By: IPP Bureau
Last updated : December 10, 2023 6:44 pm
Demonstrate significant bleed reduction in hemophilia A and B
Pfizer presented results from the pivotal Phase 3 BASIS clinical trial evaluating marstacimab for the treatment of people with severe hemophilia A and moderately severe to severe hemophilia B.
The results from the BASIS trial demonstrated a statistically significant and clinically meaningful effect on annualized bleeding rate (ABR). The findings were presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego.
“For more than five decades, the most common treatment for hemophilia A and B has been intravenous infusions that are often administered multiples times per week,” said James Rusnak, M.D., Ph.D., Senior Vice President, Chief Development Officer, Internal Medicine and Infectious Diseases, Research and Development, Pfizer.
“Based on these results and if approved, we believe marstacimab could offer a subcutaneous option with a compelling combination of efficacy and safety that may significantly reduce the risk of bleeding. We look forward to potentially bringing this treatment option to people living with hemophilia A and B without inhibitors.”
The safety profile for marstacimab was consistent with Phase 1/2 results and treatment was generally well-tolerated.
“Recognizing the uncertainty that living with hemophilia can present for patients, the results from the BASIS trial are particularly encouraging as reductions in ABR were seen in the 12-month treatment period and then retained in long-term follow-up,” said Davide Matino, M.D., M.Sc., Assistant Professor of Medicine, McMaster University. “Based on these results, marstacimab has shown the potential to address the diverse needs of appropriate patients with hemophilia A or B without inhibitors with weekly subcutaneous administration in a flat dose that is not weight-based, and with low monitoring requirements.”
Pfizer currently has three Phase 3 programs investigating novel treatment options for people living with hemophilia. In addition to the BASIS study, fidanacogene elaparvovec and giroctocogene fitelparvovec are investigational gene therapy treatments being studied for the treatment of adults living with hemophilia B and hemophilia A, respectively.