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AFTX-201

Phase 1

Dilated Cardiomyopathy (DCM) | Gene therapy | Cardiovascular |cbdMD, Inc.|Last Updated: Aug 20, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment22

FDA Designations

No designations recorded

Clinical trial landscape

AFTX-201 · 1 trial · 2 indications

Phase 1 1
NCT07426419An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)Dilated Cardiomyopathy (DCM)
RECRUITING22 Analytics
PHASE1RECRUITING
An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
Dilated Cardiomyopathy (DCM)Unlock trial analytics

Study Endpoints

Primary Endpoints

Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Baseline through 12 months

Safety and tolerability of AFTX-201 over 12 months.

Secondary Endpoints

Change from baseline in pharmacodynamic biomarkers
Baseline through 12 months
Change from baseline in measurement of cardiac function including left ventricular ejection fraction (LVEF)
Baseline through 12 months
Change from baseline in measurement of cardiac function including peak oxygen consumption (pVO2) by cardiopulmonary exercise test (CPET)
Baseline through 12 months
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
AFTX-201EXPERIMENTALParticipants will receive a single intravenous dose of AFTX-201

Interventions

NameTypeDescription
AFTX-201GENETICAFTX-201 is a gene therapy product consisting of the ATC-0187 capsid containing the BCL2-associated Athanogene 3 (BAG3) transgene. It is administered as a single intravenous infusion
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Eligibility Criteria

Age Range18 Years to 70 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: Male or female, 18 to 70 years of age Truncating mutation in BAG3 Dilated cardiomyopathy with left ventricular ejection fraction (LVEF) \< 45% NYHA Class II or III heart failure symptoms NT-proBNP ≥ 300 pg/mL for subjects in sinus rhythm or paced rhythm, or ≥ 600 pg/mL for s...

Countries:United States
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Frequently asked questions about AFTX-201

What is AFTX-201 used for?

AFTX-201 is an investigational gene therapy being developed for the treatment of dilated cardiomyopathy (DCM), specifically in adults with BAG3-associated dilated cardiomyopathy. It is designed to address the underlying genetic cause of this condition, which affects the heart's ability to pump blood effectively.

What does AFTX-201 target?

AFTX-201 targets the BAG3 gene, which is associated with dilated cardiomyopathy. The therapy is designed to deliver a functional copy of the BAG3 gene to heart cells, potentially correcting the genetic defect that leads to the disease. This mechanism is being evaluated in a Phase 1 clinical trial.

Who makes AFTX-201?

AFTX-201 is being developed by cbdMD, Inc., a company publicly traded under the ticker symbol YCBD on the NYSE American. The company is conducting clinical research on this gene therapy candidate for dilated cardiomyopathy.

What phase is AFTX-201 in?

AFTX-201 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities. The ongoing Phase 1 trial is recruiting participants to evaluate the safety and preliminary efficacy of the therapy.

What clinical trials is AFTX-201 in?

AFTX-201 is being studied in a Phase 1 clinical trial registered as NCT07426419. This open-label, uncontrolled trial is recruiting 22 adults aged 18 years and older with BAG3-associated dilated cardiomyopathy in the United States. The study is currently in the recruiting stage.

Is AFTX-201 the same as BAG3 gene therapy?

AFTX-201 is a gene therapy that targets the BAG3 gene, but it is not referred to by an alternative name in the available information. It is specifically designed for patients with BAG3 mutations that cause dilated cardiomyopathy, and it is being evaluated under the name AFTX-201.