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PFE's Hympavzi Phase III Hemophilia Study With Inhibitors Meets Goal
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Key Takeaways
PFE's Hympavzi met primary and key secondary endpoints in the BASIS study for hemophilia with inhibitors.
Hympavzi delivered a 93% reduction in annualized bleeding rates versus on-demand treatment over 12 months.
Hympavzi was superior across all bleeding-related measures, including spontaneous and joint bleeds.
Pfizer (PFE - Free Report) announced that the phase III BASIS study evaluating its anti-TFPI inhibitor Hympavzi (marstacimab) for treating patients with hemophilia A or B with inhibitors met its primary endpoint as well as key secondary endpoints.
Hympavzi is currently approved as a prophylactic treatment to prevent or reduce the frequency of bleeding episodes in adults and adolescents with hemophilia A or B without inhibitors.
The pivotal BASIS study evaluated the safety and efficacy of Hympavzi, administered once-weekly as a subcutaneous injection, in adolescent and adult participants living with hemophilia A or B with inhibitors. Data from the same showed that prophylactic treatment with Hympavzi led to a statistically significant and clinically relevant reduction in annualized bleeding rate (ABR) in people living with severe hemophilia A or hemophilia B with inhibitors.
Treatment with Hympavzi demonstrated a 93% reduction in ABR over 12 months, which was superior to on-demand treatment. Treatment with Hympavzi was also found to be superior across all bleeding-related secondary endpoints, including spontaneous bleeds, joint bleeds, target joint bleeds and total bleeds.
Treatment with Hympavzi was well-tolerated with no deaths or clotting events reported, and consistent with earlier study data and the non-inhibitor cohort of the BASIS study.
Year to date, shares of Pfizer have lost 8.6% compared with the industry’s decline of 2.1%.
Image Source: Zacks Investment Research
PFE's Next Development Plans for Hympavzi
The above-mentioned positive data show that Hympavzi could help people with hemophilia A or B who have inhibitors, offering a much-needed option for those whose bodies block most regular treatments to prevent bleeding. Patients with inhibitors are prone to frequent complications and face a higher burden of the disease.
Analysis of full phase III data from the inhibitor cohort of the BASIS study is ongoing. Pfizer plans to present additional results at an upcoming medical conference. The company plans to engage with regulatory authorities to initiate the approval process for Hympavzi for the treatment of patients with hemophilia and inhibitors.
Hemophilia, a rare genetic blood disorder, 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 does not clot properly, it can lead to painful bleeding inside the joints that can cause scarring and damage. Hympavzi targets tissue factor pathway inhibitor (TFPI), which is a natural anticoagulation protein that functions to prevent the formation of blood clots.
Pfizer is also evaluating the safety and efficacy of Hympavzi in children aged between one to less than 18 years with severe hemophilia A or B with or without inhibitors in the open-label BASIS KIDS study.
Pfizer previously marketed Beqvez/Durveqtix, a one-time gene therapy, approved for the treatment of hemophilia B. However, according to some reports, the company has now stopped the development and commercialization of Beqvez/Durveqtix owing to lower demand.
We note that BioMarin Pharmaceuticals (BMRN - Free Report) is another company that markets a gene therapy treatment for hemophilia.
BMRN’s Roctavian is the first and only one-shot gene therapy approved for treating adults with severe hemophilia A in the United States and Europe. BioMarin’s Roctavian has been designed to help the body make its own FVIII protein, reducing the need for regular treatment.
In the past 60 days, estimates for Exelixis’ earnings per share have increased from $2.31 to $2.64 for 2025. During the same time, earnings per share estimates for 2026 have increased from $2.83 to $3.13. Year to date, shares of EXEL have rallied 29.4%.
EXEL’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 48.60%.
In the past 60 days, estimates for Puma Biotechnology’s earnings per share have increased from 60 cents to 65 cents for 2025. During the same time, earnings per share estimates for 2026 have risen from 46 cents to 51 cents. Year to date, shares of PBYI have gained 8.5%.
