RARE Daily

KemPharm to Acquire Orphazyme, Expanding its Rare CNS Diseases Pipeline

May 16, 2022

Specialty U.S. pharmaceutical KemPharm reached a definitive agreement with Danish biotech Orphazyme to acquire its sole experimental candidate, arimoclomol, an orally-delivered, first-in-class heat shock protein amplifier being developed as a treatment for Niemann-Pick disease type C (NPC) and other rare neurodegenerative disorders.

Photo: Travis Mickle, president and CEO r of KemPharm

The deal comes less than a year after the U.S. Food and Drug Administration handed Orphayme a Complete Response Letter for arimoclomol for the treatment of NPC. The company then withdrew its marketing application in the European Union ahead of a final vote and opinion by the Committee for Medicinal Products for Human Use (CHMP).

The company never recovered from the setback. At the end of February, Orphazyme shed half its workforce and initiated formal negotiations in Denmark to sell itself.

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 $12.8 million. KemPharm intends to retain the majority of Orphazyme’s current employees. In addition, KemPharm has agreed to assume an estimated reserve liability of approximately $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. The EAP is expected to remain in place until arimoclomol becomes commercially available in each of the current EAP markets. The transaction is expected to close on or before June 1, 2022, subject to customary closing conditions and approval by Orphazyme’s creditors and the Danish bankruptcy court.

“The acquisition of arimoclomol aligns perfectly with our strategy to build KemPharm’s value via the advancement and commercialization of novel treatments that address rare CNS conditions, including our lead clinical candidate, KP1077 in idiopathic hypersomnia,” said Travis Mickle, president and CEO r of KemPharm. “We have carefully evaluated the CRL issued by the FDA and the minutes from the subsequent Type A meeting, as well as the data that has been generated from the development work performed to date. We believe the efficacy signal for arimoclomol in NPC is convincing and that there is a viable regulatory path that could enable a successful NDA resubmission. KemPharm has significant experience with challenging regulatory situations, having successfully led or participated in three FDA product approvals, two of which followed an initial CRL. We welcome the opportunity to work with the FDA on the resubmission of the NDA for arimoclomol in NPC, which we expect to file as early as the first quarter of 2023.”

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).

Arimoclomol is administered orally and has been studied in ten phase 1, four phase 2, and three pivotal phase 2/3 trials. Arimoclomol has received Orphan Drug designation (ODD) for NPC in the United States and the European Union. Arimoclomol has also received Fast-Track, Breakthrough, and Rare Pediatric Disease designations from the FDA for NPC.

Author: Rare Daily Staff

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