- Interim longer term data with investigational drug luspatercept show sustained increases in hemoglobin levels, and reduced transfusion burden in patients with beta-thalassemia -
“Beta-thalassemia is a severe, chronic disease with no pharmaceutical treatment options to correct or improve the underlying anemia in patients,” said
Luspatercept Beta-Thalassemia Data Presented at ASH
Results in Transfusion Dependent (TD) Beta-Thalassemia Patients
RBC transfusion reduction | Response rate (% of patients) | ||||||
3-month base study (n=31) | Long-term extension study (n=24) | ||||||
≥ 20% | 81% (25/31) | 96% (23/24) | |||||
≥ 33% | 71% (22/31) | 83% (20/24) | |||||
≥ 50% | 55% (17/31) | 71% (17/24) | |||||
Results in Non-Transfusion Dependent (NTD) Beta-Thalassemia Patients
Hemoglobin (Hb) response over | Response rate (% of patients) in patients treated with ≥ 0.6 mg/kg | ||||||
3-month base study (n=21) | Long-term extension study (n=27) | ||||||
Increase in mean Hb ≥ 1.0 g/dL | 62% (14/21) | 78% (21/27) | |||||
Increase in mean Hb ≥ 1.5 g/dL | 33% (7/21) | 52% (14/27) | |||||
In the long-term extension study, the median duration of a hemoglobin increase ≥ 1.0 g/dL maintained for at least 12 weeks in responders is 13.5 months (N=21) with treatment still ongoing.
Safety
- 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 headache (n=1 extension)
- The most common related adverse events (all grades) were bone pain, myalgia, headache, musculoskeletal pain, arthralgia, and injection site pain.
Luspatercept is an investigational product that is not approved for use in any country.
The BELIEVE Trial, a global Phase 3, double-blind, randomized, placebo-controlled, multicenter study in transfusion dependent beta-thalassemia patients, is currently enrolling.
The slides from the ASH beta-thalassemia presentation will be available immediately following the presentation at the conference on Acceleron’s website (www.acceleronpharma.com) under the Science tab.
About the Phase 2 Study
Data from two open-label Phase 2 studies were presented at the conference: the base study in which patients received treatment with luspatercept for three months and the ongoing long-term safety extension study in which patients 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, 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 three weeks.
The primary outcome measure of the three-month 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 for the long-term extension study is to evaluate the long-term safety and tolerability of luspatercept.
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
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
For more information, please visit www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.
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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.
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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
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