PBYI’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 171.43%.
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PFE's Hympavzi Phase III Hemophilia Study With Inhibitors Meets Goal
Key Takeaways
Pfizer (PFE - Free Report) announced that the phase III BASIS study evaluating its anti-TFPI inhibitor Hympavzi (marstacimab) for treating patients with hemophilia A or B with inhibitors met its primary endpoint as well as key secondary endpoints.
Hympavzi is currently approved as a prophylactic treatment to prevent or reduce the frequency of bleeding episodes in adults and adolescents with hemophilia A or B without inhibitors.
The pivotal BASIS study evaluated the safety and efficacy of Hympavzi, administered once-weekly as a subcutaneous injection, in adolescent and adult participants living with hemophilia A or B with inhibitors. Data from the same showed that prophylactic treatment with Hympavzi led to a statistically significant and clinically relevant reduction in annualized bleeding rate (ABR) in people living with severe hemophilia A or hemophilia B with inhibitors.
Treatment with Hympavzi demonstrated a 93% reduction in ABR over 12 months, which was superior to on-demand treatment. Treatment with Hympavzi was also found to be superior across all bleeding-related secondary endpoints, including spontaneous bleeds, joint bleeds, target joint bleeds and total bleeds.
Treatment with Hympavzi was well-tolerated with no deaths or clotting events reported, and consistent with earlier study data and the non-inhibitor cohort of the BASIS study.
Year to date, shares of Pfizer have lost 8.6% compared with the industry’s decline of 2.1%.
Image Source: Zacks Investment Research
PFE's Next Development Plans for Hympavzi
The above-mentioned positive data show that Hympavzi could help people with hemophilia A or B who have inhibitors, offering a much-needed option for those whose bodies block most regular treatments to prevent bleeding. Patients with inhibitors are prone to frequent complications and face a higher burden of the disease.
Analysis of full phase III data from the inhibitor cohort of the BASIS study is ongoing. Pfizer plans to present additional results at an upcoming medical conference. The company plans to engage with regulatory authorities to initiate the approval process for Hympavzi for the treatment of patients with hemophilia and inhibitors.
Hemophilia, a rare genetic blood disorder, 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 does not clot properly, it can lead to painful bleeding inside the joints that can cause scarring and damage. Hympavzi targets tissue factor pathway inhibitor (TFPI), which is a natural anticoagulation protein that functions to prevent the formation of blood clots.
Pfizer is also evaluating the safety and efficacy of Hympavzi in children aged between one to less than 18 years with severe hemophilia A or B with or without inhibitors in the open-label BASIS KIDS study.
Pfizer previously marketed Beqvez/Durveqtix, a one-time gene therapy, approved for the treatment of hemophilia B. However, according to some reports, the company has now stopped the development and commercialization of Beqvez/Durveqtix owing to lower demand.
We note that BioMarin Pharmaceuticals (BMRN - Free Report) is another company that markets a gene therapy treatment for hemophilia.
BMRN’s Roctavian is the first and only one-shot gene therapy approved for treating adults with severe hemophilia A in the United States and Europe. BioMarin’s Roctavian has been designed to help the body make its own FVIII protein, reducing the need for regular treatment.
PFE's Zacks Rank & Stocks to Consider
Pfizer currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Exelixis (EXEL - Free Report) and Puma Biotechnology (PBYI - Free Report) , each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 60 days, estimates for Exelixis’ earnings per share have increased from $2.31 to $2.64 for 2025. During the same time, earnings per share estimates for 2026 have increased from $2.83 to $3.13. Year to date, shares of EXEL have rallied 29.4%.
EXEL’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 48.60%.
In the past 60 days, estimates for Puma Biotechnology’s earnings per share have increased from 60 cents to 65 cents for 2025. During the same time, earnings per share estimates for 2026 have risen from 46 cents to 51 cents. Year to date, shares of PBYI have gained 8.5%.
PBYI’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 171.43%.