Positive Ph III results with Bayer's long-acting recombinant factor VIII product BAY94-9027

Germany’s Bayer (BAYN: DE) has released positive results from the PROTECT VIII trial evaluating the company’s investigational site-specific PEGylated recombinant human factor VIII compound BAY94-9027.

The study met its primary objective of protection from bleeds with fewer infusions. In the study, the site-specific PEGylated factor VIII provided protection from bleeds when used prophylactically every seven days, every five days, and twice per week. The compound was also effective for treatment of acute and breakthrough bleeds with 91 percent of events resolved with one or two infusions.

“These results are very encouraging,” said Jerry Powell, director of the Hemophilia Treatment Center at the University of California Davis, USA, adding: “We have found that this investigational site-specific PEGylated factor VIII could protect users from bleeds associated with hemophilia A, even when used every seven days. This is a significant advantage over the current standard of care, which requires infusion every two to three days.”

“We are pleased that Bayer’s site-specific PEGylated factor VIII has been shown to provide longer-lasting protection from bleeds. Reducing the significant burden associated with frequent infusions has long been a key goal of the entire hemophilia community,” said Joerg Moeller, a member of the Bayer HealthCare executive committee and head of global development. “This compound may lead to improvements in long-term outcomes and quality of life for people with hemophilia A,” he noted.

Extends circulating half life

The current standard treatment for severe hemophilia A is regularly scheduled prophylactic infusion of factor VIII to keep levels high enough to prevent bleeding. Due to the short half-life of currently marketed factor VIII products, prophylaxis may require treatment as often as every other day. Bayer’s compound is engineered to extend the circulating half-life while preserving full biologic activity through site-specific pegylation. This site-specific pegylation is achieved by inserting a single cysteine (amino acid) on the factor VIII surface, which serves as an attachment site for a polyethylene glycol (PEG) polymer.

Detailed data are scheduled for presentation at the World Federation of Hemophilia Meeting in May 2014 in Melbourne, Australia. Bayer plans to submit marketing authorization applications to regulatory authorities in the USA, Europe and other countries in the second half of 2015. Evaluation of safety and efficacy during major surgery and PROTECT Kids in pediatric patients are ongoing, and a study in previously untreated patients is planned.