Winter 2006 - Factor Nine News

TOPICS IN HEMOPHILIA

SPECIAL EDITION

-A Letter from John R. Taylor, Jr.
-Industry Update
-Coalition 2006 Meeting Pictures
- Future Meeting Information

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A Letter from John R. Taylor, Jr.

Dear all members,

It is with a mixture of sadness and elation that I write this letter to you all. My wife Joyce and I had the idea to start the Coalition several months after our son John was born. We wanted to provide information and support for parents like us, men, women and children with hemophilia and their families. Although filled with much anxiety, we realized the more we learned, the stronger we became to deal with the problems we faced. We set out to empower others so they would not have to deal with the same issues we faced, alone. Thus the birth of The Coalition for Hemophilia B in 1990.

While serving as Chairman for the past 16 years, we have seen many changes in the treatment and the lives of those who have been able to enjoy the progress that science has made. First with the introduction of Alphanine, which was the first product just for us, and then Mononine, the first monoclonal factor, we began to get the feeling of what life could be. The Coalition was very happy to have played a part in the FDA approval of Mononine, but we pushed hard for BeneFIX as soon as Mononine hit the market. When BeneFIX was approved in 1996, we all thought this was great, but then immediately began wondering how we could make things better again. Everyone involved in the Coalition has always desired to go the next mile, take the next step up the ladder, and eventually reach some unknown goal.

With that in mind, the Coalition has pushed prophylaxis before it was common and before MASAC said that this was the approved treatment. But we worried about the cost of this treatment. This pushed us into research, and the Coalition championed transgenic research to make factor much cheaper. It also spent money to support the search for alternate delivery methods“ pills, nasal sprays, skin patches and anything else that would eliminate the dreaded i.v. needle.

Anyone who has spoken with me knows that I think that more should be done, and after many years of talking about improving products and delivering them by something better than i.v., I have taken a big step. I have started a company, Inspiration Biopharmaceuticals, Inc., with a partner and fellow Coalition member, Scott Martin from Houston, Texas. Inspiration’s first product will be a recombinant Factor IX and we hope the second product will be an orally delivered Factor IX. Our goal is to revolutionize the treatment of hemophilia, and it will take a lot of work to reach that goal but we are on our way.

Because of my new role, I can no longer be associated with the Coalition. Upon my resignation I have passed the baton to two individuals who express the same desires and hopes that the Coalition values so highly: Dr. David Clark as Chairman. Dave has been writing the scientific end of our newsletter for many years.
He has a strong interest in hemophilia B. And Wayne Cook, who has hemophilia B and has been a long-term advocate for the cause. The Coalition has moved its offices and is now located at 825 Third Avenue here in Manhattan.

All of the Coalition’s functions, advocating for new research, serving as a resource for patients and their families, and spreading new treatment ideas, will remain as they were.

This is a very exciting time for our community as the efforts of the Coalition have led not only to my new company Inspiration, but also to new product developments at Wyeth and a new entrant in our market“ Biogen. Whatever the outcome, I am sure that our community will be better served in the years ahead and that’s the whole idea.

My experience with the Coalition has been one of the highlights of my life. It certainly helped us. It has been a pleasure serving you all.

Warm regards,

John R. Taylor, Jr.

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Industry Update

Grifols’ Alphanate approved by FDA

Grifols, a plasma fractionation company in Los Angeles, produces Alphanate, a plasma-derived factor VIII clotting factor concentrate that also contains significant amounts of von Willebrand factor (vWF) protein. Physicians know this and have used Alphanate off-label for years to treat Von Willebrand disease (vWD). However, Grifols has recently completed a clinical study of 76 patients, including children that allowed the FDA to formally license Alphanate for treatment of vWD. vWD is a disease that has a lot of similarities to hemophilia A and B. vWF is a protein that protects factor VIII and carries it around in the bloodstream. FDA approval of an indication for treatment of vWD allows Grifols to market Alphanate for treatment of vWD, and it is sometimes important in getting insurance companies to pay for the treatment. An indication is an approved use for a drug. Therefore, this potentially is a significant development for vWD patients. ~

Biogen Idec has announced that it is acquiring Syntonix Pharmaceuticals, a biotechnology company based in Waltham, Massachusetts. Syntonix has been developing a long-acting Factor IX product for the treatment of Hemophilia B. Their SynFusion technology links a factor IX molecule to an antibody molecule. Antibodies last longer in the bloodstream than may other proteins, so this helps the factor IX stay in the bloodstream longer. Antibodies also can move across cell barriers such as those in the lungs. Thus the new product may be able to be delivered by inhalation rather than injection.

Wyeth Pharmaceuticals and Nautilus Biotech, a French company, have agreed to work together to develop a recombinant factor IX that has a longer half-life. The half-life is the amount of time it takes for half of the factor IX to disappear from the bloodstream. The longer the half-life, the less frequently an infusion is needed. This is especially important for prophylactic treatment where it could lengthen the time between infusions.

Nautilus has proprietary technology that can make changes in the amino acid sequence of a recombinant protein like factor IX. Amino acids are small molecules that are strung together into long chains to create proteins. There are 21 common amino acids that the human body uses to make all of its proteins. The type and order in which the amino acids are strung together determine which protein is produced.

Small changes in the type of amino acid at a specific place in the protein molecule, which might not significantly affect the activity of the protein, can make a big difference in how long a protein lasts in the bloodstream.

Wyeth and Nautilus will look at various amino acid changes to see if a recombinant factor IX molecule can be produced that has a longer half-life without sacrificing activity.
Wyeth has also signed a research agreement to work with MediVas, a biotechnology company in San Diego to develop improved treatments for hemophilia. Although the exact nature of the research was not announced, MediVas has developed unique drug delivery systems for recombinant proteins that can improve patient convenience by giving the protein a longer half-life. MediVas technology can potentially be used for oral, inhaled or subcutaneous injection. (injection with a short needle directly under the skin). This can make the administration of the recombinant protein more convenient, more effective and safer, potentially leading to higher patient compliance and better overall health.

The news from Wyeth and Biogen is very dramatic and it is a very positive change for those of us who want Factor IX products to get better and better. Two cheers for these companies, we wish them success. Although starting a new research project to develop new kinds of factor is
a long way from that factor being available, it is
a very important first step. We are very glad that they have chosen to take that step. Hopefully, some time in the future we will have long lasting Factor IX, from either Biogen or Wyeth, and we will have orally delivered Factor IX from either Wyeth or Inspiration. We are keeping our fingers crossed.~

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Highlight pictures from a gathering and our Breakfast meeting at the NHF, October 11, 2006

Many Factor Nine families gathered at an evening event held at the Franklin Institute

 

Our Factor Nine family meeting on Saturday morning had a very good turnout!

Please join us at the HFA in New Mexico for a breakfast meeting on Saturday, March 3, 2007

And our New York Symposium on Saturday, March 31, 2007 which is posted on our website!

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For back issues of Factor Nine Newsletter or for more information on research please call or write to:
Kim Phelan, 825 Third Avenue, Suite 226, New York, New York 10022, Telephone: (212) 520-8272
Telefax: (212) 520-8501, E-mail: hemob@ix.netcom.com Website: www.coalitionforhemophiliab.org