Affinia Therapeutics has received approval from Health Canada for its clinical trial application (CTA) to begin the UPBEAT Phase I/II study of AFTX-201 as a treatment for Bcl2-associated athanogene 3 (BAG3)-related dilated cardiomyopathy (DCM).
The open-label, multi-centre, single-arm trial will examine the efficacy and safety of the investigational therapy, which employs Affinia’s capsid and delivers a fully human, full-length BAG3 transgene.
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AFTX-201 will be administered through a single intravenous infusion. Preclinical animal model data showed increased BAG3 protein concentrations in the heart and fully restored cardiac function.
This regulatory approval in Canada follows recent progress for AFTX-201, including acceptance of its investigational new drug (IND) application by the US Food and Drug Administration (FDA), which also granted fast track designation.
The European Medicines Agency (EMA) awarded orphan drug designation, further supporting ongoing development efforts.
Affinia Therapeutics chief medical officer Hideo Makimura said: “We are working diligently with multiple trial sites and investigators across the US and Canada to begin patient enrolment and dosing in the next few weeks in the UPBEAT clinical trial so that we can bring this potentially transformative treatment to patients living with BAG3 DCM and make a meaningful impact in their lives.”
The UPBEAT clinical trial will enrol adults diagnosed genetically with BAG3-associated DCM. It consists of dose-exploration and dose-expansion phases.
Safety and tolerability will be monitored over 52 weeks. Secondary and exploratory endpoints include pharmacodynamic and preliminary efficacy evaluations.
The study incorporates input from patients, clinicians, and regulators, and incorporates nonclinical safety and efficacy evidence.
Multiple sites across Canada and the US will participate in the trial.
BAG3-associated DCM is a genetic disorder leading to early onset and rapid progression of heart failure.
