Pharmaceutical giant Pfizer has announced the positive topline results of its Phase III study on Hympavzi.
The BASIS study, which was evaluating the safety and efficacy of the once-weekly subcutaneous haemophilia drug, revealed its potential to improve key bleeding outcomes better than on-demand treatment.
Inhibitors, or antibodies, which neutralise factor replacement therapies and render them ineffective, may develop in people living with haemophilia.
Of the more than 800,000 people in the world living with hemophilia A or hemophilia B, approximately 20% of people with haemophilia A and 3% of people with hemophilia B are unable to continue taking factor replacement therapies because they develop inhibitors to FVIII (Factor VIII) and FIX (Factor IX) and these therapies no longer prevent or stop bleeding episodes.
“Patients with inhibitors tend to face frequent complications, and navigating the treatment landscape can introduce complexities and increase disease burden,” said Davide Matino, BASIS Principal Investigator, Associate Professor of Medicine, McMaster University.
“The strong bleed reduction with Hympavzi compared with on-demand treatment in the Phase III BASIS study, coupled with its weekly administration method, offers exciting potential for these patients who are in critical need of treatment options.”
Through the BASIS trial, Pfizer demonstrated that prophylactic treatment with Hympavzi significantly reduced the annualised bleeding rate of study participants.
Hympavzi was also superior to on-demand treatment, with a 93% reduction in ABR over 12 months (ABR 1.39 vs ABR on-demand 19.78; p < 0.0001). Superiority of HYMPAVZI was also demonstrated across all bleeding-related secondary endpoints—spontaneous bleeds, joint bleeds, target joint bleeds, and total bleeds.
“These encouraging results demonstrate HYMPAVZI’s potential to help people living with hemophilia A or B with inhibitors, meeting an important need for patients with antibodies that neutralise most factor-based prophylactic options used to manage bleeding episodes,” said Michael Vincent, Chief Inflammation & Immunology Officer, Pfizer.
“HYMPAVZI represents Pfizer’s latest contribution in more than 40 years of working to advance hemophilia 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.”
Analyses of the full Phase III dataset from the inhibitor cohort is currently ongoing, and additional data will be presented at upcoming medical meetings.