VivoSeq
Other

Rett Syndrome

30

Pipeline Programs↗

10

Active Trials↗

16

Companies Involved↗

Rett syndrome is an emerging landscape with 30 programs across 16 companies. The most active players are Novartis, Ipsen, Neuren Pharmaceuticals Limited. 3 therapies are approved with 8 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

54/100

Pipeline Velocity

22/100

Mech. Concentration

17/100

Completion Rate

75/100

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.

Novartis logo

Novartis

1 program

Phase 1Ipsen logo

Ipsen

1 program

Phase 1N

Neuren Pharmaceuticals Limited

1 program

Phase 2E

Edison Pharmaceuticals Inc

1 program

Phase 2Teva logo

Teva

1 program

Phase 2Pfizer logo

Pfizer

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

94%

Small molecule

29 of 31 classified programs — the dominant modality here.

6%ALL OTHERSPeptide26%

Small molecule leads; the ring shows all 31 classified programs, each share of the total. Modality classified for 100% of 31 pipeline programs.

Analysis

Trial Status

39

clinical trials

Completed18 · 46%
Terminated6 · 15%
Unknown status5 · 13%
Not yet recruiting4 · 10%
Active, not recruiting3 · 8%
Recruiting3 · 8%

Clinical Trials

Trial Activity

39 trials

Repurposing Mirtazapine in Rett Syndrome
Recruiting·UUniversity of Trieste·Phase 2·NCT07430046·54 enrolled
To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
Recruiting·BBiomed Industries, Inc.·Phase 3·NCT06840496·210 enrolled
Rett Syndrome Real World Data Observational Registry
Recruiting·International Rett Syndrome Foundation·N/A·NCT05432349·3,000 enrolled
Esketamine for the Treatment of Rett Syndrome
Active, not recruiting·—·Phase 1·NCT06199700·3 enrolled
Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)
Active, not recruiting·FFenix Innovation Group·Phase 1/2·NCT06621043·14 enrolled
An Open-label Phase 1/2/3 Study Consisting of a Phase 1/2 Safety and Dose-escalation and Phase 3 Dose-expansion Study to Evaluate Safety and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy in Females With Rett Syndrome
Active, not recruiting·TTaysha Gene Therapies, Inc.·Phase 3·NCT05606614·17 enrolled
Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome
Not yet recruiting·FFenix Innovation Group·Phase 2/3·NCT07257978·40 enrolled
Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome
Not yet recruiting·UUnravel Biosciences, Inc.·Phase 1·NCT07150013·15 enrolled
A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome
Not yet recruiting·HHolland Bloorview Kids Rehabilitation Hospital·Phase 2·NCT04041713·35 enrolled
A Phase 3 Randomized, Double-Blind, Placebo Controlled, Parallel Group, Multicenter Study With Open-Label Extension to Evaluate the Efficacy And Safety of Fenfluramine Hydrochloride in Study Participants With Rett Syndrome
Not yet recruiting·UCB logoUCB·Phase 3·NCT07503444·200 enrolled
Study of ANAVEX2-73 in Patients With Rett Syndrome
Completed·AAnavex Life Sciences Corp.·Phase 2·NCT03758924·31 enrolled
ANAVEX2-73 Study in Patients With Rett Syndrome
Completed·AAnavex Life Sciences Corp.·Phase 3·NCT03941444·33 enrolled
ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome
Completed·AAnavex Life Sciences Corp.·Phase 2/3·NCT04304482·92 enrolled
To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome
Completed·BBiomed Industries, Inc.·Phase 2/3·NCT06849973·187 enrolled
Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
Completed·AACADIA Pharmaceuticals Inc.·Phase 3·NCT04181723·187 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)