Celgene’s (CELG) and Acceleron Pharma’s investigational drug luspatercept aims to treat anemia in patients suffering with diseases such as beta thalassemia and myelodysplastic syndromes (or MDS). On May 18, 2015, the FDA (U.S. Food and Drug Administration) gave luspatercept a fast track designation as an anemia therapy for transfusion-dependent as well as transfusion-independent beta thalassemia patients. On December 7, 2015, the drug was also granted fast track designation as a therapy for anemia patients suffering with MDS.
If luspatercept gets regulatory approval for these indications, it may have a positive impact on Celgene stock as well as the iShares Core S&P 500 (IVV). Celgene makes up about 0.47% of IVV’s total portfolio holdings.
Clinical trial data
Data from Celgene’s Phase 2 open-label long-term extension trial have shown that 61.0% of MDS patients achieved transfusion independence after receiving luspatercept therapy. Red blood cell (or RBC) transfusion independence is also the primary endpoint for the ongoing Phase 3 trial for this indication, so it increases confidence for a positive outcome for that trial.
In another ongoing Phase 2 open-label extension trial, luspatercept therapy is showing positive results in both transfusion-dependent and transfusion-not-dependent beta thalassemia patients. The adverse events are mild, indicating a high tolerability for the drug.
Among the 31 transfusion-dependent patients enrolled in this trial, 83.0% have seen at least a 33.0% reduction in their total number of transfusions. About 71.0% have seen at least a 50.0% reduction in transfusions. The promising data highlight luspatercept’s strong clinical profile. The drug could enable Celgene to become a key player in the anemia segment, similar to peers such as Novo Nordisk (NVO), Amgen (AMGN), and Novartis (NVS).
Let’s move on now to look at Celgene’s research programs in the myeloid disease segment.