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Afimmune Confirm Alignment with FDA on Planned Pivotal Trial of Epeleuton in Sickle Cell Disease Patients

Afimmune looking to initiate Pivotal study in 2023

Dublin, Ireland, 4 January 2023: Afimmune, a privately held, clinical stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced the successful completion and receipt of minutes from its pre-IND Meeting with the US Food and Drug Administration (FDA) with respect to Epeleuton for the treatment of Sickle Cell Disease (SCD). The minutes confirm that the FDA and Afimmune are aligned on key aspects of the Company’s planned pivotal clinical trial for Epeleuton.

Commenting on the significant company milestone, Dr. Moayed Hamza, MD, Chief Medical Officer said, “The outcome from our FDA meeting follows additional positive data showing the effect of Epeleuton on the adhesion of red blood cells of patients with Sickle Cell Disease. If approved, we believe that Epeleuton, a therapy that targets multiple aspects of the disease, has the potential to be a safe and effective oral treatment for Sickle Cell Disease”.

Epeleuton has Orphan Drug Designation for the treatment of SCD in the US and in Europe.

About Epeleuton

Epeleuton is 15-hydroxy eicosapentaenoic acid (15(S)-HEPE) ethyl ester, a novel synthetic fatty acid drug product. Afimmune is prioritising Epeleuton for SCD due to its superior preclinical efficacy, first-in-class opportunity and a significantly reduced regulatory pathway. Epeleuton has received orphan drug designation for the treatment of SCD from the FDA and EMA.

Epeleuton has been shown to have a unique mechanism of action for the treatment of SCD, targeting factors affecting severity, course of disease and vaso-occlusive crisis risk. In the humanized SCD mice, Epeleuton protected against inflammatory response, vascular activation, abnormal red cell morphology, hemolysis and sickling.

About Sickle Cell Disease
SCD is a group of inherited, progressive blood disorders carried by the β allele of the hemoglobin gene with an expected 30-year reduced life expectancy. The disease is characterized by abnormal polymerization of hemoglobin during oxygenation which results in the sickling of red blood cells. The disease is rare, with an estimated prevalence of only ~100,000 people affected in the US and ~52,000 people in the EU.

About Afimmune
Afimmune, headquartered in Dublin, Ireland, is a clinical stage drug discovery and development company working on new medicines to improve the quality of life for people with rare and inflammatory diseases.


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