Inspiration Biotherapeutics

A Promising Future with Hemophilia B

Laurie Kelley May 4, 2014

 

I’ve always been an admirer of John Taylor, and I’ve known him since both our boys with hemophilia were born in the
1980s. His story is so inspiring! I am pleased to share some of his trials and triumphs, as well as his outlook on hemophilia B and the factor IX product he is helping to bring to market. 
John Taylor Sr.
 
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by John Taylor Sr.

Nearly 25 years ago when our son, John Taylor Jr., was born, my wife Joyce and I were informed that he had hemophilia B. This diagnosis changed our lives. When your child has hemophilia, it’s all encompassing. As a family, you struggle through it every day. But what started as an unknown,
daunting journey has developed into an expedition of hope.

 
In 1990, we were told that there were few treatment options for John. Determined to find the right
treatment and support for ourselves and other families coping with hemophilia B, we reached out to the scientific community. This quest led me to incredible passages in life—founding the Coalition for Hemophilia B in 1990, becoming an activist for FDA approval of additional factor treatments, and starting a company dedicated to advancing access to hemophilia B treatment.
 
That company was called Inspiration Biopharmaceuticals, which our family co-founded with Scott Martin, another father of a son with hemophilia. Our mission was to bring product choice and access to the hemophilia B community. Because we understood the impact of this condition on our own families—as well as many other families we met also coping with hemophilia—we were determined to start a company that would address the concerns we were all living with. Thanks in part to our work, a lot has changed for families living with hemophilia B.
 
When Emergent BioSolutions, a company whose goal is to improve the lives of people with rare conditions, acquired the recombinant Factor IX we were developing, families felt empowered knowing that there was now a company that was working directly for us. 
 
One demand that is finally being addressed by pharmaceutical companies is treatment choice. Soon, people with hemophilia B will have even more product choices for bleed control and prevention than ever before.
 
One product I am particularly excited about is IB1001, from Emergent BioSolutions. IB1001 is a recombinant factor IX product that began
development at Inspiration Biopharmaceuticals. I am so proud to see Emergent continue the work we began at Inspiration, and to know that they are seeking to gain approval for IB1001. Emergent’s focus on developing treatments for rare conditions and blood disorders and their experience

in addressing the needs of small patient populations makes them a welcomed addition to the companies serving the hemophilia B community.

 
Emergent’s mission is simple—to protect and enhance life.  Although they are new to the hemophilia community, their commitment to IB1001 and to improving the lives of patients with hemophilia B makes me eager to see them bring to market a product I believe in so strongly.
 
When I reflect on this journey, I can honestly say that every stop along the way—and every person I’ve met from our community—have added incredible value to my life. My son and this amazing community have given me the drive and determination to keep going. When a company like Emergent comes along and commits to understanding and responding to the needs of the hemophilia B community, I can confidently say that my journey has been worthwhile. With dedication and the commitment that come from having lived with the condition, and a thorough knowledge of our
community, we are proud to have been part of the development of a new recombinant factor IX product. Standing with you, from a family like yours. This is a journey we are taking with all of you.
 
 
 John’s work on behalf of the hemophilia B community continues to make a positive impact. If you’d like to learn more about IB1001, the product he helped develop, and Emergent BioSolutions’ efforts to finish the work John began, please visit www.ebsi.com/hemophiliab. To learn more about the Coalition for Hemophilia B, visit http://coalitionforhemophiliab.org


This is a sponsored public awareness notice.
 

New Factor IX Products in Development

Laurie Kelley January 14, 2008

What’s going on with hemophilia B? A lot! I’ve read recently about four new studies about new products for hemophilia B patients. In a nutshell:

A. Inspiration Biopharmaceuticals (www.inspirationbio.com) has signed an agreement with Cook Pharmacia to develop an injectable recombinant factor IX. So, what’s new about that? I mean, we already have that. Well, Inspiration is focusing on revolutionizing hemophilia treatment in two ways: 1) developing lower-cost versions of existing intravenous recombinant therapies; and 2) developing non-invasive administration of therapies. Like inhaling? (Hence the name, Inspiration? Just a wild guess)

B. Baxter International (www.hemophiliagalaxy.com) has begun preclinical work on a genetically engineered product for bleeding in patients with hemophilia B. Again, we already have that, so what’s new? With its partner Nektar Therapeutics of San Carlos, Calif., Baxter wants a more long-lasting treatment for hemophilia B. Imagine infusions lasting all week instead of a day, or maybe two weeks!

C. Nastech Pharmaceutical (www.nastech.com) has begun a feasibility study with an undisclosed global leader in the production of plasma products. The study will examine the possibility of developing an alternative delivery method of factor IX without an injection. Not much said about this one: who’s the company? What’s the method? More to come, hopefully!

D. GTC Biotherapeutics, Inc., of Framingham, Massachusetts, has purchased a license to develop a new recombinant factor IX product with ProGenetics LLC of Blacksburg, Virginia.

Currently, the only existing recombinant factor IX product is BeneFIX, made by Wyeth Pharmaceuticals of King of Prussia, Pennsylvania. But there sure is a lot of commercial interest in this hemophilia population, which is a small. Of the approximately 17,000 people with hemophilia in the US, only about 15% are estimated to have factor IX deficiency.

If this is all interesting to you, then see about attending The Coalition for Hemophilia B one-day consumer symposium on Saturday, March 8, 2008, from 9:30 am–5 pm, at the Millennium Broadway Hotel in New York City. For information, call Kim at 212-520-8272 or email her hemob@ix.netcom.com

Great Book I am Reading: Dreams from My Father: A Story of Race and Inheritance by Barack Obama. I thought, oh great, a book ghostwritten for a politician seeking election. But I was wrong. He wrote this in 1995, when he was a senator-elect. I am captivated already: it is honest, intimate, frank and eloquent. This is a guaranteed great read (and not a political endorsement), and I always appreciate a well-written book. I am only on Chapter 3 but it is insightful and informative. Obama has led an amazing life, and in this book, he traces his roots, from his grandparents to his childhood, and the unusual path his genealogy has taken and why. Eventually he returns to Kenya to learn more about the father he barely recalls. This is a look at race relations in America, one man’s quest to make a difference for his race, and a quest for the meaning of family for a man who was raised in several diverse cultures. (For those of you fearing I am making a “statement,” I also have it on my list to read John McCain’s book, Faith of Our Fathers, also about the impact his grandfather and father had on his life. ). Three stars out of four.

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