– Company plans to initiate Phase 1b/Phase 2
trial in patients with systemic sclerosis-associated interstitial
lung disease (SSc-ILD) in 2021 –
– ACE-1334 previously received Fast Track
designation from the FDA –
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 United States
Food and Drug Administration (FDA) has granted Orphan Drug
designation to ACE-1334 for the treatment of patients with systemic
sclerosis. The Company intends to initiate a Phase 1b/Phase 2 trial
in patients with systemic sclerosis-associated interstitial lung
disease (SSc-ILD) in 2021.
“We’re excited that ACE-1334 has received Orphan Drug
designation, as it aligns with our vision to develop novel
therapies for pulmonary diseases of high unmet medical need,” said
Habib Dable, President and Chief Executive Officer of Acceleron.
“It’s clear that 2021 will be an important year in this regard, as
we seek to expand our portfolio in this important therapeutic area.
In addition to advancing our Phase 3 clinical program to
investigate sotatercept’s potential to reverse vascular remodeling
associated with pulmonary arterial hypertension, we’re pleased to
be planning for a Phase 1b/Phase 2 study to determine whether the
anti-fibrotic activity ACE-1334 has demonstrated in preclinical
models of fibrosis can be replicated in patients with SSc-ILD.”
Orphan designation is granted by the FDA Office of Orphan
Products Development to advance the evaluation and development of
safe and effective therapies for the treatment of rare diseases or
conditions affecting fewer than 200,000 people in the U.S. Under
the Orphan Drug Act, the FDA may provide grant funding toward
clinical trial costs, tax advantages, FDA user-fee benefits, and
seven years of market exclusivity in the United States following
marketing approval by the FDA. The granting of an orphan
designation request does not alter the standard regulatory
requirements and process for obtaining marketing approval. For more
information about orphan designation, please visit the FDA
website.
ACE-1334 is an investigational therapy that is not approved for
any use in any country.
About ACE-1334
ACE-1334 is an Acceleron-discovered, TGF-beta superfamily-based
ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands
but not TGF-beta 2. TGF-beta 1 and 3 are believed to be key
signaling factors in the pathogenesis of fibrotic disease. ACE-1334
has shown robust anti-fibrotic activity in multiple preclinical
models of fibrosis. ACE-1334 recently completed an ascending-dose
Phase 1 clinical trial in healthy volunteers. The FDA has granted
Fast Track designation to ACE-1334 in patients with systemic
sclerosis-associated interstitial lung disease (SSc-ILD) as well as
Orphan Drug designation for the treatment of systemic
sclerosis.
About SSc-ILD
Systemic sclerosis-associated interstitial lung disease
(SSc-ILD; also known as systemic scleroderma-associated
interstitial lung disease) is a rare, progressive, autoimmune
connective tissue disorder characterized by immune dysregulation.
It is reported to be the most common cause of death among patients
with systemic scleroderma. Patients with SSc-ILD ultimately
experience severely compromised pulmonary function, with the
disease initially causing cough, shortness of breath, and fatigue.
SSc-ILD, which affects an estimated 50,000 patients in the United
States and Europe, is thought to arise from a combination of
genetic susceptibility, autoimmunity, inflammation, fibrotic
activity, and vascular insult. The disease has an estimated 10-year
survival rate of approximately 56%. Current treatment for SSc-ILD
includes a range of potentially high-risk approaches, including
immunosuppression, hematopoietic stem cell transplantation, and
lung transplantation.
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) 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 ACE-1334, 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, and
that Acceleron’s compounds will not receive regulatory approval or
become commercially successful products. 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 Reports 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20201201005389/en/
Acceleron Pharma Inc.
Investors: Jamie Bernard, IRC, 617-649-9650 Associate Director,
Investor Relations
Media: Matt Fearer, 617-301-9557 Director, Corporate
Communications
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