Zevra Therapeutics(US:ZVRA)2023-11-10 13:01Acquisition and Pipeline - Zevra's acquisition of Acer Therapeutics aims to accelerate growth into a commercial organization and expand the development pipeline[10] - The acquisition includes OLPRUVA, an FDA-approved product for urea cycle disorders (UCDs), and EDSIVO for vascular Ehlers-Danlos syndrome (vEDS)[8, 10] - Arimoclomol NDA resubmission for Niemann-Pick disease Type C (NPC) is expected in Q4 2023[8, 10] - Topline data for KP1077 in idiopathic hypersomnia (IH) is expected in H1 2024[8, 10] Financial Performance and Outlook - Zevra reported net revenue of $2.9 million for Q3 2023[35] - The company expects to earn the next AZSTARYS net sales milestone of $10 million this year[35] - As of September 30, 2023, Zevra had $83.4 million in cash, cash equivalents, and investments[35] Rare Disease Focus - Zevra is focused on delivering therapies addressing significant unmet needs in rare diseases[8] - Approximately 1,800 patients are diagnosed with Niemann-Pick disease Type C (NPC) in the U S and E U [20] - There are approximately 1,100 diagnosed Urea Cycle Disorder (UCD) patients, with over 800 being treated[11, 12] - An estimated 37,000 diagnosed idiopathic hypersomnia (IH) patients are actively seeking treatment in the US[28]