SpliSense Advances Phase 2b Trial for Cystic Fibrosis Therapy SPL84

The randomized, double-blind study aims to recruit approximately 40 participants currently using Trikafta, Kaftrio, or Alyftrek. Over a 12-week period, subjects will receive a weekly 50 mg dose of SPL84 or a placebo via inhalation. This phase follows a successful Phase 2a trial where 70% of treated participants showed a lung function improvement of at least five points in ppFEV1, confirming the drug’s safety and potential efficacy.

SPL84 operates by correcting RNA splicing defects caused by the specific CFTR mutation, a mechanism distinct from traditional protein-level modulators. CEO Gili Hart noted that the combination of these two approaches offers a strong scientific rationale for treating patients who experience only moderate or variable responses to existing standard-of-care options. The company expects to release topline results in the second half of 2027. The drug has already secured Fast Track and Orphan Drug designations from the U.S. FDA, alongside PRIME status from the European Medicines Agency, underscoring its potential role in pulmonary medicine.