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CytRx Corporation Highlights Positive Results from Full Data Set of Phase 2/3 Clinical Trial in Niemann Pick Disease Type C (NPC) Conducted by Arimoclomol Licensee Orphazyme A/S

Orphazyme to Engage with FDA and EMA on Path to Approval While Preparing for Filing
CytRx Eligible to Receive up to $120 Million in Future Milestones, Plus Royalties, From Arimoclomol Licensing Agreement

LOS ANGELES, Feb. 6, 2019 /PRNewswire/ -- CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, highlighted positive Phase 2/3 clinical trial data from arimoclomol licensee Orphazyme A/S (CPH: ORPHA). Orphazyme is currently developing arimoclomol in four different indications, including amyotrophic lateral sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM).

(PRNewsfoto/CytRx Corporation)

Orphazyme announced the full data set for its Phase 2/3 trial evaluating arimoclomol in patients with NPC. In this full data set analysis, the primary endpoint showed a 74% reduction in disease progression after 12 months compared to placebo control (p-value=0.0506). Biomarker results demonstrated statistically significant biological response to treatment.

Orphazyme also announced the urgency of making arimoclomol available to NPC patients as soon as possible. They will initiate filing preparations and seek to meet with the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) mid-2019 to discuss the path to approval. Orphazyme also communicated that it plans to submit the regulatory filing to the FDA and EMA during the first half of 2020, with potential approval expected during the second half of 2020.

"When we licensed arimoclomol to Orphazyme in 2011 we were confident they had the capabilities to plan and execute outstanding research and development in heat-shock amplification as a potential treatment in a variety of protein-misfolding diseases. It is exciting to see the progress made and how their work can help patients with such high unmet medical needs in NPC," said Steven A. Kriegsman, CytRx's Chairman and Chief Executive Officer. "Should arimoclomol be approved for NPC in the U.S. and Europe, CytRx is eligible to receive $10 million in milestone payments, plus royalties. We commend the Orphazyme team for the impressive data from their Phase 2/3 clinical trial in NPC and believe the significant value of this important asset is just now beginning to be unlocked."

Detailed Results from Full Data Set of Phase 2/3 Arimoclomol Trial in NPC

In this full data set analysis, treatment with arimoclomol adjunct to routine clinical care resulted in a 74% reduction in disease progression (p-value=0.0506) as measured by the primary endpoint, 5-domain NPC Clinical Severity Scale (NPC-CSS). In the predefined subgroup of patients of 4 years and older (44 out of 50 randomized patients in the trial), the treatment difference was statistically significant with a minimal disease progression at month 12 in the arimoclomol-treated group (p-value =0.0219). A highly statistically significant treatment difference was observed in another predefined subgroup analysis, requested by the EMA, that compared arimoclomol to placebo control in patients receiving miglustat as a part of routine clinical care (p-value =0.0071).

In agreement with the FDA, treatment response defined as no change or improved on the Clinical Global Impression of Improvement scale (CGI-I) was included as a co-primary endpoint. A responder rate of more than 50% in the placebo control group impeded the ability to show an overall effect on this endpoint. However, when considering patients who severely progressed during the trial, only 10.7% of the arimoclomol-treated patients got 'much worse' or 'very much worse' compared to 26.7% in the placebo control group.

As previously reported, overall, baseline characteristics were well-balanced across treatment arms. Arimoclomol was well-tolerated with a similar incidence of adverse events (AEs) for arimoclomol (88.2%) and placebo control (81.3%). Serious AEs occurred less frequently in the arimoclomol group (14.7%) compared to placebo control (37.5%).

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. NPC 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. There are no approved treatments for NPC in the U.S. and only one approved product in Europe called miglustat.

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 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. Its research focus is on developing therapies for diseases caused by misfolding of proteins, including lysosomal storage diseases. 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, 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, 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
(212) 600-1902


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SOURCE CytRx Corporation