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).
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 www.cytrx.com.
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
www.centurionbiopharma.com.
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 www.orphazyme.com.
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
michelle@argotpartners.com
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SOURCE CytRx Corporation