– Treatment with sotatercept in the ongoing
SPECTRA Phase 2 trial was associated with improvements in resting
and exercise hemodynamics at week 24 –
– Sotatercept was generally well tolerated,
consistent with the previously reported safety profile 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 leading
biopharmaceutical company in 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) preliminary
interim data from the SPECTRA Phase 2 trial of sotatercept in
patients with pulmonary arterial hypertension (PAH).
The findings, presented during the session “Clinical Advances in
Pulmonary Hypertension: Lessons from Best Abstracts,” included
outcomes obtained from the first 10 patients evaluated among a
total of 21 trial participants. These preliminary data from the
ongoing trial, which is designed to assess resting and exercise
hemodynamics and peak oxygen uptake—as recorded by invasive
cardiopulmonary exercise testing (iCPET)—show that patients treated
with sotatercept experienced improvements in multiple key
hemodynamic measures.
“Despite the relatively small number of patients evaluated to
date, the consistency and scale of improvements seen in a range of
clinically meaningful measures are very encouraging,” said Aaron
Waxman, M.D., Ph.D.*, Director, Pulmonary Vascular Disease Program
at Boston’s Brigham and Women’s Hospital, who presented at ATS
2021. “Our analyses are ongoing, but observing such beneficial
changes among heavily pretreated patients with fairly advanced
disease suggests that sotatercept may be affecting the underlying
pathology of PAH.”
In this single-arm, open-label multi-center exploratory study, a
total of 21 patients with advanced PAH (classified as WHO
functional class III) on stable combination background therapy are
to be treated with an initial cycle of 0.3 mg/kg of sotatercept
delivered subcutaneously, followed by subsequent cycles of 0.7
mg/kg of sotatercept through a 24-week treatment period.
As measured by iCPET at baseline and at week 24, investigators
recorded improvements in peak oxygen uptake or VO2 max (the primary
endpoint) as well as in a range of secondary endpoints, including
ventilatory efficiency, total workload, and arteriovenous oxygen
content. Previously reported improvements in resting hemodynamics
included reductions in pulmonary vascular resistance (PVR; as
measured in dynes-sec/cm5) from a mean of 576 at baseline to 369 at
week 24 (35.9% reduction) and mean pulmonary arterial pressure
(mPAP) as measured in mmHg from 43.4 to 30.6 (29.5% reduction).
In addition, six-minute walk distance (6MWD) increased by an
average of 72.4 meters from baseline to week 24 in nine patients
for whom data were available.
Sotatercept was generally well tolerated in the trial. Adverse
events observed in the study were generally consistent with
previously published data on sotatercept in PAH and in other
diseases. As of an interim data cut-off of February 25, 2021,
treatment-emergent adverse events (TEAEs) were reported in 16 of 21
patients (76%). Serious TEAEs were reported in three patients
(14%), but none was considered related to the study drug and none
required dose interruption or reduction.
“It’s exciting to be able to add these positive results from the
SPECTRA trial to the growing body of clinical and preclinical
evidence showing that sotatercept has the potential to transform
the lives of patients with PAH by introducing a novel mechanism of
action to the treatment paradigm,” said Habib Dable, President and
Chief Executive Officer of Acceleron. “With our comprehensive Phase
3 clinical program now underway, we are on track to achieve our
vision of developing sotatercept as a backbone therapy for patients
with PAH across all stages of disease.”
Sotatercept is an investigational therapy that is not approved
for any use in any country.
Dr. Waxman’s detailed presentation is available on the
“Publications” page under the “Science & Pipeline” section of
Acceleron’s website, www.acceleronpharma.com.
*Dr. Waxman is the principal investigator of the SPECTRA trial
and a paid consultant to Acceleron.
About the SPECTRA Trial
The SPECTRA Phase 2 trial is a single arm, open-label,
multi-center exploratory study to determine the effects of
sotatercept plus standard of care in adults with WHO functional
class III PAH. The primary endpoint of the trial is the change from
baseline in peak oxygen uptake (VO2 max) at 24 weeks, as recorded
by invasive cardiopulmonary exercise testing (iCPET). Secondary
hemodynamic endpoints as well as endpoints of exercise capacity and
tolerance assessed via iCPET and right heart catheterization
include change from baseline at 24 weeks in: ventilatory efficiency
(VE/VCO2 slope); cardiac index (L/min/m2); mean pulmonary artery
pressure (mPAP); pulmonary vascular resistance (PVR); arteriovenous
oxygen content difference (Ca-vO2); ventilatory efficiency; “dead
space” assessment (VE/VCO2 slope); and oxygen consumption at
anaerobic threshold (VO2 at AT).
A total of 21 patients are to receive stable background
combination PAH therapy plus sotatercept at a starting dose level
of 0.3 mg/kg delivered subcutaneously for one cycle, escalating to
0.7 mg/kg at cycle 2 for the remainder of the treatment period.
Following the 6-month open-label treatment period, participants in
the trial are eligible to continue in the 18-month extension
period, which includes iCPET conducted at 48 weeks.
Conference Call and Webcast Information
The Company will host a webcast and conference call today, May
19, 2021, at 10:30 a.m. EDT, to discuss results of the
presentations at ATS 2021.
The webcast will be accessible under "Events &
Presentations" in the Investors & Media page of the Company’s
website at www.acceleronpharma.com. To participate in the
conference call, please dial 833-494-1483 (domestic) or
236-714-2620 (international) and reference code #6565123.
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 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 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/20210519005172/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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