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timolol

Phase 1

Hereditary Hemorrhagic Telangiectasia | Small molecule | Other |Unity Biotechnology, Inc.|Last Updated: Sep 21, 2023

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment5
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01752049Topical Anti-angiogenic Therapy for Telangiectasia in HHT: Proof of ConceptPHASE1 COMPLETED 5May 1, 2013Aug 30, 2019Sep 21, 20231 Canada
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Study Endpoints
Primary Endpoints
Change in Lesion Area of Treated Telangiectasia.
84 days

Change in lesion area (compared with baseline measurement) of treated telangiectasia.

Secondary Endpoints
Descriptive Changes in Histopathology in Baseline vs Treated Lesions
84 days
Serum Angiogenic Markers
84 days
Stability of Area of Untreated Telangiectasias Over the 84 Day Period
84 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Topical timolol maleateACTIVE_COMPARATORDrug: • Topical timolol maleate 0.5% drops * Topical timolol maleate 0.5% drops * Applied twice daily for 12 weeks (84 days) or until disappearance of lesions * Study drops will be applied to 3 cutaneous telangiectasias per patient telangiectasia per patient).
PlaceboPLACEBO_COMPARATORplacebo saline drops -Applied twice daily for 12 weeks (84 days) or until disappearance of lesions to one cutaneous telangiectasias per patient.
Interventions
NameTypeDescription
Topical timolol maleateDRUG* Topical timolol maleate 0.5% drops * Applied twice daily for 12 weeks (84 days) or until disappearance of lesions * Study drops will be applied to 3 cutaneous telangiectasias per patient
placebo saline dropsDRUGApplied twice daily for 12 weeks (84 days) or until disappearance of lesions to 1 cutaneous telangiectasias per patient.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Definite clinical or genetic diagnosis of HHT 2. Known ENG or ALK1 mutation (personal or familial) 3. Age\>=18 years 4. At least 5 typical (round/ovoid, not spider or linear) cutaneous telangiectasia (size range 2-5mm) on hands (not including lesions on over inter-phalangeal ...

Countries:Canada
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