- Data presented show maintained or improved
responses in multiple efficacy endpoints among patients treated
with sotatercept plus stable background therapy for up to 48 weeks
-
- Patients receiving placebo plus stable
background therapy during the 24-week placebo-controlled period who
were re-randomized to sotatercept plus stable background therapy
during weeks 24 to 48 experienced improvements consistent with the
initial results from the placebo-controlled treatment period -
- Sotatercept was generally well tolerated;
adverse events were consistent with previously published data on
sotatercept in clinical trials in PAH and in other diseases -
- Company-hosted investor and analyst
conference call and webcast with guest PAH key opinion leader to be
held today, Wednesday, May 19th at 10:30 a.m. EDT -
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 presented at the American Thoracic Society 2021
International Conference (ATS 2021) interim results from the
open-label extension of the PULSAR Phase 2 trial of sotatercept in
patients with pulmonary arterial hypertension (PAH).
During the ATS 2021 session “Clinical Advances in Pulmonary
Hypertension: Lessons from Best Abstracts,” investigators reported
that patients in the open-label extension period of the trial on
stable background PAH-specific therapies experienced consistent or
improved responses in multiple efficacy endpoints when treated with
sotatercept for up to 48 weeks. Additionally, patients
re-randomized to receive sotatercept on top of background therapies
during weeks 24 to 48—after receiving placebo plus stable
background therapy during the first 24 weeks of the
trial—experienced clinical improvements consistent with those seen
in the initial placebo-controlled treatment period.
“It’s truly exciting to be able to share data showing that
patients with PAH can continue to benefit from ongoing treatment
with sotatercept,” said Habib Dable, President and Chief Executive
Officer of Acceleron. “This duration of response among
sotatercept-treated patients and demonstrable clinical efficacy in
patients emerging from the placebo group further solidify the
potential of sotatercept and its novel mechanism of action to offer
an important new approach to treating a disease whose unmet need
remains substantial.”
In the open-label extension period of the trial, investigators
observed maintained or enhanced responses with sotatercept
treatment in multiple study endpoints evaluated at week 48,
including six-minute walk distance (6MWD) and World Health
Organization (WHO) functional class. Patients treated with
sotatercept in all cohorts also experienced reductions in levels of
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.
“These longer-term results point to the potential utility of
sotatercept as a novel treatment for PAH,” said Dr. David Badesch†,
Professor of Medicine and Clinical Director of the Pulmonary
Hypertension Center at the University of Colorado.
Badesch, who presented this open-label data during today’s ATS
2021 session, continued: “Notably, we observed this clinical
efficacy on top of current therapies in a population of heavily
pre-treated patients. This suggests that sotatercept is acting in a
manner distinct from other agents, possibly by balancing pro- and
anti-proliferative signaling in pathways known to be relevant in
PAH biology. We look forward to collecting additional, potentially
confirmatory data through this ongoing open-label extension study
and in the next phase of clinical development.”
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 PAH,
in other diseases, and in the placebo-controlled treatment period.
Overall in the trial, serious treatment-emergent adverse events
(TEAEs) were reported in 28% of patients and 9% of patients had
TEAEs that led to study discontinuation.
As of May 18, 2021, 90 of 97 patients who opted to participate
in the ongoing open-label extension period of the trial were still
enrolled. All 90 patients have been treated with sotatercept for at
least 18 months with 69 patients completing 24 months of
treatment.
Dr. Badesch’s detailed presentation from the open-label
extension of the PULSAR Phase 2 trial of sotatercept at ATS 2021 is
available in the Publications section under “Science &
Pipeline” on the Company’s website at acceleronpharma.com.
Sotatercept is an investigational therapy that is not approved
for any use in any country.
†Dr. Badesch is the principal investigator of 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.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss
results from presentations at ATS 2021 today, May 19, 2021, at
10:30 a.m. EDT.
The webcast will be accessible under "Events &
Presentations" in the Investors & Media page of the Company’s
website at acceleronpharma.com. To participate in the conference
call, please dial 833-494-1483 (domestic) or 236-714-2620
(international) and reference code #6565123.
The archived webcast will be available for replay 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 signaling in the BMP pathway, which is a
key molecular driver of PAH. In preclinical studies, sotatercept
was shown to reverse the vascular remodeling that is a hallmark 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, which were published in a
recent edition of the New England Journal of Medicine, 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
World Health Organization (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 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/20210519005163/en/
Investors: Jamie Bernard, IRC, 617-301-9650 Associate Director,
Investor Relations
Media: Matt Fearer, 617-301-9557 Senior Director, Corporate
Communications
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