Acceleron Announces Updated Results from Ongoing Phase 2 Trials of Luspatercept in Myelodysplastic Syndromes at the ASCO 2018 Annual Meeting
– Multiple patients continue on treatment with clinically meaningful increases in hemoglobin and reduction in red blood cell transfusions surpassing 3 years –
– Top-line results from the MEDALIST Phase 3 trial are on track for the middle of 2018 –
– COMMANDS Phase 3 trial in first-line, lower-risk MDS to be initiated in Q3 2018 –
“The ongoing Phase 2 trials continue to provide important insights into
luspatercept’s potential to deliver long-term benefit to thousands of
patients with lower-risk MDS,” said
Patients with MDS suffer from insufficient production of red blood cells, resulting in chronic anemia that can lead to debilitating fatigue, diminished quality of life and increased mortality. Because MDS-related chronic anemia often fails to respond to unapproved therapies which include erythropoiesis-stimulating agents, many patients require frequent red blood cell transfusions.
Phase 2 Results
A total of 101 patients with lower-risk MDS have been treated with luspatercept (dose levels ≥ 0.75 mg/kg) in the Phase 2 trials.
- 55% (55 of 101 patients) achieved a clinically meaningful erythroid improvement (IWG HI-E criteria).
44% (30 of 68 patients) with a red blood cell (
RBC) transfusion burden at baseline achieved RBCtransfusion independence (RBC-TI) for at least 8 weeks.
- The mean duration of treatment for RBC-TI responders was 18.3 months (n=30, ongoing).
- Multiple patients continue on treatment through 40 months, and continue to sustain a clinically meaningful increase in hemoglobin and reduction in transfusion burden.
Phase 2 Safety Summary
The majority of adverse events (AEs) were Grade 1 or 2. Grade 3 non-serious AEs possibly related to study drug were ascites, blood bilirubin increase, bone pain, hypertension, mucosal inflammation, platelet count increase, and transformation to AML (previously reported as a blast cell count increase). The Grade 3 non-serious AEs occurred in one patient each, with the exception of hypertension in 2 patients.
Serious AEs (SAEs) possibly related to study drug were general physical health deterioration, muscular weakness, musculoskeletal pain, and myalgia. The four SAEs occurred in three individual patients.
The ASCO MDS 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 any use in any country.
About the Ongoing MDS Phase 2 Trials
Data from two Phase 2 trials were presented at the 2018 ASCO Annual Meeting: the base study in which patients with lower-risk MDS 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.
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.
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development of the Company's compounds will take longer and/or cost more
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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