SpliSense Granted EMA EARLY PRIME Designation for SPL84 in Cystic Fibrosis

• e-PRIME recognition underscores potential of inhaled RNA therapy to address unmet need in patients with the 3849+10Kb C>T mutation
• Company advancing SPL84 to Phase 2b clinical study

JERUSALEM, March 3, 2026 /PRNewswire/ -- SpliSense, a clinical-stage biotechnology company developing transformative RNA-based therapies for pulmonary diseases, today announced that the European Medicines Agency (EMA) has granted early Priority Medicines (e-PRIME) designation to SPL84 for the treatment of cystic fibrosis (CF) in patients carrying the 3849+10Kb C>T mutation in the CFTR gene.

The e-PRIME designation follows a review by the Committee for Medicinal Products for Human Use (CHMP), which recognized the remaining unmet medical need for patients with this mutation and acknowledged the supportive non-clinical and preliminary clinical data generated to date. In its communication, the CHMP noted that despite the availability of CFTR modulators, there remains a need for novel therapies that can provide substantially improved efficacy, either as standalone treatment or in combination with modulators, as well as for patients not eligible for such therapies.

"This PRIME designation represents a major regulatory milestone for SpliSense and for the SPL84 program," said Gili Hart, PhD, Chief Executive Officer of SpliSense. "It reflects the strength of our mechanistic, non-clinical and emerging clinical data, and reinforces the potential of SPL84 to deliver meaningful benefit to people with cystic fibrosis who continue to represent a significant unmet medical need."

"SpliSense is currently advancing SPL84 to a global Phase 2b study in US and EU designed to further evaluate efficacy and safety in patients with the 3849+10Kb C>T mutation, including those receiving CFTR modulators. In Addition, the company is focused on additional programs – SPL5AC for muco-obstructive diseases (Chronic Obstructive Pulmonary Disease (COPD), asthma, Non-Cystic Fibrosis Bronchiectasis (NCFB) and CF) and SPL5B for Idiopathic Pulmonary Fibrosis (IPF) – with first-in-human expected to begin in 2026," added Dr. Hart.

The e-PRIME designation builds also on the positive results previously reported from the Phase 2 (SPL84-002) study. As announced in September 2025, evaluation of the first two cohorts demonstrated a favorable safety profile, with no treatment-related serious adverse events observed, and a clinically significant improvement in lung function (ppFEV1) in up to 70% of SPL84-treated participants. The estimated mean absolute change in ppFEV1 compared with placebo was 10. These data represent the first evidence of potential clinical benefit for an inhaled antisense oligonucleotide (ASO) therapy in a pulmonary disease.

About PRIME designation

PRIME is granted to medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. The designation provides early and enhanced scientific and regulatory support from the EMA, with the goal of accelerating development and review timelines.

About SPL84

SPL84 is an inhaled antisense oligonucleotide designed to correct the splicing defect caused by the 3849+10Kb C>T mutation in CFTR, enabling the production of functional CFTR protein in the lungs. The therapy is administered weekly by inhalation to directly target the primary site of disease pathology. In addition to e-PRIME designation from the EMA, SPL84 has previously received Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration (FDA). The clinical validation of SPL84 further supports SpliSense's broader RNA-based pulmonary platform, which includes SPL5AC for muco-obstructive diseases and SPL5B for idiopathic pulmonary fibrosis, both of which are advancing toward clinical development in 2026.

About SpliSense

SpliSense is a clinical stage company focused on the development of RNA-based treatments (ASOs) for pulmonary diseases. The Company's pioneering ASO platform is designed to target the root cause of pulmonary disease by restoring or reducing protein function. SpliSense is advancing a pipeline of programs addressing pulmonary indications, including muco-obstructive diseases (COPD, asthma, NCFB) and idiopathic pulmonary fibrosis (IPF) on top of CF. For more information, please visit: www.splisense.com

Company Contact:
Tsipi Haitovsky
Global Media Liaison
+972-52-5989-892
[email protected] 

SOURCE Splisense