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World experts in the field of Cystinosis and families affected byCystinosis attending the event can discuss Mr. Daley's presentation between3:45pm and 5:30pm in the conference hall on Friday, June 27, 2008, during theposter and round table session of the conference.
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For more information on this event please visit: http://www.cystinosis.ie.
Cystinosis is an inborn metabolic error characterized by abnormaltransport of cystine, an amino acid, out of the lysosomes. Symptom onsettypically occurs within the first year after birth, when cystine crystalsaccumulate in various tissues and organs, including the kidneys, brain, liver,thyroid, pancreas, muscles and eyes. Failure to treat the disease can causeserious health consequences, including renal failure and resultant kidneytransplant, growth failure, rickets, photophobia and blindness.
In collaboration with the University of California, San Diego ("UCSD"),supported by the Cystinosis Research Foundation ("CRF"), Raptor is developinga delayed-release version of cysteamine bitartrate ("DR Cysteamine") for thetreatment of Cystinosis. Cysteamine bitartrate, a cystine-depleting agent, isthe only Cystinosis treatment approved for sale by the U.S. Food and DrugAdministration ("FDA") and the European Medicines Agency ("EMEA").
Although it can delay or prevent kidney transplant in Cystinosis patients,the current formulation of cysteamine bitartrate is not well tolerated by manyCystinosis patients, who experience severe gastrointestinal distress from thedrug. Additionally, the strict, current four times daily dose regimen meansthat Cystinosis patients must be awakened every night to take the medicine.These side effects and frequent dosing requirements result in non-complianceor skipped doses for many patients. Studies have shown that non-compliancewith the current cysteamine bitartrate dose regimen can lead to deterioratingkidney function and other complications.
Based on safety and efficacy studies performed at UCSD, Raptor believesthat Cystinosis patients could benefit from a more convenient and superiortreatment alternative utilizing DR Cysteamine.
Mr. Daley stated, "We are pleased to have been invited to present at theInternational Cystinosis Conference. I look forward to sharing results fromstudies performed at UCSD with a delayed-release form of cysteamine bitartratewhich suggest that DR Cysteamine has the potential to become a safer and moreeffective treatment option for Cystinosis patients and could resolve currentnon-compliance issues prevalent in this patient population. These findingsare supported by the Cystinosis Research Foundation's survey results on thechallenges patients face in complying with the current drug's frequent dosingrequirements, which I also plan to present. Our goal is to improve thequality of life of the Cystinosis patients and their families."
Raptor plans to initiate an expanded study of its final proprietary DRCysteamine formulation in collaboration with the UCSD clinical researchers in2009.
About Cystinosis Foundation Ireland and the International CystinosisConference
Cystinosis Foundation Ireland was set up in 2003 to support Cystinosispatients and their families. The goal of the Foundation is to raise awarenessabout Cystinosis and support research through its fundraising efforts. TheInternational Cystinosis Conference takes place every two years in Europe. Inaddition to world experts in the field of Cystinosis, the conference attractsC
