SAN DIEGO, Feb. 22, 2016 /PRNewswire/ -- Mast Therapeutics,
Inc. (NYSE MKT: MSTX), a biopharmaceutical company developing
novel, clinical-stage therapies for sickle cell disease and heart
failure today announced that it has completed patient enrollment in
its Phase 3 clinical study of vepoloxamer for the treatment of
patients with sickle cell disease experiencing vaso-occlusive
crisis, known as the EPIC study. Consistent with prior guidance,
the Company expects to report top-line results in the second
quarter of 2016.
The EPIC study was conducted under the direction of Principal
Investigator James F. Casella, M.D.,
Rainey Professor and Chief, Division of Pediatric Hematology, Vice
Chair for Research, Pediatrics and Director of the Basic and
Translational Research Program in Sickle Cell Disease,
Comprehensive Sickle Cell Center at Johns
Hopkins University School of Medicine.
"The full enrollment of EPIC is an important achievement in the
history of sickle cell disease clinical development, as it
represents the largest placebo-controlled clinical trial conducted
to date for our patients with this disease," stated Mark T. Gladwin, M.D., Jack D. Myers Professor
and Chair, Chairman of the Department of Medicine, Director,
Pittsburgh Heart, Lung, Blood and Vascular Medicine Institute, UPMC
and the University of Pittsburgh School
of Medicine, and member of the Executive Steering Committee for
EPIC. "Interventional treatment of acute painful crisis
represents an important unmet medical need, and has remained
elusive to large, multicenter randomized trials necessary to
support registration of a new drug. EPIC has set a new
standard for the field, and the learnings from this study will have
an important impact on the design of future studies. The
patients and their families, our committed clinical investigators,
and Mast Therapeutics should be applauded for this important
achievement."
"As physicians, we are frustrated by our inability to offer
patients a disease-modifying treatment for an ongoing crisis,"
stated Gregory J. Kato, M.D.,
Professor of Medicine, Division of Hematology/Oncology, Director,
Sickle Cell Center of Excellence, UPMC and the University of Pittsburgh School of Medicine, and
member of the Executive Steering Committee for EPIC. "This is
a challenging condition for which multiple underlying pathologies
may need to be addressed simultaneously to achieve clinical
outcomes. In non-clinical studies, vepoloxamer has
demonstrated multiple activities that may improve the complex
pathologies occurring during sickle cell crisis. We are
anxious to review the data from the trial, once unblinded, to
determine how vepoloxamer's pharmacodynamic activity translates to
clinical benefit for sickle cell patients."
"We are proud to have completed enrollment in the EPIC study,"
stated Brian M. Culley, Chief
Executive Officer. "We wish to express our deep gratitude to the
courageous patients and their family members and the committed
investigators and study coordinators around the globe who have
helped us achieve this goal."
"Following the last patient's discharge from the hospital and
30-day safety observation period, there will be an extensive and
rigorous review of the blinded data for quality control, ultimately
leading to database lock. Study unblinding then will be performed
by the study biostatistician. After the biostatistician's work is
complete, Mast will learn the top-line outcome of the study from
the biostatistician and will be able to report top-line results to
the public," continued Mr. Culley.
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
hospitalized for treatment of vaso-occlusive crisis. The primary
objective of the study is to demonstrate that vepoloxamer reduces
the duration of vaso-occlusive crisis, with the duration of crisis
measured from the time a patient is randomized to the time at which
the patient receives the last dose of parenteral opioid analgesic
for the treatment of vaso-occlusive crisis prior to hospital
discharge. A total of 388 patients, ages four to 46 were enrolled
in EPIC. Secondary efficacy endpoints are to compare the rates of
re-hospitalization for vaso-occlusive crisis within 14 days of
initial hospital discharge and the occurrence of acute chest
syndrome within 120 hours of randomization between the treatment
and control groups.
About Sickle Cell Disease and Vaso-Occlusive Crisis
Sickle cell disease is a chronic, genetic blood disorder that
affects millions worldwide and an estimated 90,000 to 100,000
people in the United States, where
it is classified as a rare, or orphan, disease. The hallmark of
sickle cell disease is recurring episodes of severe, debilitating
pain commonly known as sickle cell crisis or vaso-occlusive crisis.
The intense pain experienced by patients is the result of
obstruction of blood vessels by "sickled" red blood cells, which
are rigid and highly adherent to the vessel walls and to each
other. This obstruction leads to reduced blood flow to organs,
including the bone marrow, not only causing severe pain, but also
cumulative tissue damage and, ultimately, loss of vital organ
function and significantly reduced lifespan. There are
between 80,000 to 100,000 hospitalizations annually in the U.S.
related to vaso-occlusive crisis and no approved treatment option
to shorten the duration or reduce the severity of a crisis once
underway.
About Mast Therapeutics
Mast Therapeutics, Inc.
is a publicly traded biopharmaceutical company headquartered in
San Diego, California. The
Company is leveraging its MAST (Molecular Adhesion and Sealant
Technology) platform, derived from over two decades of clinical,
nonclinical and manufacturing experience with purified and
non-purified poloxamers, to develop vepoloxamer (also known as
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 nebulization, for the treatment of heart failure
with preserved ejection fraction (HFpEF).
Vepoloxamer is an investigational new drug being evaluated in a
pivotal Phase 3 study called EPIC for the treatment of
vaso-occlusive crisis in patients with sickle cell disease and in a
Phase 2 study for the treatment of patients with chronic heart
failure. AIR001 is in Phase 2 clinical development for the
treatment of 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
within the meaning of the Private Securities Litigation Reform Act
of 1995 that are based on the Company's current expectations and
assumptions. Such forward-looking statements may be identified by
the use of forward-looking words such as "intend," "plan,"
"anticipate," "believe," "expect," among others, and include, but
are not limited to, statements relating to prospects for successful
development and commercialization of the Company's product
candidates, including vepoloxamer for the treatment of
vaso-occlusive crisis of sickle cell disease, and anticipated
timing of achievement of development milestones, such as completion
of clinical studies and announcement of study data. There are
a number of factors that could cause or contribute to material
differences between actual events or results and the expectations
indicated by the forward-looking statements. These factors include,
but are not limited to: the inherent uncertainty of outcomes in
ongoing and future studies of the Company's product candidates and
the risk that its product candidates may not demonstrate adequate
safety, efficacy or tolerability in one or more such studies,
including vepoloxamer in 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; delays in clinical
study closeouts, including blinded data review and quality control
and assurance procedures; the risk that, even if current and
planned 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 dependence on third parties to assist
with important aspects of development of its product candidates,
including conduct of its clinical studies and supply and
manufacture of clinical trial material, and, if approved,
commercial product, and the risk that such third parties may fail
to perform as expected, leading to delays in product candidate
development or approval or inability to meet market demand for
approved products, if any; the risk that the Company may be
required to repay its outstanding debt obligations on an
accelerated basis and/or at a time that could be detrimental to its
financial condition, operations and/or business strategy; risks
associated with the Company's ability to manage operating expenses
and/or obtain additional funding to support its operations 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, or partner its
product candidates at inopportune times 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
obtain and maintain effective patent coverage or other market
exclusivity protections for its products, if approved, without
infringing the proprietary rights of others; 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.