Results from ongoing study demonstrate
increases in hemoglobin and decreases in red blood cell transfusion
burden sustained for up to 24 months, with patients still active on
treatment
Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage
biopharmaceutical company focused on the discovery, development,
and commercialization of innovative therapeutics to treat serious
and rare diseases, today announced preliminary results from the
ongoing Phase 2 study of luspatercept in patients with
beta-thalassemia during an oral presentation at the 22nd Congress
of the European Hematology Association (EHA) in Madrid, Spain.
Luspatercept is being developed to treat a range of hematologic
diseases including beta-thalassemia, myelodysplastic syndromes
(MDS), and myelofibrosis as part of a global collaboration between
Acceleron and Celgene.
“With beta-thalassemia patients now remaining on study for over
two years, we continue to be highly encouraged by luspatercept’s
long-term efficacy results and safety profile,” said Habib Dable,
President and Chief Executive Officer of Acceleron. “Combined with
the rapid completion of enrollment in the BELIEVE Phase 3 trial,
the program’s momentum continues to build alongside our enthusiasm
to potentially transform the treatment of beta-thalassemia patients
globally.”
Phase 2 Results
A total of 32 transfusion-dependent beta-thalassemia patients
have been treated with therapeutic dose levels of luspatercept in
the ongoing study.
- 69% (22 of 32) achieved a reduction in
red blood cell (RBC) transfusion burden of at least 33% in any
12-week treatment interval as compared to baseline.
A 12-week fixed interval analysis was conducted to review RBC
transfusion reduction during weeks 13 to 24 and weeks 37 to 48
compared to the baseline 12-week period pre-treatment in order to
evaluate durability of response. The ongoing BELIEVE Phase 3 trial
will use this 12-week fixed interval analysis for evaluating the
proportion of patients achieving at least a 33% reduction in RBC
transfusion burden.
- 50% (12 of 24 patients with 6-20 units
RBC / 24 weeks estimated pre-treatment) achieved a reduction in RBC
transfusion burden of at least 33% in the fixed 12-week interval
from weeks 13 to 24 as compared to baseline.
- 46% (11 of 24 patients with 6-20 units
RBC / 24 weeks estimated pre-treatment) achieved a reduction in RBC
transfusion burden of at least 33% in the fixed 12-week interval
from weeks 37 to 48 as compared to baseline.
A total of 31 non-transfusion-dependent beta-thalassemia
patients have been treated with therapeutic dose levels of
luspatercept in the ongoing study.
- 71% (22 of 31) achieved a clinically
meaningful increase in hemoglobin of at least 1.0 g/dL compared to
baseline (mean increase over 12 weeks).
There are patients who remain on luspatercept with clinically
meaningful increases in hemoglobin and reductions in RBC
transfusion burden for up to 24 months.
Phase 2 Safety Summary
A total of 64 beta-thalassemia patients have been treated with
luspatercept in the ongoing Phase 2 studies (all dose levels).
- The majority of adverse events (AEs)
were Grade 1 or 2. The most common related AEs (occurring in ≥ 10%
of patients) were bone pain, headache, myalgia, arthralgia,
musculoskeletal pain, asthenia, injection site pain, and back
pain.
- Grade 3 AEs probably related to study
drug were bone pain (n=3), asthenia (n=2) and headache (n=1).
- There were no serious AEs related to
study drug.
“Beta-thalassemia remains an area of critical medical need for
many patients around the world,” said Michael Pehl, President,
Hematology/Oncology for Celgene. “These longer-term results
continue to illustrate the potential for luspatercept to affect
transfusion dependence and hemoglobin levels, making a meaningful
impact for patients with this serious blood disease.”
Luspatercept is an investigational product that is not approved
for use in any country.
The BELIEVE trial, a global Phase 3 study of luspatercept in
transfusion-dependent beta-thalassemia patients, is fully enrolled
and top-line results are expected in mid-2018.
The EHA beta-thalassemia presentation is available under the
Science page of the Company’s website at
www.acceleronpharma.com/.
