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news.Transave has released interim results from a multi-cycle Phase II open label clinical trial in cystic fibrosis (CF) patients on its lead investigational drug, Arikace (liposomal amikacin for inhalation).
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The data indicated that Arikace, delivered once daily for 28 consecutive days followed by 56 days off-treatment for four cycles, demonstrated improvement in lung function that was sustained during the 56 days off study drug.
Arikace was well-tolerated during the four cycles. Results were presented at the 33rd European Cystic Fibrosis Society (ECFS) Conference in Valencia, Spain, by Predrag Minic, professor of pediatrics and head of pediatrics pulmonology department, Mother and Child Health Institute, Belgrade, Serbia, and co-lead investigator of the study.
The open label study is an extension of a previously reported randomised, placebo controlled Phase II study, and was designed to evaluate Arikace over multiple treatment cycles in CF patients with Pseudomonas aeruginosa lung infections.
In the trial, 49 patients were enrolled to receive Arikace 560mg daily for 28 days of therapy followed by a 56-day off-treatment observation period. Arikace was administered once daily using an eFlow Nebulizer System (PARI Pharma), a novel, highly efficient and portable aerosol delivery system.
Pulmonary function (FEV1) increased among patients receiving 560mg of Arikace, with an estimated relative change from baseline in FEV1 of 9.2% at the end of treatment during cycles one to four. The improvement in lung function was sustained at the end of the 56-day off-treatment period during the four cycles with an estimated relative change from baseline in FEV1 of 4.7%.
Arikace demonstrated reduction in Pseudomonas density, including mucoid strains, which was sustained over the treatment period of four cycles for 12 months. Mucoid strains of Pseudomonas are often difficult to suppress with antibiotics and play a greater role in progression of CF lung disease.
Renu Gupta, executive vice president for development and chief medical officer of Transave, said: “The sustained improvement in lung function with significant reduction in bacterial density over multiple treatment cycles with Arikace is encouraging and indicative of benefit for cystic fibrosis patients who have chronic Pseudomonas lung infections.
“Dr Gupta indicated that preparations are underway to launch Phase III studies to confirm efficacy of Arikace. These results support the potential value of delivering amikacin through Transave’s advanced pulmonary liposome technology designed for sustained release and penetration of the mucus and bacterial biofilm in the lungs.”