Sionna Begins Phase 1 Trial for Cystic Fibrosis Drug SION-109

Sionna Therapeutics, a clinical-stage life sciences firm, has embarked on a new chapter in cystic fibrosis (CF) treatment with the initiation of a Phase 1 clinical trial for its novel small molecule, SION-109. This advancement follows the U.S. Food and Drug Administration's (FDA) approval of the company's Investigational New Drug application (IND), marking a significant milestone in the pursuit of innovative CF therapies.

The SION-109 molecule is a breakthrough in the field, specifically designed to interact with the CFTR protein's intracellular loop 4 (ICL4) and the first nucleotide-binding domain (NBD1). This approach is poised to complement Sionna's existing NBD1 modulator portfolio, exemplified by SION-638, which is already undergoing Phase 1 trials as a pioneering NBD1 stabilizer.

The company's President and Chief Executive Officer, Mike Cloonan, expressed enthusiasm about the potential of SION-109, highlighting its unique capacity to address NBD1 correction and the opportunity it presents for developing proprietary combination treatments. These treatments could potentially achieve a complete restoration of CFTR function, a significant advancement in the management of CF.

Cystic fibrosis is a genetic disorder caused by mutations in the CFTR protein, which is crucial for the production of healthy mucus in various organs, including the airways and digestive system. The ΔF508 mutation is the most prevalent, leading to a malfunctioning NBD1 domain and impaired CFTR function. Sionna's strategy to target both NBD1 and ICL4 with its small molecules aims to enable combination therapies that could correct these defects fully.

Sionna Therapeutics, founded in 2019 and grounded in over a decade of research on CF-related genetic mutations, is committed to normalizing CFTR function to halt disease progression. The company's pipeline is focused on small molecules that correct the defects caused by the ΔF508 mutation. With a first-in-class portfolio targeting NBD1 correction and additional programs for ICL4 and TMD1, Sionna is on a mission to provide superior efficacy and long-term benefits for CF patients.

The commencement of the Phase 1 clinical trial for SION-109 signifies a leap forward in the quest for more effective CF treatments. It underscores Sionna's dedication to rapid program advancement, driven by a team with profound expertise in CF and a singular goal of offering new hope to those affected by the disease and their families.