9 April 2019 - - US-based biopharmaceutical company Celgene Corp. (NASDAQ: CELG) has submitted a Biologics License Application for luspatercept, an erythroid maturation agent, for the treatment of adult patients with very low to intermediate risk myelodysplastic syndromes -associated anemia who have ring sideroblasts and require red blood cell transfusions and for the treatment of adult patients with beta-thalassemia-associated anemia who require RBC transfusions, US-based Celgene Corp. (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) said.

The submission is based on the safety and efficacy results of the pivotal phase 3 studies MEDALIST and BELIEVE, both recently presented at the American Society of Hematology annual meeting, where MEDALIST was included in the plenary session.

The companies also plan to submit a marketing application to the European Medicines Agency in the second quarter of 2019.

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

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

Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. A phase 3 trial (COMMANDS) in ESA-naïve, lower-risk MDS patients, the BEYOND phase 2 trial in non-transfusion-dependent beta-thalassemia, and a phase 2 trial in myelofibrosis are ongoing.

MEDALIST is a phase 3, randomized, double blind, placebo-controlled, multi-center study evaluating the safety and efficacy of luspatercept in adults with very low-, low-, or intermediate-risk 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) by subcutaneous injection once every 21 days. The study was conducted at 65 sites in 11 countries.

BELIEVE is a phase 3, randomized, double blind, placebo-controlled multicenter study comparing luspatercept + best supportive care versus placebo + BSC in adults with beta-thalassemia patients who require regular RBC transfusions.

The median age of the patients was 30 years in both treatment arms. 336 patients were randomized to receive either luspatercept 1.0 mg/kg (224 patients) or placebo (112 patients) by subcutaneous injection every 21 days for up to 48 weeks.

Crossover to the luspatercept treatment groups was allowed after unblinding based on the recommendation of an independent Data Safety Monitoring Committee; patients treated with luspatercept will be followed for up to three years.

The study was conducted at 65 sites in 15 countries.

Celgene, 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 clinical-stage biopharmaceutical company focused on 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 programme with ACE-083, a locally-acting Myostatin+ agent in Phase 2 development in facioscapulohumeral muscular dystrophy and Charcot-Marie-Tooth disease and is conducting a Phase 2 pulmonary programme with sotatercept in pulmonary arterial hypertension.