Affinia Therapeutics has received approval from the US Food and Drug Administration (FDA) for an investigational new drug (IND) application to initiate the UPBEAT Phase I/II trial of AFTX-201.
The medication is an investigational gene therapy for adults with bcl2-associated athanogene 3 (BAG3)-related dilated cardiomyopathy (DCM).
The single-arm, open-label, multi-centre Phase I/II clinical study will assess the tolerability, pharmacodynamics, safety, and preliminary efficacy of AFTX-201.
It includes an initial dose-exploration phase followed by a dose-expansion segment, enrolling adults with genetically confirmed BAG3-associated DCM.
Participants will each receive a single intravenous infusion of AFTX-201 at doses determined from preclinical studies.
The main objective is to assess the tolerability and safety over 52 weeks following administration. Secondary and exploratory objectives include efficacy and pharmacodynamic measurements, tracked as changes from baseline.
Study design and monitoring strategies have incorporated feedback from patients, clinicians, regulators, and data from nonclinical proof-of-concept studies.
These studies showed correction of heart ejection fraction to normal levels and adequate safety margins for all doses under investigation. An independent Data Safety Monitoring Board will oversee participant safety, with protocol-defined stopping rules and centralised safety data review procedures in place.
AFTX-201 uses Affinia’s engineered capsid for cardiac delivery at doses five to ten times lower than conventional capsids such as adeno-associated virus serotype 9 (AAV9) or adeno-associated virus serotype rh74 (AAVrh74).
Affinia chief medical officer Hideo Makimura said: “We are grateful to the FDA for the timely review of our IND application for AFTX-201 for the treatment of BAG3-associated dilated cardiomyopathy and to the patient and clinician community who have provided valuable input and support for our investigational programme to date.
“We look forward to initiating the UPBEAT clinical trial at multiple trial sites in the coming weeks and bringing a much-needed treatment option for patients and families affected by this devastating disease.”
