KemPharm Sees Promise in Arimoclomol for Niemann-Pick Disease Type C, and Plans to Resubmit to FDA as Early as Q1 2023

CytRx Corporation (OTCQB: CYTR) (“CytRx” or the “Company”), a biopharmaceutical innovator focused on research and development of life-saving cancer therapeutics, is pleased to highlight the strategic purchase of arimoclomol by KemPharm, Inc. (“KemPharm”).

KemPharm is a specialty biopharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system ("CNS") diseases. KemPharm has announced a definitive agreement with Orphazyme A/S ("Orphazyme") to acquire arimoclomol, a first-in-class heat shock protein potentiator being developed as a treatment for Niemann-Pick disease type C ("NPC"). NPC is a rare neurodegenerative disease characterized by abnormal accumulation of cholesterol and lipids in neuronal tissues. 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 transaction is expected to close on or before June 1, 2022.

CytRx licensed arimoclomol to Orphazyme for milestones and royalties on sales, and CytRx expects the terms of its license with Orphazyme to remain intact as the arimoclomol asset transfers to KemPharm.

In the press release issued by KemPharm, Travis Mickle, Ph.D., President and Chief Executive Officer of KemPharm stated, “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. 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.”

Stephen Snowdy, PhD, Chief Executive Officer of CytRx commented, “CytRx is very pleased to see that arimoclomol will enjoy the support and resources of KemPharm, and that KemPharm sees value in the human trials data collected on arimoclomol through the course of 10 Phase 1, four Phase 2 and three pivotal Phase 2/3 trials. We are especially pleased to see that KemPharm, with its experience in challenging regulatory submissions, plans to re-submit arimoclomol to the FDA as early as Q1 2023. With a mean age of death of 13 years, patients desperately need a solution to Niemann-Pick disease type C.”

Forward-Looking Statements

This press release contains forward-looking statements. These statements are not historical facts, but instead represent only CytRx’s belief regarding future events, many of which, by their nature, are inherently uncertain and outside of CytRx’s control. Forward-looking statements include statements relating to the potential receipt of EMA and FDA approval of arimoclomol and the CytRx’s potential receipt of future milestone and royalty payments from Orphazyme. Such statements involve risks and uncertainties that could cause actual events or results to differ materially from the events or results described in the forward-looking statements, including risks and uncertainties relating to the ability of Orphazyme to obtain regulatory approval for, manufacture and commercialize its products and therapies that use arimoclomol; the results of clinical trials involving arimoclomol; the amount, if any, of future milestone and royalty payments that we may receive from Orphazyme; and other risks and uncertainties described in the most recent annual and quarterly reports filed by the CytRx with the SEC, including disclosures under the heading “Risk Factors”, and current reports filed since the date of the CytRx’s most recent annual report. All forward-looking statements are based upon information available to the CytRx on the date the statements are first published. CytRx undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

About CytRx

CytRx Corporation (OTCQB: CYTR) is a biopharmaceutical company with expertise in discovering and developing new therapeutics principally to treat patients with cancer. CytRx’s most recent advanced drug conjugate, aldoxorubicin, is an improved version of the widely used anti-cancer drug doxorubicin and has been out-licensed to ImmunityBio, Inc. In addition, CytRx’s drug candidate, arimoclomol, was sold to Orphazyme A/S in exchange for milestone payments and royalties. Orphazyme is developing arimoclomol in two indications, including Niemann-Pick disease Type C (NPC). CytRx Corporation’s website is www.cytrx.com.

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