VivoSeq
Other

Fragile X Syndrome

42

Pipeline Programs↗

6

Active Trials↗

25

Companies Involved↗

Fragile X Syndrome is a moderately active landscape with 42 programs across 25 companies. The most active players are Mirum Pharmaceuticals, Inc., Neuren Pharmaceuticals Limited, Retrophin. 10 therapies are approved with 6 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

57/100

Pipeline Velocity

11/100

Mech. Concentration

6/100

Completion Rate

82/100

Attrition Rate

13/100

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.

M

Mirum Pharmaceuticals, Inc.

3 programs

Phase 2N

Neuren Pharmaceuticals Limited

1 program

Phase 2R

Retrophin

1 program

Phase 2COMPASS Pathways logo

COMPASS Pathways

1 program

Phase 2Otsuka logo

Otsuka

1 program

Phase 2I

Immedica Pharma AB

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Trial Status

55

clinical trials

Completed40 · 73%
Terminated5 · 9%
Recruiting3 · 5%
Suspended2 · 4%
Withdrawn2 · 4%
Not yet recruiting1 · 2%
Other2 · 4%

Clinical Trials

Trial Activity

55 trials

Metformin in Children With Fragile X Syndrome
Recruiting·—·Phase 4·NCT05120505·20 enrolled
Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)
Recruiting·MMirum Pharmaceuticals, Inc.·Phase 2·NCT07209462·60 enrolled
Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG- Infant
Recruiting·CChildren's Hospital Medical Center, Cincinnati·Preclinical·NCT06560242·30 enrolled
An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome
Active, not recruiting·TTetra Discovery Partners·Phase 3·NCT05367960·314 enrolled
A Study to Investigate the Effects and Safety of SPG601 for the Treatment of Fragile X Syndrome in Male Participants
Not yet recruiting·SSpinogenix·Phase 2/3·NCT07439510·248 enrolled
Piloting an Early Intervention Program for Infants With Rare Neurogenetic Disorders
Enrolling by invitation·RRTI International·Preclinical·NCT03836300·120 enrolled
A Trial of Metformin in Individuals With Fragile X Syndrome (Met)
Completed·UUniversity of Alberta·Phase 2·NCT03862950·125 enrolled
A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome
Completed·TTetra Discovery Partners·Phase 2/3·NCT05163808·163 enrolled
Combined Treatment of Minocycline and Lovastatin to Treat Individuals With Fragile X Syndrome
Completed·UUniversité de Sherbrooke·Phase 2·NCT02680379·22 enrolled
Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)
Completed·Novartis logoNovartis·Phase 1·NCT01482143·21 enrolled
Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome
Completed·FFRAXA Research Foundation·N/A·NCT00858689·20 enrolled
Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)
Completed·—·Phase 2·NCT06334419·10 enrolled
Trial of Sertraline to Treat Children With Fragile X Syndrome
Completed·RRandi J. Hagerman, MD·Phase 2·NCT01474746·57 enrolled
A Study of RO4917523 in Patients With Fragile X Syndrome
Completed·Roche logoRoche·Phase 2·NCT01517698·185 enrolled
Double-blind Placebo Controlled Study of Oxytocin in Fragile X Syndrome
Completed·SStanford University·Phase 2·NCT01254045·10 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)