- A Phase 3 clinical trial, BELIEVE, evaluating Celgene (NASDAQ:CELG) and Acceleron Pharma's (NASDAQ:XLRN) luspatercept in adults with beta-thalassemia-associated anemia met the primary endpoint. The results were presented at ASH in San Diego.
- A statistically significant proportion patients in the treatment group experienced hematological improvement compared to placebo, defined as at least a 33% reduction from baseline in red blood cell count (RBC) transfusion burden. Specifically, 21.4% (n=48/224) of patients receiving luspatercept experienced red blood cell responses compared to 4.5% (n=5/112) for control (p<0.0001).
- 7.6% (n=17/224) of patients in the treatment group experienced at least a 50% reduction in RBC transfusion burden versus 1.8% (n=2/112) for placebo (p=0.0303).
- On the safety front, 15.2% (n=34/223) of patients receiving luspatercept experienced serious treatment-emergent adverse events (TEAEs) compared to 5.5% (n=6/109) for control. 13.9% (n=31/223) of patients in the treatment group experienced life-threatening TEAEs versus 10.1% (n=11/109) for placebo. The three most common Grade 3/4 TEAEs in the treatment group were anemia (3.1%), increased liver iron concentration (2.7%) and excess uric acid in the blood (2.7%). There was one death (acute gallbladder inflammation) in the control group.
- Marketing applications in the U.S. and Europe will be submitted in H1 2019.
- Fast Track-tagged luspatercept is a protein therapeutic called a ligand trap. It promotes red blood cell formation by inhibiting members of the transforming growth factor beta superfamily, proteins that induce programmed cell death (apoptosis).
- Previously: Acceleron's luspatercept continues to show positive effects in mid-stage beta-thalassemia studies (June 15)
- Previously: Celgene, Acceleron announce positive results in blood disorder drug study (July 9)
- #ASH18
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