SpliSense announced that it has initiated the Phase 2b portion of the SPL84-02 trial of the SPL84 inhaled antisense oligonucleotide in CF patients with the 3849+10kb C→T splicing mutation in the CFTR gene. The Phase 2b study is expected to enroll approximately 40 patients who are currently receiving treatment with Trikafta / Kaftrio or Alyftrek and will compare once-a-week doses of nebulized SPL84 to placebo over12 weeks.
In June 2026, the company announced that up to $13 million in additional funding from the Cystic Fibrosis Foundation would support an ongoing Phase 2b trial. SpliSense announced data from the Phase 2a portion of the SPL84-002 trial in September 2025.
SpliSense CEO Gili Hart commented, “Following the favorable safety and efficacy profile demonstrated in the Phase 2a study, we are excited to initiate the Phase 2b study, designed to determine whether once-weekly SPL84 can provide additional clinical benefit for people with the 3849+10kb C→T mutation who are already receiving standard–of–care CFTR modulator therapy. We selected the 50 mg dose based on the favorable Phase 2a safety profile and the consistent encouraging clinical activity observed at this dose. Given SPL84’s RNA-level mechanism, which is distinct from CFTR modulators, we believe there is a strong scientific rationale for evaluating the combination. More broadly, SPL84 provides important clinical experience supporting our inhaled ASO approach for pulmonary diseases. We look forward to the Phase 2b topline results, expected in the second half of next year.”
Read the SpliSense press release
