CytRx has received a milestone payment of $250,000 from Orphazyme A/S. The payment is a result of Orphazyme dosing the first patient in their Phase 3 clinical trial evaluating arimoclomol in patients with amyotrophic lateral sclerosis (ALS).
In 2011, CytRx sold the rights to arimoclomol to Orphazyme A/S (formerly Orphazyme ApS) in exchange for a one-time, upfront payment of $150,000 (USD) and the right to receive up to a total of $120 million (USD) in milestone payments upon the achievement of certain pre-specified regulatory and business milestones, as well as royalty payments based on a specified percentage of any net sales of products derived from arimoclomol. Orphazyme, a public company trading on the Nasdaq Copenhagen exchange, is testing arimoclomol in three additional indications beyond ALS, including Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM). Orphazyme anticipates announcing top-line results for their pivotal Phase 2/3 trial in NPC during 2018, and if positive, expects to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration and a Marketing Authorization Application (MAA) to the Europeans Medicines Agency (EMA) during the second half of 2019. If approved, Orphazyme is planning to commercialize arimoclomol for the treatment of NPC during 2020.
"Receipt of this milestone payment reflects the meaningful progress being made by Orphazyme with arimoclomol, while adding to our current cash on hand," said Eric Curtis, President and Chief Operating Officer of CytRx. "Should arimoclomol be approved for NPC in Europe, CytRx will receive a $4 million milestone payment, plus royalties. Additional arimoclomol milestones include $6 million upon approval in the U.S. and $2 million upon approval in Japan, plus royalties on net sales in both territories."