VivoSeq
Neurology

Tuberous Sclerosis

19

Pipeline Programs↗

9

Active Trials↗

14

Companies Involved↗

Tuberous Sclerosis is an emerging landscape with 19 programs across 14 companies. The most active players are Pfizer, Aucta Pharmaceuticals, Inc, Aeovian Pharmaceuticals, Inc.. 4 therapies are approved with 7 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

48/100

Pipeline Velocity

11/100

Mech. Concentration

29/100

Completion Rate

89/100

Attrition Rate

6/100

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.

Pfizer logo

Pfizer

1 program

Phase 1A

Aucta Pharmaceuticals, Inc

1 program

Phase 2A

Aeovian Pharmaceuticals, Inc.

1 program

Phase 2Bayer logo

Bayer

1 program

Phase 2Eli Lilly logo

Eli Lilly

1 program

Phase 2N

Nobelpharma

1 program

Phase 3

Pipeline

Programs by Phase

Analysis

Modality Breakdown

96%

Small molecule

24 of 25 classified programs — the dominant modality here.

4%ALL OTHERSProtein / enzyme14%

Small molecule leads; the ring shows all 25 classified programs, each share of the total. Modality classified for 96% of 26 pipeline programs.

Analysis

Trial Status

32

clinical trials

Completed17 · 53%
Recruiting5 · 16%
Unknown status4 · 13%
Active, not recruiting2 · 6%
Not yet recruiting2 · 6%
Terminated1 · 3%
Other1 · 3%

Clinical Trials

Trial Activity

32 trials

Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
Recruiting·—·Phase 2/3·NCT04987463·60 enrolled
Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY
Recruiting·Jazz Pharmaceuticals logoJazz Pharmaceuticals·N/A·NCT05485831·100 enrolled
Treatment With Full-spectrum Cannabis Extract of Refractory Epilepsy Associated With Tuberous Sclerosis Complex (TSC)
Recruiting·OOils4Cure·Phase 3·NCT07403266·84 enrolled
Cutaneous Tumorigenesis in Patients With Tuberous Sclerosis
Recruiting·National Heart, Lung, and Blood Institute (NHLBI)·N/A·NCT00001975·400 enrolled
TSC Alliance Tuberous Sclerosis Complex (TSC) Biosample Repository and Natural History Database
Recruiting·NNational Tuberous Sclerosis Association·N/A·NCT05676099·5,000 enrolled
A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML)
Active, not recruiting·Novartis logoNovartis·Phase 4·NCT05252585·4 enrolled
A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex
Active, not recruiting·Jazz Pharmaceuticals logoJazz Pharmaceuticals·Phase 4·NCT05864846·79 enrolled
Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME)
Not yet recruiting·SSovargen·Phase 1/2·NCT07287202·15 enrolled
A Parallel-Group, Phase 2, Double-Blind, Placebo-Controlled, Randomized Study of the Safety and Efficacy of Oral AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy
Not yet recruiting·AAeovian Pharmaceuticals, Inc.·Phase 2·NCT07680322·42 enrolled
An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
Completed·Jazz Pharmaceuticals logoJazz Pharmaceuticals·Phase 3·NCT02544750·199 enrolled
A Placebo-controlled Study of Efficacy & Safety of 2 Trough-ranges of Everolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Partial-onset Seizures
Completed·Novartis logoNovartis·Phase 3·NCT01713946·366 enrolled
A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
Completed·Jazz Pharmaceuticals logoJazz Pharmaceuticals·Phase 3·NCT02544763·224 enrolled
Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM
Completed·—·Phase 2·NCT00457808·25 enrolled
Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex
Completed·NNobelpharma·Phase 3·NCT02634931·94 enrolled
Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)
Completed·TThe University of Texas Health Science Center, Houston·Phase 1·NCT01031901·52 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)