Sionna Therapeutics has announced the presentation of Phase 1 clinical trial data for its novel NBD1 stabilizers, SION-719 and SION-451. These findings indicate that both compounds were generally well tolerated among participants and successfully surpassed the desired pharmacokinetic targets.
The Phase 1 data highlights the potential of these first-in-class stabilizers, which are designed to enhance the functionality of the F508del-CFTR protein, a common mutation associated with cystic fibrosis. In addition to the positive tolerability profile, recent preclinical studies have shown that these NBD1 stabilizers can restore the half-life of F508del-CFTR to levels comparable to that of the wild-type protein.
This progress demonstrates a significant step forward in developing effective treatments for individuals with cystic fibrosis, particularly those carrying the F508del mutation. Sionna Therapeutics remains committed to advancing its research and bringing innovative therapies to market for this population.
