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CytRx Corporation Highlights Enrollment Completion in Phase 2/3 Clinical Trial of Sporadic Inclusion Body Myositis Conducted by Arimoclomol Licensee Orphazyme A/S

CytRx Eligible to Receive up to $120 Million in Future Milestones, Plus Royalties, From Arimoclomol Licensing Agreement

LOS ANGELES, April 29, 2019 /PRNewswire/ -- CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, today highlighted that arimoclomol licensee Orphazyme A/S (CPH: ORPHA) has completed enrollment in its Phase 2/3 clinical trial evaluating arimoclomol for the treatment of sporadic Inclusion Body Myositis (sIBM). Orphazyme is currently developing arimoclomol in four different indications, including amyotrophic lateral sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sIBM.

(PRNewsfoto/CytRx Corporation)

"CytRx is proud to see the progress our partner Orphazyme is making with arimoclomol's clinical development," said Eric Curtis, CytRx's President and Chief Operating Officer. "The impressive rate of enrollment in this trial is made possible by both the support and enthusiasm from the sIBM patient community and Orphazyme's own sense of urgency in developing and bringing to market arimoclomol. As Orphazyme makes further progress with the drug, CytRx is eligible to receive future royalty payments from our licensing agreement."

The Phase 2/3 trial of arimoclomol for the treatment of sIBM is a 150-patient, 20-month, randomized, double-blind, placebo-controlled trial in 11 centers in the United States and one in the United Kingdom. Orphazyme expects to conduct an interim analysis in the first half of 2020 and to complete the study by the end of 2020, with results anticipated to be announced in the first half of 2021. Orphazyme has additionally initiated an open-label extension trial to which patients from the Phase 2/3 trial may enroll.

About Sporadic Inclusion Body Myositis (sIBM)

Sporadic Inclusion Body Myositis (sIBM) is a progressively debilitating muscle-wasting disease. sIBM is characterized by a build-up of protein aggregates and atrophy of muscle cells, which leads to weakness and over time severe disability. The estimated prevalence of sIBM is 24.8-45.6 per million or 17,000-31,000 patients in the USA and Europe. There are no approved treatments for sIBM. Arimoclomol has been granted Orphan Drug Designation (EU and USA) for the treatment of sIBM.

About Arimoclomol

Arimoclomol is an investigational drug candidate that amplifies the production of heat-shock proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally, crosses the blood brain barrier, and has been studied in seven Phase 1 and three Phase 2 clinical trials. Arimoclomol is in clinical development at Orphazyme for the treatment of Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and amyotrophic lateral sclerosis.

About CytRx Corporation

CytRx Corporation (NASDAQ: CYTR) is a biopharmaceutical company with expertise in discovering and developing new therapeutics to treat patients with cancer. CytRx's most advanced drug conjugate, aldoxorubicin, is an improved version of the widely used anti-cancer drug doxorubicin and has been out-licensed to NantCell, Inc.  In addition, CytRx's other drug candidate, arimoclomol has been out-licensed to Orphazyme A/S (Nasdaq Copenhagen exchange: ORPHA). Orphazyme is testing arimoclomol in four indications including amyotrophic lateral sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM).  CytRx Corporation's website is

About Orphazyme A/S

Orphazyme is a biopharmaceutical company focused on bringing novel treatments to patients living with life-threatening or debilitating rare diseases. Our research focuses on developing therapies for diseases caused by misfolding of proteins and lysosomal dysfunction. Arimoclomol, the company's lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. The Denmark-based company is listed on Nasdaq Copenhagen (ORPHA.CO). For more information, please visit

Forward-Looking Statements

This press release contains forward-looking statements. 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 A/S to obtain regulatory approval for, manufacture and commercialize its products and therapies that use arimoclomol; the results of future clinical trials involving arimoclomol; the amount, if any, of future milestone and royalty payments that we may receive from Orphazyme A/S; and other risks and uncertainties described in the most recent annual and quarterly reports filed by CytRx with the Securities and Exchange Commission and current reports filed since the date of CytRx's most recent annual report. All forward-looking statements are based upon information available to 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.

Investor Relations Contact:
Argot Partners
Michelle Carroll/Maghan Meyers
(212) 600-1902


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SOURCE CytRx Corporation