FDA Approves Phase 2 Trial for SpliSense's Cystic Fibrosis Drug SPL84

SpliSense, a clinical-stage biotech firm, has received FDA approval to begin a Phase 2 trial for SPL84, a drug designed to treat cystic fibrosis (CF). The drug is an antisense oligonucleotide (ASO) targeting individuals with a specific CFTR gene mutation. The FDA's green light came after Phase 1 trials and preclinical data showed SPL84 could fully restore CFTR protein and activity, a crucial step in treating CF.

SpliSense has also secured funding from the Cystic Fibrosis Foundation and other investors for the SPL84 trial and other respiratory disease programs. This marks the foundation's third investment in the SPL84 initiative.

Gili Hart, SpliSense's CEO, expressed enthusiasm about the Phase 2 study, noting that while existing CF therapies have improved life expectancy, there's a need for treatments that can modify the disease's course. SPL84 has demonstrated its ability to restore CFTR activity fully in a standard pharmacological model of CF. The company hopes to provide a transformative treatment for CF patients with the 3849+10 Kb C->T mutation and plans to extend its technology to other CF mutations where needs are unmet.

SpliSense is also working on two other ASOs, SPL5AC and SPL5B, targeting muco-obstructive diseases and idiopathic pulmonary fibrosis (IPF), respectively. SPL5AC is designed to reduce mucus viscosity and inflammation, while SPL5B aims to decrease MUC5B overexpression linked to IPF development. Both are expected to enter clinical trials within a year.

CF is a genetic disorder caused by mutations in the CFTR gene, impacting the production of the CFTR protein, essential for chloride transport in the lungs and other tissues. Despite advancements with CFTR modulators, they do not benefit all CF patients or cure the disease. New therapeutic approaches are necessary for those partially or non-responsive to current treatments, particularly those with the 3849+10 Kb C->T mutation.

SpliSense's ASOs are short, targeted RNA sequences that can modulate mRNA sequences or correct mutations, potentially enabling cells to produce functional CFTR proteins. SPL84 is inhaled directly into the lungs, where it targets lung cells to produce corrected CFTR mRNA and proteins.

The company's investors include prominent names in the biotech and healthcare sectors, reflecting the significance of its work in RNA-based treatments for pulmonary diseases. SpliSense's innovative approach aims to address the root causes of diseases by restoring or reducing protein function affected by genetic mutations or deficiencies.