Winter 07 Factor Nine Newsletter

TOPICS IN HEMOPHILIA

- Factor Nine Family Meeting – Orlando, Florida
- NHF – Coalition Award
- Factor Nine Products in Development
- Coalition for Hemophilia B 2008 Fundraising Event & Symposium

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FACTOR NINE FAMILY MEETING, ORLANDO FLORIDA

We had a wonderful turnout at our Factor Nine Family meeting held in Orlando Florida in conjunction with the National Hemophilia Foundation Conference in November 2007. Over 65 people attended this meeting. We will keep you informed of upcoming meetings in 2008 in our spring edition of Factor Nine News !

 

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NHF AWARD S THE COALITION FOR HEMOPHILIA B

Wayne Cook, Ray Stanhope, David Clark

On Thursday, November 1, 2007, The Coalition for Hemophilia B attended the Celebration of Giving cocktail party hosted by The National Hemophilia Foundation (NHF). Ray Stanhope, Chairman of the Board of NHF presented Wayne Cook and David Clark from The Coalition for Hemophilia B with an award in recognition of our support of the National Hemophilia Foundation in the past year. Areas of support include ongoing scientific research and education.

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FACTOR NINE PRODUCTS IN DEVELOPMENT

After being relatively quiet for a few years, there is currently a flurry of activity by several companies in developing new factor IX products and therapies.

Inspiration Biopharmaceuticals filed an Investigational New Drug (IND) application in November 2007 to begin testing a new recombinant factor IX concentrate in patients. An IND is the document describing all of the laboratory and safety testing of a new drug that the FDA uses to determine whether the drug appears safe enough to test in humans. Inspiration plans to test their product for both on-demand and prophylactic use, as well as for use in surgery.

Inspiration has proprietary manufacturing technology that can produce recombinant proteins more efficiently and with higher yields. They have also been doing research to increase the recovery of recombinant factor IX after infusion. Their goal is to be able to produce factor IX and other factor products at much lower cost to make them available to larger numbers of patients all over the world. Lower cost factor IX also makes it possible to explore non-intravenous delivery methods, which potentially require larger amounts of factor IX.

Baxter Healthcare developed the first factor IX product in the U.S. in 1969 but has been largely out of the factor IX market for several years. Now they have announced that they are developing a recombinant factor IX concentrate and also starting research on a longer-acting factor IX. A longer-acting factor IX would require less frequent infusions and potentially make prophylactic treatment easier. One of the methods Baxter is reportedly exploring is PEGylation, attaching polyethylene glycol (PEG) polymer molecules to the factor IX molecule. Attaching PEG to a protein tends to protect the protein from being broken down and cleared from the bloodstream. PEGylation has already been used successfully to increase the half-life of several other protein drugs.

GTC Biotherapeutics is developing a recombinant factor IX made in the milk of transgenic animals. Currently, recombinant products are made by genetically engineered cells that are grown in large tanks. GTC is genetically engineering cells in the mammary glands of pigs so that they produce factor IX in their milk. The milk is collected and processed to purify the factor IX. Because huge amounts of protein can be made this way, it is another source of potentially low-cost and abundant factor IX. GTC previously developed a transgenic recombinant antithrombin, which was licensed in Europe in 2005; the first transgenic product ever approved anywhere. Antithrombin is an anti-clotting protein. People with antithrombin deficiencies clot too easily.

Nastech Pharmaceutical Company is also looking at the possibility of developing a non-intravenous delivery method for factor IX. Very few details are available, but one of their specialties is nasal delivery of drugs. They are working with one of the major factor IX manufacturers, so far unidentified.

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THE COALITION FOR HEMOPHILIA B
2ND ANNUAL
NEW YORK SYMPOSIUM
SATURDAY, MARCH 8, 2008

MILLENNIUM BROADWAY HOTEL
145 WEST 44TH STREET
NEW YORK, NEW YORK

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THE COALITION FOR HEMOPHILIA B
1ST ANNUAL
FUNDRAISING DINNER
FRIDAY, MARCH 7, 2008

MILLENNUM BROADWAY HOTEL
145 WEST 44TH STREET
NEW YORK, NEW YORK

 

 

 

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For back issues of Factor Nine Newsletter of for more information on research please call or write to:
Kim Phelan, 825 Third Ave., Suite 226, New York, New York 10022, Telephone (212) 520-8272
E-mail: hemob@ix.netcom.com Website: www.coalitionforhemophiliab.org