Celgene Corporation and Acceleron Pharma Announce Results of the Phase 3 MEDALIST Trial Evaluating Luspatercept in Patients with Myelodysplastic Syndromes at the ASH 2018 Plenary Session

SUMMIT, N.J. CAMBRIDGE, Mass.–(BUSINESS WIRE)–Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ:
XLRN) today announced results from the pivotal, phase 3 MEDALIST trial
evaluating the efficacy and safety of investigational luspatercept to
treat patients with ring sideroblast (RS+) myelodysplastic syndromes
(MDS)-associated anemia who require red blood cell transfusions and who
had failed, were intolerant to, or ineligible for erythropoietin
therapy. Results were presented by Alan F. List, M.D. during the Plenary
Scientific Session at the 60th American Society of Hematology
(ASH) Annual Meeting and Exposition in San Diego, C.A. (Abstract #1).

“Severe anemia resulting in red blood cell transfusion dependence is a
significant challenge for patients with low- and intermediate-risk MDS.
Those who become resistant or refractory to currently available
treatments have limited alternatives,” said Dr. List, President and CEO
of Moffitt Cancer Center. “The findings from MEDALIST are very exciting
as they support the hypothesis that targeting red blood cell precursor
maturation could help to address patients’ anemia and allow them to
achieve transfusion independence.”

MEDALIST met the primary endpoint of red blood cell transfusion
independence (RBC-TI) for 8 or more weeks during the first 24 weeks of
the study. Treatment with luspatercept resulted in a statistically
significantly greater proportion of patients achieving RBC-TI ≥ 8 weeks
compared to placebo. The study also found in secondary endpoints that
treatment with luspatercept resulted in a statistically significant
higher percentage of patients achieving RBC-TI of 12 or more weeks in
the first 24 or 48 weeks of the study, as well as hematologic
improvement-erythroid (HI-E) of 8 or more weeks.

MEDALIST Safety Summary

Treatment-emergent adverse events (TEAEs) of Grade 3 or 4 were reported
in 42.5% (65/153) of patients receiving luspatercept and 44.7% (34/76)
of patients receiving placebo. Progression to acute myeloid leukemia
(AML) occurred in four patients, three patients (2.0%) receiving
luspatercept and one patient (1.3%) receiving placebo. Five patients
receiving luspatercept (3.3%) and four patients receiving placebo (5.3%)
experienced one or more TEAE that resulted in death.

Most common TEAEs of any Grade in Greater than
10% of Patients in Either Arm

 

 

Luspatercept

N=153

 

Placebo

N=76

Fatigue

 

26.8

%

 

13.2

%

Diarrhea

 

22.2

%

 

9.2

%

Asthenia

 

20.3

%

 

11.8

%

Nausea

 

20.3

%

 

7.9

%

Dizziness

 

19.6

%

 

5.3

%

Back pain

 

19.0

%

 

6.6

%

 

 

“The MEDALIST results demonstrate the potential clinical benefit of
luspatercept in achieving red blood cell transfusion independence in
patients with low-to-intermediate risk RS+ MDS, an area in need of new
treatments,” said Alise Reicin, MD, President, Global Clinical
Development for Celgene. “Based on these results, we are encouraged that
this first-in-class erythroid maturation agent may help these patients
address the underlying cause of their disease-related chronic anemia.”

“It’s truly an honor to showcase the results from the MEDALIST trial as
the first presentation of the ASH Plenary Session,” said Habib Dable,
President and Chief Executive Officer of Acceleron. “The results from
the MEDALIST trial increase our confidence in the potential of
luspatercept to provide a meaningful treatment option for patients
suffering from lower-risk RS+ MDS worldwide. We’re excited to continue
our clinical development program in MDS, beta-thalassemia, and
myelofibrosis, while also exploring additional applications for
luspatercept in a range of diseases associated with anemia.”

Luspatercept is not approved in any region for any indication. The
companies are planning regulatory application submissions of
luspatercept in the United States and Europe in the first half of 2019.

About MEDALIST

MEDALIST is a phase 3, randomized, double blind, placebo-controlled,
multi-center study evaluating the safety and efficacy of luspatercept in
patients with very low-, low-, or intermediate-risk non-del(5q)
myelodysplastic syndromes (MDS). All patients were RBC transfusion
dependent and were either refractory or intolerant to prior
erythropoiesis-stimulating agent (ESA) therapy, or were ESA naïve with
endogenous serum erythropoietin ≥ 200 U/L, and had no prior treatment
with disease modifying agents. The median age of the patients enrolled
in the trial was 71 years in the luspatercept treatment group and 72
years in the placebo group. Median transfusion burden in both treatment
arms was 5 RBC units/8 weeks. 229 patients were randomized to receive
either luspatercept 1.0 mg/kg (153 patients) or placebo (76 patients)
via subcutaneous injection once every 21 days. The study was conducted
at 65 sites in 11 countries.

About Luspatercept

Luspatercept is a first-in-class erythroid maturation agent (EMA) that
is believed to regulate late-stage red blood cell maturation. Acceleron
and Celgene are jointly developing luspatercept as part of a global
collaboration. Phase 3 clinical trials continue to evaluate the safety
and efficacy of luspatercept in patients with MDS (the MEDALIST trial)
and in patients with beta-thalassemia (the BELIEVE trial). The COMMANDS
phase 3 trial in first-line, lower-risk, MDS patients, the BEYOND phase
2 trial in non-transfusion-dependent beta-thalassemia, and a phase 2
trial in myelofibrosis are ongoing. For more information, please visit www.clinicaltrials.gov.

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: Twitter,
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and YouTube.

About Acceleron

Acceleron is a Cambridge-based, clinical-stage biopharmaceutical company
dedicated to the discovery, development, and commercialization of
therapeutics to treat serious and rare diseases. The Company’s
leadership in the understanding of TGF-beta biology and protein
engineering generates innovative compounds that engage the body’s
ability to regulate cellular growth and repair.

Acceleron focuses its research and development efforts in hematologic,
neuromuscular, and pulmonary diseases. In hematology, the Company and
its global collaboration partner, Celgene, are developing luspatercept
for the treatment of chronic anemia in myelodysplastic syndromes,
beta-thalassemia, and myelofibrosis. Acceleron is also advancing its
neuromuscular franchise with two distinct Myostatin+ agents, ACE-083 and
ACE-2494, and a Phase 2 pulmonary program with sotatercept in pulmonary
arterial hypertension.

For more information, please visit www.acceleronpharma.com.
Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.

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,” “will,” “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, that the results of any clinical study will be predictive for
other clinical studies of the same product candidate, 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.

Hyperlinks are provided as a convenience and for informational
purposes only. Neither Celgene nor Acceleron bears responsibility for
the security or content of external websites or websites outside of
their respective control.

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