Benzinga | Feb 3, 2021 08:03AM EST

Catalyst Biosciences Reports Presentation Of Pre-Clinical Subcutaneously Administered Engeineered Activated Coagulation Factor VII For Treatment Of Episodic Bleeding

Catalyst Biosciences, Inc. (NASDAQ:CBIO) today presented positive pre-clinical data from its Marzeptacog alfa (activated) -- MarzAA, program, the Company's subcutaneously (SQ) administered next-generation engineered activated coagulation Factor VII (FVIIa) for the treatment of episodic bleeding that is entering a Phase 3 registration trial. The data were presented by Tom Knudsen, D.V.M., Ph.D., vice president of translational research and Howard Levy, M.B.B.Ch., Ph.D., M.M.M., chief medical officer in poster sessions at the virtual 14th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD).

The poster, "Subcutaneous Marzeptacog Alfa (Activated) is Effective for On-Demand Treatment of Spontaneous Bleeding in Hemophilia A (HA) Rats," showed that SQ MarzAA was superior to vehicle (p=0.007) and effective for the management of spontaneous mild, moderate, and severe bleeding in HA rats. Notably, the rats have a human-like bleeding phenotype and most rats needed only a single SQ dose of MarzAA to achieve efficacious bleeding control.

The poster, "Subcutaneous Marzeptacog Alfa (Activated) is Effective for On-Demand Treatment in Dogs with Hemophilia A," demonstrated that SQ MarzAA appeared to be consistently effective as an on-demand treatment of spontaneous bleeding in dogs with Hemophilia A. SQ MarzAA treatment rapidly improved all parameters of the thromboelastography (TEG), controlled bleeding, and improved the general condition of the dogs. TEG normalized in 3 of 5 dogs, was confounded by canine FVIII administration in one dog, and did not respond in another dog despite rapid cessation of bleeding. TEG R-time shortened to the same or shorter time than high dose (270 g/kg) intravenous NovoSeven.

"In hemophilia, spontaneous bleeding can occur even on prophylaxis, and there is a need for a safe and effective treatment that can be easily administered subcutaneously to manage breakthrough bleeding. The pre-clinical data we presented at EAHAD showed efficacy in controlling spontaneous bleeding," said Nassim Usman, Ph.D., president and chief executive officer of Catalyst. "We are dedicated to developing meaningful SQ therapy options for individuals living with hemophilia and other rare bleeding disorders."

MarzAA is entering a registrational Phase 3 trial (MAA-304) to treat spontaneous bleeds in patients with Hemophilia A or B with inhibitors (Crimson 1). Catalyst also plans to initiate a Phase 1/2 trial of MarzAA in Factor VII Deficiency, Glanzmann Thrombasthenia, and Hemophilia A with inhibitor patients on Hemlibra prophylaxis for treatment of episodic bleeding (MAA-202).

A copy of the presentation materials can be accessed on the Events and Presentations section of the Catalyst website.