CytRx
Corporation (NASDAQ: CYTR), a biopharmaceutical research and
development company specializing in oncology, today announced that the
first patient has been dosed in the Company’s Phase 2 clinical trial
with aldoxorubicin for the treatment of unresectable glioblastoma
multiforme (GBM), a deadly form of brain cancer. The open-label,
multisite trial is designed to investigate the preliminary efficacy and
safety of aldoxorubicin in patients whose tumors have progressed
following prior treatment with surgery, radiation and temozolomide.
“With the dosing of the first patients we are on schedule with this
important Phase 2 GBM trial, and look forward to reporting preliminary
results in the second half of this year,” said CytRx President and CEO
Steven A. Kriegsman. “Aldoxorubicin has been shown to have the unique
ability to cross the blood-brain barrier in animal models of human
glioblastoma, potentially creating a new approach to attacking brain
tumors. Should the data from this trial be positive, we will pursue the
rapid development of aldoxorubicin for unresectable GBM, including
filing for ‘breakthrough therapy’ designation with the U.S. Food and
Drug Administration, which could expedite marketing approval.”
The primary objective of this Phase 2 trial is to determine
progression-free survival (PFS) and overall survival (OS) according to
RANO Working Group Criteria. The principal secondary objective is to
evaluate the safety of aldoxorubicin in study patients as assessed by
the frequency and severity of adverse events. The clinical trial is
expected to enroll up to 28 patients randomly assigned equally to
receive either 350 mg/m2 (260 mg/m2 doxorubicin
equivalent) or 250 mg/m2 (185 mg/m2 doxorubicin
equivalent) of aldoxorubicin intravenously on Day 1, and every 21 days
thereafter until evidence of tumor progression, unacceptable toxicity or
withdrawal of consent. Tumor response will be monitored every 6 weeks by
MRI until disease progression occurs. The trial is being conducted at
the John Wayne Cancer Center/Sarcoma Oncology Center in Santa Monica,
Calif., City of Hope in Duarte, Calif. and the Louisiana State
University Health Sciences Center in New Orleans.
This Phase 2 study follows positive confirmatory results reported in
2013 from a preclinical study in which aldoxorubicin demonstrated
statistically significant efficacy (p<.0001) in the treatment of rapidly
growing human brain (glioblastoma) cancer in the brains of animals. In
that study, animals treated with aldoxorubicin had median survival of
more than 63 days, compared with approximately 25 days for animals
treated with doxorubicin or saline. In addition, because aldoxorubicin
uptake was confined to the tumor in the brain rather than normal brain
tissue, the principal investigator concluded that aldoxorubicin has the
potential to safely shrink glioblastoma tumors, which could dramatically
prolong patient survival.
About Glioblastoma Multiforme
Glioblastoma multiforme (GBM) is the most common and most malignant
brain tumor in adults, and afflicts more than 12,500 new patients in the
U.S. annually. The five-year survival rate is approximately 4%. Despite
surgical resection, radiotherapy and chemotherapy, the median survival
after diagnosis is about 12 to 14 months. Although treatment failure may
be due to several factors, limited efficacy of chemotherapeutic agents
has been attributed to several contributing factors, including
insufficient drug delivery to the tumor site through the blood-brain
barrier.
About Aldoxorubicin
The widely used chemotherapeutic agent doxorubicin is delivered
systemically and is highly toxic, which limits its dose to a level below
its maximum therapeutic benefit. Doxorubicin also is associated with
many side effects, especially the potential for damage to heart muscle
at cumulative doses greater than 500 mg/m2. Aldoxorubicin
combines doxorubicin with a novel single-molecule linker that binds
directly and specifically to circulating albumin, the most plentiful
protein in the bloodstream. Protein-hungry tumors concentrate albumin,
thus increasing the delivery of the linker molecule with the attached
doxorubicin to tumor sites. In the acidic environment of the tumor, but
not the neutral environment of healthy tissues, doxorubicin is released.
This allows for greater doses (3 ½ to 4 times) of doxorubicin to be
administered while reducing its toxic side effects. In studies thus far
there has been no evidence of clinically significant effects of
aldoxorubicin on heart muscle, even at cumulative doses of drug well in
excess of 2 g/m2.
About CytRx Corporation
CytRx Corporation is a biopharmaceutical research and development
company specializing in oncology. CytRx currently is focused on the
clinical development of aldoxorubicin (formerly known as INNO-206), its
improved version of the widely used chemotherapeutic agent doxorubicin.
CytRx has completed a global Phase 2b clinical trial with aldoxorubicin
as a first-line therapy for soft tissue sarcomas, a Phase 1b/2 clinical
trial primarily in the same indication, a Phase 1b study of
aldoxorubicin in combination with doxorubicin in patients with advanced
solid tumors and a Phase 1b pharmacokinetics clinical trial in patients
with metastatic solid tumors. CytRx plans to initiate under a special
protocol assessment a pivotal Phase 3 global trial with aldoxorubicin as
a therapy for patients with soft tissue sarcomas whose tumors have
progressed following treatment with chemotherapy. CytRx has initiated a
Phase 2 clinical trial with aldoxorubicin in patients with late-stage
glioblastoma (brain cancer), and plans to initiate a Phase 2 clinical
trial in HIV-related Kaposi’s sarcoma. CytRx plans to expand its
pipeline of oncology candidates based on a linker platform technology
that can be utilized with multiple chemotherapeutic agents and may allow
for greater concentration of drug at tumor sites. CytRx also has rights
to two additional drug candidates, tamibarotene and bafetinib. CytRx
completed its evaluation of bafetinib in the ENABLE Phase 2 clinical
trial in high-risk B-cell chronic lymphocytic leukemia (B-CLL), and
plans to seek a partner for further development of bafetinib. For more
information about CytRx Corporation, visit www.cytrx.com.
Forward-Looking Statements
This press release contains forward-looking statements within the
meaning of Section 21E of the Securities Exchange Act of 1934, as
amended. 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
relating to the outcome, timing and results of CytRx's clinical trials,
the risk that any future human testing of aldoxorubicin, including the
Phase 2 study of aldoxorubicin for the treatment of unresectable
glioblastoma multiforme (GBM), might not produce results similar to
those seen in past human or animal testing, including the mouse study
described in this press release, risks related to CytRx's ability to
manufacture its drug candidates in a timely fashion, cost-effectively or
in commercial quantities in compliance with stringent regulatory
requirements, risks related to CytRx's need for additional capital or
strategic partnerships to fund its ongoing working capital needs and
development efforts, including the Phase 3 clinical development of
aldoxorubicin, and the 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.
Copyright Business Wire 2014