Baxter completes patient enrollment in phase III trial of BAX 855, extended half-life rFVIII to treat haemophilia A

Baxter International Inc. has completed enrollment in its phase III clinical trial of BAX 855, an investigational extended half-life, recombinant factor VIII (rFVIII) treatment for haemophilia A. The ongoing trial is aimed at assessing the efficacy of the compound in reducing annualized bleed rates (ABR) in both prophylaxis and on-demand treatment schedules, and will also evaluate its safety and pharmacokinetic profile.

BAX 855 was designed based on the full-length ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] molecule, a product with 10 years of real-world experience. The BAX 855 molecule was modified with PEGylation technology designed to extend its duration of activity in the body.

"The BAX 855 development programme is a priority for Baxter as we evaluate the potential to provide an efficacious and safe treatment with an extended half-life for patients with hemophilia," said Anders Ullman, MD, Ph.D., vice president of global research and development in Baxter's BioScience business. "We are focused first and foremost on strategies to address optimal efficacy and minimize patients' bleeding episodes, while at the same time delivering on the convenience of less frequent dosing for this population with severe disease."

The phase II/III multi-centre, open-label study called PROLONG-ATE is evaluating BAX 855 among 146 adult patients with previously-treated severe haemophilia A. Patients participating in PROLONG-ATE receive treatment twice weekly (45 IU/kg) and are followed for six months. The primary endpoint of the study is the annualized bleed rate (ABR) during the treatment period. The study is also evaluating the safety and immunogenicity of the compound when administered on either prophylaxis and on-demand treatment regimens. Other outcome measures include number of infusions needed to treat bleeding episodes, time intervals between these episodes, pharmacokinetics and patient reported outcomes. To date, no inhibitors or safety issues have been reported in the study.

Based upon the results of the study, the company expects to complete the trial and file for regulatory approval late in 2014. Baxter is also initiating a continuation study for all patients who complete the pivotal phase II/III study, and expects to initiate a study of BAX 855 among pediatric patients in 2014.

The treatment protocol is based on the results of a phase I trial of BAX 855, assessing its safety, tolerability and pharmacokinetics. That trial found that the half-life (measuring the duration of activity of the drug in the body) of the investigational compound was approximately 1.5-fold higher compared to ADVATE. An extended half-life was achieved in all patients in the study using BAX 855, no patients developed inhibitors to either the base molecule, BAX 855 or to PEG, and no patients had allergic reactions. No treatment-related or serious adverse events were reported, and no patients withdrew from the study due to adverse events.

BAX 855 is built from the same native FVIII protein used in the production of ADVATE, and employs proprietary PEGylation technology from Nektar Therapeutics designed to extend the duration of activity of proteins. PEGylation technology has been widely used in various approved treatments.

ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] is indicated for the control and prevention of bleeding episodes in adults and children (0-16 years) with haemophilia A. ADVATE is also indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children (0-16 years) with haemophilia A. ADVATE is not indicated for the treatment of von Willebrand disease.

ADVATE has a demonstrated efficacy profile and a low rate of inhibitor development. ADVATE is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives. Because no blood-derived components are added at any stage of the manufacturing process, the potential risk of transmitting pathogens that may be carried in blood-based additives is eliminated. There have been no confirmed reports of transmission of HIV, HBV or HCV with rFVIII treatments.

ADVATE is approved in 60 countries worldwide including the United States, Canada, 27 countries in the European Union, Argentina, Australia, Brazil, Chile, China, Colombia, Croatia, Ecuador, Hong Kong, Iceland, Iraq, Japan, Kuwait, Macau, Malaysia, Mexico, New Zealand, Norway, Panama, Puerto Rico, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.

Baxter (BAX) To Expand Access To Recombinant FVIII Hemophilia Treatment In Brazil

Baxter International Inc. (NYSE: BAX) announced an exclusive 20-year partnership with Hemobrás (Empresa Brasileira de Hemoderivados e Biotechnologia) to expand greater access to recombinant factor VIII (rFVIII) therapy for the treatment of hemophilia A in Brazil.

Hemophilia A is a genetic condition in which the body does not produce enough clotting protein factor VIII. It is estimated that more than 10,000 people in Brazil are living with hemophilia A, and today the vast majority are treated with plasma-derived FVIII therapy.

Recently, Baxter highlighted the importance of developing innovative business models, including public and private partnerships, aimed at improving the quality of and access to care in both developed and emerging markets.

