SpliSense has announced results from the Phase 2 SPL84-002 study of SPL84 inhaled antisense oligonucleotide (ASO) for the treatment of people with cystic fibrosis who carry the 3849+10 Kb C->T mutation in the CFTR gene. The FDA has granted Fast Track and Orphan Drug designations to SPL84 for this indication. According to the company, following treatment of the first two cohorts, a total of 12 patients, “Improvement in lung function (ppFEV1) was observed in up to 70% of SPL84-treated participants compared with placebo” with no observed safety concerns.
The SPL84-002 trial is expected to enroll a total of 24 CF patients with the 3849+10 Kb C->T mutation and is evaluating 25, 50, and 100mg doses of nebulized SPL84 versus placebo. Each of the cohorts receives either SPL84 or placebo via nebulization once weekly over 9 weeks. SpliSense notes that enrollment for the third cohort is underway.
SpliSense CEO Gili Hart commented, “The SPL84-002 data mark an important milestone, not only for SpliSense but for the entire field of pulmonary ASO therapeutics. They represent the first time an antisense oligonucleotide administered directly to the lungs by inhalation has demonstrated potential efficacy in treating a pulmonary disease.”
Hart added, “The favorable safety and efficacy profile emerging from our SPL84 program provides clinical validation of our ASO platform. Importantly, this readout supports advancement of our additional pipeline programs (SPL5AC for muco-obstructive diseases such as chronic obstructive pulmonary disease (COPD), asthma, non-cystic fibrosis bronchiectasis (NCFB) and CF, and SPL5B for idiopathic pulmonary fibrosis (IPF)) with first-in-human studies expected to begin in early 2026. We remain focused on our mission of developing new treatment options for people suffering from serious pulmonary conditions.”
Read the SpliSense press release
