Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel protein therapeutics for cancer and rare diseases, today announced that its collaboration partner, Celgene Corporation, presented interim data demonstrating dose dependent increases in hemoglobin in patients with end stage renal disease (ESRD) on hemodialysis. The data are from an ongoing phase 2a study of sotatercept and were presented at the National Kidney Foundation 2014 Spring Clinical Meeting in Las Vegas, NV. The phase 2a clinical trial of sotatercept is designed as a randomized, placebo-controlled, dose escalation study to evaluate sotatercept for the correction of anemia in patients with end stage renal disease on hemodialysis. There are four sotatercept dose groups (0.3 mg/kg, 0.5 mg/kg, 0.7 mg/kg, or 0.7 mg/kg loading dose followed by 0.4 mg/kg). Highlights of the data from the two lowest dose level cohorts (0.3 mg/kg and 0.5 mg/kg) are summarized below:
- Hemoglobin increase ≥1.0 g/dL was achieved by 20%, 37% and 40% of patients in the placebo, sotatercept 0.3 mg/kg and sotatercept 0.5 mg/kg cohorts, respectively, in the first 28-day cycle following a single dose.
- The mean peak hemoglobin increase in the first 28-day dose cycle was 0.1 g/dL, 0.5 g/dL and 0.8 g/dL in patients in the placebo, sotatercept 0.3 mg/kg and sotatercept 0.5 mg/kg cohorts, respectively.
- Rescue therapy with erythropoietin, if hemoglobin levels fell to less than 9 g/dL, was required in 40% of placebo patients, 13% of patients at sotatercept 0.3 mg/kg, and 0% of patients at sotatercept 0.5 mg/kg.
About SotaterceptSotatercept is an activin receptor type IIA fusion protein that acts as a ligand trap for members in the Transforming Growth Factor-Beta (TGF-β) superfamily involved in the late stages of erythropoiesis (red blood cell production). Sotatercept 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 Celgene are jointly developing sotatercept as part of a global collaboration. Sotatercept is currently in multiple phase 2 clinical trials. For more information, please visit www.clinicaltrials.gov. About Acceleron Acceleron is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel protein therapeutics for cancer and rare diseases. The company is a leader in understanding the biology of the Transforming Growth Factor-Beta (TGF-β) protein superfamily, a large and diverse group of molecules that are key regulators in the growth and repair of tissues throughout the human body, and in targeting these pathways to develop important new medicines. Acceleron has built a highly productive R&D platform that has generated innovative clinical and preclinical protein therapeutic candidates with novel mechanisms of action. These protein therapeutic candidates have the potential to significantly improve clinical outcomes for patients with cancer and rare diseases. For more information, please visit www.acceleronpharma.com.