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A Opening in the Market. Why develop a new PAH drug. Pulmonary Arterial Hypertension. Increase in pulmonary arterial resistance and pressure Pathophysiology: vasoconstriction, thickening and occlusion of pulmonary arteries and arterioles Symptoms: Shortness of breath
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A Opening in the Market Why develop a new PAH drug
Pulmonary Arterial Hypertension • Increase in pulmonary arterial resistance and pressure • Pathophysiology: vasoconstriction, thickening and occlusion of pulmonary arteries and arterioles • Symptoms: • Shortness of breath • Decreasing exercise tolerance • Right heart failure • Marked functional limitation • Right side of the lungs becomes enlarged
Inhaled Nitrite Decrease Pulmonary Arterial Hyperplasia Normal artery Artery with PAH Treated artery
Prevalence/ Treatment • Fatal outcome: <3 yrs (w/o treatment) 5-7 yrs (w/ treatment) • US prevalence: 50,000-100,000 (est) 16,500 diagnosed & treated • Three main classes of drugs- each targets different pathway • This allows for combination therapy • PAH is a growing niche market • New drugs have been approved, but there is still room for improvement • efficacy and safety
PAH Market • Global Market worth approx. 3.3 billion • grew due to: • drug development activity • increased awareness • Limited Market • High intensity leads to competition
Why Aironite? • Identified nitrite (NO pro-drug) technology at the NIH and felt it had great potential • Nitric oxide approved in pediatric pulmonary hypertension • Newly recognized nitrite biology • Animal & human data support further development • Pulmonary specific vasodilator and delivery system; minimal, if any, side effects • Compare to Traceleer • Current therapies are not curative; median survival improved from 3+ to 5+years. New drugs are needed. • Expect additive or synergisitic efficacy when combined with oral and inhaled Rx targeting other pathways (prostacyclin, endothelin) • Possibility of one dose a day • Convenient and cost effective
Why Aironite? • Current therapies are not curative; median survival improved from 3+ to 5+years. New drugs are needed.