Catalyst Biosciences presents interim FVIIa data at EAHAD
Catalyst Biosciences presented updated interim data from the Phase 2/3 trial of subcutaneous prophylactic Factor VIIa, or FVIIa, variant marzeptacog alfa currently being developed for the treatment of hemophilia A or B with inhibitors. The data were delivered in an oral presentation at the annual congress of the European Association for Haemophilia and Allied Disorders, or EAHAD. The company presented the updated results which include data from a total of 11 subjects. Seven subjects have completed dosing in the trial, two are currently dosing, others are completing screening and enrollment is now complete. To date, for all enrolled patients, the mean annualized bleed rate, or ABR, prior to the trial was 19 bleeds per year. All subjects who have completed dosing have had clinically significant reductions in ABR and five experienced no bleeds with individualized dosing of either 30 microg/kg MarzAA or 60 microg/kg MarzAA for 50 days. Six subjects had no spontaneous bleeds at their final dose level. The median proportion of days with bleeding for the seven subjects during the pre-study period was 11% and this was significantly reduced to a median of 1% during the treatment period. A total of more than 450 days of subcutaneous dosing of MarzAA have been completed with only six localized skin reactions in two subjects, and no anti-drug antibodies to MarzAA have been detected to date. An additional aim of the study is to examine the quality of life, or QOL, of individuals who have inhibitors. Results of the analysis have shown that QOL is measurably worse in those with inhibitors compared with those without inhibitors. Interim results after treatment for 50 days with subcutaneous MarzAA showed an improvement in the QOL of individuals, as measured using the Haemophilia quality of life questionnaire and the Haemophilia activity list.