| 2 min read
Register for free to listen to this article
Listen with Speechify
0:00
2:00
SOUTH SAN FRANCISCO, Calif.—Catalyst Biosciences, Inc., a biopharmaceutical company focused on developing novel subcutaneous (SQ) therapies for hemophilia and other inherited bleeding disorders, announced today that it has received guidance from the U.S. Food and Drug Administration and the European Medicines Agency on a pivotal Phase 3 trial design for Marzeptacog alfa (activated), known as MarzAA. The open-label trial will evaluate the efficacy of SQ MarzAA to treat episodic bleeding in individuals with hemophilia A or B with inhibitors.
 
MarzAA is a potent, subcutaneously administered, next-generation recombinant Factor VIIa variant. Catalyst previously completed a Phase 2 open-label SQ prophylaxis trial of MarzAA, which met the trial’s primary and secondary endpoints in 2019. The preclinical and clinical data support not only MarzAA’s potential use for treatment of episodic bleeding episodes, but also further clinical testing for on-demand treatment of bleeds in individuals with hemophilia, Glanzmann Thrombasthenia, Factor VII deficiency and other bleeding disorders.
 
“Now that we have received regulatory feedback, we have initiated preparations for a Phase 3 trial of MarzAA in individuals with hemophilia A or B with inhibitors,” stated Nassim Usman, Ph.D., president and chief executive officer of Catalyst. “We plan to enroll the first patient before year end, however that date is dependent on the readiness of our trial sites and improvements in the current effects of COVID-19 on clinical trial execution. At present, manufacturing and drug supply are unaffected for our clinical trials.”
 
The Phase 3 Crimson-1 (Subcutaneous Marzeptacog Alfa [Activated] For On demand Treatment and Control of Bleeding Episodes in Subjects with Hemophilia A or Hemophilia B with Inhibitors) study will enroll individuals who experience episodic bleeding. Crimson-1 will be an open-label global trial, evaluating the safety and efficacy of SQ MarzAA in the treatment of approximately 230 bleeding episodes in approximately 75 patients, compared with their prior standard of care in a similar number of bleeding episodes.
 
The study will assess the effectiveness of SQ MarzAA, using up to 3 doses to treat a bleeding episode. The primary endpoint will be hemostatic efficacy, using a standard 4-point assessment scale.

Related Topics

Loading Next Article...
Loading Next Article...
Subscribe to Newsletter

Subscribe to our eNewsletters

Stay connected with all of the latest from Drug Discovery News.

Subscribe

Sponsored

Reliable fluid biomarkers strategies for clinical neuroscience research

Reliable fluid biomarkers strategies for clinical neuroscience research

Explore how validated fluid biomarker assays advance clinical research for neurological diseases.
A group of blue capsules is scattered on a bright yellow surface, with one capsule opened to reveal white powder inside.

Understanding drug impurities: types, sources, and analytical strategies

Unseen and often unexpected, drug impurities can slip in at every drug development stage, making their detection and control essential.
Laboratorian with a white coat and blue gloves pipettes green liquid into a beaker with multicolored liquids in beakers and tubes in the blue-tinged, sterile laboratory background.

Discovering cutting-edge nitrosamine analysis in pharmaceuticals

New tools help researchers detect and manage harmful nitrosamine impurities in drugs such as monoclonal antibodies.
Drug Discovery News March 2025 Issue
Latest IssueVolume 21 • Issue 1 • March 2025

March 2025

March 2025 Issue

Explore this issue