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ACER-002

Phase 3

Vascular Ehlers-Danlos Syndrome | Small molecule | Rare Disease |Zevra Therapeutics, Inc.|Last Updated: Aug 24, 2025

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-BlindCONTROLLEDDMC
Total Trials1
Total Enrollment150
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05432466Clinical Trial to Compare the Efficacy of Celiprolol to Placebo in Patients With Vascular Ehlers-Danlos SyndromePHASE3 RECRUITING 150Nov 7, 2022Apr 1, 2029Aug 24, 20251 United States
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Study Endpoints
Primary Endpoints
Time to first occurrence of a vEDS-related clinical event requiring medical attention: Fatal/nonfatal cardiac or arterial events [including dissection or rupture], uterine rupture, intestinal rupture, and/or unexplained sudden death
Over the double-blind period (estimated to be 40 months)
Secondary Endpoints
Number and proportion of patients reporting a vEDS related clinical event requiring medical attention: Fatal/nonfatal cardiac or arterial events [including dissection or rupture], uterine rupture, intestinal rupture, and/or unexplained sudden death
Over the double-blind period (estimated to be 40 months)
Number and percentage of patients with adverse events
Over the double-blind period (estimated to be 40 months)
Number and percentage of Serious Adverse Events (SAE)
Over the double-blind period (estimated to be 40 months)
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ACER-002 (celiprolol) 200 mg BIDEXPERIMENTALACER-002 200 mg twice daily (BID) (after titration): 200 mg morning and 200 mg evening: 400 mg total daily dose Titration: Day 1 to Month 1 - 100 mg once daily (QD) evening: 100 mg total daily dose Month 2 to Month 3 - 100 mg morning and 100 mg evening: 200 mg total daily dose Month 3 to Month 4 - 100 mg morning and 200 mg evening: 300 mg total daily dose Month 4 to End of Treatment Period (BID) - 200 mg morning and 200 mg evening: 400 mg total daily dose
Placebo BIDEXPERIMENTALPlacebo twice daily (BID) Placebo given orally to mimic ACER-002 (celiprolol) administration
Interventions
NameTypeDescription
ACER-002 (celiprolol) 200 mg BIDDRUGACER-002 (celiprolol) 200 mg BID
Placebo BIDDRUGplacebo for ACER-002
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Eligibility Criteria
Age Range15 Years — 64 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Willingness to obtain magnetic resonance angiogram (MRA) image at local imaging facility. 2. A genetic test confirming the presence of a pathogenic COL3A1 variant (classified as likely pathogenic or pathogenic according to ACMG/AMP Guidelines. 3. Patients must be ≥ 15 years o...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT05432466studyFirstPostDate: changed