About the Phase 2 Study
Data from two open-label Phase 2 studies were presented at the
conference: the base study in which patients received treatment
with luspatercept for three months and the ongoing long-term safety
extension study in which patients may receive treatment with
luspatercept for up to an additional five years. In both the
three-month base study and the long-term extension study, red blood
cell (RBC) transfusion-dependent patients (≥ 4 units RBC / 8 weeks)
and non-transfusion-dependent patients (< 4 units RBC / 8 weeks)
were enrolled and treated with open-label luspatercept, dosed
subcutaneously once every three weeks.
The primary outcome measure of the three-month base study was
the proportion of patients who have an erythroid response, defined
as 1) a hemoglobin increase of ≥ 1.5 g/dL from baseline for ≥ 14
days (in the absence of RBC transfusions) in non-transfusion
dependent patients, or 2) ≥ 20% reduction in RBC transfusion burden
compared to pretreatment in transfusion-dependent patients. The
primary outcome for the long-term extension study is to evaluate
the long-term safety and tolerability of luspatercept.
About Luspatercept
Luspatercept is a modified activin receptor type IIB fusion
protein that acts as a ligand trap for members in the transforming
growth factor-beta superfamily involved in the late stages of
erythropoiesis (red blood cell production). Luspatercept regulates
late-stage erythrocyte (red blood cell) precursor cell
differentiation and maturation. This mechanism of action is
distinct from that of erythropoiesis stimulating agents (ESAs),
which stimulate the proliferation of early-stage erythrocyte
precursor cells. Acceleron and Celgene are jointly developing
luspatercept as part of a global collaboration. Phase 3 clinical
trials are underway to evaluate the safety and efficacy of
luspatercept in patients with myelodysplastic syndromes (the
“MEDALIST” study) and in patients with beta-thalassemia (the
“BELIEVE” study). For more information, please visit
www.clinicaltrials.gov.
About Acceleron
Acceleron is a clinical stage biopharmaceutical company focused
on the discovery, development and commercialization of innovative
therapeutics to treat serious and rare diseases. Its pioneering
research platform leverages the powerful biology behind the body’s
ability to rebuild and repair its own cells and tissues. This
approach to drug discovery has generated four therapeutic
candidates that are currently in clinical trials. The Company’s
lead therapeutic candidate, luspatercept, is being evaluated in
Phase 3 studies for the treatment of the hematologic diseases
myelodysplastic syndromes (MDS) and beta-thalassemia under a global
partnership with Celgene. Acceleron is also advancing its ACE-083
clinical program in the field of neuromuscular disease, and has a
comprehensive preclinical research effort targeting fibrotic and
other serious diseases.
For more information, please
visit www.acceleronpharma.com/. Follow Acceleron on Social
Media: @AcceleronPharma and LinkedIn.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements about
Acceleron's strategy, future plans and prospects, including
statements regarding the development of luspatercept, the timeline
for clinical development and regulatory approval of Acceleron’s
compounds, the expected timing for the reporting of data from
ongoing trials, and the structure of Acceleron’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
Acceleron’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 Acceleron expects it to
be, that Acceleron or its collaboration partner, Celgene, will be
unable to successfully complete the clinical development of
Acceleron’s compounds, that the development of Acceleron’s
compounds will take longer or cost more than planned, that
Acceleron or Celgene may be delayed in initiating or completing any
clinical trials, and that Acceleron'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 Acceleron's Annual Report on
Form 10-K which was filed with the Securities and Exchange
Commission (SEC) on March 1, 2017, 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 reflect
Acceleron’s current views 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: http://www.businesswire.com/news/home/20170622006298/en/
Acceleron Pharma Inc.Todd James, IRC, 617-649-9393Vice
President, Investor Relations and Corporate CommunicationsorCandice
Ellis, 617-649-9226Manager, Investor Relations and Corporate
CommunicationsorMedia:BMC CommunicationsBrad Miles,
646-513-3125
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