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).
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 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. 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 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
(212) 600-1902
michelle@argotpartners.com
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