Acceleron and Celgene Announce Updated Results from an Ongoing Phase 2 Study of Luspatercept in Beta-Thalassemia Presented at the 21st Congress of the European Hematology Association

CAMBRIDGE, Mass. & SUMMIT, N.J.–(BUSINESS WIRE)–Acceleron Pharma Inc. (NASDAQ:XLRN) and Celgene Corporation (NASDAQ:
CELG) today announced that preliminary results will be presented from an
ongoing long-term Phase 2 extension study in patients with
beta-thalassemia during an oral presentation and a separate poster
presentation at the 21^st Congress of the European Hematology
Association (EHA). Results from the presentations showed that 80%
(24/30) of patients achieved at least a 20% reduction in transfusion
burden and 36% (8/22) of patients achieved a hemoglobin increase of at
least 1.5 g/dL in the luspatercept 3-month base study. In the ongoing
long-term extension study, 96% (23/24) of patients achieved at least a
20% reduction in transfusion burden and 56% (15/27) of patients achieved
a hemoglobin increase of at least 1.5 g/dL. Luspatercept is being
developed as part of the global collaboration between Acceleron and
Celgene.
“The results from the 3-month luspatercept clinical trial were very
promising and the data from longer-term treatment are even more
encouraging,” said Professor Antonio Piga, M.D., Ph.D., Director of
Pediatrics at San Luigi Gonzaga University Hospital in Torino, Italy and
coordinating principal investigator of the study. “Clinicians and
patients are in need of safe and effective therapies for the treatment
of beta-thalassemia. These Phase 2 luspatercept data provide strong
rationale for the ongoing Phase 3 BELIEVE study of luspatercept in
regularly transfused beta-thalassemia patients.”
Highlights of the Luspatercept Beta-Thalassemia Data Presented at EHA
Study Design
Data from two Phase 2 studies will be presented at the conference: the
completed dose-escalation study in which patients received treatment
with luspatercept for three months and the ongoing long-term extension
study in which patients receive treatment with luspatercept for up to an
additional 24 months. In both the 3-month 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 3 weeks. The primary outcome measure of
the 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 measure of the long-term extension was the
safety and tolerability assessed by adverse events.
Efficacy
Results in Transfusion Dependent (TD) Beta-Thalassemia Patients

Durability of Response:
In the long-term extension study, the duration of the reduction in
transfusion burden of ≥ 33% ranged from 12 to 48+ weeks.
Results in Non-Transfusion Dependent (NTD) Beta-Thalassemia Patients

Durability of Response:
In the long-term extension study, the duration of hemoglobin increase (≥
1.0 g/dL) ranged from 113 to 505+ days.
Improvement in patient-reported quality of life (QoL) measures in NTD
patients:

• Increases in mean hemoglobin over a 12-week period correlated (r=0.67,
  p=0.001) with increases in
  FACIT-F, a patient-reported outcome
  (PRO) questionnaire used to assess anemia related symptoms

Safety Results in TD and NTD Patients
TD Patients

• 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 myalgia (n=1 extension)
• The most common related adverse events (all grades) were bone pain,
  myalgia, arthralgia, headache, asthenia, and musculoskeletal pain

NTD Patients

• There were no related serious adverse events and one grade 3 related
  adverse event of headache (n=1) in the extension study
• The most common related adverse events were bone pain, headache,
  musculoskeletal pain and arthralgia

Luspatercept is an investigational product that is not approved for use
in any country.
The BELIEVE Trial, a global Phase 3 study in regularly transfused
beta-thalassemia patients, is currently enrolling.
The slides from the EHA beta-thalassemia presentations will be available
immediately following the presentations at the conference on Acceleron’s
website (www.acceleronpharma.com)
under the Science tab.
Acceleron EHA Conference Call Information
Acceleron will host a conference call and live webcast from EHA today at
8:00 a.m. EDT (2:00 p.m. CEST). To participate by teleconference, please
dial 877-312-5848 (domestic) or 253-237-1155 (international) and refer
to the Acceleron EHA Congress Review.
To access the live webcast, please select “Events & Presentations” in
the Investor 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 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 discovers and develops novel therapies to treat a wide range
of 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 innovative approach to drug discovery has
generated four therapeutic candidates currently in clinical trials.
Acceleron’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 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.
Celgene:
This press release contains forward-looking statements, which are
generally statements that are not historical facts. Forward-looking
statements can be identified by the words “expects,” “anticipates,”
“believes,” “intends,” “estimates,” “plans,” “will,” “outlook” and
similar expressions. Forward-looking statements are based on
management’s current plans, estimates, assumptions and projections, and
speak only as of the date they are made. We undertake no obligation to
update any forward-looking statement in light of new information or
future events, except as otherwise required by law. Forward-looking
statements involve inherent risks and uncertainties, most of which are
difficult to predict and are generally beyond our control. Actual
results or outcomes may differ materially from those implied by the
forward-looking statements as a result of the impact of a number of
factors, many of which are discussed in more detail in our Annual Report
on Form 10-K and our other reports filed with the Securities and
Exchange Commission.