Catalyst Completes Phase 1 Trial of MarzAA, Is on Track to Start Phase 3

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by Joana Carvalho |

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Despite the COVID-19 pandemic, Catalyst Biosciences has successfully completed a Phase 1 trial of marzeptacog alfa activated (MarzAA), with results supporting the treatment as an on-demand option for bleeding episodes in people with hemophilia A or B.

The study, MAA-102 (NCT04072237), was designed to assess the safety and pharmacological properties of  MarzAA in patients with moderate-to-severe hemophilia A or B, with or without inhibitors — neutralizing antibodies that render conventional replacement therapies useless. A first administration directly into the bloodstream was followed by single increasing doses of subcutaneous (under-the-skin) injections.

Early findings from MAA-102 presented at the 13th Annual Congress of the European Association of Haemophilia and Allied Disorders in February had shown that when given subcutaneously, MarzAA was well-tolerated and rapidly reached and maintained the levels required to stop ongoing bleeding. Final study results will be presented at an upcoming scientific meeting.

In line with a recent update after guidance from the U.S. Food and Drug Administration and the European Medicines Agency, Catalyst also said it remains on track to enroll the first patient into an upcoming Phase 3 trial of MarzAA before the end of the year.

The study, called Crimson-1, will investigate the safety and effectiveness of subcutaneous MarzAA as an on-demand treatment for bleeds in approximately 75 patients with hemophilia A or B with inhibitors. Each patient may receive up to three MarzAA injections for each bleeding episode.

The main goal of the study is to assess the effectiveness of MarzAA at controlling acute bleeding episodes compared to standard therapies.

“Successful completion of our Phase 1 study, in the midst of the global coronavirus pandemic, demonstrates the commitment of our team and is one more step towards enrolling the first patient in our pivotal Phase 3 trial for MarzAA later this year,” Nassim Usman, PhD, president and CEO of Catalyst, said in a press release.

MarzAA is a recombinant (man-made) version of the blood-clotting protein factor VIIa (FVIIa) designed specifically to prevent acute bleeds in hemophilia patients with inhibitors. According to Catalyst, the therapy combines a potent blood-clotting activity with sustained activity at the bleeding site.

Preclinical studies showed that when given subcutaneously to a mouse model of hemophilia A minutes before injury, MarzAA was as effective at reducing bleeding as NovoSeven RT (recombinant FVIIa), an approved bypassing agent marketed by Novo Nordisk.

These findings propelled MarzAA’s clinical development as an on-demand treatment for people with hemophilia or FVII deficiency.

In a Phase 2/3 trial (NCT03407651), daily preventive treatment with MarzAA safely reduced the number of bleeds per year and lowered the proportion of days with bleeding.

“We believe MarzAA’s rapid uptake and extended half-life with SQ [subcutenous] administration has the potential to disrupt the multi-billion dollar hemophilia and bleeding disorders market by addressing clear unmet needs for treatment and prevention of bleeding,” Usman said.