SpliSense Receives EMA e-PRIME Designation for SPL84 Antisense Therapy in Cystic Fibrosis
核心洞察
SpliSense (搜索)'s inhaled antisense oligonucleotide SPL84 received early Priority Medicines (e-PRIME) designation from the European Medicines Agency (搜索) for treating cystic fibrosis (搜索) patients with the 3849+10Kb C>T mutation.
Phase 2 study results demonstrated clinically significant lung function improvements in up to 70% of SPL84-treated participants, with an estimated mean absolute change in ppFEV1 of 10 compared to placebo.
The company is advancing SPL84 to a global Phase 2b study while developing additional RNA-based therapies for pulmonary diseases including COPD (搜索), asthma (搜索), and idiopathic pulmonary fibrosis (搜索).
SpliSense (搜索), a clinical-stage biotechnology company developing RNA-based therapies for pulmonary diseases, announced that the European Medicines Agency (搜索) (EMA) has granted early Priority Medicines (e-PRIME) designation to SPL84 for treating cystic fibrosis (搜索) in patients carrying the 3849+10Kb C>T mutation in the CFTR (搜索) 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. The CHMP noted that despite the availability of CFTR modulators (搜索), 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.
Promising Phase 2 Results Drive Regulatory Recognition
The designation builds on positive results from the Phase 2 (SPL84-002) study reported 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, marking a significant milestone for the field.
"This PRIME designation represents a major regulatory milestone for SpliSense (搜索) 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 (搜索) who continue to represent a significant unmet medical need."
Advancing to Phase 2b and Expanding Pipeline
SpliSense (搜索) is currently advancing SPL84 to a global Phase 2b study in the US and EU designed to further evaluate efficacy and safety in patients with the 3849+10Kb C>T mutation, including those receiving CFTR modulators (搜索). The company is also focused on additional programs including SPL5AC for muco-obstructive diseases (Chronic Obstructive Pulmonary Disease, asthma (搜索), Non-Cystic Fibrosis Bronchiectasis (搜索) and CF) and SPL5B for Idiopathic Pulmonary Fibrosis (搜索), with first-in-human studies expected to begin in 2026.
Novel Mechanism of Action
SPL84 is an inhaled antisense oligonucleotide designed to correct the splicing defect caused by the 3849+10Kb C>T mutation in CFTR (搜索), 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 (搜索) (FDA). The clinical validation of SPL84 further supports SpliSense (搜索)'s broader RNA-based pulmonary platform.
Regulatory Pathway Acceleration
PRIME designation 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.
SpliSense (搜索)'s pioneering ASO platform is designed to target the root cause of pulmonary disease by restoring or reducing protein function, addressing multiple pulmonary indications including muco-obstructive diseases and idiopathic pulmonary fibrosis (搜索) beyond cystic fibrosis (搜索).