- Preliminary results show that treatment with
investigational drug luspatercept achieves meaningful erythroid
response rates and transfusion independence in first-line,
lower-risk myelodysplastic syndromes patients -
- Celgene and Acceleron plan to initiate a
Phase 3 trial in first-line, lower-risk MDS patients in early 2018
-
- Acceleron to review MDS Symposium
presentation in conjunction with its first quarter earnings call on
Monday, May 8th at 8:00 a.m. EDT -
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 14th International Symposium
on MDS in Valencia, Spain. Luspatercept is being developed as part
of the global collaboration between Acceleron and Celgene.
“These positive data presented in lower-risk MDS confirm our
optimism in new opportunities for luspatercept beyond our ongoing
Phase 3 trials,” said Michael Pehl, President, Hematology and
Oncology for Celgene. “We are now planning a Phase 3 clinical trial
to expand the development of luspatercept into this lower-risk MDS
patient population.”
“There is a high unmet medical need for a drug to treat patients
earlier in the MDS treatment paradigm,” said Habib Dable, President
and CEO of Acceleron. “We continue to be motivated to find
additional opportunities for luspatercept to treat anemia due to
rare blood disorders and remain on track to initiate Phase 2 trials
in myelofibrosis and non-transfusion dependent beta-thalassemia by
year-end.”
Luspatercept Phase 2 Data in First-Line, Lower-Risk
MDS Patients
In lower-risk, erythropoiesis-stimulating agent
(ESA)-naïve MDS patients, 48% (11/23) of patients treated with
luspatercept achieved red blood cell transfusion independence
(RBC-TI) and 51% (20/39) of patients achieved a clinically
meaningful erythroid hematological improvement (HI-E) response per
the International Working Group’s (IWG) criteria. The response
rates were positive in patients treated with luspatercept in both
ESA-naïve and prior ESA-treated patients.
IWG HI-E, N=82n (%)
RBC-TI, N=56n (%)
ESA-Naïve 20/39 (51%) 11/23
(48%)
Prior ESA 22/43 (51%)
11/33 (33%)
Luspatercept Phase 2 Data in RS+ and RS-
Lower-Risk MDS Patients
In patients with baseline erythropoietin (EPO)
levels ≤ 500 international units per liter (IU/L), RBC-TI and IWG
HI-E response rates were positive in both ring sideroblast-positive
(RS+) and –negative (RS-) patients.
Baseline EPO (IU/L)
RS Status
IWG HI-E, N=82n (%)
RBC-TI, N=56n (%)
≤ 500 RS+ 30/46 (65%)
16/29 (55%) RS- 6/14 (43%)
4/7 (57%) > 500 RS+ 5/9 (56%)
2/9 (22%) RS- 1/11 (9%)
0/9 (0%) Unknown 0/2 (0%)
0/2 (0%)
*Table includes both ESA refractory and ESA
naïve patients. Patients treated at dose levels ≥ 0.75 mg/kg.
Luspatercept Phase 2 Safety Data
The majority of adverse events (AEs) were grade 1 or 2. AEs at
least possibly related to study drug that occurred in at least 3
patients during the studies were fatigue, headache, hypertension,
diarrhea, arthralgia, bone pain, injection site erythema, myalgia,
and edema peripheral. Grade 3 non-serious AEs possibly or probably
related to study drug were ascites, blast cell count increase,
blood bilirubin increase, hypertension, platelet count increase,
and pleural effusion. Grade 3 serious AEs possibly or probably
related to study drug were general physical health deterioration
and myalgia.
Luspatercept is an investigational product that is not approved
for use in any country.
The oral presentation given at the 14th International Symposium
on MDS is available on Acceleron's website
(www.acceleronpharma.com) under the Science tab.
Acceleron MDS Symposium Conference Call Information
Acceleron will host a conference call and live webcast to
discuss data presented at the MDS Symposium and its first quarter
operational and financial results on May 8, 2017, at 8:00 a.m. EDT.
To participate by teleconference, please dial 877-312-5848
(domestic) or 253-237-1155 (international) and refer to the
Acceleron Earnings Call.
To access the live webcast, please select “Events &
Presentations” in the Investors/Media 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, lower-risk MDS patients were enrolled
and treated with open-label luspatercept, dosed subcutaneously once
every three weeks.
The outcome measures for the studies included the proportion of
patients who had an erythroid response (IWG HI-E) or achieved RBC
transfusion independence (RBC-TI). IWG HI-E was defined as
hemoglobin increase ≥ 1.5 g/dL sustained for ≥ 8 weeks in patients
with < 4 units RBC / 8 weeks transfusion burden at baseline and
hemoglobin levels below 10 g/dL. For patients with a ≥ 4 units RBC
/ 8 weeks transfusion burden at baseline, erythroid response was
defined as a reduction of ≥ 4 units RBC sustained for ≥ 8 weeks.
RBC-TI was defined as no RBC transfusions for ≥ 8 weeks in patients
with a ≥ 2 units RBC / 8 weeks baseline transfusion burden.
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 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 http://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
Celgene and Acceleron:
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. Each
company’s 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; 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20170506005003/en/
For Acceleron:Investors:Todd James,
IRC, 617-649-9393Senior Director, Investor Relations and Corporate
CommunicationsorMedia:BMC CommunicationsBrad Miles,
646-513-3125orFor
Celgene:Investors:908-673-9628investors@celgene.comorMedia:908-673-2275media@celgene.com
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