– Treatment with sotatercept in the ongoing
PULSAR Phase 2 trial was associated with improvements in cardiac
and pulmonary function at week 24 –
– Presentation of echocardiography data from
the PULSAR trial received AHA’s “Cardiopulmonary Best Abstract”
Award –
– Preclinical research shows sotatercept
inhibits cardiac remodeling, restores function in experimental
model of severe PAH –
– Acceleron to host investor and analyst
conference call and webcast with guest PAH key opinion leaders
today, Friday, November 13, at 11:00 a.m. EST –
Acceleron Pharma Inc. (Nasdaq: XLRN), a leading
biopharmaceutical company in the discovery, development, and
commercialization of TGF-beta superfamily therapeutics to treat
serious and rare diseases, today presented new cardiac and
pulmonary function data from the ongoing PULSAR Phase 2 trial of
sotatercept in patients with pulmonary arterial hypertension
(PAH).
Echocardiography data obtained during the PULSAR trial and
presented virtually during the American Heart Association (AHA)
2020 Scientific Sessions showed that patients on stable background
PAH-specific therapies treated with sotatercept experienced
improvement in a measure of cardiopulmonary function known as right
ventricular-pulmonary arterial (RV-PA) coupling, which represents
the match between the output of the RV and the resistance of the
pulmonary vasculature. These patients also experienced improvement
in RV function. In patients with PAH, RV function deteriorates as a
result of the pulmonary vascular remodeling that is a hallmark of
the disease.
“Progression of PAH may lead to RV failure, which is ultimately
fatal in patients with this disease,” said Dr. Vallerie
McLaughlin*, Professor of Medicine and Director of the Pulmonary
Hypertension Program at the University of Michigan. McLaughlin’s
presentation today of the PULSAR echocardiography data received
AHA’s “Cardiopulmonary Best Abstract” award.
"The measurement of RV-PA coupling may offer important insights
into how the RV is coping with increased pulmonary pressure.
Although the assessment of RV-PA coupling noninvasively is a
relatively new approach, these data are encouraging, as they
demonstrate potential for RV remodeling,” McLaughlin continued.
“Taken together with previously reported results of sotatercept’s
hemodynamic and functional improvements—as measured by reduced
pulmonary vascular resistance and increased six-minute walk
distance—these outcomes suggest that sotatercept has the potential
to become a paradigm-shifting new treatment option for patients
with PAH.”
In the PULSAR Phase 2 double-blind, placebo-controlled study,
106 patients were randomized to receive placebo, 0.3 mg/kg of
sotatercept, or 0.7 mg/kg of sotatercept subcutaneously every 21
days in combination with stable background PAH-specific therapies
over a 24-week treatment period. The echocardiography results
presented are from 94 patients and additional data analyses are
ongoing. In addition to RV-PA coupling (measured in 51 patients)
and RV function, patients treated with sotatercept experienced
improvements in a number of other cardiopulmonary measures,
including pulmonary artery systolic pressure and right ventricular
fractional area change.
Sotatercept was generally well tolerated in the trial. Adverse
events observed in the study were generally consistent with
previously published data on sotatercept in other diseases.
“As we seek to deliver a much-needed, transformative therapy to
patients with this debilitating disease, we are increasingly
encouraged by a growing body of clinical and preclinical evidence
that sotatercept does indeed have such potential,” said Habib
Dable, President and Chief Executive Officer of Acceleron, who also
referenced a preclinical research presentation at AHA showing that
a murine version of sotatercept prevented RV remodeling and
restored RV function in an animal model of severe disease.
“We look forward to confirming this early success with
sotatercept in PAH in a comprehensive Phase 3 clinical development
program, beginning with the registrational STELLAR trial, which we
expect to initiate before the end of this year,” Dable added.
Sotatercept is an investigational therapy that is not approved
for any use in any country.
The presentations referenced above are available on the
“Publications” page under the “Science & Pipeline” section of
Acceleron’s website, www.acceleronpharma.com.
*Dr. McLaughlin is an investigator in the PULSAR trial and a
paid consultant to Acceleron.
About the PULSAR Trial
The PULSAR Phase 2 trial is a randomized, double-blind,
placebo-controlled study designed to evaluate the efficacy and
safety of sotatercept in PAH patients. The primary endpoint of the
trial is the change from baseline in pulmonary vascular resistance
(PVR) over a 24-week treatment period. PVR, as measured by right
heart catheterization, is the resistance that the heart must
overcome to pump blood through the pulmonary circulatory system.
The key secondary endpoint was six-minute walk distance (6MWD); a
measure of functional capacity/endurance. Other exploratory
analyses included change in amino-terminal brain natriuretic
propeptide (NT-proBNP), a hormone secreted by cardiac muscle cells
in response to stretching caused by increased blood volume in the
heart; mean pulmonary arterial pressure, a hemodynamic measure of
average pressure in the main pulmonary arteries, which is elevated
in PAH patients; and WHO functional class. A total of 106 patients
were randomized in a 3:3:4 ratio to receive placebo, sotatercept
0.3 mg/kg, or sotatercept 0.7 mg/kg subcutaneously every 21 days
with standard-of-care therapies in combination.
Following the 6-month double-blind treatment period,
participants in the trial were eligible to continue in the 18-month
extension period.
As of October 28, 2020, 93 of 97 patients who opted to
participate in the 18-month extension period of the trial were
still enrolled; 94 patients have now been treated with sotatercept
for at least 12 months.
Conference Call and Webcast Information
The Company will host a webcast and conference call today,
November 13, 2020, at 11:00 a.m. EST, to review the presentations
of sotatercept at AHA.
