- Preliminary results show that treatment with investigational drug
sotatercept can increase hemoglobin and achieve transfusion independence
in patients with myelofibrosis -
- Acceleron to host conference call and live webcast on Monday,
December 5th at 9:00 a.m. EST (6:00 a.m. PST) -
CAMBRIDGE, Mass. & SUMMIT, N.J.--(BUSINESS WIRE)--
Acceleron Pharma Inc. (NASDAQ:XLRN) and Celgene Corporation
(NASDAQ:CELG), today announced preliminary results from an ongoing
investigator initiated Phase 2 study with investigational drug
sotatercept in patients with myelofibrosis at the 58th Annual
Meeting of the American Society of Hematology (ASH) in San Diego,
California. Sotatercept is being developed as part of the global
collaboration between Acceleron and Celgene.
"These initial and encouraging data support our efforts for further
clinical development work with our Acceleron-partnered programs in
myelofibrosis," said Michael Pehl, President, Hematology and Oncology
for Celgene. "We believe these programs have the potential to address a
major unmet need in patients with myelofibrosis."
Highlights of the Sotatercept Myelofibrosis Phase 2 Data Presented at
19 patients were enrolled and treated with sotatercept (12 patients at
0.75 mg/kg and 7 at 1 mg/kg) and 14 of these patients have received at
least 5 doses of sotatercept and are evaluable for response
36% (5/14) of the evaluable patients achieved an anemia response,
defined as a composite of RBC-transfusion-independence and hemoglobin
response (increase of ≥1.5 g/dL from baseline on every determination
consecutively over ≥84 days without RBC transfusions).
Most adverse events were grades 1 or 2.
Adverse events at least possibly related to study drug that occurred
include grade 3 hypertension leading to discontinuation, grade 1
myalgia, bone pain, pain in extremity and injection site reaction.
13 patients have discontinued from the study: 5 due to no response, 2
proceeded to stem cell transplantation, 2 had MF progression, 1
transformed to AML, 1 each withdrew consent, had unrelated medical
problems and hypertension
Sotatercept is an investigational product that is not approved for use
in any country.
Acceleron ASH Conference Call Information
Acceleron will host a conference call and live webcast to discuss data
presented at the ASH meeting on December 5, 2016, at 9:00 a.m. EST (6:00
a.m. PST). To participate by teleconference, please dial 877-312-5848
(domestic) or 253-237-1155 (international) and refer to the Acceleron
To access the live webcast, please select "Events & Presentations" in
the Investors section on Acceleron's website (www.acceleronpharma.com)
at least 10 minutes beforehand to ensure time for any downloads that may
An archived webcast recording will be available on the Acceleron website
beginning approximately two hours after the event.
About the Phase 2 Study
Data were presented from an ongoing investigator sponsored Phase 2 study
of sotatercept in subjects with myelofibrosis at the conference.
Subjects enrolled were red blood cell (RBC) transfusion-dependent, have
hemoglobin < 10 g/dL on every determination during the 84 days preceding
study entry without RBC transfusions, or have hemoglobin < 10 g/dL
despite intermittent RBC transfusions without fulfilling the criteria
for transfusion dependence. Patients received open-label sotatercept at
0.75 mg/kg or 1 mg/kg, dosed subcutaneously once every three weeks.
The primary outcome measures for the study include anemia response and
safety. Anemia response is a composite of RBC-transfusion-independence
and hemoglobin response (increase of ≥1.5 g/dL from baseline on every
determination consecutively over ≥84 days without RBC transfusions).
Subjects must have received ≥5 cycles of sotatercept to be evaluable for
Sotatercept is an activin receptor type IIA fusion protein that acts as
a ligand trap for members in the Transforming Growth Factor-Beta (TGF-β)
superfamily involved in fibrosis and late stage erythropoiesis (red
blood cell production). Acceleron and Celgene are jointly developing
sotatercept as part of a global collaboration. Sotatercept is currently
in multiple Phase 2 investigator initiated trials. For more information,
please visit www.clinicaltrials.gov.
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
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.
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 sotatercept, 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
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
Cautionary Note Regarding Forward-Looking Statements
This 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/20161205005432/en/
James, IRC, 617-649-9393
Senior Director, Investor Relations and
BMC Communications LLC
Source: Acceleron Pharma Inc. and Celgene Corporation
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