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news.Raptor Pharmaceutical (Raptor) has reported the publication of the results from a Phase 2a clinical trial of a prototype formulation of its proprietary delayed-release cysteamine bitartrate (DR Cysteamine) in patients with nephropathic cystinosis (cystinosis).
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The Phase 2a study has demonstrated proof-of-concept for DR Cysteamine, which is Raptor’s proprietary, delayed-release, enteric-coated microbead formulation of immediate release cysteamine bitartrate contained in a gelatin capsule.
The results indicated that when given twice daily, the prototype DR Cysteamine formulation was effective at maintaining low white blood cell (WBC) cystine levels in subjects with cystinosis.
The results also indicated that the prototype DR Cysteamine effectively maintained trough WBC cystine levels within a satisfactory range when patients received approximately 60% of the previous total daily dose of IR Cysteamine.
In the Phase 2b study, DR Cysteamine has demonstrated improved tolerability and the potential to reduce total daily dosage and administration frequency compared to IR Cysteamine. Pharmacokinetic evaluation showed that DR Cysteamine had a terminal half-life more than three times longer than the terminal half-life of IR Cysteamine.
During the first quarter of 2010, Raptor plans to meet with the FDA and European Medicines Agency (EMEA) to discuss plans for a repeat-dose, pivotal, Phase 3 clinical trial in cystinosis patients. Upon receiving FDA and EMEA agreements on protocol, Raptor intends to initiate its Phase 3 clinical trial at multiple sites in the US and Europe.
Ranjan Dohil, lead author of the study, said: “We believe the results of the Phase 2a study bring us significantly closer to a potential treatment solution for cystinosis patients. The results from our trial indicate prototype delayed-release cysteamine formulation lead to improved tolerability and efficacy when administered twice-daily and at a lower total daily dose than IR Cysteamine.
“Based on these results, I believe that Raptor’s final DR Cysteamine formulation has the potential to improve compliance and long-term treatment outcomes for cystinosis patients.”