Helen Usansky, Anthony Cammarata, ChongHua Li, Sam Au Yeung, Christopher M Rubino, Lin Wang, Ariel Teper, Carlos Fernandez, Kevin Mange
Brensocatib, a competitive and reversible oral DPP1 inhibitor, at 10 and 25 mg significantly reduced pulmonary exacerbation burden, and 25 mg also reduced lung function decline vs. placebo in patients with non-cystic fibrosis bronchiectasis, hereafter bronchiectasis, in ASPEN (NCT04594369). A population pharmacokinetic (PPK) model was developed to support brensocatib dose selection for adults and adolescents. Data from 11 clinical trials were pooled for analysis. The covariate model was developed via a stepwise approach. Clinical relevance of statistically significant covariates was assessed by simulated brensocatib exposure at steady state in various subgroups with different characteristics relative to a reference subject (healthy White male, 49 years, 70 kg, fasted, no concomitant medications). Covariate effects on brensocatib PK were evaluated. From 291 healthy participants and 807 patients, 11,643 brensocatib plasma concentrations were used for PPK model construction. Body weight was integrated using standard allometric scaling coefficients of 0.75 on total and intercompartment clearance (CL/F and Q/F) and 1.0 on central and peripheral volume of distribution (Vc/F and Vp/F). Statistically significant covariates included hepatic impairment, sex, race, and co-medication use (strong CYP3A and Pgp modulators) on CL/F; age on Vc/F; food effect on Ka; and co-medications (strong CYP3A and Pgp inhibitors) on relative bioavailability. Disease populations, renal impairment, and tablet formulations were not statistically significant covariates. The PPK model provided a robust fit to observed brensocatib concentration time profiles in bronchiectasis patients. No identified covariates were clinically relevant. This analysis supported brensocatib dosage of 10 and 25 mg in patients with bronchiectasis ≥ 12 years without dose adjustment.