Acceleron Announces Updated Results from Ongoing Phase 2 Trials of Luspatercept in Beta-Thalassemia at the 23rd Congress of the European Hematology Association
- Clinically meaningful increases in hemoglobin and reductions in red blood cell transfusion burden observed through 36 months –
- Top-line results from the BELIEVE Phase 3 trial are on track for the middle of 2018 -
“These results reinforce our enthusiasm for luspatercept’s potential as
a safe, efficacious therapy for beta-thalassemia patients over the long
Beta-thalassemia is caused by a genetic defect in the production of hemoglobin, a protein that carries oxygen to red blood cells throughout the body. Patients suffer from severe, chronic anemia and often experience fatigue, organ enlargement, and bone complications. Patients require lifelong therapy that includes frequent red blood cell transfusions and corresponding treatment for the iron overload that results.
“We now have patients with both non-transfusion- and transfusion-dependent beta-thalassemia continuing on treatment for three years,” Dable continued. “We look forward to sharing top-line results from the BELIEVE Phase 3 trial over the next few months.”
Phase 2 Results
A total of 31 non-transfusion-dependent patients have been treated with luspatercept (dose levels ≥ 0.6 mg/kg) in the trials.
- 53% (16 of 30) achieved a ≥ 1 gram per deciliter increase in hemoglobin in the fixed 12-week intervals including weeks 13 to 24 and weeks 37 to 48 when compared to baseline.
- At week 48, the mean improvement in 6-minute walk distance was 18.6% compared to baseline (n=9).
- Multiple patients remain on treatment through 36 months, and continue to sustain clinically meaningful increases in hemoglobin.
A total of 32 transfusion-dependent patients have been treated with luspatercept (dose levels ≥ 0.6 mg/kg) in the trials.
41% (12 of 29) achieved a reduction in
RBCtransfusion burden of at least 33% in the fixed 12-week intervals including weeks 13 to 24 and weeks 37 to 48 when compared to baseline.
Phase 2 Safety Summary
The majority of adverse events (AEs) were Grade 1 or 2. Grade 3 AEs possibly or probably related to study drug were bone pain (n=3 patients), asthenia (n=2 patients), bone infarction (n=1 patient), headache (n=1 patient), and presyncope (n=1 patient). One serious AE of biliary colic was reported as possibly related to study drug.
The EHA beta-thalassemia presentation will be available immediately
following the presentation on
Luspatercept is an investigational product that is not approved for any use in any country.
About the Phase 2 Trial
Data from two Phase 2 trials were presented at the 23rd
Luspatercept is a first-in-class erythroid maturation agent (EMA) that
regulates late-stage red blood cell maturation. Acceleron and
Acceleron is a
Acceleron focuses its research and development efforts in hematologic,
neuromuscular, and pulmonary diseases. In hematology, the Company and
its global collaboration partner,
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,
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.
Acceleron Pharma Inc.
Todd James, IRC, (617) 649-9393
Vice President, Investor Relations and Corporate Communications
Candice Ellis, 617-649-9226
Manager, Investor Relations and Corporate Communications
Matt Fearer, 617-301-9557
Director, Corporate Communications