Boehringer Ingelheim Discontinues Cystic Fibrosis Gene Therapy While Advancing €25 Billion R&D Investment Plan
Key Insights
Boehringer Ingelheim has discontinued its inhaled gene therapy BI 3720931 for cystic fibrosis after the phase 1/2 Lenticlair 1 trial failed to generate efficacy data supporting further development.
The German pharmaceutical company is investing €25 billion over five years in R&D, driven by strong sales from Jardiance which increased 28% to €3.9 billion in 2021.
Despite the cystic fibrosis setback, Boehringer continues advancing multiple late-stage programs including zongertinib for lung cancer and nerandomilast for pulmonary fibrosis.
Boehringer Ingelheim has discontinued its inhaled gene therapy for cystic fibrosis after the phase 1/2 Lenticlair 1 trial failed to demonstrate efficacy, marking a setback in the company's respiratory disease portfolio even as it pursues an ambitious €25 billion R&D investment strategy over the next five years.
The candidate, BI 3720931, a lentiviral vector designed for inhalation, was halted after enrolling five patients in the study launched in late 2024. According to the company, while the safety profile aligned with expectations for an innovative inhaled genetic therapy, efficacy data did not justify continuation of the program.
Strong Financial Foundation Drives R&D Expansion
The discontinuation comes as Boehringer reported robust financial performance, with Jardiance (empagliflozin) sales increasing 28% to €3.9 billion in 2021. The SGLT2 inhibitor, partnered with Eli Lilly, has been buoyed by new approval in heart failure. Additionally, pulmonary fibrosis therapy Ofev (nintedanib) achieved a 25% gain to €2.5 billion.
This strong cash flow enabled Boehringer to invest €4.1 billion in R&D last year, the highest spend in the company's 137-year history. The investment rivals spending by much larger pharmaceutical groups as Boehringer pursues a goal of bringing 15 new products to market by 2025.
Pipeline Progress Despite Setbacks
The cystic fibrosis discontinuation does not affect other research programs. Boehringer continues to evaluate verducatib (search), a DPP1 (search)/Cathepsin C (search) inhibitor, in a phase 3 trial for cystic fibrosis-associated and non-CF bronchiectasis.
The company's late-stage pipeline includes several promising candidates. Spesolimab, an IL-36 (search)-targeted antibody, has been filed for approval as a treatment for generalised pustular psoriasis (GPP), a rare and sometimes life-threatening disease with no approved therapies.
Regulatory Milestones and Future Prospects
Boehringer is awaiting key regulatory decisions in the United States. The FDA is expected to rule on full approval of zongertinib, a HER2 (search)-selective tyrosine kinase inhibitor for previously treated HER2-mutated non-small cell lung cancer. This represents the first oral targeted therapy in this setting and has already won accelerated approval.
The company also received FDA approval for nerandomilast, a PDE4B (search) inhibitor for idiopathic pulmonary fibrosis, where phase 3 data suggested potential advantages over existing treatments. Additionally, BI 425809, a Gly-T1 (search) inhibitor, received breakthrough status for cognitive impairment caused by schizophrenia.
Oncology and Digital Health Initiatives
Boehringer's oncology push is headed by MDM2-p53 (search) antagonist BI 907828 in phase 2/3 testing both alone and in combination with the company's experimental PD-1 (search) inhibitor ezabenlimab in advanced solid tumors.
The company is also advancing digital health initiatives, including CT-155, a cognitive behavioral therapy app developed with Click Therapeutics (search) as an adjunct to drug treatment for patients with schizophrenia.
Beyond respiratory disease and oncology, Boehringer is developing survodutide, a dual glucagon/GLP-1 (search) receptor agonist partnered with Zealand Pharma, in late-stage trials for obesity and metabolic dysfunction-associated steatohepatitis.
With more than ten late-stage trials expected to read out within the next 12 to 18 months, the company describes its pipeline as entering a more mature phase, positioning it for continued growth despite individual project discontinuations.