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CytRx Corporation Announces Receipt of Milestone Payment from Orphazyme A/S

Orphazyme Milestones Payments Could Deliver up to $120 Million, Plus Royalties

LOS ANGELES, Sept. 13, 2018 /PRNewswire/ -- CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, today announced it has received a milestone payment of $250,000 (USD) from Orphazyme A/S (CPH: ORPHA). 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).

(PRNewsfoto/CytRx Corporation)

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."

About Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a rare, rapidly progressive, and always fatal neurodegenerative disease.  Protein misfolding and aggregation in motor neurons are important contributors to the disease process, which ultimately leads to paralysis of skeletal muscles as well as the muscles that enable breathing.  The patient population in Europe and the United States is estimated to be approximately 50,000 patients.  Currently, there are only limited treatment options available.  Arimoclomol has been granted Orphan Drug Designation (EU and U.S.) for the treatment of ALS.

About Niemann-Pick Disease Type C

Niemann-Pick disease Type C (NPC) is a rare, genetic and progressive disease that impairs the ability of the body to move cholesterol and other fatty substances (lipids) inside the cells. The result is an accumulation of lipids within the body's tissue, including the brain tissue, causing damage to the affected areas. The symptoms upon onset of NPC vary from fatality during the first months after birth to a progressive disorder not diagnosed until adulthood. The disease affects neurologic and psychiatric functions as well as various internal organs. Systemic symptoms of NPC are more common in infancy or childhood and the rate of progression is usually much slower in individuals with onset of symptoms during adulthood. NPC is usually fatal and the majority of individuals with the disease die before the age of 20. Arimoclomol has been granted Orphan Drug Designation (EU and U.S.) for the treatment of NPC.

It is conservatively estimated that the number of potential NPC patients in the United States and in the EU is between 1,000 and 2,000 individuals in total. Diagnostic challenges may affect the number of potential patients. However, a treatment option could also increase awareness of the disease and assist in identifying more patients.

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.  CytRx's website is

About Centurion BioPharma Corporation

CytRx's wholly owned subsidiary, Centurion BioPharma Corporation, is focused on advancing a portfolio of novel, anti-cancer drug candidates that employ its LADR™ (Linker Activated Drug Release) technology, a discovery engine designed to leverage the Company's expertise in albumin biology and linker technology for the development of a new class of potential breakthrough anti-cancer therapies. A critical element of the LADR™ platform is its ability to bind anti-cancer molecules to albumin, the most ubiquitous protein in human blood plasma, and then to release the highly potent cytotoxic payload at the tumor site. This technology allows for the delivery of higher doses of drug directly to the tumor, while avoiding much of the off-target toxicity observed with the parent molecules.  Centurion'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. Its research focus is on developing therapies for diseases caused by misfolding of proteins, including lysosomal storage diseases. Arimoclomol, the company's lead drug 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, go to

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 its products that use arimoclomol; the ability of Orphazyme A/S to manufacture and commercialize products or therapies that use arimoclomol; the amount, if any, of future milestone and royalty payments that we may receive from Orphazyme A/S; Centurion BioPharma Corporation's ability to develop new ultra-high potency drug candidates based on its LADR™ technology platform; our ability to attract potential licensees; 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
(212) 600-1902


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SOURCE CytRx Corporation