Boehringer Ingelheim Announces First Patient Enrolled in Phase II Trial of Novel Cystic Fibrosis Treatment (BALANCE-CFTM 1)

Ingelheim, Germany, 16 October, Wed, 10/16/2019 - 14:00

Investigational compound BI ENaC inhibitor delivered twice daily via the Respimat®, a handheld inhaler

Ingelheim, Germany, 16 October, 2019 – Boehringer Ingelheim today announced the first patient enrolled in its Phase II clinical trial BALANCE-CF^TM 1 to evaluate a new potential treatment for cystic fibrosis.^1,2 The trial will investigate how different doses of the compound, an inhaled epithelial sodium channel (ENaC) inhibitor, impact lung function compared to placebo when added to the standard of care in adults and adolescents with cystic fibrosis.^1,3,4

People with cystic fibrosis have genetic mutations that cause mucus in various organs to become thick and sticky.⁵ In the lungs, thick and sticky mucus clogs the airways and traps germs, like bacteria, leading to infections, inflammation, respiratory failure and other complications.⁶ The inhaled ENaC inhibitor (BI 1265162) is designed to block the absorption of sodium that may keep the surface of the airways hydrated, which may help make mucus less thick, making it easier to keep airways clear in cystic fibrosis.^7,8

The ENaC inhibitor is intended to treat patients with all types of cystic fibrosis mutations.^1,9 It is inhaled via the Respimat^®1, which is the platform inhaler of Boehringer Ingelheim. The Respimat^® inhaler actively delivers medicine in a slow-moving soft mist that is easy to inhale even for people who have difficulty breathing.^10,11 People with cystic fibrosis often have a high treatment burden, sometimes spending hours administrating multiple treatments.¹² The BI 1265162 ENaC inhibitor is delivered via the Respimat^®1 to offer a quick inhalation that can be taken anywhere and is intended to be added to the standard of care.

“We are pleased to enrol our first patient into this Phase II trial, and we hope our ENaC inhibitor will be proven to help people affected by cystic fibrosis,” said Dr Kay Tetzlaff, Head of Medicine, Therapeutic Area Inflammation, Boehringer Ingelheim. “Boehringer Ingelheim is committed to researching new treatments that address serious unmet needs. We are hopeful that this treatment, along with its delivery method, will make an important difference for patients affected by this debilitating condition.”

Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time.⁶ Despite improved treatment options in recent years, patients still face deteriorating health and have a median life expectancy in their 40s.¹²

Boehringer Ingelheim
Improving the health and quality of life of patients is the goal of the research-driven pharmaceutical company Boehringer Ingelheim. The focus in doing so is on diseases for which no satisfactory treatment option exists to date. The company therefore concentrates on developing innovative therapies that can extend patients’ lives. In animal health, Boehringer Ingelheim stands for advanced prevention.

Family-owned since it was established in 1885, Boehringer Ingelheim is one of the pharmaceutical industry’s top 20 companies. Some 50,000 employees create value through innovation daily for the three business areas human pharmaceuticals, animal health and biopharmaceuticals. In 2017, Boehringer Ingelheim achieved net sales of nearly 18.1 billion euros. R&D expenditure, exceeding three billion euros, corresponded to 17.0 per cent of net sales.

As a family-owned company, Boehringer Ingelheim plans in generations and focuses on long-term success. The company therefore aims at organic growth from its own resources with simultaneous openness to partnerships and strategic alliances in research. In everything it does, Boehringer Ingelheim naturally adopts responsibility towards mankind and the environment.

More information about Boehringer Ingelheim can be found on www.boehringer-ingelheim.com or in our annual report: http://annualreport.boehringer-ingelheim.com.

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This press release is issued from our Corporate Headquarters in Ingelheim, Germany and is intended to provide information about our global business. Please be aware that information relating to the approval status and labels of approved products may vary from country to country, and a country-specific press release on this topic may have been issued in the countries where we do business.

References

1 Clinical Trials.gov. Available at: https://clinicaltrials.gov/ct2/show/NCT04059094?term=BI+1265162&rank=1. [Last accessed October 2019].

2 Boehringer Ingelheim. Data on file. 2019.

3 EU Clinical Trials Register. Available at: https://www.clinicaltrialsregister.eu/ctr-search/trial/2019-000261-21/FR [Last accessed October 2019]

4 ECFS Clinical Trial Network. Available at: https://www.ecfs.eu/boehringer-1399-0003. [Last accessed October 2019].

5 Cystic Fibrosis Trust. What is cystic fibrosis? Available at: https://www.cysticfibrosis.org.uk/what-is-cystic-fibrosis. [Last accessed October 2019].

6 Cystic Fibrosis Foundation. About Cystic Fibrosis. Available at: https://www.cff.org/What-is-CF/About-Cystic-Fibrosis/. [Last accessed October 2019].

7 Althaus M. ENaC inhibitors and airway re-hydration in cystic fibrosis: state of the art. Curr Mol Pharmacol. 2013 Mar;6(1):3–12. Available at: https://www.ncbi.nlm.nih.gov/pubmed/23547930. [Last accessed October 2019].

8 Cystic Fibrosis Foundation. Drug Development Pipeline. Available at: https://www.cff.org/Trials/Pipeline/details/10164/BI-1265162 [Last accessed September 2019].

9 Mall M.A., et al. Targeting ion channels in cystic fibrosis. J Cystic Fibrosis. 2015;14(5):561–570.

10 Zierenberg, B. Optimizing the in vitro performance of Respimat. J Aerosol Med. 1999;12(suppl 1):S19–S24.

11 Dalby R, et al. A review of the development of Respimat® Soft MistTM Inhaler. Int J Pharm. 2004;283(1–2):1-9.

12 Olufunmilola A, Morris A. Opportunities for Outpatient Pharmacy Services for Patients with Cystic Fibrosis: Perceptions of Healthcare Team Members. Pharmacy (Basel). 2019 Jun; 7(2): 34. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6631244/. [Last accessed October 2019].