Sionna Therapeutics announced that it has finished enrolling patients in its Phase 2a PreciSION CF trial, a randomized, double‑blind, placebo‑controlled crossover study that tests the NBD1 stabilizer SION‑719 as an add‑on to the standard of care Trikafta in adults homozygous for the F508del mutation.
The trial is the first human evaluation of an NBD1 stabilizer, a novel mechanism that directly targets the unstable NBD1 domain of the CFTR protein. By stabilizing this domain, SION‑719 aims to improve CFTR function beyond what is achieved with Trikafta alone, addressing the unmet need of the roughly two‑thirds of cystic fibrosis patients who do not reach normal CFTR activity with current therapies.
Sionna’s cash position—$310.3 million in cash and cash equivalents as of December 31 2025—provides a runway through 2028, giving the company flexibility to continue development of its NBD1 stabilizer pipeline, which includes SION‑451 and complementary modulators. The enrollment milestone de‑risks the program and positions Sionna to attract future investment as it moves toward its first topline readout in summer 2026.
"Completion of enrollment in our PreciSION CF proof‑of‑concept trial represents a significant milestone for both Sionna and the cystic fibrosis community. This is the first clinical trial evaluating an NBD1 stabilizer in people with CF, marking an important step toward directly addressing the underlying defect of the most common CF‑causing mutation. We are thankful to the patients, investigators, and the broader CF community for their strong engagement as we advance this trial. We remain focused on executing and look forward to our first data readout in CF patients this summer," said Charlotte McKee, M.D., Chief Medical Officer of Sionna.
With enrollment complete, the company can now begin dosing and data collection, bringing it closer to the anticipated topline readout. The successful recruitment demonstrates Sionna’s operational capability and strengthens the case for the NBD1 stabilization approach, potentially expanding the therapeutic landscape for cystic fibrosis and enhancing the company’s long‑term growth prospects.
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