Adam Walsh

Adam Walsh asked about brensocatib persistence, referencing a pulmonologist's view that it prevents long-term damage rather than providing immediate symptomatic relief, and flagged the one-year mark as a key discontinuation risk. He requested aggregate persistence or refill data and the company's strategy for ensuring persistence.

Answer

Will Lewis, Chair and CEO, stated that BRINSUPRI refills are 'going very well,' which is a positive early sign. He highlighted the low treatment burden of a once-a-day pill and positive patient feedback on feeling better. He also noted that the statistically significant preservation of lung function at the 25mg dose, even without immediate symptomatic relief, is a compelling driver for continued use, which is supported by the current refill rates.

Adam Walsh from Stifel, Nicolaus & Company, Inc. asked about the specific clinical thresholds for announcing PTG-300 data, whether a twice-weekly dosing regimen is being considered for indications other than beta-thalassemia, and for comments on competition from Morphic's oral alpha-4-beta-7 integrin competitor.

Answer

CFO Donald Kalkofen and President and CEO Dinesh Patel explained that the threshold for announcing PTG-300 data is reaching a point of certainty that warrants moving into a registration program, with the primary goal of selecting the first pivotal indication in 2020. Regarding dosing, they noted the study design allows for flexibility, but the goal is always the lowest, least frequent dose possible. Mr. Patel acknowledged awareness of competitors like Morphic but declined to comment on their programs.