Policy & Regulation
Celgene, Acceleron Pharma Present Results of the Phase 3 MEDALIST Trial Evaluating Luspatercept in Patients with Myelodysplastic Syndromes
6 December 2018 - - US-based biopharmaceutical company Celgene Corp. (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) have received results from the pivotal, phase 3 MEDALIST trial evaluating the efficacy and safety of investigational luspatercept to treat patients with ring sideroblast myelodysplastic syndromes -associated anemia who require red blood cell transfusions and who had failed, were intolerant to, or ineligible for erythropoietin therapy, the companies said.

Results were presented by Alan F. List, M.D. during the Plenary Scientific Session at the 60th American Society of Hematology annual meeting and Exposition in San Diego, C.A. (Abstract #1).

MEDALIST met the primary endpoint of red blood cell transfusion independence (RBC-TI) for 8 or more weeks during the first 24 weeks of the study.

Treatment with luspatercept resulted in a statistically significantly greater proportion of patients achieving RBC-TI ≥ 8 weeks compared to placebo.

The study also found in secondary endpoints that treatment with luspatercept resulted in a statistically significant higher %age of patients achieving RBC-TI of 12 or more weeks in the first 24 or 48 weeks of the study, as well as hematologic improvement-erythroid of eight or more weeks.

Treatment-emergent adverse events (TEAEs) of Grade 3 or 4 were reported in 42.5% (65/153) of patients receiving luspatercept and 44.7% (34/76) of patients receiving placebo.
Progression to acute myeloid leukemia occurred in four patients, three patients receiving luspatercept and one patient receiving placebo. Five patients receiving luspatercept and four patients receiving placebo experienced one or more TEAE that resulted in death.

Luspatercept is not approved in any region for any indication. The companies are planning regulatory application submissions of luspatercept in the United States and Europe in the first half of 2019.

MEDALIST is a phase 3, randomised, double blind, placebo-controlled, multi-center study evaluating the safety and efficacy of luspatercept in patients with very low-, low-, or intermediate-risk non-del(5q) myelodysplastic syndromes.

All patients were RBC transfusion dependent and were either refractory or intolerant to prior erythropoiesis-stimulating agent therapy, or were ESA naïve with endogenous serum erythropoietin ≥ 200 U/L, and had no prior treatment with disease modifying agents.

The median age of the patients enrolled in the trial was 71 years in the luspatercept treatment group and 72 years in the placebo group.

Median transfusion burden in both treatment arms was 5 RBC units/8 weeks. 229 patients were randomised to receive either luspatercept 1.0 mg/kg (153 patients) or placebo (76 patients) via subcutaneous injection once every 21 days. The study was conducted at 65 sites in 11 countries.

Luspatercept is a first-in-class erythroid maturation agent that is believed to regulate late-stage red blood cell maturation.

Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. Phase 3 clinical trials continue to evaluate the safety and efficacy of luspatercept in patients with MDS (the MEDALIST trial) and in patients with beta-thalassemia (the BELIEVE trial).

The COMMANDS phase 3 trial in first-line, lower-risk, MDS patients, the BEYOND phase 2 trial in non-transfusion-dependent beta-thalassemia, and a phase 2 trial in myelofibrosis are ongoing.

Celgene Corp., headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialisation of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation.

Acceleron is a Cambridge-based, clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialisation 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 Phase 2 pulmonary programme with sotatercept in pulmonary arterial hypertension.
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