– 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 –
CAMBRIDGE, Mass.--(BUSINESS WIRE)--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.
“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.”
“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, .
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 .
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.
Contacts
Acceleron Pharma Inc.Todd James, 617-649-9393Senior
Director, Corporate CommunicationsorMedia contact:Suda
Communications LLCMaureen L. Suda, 585-387-9248
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