Pthena C Corporation plc announced the publication of the Phase 3 VITAL clinical trial in Blood, a journal of the American Society of Hematology (ASH). Based on the totality of data generated to date, including results from the VITAL clinical trial, Prothena has advanced birtamimab into the confirmatory Phase 3 AFFIRM-AL clinical trial in patients with Mayo Stage IV AL amyloidosis under a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA) with a primary endpoint of all-cause mortality at a significance level of 0.10. Phase 3 AFFIRM -AL topline data is expected in 2024.

Birtamimab has also been granted orphan drug designation for AL amyloidosis by both the FDA and the European Medicines Agency and has been granted Fast Track designation by the FDA.