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ATB200

Phase 1

Pompe Disease | Small molecule | Metabolic |Amicus Therapeutics, Inc.|Last Updated: Oct 23, 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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment29
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02675465First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221PHASE1 COMPLETED 29 — — Apr 1, 2016Aug 22, 2024Oct 23, 202519 United States, Australia +4
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Study Endpoints
Primary Endpoints
Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug
Stage 3 (2 year treatment) and Stage 4 (Extension) combined, (mean = 71 months on treatment)

Number of subjects with TEAE, TESAE, and AE leading to discontinuation during the 2 year treatment period and extension (Stage 3 and 4 combined)

Plasma Human Acid α-glucosidase (GAA) Activity Levels as Measured by Maximum Observed Plasma Concentration (Cmax).
18 Weeks

Plasma GAA levels (Cmax) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat

Plasma GAA Activity Levels as Measured by Time to Reach the Maximum Observed Plasma Concentration (Tmax).
18 Weeks

Plasma GAA levels (Tmax) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat

Plasma GAA Activity Levels as Measured by Area Under the Plasma Drug Concentration-time Curve (AUC).
18 Weeks

Plasma GAA levels (AUC) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat

Secondary Endpoints
Change From Baseline in 6-minute Walk Distance (6MWD)
Baseline, Month 60
Change From Baseline in Pulmonary Function Tests
Baseline, Month 60
Change From Baseline in Muscle Strength Tests
Baseline, Month 60
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ATB200EXPERIMENTALSequential single ascending doses of intravenously infused ATB200 for 3 dosing periods
ATB200 + AT2221EXPERIMENTALATB200 co-administered with AT2221 (Miglustat)
Interventions
NameTypeDescription
ATB200DRUG -
AT2221DRUG -
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites19

Adults with Diagnosis of Pompe disease Cohort 1: Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory): * Male and female subjects between 18 and 65 years of age, inclusive * Received ERT with alglucosidase alfa (Myozyme/Lumizyme) for the previous 2-6 years, inclusive * Was receiving a...

Countries:United StatesAustraliaGermanyNetherlandsNew ZealandUnited Kingdom
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