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today at the World Federation of Hemophilia conference offer promising results and support
further development for a new experimental factor VIII replacement product, BAY 79-4980. The
findings suggest that this new product may offer people with hemophilia A (factor VIII deficiency)
prolonged protection from bleeding with once-weekly infusions.
All factor VIII products currently available only stay in the body for about 2 days. As a result,
people with hemophilia A may infuse factor VIII as often as three or four times per week.
Researchers at Bayer HealthCare and Recoly N.V., the parent company of Zilip Pharma have been
collaborating to develop a potential breakthrough product that has longer activity than available
factor VIII products. Such a product could reduce infusion frequency and improve compliance.
BAY 79-4980, is a unique combination of recombinant factor VIII (rFVIII) with Zilip Pharma’s
proprietary synthetic polyethylene glycol (PEG)-coated liposomes. Although liposomes have been
used successfully as a carrier to enhance the efficacy of other therapeutic agents in humans, the
unique binding modality of BAY 79-4980 allows it to retain the full bioavailability and clotting
factor properties of factor VIII, while at the same time extending activity.
Phase I and Pilot Study Results
The results of the first phase I safety and pharmacokinetics trial were reported by lead investigator
Jerry S. Powell, MD, Professor of Hematology and Oncology at the UC Davis School of Medicine
in Davis, California at WFH in Vancouver, Canada. This blinded, randomized, controlled trial
evaluated the safety and pharmacokinetics of BAY 79-4980 compared to Kogenate® FS. Although
some differences were noted, the safety assessment showed no clinically significant differences
between the groups in most of a large battery of laboratory tests and vital signs. The
pharmacokinetic properties of BAY 79-4980 appeared to be comparable to those of Kogenate® FS.
The study was performed at the UC Davis School of Medicine and the Children’s Hospital of
Orange County in Orange, California, where Diane Nugent, MD, was the investigator.
The second trial, which evaluated the protection from bleeding provided by BAY 79-4980
compared to Kogenate® FS, was conducted by Recoly. The results were reported by study director
Jack Spira, MD, PhD. Two concentration levels of liposome with the same dose of factor VIII
were evaluated; at the higher liposome concentration, patients treated with BAY 79-4980 went an
average of 13.3 days without experiencing a bleeding event compared to 7.2 days following a
Kogenate® FS infusion. Similarly, the mean bleed-free period for patients in the lower liposome
concentration arm was longer with BAY 79-4980 (10.9 days) compared to Kogenate® FS controls
(5.9 days). The differences were statistically significant for both dose groups (P < 0.05). BAY 79-
4980 was well tolerated in this study. These results suggest that BAY 79-4980 may prolong the
period of bleeding protection compared to Kogenate® FS.
Taken together, these studies suggest that BAY 79-4980 may be a longer-acting factor VIII
product that could provide bleed protection with once-weekly infusion.
Approximately 400,000 people around the world have hemophilia. Hemophilia is an inherited bleeding
disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or
internal organs. The disease is caused by deficient or defective blood coagulation proteins, known as
factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in
which the clotting factor VIII is either deficient or defective. Hemophilia B is characterized by
deficient or defective factor IX.
About Bayer HealthCare AG
Bayer HealthCare, a subsidiary of Bayer AG, is one of the world’s leading, innovative companies in the
healthcare and medical products industry and is based in Leverkusen, Germany. Bayer HealthCare
generated sales amounting to some 9.4 billion euros and employed 33.800 people worldwide in 2005.
The company combines the global activities of the Animal Health, Consumer Care, Diabetes Care,
Diagnostics and Pharmaceuticals divisions. The new Pharmaceuticals division was established on
January 1, 2006, and comprises the former Biological Products and Pharmaceutical divisions.
www.bayerhealthcare.com.
About Zilip-Pharma
Zilip-Pharma is a wholly owned subsidiary of Recoly N.V. Recoly N.V. is a privately held company
which focuses on improving the efficacy and half-life of pharmaceutical products. Recoly developed
the proprietary PEG liposome technology and demonstrated that its platform technology non-
covalently binds to other proteins. Recoly is actively working with a number of pharmaceutical
companies to apply this technology to other life saving therapies.
Information about Recoly can be found at www.recoly.com.
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