Sionna Therapeutics announces data from Phase 1 trials of SION-719, SION-451

Sionna Therapeutics (SION) announced positive data from its Phase 1 clinical trials of SION-719 and SION-451, the company’s first-in-class nucleotide-binding domain 1 stabilizers, in healthy volunteers. Both NBD1 stabilizers were generally well tolerated and achieved pharmacokinetic concentration targets established using Sionna’s preclinical CF human bronchial epithelial model. These data reinforce the potential of both NBD1 stabilizers to provide clinically meaningful benefit for CF patients, including the potential for wild-type CFTR function, either as an add-on to standard of care, or in a proprietary dual combination with complementary modulators. Sionna plans to advance both NBD1 stabilizers to the next stage of clinical development – SION-719 to a Phase 2a proof-of-concept trial in CF patients as an add-on to SOC, and SION-451 to a Phase 1 proprietary dual combination trial in healthy volunteers. SION-719 and SION-451 were generally well tolerated and achieved desired pharmacokinetic targets that reinforce their potential as either an add-on to standard of care or in a Sionna dual combination. Sionna plans to advance SION-719 to a Phase 2a proof-of-concept add-on to standard of care trial in cystic fibrosis patients, and SION-451 to a Phase 1 healthy volunteer dual combination trial.