Onto the Fast Track for MarzAA
Catalyst Biosciences gains FDA Fast Track designation for subcutaneous MarzAA for the treatment of Hemophilia
SOUTH SAN FRANCISCO, Calif.—Today, Catalyst Biosciences, Inc. announced that the FDA has now granted Fast Track Designation for Marzeptacog alfa (activated), also known as MarzAA.
“We are entering into a Phase 3 clinical trial to evaluate the safety and efficacy of MarzAA for on-demand treatment and control of episodic bleeding in subjects with Hemophilia A or Hemophilia B with inhibitors,” notes Catalyst’s website.
MarzAA is Catalyst Biosciences’ subcutaneously (SQ) administered next-generation engineered coagulation Factor VIIa (FVIIa). The drug candidate is expected to enter the pivotal Phase 3 study this month.
The Phase 3 CRIMSON 1 study will enroll approximately 60 subjects to treat 244 eligible bleeding episodes with each treatment.
“The study will assess the effectiveness of SQ MarzAA, using up to three doses to treat a bleeding episode. The primary endpoint will be hemostatic efficacy using a standard 4-point assessment scale,” the website adds.
The primary endpoint for the trial is the percentage of treated bleeds resulting in effective hemostasis at the 24-hour timepoint. The trial’s objective is to demonstrate non-inferiority of MarzAA, compared with the current standard of care.
MarzAA met all primary and secondary endpoints of its previous Phase 2 trial. The drug candidate significantly reduced the annualized bleed rate in patients with Hemophilia A or B with inhibitors. The results of the trial were presented in an oral presentation at the 2019 Congress of the International Society on Thrombosis and Haemostasis.
“We also plan to initiate a Phase 1/2 trial of MarzAA in Factor VII Deficiency, Glanzmann Thrombasthenia, and patients with Hemophilia A with inhibitors treated with Hemlibra for treatment of bleeding in late 2020,” notes the website. “Our preclinical data suggest that MarzAA has the potential to be used for treatment of episodic bleeding and supports further clinical testing in individuals with hemophilia with inhibitors or for other conditions.”
“We believe the FDA Fast Track Designation validates MarzAA’s potential to improve patient care. As the only SQ delivered therapy in development for on-demand treatment of bleeding events, MarzAA is uniquely positioned to become an important addition to the treatment landscape,” stated Nassim Usman, Ph.D., president and chief executive officer of Catalyst Biosciences.