SpliSense’s SPL84 Study Shows Promise for Cystic Fibrosis Treatment

Positive Results from SPL84 Phase 2 Study in Cystic Fibrosis

Results mark first evidence of potential clinical benefit of antisense oligonucleotide therapy in a pulmonary disease.

Encouraging safety profile with no identified safety signals to date.

Improvement in lung function observed in up to 70% of participants treated with SPL84.

This readout validates SpliSense's platform, supporting advancement of the Company's additional pulmonary programs.

SpliSense, a clinical-stage biotechnology company dedicated to developing innovative RNA-based therapies for pulmonary diseases, has recently shared positive outcomes from its SPL84-002 study. This Phase 2 trial is evaluating the efficacy of SPL84, which is an antisense oligonucleotide (ASO), specifically targeted toward individuals affected by cystic fibrosis (CF) with a distinct mutation in the CFTR gene.

The study, which includes numerous global sites, demonstrated promising results for the first two cohorts with a total of 12 participants. The findings suggest that up to 70% of the individuals treated with SPL84 exhibited a measurable improvement in lung function, a critical marker for assessing treatment efficacy in respiratory diseases.

Safety and Efficacy of SPL84

Regarding safety, the SPL84-002 study presented a favorable profile with no significant safety concerns or severe adverse events reported thus far. Gili Hart, PhD, the CEO of SpliSense, highlighted the study as a pivotal moment in the journey towards potential treatments for pulmonary conditions. He stated that this is the first instance where an ASO has shown efficacy when administered directly via inhalation for a pulmonary ailment.

The data also underscore the potential of the SPL84 program to validate SpliSense's overall ASO platform. With the positive results in lung function, the company is looking forward to expanding its research into other pipeline projects aimed at muco-obstructive diseases such as Chronic Obstructive Pulmonary Disease (COPD), asthma, and idiopathic pulmonary fibrosis (IPF), with human trials anticipated to commence in the near future.

Understanding the SPL84-002 Study

The SPL84-002 clinical trial is structured as a randomized, double-blind, placebo-controlled study focusing on the safety and effectiveness of the SPL84 treatment. The participants include those diagnosed with cystic fibrosis who carry the specific 3849+10 Kb C->T splicing mutation.

The trial evaluates various dosage levels of SPL84, where participants receive treatments once a week over a nine-week period. The key goals of the study involve assessing changes in lung function, particularly the percent predicted FEV1. Secondary assessments will also include pharmacokinetics and other relevant measures to evaluate the overall impact of the treatment.

SpliSense is currently finalizing recruitment for the third cohort, eagerly anticipating the completion of data analysis to share with the cystic fibrosis community. The institution prioritizes transparency and is committed to enhancing public awareness regarding ongoing clinical trials.

Insights into SPL84

SPL84 represents a significant advancement in inhaled ASO therapy, specifically designed to rectify the splicing defect attributed to the CFTR gene mutation. By binding to the mutated RNA, SPL84 facilitates the production of functional CFTR protein, aiming to restore normal cellular function.

Preliminary studies indicate that SPL84 effectively restores CFTR activity in validated pharmacological models, offering hope for those affected by cystic fibrosis. The treatment's design enables direct delivery to the lungs, thereby targeting the primary site of disease pathology.

SPL84 has received Orphan Drug and Fast Track designations from the U.S. FDA, highlighting its promise and the urgency for developing effective treatments for this serious condition.

About SpliSense

SpliSense is committed to developing pioneering RNA therapies for various pulmonary diseases. The organization's ASO platform addresses the core mechanisms responsible for such conditions, focusing on restoring or enhancing protein function. As part of its broader mission, SpliSense aims to deliver innovative solutions for individuals living with muco-obstructive diseases, including cystic fibrosis, helping them lead healthier lives.

Company Contact:

Tsipi Haitovsky
Global Media Liaison
+972-52-5989-892

Frequently Asked Questions

What is the SPL84-002 study?

The SPL84-002 study is a Phase 2 clinical trial evaluating the safety and efficacy of SPL84 in patients with cystic fibrosis.

How does SPL84 work?

SPL84 is designed to correct splicing defects in the CFTR gene, enabling production of functional CFTR protein.

What were the main findings of the study?

About 70% of participants showed improvement in lung function with no significant safety concerns reported.

What future programs does SpliSense plan to advance?

SpliSense plans to advance additional therapies targeting conditions like COPD, asthma, and idiopathic pulmonary fibrosis starting in early 2026.

How can I learn more about SpliSense?

For detailed information, you can visit the SpliSense website where they provide updates on their research and clinical trials.

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