Hypertonic Saline for Cystic Fibrosis

DESCRIPTION (provided by applicant): Cystic fibrosis (CF) is an autosomal lethal disease characterized that affects ~30,000 individuals in the US. CF lung disease, the major cause of mortality in CF, is caused by dehydration of airway secretions that leads to mucous adhesion, infection and airways inflammation. A simple means to restore hydration and mucous clearance in CF airways is the inhalation of hypertonic saline (HS), which osmotically draws water onto the airway surface. Rehydration of the lubricant layer of the airway surface liquid facilitates mucociliary clearance (MCC) and therefore the removal of inhaled infectious agents. Previously published evidence has shown that short-term administration of HS improves the rheologic properties and transportability of sputum, hydrates the airway surface, and improves MCC and lung function in patients with CF. Recent clinical studies support the efficacy of daily HS treatments for CF demonstrating improvements in lung function, a reduction in pulmonary exacerbations, and improvements in quality of life. As a result, inhaled HS is becoming a standard of care for CF and is now used by ~50% of patients nationwide. One consequence of the addition of HS to CF treatment regimens is the time required by CF patientsfor its administration. Daily HS treatments require ~1 hour of time commitment, which is superimposed on the ~2 hours of care typically asked of these patients for other aerosols and physiotherapy. Thus, there is a great need to reduce the treatment burden(time commitment) of this therapy to improve the quality of life of CF patients. Parion Science's goal is to develop therapies that provide major improvements in efficacy over current treatments for CF. Parion is developing a specialized device of overnight administration of HS that will reduce daytime treatment requirements for CF patients. Parion has developed a novel nasal aerosol delivery device designed to administer a therapeutic dose of HS overnight that is comfortable, quiet, and compatible with sleep. The device was tested in a clinical deposition study where it achieved a higher than expected pulmonary deposition of HS aerosol, confirming that the device will be able to achieve a sufficiently high deposited pulmonary dose of HS to support futureclinical trials. Based on the success of the deposition study, Parion will continue the development program by evaluating the tolerability and efficacy of HS delivery via the device in a Phase 2 clinical study. If successful, we believe that this approachwill improve the quality of life of CF patients by decreasing the treatment burden associated with daily HS treatments and improve compliance of this therapy. PUBLIC HEALTH RELEVANCE PUBLIC HEALTH RELEVANCE: Cystic Fibrosis (CF) is one of the most common lethal genetic disorders in the US. Inhaled hypertonic saline is a simple, safe, and effective therapy for CF, but it also adds a significant time burden to patients, requiring an additional hour per day on top of the ~2 hours spent taking other treatments. Parion Sciences is developing a device to administer hypertonic saline overnight to patients in order to reduce the treatment burden and improve compliance with this therapy.