News Feed Item

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.

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
SYS-CON Events announced today that Impiger Technologies will exhibit in Booth #109 at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. Impiger Technologies is a world-class, enterprise software product engineering company specializing in Mobile Application Development, Cloud Applications, Microsoft Technology Solutions, Web Technology and Telecom Services. Impiger Technologies helps enterprises improve busi...
Intelligent machines are here. Robots, self-driving cars, drones, bots and many IoT devices are becoming smarter with Machine Learning. In her session at @ThingsExpo, Sudha Jamthe, CEO of IoTDisruptions.com, will discuss the next wave of business disruption at the junction of IoT and AI, impacting many industries and set to change our lives, work and world as we know it.
SYS-CON Events announced today that Enzu will exhibit at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. Enzu’s mission is to be the leading provider of enterprise cloud solutions worldwide. Enzu enables online businesses to use its IT infrastructure to their competitive advantage. By offering a suite of proven hosting and management services, Enzu wants companies to focus on the core of their online busine...
In the 21st century, security on the Internet has become one of the most important issues. We hear more and more about cyber-attacks on the websites of large corporations, banks and even small businesses. When online we’re concerned not only for our own safety but also our privacy. We have to know that hackers usually start their preparation by investigating the private information of admins – the habits, interests, visited websites and so on. On the other hand, our own security is in danger bec...
The explosion of new web/cloud/IoT-based applications and the data they generate are transforming our world right before our eyes. In this rush to adopt these new technologies, organizations are often ignoring fundamental questions concerning who owns the data and failing to ask for permission to conduct invasive surveillance of their customers. Organizations that are not transparent about how their systems gather data telemetry without offering shared data ownership risk product rejection, regu...
The Open Connectivity Foundation (OCF), sponsor of the IoTivity open source project, and AllSeen Alliance, which provides the AllJoyn® open source IoT framework, today announced that the two organizations’ boards have approved a merger under the OCF name and bylaws. This merger will advance interoperability between connected devices from both groups, enabling the full operating potential of IoT and representing a significant step towards a connected ecosystem.
Kubernetes, Docker and containers are changing the world, and how companies are deploying their software and running their infrastructure. With the shift in how applications are built and deployed, new challenges must be solved. In his session at @DevOpsSummit at19th Cloud Expo, Sebastian Scheele, co-founder of Loodse, will discuss the implications of containerized applications/infrastructures and their impact on the enterprise. In a real world example based on Kubernetes, he will show how to ...
November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. Penta Security is a leading vendor for data security solutions, including its encryption solution, D’Amo. By using FPE technology, D’Amo allows for the implementation of encryption technology to sensitive data fields without modification to schema in the database environment. With businesses having their data become increasingly more complicated in their mission-critical applications (such as ERP, CRM, HRM), continued ...
Enterprises have been using both Big Data and virtualization for years. Until recently, however, most enterprises have not combined the two. Big Data's demands for higher levels of performance, the ability to control quality-of-service (QoS), and the ability to adhere to SLAs have kept it on bare metal, apart from the modern data center cloud. With recent technology innovations, we've seen the advantages of bare metal erode to such a degree that the enhanced flexibility and reduced costs that cl...
SYS-CON Events announced today that Embotics, the cloud automation company, will exhibit at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. Embotics is the cloud automation company for IT organizations and service providers that need to improve provisioning or enable self-service capabilities. With a relentless focus on delivering a premier user experience and unmatched customer support, Embotics is the fas...
SYS-CON Events announced today that Cloudbric, a leading website security provider, will exhibit at the 19th International Cloud Expo, which will take place on November 1–3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. Cloudbric is an elite full service website protection solution specifically designed for IT novices, entrepreneurs, and small and medium businesses. First launched in 2015, Cloudbric is based on the enterprise level Web Application Firewall by Penta Security Sys...
Smart Cities are here to stay, but for their promise to be delivered, the data they produce must not be put in new siloes. In his session at @ThingsExpo, Mathias Herberts, Co-founder and CTO of Cityzen Data, will deep dive into best practices that will ensure a successful smart city journey.
Governments around the world are adopting Safe Harbor privacy provisions to protect customer data from leaving sovereign territories. Increasingly, global companies are required to create new instances of their server clusters in multiple countries to keep abreast of these new Safe Harbor laws. Is it worth it? In his session at 19th Cloud Expo, Adam Rogers, Managing Director of Anexia, Inc., will discuss how to keep your data legal and still stay in business.
In his session at 19th Cloud Expo, Nick Son, Vice President of Cyber Risk & Public Sector at Coalfire, will discuss the latest information on the FedRAMP Program. Topics will cover: FedRAMP Readiness Assessment Report (RAR). This new process is designed to streamline and accelerate the FedRAMP process from the traditional timeline by initially focusing on technical capability instead of documentation preparedness. FedRAMP for High-impact level systems. Early in 2016 FedRAMP officially publishe...
Qosmos, the market leader for IP traffic classification and network intelligence technology, has announced that it will launch the Launch L7 Viewer at CloudExpo | @ThingsExpo Silicon Valley, being held November 1 – 3, 2016, at the Santa Clara Convention Center in Santa Clara, CA. The L7 Viewer is a traffic analysis tool that provides complete visibility of all network traffic that crosses a virtualized infrastructure, up to Layer 7. It facilitates and accelerates common IT tasks such as VM migra...