SAN DIEGO, April 21, 2015 /PRNewswire/ -- Mast
Therapeutics, Inc. (NYSE MKT: MSTX), a clinical-stage
biopharmaceutical company leveraging its molecular adhesion and
sealant technology (MAST) platform to develop novel therapies for
sickle cell disease, heart failure, and arterial disease, today
reported that patient enrollment in its pivotal EPIC study of its
lead product candidate, vepoloxamer (MST-188), in sickle cell
disease has surpassed the halfway point. Consistent with prior
guidance, the Company anticipates reporting top-line results in the
first quarter of 2016.
"We are extremely pleased to report that, as of yesterday, 205
patients had been randomized to the EPIC study," stated
Brian M. Culley, Chief Executive
Officer. "Consistent with projections we set for ourselves
more than 18 months ago, we remain on track to announce
top-line data in the first quarter of 2016. Our clinical operations
team has opened more than 70 sites in ten countries, with more than
two-thirds of those sites located in the U.S. In the next few
months, we will be opening new sites in at least four additional
countries."
"We have cultivated relationships with the sickle cell medical
and patient communities and continued to add new sites, which has
increased awareness about our study. As a result, we are
seeing consistent growth in enthusiasm and enrollment," stated Dr.
Edwin L. Parsley, the Company's
Chief Medical Officer. "Of note, more than 70% of the EPIC sites
have enrolled at least one subject and 75% of those sites have
enrolled two or more subjects. A majority of patients have come
from our U.S. sites and we believe that at the conclusion of EPIC,
the majority of the study population will have come from the
U.S."
"Notably, although EPIC is open to patients aged 4-65, the
average age of randomized patients is 14. We believe the
potential benefits of vepoloxamer are not limited to age, but that
younger patients may experience a more distinct crisis, providing
the opportunity to assess differences accross age groups. In
additon, approximately 20% of the randomized patients are 18 and
over. Exposures to vepoloxamer across a wide age range will provide
safety and efficacy data supportive of a broad label," continued
Dr. Parsley.
Mr. Culley added, "There has never been a drug approved to treat
an ongoing sickle cell crisis, however, there have been hundreds of
clinical administrations with vepoloxamer even prior to this Phase
3 and it is years ahead of other new treatments being developed for
sickle cell disease, so I am confident the sickle cell community is
hopeful and excited for a positive outcome from this important
study."
About the EPIC Study
The EPIC study is a randomized,
double-blind, two-arm, placebo-controlled, Phase 3 clinical trial
of vepoloxamer in patients with sickle cell disease. The primary
objective of the study is to demonstrate that vepoloxamer reduces
the duration of vaso-occlusive crisis. Vaso-occlusive crises are
recurring episodes of severe pain and the leading cause of
hospitalization for patients with sickle cell disease. A total of
388 patients, ages four to 65, will be enrolled in EPIC. The study
also will compare the rates of re-hospitalization for
vaso-occlusive crisis and occurrence of acute chest syndrome
between the treatment and control groups.
About Mast Therapeutics
Mast Therapeutics, Inc. is a
publicly traded biopharmaceutical company headquartered in
San Diego, California. The
Company is leveraging its MAST platform, derived from over two
decades of clinical, nonclinical and manufacturing experience with
purified and non-purified poloxamers, to develop vepoloxamer
(MST-188), its lead product candidate, for serious or
life-threatening diseases and conditions typically characterized by
impaired microvascular blood flow and damaged cell membranes.
The Company is also developing AIR001, a sodium nitrite solution
for inhalation via nebulizer, for the treatment of heart failure
with preserved ejection fraction (HFpEF).
In addition to EPIC, vepoloxamer is being tested in a Phase 2
study to evaluate whether it improves the effectiveness of
recombinant tissue plasminogen activator therapy in patients with
acute limb ischemia. The Company plans to initiate a Phase 2
study of vepoloxamer in chronic heart failure in the third quarter
of this year. AIR001 is being tested in multiple
institution-sponsored Phase 2a studies in patients with HFpEF. More
information can be found on the Company's web site at
www.masttherapeutics.com. (Twitter: @MastThera)
Mast Therapeutics™ and the corporate logo are trademarks of Mast
Therapeutics, Inc.
Forward Looking Statements
Mast Therapeutics cautions
you that statements included in this press release that are not a
description of historical facts are forward-looking statements that
are based on the Company's current expectations and assumptions.
Such forward-looking statements include, but are not limited to,
statements concerning the Company's anticipated product development
activities, such as expected clinical study commencement and
completion and announcement of study results, as well as prospects
for and anticipated timing of successful development, approval and
commercialization of our product candidates, including vepoloxamer
in sickle cell disease. Among the factors that could cause or
contribute to material differences between the Company's actual
results and the expectations indicated by the forward-looking
statements are risks and uncertainties that include, but are not
limited to: the uncertainty of outcomes in ongoing and future
studies of the Company's product candidates and the risk that its
product candidates, including vepoloxamer, may not demonstrate
adequate safety, efficacy or tolerability in one or more such
studies, including EPIC; delays in the commencement or completion
of clinical studies, including as a result of difficulties in
obtaining regulatory agency agreement on clinical development plans
or clinical study design, opening trial sites, enrolling study
subjects, manufacturing sufficient quantities of clinical trial
material, being subject to a "clinical hold," and/or suspension or
termination of a clinical study, including due to patient safety
concerns or lack of funding; the potential for additional
nonclinical or clinical studies to be required prior to initiation
of a planned clinical study; the risk that, even if clinical
studies are successful, the FDA or other regulatory agencies may
determine they are not sufficient to support a new drug
application; the potential that, even if clinical studies of a
product candidate in one indication are successful, clinical
studies in another indication may not be successful; the Company's
reliance on contract research organizations (CROs), contract
manufacturing organizations (CMOs), and other third parties to
assist in the conduct of important aspects of development of its
product candidates, including clinical studies, manufacturing, and
regulatory activities for its product candidates, and that such
third parties may fail to perform as expected; the Company's
ability to obtain additional funding on a timely basis or on
acceptable terms, or at all; the potential for the Company to
delay, reduce or discontinue current and/or planned development
activities, including clinical studies, partner its product
candidates at inopportune times or pursue less expensive but
higher-risk and/or lower return development paths if it is unable
to raise sufficient additional capital as needed; the risk that,
even if the Company successfully develops a product candidate in
one or more indications, it may not realize commercial success and
may never achieve profitability; the risk that the Company is not
able to adequately protect its intellectual property rights and
prevent competitors from duplicating or developing equivalent
versions of its product candidates; and other risks and
uncertainties more fully described in the Company's press releases
and periodic filings with the Securities and Exchange Commission.
The Company's public filings with the Securities and Exchange
Commission are available at www.sec.gov.
You are cautioned not to place undue reliance on forward-looking
statements, which speak only as of the date when made. Mast
Therapeutics does not intend to revise or update any
forward-looking statement set forth in this press release to
reflect events or circumstances arising after the date hereof,
except as may be required by law.
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SOURCE Mast Therapeutics, Inc.