Pfizer has reported positive topline outcomes from the multicentre, open-label Phase III BASIS trial of Hympavzi (marstacimab) for adults and adolescents with haemophilia A or B with inhibitors.
The primary endpoint and key secondary bleeding endpoints were met, showing that the subcutaneous (SC) therapy improved key bleeding outcomes, compared to on-demand treatment.
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The trial aims to assess the therapy’s safety and efficacy in participants aged 12 to less than 75 years old with severe haemophilia A or moderately severe to severe haemophilia B, with or without inhibitors.
During the active treatment period (ATP), subjects were given a 300mg SC loading dose of the therapy, followed by 150mg subcutaneously once a week, with a potential dose escalation to 300mg once a week.
The trial revealed that prophylactic treatment with the therapy led to a statistically significant decrease in annualised bleeding rate (ABR) of treated bleeds in individuals with severe haemophilia A or haemophilia B with inhibitors.
Over 12 months, 48 subjects received Hympavzi, showing a 93% decrease in ABR, compared to an on-demand intravenous regimen with bypassing agents used in the six-month lead-in duration.
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By GlobalDataDiscovered by the company’s scientists, the therapy’s superiority was observed across all bleeding-related secondary endpoints, including joint bleeds, target joint bleeds, spontaneous bleeds, and total bleeds.
The treatment was found to be generally well-tolerated, without any mortalities or thromboembolic events reported. These results are in line with the Phase I/II outcomes from the non-inhibitor cohort of the BASIS trial.
Pfizer chief inflammation and immunology officer Michael Vincent said: “Hympavzi represents Pfizer’s latest contribution in more than 40 years of working to advance haemophilia care, as a generally well-tolerated treatment option that could offer bleed protection with a straightforward, once-weekly subcutaneous administration in a pre-filled pen for patients with inhibitors, if approved in this patient population.”
In addition, Pfizer is carrying out the open-label BASIS KIDS trial, examining the therapy’s efficacy and safety in paediatric patients aged between one and 18 years old with severe haemophilia A or moderately severe to severe haemophilia B with or without inhibitors.
Last month, Pfizer’s Braftovi (encorafenib) combination therapy demonstrated a reduction in the risk of mortality for metastatic colorectal cancer patients with the BRAF V600E mutation, when compared to standard of care.
