Longer Acting Recombinant Factor IX

Recombinant Factor IX Monomer Formulation Yields Good Results in Patients With Severe Haemophilia B: Presented at Hemophilia 2010

By Thomas R. Collins
From DOC Guide 

BUENOS AIRES — July 13, 2010 — In the first human trial of rFIX-Fc using monomer technology — a recombinant molecular fusion of coagulation factor IX (FIX) and the Fc region of immunoglobulin G– the treatment was found to perform well in patients with severe haemophilia B and had an encouraging safety profile, researchers said here at the Hemophilia 2010 World Congress.The treatment had a 3-fold increase in half-life and mean residence time compared with an established therapy (BeneFIX) along with a 24% improvement in incremental recovery.

Researchers said the findings are a sign that rFIX-Fc might not have to be treated as often.

“Monomer technology is being applied to FIX for treatment of haemophilia B,” said Amy Shapiro, MD, Indiana Hemophilia & Thrombosis Center, Indianapolis, Indiana, on July 11. “Monomer configuration has demonstrated a range of improvements for a variety of proteins.”

This trial, she said, showed that “activity of rFIX-Fc is preserved over time.”

Fourteen patients were enrolled at 7 haemophilia centres in the United States and Hong Kong. Subjects participated for 60 days, with 13 visits.

The half-life of rFIX-Fc averaged 52.5 hours, compared with historical data from another study that showed the half-life of BeneFIX averaged 19.3 hours.

The mean response time was 68 hours, which was also about 3 times longer than historical data for BeneFIX.

Incremental recovery was also improved with rFIX-Fc – 0.93 IU/dL per IU/kg compared with 0.75 IU/dL per IU/kg.

Six doses were tested in the study, ranging from 1 to 100 IU/kg.

Drug-related adverse events included an abnormal taste in the mouth and headache. There were no serious adverse events linked to the drug, Dr. Shapiro said.

Researchers found that the effects corresponded to dosage, with maximum thrombin concentrations rising evenly with the amount of the treatment administered.

Dr. Shapiro said the results give rise to new possibilities for treatment. “There are implications here both for this specific product as well as for all long-acting products for future development,” she said. “Specifically related to the recombinant IX-Fc protein, this data supports administration at less frequent intervals, perhaps once weekly, or at higher doses with even longer intervals, based upon specific patients.”

For all longer-acting products, she said, “Fewer injections, especially in a younger patient, lead to a decreased need for placement of a venous access device.”

Funding for this study was provided by Biogen Idec.

[Presentation title: Safety and Prolonged Biological Activity Following a Single Administration of a Recombinant Molecular Fusion of Native Human Coagulation Factor IX and the Fc Region of Immunoglobulin G (IgG)(rFIXFc) to Subjects With Hemophilia B]

Prophylaxis Vs. Timely On-Demand Therapy: Joint Bleeds

Prophylaxis More Effective at Preventing Joint Bleeds Than Timely, On-Demand Therapy: Presented at Hemophilia 2010

By Thomas R. Collins
Taken from: DOC Guide

BUENOS AIRES — July 13, 2010 — Prophylaxis treatment resulted in half as many bleeding events in children aged younger than 7 years with haemophilia A, compared with those getting timely, on-demand treatment, according to the latest results from a 10-year study out of Italy presented July 12 at the Hemophilia 2010 World Congress.

Prophylaxis also resulted in half as much joint damage, reported Alessandro Gringeri, MD, Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre, Milan, Italy.

In the Evaluation Study on Prophylaxis: A Randomized Italian Trial (ESPRIT) study, 23 children with haemophilia A were randomised to the prophylaxis group and 22 to the on-demand treatment group. Two patients in the prophylaxis group dropped out after randomisation, as did 3 in the on-demand group.

The young patients averaged 49.7 months in the prophylaxis group and 48.8 months in the episodic treatment group.

The on-demand group was treated with a recombinant FVIII concentrate of at least 25 IU/kg within 6 hours from a bleeding occurrence. The prophylaxis group was treated with 25 IU/kg 3 times a week on nonconsecutive days, but dosage could be adjusted if needed.

Breakthrough bleeds were treated like those in the on-demand group.

Patients were followed for an average of 74 months.

Ultimately, in the prophylaxis group, the starting dose was able to maintain FVIII trough levels >1% in only a third of the patients, so the dose had to be increased.

The total average number of bleeding events per patient in the prophylaxis group was 38, averaging .52 events per patient per month. There was an average of 82 bleeding events per patient in the other group, averaging 1 event per patient per month (P < .01).

There were 15 instances of joint bleeds per patient, averaging .2 per patient per month in the prophylaxis group. There were 40 per patient in the on-demand group, for an average of .53 per patient per month (P < .01).

According to radiographic findings, 6 patients suffered joint damage in the prophylaxis group, compared with 14 in the on-demand treatment group (P < .05).

Of the 10 patients in the prophylaxis group who needed port-a-caths, 6 developed infections.

“Use of CVC [central venous catheters] is complicated by a higher risk of infection,” Dr. Gringeri said in his presentation.

