Pharma Focus Asia

SpliSense Granted FDA Clearance for Investigational New Drug Application to Initiate Phase 2 Trials of SPL84 in Cystic Fibrosis Treatment

Thursday, April 04, 2024

SpliSense, a biotechnology company specializing in RNA-based therapies for pulmonary diseases, has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application of SPL84, its leading antisense oligonucleotide (ASO) product targeting cystic fibrosis (CF). This clearance allows the commencement of a Phase 2 study for CF patients carrying the 3849+10 Kb C->T splicing mutation in the CFTR gene.

The FDA clearance follows positive safety data from Phase 1 trials and preclinical studies showing SPL84's potential to fully restore CFTR protein and activity. The company has secured funding from the CF Foundation and other investors to support the Phase 2 study and further pulmonary programs, with the CF Foundation making its third investment in SPL84.

Dr. Gili Hart, CEO of SpliSense, expressed enthusiasm for advancing SPL84, emphasizing the significant unmet medical need for CF treatments despite advancements in recent decades. SPL84 has shown promise in restoring CFTR activity in pharmacological models, and with FDA clearance, the company aims to deliver a transformative treatment for CF patients with specific mutations.

Furthermore, SPL84 serves as a proof-of-concept for SpliSense's platform in treating other pulmonary diseases, including muco-obstructive diseases and idiopathic pulmonary fibrosis (IPF), with plans to enter clinical trials within the next year.

Cystic Fibrosis (CF) is a genetic disorder resulting from mutations in the CFTR gene, leading to impaired CFTR protein function. While recent years have seen progress with CFTR modulators, there remains a need for treatments addressing non-responsive or partially responsive CF cases, particularly those with specific mutations like the 3849+10 Kb C->T splicing mutation.

SpliSense's approach involves utilizing antisense oligonucleotides (ASOs) to modulate mRNA sequences or correct mutations, with SPL84 specifically targeting mutated CFTR RNA to potentially restore functional CFTR protein production. Administered via inhalation, SPL84 is designed to reach lung cells and facilitate the production of corrected CFTR mRNA and functional proteins.

In addition to CF, SpliSense is developing ASO assets for muco-obstructive diseases (SPL5AC) and IPF (SPL5B). SPL5AC aims to reduce MUC5AC expression, thereby improving lung function and reducing inflammation, while SPL5B targets MUC5B overexpression associated with IPF to potentially slow disease progression and enhance patient quality of life.



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