On track to initiate global pivotal Phase 3
studies and CVOT in the fourth quarter Aligned with global
regulatory authorities on a consistent definition of statin
intolerance Conference Call and Webcast on Tuesday, June 28,
2016 at 4:30 p.m. Eastern Time
Esperion Therapeutics, Inc. (NASDAQ:ESPR), a late-stage
pharmaceutical company focused on developing and commercializing
first-in-class oral therapies for the treatment of patients with
elevated low density lipoprotein cholesterol (LDL-C), today
provided updates on the bempedoic acid (ETC-1002) clinical
development program and regulatory plans. The clinical program has
two major components: the global pivotal Phase 3 efficacy and
safety studies, and the planned global cardiovascular outcomes
trial (CVOT) in patients with elevated LDL-C levels who are unable
to tolerate statins (statin intolerance). The Company intends to
initiate global pivotal Phase 3 efficacy studies for LDL-C lowering
in patients with hypercholesterolemia, and a CVOT specifically in
statin intolerant patients who are at high risk for cardiovascular
(CV) disease, in the fourth quarter of 2016.
For Europe, the Company engaged with the European Medicines
Agency (EMA) resulting in alignment on the definition of statin
intolerance and key design features of the global Phase 3 LDL-C
lowering program and CVOT. The planned LDL-C lowering efficacy
studies, together with the ongoing long-term safety and
tolerability study (1002-040) initiated in January 2016, are
designed to support a submission for an LDL-C lowering Marketing
Authorization Application (MAA) for bempedoic acid in patients with
elevated LDL-C, including statin intolerant patients, in Europe by
2019. The CVOT is designed to support a MAA submission for
bempedoic acid for CV disease risk reduction in statin intolerant
patients in Europe by 2022.
For the United States (U.S.), the Company engaged with the U.S.
Food and Drug Administration (FDA or the Agency) resulting in
alignment on the definition of statin intolerance, key design
features of the CVOT, and the regulatory approval pathway for a CV
disease risk reduction indication in statin intolerant patients for
bempedoic acid. However, the FDA did not provide clarity on a
regulatory pathway for an LDL-C lowering indication in the U.S. in
statin intolerant patients at this time. The Agency indicated its
position regarding an LDL-C lowering indication could be impacted
by potential future changes in their view of LDL-C lowering as a
surrogate endpoint or the possibility of a shift in the future
standard-of-care for statin intolerant patients with elevated LDL-C
levels. In the event LDL-C lowering is no longer a surrogate
endpoint for initial approval in the future, Esperion would plan to
submit a New Drug Application (NDA) to FDA for a CV disease risk
reduction indication on the basis of a successful completion of the
CVOT, which would include the results of the LDL-C lowering
efficacy studies, by 2022.
“We are pleased with our discussions and engagement with the FDA
and EMA on the definition of statin intolerance and key design
features of our planned CVOT. Scientific advice received from EMA
provided clarity on the regulatory approval pathways for both an
LDL-C lowering indication and a cardiovascular disease risk
reduction indication in statin intolerance for bempedoic acid in
Europe,” said Tim Mayleben, president and chief executive officer
of Esperion Therapeutics. “Following engagement with FDA on the
LDL-C lowering statin intolerance program for bempedoic acid in the
U.S., the regulatory pathway for an LDL-C lowering indication is
not well defined at this time due to the Agency’s view of a
potentially evolving landscape. However, we remain confident in our
ability to recruit and treat a patient population that will
continue to be relevant to care in the U.S. at the time we seek
approvals for bempedoic acid. We look forward to advancing our
global Phase 3 and CVOT clinical programs later this year, which we
anticipate will support our future global regulatory and marketing
application submissions.”
Esperion Global Cardiovascular Outcomes
Trial
The Company intends to initiate the planned CVOT in statin
intolerant patients who are at high risk for CV disease in the
fourth quarter of 2016. The global CVOT – Cholesterol Lowering via
BEmpedoic Acid, an ACL-inhibiting Regimen (CLEAR) Outcomes – will
be conducted in cooperation with the Cleveland Clinic Coordinating
Center for Clinical Research (C5Research), with Steven E. Nissen,
MD, Department Chair, Cardiovascular Medicine, Cleveland Clinic,
serving as study chairman.