Seven of the children in the prophylaxis group had no joint bleeds at all, compared with 2 in the on-demand group.

Five of the 40 patients developed an inhibitor — 3 of the 21 in the prophylaxis group and 2 of the 19 in the on-demand group.

“Prophylaxis can reduce joint damage at the average dose of 30 IU/kg 3 times a week,” Dr. Gringeri said. “Prophylaxis might prevent joint damage also in those patients who start prophylaxis later, but with a lower rate of efficacy.”

Funding for this study was provided by Baxter International Inc.

[Presentation title: A Randomized Clinical Trial on Prophylaxis vs Episodic Treatment in Children With Haemophilia A: The ESPRIT Study. Abstract 07FP06]

Bayer Clinical Data for Long-acting Recombinant Factor VIIa Molecule

Bayer HealthCare Presents Phase I Clinical Data for BAY 86-6150, a Novel Long-acting Recombinant Factor VIIa Molecule

WAYNE, N.J., July 8 /PRNewswire/ — Phase I clinical data for BAY86-6150, a novel recombinant factor VIIa protein (rFVIIa) being developed by Bayer HealthCare, will be presented at the XXIX International Congress of the World Federation of Hemophilia (WFH) to be held in Buenos Aires, Argentina from Saturday, July 10-Wednesday, July 14.(1)

Data to be presented are from a phase I, randomized, double-blind, placebo-controlled, single-dose escalation study of the rFVIIa variant (BAY 86-6150) in hemophilia A or B with or without inhibitors (Abstract #07P14). This study sought to evaluate the safety, tolerability, pharmacodynamic and pharmacokinetic profiles and immunogenicity of BAY 86-6150 in non-bleeding subjects with hemophilia A or hemophilia B.

Bayer is committed to expanding scientific and clinical knowledge that improves patient care. At the Congress, Bayer will be presenting data from other globally sponsored studies and country-sponsored programs as well as hosting two satellite symposia.

About Bayer HealthCare Pharmaceuticals Inc. Bayer HealthCare Pharmaceuticals Inc. in the U.S.-based pharmaceuticals operation of Bayer HealthCare, an affiliate of Bayer AG. One of the world’s leading, innovative companies in the healthcare and medical products industry, Bayer HealthCare combines the global activities of the Animal Health, Consumer Care, Diabetes Care, and Pharmaceuticals divisions. In the United States, Bayer HealthCare Pharmaceuticals comprises the following business units: Women’s Healthcare, Diagnostic Imaging, General Medicine, Hematology/Neurology, and Oncology. The company’s aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases.

This news 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 http://www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

BAYER and the Bayer Cross are registered trademarks of Bayer.(1) Mahlangu J, Coetzee M, Laffan M, Windyga J, Yee T, Schroeder J. Phase I, randomized, double-blind, placebocontrolled, single-dose escalation study of the rFVIIa variant (BAY86-6150) in haemophilia A or B with or without inhibitors. Haemophilia 2010;16(Suppl. 4): Abstract 07P14. Presented at the Sunday and Monday poster sessions.

SOURCE Bayer HealthCare

Source: PR Newswire

Young Women Urged to Recognize Symptoms of Widely Undiagnosed Bleeding Disorder

AUSTIN, Texas, May 13 /PRNewswire/ — To promote the importance of screening young women for von Willebrand disease (VWD), the American Academy of Nurse Practitioners (AANP) today announced the launch of its VWD Young Women’s Education Campaign. This educational initiative is designed to inform teenage girls and their healthcare providers about the five signs and symptoms of VWD, the most common hereditary bleeding disorder.

VWD is caused by a lack of working von Willebrand factor (VWF), a protein in the blood that is necessary for clotting. The Centers for Disease Control and Prevention (CDC) estimates that VWD affects one to two percent or three to six million people in the U.S. alone. While the disorder affects men and women equally, women are at a greater risk for miscarriage, life-threatening bleeding following childbirth, and for undergoing unnecessary hysterectomies.

“Women struggle with the consequences of this disorder for an average of 16 years before they receive a proper diagnosis because sometimes healthcare professionals interpret VWD symptoms as gynecologic, when in fact the underlying cause is hematologic,” said Josie Weiss, PhD, FNP-BC, Associate Professor, Christine E. Lynn College of Nursing at Florida Atlantic University and Fellow of the American Academy of Nurse Practitioners. “The teenage years are both the best time and provide the best opportunity to recognize von Willebrand disease because the onset of menstruation often reveals a commonly overlooked symptom — heavy and prolonged bleeding.”

The AANP urges young women to visit their healthcare provider if they are experiencing any of these five signs and symptoms of VWD:

• Easy bruising
• Frequent or prolonged nosebleeds
• Heavy, prolonged menstruation
• Prolonged bleeding following injury, childbirth or surgery
• Prolonged bleeding during dental procedures

AANP’s VWD Young Women’s Education Campaign includes educational materials developed for both healthcare professionals and patients. These materials include VWD fact sheets, a screening questionnaire and links to hemophilia treatment centers (HTCs) that specialize in treating bleeding disorders. The virtual toolkit can be found in the AANP Toolkits section of the organization’s website.

