– Phase 3 Studies in Myelodysplastic Syndromes
(“MEDALIST”) and Beta-thalassemia (“BELIEVE”) Outlined –
– ACE-083 Preliminary Phase 1 Results
Demonstrate 14% Increase in Muscle Volume –
– Acceleron Unveils IntelliTrap™ Platform to
Design New Class of Highly Selective Inhibitors of TGF-beta
Superfamily Ligands –
Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage
biopharmaceutical company focused on the discovery, development and
commercialization of novel therapeutic candidates that regulate
cellular growth and repair, today announced the phase 3 clinical
trial designs for the luspatercept program in myelodysplastic
syndromes (“MEDALIST” study) and beta-thalassemia (“BELIEVE”
study), phase 1 preliminary results from the ACE-083 program, and
its new IntelliTrap™ drug discovery platform.
“We are extremely excited to present the plans for Acceleron’s
first phase 3 clinical trials, the unprecedented increases in
muscle mass demonstrated in the ACE-083 phase 1 clinical trial
supporting its advancement into phase 2 trials next year and our
new IntelliTrap™ drug discovery platform which is already
generating promising new therapeutic candidates such as ACE-2494,”
said John Knopf, Ph.D., Chief Executive Officer of Acceleron.
“Acceleron is making great strides across its entire pipeline from
our late stage phase 3 programs to our highly productive discovery
organization and I am proud of the tremendous progress we are
making.”
Luspatercept Phase 3 Clinical Trials in Myelodysplastic
Syndromes (MDS) and Beta-Thalassemia
Acceleron announced that the phase 3 MDS trial will be a
double-blind, randomized, placebo-controlled study of luspatercept
in 210 very low to intermediate risk MDS patients (the “MEDALIST”
study). The primary endpoint is the proportion of patients that
become red blood cell transfusion independent (≥ 8 weeks) during
the first 24 weeks of the study.
The phase 3 beta-thalassemia trial will be a double-blind,
randomized, placebo-controlled study of luspatercept in 300
regularly transfused beta-thalassemia patients (the “BELIEVE”
study). The primary endpoint is the proportion of patients with ≥
33% reduction in transfusion burden from weeks 13 to 24 compared to
the 12 weeks preceding treatment.
Both the MEDALIST and BELIEVE studies are planned to begin by
the end of the year.
ACE-083 Phase 1 Preliminary Results
Acceleron reported positive top-line data from the phase 1
randomized, double-blind, placebo-controlled, dose-ranging study in
healthy volunteers. ACE-083 is designed to selectively increase
muscle mass and strength in the muscles in which the drug is
administered. The results showed a dose dependent increase in
muscle volume, assessed by MRI, with the highest dose level
generating a 14% increase in volume of the injected muscle, the
rectus femoris, in the thigh.
Based on these exciting results, Acceleron announced it intends
to advance ACE-083 into a phase 2 clinical trial in patients with
facioscapulohumeral muscular dystrophy in mid-2016.
IntelliTrap™ Drug Discovery Platform
Acceleron introduced its new IntelliTrap™ drug discovery
platform from which it is creating a large and diverse library of
new, selective therapeutic candidates targeting the TGF-beta
superfamily. This platform has already generated several new
therapeutic candidates including ACE-2494, a systemic muscle
therapeutic and the first clinical candidate to emerge from this
platform. Acceleron aims to initiate its first clinical trial of
ACE-2494 by the end of 2016.
A replay of the live webcast of the Research and Development Day
event will be accessible from the “Investors & Media” section
of the company's website, www.acceleronpharma.com.
About Acceleron
Acceleron is a clinical stage biopharmaceutical company focused
on the discovery, development and commercialization of novel
therapeutic candidates that regulate cellular growth and repair.
The company is a leader in understanding the biology of the
Transforming Growth Factor-Beta (TGF-beta) protein superfamily, a
large and diverse group of molecules that are key regulators in the
growth and repair of tissues throughout the human body, and in
targeting these pathways to develop important new medicines.
Acceleron has built a highly productive R&D platform that has
generated innovative clinical and preclinical therapeutic
candidates with novel mechanisms of action. These therapeutic
candidates have the potential to significantly improve clinical
outcomes for patients with cancer and rare diseases. For more
information, please visit www.acceleronpharma.com.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements about the
Company's strategy, future plans and prospects, including
statements regarding the development of the Company's compounds,
including sotatercept, luspatercept, dalantercept, ACE-083,
ACE-2494, the Company’s IntelliTrap™ drug discovery platform, and
the Company's TGF-beta superfamily program generally, the timeline
for clinical development and regulatory approval of the Company's
compounds, the expected timing for the reporting of data from
ongoing trials, and the structure of the Company's planned or
pending clinical trials. The words “anticipate,” “appear,”
“believe,” “continue,” “could,” “estimate,” “expect,” “forecast,”
“goal,” “intend,” “may,” “plan,” “potential,” “predict,” “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.
Each forward-looking statement is subject to risks and
uncertainties that could cause actual results to differ materially
from those expressed or implied in such statement. Applicable risks
and uncertainties include the risks that preclinical testing of the
Company's compounds and data from clinical trials may not be
predictive of the results or success of ongoing or later clinical
trials, that data may not be available when the Company expects it
to be, that the Company or its collaboration partner, Celgene, will
be unable to successfully complete the clinical development of the
Company’s compounds, that the development of the Company's
compounds will take longer or cost more than planned, that the
Company or Celgene may be delayed in initiating or completing any
clinical trials, and that the Company's compounds will not receive
regulatory approval or become commercially successful products.
Other risks and uncertainties include those identified under the
heading "Risk Factors" included in the Company's Annual Report on
Form 10-K which was filed with the Securities and Exchange
Commission (SEC) on March 2, 2015, and other filings that the
Company has made and may make with the SEC in the future. The
forward-looking statements contained in this press release reflect
the Company's current views with respect to future events, and the
Company does not undertake and specifically disclaims any
obligation to update any forward-looking statements.
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version on businesswire.com: http://www.businesswire.com/news/home/20151023005517/en/
Acceleron Pharma Inc.Todd James, 617-649-9393Senior Director,
Corporate CommunicationsorMedia contact:Suda Communications
LLCMaureen L. Suda, 585-387-9248
Acceleron Pharma (NASDAQ:XLRN)
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