- Results from ongoing trials demonstrate
increases in hemoglobin and reductions in red blood cell
transfusion burden sustained for more than 26 months -
Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage
biopharmaceutical company focused on the discovery, development,
and commercialization of innovative therapeutics to treat serious
and rare diseases, today announced preliminary results from the
ongoing Phase 2 studies of luspatercept in patients with lower-risk
myelodysplastic syndromes (MDS) at the 22nd Congress of the
European Hematology Association (EHA) in Madrid, Spain.
Luspatercept is being developed to treat a range of hematologic
diseases including MDS, beta-thalassemia, and myelofibrosis as part
of a global collaboration between Acceleron and Celgene.
“This Phase 2 update further supports our confidence that
luspatercept could become a potential first-in-class treatment for
lower-risk MDS patients. With some patients continuing on study for
more than 26 months, we are very encouraged by both the durability
of response and safety profile of luspatercept,” said Habib Dable,
President and Chief Executive Officer of Acceleron. “With Phase 3
trials across two indications ongoing and new studies planned, we
and Celgene remain committed to exploring the full opportunity for
luspatercept to transform patients’ lives.”
Phase 2 Results
A total of 88 lower-risk MDS patients have been treated with
therapeutic dose levels of luspatercept in the ongoing Phase 2
study.
- 50% (44 of 88) achieved a clinically
meaningful erythroid response of an increase in hemoglobin or
reduction in red blood cell (RBC) transfusion burden as per the
International Working Group’s Hematologic Improvement Erythroid
(IWG HI-E) response criteria.
- 38% (23 of 60 patients with ≥ 2 units
RBC / 8 weeks transfusion burden at baseline) achieved RBC
transfusion independence (RBC-TI) for ≥ 8 weeks.
- Patients with a low transfusion burden
(< 4 units / 8 weeks and hemoglobin < 10 g/dL) demonstrated a
clinically meaningful increase in hemoglobin for up to 26 months,
with several remaining on treatment.
The results presented at EHA confirm and extend previously
reported results across the lower-risk MDS patient subpopulations,
showing erythroid responses regardless of prior use of
erythropoiesis-stimulating agents (ESA), baseline erythropoietin
(EPO) levels, and ring sideroblast (RS) status.
Phase 2 Safety Summary
A total of 95 lower-risk MDS patients have been treated with
luspatercept in the ongoing Phase 2 studies (all dose levels).
- The majority of adverse events (AEs)
were Grade 1 or 2. AEs possibly related to study drug that occurred
in at least three patients during the studies were headache,
fatigue, hypertension, bone pain, diarrhea, arthralgia, injection
site erythema, myalgia, and edema peripheral.
- Grade 3 non-serious AEs possibly
related to study drug were ascites, blast cell count increase,
blood bilirubin increase, bone pain, hypertension, platelet count
increase, and pleural effusion. These Grade 3 non-serious AEs
occurred in six individual patients with one patient accounting for
both the ascites and pleural effusion AEs.
- Grade 3 serious AEs (SAEs) possibly
related to study drug were ataxia, general physical health
deterioration, and myalgia; a Grade 2 SAE possibly related to study
drug of muscle weakness was reported.
“The longer term results of these Phase 2 studies reinforce the
potential of inhibiting ligands in the TGF-beta superfamily for
patients with lower-risk MDS,” said Michael Pehl, President,
Hematology/Oncology for Celgene. “With the Phase 3 study now fully
enrolled, we look forward to advancing luspatercept as part of our
ongoing commitment to individuals with MDS around the world.”
Luspatercept is an investigational product that is not approved
for use in any country.
The MEDALIST trial, a global Phase 3 study of luspatercept in
patients with lower-risk MDS who require red blood cell
transfusions, is fully enrolled and top-line results are expected
in mid-2018.
The MDS poster presentation is available under the Science page
of the Company’s website at www.acceleronpharma.com/.
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 who completed the base study 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 erythropoiesis stimulating agents (ESAs),
which stimulate the proliferation of early-stage erythrocyte
precursor cells. Acceleron and Celgene are jointly developing
luspatercept as part of a global collaboration. Phase 3 clinical
trials are underway 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. Acceleron is also advancing its ACE-083
clinical program in the field of neuromuscular disease, 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.
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
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 March 1, 2017, 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20170622006288/en/
Acceleron Pharma Inc.Todd James, IRC, 617-649-9393Vice
President, Investor Relations and Corporate CommunicationsorCandice
Ellis, 617-649-9226Manager, Investor Relations and Corporate
CommunicationsorMedia:BMC CommunicationsBrad Miles,
646-513-3125
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