Through this innovative partnership, Baxter will be the exclusive provider of Brazil's recombinant FVIII treatment over the next 10 years while the companies work together on the technology transfer to support development of local manufacturing capacity by Hemobrás.

Baxter will receive cash payments for product it supplies to Hemobrás and, following completion of the technology transfer, royalties on recombinant FVIII produced by Hemobrás. The company expects peak annual sales to exceed $200 million.

''Our unique collaboration with Hemobrás is a demonstration of Baxter's leadership in hemophilia, reflects our expertise and commitment to the community, and positions us as an attractive partner that can make a significant impact on expanding access to quality care to patients around the world,'' said Robert Parkinson Jr., chief executive of Baxter.

China Biologic Receives SFDA Manufacturing Approval Certificate for Human Coagulation Factor VIII

China Biologic Products, Inc., a leading fully integrated plasma-based biopharmaceutical company in China, today announced that its indirectly owned subsidiary, Shandong Taibang Biological Products Co., Ltd. ("Taibang") has received a manufacturing approval certificate from the China State Food and Drug Administration ("SFDA") for Human Coagulation Factor VIII ("FVIII"). With this certificate, the only approval remaining for Taibang's commercial production of FVIII is the SFDA's good manufacturing practice ("GMP") certification of the FVIII production line itself.

Taibang began research for FVIII in 2007 and successfully developed the technology in 2008. The Company conducted clinical trials from 2009 to 2010. In June 2010, the Company submitted required materials to Center for Drug Evaluation for approval to start manufacturing and passed on-site products verification in January 2011. The Company received official manufacturing approval certificate on June 21, 2012. FVIII will be primarily used in the treatment of hemophilia A.

Mr. David Gao, Chairman & CEO, said, "Receiving SFDA manufacturing approval represents the culmination of more than five years of work and the first technological approval of a coagulation factor product developed by Taibang. With the addition of Factor VIII, we should soon be able to offer three major categories of blood products: albumin products, immunoglobulin products and coagulation factor products. This will further strengthen our competitive position as a leading plasma-based biopharmaceutical company in China."

"We are very proud to bring FVIII to hemophilia A patients in China," continued Mr. Gao. "We believe that Human Coagulation Factor VIII addresses a critically unmet need of a large patient population and helps ease under-supply of coagulation products. Although it is not possible to specify a definitive timeframe in which we will receive GMP approval, we expect our FVIII production to be inspected by SFDA and then GMP certified by the end of 2012. Going forward, we intend to continue to develop and manufacture life-enhancing, effective, quality-assured products."

About China Biologic Products, Inc.

China Biologic Products, Inc., is a leading fully integrated plasma-based biopharmaceutical company in China. The Company's products are used as critical therapies during medical emergencies and for the prevention and treatment of life-threatening diseases and immune-deficiency related diseases. China Biologic is headquartered in Beijing and manufactures over 20 plasma-based products through its indirect majority-owned subsidiaries, Shandong Taibang Biological Products Co., Ltd. and Guiyang Dalin Biologic Technologies Co., Ltd. The Company also has an equity investment in Xi'an Huitian Blood Products Co., Ltd. The Company sells its products to hospitals and other healthcare facilities in China. For additional information, please see the Company's website www.chinabiologic.com .

CSL Behring Marks World Hemophilia Day with Coagulation Factor Donation to World Federation of Hemophilia

KING OF PRUSSIA, Pa. and MONTREAL, Feb. 16, 2011 /CNW/ -- As a means of aiding the World Federation of Hemophilia's (WFH) progress in improving the diagnosis and treatment of hemophilia in developing countries through its Global Alliance for Progress (GAP) program, CSL Behring has committed to donating more factor VIII concentrate (FVIII) to WFH. The donated product will be used in South America, Thailand, thePhilippines, parts of eastern Europe, parts of the Middle East, and in Tunisia. The donation, the total market value of which is approximately $500,000, will be made using coagulation factor concentrate with a minimum shelf-life of one year.
CSL Behring has pledged to support the WFH with separate financial contributions totaling nearly $1 million over a period of three years. This most recent donation is part of that commitment.
"As a long-standing contributor to GAP, CSL Behring is pleased to once again be in a position to assist hemophilia patients who have a critical need for safe, effective medical therapies," said Peter Turner, President and Chief Executive Officer of CSL Behring. "We are also gratified to be a partner with WFH in this effort, an organization that continues to make a large and positive difference in the world. Our commitment to their progress remains strong."
"The WFH relies on product donations to help introduce clotting factor concentrates in developing countries and these donations serve as a catalyst for improving care," said Claudia Black, Chief Executive Officer of the WFH. "We are grateful to our industry partners such as CSL Behring, who provide, among other resources, valuable and life-saving medicine, including this most recent donation of coagulation factor. "
Factor VIII is an essential blood clotting factor that can be given to hemophiliacs to restore hemostasis.

