Pfizer succeeds in Phase 3 trial for hemophilia candidate
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Pfizer (NYSE:PFE) announced Tuesday that a pivotal Phase 3 trial for marstacimab, a candidate targeted at certain hemophilia patients as a prophylactic regimen, reached its primary endpoints.
The global study involving about 145 adolescent and adult participants was designed to evaluate the annualized bleed rate through 12 months in patients treated with marstacimab. The BASIS trial included those with severe hemophilia A or moderately severe to severe hemophilia B.
According to the company, BASIS showed that marstacimab as a prophylactic treatment led to a statistically significant and clinically relevant reduction in annualized bleeding rate in severe hemophilia A and hemophilia B without inhibitors.
Pfizer (PFE) said that the experimental therapy was generally well-tolerated indicating a safety profile consistent with Phase 1/2 data.
Subcutaneously delivered marstacimab has a different mechanism of action than FVIII and FIX replacement treatments, the current standard of care for hemophilia.
“These results support the potential for marstacimab to become the first once-weekly non-factor treatment for people with hemophilia B and a treatment option that helps address the diverse needs of patients with hemophilia A or B without inhibitors,” said Chris Boshoff, the company’s head of development for Oncology and Rare Disease.
The full dataset from BASIS is currently under review, and Pfizer (PFE) plans to present the data at an upcoming scientific conference.