Press release
KemPharm Announces Strategic Acquisition of Arimoclomol from Orphazyme, Expanding its Rare CNS Diseases Pipeline
Arimoclomol is an NDA-stage, revenue-generating investigational drug candidate being developed for the treatment of Niemann-Pick disease type C (NPC), a rare
About this update from Zevra Therapeutics, Inc.
[{"type":"text","content":"Arimoclomol is an NDA-stage, revenue-generating investigational drug candidate being developed for the treatment of Niemann-Pick disease type C (NPC), a rare progressive neurodegenerative disease KemPharm plans to refile the New Drug Application (NDA) for arimoclomol in NPC with the U.S. Food and Drug Administration (FDA) as early as the First Quarter of 2023 Conference call and live audio webcast with slide presentation is scheduled for tomorrow, May 16, 2022, at 8:30 a.m., EDT CELEBRATION, Fla., May 15, 2022 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a specialty pharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system (CNS) diseases, today announced a definitive agreement with Orphazyme A/S (in reconstruction) (ORPHA.CO; ORPH) (Orphazyme) to acquire arimoclomol, an orally-delivered, first-in-class heat shock protein (HSP) amplifier being developed as a treatment for Niemann-Pick disease type C (NPC). NPC is a rare progressive neurodegenerative disease that impacts children, adolescents, and adults, and is characterized by an inability of the body to transport cholesterol and lipids inside of cells, which leads to the abnormal accumulation of these substances within various tissues of the body, including the brain. Arimoclomol is currently being made available to NPC patients in the U.S., France and Germany under Orphazyme’s Early Access Programs (EAP). Under the terms of the agreement, KemPharm will purchase substantially all of the assets and operations of Orphazyme, including arimoclomol, for a cash payment of USD $12.8 million. The Company expects to finance the cash payment with a revolving line of credit secured by KemPharm’s balance sheet. KemPharm intends to retain the majority of Orphazyme’s current employees. In addition, KemPharm has agreed to assume an estimated reserve liability equal to approximately USD $5.2 million, which is an estimated future rebate due to the French regulatory authorities based on the revenue generated from the EAP in France. For the year ending December 31, 2022, the EAP is expected to generate at least USD $12 million in revenue based upon enrollment in France as of March 2022. The EAP is expected to remain in place until arimoclomol becomes commercially available in each of the current EAP marke...