LOS ANGELES, May 2, 2019 /PRNewswire/ -- CytRx Corporation
(NASDAQ: CYTR), a biopharmaceutical research and development
company specializing principally in oncology, today highlighted
that arimoclomol licensee Orphazyme A/S (ORPH.CO) has provided
clinical guidance on arimoclomol development milestones through
2021 in its four indications, amyotrophic lateral sclerosis (ALS),
sporadic Inclusion Body Myositis (sIBM), Niemann-Pick disease Type
C (NPC) and Gaucher Disease.
"The outlook for our arimoclomol licensee Orphazyme A/S is
encouraging due to their team's commitment to treating these
debilitating diseases," said Eric
Curtis, CytRx's President and Chief Operating Officer.
"Orphazyme's success would be rewarding to patients
worldwide. Should Orphazyme achieve certain clinical
milestones with the development of arimoclomol, CytRx would be
eligible to receive future milestone and royalty payments from our
licensing agreement."
CytRx is eligible to receive $6
million in the U.S. and $4
million in Europe upon
approval of arimoclomol in Orphazyme's first non-ALS indication,
plus royalties. As previously announced, Orphazyme is engaging with
both the U.S. Food and Drug Administration (FDA) and European
Medicines Agency (EMA) on path to approval while preparing for
filing for Niemann-Pick disease Type C.
The following are Orphazyme's expected pipeline milestones (as
stated by Orphazyme):
Niemann-Pick disease Type C (NPC):
- Communication with FDA and EMA on pivotal clinical trial data
in NPC – mid-2019
- Regulatory submission of arimoclomol in NPC – 2019
- Approval of arimoclomol in NPC and launch – 2020 and
2021, respectively
Amyotrophic Lateral Sclerosis (ALS):
- Enrollment completion in Phase 3 trial of ALS – H2
2019
- Announcement of Phase 3 results in ALS – H1 2021
- Regulatory submission of arimoclomol in ALS – H2
2021
Inclusion Body Myositis (sIBM):
- Sporadic inclusion body myositis (sIBM) Announced –
completion of enrollment in April
2019
- Announcement of interim results of Phase 2/3 trial in sIBM
– H1 2020
- Announcement of full data in Phase 2/3 trial in sIBM –
H1 2021
- Regulatory submission of arimoclomol in sIBM – H2
2021
Gaucher Disease
- Announcement of results of Phase 2 trial in Gaucher disease
– H2 2019
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.
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 cases.
About ALS
The rare neuromuscular disease Amyotrophic Lateral Sclerosis
(ALS), also called Lou Gehrig's
disease, is rapidly progressive and fatal, usually within two to
five years. The disease attacks the neurons responsible for
controlling muscles leading to paralysis of all skeletal muscles,
eventually also affecting breathing, speaking, and swallowing. The
cause of damage to the neurons includes protein misfolding and
aggregation. Familial and sporadic ALS Approximately 10% of ALS
cases are associated with familial ALS, while the rest have no
identified genetic component (sporadic ALS). Amongst familial ALS
cases, 20% harbor mutations in a SOD1 enzyme. SOD1 ALS is often
very aggressive with a life expectancy of less than 2 years from
diagnosis. Arimoclomol has so far been tested in two Phase II ALS
trials, one dose-ranging trial in sporadic ALS, and one trial in
ALS caused by SOD1 mutations.
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 Gaucher Disease
Gaucher disease is an inherited metabolic disorder caused by
mutations in a protein called glucocerebrosidase, which leads to
the accumulation of certain sugar-containing lipids. The usual
symptoms of Gaucher disease include an abnormally enlarged liver
and/or spleen (hepatosplenomegaly), low levels of circulating red
blood cells (anemia), blood cells promoting clotting
(thrombocytopenia), and skeletal abnormalities. Disease of the
nervous system is observed in a significant subpopulation of
Gaucher disease (neuropathic Gaucher). There are three types of
Gaucher disease, type II being the most severe.
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
principally 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 www.cytrx.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. 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
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, 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
michelle@argotpartners.com
maghan@argotpartners.com
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SOURCE CytRx Corporation