KemPharm (KMPH) Investor Presentation - Slideshow

Company Overview - KemPharm is focused on developing and commercializing treatments for rare CNS, neurodegenerative, and lysosomal storage diseases[7] - The company has a diverse product portfolio with clinical-stage pipeline assets, revenue-generating NDA-stage assets, and commercial assets[7] - KemPharm's strategic focus is on CNS/rare diseases, with potential for internal commercialization[15] Arimoclomol (Niemann-Pick Disease Type C) - KemPharm acquired arimoclomol, an NDA-stage drug candidate for Niemann-Pick disease type C (NPC), for $128 million[24] - The French EAP for arimoclomol is expected to generate revenue exceeding $12 million in FY 2022[22, 24] - The company plans to resubmit the NDA for arimoclomol in NPC as early as Q1 2023[15, 39] KP1077 (Idiopathic Hypersomnia) - KP1077 is being developed for the treatment of Idiopathic Hypersomnia (IH), a high-value opportunity with significant unmet need[16] - A Phase 1 clinical trial assessing the cardiovascular safety of SDX (KP1077) is expected to have topline results in Q3 2022[59] - Approximately 37,000 patients are diagnosed with IH and actively seeking treatment[46, 68] AZSTARYS® (ADHD) - AZSTARYS® is a partnered medication for ADHD, providing ongoing revenue potential from royalties and milestones[7, 17] - Over 110 million commercial and Medicaid lives have access to AZSTARYS as of March 1, 2021[79] Financial Position - As of March 31, 2022, KemPharm had $1191 million in cash, cash equivalents, marketable securities, and long-term investments[17, 84, 90] - The company's cash runway extends beyond 2025[17, 84, 90]