The CLEAR Outcomes trial is a randomized, double-blind,
placebo-controlled study to assess the effects of bempedoic acid in
statin intolerant patients who are at high risk of CV disease. The
global study is expected to enroll about 12,600 patients at up to
1,000 sites in approximately 30 countries. Patients enrolled in the
study will be required to have a history of, or be at high risk
for, CV disease with LDL-C levels between 100 mg/dL and 190 mg/dL
despite background lipid-lowering therapy. The trial will be an
event-driven study with the primary efficacy endpoint of the effect
of bempedoic acid versus placebo on the risk of major adverse
cardiovascular events (cardiovascular death, non-fatal myocardial
infarction, non-fatal stroke, hospitalization for unstable angina,
or coronary revascularization; also referred to as “five-component
MACE”).
“The CLEAR Outcomes study for bempedoic acid is a critical step
in identifying patients who are unable to tolerate statins,” said
A. Michael Lincoff, MD, Director of C5Research, Vice Chairman for
Research, Department of Cardiovascular Medicine, Cleveland Clinic.
“This study is a well-designed and appropriately powered
cardiovascular outcomes trial in these patients. We are highly
confident that we will be able to identify these patients in this
outcomes study. Those of us in clinical practice see these patients
frequently and we need more accessible therapeutic options for them
in our treatment armamentarium. As an oral therapy which provides
30 percent LDL-C lowering as monotherapy, is well-tolerated, and
can easily be combined with currently available oral LDL-C lowering
therapies, we hope to learn whether bempedoic acid can benefit
these patients with statin intolerance.”
Esperion Global Pivotal Phase 3 LDL-C
Lowering Program
In the fourth quarter of 2016, the Company plans to initiate
global pivotal Phase 3 efficacy studies in patients with elevated
LDL-C levels, including statin intolerant patients. The global
Phase 3 LDL-C lowering program is anticipated to include
approximately 2,000 patients on optimized background
lipid-modifying therapy with LDL-C levels of ≥130 mg/dL for
patients without atherosclerotic cardiovascular disease (ASCVD) and
≥100 mg/dL for patients with ASCVD or heterozygous familial
hypercholesterolemia (HeFH).
The global pivotal Phase 3 efficacy studies are expected to
measure the change in LDL-C from baseline at 12 and 24 weeks. This
program is also designed to produce 52 weeks of safety data,
comprising an appropriately sized safety database at the time of
initial MAA submission for the LDL-C lowering indication in Europe
by 2019.
Esperion 2016 Anticipated Milestones and Financial
Outlook
Milestone |
Date |
Initiate bioavailability study for Fixed Dose Combination of
bempedoic acid and ezetimibe |
July |
Announce top-line results from high-dose statin studies (-035
and -037) |
September |
Initiate CLEAR Outcomes global CVOT |
Q4 2016 |
Initiate global pivotal Phase 3 LDL-C efficacy studies |
Q4 2016 |
Esperion expects full-year 2016 net cash used in operating
activities to be between $65 to $75 million and its cash and cash
equivalents and investment securities to be approximately $220
million at December 31, 2016. Current cash resources are expected
to be sufficient to fund operations into early 2019 and the
anticipated announcement of top-line results from the global
pivotal Phase 3 LDL-C lowering and long-term safety clinical
studies.
Conference Call and Webcast Information
Esperion's management will host a conference call to discuss
these updates. The call can be accessed by dialing (877) 831-3840
(domestic) or (253) 237-1184 (international) five minutes prior to
the start of the call and providing access code 41576970. A live,
listen-only webcast of the conference call can be accessed on the
investor relations section of the Esperion website at
investor.esperion.com, along with slides to accompany this update.
A webcast replay of the call will be available approximately two
hours after completion of the call and will be archived on the
Company's website for two weeks.
