FDA Grants Fast Track for SpliSense's SPL84 for Cystic Fibrosis

Jerusalem, May 29, 2024 – SpliSense, a clinical-stage biotech company specializing in RNA-based therapies for various pulmonary diseases such as cystic fibrosis (CF), COPD, asthma, NCFB, and idiopathic pulmonary fibrosis (IPF), revealed that its lead product SPL84 has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for treating CF.

SPL84: A Promising Treatment for CF

SPL84 is an antisense oligonucleotide (ASO) product designed to treat CF patients who have the 3849+10 kilobase (Kb) C->T splicing mutation in the CFTR gene. This development was heralded as a significant milestone by SpliSense CEO, Gili Hart, PhD. He mentioned that the FDA’s Fast Track Designation highlights the urgent necessity for an effective treatment for CF patients with this specific mutation, a condition marked by insufficient treatment options.

Dr. Hart emphasized that the combination of the recent Investigational New Drug (IND) clearance from the FDA and the Fast Track status for SPL84, which is currently under evaluation in a global Phase 2 study, will facilitate the expedited development of a potentially transformative treatment for CF patients with the 3849+10 Kb C->T mutation. He also noted that this advancement could extend their unique technology to other pulmonary diseases with significant unmet needs.

Understanding Fast Track Designation

The FDA’s Fast Track Designation is aimed at accelerating the development and review of drugs intended to treat severe conditions with unmet medical needs. This status offers enhanced communication and collaboration between the FDA and drug developers, potentially hastening the delivery of critical treatments to patients.

Cystic Fibrosis: A Genetic Challenge

CF is a genetic disorder that affects multiple systems, originating from mutations in the CFTR gene responsible for producing the CFTR protein – a chloride channel expressed in the lungs and other tissues. Over the past decade, CF care has evolved with the approval of new CFTR modulators, yet these do not benefit all CF patients and do not provide a cure. Therefore, new therapeutic strategies are essential, particularly for those with the 3849+10 Kb C->T splicing mutation who remain unresponsive or only partially responsive to current treatments.

The Mechanism and Potential of SPL84

SpliSense employs short, targeted RNA sequences called Antisense Oligonucleotides (ASOs) to modulate specific mRNA sequences or correct mutations in the target mRNA. SPL84 targets the mutated CFTR RNA, modulating the mutated mRNA region, which can potentially enable cells to produce fully functional CFTR proteins. Administered directly to the lungs via inhalation, SPL84 is designed for uptake by lung cells to drive the production of corrected CFTR mRNA and fully functional CFTR proteins. SPL84 has demonstrated the ability to fully restore CFTR activity in the gold standard pharmacological model for CF and is currently being tested in a global Phase 2 study for CF patients with the 3849+10 Kb C->T mutation through a weekly inhalation regimen.

SpliSense’s Vision

SpliSense is dedicated to developing RNA-based treatments for pulmonary diseases using its innovative ASO platform. The company’s approach aims to tackle specific genetic mutations or deficiencies by restoring or reducing protein function, addressing the root causes of diseases like CF, muco-obstructive diseases, and IPF.

This announcement marks a significant step forward in the treatment of CF, offering hope for more effective and targeted therapies for those with challenging genetic mutations.

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