– Sotatercept received Orphan Drug designation
from the U.S. Food and Drug Administration (FDA) in PAH in 2019
–
– In the spring of 2020, sotatercept received
Breakthrough Therapy designation from the FDA and Priority
Medicines (PRIME) designation from the European Medicines Agency
(EMA), both in PAH –
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 European
Commission (EC) has granted orphan designation to sotatercept for
the treatment of patients with pulmonary arterial hypertension
(PAH).
“We’re thrilled at the European Commission’s decision to grant
orphan designation to sotatercept in PAH,” said Habib Dable,
President and Chief Executive Officer of Acceleron. “We fully
intend to take advantage of the benefits that this and other
special statuses—including Orphan Drug and Breakthrough Therapy
designations in the United States and PRIME designation in
Europe—provide to drug developers as we work with health
authorities to deliver this potential new backbone therapy in PAH
to patients in need as quickly as possible.”
The EC grants orphan designation to medicines intended to treat,
prevent or diagnose a disease of low prevalence (fewer than 5
individuals per 10,000 population) that is life-threatening or
chronically debilitating. To encourage the development of such
medicines, the designation carries with it certain incentives,
including scientific advice and assistance with clinical trial
protocols, and the potential for a 10-year period of market
exclusivity. For additional information about orphan designation,
visit the EMA website.
Acceleron is currently advancing a Phase 3 development plan for
sotatercept, beginning with the registrational trial known as
STELLAR expected to initiate by the end of this year.
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 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 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/2 trial in systemic sclerosis-associated
interstitial lung disease (SSc-ILD) next 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, 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.
Source: Acceleron Pharma Inc.
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version on businesswire.com: https://www.businesswire.com/news/home/20201214005111/en/
Investors: Jamie Bernard, IRC, 617-301-9650 Associate Director,
Investor Relations
Media: Matt Fearer, 617-301-9557 Director, Corporate
Communications
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