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Pfizer (PFE) Posts Upbeat Data From Hemophilia Drug Study

Pfizer PFE announced positive data from the phase III BASIS study, which evaluated its investigational anti-tissue factor pathway inhibitor (“anti-TFPI”) marstacimab as a treatment for hemophilia A or B patients, without inhibitors to Factor VIII (“FVIII”) or Factor IX (“FIX”).

The BASIS study achieved its primary endpoints. Data from the study showed that participants treated with marstacimab achieved statistically significant and clinically relevant reduction in annualized bleeding rate (“ABR”) compared with the current standard-of-care treatment for hemophilia patients, during a 12-month period.

Study participants, who received factor replacement therapy in the six-month lead-in period, achieved a 92% reduction in bleeds upon switching to marstacimab. Data from the BASIS study also showed superiority of marstacimab over prophylaxis regimen, achieving a 35% reduction in ABR.

Based on the above results, management believes that marstacimab can potentially become the first once-weekly subcutaneous treatment for hemophilia B patients and a treatment option that addresses the diverse needs of patients with hemophilia A or B without inhibitors.


Shares of Pfizer have lost 30.2% in the year so far against the industry’s 5.7% growth.

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The BASIS study is evaluating marstacimab in adults and adolescents (aged 12 years to less than 75 years) with severe hemophilia A or moderately severe to severe hemophilia B, with or without inhibitors. Data from the inhibitor cohort is expected in the next year.

Pfizer plans to complete the ongoing analyses of full data from the BASIS study. Based on this data, management will start discussions with regulatory authorities with a goal to initiate filing for marstacimab in the near future.

A rare genetic blood disorder, hemophilia is caused by a missing clotting protein (FVIII in case of hemophilia A and FIX in case of hemophilia B), which prevents normal blood clotting. If the blood is not clot properly, it can lead to painful bleeding inside the joints that can cause scarring and damage.

Per Pfizer, marstacimab targets TFPI, which is a natural anticoagulation protein that functions to prevent the formation of blood clots.

Like marstacimab, Novo Nordisk NVO also developed its own anti-TFPI inhibitor, concizumab, for treating hemophilia A and B with inhibitors. Last month, Novo Nordisk received a complete response letter (“CRL”) from the FDA on the regulatory filing for concizumab in hemophilia A or B patients. Per the CRL, the agency requested additional data from Novo Nordisk on the drug’s administration and manufacturing process. Novo Nordisk intends to work closely with the FDA to provide the data requested by the latter.

Other than marstacimab, Pfizer is developing two other investigational gene therapies for hemophilia patients — fidanacogene elaparvovec (for hemophilia B) and giroctocogene fitelparvovec (for hemophilia A) — in separate phase III studies.

Last December, Pfizer reported positive top-line data from a phase III study (BENEGENE-2) which evaluated fidanacogene elaparvovec for the treatment of male adult patients with moderately severe to severe hemophilia B. The study achieved its primary endpoint of non-inferiority and superiority in the ABR post-infusion with the gene therapy.

Several companies have developed their own gene therapies in hemophilia indication.

Last November, the FDA approved Hemgenix, a gene therapy for treating hemophilia B in adults aged 18 years and older. This gene therapy has been developed by uniQure QURE in partnership with CSL Behring, the latter being a subsidiary of CSL Limited. Following approval, uniQure/CSL’s Hemgenix became the first FDA-approved gene therapy for treating hemophilia B. The FDA’s approval of uniQure/CSL’s Hemgenix was based on results from the pivotal HOPE-B study, which demonstrated that following infusion with the gene-therapy, patients produced mean FIX activity of 39% at six months and 36.7% at 24 months. The clinical development program for Hemgenix was initially led by uniQure, which was subsequently transferred to CSL after it acquired global rights to commercialize the treatment.

The newest drug in BioMarin Pharmaceuticals’ BMRN portfolio is Roctavian (valoctocogene roxaparvovec, or valrox), a gene therapy for severe hemophilia A, which received conditional marketing authorization in the European Union in August 2022. In the United States, BioMarin had previously submitted a biologics license application (BLA) in 2019 for valrox to address hemophilia A. However, the FDA issued a CRL to the BLA. BioMarin refiled the BLA in September 2022 and a decision is expected by the end of next month.

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