 7%

Clinical Trials

Trial Activity

59 trials

Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial
Recruiting·UUniversity Hospital, Toulouse·Phase 3·NCT05032326·80 enrolled
A Study of Pitolisant in Patients With Prader-Willi Syndrome
Recruiting·HHarmony Biosciences Management, Inc.·Phase 3·NCT06366464·134 enrolled
The Safety and Effectiveness Study of Prebiotics and Probiotics in the Intervention of Obesity in Children With Prader-Willi Syndrome
Recruiting·CChildren's Hospital of Fudan University·Preclinical·NCT05791604·60 enrolled
Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome
Active, not recruiting·SSoleno Therapeutics, Inc.·Phase 3·NCT05701774·83 enrolled
A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension
Active, not recruiting·HHarmony Biosciences Management, Inc.·Phase 2·NCT04257929·65 enrolled
Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Active, not recruiting·AACADIA Pharmaceuticals Inc.·Phase 3·NCT06173531·170 enrolled
A Study of Setmelanotide in Patients With Prader-Willi Syndrome
Active, not recruiting·Rhythm Pharmaceuticals logoRhythm Pharmaceuticals·Phase 2·NCT06772597·18 enrolled
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
Not yet recruiting·CConSynance Therapeutics·Phase 2·NCT07348601·12 enrolled
A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.
Not yet recruiting·BBright Minds Biosciences Pty Ltd·Phase 2·NCT07266324·16 enrolled
A Study of Pitolisant in Participants With Prader-Willi Syndrome
Enrolling by invitation·HHarmony Biosciences Management, Inc.·Phase 3·NCT07219485·150 enrolled
Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome
Enrolling by invitation·SSoleno Therapeutics, Inc.·N/A·NCT07450664·200 enrolled
OLE Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
Enrolling by invitation·AACADIA Pharmaceuticals Inc.·Phase 3·NCT06420297·160 enrolled
Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
Completed·LLevo Therapeutics, Inc.·Phase 3·NCT03649477·130 enrolled
Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome
Completed·Rhythm Pharmaceuticals logoRhythm Pharmaceuticals·Phase 2·NCT02311673·40 enrolled
An Efficacy, Safety, and Pharmacokinetics Study of Beloranib in Obese Subjects With Prader-Willi Syndrome
Completed·ZZafgen, Inc.·Phase 2·NCT01818921·17 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)