Who We Are
KemPharm (NASDAQ: KMPH) is a rare disease company focused on the discovery, development and commercialization of novel treatments for rare CNS and neurodegenerative diseases, lysosomal storage disorders and related treatment areas. KemPharm has a diverse product portfolio, combining a clinical-stage development pipeline with NDA-stage and commercial assets.
What We Do
KemPharm, Inc. is a biotechnology company focused on the discovery, development and commercialization of novel treatments for rare central nervous system and neurodegenerative diseases, lysosomal storage disorders and related treatment areas. The Company has a diverse product portfolio, combining a clinical-stage pipeline with new drug application stage and commercial assets. The Company’s pipeline includes arimoclomol, an orally-delivered, first-in-class investigational product candidate for Niemann-Pick disease type C, and KP1077, which the Company is developing as a treatment for idiopathic hypersomnia, a rare neurological sleep disorder, and narcolepsy.
In addition, the U.S. Food and Drug Administration has approved AZSTARYS®, formally referred to as KP415, a once-daily treatment for attention deficit hyperactivity disorder in patients age six years and older containing the Company’s prodrug, serdexmethylphenidate, which is being commercialized by Corium, Inc., an affiliate of Gurnet Point Capital, L.P., in the U.S. The FDA has also approved APADAZ®, an immediate-release combination product containing benzhydrocodone, the Company’s prodrug of hydrocodone, and acetaminophen, which is being commercialized by KVK-Tech, Inc. in the U.S.
We employ our Ligand Activated Therapy (LAT®) technology to create prodrugs that are new molecules in order to improve one or more of the attributes of approved drugs, such as susceptibility to abuse, bioavailability and safety. We create these new prodrugs by chemically attaching one or more molecules, or ligands, to an FDA-approved parent drug. When combined with the parent drug, our ligands create prodrugs designed to have improved drug attributes while maintaining efficacy equivalent to the parent drug. Once administered, targeted human metabolic processes, such as those in the GI tract, separate the ligand from the prodrug and release the parent drug, which can then exert its therapeutic effect.
KemPharm’s prodrug product candidate pipeline is focused on the high need areas of idiopathic hypersomnia (IH) and other CNS/rare diseases. In addition, the FDA has approved AZSTARYS®, a new once-daily treatment for ADHD in patents age six years and older containing KemPharm’s prodrug, serdexmethylphenidate (SDX), and APADAZ®, an immediate-release combination product containing benzhydrocodone, KemPharm’s prodrug of hydrocodone, and acetaminophen.
Our strategic focus on CNS/rare disease indications guides our business development efforts to expand our pipeline with the goal of developing and potentially commercializing innovative therapies for patients living with CNS/rare neurological disorders.