"In previous clinical studies, sotatercept has shown encouraging
activity by increasing red blood cells as well as bone mass in
About the Phase 2 Clinical Trial
The phase 2 clinical trial in patients with end stage renal disease (ESRD) who are on hemodialysis is designed as a two-part study to assess the safety and efficacy of sotatercept as a therapy to treat anemia and potentially to control the adverse manifestations of chronic kidney disease mineral and bone disorder (CKD-MBD). Patients in both parts of the study must first be on a stable dose of an erythropoiesis stimulating agent (ESA) to maintain hemoglobin levels and will then be switched to treatment with sotatercept after an ESA treatment free period of approximately five days.
The first part is a dose-escalation study of intravenous (IV) and subcutaneous (SC) routes of administration of sotatercept in approximately 60 patients to evaluate pharmacokinetics, safety and tolerability. Patients in the dose escalation part of the study will be given sotatercept once every fourteen days up to a total of eight doses and followed for approximately four months after their last dose. The first part of the study is designed to inform the dosing regimens to be tested in the second part of the clinical trial. The second part will be a randomized, controlled study of approximately 230 patients to evaluate the efficacy and safety of sotatercept versus an erythropoiesis stimulating agent (ESA). Efficacy measures for part two of the study include the change in mean hemoglobin concentration from baseline and the ability of sotatercept to maintain patients' hemoglobin levels within a target range after switching from an ESA to sotatercept. Measures of biomarkers for bone remodeling and mineral metabolism also will be studied.
For additional information on this clinical trial, please visit www.clinicaltrials.gov, identifier NCT01999582.
About Chronic Kidney Disease
Chronic Kidney Disease (CKD) is a serious condition characterized by the progressive loss of kidney function and is most often caused by diabetes or high blood pressure. CKD is classified in five stages according to the degree of kidney impairment. End Stage Renal Disease (ESRD), also known as kidney failure, is the most advanced stage of CKD and a life-threatening condition in which patients require a either kidney transplant or dialysis for survival. Anemia, low levels of red blood cells, is a common secondary disorder to Chronic Kidney Disease and worsens with progression of the disease. Disturbance in mineral and bone metabolism, known as Chronic Kidney Disease Mineral and Bone Disorder (CKD-MBD), is also a common complication in CKD patients that affects almost all patients who are on dialysis. CKD-MBD can lead to bone abnormalities and/or calcification in soft tissue, especially blood vessels, and is associated with increased morbidity and mortality in CKD patients.
Sotatercept 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.
For more information, please visit www.acceleronpharma.com.
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements about the
Company's strategy, future plans and prospects, including statements
regarding the development of the Company's compounds, including
sotatercept, ACE-536, dalantercept, or ACE-083 and the Company's TGF-β
superfamily program generally, the timeline for clinical development and
regulatory approval of the Company's compounds, the expected timing for
the reporting of data from ongoing trials, and the structure of the
Company's planned or pending clinical trials. The words "anticipate,"
"appear," "believe," "estimate," "expect," "intend," "may," "plan,"
"predict," "project," "target," "potential," "will," "would," "could,"
"should," "continue," and similar expressions are intended to identify
forward-looking statements, although not all forward-looking statements
contain these identifying words. Each forward-looking statement is
subject to risks and uncertainties that could cause actual results to
differ materially from those expressed or implied in such statement.
Applicable risks and uncertainties include the risks that the
preclinical testing of the Company's compounds and preliminary data from
clinical trials may not be predictive of the results or success of
ongoing or later clinical trials, that data may not be available when we
expect it to be, that the Company or its collaboration partner, Celgene,
will be unable to successfully complete the clinical development of its
compounds, that the development of the Company's compounds will take
longer or cost more than planned, and that the Company's compounds will
not receive regulatory approval or become commercially successful
products. Other risks and uncertainties include those identified under
the heading "Risk Factors" included in the Company's Registration
Statement on Form S-1 which was declared effective by the
Senior Vice President and Chief Business Officer
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