jeudi 3 décembre 2020


Acceleron Announces Updated Results from Ongoing Phase 2 Trials of Luspatercept in Myelodysplastic Syndromes at the 59th Annual Meeting of the American Society of Hematology

Par Rédaction , dans Communiqués , le 10 décembre 2017

Sunday, December 10th 2017 at 2:00pm UTC

– Preliminary results show treatment with luspatercept increases
hemoglobin and achieves durable red blood cell transfusion independence
in patients with lower-risk myelodysplastic syndromes –

– Conference call and webcast to be held on Monday, December 11th
at 7:00 a.m. EST –

CAMBRIDGE, Mass.–(BUSINESS WIRE)– Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company
in the discovery and development of TGF-beta therapeutics to treat
serious and rare diseases, today announced preliminary results from the
ongoing Phase 2 trials with luspatercept in patients with lower-risk
myelodysplastic syndromes (MDS) at the 59th Annual Meeting of
the American Society of Hematology (ASH) in Atlanta, Georgia.
Luspatercept is being developed as part of a global collaboration
between Acceleron and Celgene.

“As the Phase 2 results in MDS mature, we are excited to see
luspatercept achieving a clinically meaningful erythroid response in
over 50% of patients. Luspatercept continues to provide long-term
benefit to multiple patients now nearing three years on treatment. These
results further reinforce luspatercept’s potential to be a
transformative treatment option for patients living with lower-risk
MDS,” said Habib Dable, President and Chief Executive Officer of
Acceleron. “We look forward to the upcoming MEDALIST and BELIEVE Phase 3
trial top-line data readouts in mid-2018, and we and Celgene continue to
make considerable progress toward initiating the COMMANDS Phase 3 trial
during the first half of 2018.”

Phase 2 Results

A total of 99 lower-risk MDS patients have been treated with therapeutic
dose levels of luspatercept (? 0.75 mg/kg) in the ongoing Phase 2 trials.

  • 53% (52 of 99 patients) 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.
  • 43% (29 of 67 patients) with an RBC transfusion burden at baseline of
    ? 2 units per 8 weeks achieved RBC transfusion independence (RBC-TI)
    for ? 8 weeks.

    • In an updated analysis of the 23 RBC-TI responders previously
      reported at EHA 2017, the median duration of treatment increased
      to 19.0 months from 14.7 months. The current duration of treatment
      for RBC-TI responders ranges from 2.8 months to 37.3 months.
  • Patients with a low transfusion burden at baseline (< 4 RBC units per
    8 weeks and hemoglobin < 10 g/dL) demonstrated a clinically meaningful
    increase in hemoglobin for up to 34 months, with multiple ongoing.

The results presented at ASH 2017 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 106 lower-risk MDS patients have been treated with
luspatercept in the ongoing Phase 2 trials (all dose levels).

  • The majority of adverse events (AEs) were Grade 1 or 2. AEs possibly
    or probably related to study drug that occurred in at least three
    patients during the studies were headache, hypertension, fatigue, 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, mucosal inflammation, platelet count increase, and
    pleural effusion. These Grade 3 non-serious AEs occurred in seven
    individual patients with one patient reporting both the ascites and
    pleural effusion.
  • Serious AEs (SAEs) possibly related to study drug were general
    physical health deterioration, muscular weakness, musculoskeletal
    pain, and myalgia. These SAEs occurred in three individual patients
    with one patient reporting both the muscular weakness and
    musculoskeletal pain.

The MEDALIST trial, a global Phase 3 trial of luspatercept in lower-risk
MDS patients, is fully enrolled with top-line results expected in
mid-2018. The MEDALIST trial enrolled patients who are RS-positive, RBC
transfusion dependent, and are ESA-refractory or ESA-treatment
ineligible, based on EPO levels greater than 200 units per liter at
baseline. Acceleron and Celgene plan to initiate the COMMANDS Phase 3
trial in first-line, lower-risk MDS patients during the first half of
2018.

The MDS clinical poster presentation is available under the Science page
of the Company’s website at www.acceleronpharma.com.

Luspatercept is an investigational product that is not approved for use
in any country.

Acceleron ASH Conference Call Information

Acceleron will host a conference call and live webcast to discuss data
presented at the ASH meeting on December 11, 2017, at 7:00 a.m. EST.

Individuals can participate in the conference call by dialing
877-312-5848 (domestic) or 253-237-1155 (international)
and refer to the “Acceleron ASH 2017 Update.”

The webcast will be accessible under « Events & Presentations » in the
Investors/Media page of the Company’s website at www.acceleronpharma.com.

A replay of the webcast will be available approximately two hours after
the event.

About the Ongoing MDS Phase 2 Studies

Data from two Phase 2 trials 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 trials 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 receiving 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 of the TGF-beta superfamily involved
in the late stages of erythropoiesis (red blood cell production).
Luspatercept is a first-in-class erythroid maturation agent (EMA) that
regulates late-stage erythrocyte (red blood cell) precursor cell
differentiation. 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 trial) and in patients with beta-thalassemia
(the BELIEVE trial). A Phase 3 trial is being planned in first-line,
lower-risk, myelodysplastic syndromes patients (the COMMANDS trial). For
more information, please visit www.clinicaltrials.gov.

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 pulmonary program with a Phase 2 trial of sotatercept
planned 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 about the
Company’s strategy, future plans and prospects, including statements
regarding the development of the Company’s compounds, the timeline for
clinical development and regulatory approval of the Company’s compounds
and the expected timing for reporting of data from ongoing clinical
trials. The words « anticipate, » « believe, » « could, » « estimate, »
« expect, » “goal”, « intend, » « may, » « plan, » « potential, » « 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.

Actual results could differ materially from those included in the
forward-looking statements due to various risks and uncertainties,
including, but not limited to, that preclinical testing of the Company’s
compounds and data from clinical trials may not be predictive of the
results or success of ongoing or later clinical trials, that the
development of the Company’s compounds will take longer and/or cost more
than planned, that the Company or its collaboration partner, Celgene,
will be unable to successfully complete the clinical development of the
Company’s compounds, that the Company or Celgene may be delayed in
initiating, enrolling or completing any clinical trials, and that the
Company’s compounds will not receive regulatory approval or become
commercially successful products. These and other risks and
uncertainties are identified under the heading « Risk Factors » included
in the Company’s most recent Annual Report on Form 10-K, and other
filings that the Company has made and may make with the SEC in the
future.

The forward-looking statements contained in this press release are based
on management’s current views, plans, estimates, assumptions and
projections with respect to future events, and the Company does not
undertake and specifically disclaims any obligation to update any
forward-looking statements.

Contacts

Acceleron Pharma Inc.
Todd James, IRC, 617-649-9393
Vice
President, Investor Relations and Corporate Communications
or
Candice
Ellis, 617-649-9226
Manager, Investor Relations and Corporate
Communications
or
Media:
BMC Communications
Brad
Miles, 646-513-3125

Source: Acceleron Pharma

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