This campaign is supported through an educational grant from CSL Behring, a leader in the plasma protein therapeutics industry. CSL Behring is committed to the bleeding disorders community through outreach, education, research and support of a wide variety of activities and programs including Von U, its VWD community of care.

More information about VWD and links to HTCs can be found at www.AANP.org or www.allaboutbleeding-US.com

About von Willebrand Disease (VWD)

Von Willebrand disease (VWD) is caused by a deficiency or abnormality of von Willebrand factor (VWF), a protein in the blood that is necessary for normal blood clotting. Men and women are equally likely to be affected by VWD. VWD is classified by types, ranging from Type 1 (the most common and mild) to Type 3 (the most severe).

Women with VWD are more likely to experience heavy, prolonged menstruation. Other common symptoms of VWD include frequent nosebleeds and easy bruising. Bleeding can be mild or serious and can occur as a result of injury, or without any obvious cause. More serious symptoms include bleeding into joints and internal organs. The VWD patient may require special care during dental procedures, surgery and childbirth.

Treatments for VWD may include desmopressin acetate to release stored von Willebrand factor, von Willebrand factor replacement therapies to raise von Willebrand factor in the bloodstream and oral contraceptives to reduce menstrual bleeding.

About the American Academy of Nurse Practitioners

Created in 1985 to provide nurse practitioners with a unified way to network and to advocate for nurse practitioner issues across all specialties at the local, state, and national levels, the American Academy of Nurse Practitioners (AANP) has continually served as a major resource for NPs, their patients and other healthcare consumers, to promote excellence in practice, education and research; to provide legislative leadership to advance health policy; to establish healthcare standards and to advocate for access to high-quality healthcare. AANP is the oldest, largest and only full-service national professional membership organization for NPs of all specialties, representing the interests of the 135,000 NPs practicing in the United States today. For more information about AANP, visit www.aanp.org.

About CSL Behring

CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufacturers and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in newborns. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (ASX: CSL), a biopharmaceutical company headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.

CONTACT: Elaine Andrecovich MCS Healthcare Public Relations on behalf of AANP (800) 477-9626

SOURCE American Academy of Nurse Practitioners

Source: PR Newswire

Mobile Application to Help Manage Hemophilia Treatment

WAYNE, N.J., June 28 /PRNewswire/ — Bayer HealthCare Pharmaceuticals today launched FactorTrack™, the first customizable mobile application for people with hemophilia A.  FactorTrack is a free, personal and interactive mobile application that helps make it easier to track and record hemophilia factor VIII infusions.  

FactorTrack captures dosing history, frequency and locations of bleeds.  It allows people with hemophilia A to customize their infusion schedule based on their prescribed regimen, view their infusion history and, with an Internet connection, display an alert when it’s time for the next infusion.  It also gives patients the option of emailing infusion and bleed history to themselves or their healthcare team if email is configured on their device.  Further, FactorTrack links people with hemophilia A to educational tools and online resources.  

Any person who treats his or her bleeds with factor VIII infusions can use FactorTrack, regardless of the specific product or therapy.  For those on a prophylaxis (preventive) regimen, the application offers a reminder system to help make it easier to remember when to infuse.  For those who infuse factor VIII on demand (when needed), the application serves as a diary to record bleeds and infusions.  

“FactorTrack is the latest innovation from Bayer that illustrates our ongoing commitment to helping enhance the lives of people who live with bleeding disorders,” said Paul Bedard, vice president and general manager, Hematology, Bayer HealthCare Pharmaceuticals Inc.  “We worked alongside the hemophilia community, physical therapists and physical educators to develop the application in response to the needs of people with hemophilia A.”

Designed to be compatible with a number of smart phone platforms, FactorTrack is available today on an iPhone, iPod touch or iPad.  People can get information about downloading FactorTrack by going to LivingWithHemophilia.com/FactorTrack.  In the near future, FactorTrack will be made available on other smart phones and the Web.

Bayer doesn’t collect personal information when people use the FactorTrack application.  People can use the application without entering information that identifies them.  Recorded data are completely private and will only be stored on a personal iPhone or iPod touch.

About Bayer HealthCare Pharmaceuticals Inc.

Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals operation of Bayer HealthCare, an affiliate of Bayer AG.  One of the world’s leading, innovative companies in the healthcare and medical products industry, Bayer HealthCare combines the global activities of the Animal Health, Consumer Care, Diabetes Care, and Pharmaceuticals divisions. In the United States, Bayer HealthCare Pharmaceuticals comprises the following business units: Women’s Healthcare, Diagnostic Imaging, General Medicine, Hematology/Neurology, and Oncology.  The company’s aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases.

This news 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.

BAYER and the Bayer Cross are registered trademarks and FactorTrack is a trademark of Bayer.

iPhone and iPod touch are registered trademarks and iPad is a trademark of Apple.

SOURCE Bayer HealthCare Pharmaceuticals Inc.