"Safety, Tolerability and Pharmacokinetics of MP-376 Administered for 14 Days to Stable Pediatric (CF) Patients"

Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory
tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which
has been particularly problematic to eradicate and been implicated as the major cause of
morbidity and mortality in CF patients. Aerosol delivery of antibiotics directly to the lung
increases the local concentrations of antibiotic at the site of infection resulting in
improved antimicrobial effects compared to systemic administration. Bacterial resistance to
current aerosol antibiotic treatments indicate a need for improved therapies to treat CF
patients with pulmonary infections caused by multi-drug resistant Pseudomonas aeruginosa and
other bacteria. High concentrations of MP-376 delivered directly to the lung are projected to
have antimicrobial effects on even the most resistant organisms. MP-376 is a novel
formulation of the fluoroquinolone levofloxacin that has been optimized for aerosol delivery
using the PARI electronic eFlow® nebulizer. Preclinical and early clinical studies in adults
show that aerosol doses of MP-376 appear to be safe and well tolerated, and exert an
antimicrobial effect when administered once or twice daily. High concentrations of
levofloxacin in the lung delivered using MP-376 are expected to be active against CF
pathogens such as P. aeruginosa and S. aureus, including those resistant to aminoglycosides
(such as TOBI®) and other inhaled antimicrobial agents. Inhaled MP-376 can be delivered
rapidly and efficiently using the PARI eFlow® nebulizer system. This Phase 1 study is being
performed to obtain safety, tolerability and PK data in children ages 6-16 in order to
determine if MP-376 is safe, prior to enrolling children of these ages in the planned pivotal
Phase 3 studies.