– Results show patients treated with
sotatercept experienced improvements in hemodynamic and functional
endpoints compared with placebo –
– Sotatercept was generally well tolerated;
adverse events were consistent with previously published data on
sotatercept in clinical trials of other patient populations –
Acceleron Pharma Inc. (Nasdaq: XLRN), a biopharmaceutical
company dedicated to the discovery, development, and
commercialization of TGF-beta superfamily therapeutics to treat
serious and rare diseases, today announced that the New England
Journal of Medicine has published results of the PULSAR Phase 2
trial of sotatercept in patients with pulmonary arterial
hypertension (PAH).
“We couldn’t be more excited that the editors of the New England
Journal of Medicine have selected the results of the PULSAR trial
for publication,” said Habib Dable, President and Chief Executive
Officer of Acceleron. “With our STELLAR Phase 3 registrational
trial of sotatercept now enrolling patients with PAH, having such
robust data highlighted in one of the world’s most prestigious
peer-reviewed medical journals will help raise awareness of
sotatercept’s potential to alter treatment paradigms for this
disease of high unmet medical need.”
During a special session of the American Thoracic Society 2020
virtual meeting last June, study investigators reported that
patients on stable background PAH-specific therapies treated with
sotatercept experienced a statistically significant reduction in
pulmonary vascular resistance, the trial’s primary endpoint, at
week 24 versus placebo. The trial also achieved an all-dose mean
improvement from baseline in the key secondary endpoint of
six-minute walk distance.
“The PULSAR data show that sotatercept has the potential to
deliver significant benefit on top of currently available
therapies,” said Dr. Marc Humbert*, Professor of Medicine and
Director of the French Pulmonary Hypertension Reference Center at
the Université Paris-Saclay and a first author of the New England
Journal of Medicine article. “This is a result that suggests that
sotatercept could bring an important new mechanistic approach to
the treatment of patients with PAH.”
Sotatercept was generally well tolerated in the trial. Adverse
events observed in the study were generally consistent with
previously published data on sotatercept in clinical trials in
other patient populations. In addition to the ongoing STELLAR
trial, Acceleron is planning two additional Phase 3 studies in
PAH—HYPERION and ZENITH—evaluating sotatercept in newly diagnosed
patients and in patients diagnosed with World Health Organization
(WHO) functional class IV disease, respectively.
The PULSAR publication marks the third time that trial results
of an Acceleron-discovered therapy have been published in the New
England Journal of Medicine in the past 15 months. In the first
quarter of 2020, the journal published results of the pivotal Phase
3 MEDALIST and BELIEVE trials of REBLOZYL® (luspatercept-aamt), an
Acceleron-discovered erythroid maturation agent, in populations of
patients with myelodysplastic syndromes and beta-thalassemia,
respectively.
*Dr. Humbert 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 patients with PAH. The primary endpoint of
the trial was 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 three weeks on top of standard-of-care
therapies.
Following the 6-month double-blind treatment period,
participants in the trial were eligible to continue in the ongoing
open-label extension period of the study. Of the 97 patients
initially enrolled in the extension, 91 continue to
participate.
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.
The Company recently presented details of its Phase 3
development plan, including the design for the registrational
STELLAR trial, which is currently enrolling patients with PAH.
Acceleron is planning two additional Phase 3 studies in patients
with PAH: the HYPERION trial in newly diagnosed patients, and the
ZENITH trial assessing intervention in patients diagnosed with WHO
functional class IV disease.
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 research, development, and
commercialization efforts in pulmonary and hematologic diseases. In
pulmonary, Acceleron is developing sotatercept for the treatment of
pulmonary arterial hypertension (PAH), having reported 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. Acceleron is also investigating the potential of its
early-stage pulmonary candidate, ACE-1334, which it plans to
advance into a Phase 1b/Phase 2 trial in systemic
sclerosis-associated interstitial lung disease (SSc-ILD) this
year.
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 myelodysplastic
syndromes, beta-thalassemia, and myelofibrosis.
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 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/20210401005264/en/
Investors: Jamie Bernard, IRC, 617-301-9650 Associate Director,
Investor Relations
Media: Matt Fearer, 617-301-9557 Senior Director, Corporate
Communications
Acceleron Pharma (NASDAQ:XLRN)
Gráfica de Acción Histórica
De Jun 2024 a Jul 2024
Acceleron Pharma (NASDAQ:XLRN)
Gráfica de Acción Histórica
De Jul 2023 a Jul 2024