Scientists Honored for Role in Breakthrough Drug

June 8, 2017

NEW YORK, June 8, 2017 - Two scientists who spearheaded the research that brought a life-saving drug to patients around the world are being honored at the annual Cooley's Anemia Foundation Gala tonight in New York City. Drs. Michael Spino and Fernando Tricta will jointly receive the Humanitarian of the Year Award for their work on deferiprone (Ferriprox™), the first oral medication used to treat iron overload caused by blood transfusions in people with certain hereditary red blood cell disorders (thalassemia syndromes).

Prior to the approval of Ferriprox™, many patients died from cardiac failure and other complications from iron overload, as well as the inadequacy of the only existing treatment. Patients also suffered a significant burden on their quality of life due to the need of a treatment process that required lifelong, daily 8-12 hour injections to remove excess iron from the body.

In making this announcement, Anthony Viola, National President of the Cooley's Anemia Foundation Board of Directors, said, "Doctors Spino and Tricta are true heroes in the thalassemia community. Their efforts have led directly to prolonging the lives of our patients and enhancing the quality of their lives. We are so grateful that they have agreed to let us publically acknowledge them."

"It is humbling to have this honor bestowed on us, but the impact that this drug has had on so many lives would not have been possible without the unwavering support of Dr. Barry Sherman, the Founder of Apotex," said Dr. Spino. "To this day, we still receive personal, touching stories from patients living with thalassemia, telling us how this drug has positively changed their lives."

Co-recipient of the award is Dr. Fernando Tricta, a pediatric hematologist who was instrumental in the clinical studies for the assessment of the safety and efficacy of deferiprone in his pursuit of better treatment options for people with thalassemia. Almost 20 years after its first approval, he still reflects on his first experiences with the drug. "When I first started treating patients with this drug on a compassionate basis, I could not believe the improved quality of life it had versus the available treatment at the time. Now we have deferiprone being recognized by the American Heart Association for its effectiveness in removing excess iron from the heart, which was the main cause of death in transfused patients with thalassemia."

The Cooley's Anemia Foundation is also honoring Dino Philippou, recipient of the 2017 Young Leadership Award, an exceptional young man who has become not only a successful restaurateur but also an inspiring and untiring philanthropist and activist for the Foundation and other causes. Mr. Philippou also happens to be a thalassemia patient.

According to Mr. Viola, "Dino is a great benefactor and friend of Cooley's Anemia Foundation. The fact that he is also a patient is almost coincidental and we would honor him anyway because of his support for our cause. We are happy to acknowledge him with this award."

Dino Philippou says, "I am so happy to accept this recognition from Cooley's Anemia Foundation because they have been so instrumental in improving the lives of all of us with this disorder."

The 2017 Gala Chairman, Frank Fusaro, President, The Forum Group; Chairman, Columbus Citizens Foundation; and Member of the Board of Directors of Cooley's Anemia Foundation announced, "Your support of our efforts on behalf of our patients and their families is very appreciated. Please join us on June 8th to help us honor these deserving individuals and to meet some of the members of our thalassemia family and many of us who support them. I hope to see you there!"

For more information or to arrange an interview with Anthony Viola, Dr. Fernando Tricta or a patient with thalassemia, please contact:

Henry Ackermann
hackermann@thalassemia.org
(212) 279-8090 Ext. 204

For further information about the 2017 Cooley's Anemia Foundation Gala and for information about tickets for the June 8th event visit the Cooley's Anemia Foundation website http://www.thalassemia.org or e-mail darlene@suffolkcaf.com.

About Cooley's Anemia Foundation:
Since 1954, the mission of Cooley's Anemia Foundation is to increase life expectancy and enhance the quality of life for those impacted by thalassemia, a class of genetic blood disorders, most of which require regular blood transfusions and aggressive management of chronic iron overload, the predominant cause of early death. We do so by funding medical research to advance treatment and curative approaches, by supporting and advising patients and their families and advocating on their behalf, and by educating medical professionals and the general public. Every day, we strive for longer and healthier lives for all patients with thalassemia until a universal cure is found.

About Ferriprox™:
The first oral iron chelator used to treat iron overload caused by blood transfusions in people with certain hereditary red blood cell disorders (thalassemia). Prior to the approval of Ferriprox™, many patients died from cardiac failure and other complications from iron overload, as well as the inadequacy of the only existing treatment. Patients also suffered a significant burden on their quality of life due to the need for a treatment process that required lifelong, daily 8-12 hour treatments using an infusion pump to remove excess iron from the body. Ferriprox™ has been used to treat patients for almost 30 years and was first approved in Europe almost 20 years ago. It is now approved in more than 60 countries.

Endnotes: Quotes from thought leaders on the occasion of Drs. Spino and Tricta receiving this award.

"Due to many scientific and economic issues, deferiprone needed a strong commitment from an 'illuminated and high-spirited' developer, or it would have remained an inaccessible orphan drug. Michael threw his heart into the process, brushing aside all the obstacles, and responding to the patients' needs and the request of the medical and scientific community.

Determined to make this drug available to our patients, he not only took on its economic and research challenges but also accepted a great deal of Italian bureaucracy and regulatory burdens. Fernando strongly contributed to the clinical research on deferiprone. He solidified and strengthened collaborations with clinicians and researchers. Remarkably, he always acted as part of the patient community, completely involved in their needs, fears, and hopes!

Thanks to this, the Italian population was provided with a new drug which reduced the patients' morbidity and, overall, the cardiac mortality."

Prof. Adriana Ceci
Gianni Benzi Pharmacological Research Foundation- President
CVBF- TEDDY Scientific Director

"Congratulations to Michael and Fernando, who had the vision to recognize the potential of deferiprone and the need for properly conducted clinical trials so that it could be licensed. Despite enormous obstacles and the need for major financial investment, their vision and tenacity has resulted in Ferriprox™ now being available to patients worldwide, including in North America. With deferiprone proven to be the best chelating drug for extracting iron from the heart, its widespread availability has changed the lives of many."

Victor Hoffbrand
Professor A.V. Hoffbrand
Emeritus Professor of Haematology
University College, London

Contact: Henry Ackermann
hackermann@thalassemia.org
(212) 279-8090 Ext. 204

SOURCE:

Cooleys Anemia Foundation