#Israel #Jerusalem #cystic fibrosis #SpliSense #SPL84

SpliSense Reports Positive Results from Phase 2 Study of SPL84 in Cystic Fibrosis

SpliSense, a pioneering clinical-stage biotechnology company dedicated to developing revolutionary RNA-based treatments, has announced promising results from its Phase 2 trial of SPL84. This innovative antisense oligonucleotide therapy aims to treat cystic fibrosis (CF) patients who carry the rare 3849+10 Kb C->T mutation in the CFTR gene. The latest data suggests that nearly 70% of participants experienced improvement in lung function, marking a significant milestone for both the company and pulmonary disease therapeutics.

Key Highlights from the Trial

Conducted across various centers in the United States and Europe, the SPL84-002 study is a randomized, double-blind, placebo-controlled Phase 2 trial that assesses the safety and efficacy of SPL84. The results reveal not only the potential benefits of this therapy but also its safety profile. Not one severe adverse event was recorded, indicating a favorable outcome for the participants involved in this critical study.

• - Improved Lung Function: Analysis of the study’s first two cohorts, totaling 12 participants, showcased that approximately 70% who received SPL84 treatment displayed a marked improvement in lung function (measured as ppFEV1) compared with those in the placebo group.
• - Safety Assessment: Alongside efficacy, the safety of SPL84 was under scrutiny, with the absence of concerning safety signals noted during the trial, affirming its potential as a viable treatment option.

Dr. Gili Hart, CEO of SpliSense, expressed that the results from the SPL84-002 study hold substantial significance not just for their company but for the pulmonary therapeutics field as a whole. “This data is groundbreaking, as it shows for the first time that an inhaled antisense oligonucleotide therapy can effectively target and treat a serious pulmonary disease,” stated Hart.

Future Implications

The study outcomes not only validate SpliSense’s unique antisense oligonucleotide platform but also clear the path for additional programs focused on treating various pulmonary conditions, including Chronic Obstructive Pulmonary Disease (COPD), asthma, and idiopathic pulmonary fibrosis (IPF). SpliSense is gearing up for more studies expected to begin in early 2026, aiming to advance treatments for these conditions and bring hope to affected individuals.

About SPL84

SPL84 works by addressing the specific splicing defect caused by the 3849+10 Kb C->T mutation, enabling the production of a functional CFTR protein. This inhaled therapy has demonstrated its capability to restore CFTR activity in preclinical models effectively. Recognized by the U.S. Food and Drug Administration (FDA) with Orphan Drug and Fast Track designations, SPL84 has been acknowledged for its potential to provide meaningful clinical benefits.

SpliSense’s commitment lies in developing innovative treatment solutions for patients burdened by severe pulmonary conditions, proving that the future of CF therapies may indeed arrive sooner than once anticipated. As the company approaches the completion of the SPL84-002 study’s third cohort, the anticipation builds around the full data presentation to the cystic fibrosis community.

For more updates and information about SpliSense and its pioneering therapies, visit www.splisense.com.