The shares of Agios Pharmaceuticals Inc (NASDAQ:AGIO) fell following the firm’s announcement of the results obtained from a midstage trial for therapy that seeks to treat refractory or relapsed acute myeloid leukemia. The results which were meant to evaluate investigational oral IDHIFA were disappointing as they did not meet expectations. The treatment is being developed in conjunction with Celgene Corp.
“We are working with our partner Celgene to quickly bring this oral, targeted therapy to patients with limited treatment options,” said Agios’ chief medical officer Chris Bowden.
Overall response rate
Statistics that were presented during the European Hematology Association’s 22nd Congress showed that the drug’s overall response rate was 37%. The complete response rate was 20.1% in patients numbering 214 who were given enasidenib.
In the study the maximum tolerated dosage was never reached despite the fact that the safety profile that had to be observed in the case of enasidenib was the same as in the data that had been previously reported. Enrollment for the study was finalized last year in November and 52 patients were enrolled. The patients were started on a dose of 50 milligrams or 300 milligrams taken twice a day.
Adverse events
Some of the side effects that were observed in those who took the treatment include nausea, increase in blood bilirubin, decline in appetite, fatigue and diarrhea. 48% of the participants reported nausea while 41% of the participants reported diarrhea. There was a decline in appetite for 34% of the participants and an increase in blood bilirubin for 33% of the participants.
In all patients who participated in the trial, 26.1% of them reported serious adverse events that were related to the therapy. Notably this was IDH differentiation syndrome in 7% of the cases and leukocytosis in 4% of the cases. 2% of the cases reported hyperbilirubinemia and 3% of the cases reported tumor lysis syndrome. About five participants in the study experienced serious adverse events and this included withdrawal hemolysis which was followed by anemia, osteoporosis, pharyngitis and hypertriglyceridemia.
On Monday shares of Agios Pharmaceuticals Inc fell by 7.57% to close the day at $54.70.