Catalyst Biosciences Presents Updated Data from Its Phase 2/3 Trial of Subcutaneous Marzeptacog Alfa

Catalyst Biosciences (NASDAQ:CBIO) has announced it has presented updated interim data from its Phase 2/3 trial of subcutaneous prophylactic Factor VIIa (FVIIa) variant marzeptacog alfa (activated) (MarzAA).

As quoted in the press release:

The data were delivered in an oral presentation at the 12^th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) being held Feb. 6-8, 2019 in Prague.

“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. “To date, each participant in the trial has experienced a clinically significant reduction in annualized bleed rate and the percentage of days with bleeding, highlighting the potential for MarzAA as a safe and effective subcutaneous treatment to reduce spontaneous bleed rates and improve the quality of life of affected individuals. We are pleased to announce that we have completed enrollment and look forward to providing topline results in the third quarter of 2019.”

Dr. Howard Levy, chief medical officer of Catalyst, 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 (ABR) prior to the trial was 19.0 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 µg/kg MarzAA or 60 µg/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 11.0% and this was significantly reduced to a median of 1.0% during the treatment period (p = 0.016). 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.

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