Sionna Therapeutics, a US-based life sciences company, announced on Wednesday. that it has received approval from the US Food and Drug Administration (FDA) for its Investigational New Drug application (IND) for SION-638, a novel small molecule that targets the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein.

The company has dosed its first subject in a phase one study to assess the safety and pharmacokinetics of SION-638 in healthy volunteers.

Mike Cloonan, Sionna Therapeutics president and chief executive officer, said, 'Despite advances in CF treatment, the unmet need is high as current therapies can only partially correct the function of the CFTR protein, which means many patients are still at risk of experiencing exacerbations, complications, and a burden on daily life. We have achieved an important milestone of advancing SION-638, a first-in-class NBD1 modulator, into a Phase 1 study. We are encouraged by data from clinically predictive CF models demonstrating the potential to normalise folding, maturation, and function of the CFTR protein, and we look forward to evaluating our ability to drive improved outcomes for people with CF in clinical trials.'