1st Patient Enrolled in Phase 2 Study of Inhalation Treatment for All with CF, Boehringer Says

Boehringer Ingelheim announced the enrollment of a first patient in its Phase 2 BALANCE-CF 1 trial assessing the safety and efficacy of BI-1265162 to treat cystic fibrosis (CF) regardless of a person’s disease-causing mutations.

BI-1265162 is an epithelial sodium channel (ENaC) inhibitor that works to prevent cells lining the lungs’ airways from absorbing sodium. This should help to lessen the viscosity of mucus in patients’ lungs, making it easier for them to clear their airways.

CF is marked by the build-up of thick and sticky mucus in the lungs and other organs, which can trap microbes and promote serious infections and inflammation.

BI-1265162 is inhaled using the Respimat inhaler, Boehringer Ingelheim’s proprietary inhaler. Respimat is a small, plastic, portable device that measures the exact amount of medication that needs to be administered and then, once it is pressed, slowly releases the medicine in puffs that patients breathe in.

BALANCE-CF 1 (NCT04059094) aims to evaluate the safety, efficacy, and pharmacokinetic properties of BI-1265162 in adolescents and adults, ages 12 and older, with CF. (Pharmacokinetics is the study of how a drug is absorbed, distributed, metabolized, and eliminated from the body.)

The randomized, double-blind, and placebo-controlled trial is expected to enroll up to 98 patients six clinical sites in the U.S. and France; information is available here.

Study participants will be randomly assigned to either BI-1265162, inhaled at different doses twice-a-day, or to a similarly administered placebo for four weeks.

Its primary goal is to assess changes in participants’ lung function from the beginning of the study until the end of treatment, and compared to placebo. This will be assessed by changes in forced expiratory volume in one second (FEV1), which measures the total amount of air a person is able to exhale in one second after taking a deep breath.

Secondary goals include safety assessments, several patient-reported outcomes (like quality of life, and cough and sputum assessments), and pharmacokinetic parameters.

“We are pleased to enroll our first patient into this Phase 2 trial, and we hope our ENaC inhibitor will be proven to help people affected by cystic fibrosis,” Kay Tetzlaff, head of medicine, therapeutic area inflammation at Boehringer Ingelheim, said in a press release.

“We are hopeful that this treatment, along with its delivery method, will make an important difference for patients affected by this debilitating condition,” Tetzlaff concluded.

The trial is due to fully conclude in September 2020.