The webcast will be accessible under “Events &
Presentations” in the Investors/Media page of the company's website
at www.acceleronpharma.com. Individuals can participate in the live
conference call by dialing 877-312-5848 (domestic) or 253-237-1155
(international) and referring to the “AHA Sotatercept Conference
Call.”
A replay of the webcast will be available on the Acceleron
website approximately two hours after the event.
About Sotatercept
Sotatercept is an investigational reverse-remodeling agent
designed to be a selective ligand trap for members of the TGF-beta
superfamily to rebalance BMPR-II signaling, which is a key
molecular driver of PAH. The PULSAR Phase 2 trial evaluating
sotatercept in combination with approved PAH-specific medicines in
patients with PAH achieved its primary endpoint of improvement in
pulmonary vascular resistance and its key secondary endpoint of
improvement in 6-minute walk distance. Sotatercept was generally
well tolerated in the trial. Adverse events observed in the study
were generally consistent with previously published data on
sotatercept in other diseases. Following the PULSAR results,
sotatercept was granted Breakthrough Therapy designation from the
FDA and Priority Medicines designation from the EMA in PAH.
Sotatercept is also being evaluated in the SPECTRA Phase 2
exploratory trial.
In preclinical research published in Science Translational
Medicine, sotatercept exhibited consistent effects across multiple
components of disease, including suppressed proliferation of
pulmonary arterial smooth muscle and microvascular endothelial
cells, reduced pulmonary pressures, lessened right ventricular
hypertrophy, improved right ventricular function, and attenuated
vascular remodeling.
The Company recently presented details of its Phase 3
development plan, including the design for the registrational
STELLAR trial, which is expected to be initiated before the end of
2020. Acceleron is planning two additional Phase 3 studies in
patients with PAH: the HYPERION trial, exploring early intervention
with sotatercept, and the ZENITH trial assessing later-stage
intervention.
Sotatercept is an investigational therapy that is not approved
for any use in any country. Sotatercept is part of a licensing
agreement with Bristol Myers Squibb.
About PAH
PAH is a rare and chronic, rapidly progressing disorder
characterized by the constriction of small pulmonary arteries and
elevated blood pressure in the pulmonary circulation. PAH results
in significant strain on the heart, often leading to limited
physical activity, heart failure, and reduced life expectancy. The
5-year survival rate for patients with PAH is approximately 57%.
Available therapies generally act by promoting the dilation of
pulmonary vessels without addressing the underlying cause of the
disease. As a result, PAH often progresses rapidly for many
patients despite standard of care treatment. A growing body of
research has implicated imbalances in BMP and TGF-beta signaling as
a primary driver of PAH in familial, idiopathic, and acquired forms
of the disease.
About Acceleron
Acceleron is a biopharmaceutical company dedicated to the
discovery, development, and commercialization of therapeutics to
treat serious and rare diseases. Acceleron’s leadership in the
understanding of TGF-beta superfamily biology and protein
engineering generates innovative compounds that engage the body's
ability to regulate cellular growth and repair.
Acceleron focuses its commercialization, research, and
development efforts in hematologic and pulmonary diseases. In
hematology, REBLOZYL® (luspatercept-aamt) is the first and only
erythroid maturation agent approved in the United States, Europe,
and Canada for the treatment of anemia in certain blood disorders.
REBLOZYL is part of a global collaboration partnership with Bristol
Myers Squibb. The Companies co-promote REBLOZYL in the United
States and are also developing luspatercept for the treatment of
anemia in patient populations of MDS, beta-thalassemia, and
myelofibrosis. In pulmonary, Acceleron is developing sotatercept
for the treatment of pulmonary arterial hypertension (PAH), having
recently presented positive topline results of the PULSAR Phase 2
trial. The Company is currently planning multiple Phase 3 trials
with the potential to support its long-term vision of establishing
sotatercept as a backbone therapy for patients with PAH at all
stages of the disease.
For more information, please visit www.acceleronpharma.com.
Follow Acceleron on Social Media: @AcceleronPharma and
LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements about
Acceleron’s strategy, future plans and prospects, including
statements regarding the development of sotatercept in PAH, the
timeline for clinical development and regulatory approval of
sotatercept in PAH, the expected timing for reporting of data from
ongoing clinical trials, and the potential of Acceleron’s compounds
as therapeutic drugs. The words "anticipate," "believe," "could,"
"estimate," "expect," "goal," "intend," "may," "plan," “possible,”
"potential," "project," "should," "target," "will," "would," and
similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words.
Actual results could differ materially from those included in
the forward-looking statements due to various factors, risks and
uncertainties, including, but not limited to, that preclinical
testing of Acceleron’s compounds and data from clinical trials may
not be predictive of the results or success of ongoing or later
clinical trials, that regulatory approval of Acceleron’s compounds
in one indication or country may not be predictive of approval in
another indication or country, that the development of Acceleron’s
compounds will take longer and/or cost more than planned, that
Acceleron will be unable to successfully complete the clinical
development of Acceleron’s compounds, that Acceleron may be delayed
in initiating, enrolling or completing any clinical trials, that
Acceleron’s compounds will not receive regulatory approval or
become commercially successful products, and that Breakthrough
Therapy or PRIME designation may not expedite the development or
review of sotatercept. These and other risks and uncertainties are
identified under the heading “Risk Factors” included in Acceleron’s
most recent Annual Report on Form 10-K, Quarterly Report on Form
10-Q, and other filings that Acceleron has made and may make with
the SEC in the future.
The forward-looking statements contained in this press release
are based on management's current views, plans, estimates,
assumptions, and projections with respect to future events, and
Acceleron does not undertake and specifically disclaims any
obligation to update any forward-looking statements.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20201113005115/en/
Investors: Jamie Bernard, IRC, 617-649-9650 Associate Director,
Investor Relations Media: Matt Fearer, 617-301-9557 Director,
Corporate Communications
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