The introduction of CFTR modulators has transformed the treatment of cystic fibrosis (CF), greatly improving lung function and quality of life for people with the disease.
Despite these major advances, however, airway inflammation is not fully resolved and continues to drive the progression of lung damage. Existing anti-inflammatory drugs such as ibuprofen and azithromycin can cause side effects that limit their long-term use. This creates an urgent need for new anti-inflammatory therapies that are both effective and safe, and that can be used alongside CFTR modulators.
The anti-inflammatory compound GY971, previously identified by the research team (FFC#22/2019 e FFC#10/2022) and intended for direct delivery to the lungs, has already been granted Orphan Drug Designation for cystic fibrosis by the European Medicines Agency (EMA).

With this project, FFC Ricerca aims to support the pharmacological development of GY971. For the compound to become an actual drug, it is essential to minimize potential risks of failure along the development pathway.
In drug development, the de-risking phase involves collecting robust and reliable data to assess the compound’s potential as early as possible and to increase the likelihood of success in later stages.

Thanks to the expertise of the research teams at the Universities of Padua and Ferrara, and in collaboration with SINTEF, GY971 will be produced in larger quantities, its toxicity will be evaluated in several experimental models, and studies will be carried out to identify the most effective routes of administration.

Together, these studies aim to lay the foundation for future clinical trials and ultimately for the development of a new, safe, and targeted anti-inflammatory treatment for people with cystic fibrosis.