- Preliminary results show that treatment with
investigational drug luspatercept increases hemoglobin and achieves
durable transfusion independence in patients with lower risk
myelodysplastic syndromes –
- Preliminary data in ESA naïve and RS-
patients are encouraging -
- Acceleron to host conference call and live
webcast on Monday, December 5th at 9:00 a.m. EST (6:00 a.m. PST)
-
Acceleron Pharma Inc. (NASDAQ:XLRN) and Celgene Corporation
(NASDAQ:CELG), today announced preliminary Phase 2 results from the
ongoing three-month base and long-term extension studies with
investigational drug luspatercept in patients with lower risk
myelodysplastic syndromes (MDS) 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.
“We are encouraged by the additional luspatercept data from the
ongoing Phase 2 studies,” said Michael Pehl, President, Hematology
and Oncology for Celgene. “These data further support
luspatercept’s potential in treating a broader spectrum of MDS
patients. We are evaluating opportunities to expand our clinical
program to include additional MDS patient populations, as we
advance our Phase 3 MEDALIST trial in RS+ patients.”
Luspatercept Phase 2 Data in First-line, ESA treatment-naive
MDS Patients
In lower-risk MDS patients who have not received prior treatment
with an erythropoiesis-stimulating agent (ESA) and have
erythropoietin (EPO) levels ≤ 500 IU, luspatercept three-month base
study data demonstrated encouraging rates of transfusion
independence and International Working Group Hematologic
Improvement – Erythroid (IWG HI-E) response criteria.
Transfusion Burden IWG HI-E, n/N (%)
RBC-TI, n/N (%)
Base N=64 Extension N=42
Base N=49 Extension N=28
All 12/20 (60%) 13/16 (81%)
9/12 (75%) 8/10 (80%)
Low Transfusion Burden 6/13 (46%)
8/11 (73%) 5/5 (100%)
5/5 (100%)
High Transfusion Burden 6/7
(86%) 5/5 (100%) 4/7
(57%) 3/5 (60%)
Luspatercept Phase 2 Data in Ring Sideroblast Positive (RS+)
and Negative (RS-) in MDS Patients
- In patients with EPO levels < 200,
response rates were similar in both RS+ and RS- patients
- In the patients with EPO levels ≥ 200
to ≤ 500, luspatercept response rates remained encouraging in those
patients who are RS+
Baseline EPO(U/L)
RS Status
IWG HI-E, n/N
(%) RBC-TI,
n/N (%)
Base
N=64*
ExtensionN=42*
Base
N=49*
Extension N=28*
< 200 RS+ 18/29 (62%) 19/23
(83%) 13/19 (68%)
10/14 (71%) RS-
2/5 (40%)
3/3 (100%) 1/4 (25%)
1/2 (50%) ≥ 200 to ≤ 500 RS+
5/11 (46%) 7/8 (88%) 3/9 (33%) 3/5 (60%)
RS- 0/3 (0%)
0/1 (0%)
2/2 (100%) 1/1 (100%)
*Table includes both ESA refractory and ESA naïve patients.
Subjects treated at dose levels ≥ 0.75 mg/kg.
Luspatercept Phase 2 Safety Data
- The majority of adverse events (AEs)
were grade 1 or 2
- There were four grade 3/serious AEs
possibly or probably related to study drug as of November 28, 2016:
blast cell count increase, myalgia, worsening of general condition,
progression to AML
- Adverse events at least possibly
related to study drug that occurred in at least 2 patients during
studies were diarrhea, fatigue, headache, hypertension, arthralgia,
bone pain, injection site erythema, myalgia and peripheral
edema.
Luspatercept is an investigational product that is not approved
for use in any country.
The MEDALIST Trial, a global Phase 3 study in patients with very
low, low, or intermediate risk, MDS with ring sideroblasts who
require red blood cell transfusions, is currently enrolling.
The poster presentation of the ongoing Phase 2 studies is
available on Acceleron's website (www.acceleronpharma.com) under
the Science tab.
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 ASH Review.
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 be required.
An archived webcast recording will be available on the Acceleron
website beginning approximately two hours after the event.
About the MDS Phase 2 Studies
Data from two Phase 2 studies were presented at the conference:
the base study in which patients received treatment with
luspatercept for three months and the long-term 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, high transfusion burden patients (≥ 4
units RBC / 8 weeks) and low transfusion burden patients (< 4
units RBC / 8 weeks) were enrolled and treated with open-label
luspatercept, dosed subcutaneously once every three weeks.
The primary outcome measure for the three-month base study was
the proportion of patients who had an erythroid response. Erythroid
response was defined as hemoglobin ≥ 1.5 g/dL from baseline for ≥
14 days in low-transfusion burden patients and for high-transfusion
burden patients a reduction of either ≥ 4 units or ≥ 50% of units
of RBCs transfused while receiving luspatercept compared to the
patient’s pretreatment transfusion burden. 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
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.
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 the
Company 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.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20161204005056/en/
For Acceleron:Investors:Todd James,
IRC, 617-649-9393Senior Director, Investor Relations and Corporate
CommunicationsorMedia:BMC CommunicationsBrad Miles,
917-570-7340orFor
Celgene:Investors:908-673-9628investors@celgene.comorMedia:908-673-2275media@celgene.com
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