- Interim longer term data with investigational
drug luspatercept show sustained increases in hemoglobin levels,
and reduced transfusion burden in patients with beta-thalassemia
–
Acceleron Pharma Inc. (NASDAQ: XLRN) and Celgene Corporation
(NASDAQ: CELG) today announced Phase 2 results from an open-label
three-month base study and the ongoing long-term safety extension
study with luspatercept in patients with beta-thalassemia during an
oral presentation at the 58th Annual Meeting of the American
Society of Hematology (ASH) in San Diego, California. Luspatercept
is being developed as part of the global collaboration between
Acceleron and Celgene.
“Beta-thalassemia is a severe, chronic disease with no
pharmaceutical treatment options to correct or improve the
underlying anemia in patients,” said Michael Pehl, President,
Hematology and Oncology for Celgene. “These longer term
luspatercept Phase 2 data are encouraging, and we are continuing to
enroll patients in the Phase 3 BELIEVE study in transfusion
dependent beta-thalassemia patients.”
Luspatercept Beta-Thalassemia Data
Presented at ASH
Results in Transfusion Dependent (TD) Beta-Thalassemia
Patients
RBC transfusion reductionover
any 12 weeks versus 12weeks pre-treatment
Response rate (% of patients)
3-month base study
(n=31)
Long-term extension study
(n=24)
≥ 20% 81% (25/31) 96%
(23/24)
≥ 33% 71% (22/31)
83% (20/24)
≥ 50% 55% (17/31)
71% (17/24)
Results in Non-Transfusion Dependent (NTD) Beta-Thalassemia
Patients
Hemoglobin (Hb) response overany
12 weeks versus 12 weekspre-treatment
Response rate (% of patients)
in patients treated with ≥ 0.6
mg/kg
3-month base study
(n=21)
Long-term extension study
(n=27)
Increase in mean Hb ≥ 1.0 g/dL 62%
(14/21) 78% (21/27)
Increase in mean Hb ≥
1.5 g/dL 33% (7/21) 52% (14/27)
In the long-term extension study, the median duration of a
hemoglobin increase ≥ 1.0 g/dL maintained for at least 12 weeks in
responders is 13.5 months (N=21) with treatment still ongoing.
Safety
- There were no related serious adverse
events and related grade 3 adverse events included: bone pain (n=2
base, n=1 extension), asthenia (n=2 base) and headache (n=1
extension)
- The most common related adverse events
(all grades) were bone pain, myalgia, headache, musculoskeletal
pain, arthralgia, and injection site pain.
Luspatercept is an investigational product that is not approved
for use in any country.
The BELIEVE Trial, a global Phase 3, double-blind, randomized,
placebo-controlled, multicenter study in transfusion dependent
beta-thalassemia patients, is currently enrolling.
The slides from the ASH beta-thalassemia presentation will be
available immediately following the presentation at the conference
on Acceleron's website (www.acceleronpharma.com) under the Science
tab.
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 (TGF-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 erythropoietin (EPO), which stimulates the
proliferation of early-stage erythrocyte precursor cells. Acceleron
and Celgene are jointly developing luspatercept as part of a global
collaboration. Acceleron and Celgene are enrolling Phase 3 clinical
trials that are designed 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 Corp. Acceleron is also advancing clinical
programs in the fields of oncology and neuromuscular diseases 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.
About Celgene
Celgene Corporation, headquartered in Summit, New
Jersey, is an integrated global biopharmaceutical company engaged
primarily in the discovery, development and commercialization of
innovative therapies for the treatment of cancer and inflammatory
diseases through next-generation solutions in protein homeostasis,
immuno-oncology, epigenetics, immunology and neuro-inflammation.
For more information, please visit www.celgene.com. Follow
Celgene on Social
Media: @Celgene, Pinterest, LinkedIn, FaceBook and YouTube.
Forward-Looking Statements
Acceleron: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 February 25, 2016, 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.
Celgene:
Cautionary Note Regarding Forward-Looking StatementsThis press
release contains forward-looking statements within the meaning of
The Private Securities Litigation Reform Act of 1995. Such
forward-looking statements include those regarding the potential
benefits of, and plans relating to the collaboration between
Acceleron and Celgene; the potential of luspatercept as a
therapeutic drug; and the benefit of each company’s strategic plans
and focus. The words “anticipate,” “believe,” “estimate,” “expect,”
“intend,” “may,” “plan,” “predict,” “project,” “would,” “could,”
“potential,” “possible,” “hope” and similar expressions are
intended to identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Such
statements are subject to numerous important factors, risks and
uncertainties that may cause actual events or results to differ
materially from current expectations and beliefs. For example,
there can be no guarantee that any product candidate will be
successfully developed or complete necessary preclinical and
clinical phases, or that development of any of product candidates
will successfully continue. There can be no guarantee that any
positive developments will result in stock price appreciation.
Management's expectations and, therefore, any forward-looking
statements in this press release could also be affected by risks
and uncertainties relating to a number of other important factors,
including: results of clinical trials and preclinical studies,
including subsequent analysis of existing data and new data
received from ongoing and future studies; the content and timing of
decisions made by the U.S. FDA and other regulatory authorities,
investigational review boards at clinical trial sites and
publication review bodies; the ability to obtain and maintain
requisite regulatory approvals and to enroll patients in planned
clinical trials; unplanned cash requirements and expenditures;
competitive factors; the ability to obtain, maintain and enforce
patent and other intellectual property protection for any product
candidates ; the ability to maintain key collaborations; and
general economic and market conditions. These and other risks are
described in greater detail under the caption "Risk Factors"
included in each company’s public filings with the Securities and
Exchange Commission. Any forward-looking statements contained in
this press release speak only as of the date hereof, and neither
company has any obligation to update any forward-looking
statements, whether as a result of new information, future events
or otherwise, except as may be required by law.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20161205006339/en/
For Acceleron:Investors:Todd James,
IRC, (617) 649-9393Senior Director, Investor Relations and
Corporate CommunicationsorMedia:BMC CommunicationsBrad Miles, (917)
570-7340orFor Celgene:Investors:(908)
673-9628 investors@celgene.comorMedia:(908) 673-2275
media@celgene.com
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