February 5, 2018

Cerus reported that the primary efficacy and safety endpoints were successfully achieved in the company’s Phase III transfusion study of chronic anemia evaluating INTERCEPT-treated red blood cells (RBCs) in thalassemia patients, SPARC. A total of 86 patients were enrolled at three participating international sites in the double blinded, cross-over study. Subjects were randomly assigned to a sequential treatment period of either INTERCEPT-treated RBCs or conventional RBCs with cross over to the other treatment upon completion of the first treatment period. The study’s primary efficacy endpoint used a non-inferiority design to assess up to a 15% relative difference in the mean consumption of hemoglobin between INTERCEPT-treated RBC and conventional RBC.

February 16, 2015

Emmaus Life Sciences released results of a phase III study of oral pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell anemia and sickle beta-0 thalassemia. The multi-center, double-blind clinical trial studied PGLG for sickle cell disease (SCD) in 230 pediatric patients as young as five years old and adults. Study participants were randomized to receive daily PGLG (152 patients) or placebo (78 patients) for 48 weeks, after which treatment levels were tapered to zero. Researchers observed patients who received PGLG experienced fewer painful crises (three v. four events during the study period, a 25% reduction) and a longer time to a pain crisis than patients receiving placebo. Treated patients also were less likely to be hospitalized (two v. three events during the study period, a 33% reduction) and spent less time in the hospital for these events (6.5 v. 11 days, a 41% reduction) than those receiving placebo. The percentage of patients experiencing acute chest syndrome, a severe complication of SCD, was less than half among the PGLG group as compared to the placebo group (11.9% v. 26.9%, or 58% fewer cases). The therapy has Orphan Drug designation in the U.S. and Europe and Fast Track designation from the FDA.

December 19, 2011

Novartis reported results from a phase III trial of Exjade for non-transfusion-dependent thalassemia. This one-year, randomized, double-blind, placebo-controlled pivotal study, THALASSA, enrolled 166 subjects 10 years of age and older with beta-thalassemia intermedia, alpha-thalassemia or Hemoglobin E/beta-thalassemia. The subjects were randomized to Exjade doses of 5 mg/kg/day or 10 mg/kg/day or matching placebo. Exjade at a 10 mg/kg/day starting dose significantly reduced liver iron concentration (LIC) from baseline by 3.8 mg of iron per gram of liver dry weight (Fe/g dw) compared to an increase of 0.38 mg Fe/g dw for placebo (p<0.001). The 10 mg/kg/day dose was superior to a 5 mg/kg/day dose (p≡0.009). In the 10 mg/kg arm, 49% of subjects had a LIC decrease of at least 30% from baseline versus only 2% in the placebo arm. In addition, 56% of subjects in the 10 mg/kg arm had a LIC decrease of greater than or equal to 3 mg at one year compared to 11% in the placebo arm.