Raptor Pharma Reports Data from Phase 3 Study of QUINSAIR

Raptor Pharmaceutical Corp.

, a biopharmaceutical company developing and commercializing transformative treatments for rare diseases, today announced new data analyses of its Phase 3 clinical study (MPEX-209) comparing QUINSAIRTM to inhaled tobramycin solution in patients with cystic fibrosis and chronic Pseudomonas aeruginosa infections that were presented at the 39th European Cystic Fibrosis Conference in Basel, Switzerland. "Patients with cystic fibrosis have few approved inhaled antimicrobial options for the treatment of chronic Pseudomonas infection and in many cases, continue to have pulmonary exacerbations despite available therapies," said Krishna R. Polu, M.D., Chief Medical Officer of Raptor. "We believe it is critically important to provide physicians with additional data that suggest that QUINSAIR may reduce the number of pulmonary exacerbations in patients with a history of frequent pulmonary exacerbations. Further analyses suggest that QUINSAIR administration did not increase the risk for colonization with new pathogens or lead to increased antimicrobial susceptibilities." History of Pulmonary Exacerbations (PEx) as a Predictor of Response to Nebulized Levofloxacin Compared with Nebulized Tobramycin, P.A. Flume – Poster number 38. Key findings: MPEX-209 was a randomized, controlled, open-label study of levofloxacin inhalation solution, also known as MP-376/QUINSAIR, versus tobramycin inhalation solution over three 28-day cycles alternating with intervals of 28 days off treatment. Subjects were categorized by prior-year pulmonary exacerbation (PEx) history as zero, one to two, or three or more. Study subjects were analyzed by these categories for PEx occurring during the study, which were defined as events resulting in treatment with antibiotics among patients meeting ≥4 of 12 respiratory signs and symptoms. The analyses showed that subjects with three or more prior year PEx who were randomized to receive QUINSAIR had a significantly lower incidence of PEx when compared with their peers who were randomized to receive the active comparator, tobramycin inhalation solution (p=0.026). The most frequently reported adverse reactions were cough/productive cough, dysgeusia and fatigue/asthenia. Microbiologic Changes Observed over 6 months in a Randomized, Open-Label Comparison of Inhaled Levofloxacin and Inhaled Tobramycin in Persons with CF and Chronic P. aeruginosa (Pa) Airway Infection, D.R. VanDevanter – Poster number 37. Key findings: In MPEX-209, over the course of the three on/off cycle 168-day study of inhaled tobramycin or levofloxacin, no significant changes within treatment groups or differences between treatment groups were observed by selective culture with respect to: the proportion of P. aeruginosa (Pa) isolates susceptible to levofloxacin, tobramycin, aztreonam, or meropenem using EUCAST breakpoints and the proportion of Pa isolates not susceptible to at least 3 of these 4 classes. The prevalence of CF bacterial opportunists cultured from study patients did not change significantly within treatment groups or between treatment groups over the course of the study. The most frequently reported adverse reactions were cough/productive cough, dysgeusia and fatigue/asthenia.