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ATB200

Phase 1

Pompe Disease | Small molecule | Rare Disease |Amicus Therapeutics, Inc.|Last Updated: Oct 23, 2025

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment29

FDA Designations

No designations recorded

Clinical trial landscape

ATB200 · 1 trial · 1 indication

Phase 1 1
NCT02675465First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221Pompe Disease
COMPLETED29 Analytics
PHASE1COMPLETED
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
Pompe Disease
NCT0267546529
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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 to 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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Frequently asked questions about ATB200

What is ATB200 used for in Pompe Disease?

ATB200 is an investigational small molecule being studied for the treatment of Pompe Disease, a rare genetic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is administered intravenously and is being evaluated for its safety, tolerability, and pharmacokinetic profile.

What does ATB200 target?

ATB200 is a small molecule designed to treat Pompe Disease, a lysosomal storage disorder. While the specific molecular target is not disclosed, the drug is being studied as a potential therapy for this condition. It is administered intravenously and is currently in Phase 1 clinical trials.

Who makes ATB200?

ATB200 is being developed by Amicus Therapeutics, Inc., a biopharmaceutical company listed on NASDAQ under the ticker symbol FOLD. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Pompe Disease.

What phase is ATB200 in?

ATB200 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The Phase 1 trial has been completed, and the drug is still in the early stages of clinical testing for Pompe Disease.

What clinical trials is ATB200 in?

ATB200 has been studied in one clinical trial, identified as NCT02675465. This was a first-in-human study to evaluate the safety, tolerability, and pharmacokinetics of intravenous ATB200 alone and when co-administered with oral AT2221. The trial enrolled 29 participants with Pompe Disease and has been completed.

Is ATB200 the same as AT2221?

No, ATB200 and AT2221 are different drugs. ATB200 is the investigational drug being studied for Pompe Disease, while AT2221 is an oral medication that was co-administered with ATB200 in the clinical trial. The study evaluated the effects of ATB200 alone and in combination with AT2221.