ADDITIONAL RARE INFLAMMATORY DISEASE OPPORTUNITIES

We are evaluating other rare inflammatory conditions in which there is promising scientific evidence to support an important role for inflammation driven by LTB4 in the pathogenesis of disease. We believe that modulating LTB4 by inhibiting LT4AH may have therapeutic benefits in other rare pulmonary conditions besides CF, including pulmonary hypertension, or PH, a condition that is characterized by narrowing of the blood vessels connecting the heart to the lungs, and neutrophil driven non-CF bronchiectasis, or non-CF BE, a condition characterized by symptoms similar to CF but without the underlying gene defect associated with CF. Based on third party sources and internal estimates, we believe that inflammatory forms of PH affect at least 41,800 people in the United States and the European Union, and bronchiectasis affects approximately 211,000 people in the United States and the European Union.

In addition, there are several rare, neutrophil driven inflammatory dermatoses, or bullous dermatoses, where there is scientific evidence that neutrophils and LTB4 may drive the pathogenesis of disease.  Based on third party sources and internal estimates, we believe the most common form of bullous dermatoses, bullous pemphigoid, affects approximately 24,500 people annually in the United States and the European Union. We are currently conducting exploratory work in PH, non-CF BE and bullous dermatoses to identify patient populations in those indications that could benefit most from treatment with acebilustat with the goal of advancing acebilustat into Phase 2 clinical development for one or more of these indications in 2018. Importantly, there are currently no drugs approved to treat non-CF BE or bullous dermatoses and no anti-inflammatory therapies approved to treat inflammatory forms of PH.