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Hereditary Hemorrhagic Telangiectasia

3
Pipeline Programs
7
Companies
6
Clinical Trials
2 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
0
0
2
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
1100%
+ 7 programs with unclassified modality

Competitive Landscape

7 companies ranked by most advanced pipeline stage

Baxter International
2 programs
1
Tranexamic acid first, than placeboPhase 3
Tranexamic acid first, than placeboPHASE_31 trial
Active Trials
NCT01031992Completed23Est. Oct 2002
Vaderis Therapeutics
Vaderis TherapeuticsSwitzerland - Basel
1 program
1
EngasertibPhase 3Small Molecule2 trials
Active Trials
NCT07743671Not Yet Recruiting240Est. Jun 2028
NCT07800429Not Yet Recruiting27Est. Oct 2026
Diagonal Therapeutics
Diagonal TherapeuticsMA - Watertown
1 program
1
DIAG723Phase 1/21 trial
Active Trials
NCT07623525Recruiting93Est. Dec 2027
Colorado Therapeutics
Colorado TherapeuticsCO - Louisville
1 program
Comprehensive HHT Outcomes Registry of the United States (CHORUS)N/A1 trial
Active Trials
NCT06259292Recruiting10,000Est. Nov 2033
Angeles Therapeutics
Angeles TherapeuticsCA - Los Angeles
1 program
Comprehensive HHT Outcomes Registry of the United States (CHORUS)N/A
Oregon Therapeutics
Oregon TherapeuticsFrance - Paris
1 program
Comprehensive HHT Outcomes Registry of the United States (CHORUS)N/A
HELP Therapeutics
HELP TherapeuticsChina - Nanjing
1 program
Nationwide Awareness Campaign and Call for Dental Screening for HHT in GermanyN/A1 trial
Active Trials
NCT03549949UnknownEst. Jul 2022

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Vaderis TherapeuticsEngasertib
Baxter InternationalTranexamic acid first, than placebo
Diagonal TherapeuticsDIAG723
Vaderis TherapeuticsEngasertib
Colorado TherapeuticsComprehensive HHT Outcomes Registry of the United States (CHORUS)
HELP TherapeuticsNationwide Awareness Campaign and Call for Dental Screening for HHT in Germany

Clinical Trials (6)

Total enrollment: 10,383 patients across 6 trials

NCT07743671Vaderis TherapeuticsEngasertib

A Trial to Assess the Efficacy and Safety of Engasertib in Participants With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)

Start: Sep 2026Est. completion: Jun 2028240 patients
Phase 3Not Yet Recruiting
NCT01031992Baxter InternationalTranexamic acid first, than placebo

Tranexamic Acid and Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)

Start: Mar 2002Est. completion: Oct 200223 patients
Phase 3Completed
NCT07623525Diagonal TherapeuticsDIAG723

DIAG723 in Adults With Hereditary Hemorrhagic Telangiectasia

Start: Jun 2026Est. completion: Dec 202793 patients
Phase 1/2Recruiting
NCT07800429Vaderis TherapeuticsEngasertib

Effect of a Known P-glycoprotein (P-gp) Inhibitor, Itraconazole, on the Pharmacokinetics (PK) of Engasertib

Start: Sep 2026Est. completion: Oct 202627 patients
Phase 1Not Yet Recruiting
NCT06259292Colorado TherapeuticsComprehensive HHT Outcomes Registry of the United States (CHORUS)

Comprehensive HHT Outcomes Registry of the United States (CHORUS)

Start: Nov 2023Est. completion: Nov 203310,000 patients
N/ARecruiting
NCT03549949HELP TherapeuticsNationwide Awareness Campaign and Call for Dental Screening for HHT in Germany

Nationwide Awareness Campaign and Call for Dental Screening for HHT in Germany

Start: Jun 2018Est. completion: Jul 2022
N/AUnknown

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
2 actively recruiting trials targeting 10,383 patients
7 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.