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Bayer's Long-Acting Recombinant Factor VIII Demonstrated Prophylaxis with Less Frequent Infusions in Hemophilia A in Phase III Trial

- The PROTECT VIII study met its primary efficacy and safety objectives for the treatment of patients with hemophilia A

WHIPPANY, N.J., Feb. 18, 2014 /PRNewswire/ -- Bayer HealthCare today announced positive results from the PROTECT VIII trial evaluating the company's investigational long-acting site-specific PEGylated recombinant human factor VIII compound BAY 94-9027. The study met its primary objective of protection from bleeds with fewer infusions. In the study, the site-specific PEGylated factor VIII helped protect against 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 resolving with one or two infusions.

"These results are very encouraging," said Jerry Powell, MD and Director of the Hemophilia Treatment Center at the University of California Davis. "We have found that this investigational site-specific PEGylated factor VIII may help protect patients from bleeds associated with hemophilia A, even when used every seven days. The current standard of treatment requires infusion every two to three days."

"These data suggest that Bayer's site-specific PEGylated factor VIII may provide longer-lasting protection from bleeds," said Pamela Cyrus, MD, Vice President and Head, U.S. Medical Affairs, Bayer HealthCare Pharmaceuticals.  "We are hopeful that less frequent infusions will further support patients in adhering to prophylaxis regimens."

The current standard treatment for severe hemophilia A is regularly scheduled prophylactic infusion of factor VIII to keep factor VIII levels high enough to prevent bleeding. Due to the shorter half-life of currently marketed factor VIII products, prophylaxis may require treatment as often as every other day. Bayer's investigational 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, including in the U.S., in the second half of 2015. 

Studies evaluating the safety and efficacy during major surgery and in pediatric patients are ongoing.  Bayer is also planning a study in previously untreated patients.

About the PROTECT VIII study and results

PROTECT VIII (PROphylaxis in hemophilia A patienTs via directly pEgylated long-aCTing rFVIII) is a multicenter, multinational, partially randomized, open-label trial with four treatment arms evaluating the safety and efficacy of the site-specific PEGylated factor VIII in previously treated adults and adolescents with severe hemophilia A. 134 patients were treated in the study. Patients selected either on-demand or prophylactic treatment upon enrollment. All patients in the three prophylaxis arms began treatment with the site-specific PEGylated recombinant factor VIII twice per week. After a ten-week period patients experiencing more than one bleed during this assessment period stayed on two infusions per week at a higher dose and all other patients were randomized to either every five- or seven-day treatment for six months. After randomization, patients who assessed their bleeding control as not adequate could leave the assigned treatment regimen and increase their infusion frequency.

88 percent of patients met the pre-defined criterion of bleeding control in the ten-week initial assessment period and qualified for randomization.  All patients receiving infusion every five days (n=43) remained in this treatment arm.  44 percent of patients in the every five-day treatment arm experienced no bleeds.  A median annualized bleeding rate (ABR) of 1.9 was observed in this treatment arm.  

74 percent of the patients receiving infusion every seven days (n=43) remained in their treatment arm. 37 percent experienced no bleeds. A median ABR of 3.9 (including non-completers) was observed in this treatment arm.  

The 13 patients who remained in the two times per week treatment arm, because of their high bleeding rate during the assessment period, reduced their median ABR from 17.4 to 4.1.  Patients who received on-demand treatment (n=20) had a median ABR of 23.

The safety objectives were also met. Patients were treated for up to 36 weeks; no inhibitors to factor VIII were confirmed.  Two drug-related cases of hypersensitivity reactions were reported. One was assessed as serious, but resolved without medical intervention. No other serious drug-related adverse events were reported.

About Hemophilia A
Hemophilia A, also known as factor VIII deficiency or classic hemophilia, is a largely inherited bleeding disorder in which one of the proteins needed to form blood clots in the body is missing or reduced. Hemophilia A, the most common type of hemophilia, is caused by a deficient or defective blood coagulation protein, known as factor VIII. Hemophilia A is characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs.

About Bayer HealthCare Pharmaceuticals Inc.

Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world's leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Medical Care, and Pharmaceuticals divisions. As a specialty pharmaceutical company, Bayer HealthCare provides products for General Medicine, Hematology, Neurology, Oncology and Women's Healthcare. The company's aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases.

Forward-Looking Statements
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer's public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

Intended for U.S. Media Only

SOURCE Bayer HealthCare Pharmaceuticals Inc.

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