SAN DIEGO, Sept. 20, 2016 /PRNewswire/ -- Mast
Therapeutics, Inc. (NYSE MKT: MSTX), a biopharmaceutical company
developing novel, clinical-stage therapies for sickle cell disease
and heart failure, today reported top-line results from EPIC, a
Phase 3 clinical study of its investigational new drug vepoloxamer
(also known as MST-188) for the treatment of individuals with
sickle cell disease experiencing vaso-occlusive crisis (VOC).
The study did not meet its primary efficacy endpoint of
demonstrating a statistically significant reduction in the mean
duration of VOC (82 hours in the vepoloxamer group compared to 78
hours in the placebo group in the intent-to-treat population
(p=0.09)). There were no statistically significant
differences between treatment groups in the intent-to-treat
population across the two secondary efficacy endpoints, rate of
re-hospitalization for VOC and the occurrence of acute chest
syndrome. Consistent with previously conducted studies, vepoloxamer
was generally well tolerated with no statistically significant
differences in treatment-related serious adverse events in the
vepoloxamer group compared to the placebo group. No deaths occurred
on the study.
"We are exceedingly disappointed with these top-line results.
While clearly not the outcome we wanted, we believe the insights
and data from the largest placebo-controlled clinical trial ever
completed in sickle cell disease will substantially advance the
understanding of vaso-occlusive crisis and the still maturing
clinical science necessary to support the development of new
therapeutics for this debilitating disease," stated Brian M. Culley, the Company's Chief Executive
Officer. "We wish to reiterate our sincere appreciation for all of
the patients, caregivers, and others who aided us in conducting
this informative study."
"These analyses are limited to just top-line data, so in the
coming weeks the Company intends to review the full data set from
EPIC. In addition, we plan to perform an interim analysis of the
ongoing heart failure trial of vepoloxamer. However, based on the
data we've seen to date, we expect we will terminate all clinical
development of vepoloxamer. Consequently, while we evaluate our
options, we intend to significantly and immediately reduce our
operating expenses and continue our efforts with AIR001, our lead
asset in heart failure with preserved ejection fraction, which
currently is the subject of a 100-patient phase 2 study expected to
complete enrollment by the end of 2017," continued Mr. Culley.
Investor Conference Call
The Company will hold a
conference call tomorrow, September 21,
2016, at 8:00 a.m. ET /
5:00 a.m. PT to discuss the Phase 3
results. Interested parties may access the conference call by
dialing (855) 239-3120 from the U.S., (855) 669-9657 from
Canada, and (412) 542-4127 from
outside the U.S. and should request the Mast Therapeutics, Inc.
Call. A live webcast of the conference call will be available
online from the Investors section of Mast's website at
http://www.masttherapeutics.com/investors/events/. Replays of the
webcast will be available on the Company's website for 30 days and
a telephone replay will be available through September 28, 2016 by dialing (877) 344-7529 from
the U.S., (855) 669-9658 from Canada, and (412) 317-0088 from elsewhere
outside the U.S. and entering replay access code 10093117.
About the EPIC Study
The EPIC study was a
randomized, double-blind, two-arm, placebo-controlled, Phase 3
clinical trial of vepoloxamer in individuals with sickle cell
disease hospitalized for acute pain typical of vaso-occlusive
crisis who required treatment with parenteral opioid analgesia. The
primary objective of the study was to evaluate the efficacy of
vepoloxamer in reducing the duration of vaso-occlusive crisis, with
the duration of crisis measured from the time of randomization to
the time at which the patient received the last dose of parenteral
opioid analgesia for the treatment of vaso-occlusive crisis prior
to hospital discharge. Vepoloxamer or placebo (0.45% saline)
was administered intravenously as a 1 hour loading dose infusion
(100 mg/kg), immediately followed by a continuous maintenance
infusion (30 mg/kg/hr) for at least 12 hours and up to 48
hours. Randomization was stratified by age (≥4 to <16
years or ≥16 to ≤65 years), use of hydroxyurea (yes or no), and
pain score (measured using the Wong-Baker FACES® Pain Rating Scale
at time of randomization: <8 or ≥8). The study was 90% powered
to detect a 17% (16-hour) difference in treatment arms, with a
statistical significance level of p=0.05 (assuming an average
crisis duration of 96 hours in the control arm and a coefficient of
variation > 50%). Secondary efficacy endpoints were 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.
A total of 388 patients, ages four to 46, were randomized in
EPIC. More than 75 study sites in 14 countries
participated. The average age was 15 years. Patients
under age 18 accounted for approximately 71% of total
subjects. Approximately 61% of patients were concurrently on
hydroxyurea therapy.
