AB Science announces that EMA granted orphan drug status to AB8939 for the treatment of acute myeloid leukemia

Core Viewpoint - The European Medicines Agency (EMA) has granted orphan drug designation to AB8939 for the treatment of acute myeloid leukemia (AML), indicating its potential significant benefit over existing therapies [1][2]. Group 1: Orphan Drug Designation - AB8939 previously received orphan drug designation from the US FDA, and the EU designation is a significant milestone, suggesting that it offers substantial benefits to AML patients [2]. - The criteria for orphan drug designation at EMA are stringent, requiring evidence of significant benefit compared to existing treatments [2][4]. Group 2: Efficacy and Safety Data - Preclinical data from mouse models show that AB8939 provides a significant benefit over current therapies like cytarabine, azacitidine, and venetoclax [3]. - Preliminary efficacy and safety data from Phase 1 trials indicate that AB8939 is effective as a monotherapy with different treatment cycles [3]. - AB8939 demonstrates efficacy against drug-resistant AML cells, with 45% of vincristine-resistant and 66% of cytarabine-resistant cells responding to treatment [4]. Group 3: Mechanism of Action - AB8939 is a synthetic molecule that targets cancer cells by destabilizing microtubules essential for cell division and inhibiting enzymes crucial for cancer stem cell survival [6]. - The chemical name of AB8939 is '1-{4-[2-(5-ethoxymethyl-2-methylphenylamino)-oxazol-5-yl]phenyl}imidazolidin-2-one' [6]. Group 4: Benefits of Orphan Drug Designation - Orphan drug designation in the EU provides various benefits, including scientific advice on product development, access to centralized marketing authorization, and potential financial incentives [8]. - If approved, AB8939 will enjoy 10 years of marketing exclusivity from the date of registration, provided the orphan designation remains valid [8].