Novato, CA-based Raptor Pharmaceutical has reported positive results from a Phase II trial of delayed-release cysteamine bitartrate in patients with nephropathic cystinosis. Cystinosis is a metabolic error that, if left untreated, can cause renal failure and resultant kidney transplant, growth failure, rickets, photophobia and blindness. According to Raptor, DR Cysteamine demonstrated improved tolerability and the potential to reduce total daily dosage and administration frequency compared with the immediate-release version of the drug.
"The results from our Phase IIb trial are consistent with previous clinical findings... [and] support our plans to provide a twice-daily cysteamine therapy for cystinosis patients," noted Patrice Rioux, chief medical officer of Raptor. "We look forward to continuing our development program of DR Cysteamine in cystinosis with a Phase III clinical trial." The late-stage trial is expected to begin in the first quarter of 2010.
In a separate release, Raptor said its neuropathic pain drug NGX426 performed well in an early-stage trial. In a study of 18 patients, the company found that NGX426 could be effective in a variety of neuropathic pain states that are caused by damage to or dysfunction of the peripheral or central nervous system rather than stimulation of pain receptors. Raptor acquired NGX426 when it merged with the now-defunct TorreyPines Therapeutics.
- read Raptor's release on DR Cysteamine
- here's the NGX426 release