About Sickle Cell Disease and Vaso-Occlusive
Crisis
Sickle cell disease is a chronic, genetic blood
disorder that affects millions worldwide and an estimated 100,000
people in the United States, where
it is classified as a rare, or orphan, disease. The hallmark
of sickle cell disease is vaso-occlusive crisis, which results from
obstruction of blood vessels by sickled red blood cells causing
tissue ischemia and injury. Vaso-occlusions can occur
everywhere blood flows and lead to an accumulating disease burden
in every organ system with the ultimate loss of vital organ
function and significantly reduced lifespan. Vaso-occlusive
crisis is also characterized by intense and debilitating pain which
can last for days and even weeks. There are between 80,000 to
100,000 hospitalizations annually in the U.S. related to
vaso-occlusive crisis and no FDA approved treatment to shorten its
duration or reduce the risk of ischemic injury related to
crisis.
About Mast Therapeutics
Mast Therapeutics, Inc. is a
publicly traded biopharmaceutical company headquartered in
San Diego, California. The Company
is developing two clinical-stage investigational new drugs for
serious or life-threatening diseases and conditions. Vepoloxamer,
the Company's lead product candidate, is in Phase 3 clinical
development for the treatment of vaso-occlusive crisis in patients
with sickle cell disease and in Phase 2 clinical development for
the treatment of patients with heart failure. Enrollment in
the Company's Phase 2 study of vepoloxamer in patients with chronic
heart failure is ongoing. AIR001, the Company's second
product candidate, is in Phase 2 clinical development for the
treatment of patients with heart failure with preserved ejection
fraction (HFpEF). Enrollment in Phase 2 studies of AIR001 in
patients with HFpEF are ongoing, including a 100-patient,
multicenter, randomized, double-blind, placebo-controlled, Phase 2
study in patients with HFpEF being conducted by the Heart Failure
Clinical Research Network. More information can be found on
the Company's web site at www.masttherapeutics.com.
Mast Therapeutics™ and the corporate logo are trademarks of Mast
Therapeutics, Inc.
Forward Looking Statements
Mast Therapeutics cautions
you that statements in this press release and statements made
during the conference call to be held on September 21, 2016 that are not a description of
historical fact are forward-looking statements within the meaning
of the Private Securities Litigation Reform Act of 1995.
Forward-looking statements may be identified by the use of words
referencing future events or circumstances such as "expect,"
"intend," "plan," "anticipate," "believe," and "will," among
others. Examples of forward-looking statements in this press
release include statements relating to the Company's development
plans for its product candidates, the Company's business plans and
objectives, and its anticipated results of operations, financial
condition and funding needs. Forward-looking statements
should not be read as guarantees of future performance or results
because they involve the Company's beliefs and assumptions based on
currently available information and are subject to significant
known and unknown risks and uncertainties that may cause actual
performance and results to differ materially from expectations
indicated by the forward-looking statements. Some of the factors
that could cause actual performance or results to differ include,
without limitation: the Company's need for additional funding to
continue to operate as a going concern; risks associated with the
Company's ability to manage operating expenses and obtain
additional capital as needed; uncertainty related to the Company's
ability to remain in compliance with the terms and conditions under
its debt facility and risk that, in addition to the $10 million prepayment required within three days
of announcement of negative EPIC results, the Company may be
required to repay its remaining outstanding debt obligations on an
accelerated basis and/or at a time that could be detrimental to the
Company's financial condition, operations and/or business strategy;
the impact of significant redutions in the Company's operations on
its ability to develop its product candidates or maintain
compliance with laws and regulations relating to public companies;
completion of a more detailed analysis of EPIC data and
announcement of additional data from the study; uncertainties
inherent in the conduct of clinical studies and the risk that the
Company's product candidates may not demonstrate adequate safety,
efficacy or tolerability in one or more clinical studies; the
potential for the Company to significantly delay, reduce or
discontinue current and/or planned development activities or sell
or license its assets at inopportune times if it is unable to raise
sufficient additional capital as needed; that the Company is not
the sponsor of the ongoing Phase 2 clinical studies of AIR001 and
has no control over the conduct of those studies, including whether
they will be completed on anticipated timelines, or at all; the
Company's dependence on third parties to assist with important
aspects of development of the Company's product candidates,
including the conduct of its clinical studies, the manufacture and
supply of its clinical trial material and, if approved, commercial
product, and the conduct of regulatory activities, including
preparation of new drug applications, and the risk that such third
parties may fail to perform as expected leading to delays in
product candidate development, regulatory approval, commercial
launch and/or inability to meet future market demand for any
approved products; the risk that vepoloxamer may not realize
commercial success even if it receives regulatory approval, and
that the Company 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, or that the use or manufacture of the Company's
products may infringe 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 or made
during its September 21, 2016
conference call to reflect events or circumstances arising after
the date hereof, except as may be required by law.
Logo -
http://photos.prnewswire.com/prnh/20120612/LA22456LOGO-a
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/mast-therapeutics-reports-top-line-results-from-phase-3-study-in-sickle-cell-disease-300331289.html
SOURCE Mast Therapeutics, Inc.