About Bempedoic Acid
Bempedoic acid is a first-in-class ACL inhibitor inhibiting
cholesterol biosynthesis and works to lower elevated levels of
LDL-C by up-regulating the LDL receptor, but with reduced potential
for muscle-related side effects. Phase 1 and 2 studies conducted
previously in more than 700 patients treated with bempedoic acid
have produced clinically relevant LDL-C lowering results of up to
30 percent as monotherapy, approximately 50 percent in combination
with ezetimibe, and an incremental 24 percent when added to stable
statin therapy. It is expected that bempedoic acid will provide
incremental LDL-C reduction on top of currently available
background lipid-regulating therapies.
Esperion’s Commitment to Patients with
Hypercholesterolemia
In the U.S., 78 million people, or more than 20 percent of the
population, have elevated LDL-C; an additional 73 million people in
Europe and 30 million people in Japan also live with elevated
LDL-C. Esperion’s mission is to provide patients and physicians
with a new therapy to significantly decrease elevated levels of
LDL-C, without muscle-related side effects. Esperion-discovered and
developed, bempedoic acid is an oral LDL-C lowering therapy in
Phase 3 development. The Company plans to develop bempedoic acid as
a monotherapy as well as a fixed-dose combination with ezetimibe,
with a particular focus on patients with elevated LDL-C who are
unable to tolerate statin therapy. It is estimated that
approximately 5-20 percent of patients who are prescribed statins
are considered statin intolerant.
About Esperion Therapeutics
Esperion Therapeutics, Inc. is a late-stage pharmaceutical
company focused on developing and commercializing oral therapies
for the treatment of patients with elevated LDL-C. Through
scientific and clinical excellence, and a deep understanding of
cholesterol biology, the team at Esperion is committed to
developing new LDL-C lowering therapies that will make a
substantial impact on reducing global cardiovascular disease; the
leading cause of death around the world. Bempedoic acid, the
Company’s lead product candidate, dramatically lowers LDL-C levels
and could potentially reduce elevated levels of LDL-C in patients
with hypercholesterolemia, especially those that can’t tolerate
statins. For more information, please
visit www.esperion.com and follow us on Twitter
at https://twitter.com/EsperionInc.
Forward-Looking Statements
This press release contains forward-looking statements that are
made pursuant to the safe harbor provisions of the federal
securities laws, including statements regarding the therapeutic
potential of, and clinical development plan for, bempedoic acid,
the Company’s plans regarding its Phase 3 program and planned CVOT,
in each case including estimates regarding the timing that the
correlative NDA or MAA could be filed, and the Company’s expected
cash and liquidity position and outlook. Any express or implied
statements contained in this press release that are not statements
of historical fact may be deemed to be forward-looking statements.
Forward-looking statements involve risks and uncertainties that
could cause Esperion’s actual results to differ significantly from
those projected, including, without limitation, delays or failures
in patient enrollment in the Company’s studies, the risk that FDA
may require additional studies or data that Esperion may need to
change the design of its Phase 3 program, the impact of future
changes in FDA’s view of LDL-C lowering as a surrogate endpoint or
standard-of-care treatment for patients with elevated LDL-C levels,
that positive results from a clinical study of bempedoic acid may
not necessarily be predictive of the results of future clinical
studies, particularly in different or larger patient populations,
that existing cash resources may be used more quickly than
anticipated, that the planned cardiovascular outcomes trial may not
demonstrate that bempedoic acid leads to cardiovascular risk
reduction, or the risk that other unanticipated developments or
data could interfere with the scope of development and
commercialization of bempedoic, as well as other risks detailed in
Esperion’s filings with the Securities and Exchange Commission,
including its Annual Report on Form 10-K. You are cautioned not to
place undue reliance on the forward-looking statements, which speak
only as of the date of this release. Esperion disclaims any
obligation or undertaking to update or revise any forward-looking
statements contained in this press release, other than to the
extent required by law.
Media Contact:
Elliot Fox
W2O Group
212.257.6724
efox@w2ogroup.com
Investor Contact:
Mindy Lowe
Esperion Therapeutics, Inc.
734.887.3903
mlowe@esperion.com
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