Mast Therapeutics Reports Top-Line Results From Phase 3 Study In Sickle Cell Disease
"We are exceedingly disappointed with these top-line results. While clearly not the outcome we wanted, we believe the insights and data from the largest placebo-controlled clinical trial ever completed in sickle cell disease will substantially advance the understanding of vaso-occlusive crisis and the still maturing clinical science necessary to support the development of new therapeutics for this debilitating disease," stated
"These analyses are limited to just top-line data, so in the coming weeks the Company intends to review the full data set from EPIC. In addition, we plan to perform an interim analysis of the ongoing heart failure trial of vepoloxamer. However, based on the data we've seen to date, we expect we will terminate all clinical development of vepoloxamer. Consequently, while we evaluate our options, we intend to significantly and immediately reduce our operating expenses and continue our efforts with AIR001, our lead asset in heart failure with preserved ejection fraction, which currently is the subject of a 100-patient phase 2 study expected to complete enrollment by the end of 2017," continued Mr. Culley.
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About the EPIC Study
The EPIC study was a randomized, double-blind, two-arm, placebo-controlled, Phase 3 clinical trial of vepoloxamer in individuals with sickle cell disease hospitalized for acute pain typical of vaso-occlusive crisis who required treatment with parenteral opioid analgesia. The primary objective of the study was to evaluate the efficacy of vepoloxamer in reducing the duration of vaso-occlusive crisis, with the duration of crisis measured from the time of randomization to the time at which the patient received the last dose of parenteral opioid analgesia for the treatment of vaso-occlusive crisis prior to hospital discharge. Vepoloxamer or placebo (0.45% saline) was administered intravenously as a 1 hour loading dose infusion (100 mg/kg), immediately followed by a continuous maintenance infusion (30 mg/kg/hr) for at least 12 hours and up to 48 hours. Randomization was stratified by age (≥4 to <16 years or ≥16 to ≤65 years), use of hydroxyurea (yes or no), and pain score (measured using the Wong-Baker FACES® Pain Rating Scale at time of randomization: <8 or ≥8). The study was 90% powered to detect a 17% (16-hour) difference in treatment arms, with a statistical significance level of p=0.05 (assuming an average crisis duration of 96 hours in the control arm and a coefficient of variation > 50%). Secondary efficacy endpoints were to compare the rates of re-hospitalization for vaso-occlusive crisis within 14 days of initial hospital discharge and the occurrence of acute chest syndrome within 120 hours of randomization between the treatment and control groups.
A total of 388 patients, ages four to 46, were randomized in EPIC. More than 75 study sites in 14 countries participated. The average age was 15 years. Patients under age 18 accounted for approximately 71% of total subjects. Approximately 61% of patients were concurrently on hydroxyurea therapy.
About Sickle Cell Disease and Vaso-Occlusive Crisis
Sickle cell disease is a chronic, genetic blood disorder that affects millions worldwide and an estimated 100,000 people in
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