CSL Behring Marks World Hemophilia Day with $2M Coagulation Factor Donation to World Federation of Hemophilia

In recognition of the World Federation of Hemophilia’s (WFH) progress in improving the diagnosis and treatment of hemophilia in developing countries through its Global Alliance for Progress (GAP) program,CSL Behring has committed to donating to WFH two (2) million units of factor VIII concentrate (FVIII) each year for the next three (3) years. The donation, the total value of which will be approximately $2 million, will be made using coagulation factor concentrate with a minimum shelf-life of one year. CSL Behring has also renewed its pledge to continue supporting the WFH with separate financial contributions totaling nearly $1 million over a period of three years. “As a long-standing contributor to GAP, CSL Behring applauds WFH’s critically important efforts in bringing about improvements and change in the delivery of healthcare services and medicines to hemophilia patients in areas of the world where the need is greatest,” said Peter Turner, President and Chief Executive Officer of CSL Behring. “Serving patients with rare bleeding disorders is a core focus for CSL Behring, as well, and we are extremely pleased to offer our support to GAP, a program that is truly making a large and positive difference.”

Irish Create Helpline Over Mad Cow fears

IRELAND - THE DISCOVERY that a haemophiliac who died in Britain last year had evidence of variant CJD or vCJD infection has led to just over 30 people ringing a helpline specifically set up to respond to the issue. The helpline was aimed at people who had received UK-derived plasma products or had concerns about blood products they received. Two information meetings were held in Dublin and Cork at the weekend to further allay concerns and were attended by a handful of people. It emerged two weeks ago that the elderly man, who did not die from vCJD, was infected from a UK plasma-derived product known as Factor VIII. Haemophilia and related bleeding disorders are caused by a lack of clotting factors and are treated by products containing clotting factor concentrate such as Factor VIII. Since the late 1990s, Ireland has been using a synthetic clotting concentrate. On hearing the details of the man’s death, the National Haemophilia director Dr Barry White and the Irish Haemophilia Society’s Brian O’Mahony wrote to the 50 Irish patients who had received UK plasma-derived products to inform them of developments. Only two of those had received a product that was subsequently identified as being at risk of vCJD. Thousands of people in the UK have received products where some of the donors developed vCJD, but none of the recipients have developed symptoms. There have been 167 cases of vCJD in the UK and four in Ireland since the link between eating infected meat and a new variant of CJD was made in the mid-1990s. Some nine people who died of vCJD in the UK had donated blood for the manufacture of clotting factor concentrates.

Researchers Use New Method to Control Bleeding in Hemophilia

MILWAUKEE, Nov 13, 2008 /PRNewswire via COMTEX/ -- Investigators at Children's Research Institute, BloodCenter of Wisconsin's Blood Research Institute and the Medical College of Wisconsin have discovered a new way to help the blood clot by having the missing clotting factor packaged in the patient's own platelets. In the October 2008 edition of Blood, investigators describe how a gene-modified bone marrow transplant can be used to initiate clotting in hemophilia. This type of approach may work in the 30 to 35 percent of hemophilia patients that have developed inhibitory antibodies against the missing clotting protein.
The bone marrow is removed from the patient and stem cells are treated with Factor VIII, a clotting factor, which is placed in the platelets. The marrow is given back to the patient, who then retains the essential clotting mechanisms to stop bleeding that otherwise would lead to complications.
For people suffering from hemophilia, this research means the potential relief of a constant, burdening disease. People who have hemophilia previously had to be treated every time they bled. Currently, they can receive treatments three times a week, but these are very costly and time consuming. The results from this study provide hope that people with hemophilia could potentially lead a disease-free life.
The scientific community once believed that hemophilia would be treated successfully by gene therapy. Research then showed that gene therapy typically resulted in the patient not retaining a substantial amount of clotting factor, which is integral in preventing serious bleeding.
This research was a collaborative effort that included investigators Qizen Shi, PhD, MD; David A. Wilcox, PhD; and Robert Montgomery, MD.