Sionna Therapeutics, Inc. (NASDAQ: SION), a clinical-stage biopharmaceutical company focused on revolutionizing the treatment for cystic fibrosis (CF), has 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 (NBD1) stabilizers.
The phase 1 trials evaluated the safety, tolerability, and pharmacokinetic (PK) profiles of single ascending doses (SAD) and multiple ascending doses (MAD) of SION-719 and SION-451 in healthy volunteers. The trials demonstrated that both compounds were generally well tolerated, with no serious adverse events or treatment emergent adverse events that led to discontinuation of the drug.
Both stabilizers achieved desired target PK concentrations at multiple dose levels in a twice daily regimen, meeting exposure thresholds that have the potential to provide clinically meaningful benefit if administered as an add-on to standard of care or in a proprietary dual combination with complementary modulators.
Sionna is now planning to advance both NBD1 stabilizers to the next stage of clinical development. SION-719 will move to a phase 2a proof-of-concept trial as an add-on to standard of care in CF patients, with the trial expected to initiate in the second half of 2025 and topline data anticipated in mid-2026. Additionally, SION-451 is set to advance to a phase 1 healthy volunteer dual combination trial with complementary modulators, with the trial also expected to initiate in the second half of 2025 and topline data anticipated in mid-2026.
The positive results from the phase 1 trials have positioned Sionna to progress its vision of developing novel NBD1-led proprietary dual combinations to transform the treatment paradigm for CF patients. The company aims to provide new options for CF patients, potentially addressing the unmet needs that still exist in the current treatment landscape.
Sionna's successful completion of the phase 1 trials and its plans to advance the clinical development of both NBD1 stabilizers while maintaining its timelines and cash runway into 2028 have been seen as important milestones for the company. The U.S. Food and Drug Administration (FDA) has also cleared the investigational new drug (IND) application for SION-719, marking another significant step in the progress of the program.
The market has reacted to these announcements by moving the company's shares 8.87% to a price of $15.765. If you want to know more, read the company's complete 8-K report here.
