Phase II Development of lancovutide (Moli1901)

Cystic Fibrosis

Cystic fibrosis is a debilitating and fatal genetic disease, affecting 30,000 children and adults in the United States and 37,000 in Europe. Those affected are predominantly Caucasians of Northern European descent.  In the United States, the disorder occurs in roughly one out of every 3,000 live births and leads to death during childhood if not treated. The highest prevalence of cystic fibrosis is found in North America (United States and Canada), Western Europe, including Ireland and the Basque region of Spain. These geographic regions have the largest Caucasian populations and the longest patient life expectancy due to high quality medical care and an economic base of support.

CF involves a genetic mutation of the instrumental protein named cystic fibrosis transmembrane regulator (CFTR) that moves salt and water to the surface of the linings of the lungs, thus providing hydration and facilitate cleansing of the lung's surface. In healthy individuals, this protein acts as an ion-specific channel that modulates salt and water movement. In cystic fibrosis patients, however, there is a defect in this protein channel which leads to poorly hydrated, thickened mucus secretions in the airways, and therefore severely impaired mucociliary flow due to impaired clearance. Chronic secondary infections almost invariably occur, resulting in progressive lung damage and deterioration. Cystic fibrosis-induced damage to the respiratory tract accounts for more than 95% of the morbidity and mortality associated with this disease.

There are currently no FDA-approved treatments specifically designed and indicated to address the underlying ion-transport defect in cystic fibrosis. The focus of available treatments is the relief of the symptoms of the disease.  Current treatments which target the symptoms of CF include Pulmozyme® (dornase alfa by Genentech) and TOBI® (tobramycin for inhalation by Chiron). These primary treatments focus on the respiratory problems that affect CF patients involve clearing the mucus, thinning the mucus, facilitating breathing, controlling inflammation and fighting infection. These drugs are being used in combination for the treatment of CF. Due to the proposed treatment mechanism, it is likely that therapy with Moli1901, Lantibio's lead compound, will postpone or preclude the use of these products.