– Treatment with sotatercept in first set of
patients in the ongoing SPECTRA Phase 2 trial was associated with
substantial improvements in hemodynamics, exercise tolerance and
exercise capacity at week 24 –
– Sotatercept was generally well tolerated,
consistent with the previously reported safety profile in PAH and
in other diseases –
– 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 a set of preliminary
interim data from the ongoing SPECTRA Phase 2 trial of sotatercept
in patients with pulmonary arterial hypertension (PAH).
Initial data from the 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 substantial
improvements in multiple hemodynamic measures as well as in
exercise tolerance and exercise capacity. These early outcomes,
obtained from the first 10 patients participating in the trial,
were shared as part of an Invited Talk entitled, “SPECTRA and
Beyond: Signs of Disease Modification?” presented virtually during
the American Heart Association (AHA) 2020 Scientific Sessions.
Among the largest resting hemodynamic improvements observed were
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).
“The SPECTRA trial’s innovative design is meant to help us
better understand and detect sotatercept’s potential effects on
underlying disease pathology,” said Aaron Waxman, M.D., Ph.D.*,
Director, Pulmonary Vascular Disease Program at Boston’s Brigham
and Women’s Hospital, who delivered the Invited Talk at the AHA.
“Despite the limited number of patients evaluated to date, it’s
difficult not to be encouraged by the range and extent of positive
changes seen in key hemodynamic and exercise capacity measures thus
far.”
In this single-arm, open-label multi-center exploratory study, a
total of up to 25 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. The
protocol includes iCPET at baseline and at week 24 to assess change
from in peak oxygen uptake or VO2 max (the primary endpoint) as
well as changes from baseline in a range of secondary endpoints,
including mPAP and VO2 at anaerobic threshold.
During his AHA talk, Dr. Waxman also provided an in-depth
profile of the first patient treated in the SPECTRA trial: a
25-year-old woman diagnosed with idiopathic PAH nearly five years
prior to enrollment. This patient, who was classified as WHO
functional class III and on combination background therapy,
experienced substantial hemodynamic and functional improvements.
Perhaps most notably, this patient was reclassified from WHO
functional class III at baseline to class I at week 24. The patient
retained functional class I status in follow-up testing at 48
weeks.
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.
“We’re thrilled to see such positive preliminary outcomes from
the SPECTRA trial, which serves as another important exploration of
sotatercept and the potential of its unique mechanism to alter the
course and treatment of PAH,” said Habib Dable, President and Chief
Executive Officer of Acceleron. “These results, combined with new
PULSAR trial data presented at AHA and the topline PULSAR findings
announced earlier this year, position us well to initiate a robust
Phase 3 development program to realize our vision of 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.
The presentation referenced above 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 up to 25 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,
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/20201113005114/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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