Remarkable Results From a Phase 2 Trial for Beta Thalassemia

As reported in Biospace; a Phase 2 study by Bristol-Myers Squibb and Acceleron Pharma Inc. earned itself recognition as among the top six abstracts received by the Presidential Symposium of European Hematology Association’s Virtual Congress for its success alleviating symptoms of patients with non-transfusion dependent (NTD) beta thalassemia. 
The study evaluated the new treatment Rebloyzl, the first-in-class erythroid maturation agent that increases hemoglobin levels in the blood. Patients with this order have a mutation preventing the blood cells from developing enough hemoglobin, slowing the transportation of oxygen to the body. The treatment was paired with the best supportive care for adult patients with non-transfusion dependent (NTD) beta thalassemia. 
 
They found the treatment was overall well tolerated and with high efficacy, helping patients achieve healthier levels of hemoglobin and avoid transfusions. Habib Dable, President and Chief Executive Officer of Acceleron, said,
 
“We are very encouraged by the magnitude of improvement seen among Reblozyl-treated patients in the BEYOND trial. These data further strengthen our confidence in Reblozyl’s potential to become a meaningful option for this important, underserved patient population around the world.”
 

Beta thalassemia

 Beta thalassemia is a rare blood disorder in which a mutation causes lower levels of hemoglobin. Hemoglobin is a protein found in red blood cells that carry’s oxygen through the body. While symptoms vary in severity, the disease is divided into three subtypes (minor, intermedia, and major). The disorder is characterized by anemia, causing fatigue, weakness, dizziness, shortness of breath, blood clots, headaches, pallor, and splenomegaly. The genetic disorder has an FDA-approved treatment, Thiotepa. However, patients may also use other treatments to manage symptoms, including blood transfusions. 
 
 The BEYOND study intended to raise hemoglobin levels to help patients avoid transfusions and other medical flare-ups. They set out to see patients achieve a primary endpoint of  ≥1.0 g/dL mean Hb increase from the baseline levels, observed of a continuous 12-week period, without the need for the symptomatic RBC transfusions.
 
The researchers observed 145 patients over 48 weeks, with two out of two patients receiving Rebloyzl; the others received a placebo. Every three weeks they received 1 milligram/kilogram of the treatment, and continued the recommended supportive care of iron chelation therapy and transfusions as needed.
This blood deficiency has consequences on many systems throughout the body. This means patients with this disorder rely on a range of symptomatic treatments, as there’s no widely available cure still to get to the roots.
“Patients with non-transfusion dependent beta thalassemia experience chronic anemia and iron overload, which may lead to a range of clinical complications, and treatment options are greatly needed,” – Ali Taher, M.D., Ph.D., FRCP, American University of Beirut and BEYOND study investigator.
 

The Treatment Finds Success

Less than mid-way into the trial, the scientists had already achieved their goals. At the conference, Dr. Noah Berkowitz, senior vice president at Bristol-Myers Squibb, explained, 
“The results we are presenting at EHA continue to highlight multiple benefits observed with Reblozyl to treat anemia and achieve transfusion independence, as well as show its potential for patients with non-transfusion dependent disease who face a range of serious, often lifelong health complications. Along with our partners at Acceleron, we are committed to advancing our clinical program for Reblozyl for patients living with anemia-associated blood disorders.”
 
They found the treatment group achieved remarkable hemoglobin increases, with 77.7% of patients experiencing a uptake of ≥1.0 gram/deciliter while none of the placebo group experienced any hemoglobin increase. They also found 89.6% of patients in the treatment group passed through the trial transfusion free, while only 67.3% of the placebo group remained without transfusions.
Dr. Ali Taher of American University of Beirut and BEYOND study investigator concluded:
“Results from the BEYOND study show the clinical potential of luspatercept to sustain the elevation of hemoglobin levels in a majority of patients regardless of their baseline hemoglobin status, and improvements were noted in quality of life outcomes in adults with non-transfusion dependent beta thalassemia.”
 
 

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