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Pfizer (PFE) Begins Phase III Study on Hemophilia Candidate

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Pfizer Inc. (PFE - Free Report) announced that it has dosed the first participant in a phase III study, which is evaluating its anti-tissue factor pathway inhibitor (anti-TFPI) marstacimab for the treatment of patients with severe hemophilia A or B with or without inhibitors.

This open-label BASIS study will investigate the annualized bleed rate (ABR) through 12 months on prophylaxis treatment with marstacimab in adolescents and adult patients with hemophilia A or B. In the study, marstacimab will be compared to replacement therapy with essential blood-clotting proteins FVIII or FIX, respectively, or bypass therapy.

The primary endpoint of the study is to see the impact on ABR following prophylaxis treatment with marstacimab through 12 months of treatment. The study will also assess the incidence and severity of thrombotic events or blood clots.

Notably, this investigational, novel subcutaneous therapy marstacimab demonstrated significant reductions in ABR for all participants in a phase II study, thereby eradicating the need for factor replacement therapy.

Shares of Pfizer have lost 6.8% so far this year compared with the industry’s decrease of 0.2%.

price chart for PFE

We remind investors that Pfizer is currently evaluating its investigational gene therapy fidanacogene elaparvovec/PF-06838435 in a phase III study for the treatment of hemophilia B.

Meanwhile, in October 2020, Pfizer along with partner Sangamo Therapeutics, Inc. (SGMO - Free Report) initiated a phase III study to evaluate giroctocogene fitelparvovec/SB-525, an investigational gene therapy in patients with severe hemophilia A.

Hemophilia A is a genetic disorder caused by the missing or defective FVIII. Many companies are developing gene therapy products to treat severe hemophilia A.

BioMarin Pharmaceutical (BMRN - Free Report) has an investigational gene therapy candidate Roctavian (valoctocogene roxaparvovec/valrox) for treating patients with severe hemophilia A. Meanwhile, the Netherlands-based uniQure N.V. (QURE - Free Report) is developing a gene therapy candidate to treat patients with hemophilia B.

Zacks Rank

Pfizer currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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