Afimmune says epeleuton cut sickle cell crisis rate in Phase 2

By AI, Created 9:36 PM UTC, June 01, 2026, /AGP/ – Afimmune reported positive Phase 2 results for oral epeleuton in adults with sickle cell disease on June 2, 2026 in Dublin. The drug cut healthcare-visit vaso-occlusive crises by 57.4% from baseline and the company plans a randomised Phase 3 study in adults and adolescents.

Why it matters: - Sickle cell disease causes painful vaso-occlusive crises that often lead to emergency care, hospitalization and long-term complications. - Afimmune’s Phase 2 data suggest epeleuton could reduce crisis burden while improving red blood cell function and adhesion biology. - The company plans to use the crisis-rate endpoint as the primary efficacy measure in its planned Phase 3 study.

What happened: - Afimmune announced positive Phase 2 results for oral epeleuton in adults with sickle cell disease. - The 16-week trial tested epeleuton 2g twice daily in adults with recurrent vaso-occlusive crises. - The study compared treatment-period crisis rates with each participant’s documented rate in the 12 months before enrollment. - The mean annualized rate of vaso-occlusive crises leading to a healthcare visit fell from 3.97 to 1.95 events. - That represented a 57.4% reduction versus baseline, with p=0.0001. - 62.1% of evaluable participants achieved at least a 50% reduction in annualized healthcare-visit crisis rate.

The details: - Epeleuton also significantly reduced abnormal cell adhesion implicated in vaso-occlusion. - The therapy improved red blood cell function, including reduced sickling and improved hydration. - The safety profile matched previous epeleuton trials and the drug was well tolerated overall. - Afimmune said epeleuton is a novel synthetic fatty acid therapy and an orally administered small molecule. - The active ingredient is 15-hydroxy eicosapentaenoic acid (15(S)-HEPE) ethyl ester. - Afimmune said the program targets impaired red blood cell function, sickling and abnormal cellular adhesion. - The trial was identified as NCT05861453. - Dr. Biree Andemariam, lead investigator and professor of medicine at UConn Health, said the findings support further evaluation in Phase 3.

Between the lines: - The data matter because the primary signal was not just statistical; it also tracked with biomarker changes tied to the biology of vaso-occlusion. - A once-daily or twice-daily oral therapy could be easier for patients to use than more complex treatment approaches, which may matter in a chronic disease with recurrent crises. - Afimmune is framing the result as a step toward late-stage development, not a final proof point.

What’s next: - Afimmune plans to advance epeleuton into a randomized Phase 3 study in adults and adolescents with sickle cell disease. - The company said it will share detailed data with industry colleagues at future scientific conferences. - Epeleuton already holds Orphan Drug Designation, Fast Track Designation and Rare Pediatric Disease Designation from the FDA. - Afimmune said its lead program is in late-stage development for sickle cell disease.

The bottom line: - Afimmune now has Phase 2 evidence that epeleuton may materially reduce sickle cell crisis rates, setting up a larger trial to test whether the benefit holds in a randomized study.