Catalyst Reports Positive Trial Updates for Subcutaneous Therapy for Hemophilia

Catalyst Biosciences, Inc recently reported updated interim data from their phase 2/3, multinational clinical study of marzeptacog alfa (MarzAA), a subcutaneous (administered under the skin) factor VIIa therapy. MarzAA is being developed by Catalyst for the prophylactic treatment of individuals with hemophilia A or B with inhibitors. The data were communicated via an oral presentation at the 12thAnnual Congress of the European Association for Haemophilia and Allied Disorders, held February 6-8, 2019 in Prague, Czechoslovakia.

According to a Catalyst press release, the data was based on a total of 11 trial subjects. Seven subjects have completed dosing in the trial, two are currently dosing, others are completing screening and enrollment is now complete. The mean annualized bleed rate (ABR) among trial participants prior to the study was 19.0 bleeds per year. All subjects who have completed dosing have had “clinically significant” reductions in ABR, while six subjects had no spontaneous bleeds at their final dose level. Study investigators are also looking at quality of life (QOL) in trial participants using various measures. Interim results showed that participants demonstrated improvements in QOL after treatment for 50 days with MarzAA.

“The positive data from our clinical trial demonstrate clinical proof-of-concept for MarzAA as a subcutaneous treatment option for individuals with hemophilia A or B with inhibitors,” said Dr. Nassim Usman, chief executive officer of Catalyst. “We are pleased to announce that we have completed enrollment and look forward to providing topline results in the third quarter of 2019.”

Source: Catalyst press release dated February 8, 2019