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MZE001

Phase 1

Healthy | Small molecule | Other |Maze Therapeutics, Inc.|Last Updated: Feb 6, 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 Enrollment121
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05249621A Randomized Phase 1 of of MZE001 in Healthy VolunteersPHASE1 COMPLETED 121Feb 7, 2022Dec 21, 2022Feb 6, 20231 United States
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Study Endpoints
Primary Endpoints
Number of participants with adverse events as a measure of safety and tolerability of MZE001
14 days

Occurrence of adverse events, serious adverse events, adverse events of special interest

Secondary Endpoints
Maximum concentration following multiple doses of MZE001
14 days
AUC following multiple doses of MZE001
14 days
Accumulation ratio following multiple doses of MZE001
14 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
MZE001EXPERIMENTALMZE001 is a small molecule inhibitor of muscle glycogen synthase for the potential treatment of Pompe disease.
PlaceboPLACEBO_COMPARATORExcipients containing no active ingredients.
Interventions
NameTypeDescription
MZE001DRUGSmall molecule inhibitor of muscle glycogen synthase
PlaceboDRUGProduct containing excipients with no active ingredients
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Eligibility Criteria
Age Range18 Years — 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: 1. Male and female subjects, ages 18 - 55 years, inclusive; 2. Must provide written informed consent prior to any study assessments and be willing and able to comply with all study procedures; 3. Body mass index (BMI) between 18.0 and 32.0 kg/m2 inclusive; 4. Healthy as determin...

Countries:United States
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