SpliSense’s SPL84 Receives EMA Early PRIME Designation to Treat Cystic Fibrosis

SpliSense’s SPL84 Granted EMA Early PRIME Designation

In a significant advancement for cystic fibrosis (CF) treatment, SpliSense, a clinical-stage biotechnology company, has announced that the European Medicines Agency (EMA) has awarded its investigational product SPL84 an Early Priority Medicines (e-PRIME) designation. This milestone, announced on March 3, 2026, is aimed at addressing the needs of patients suffering from CF due to the 3849+10Kb C>T mutation in the CFTR gene.

SpliSense focuses on innovative RNA-based therapies targeting pulmonary diseases. The e-PRIME designation underscores the recognition from the EMA of the substantial unmet medical needs for patients with this specific genetic mutation. The Committee for Medicinal Products for Human Use (CHMP) noted that, despite the existing CFTR modulators, there remains a demand for more effective therapies—whether used alone or alongside current treatments.

Importance of e-PRIME Designation

This designation not only reflects the importance of SPL84 in the therapeutic landscape for CF but also underscores the potential of this inhaled RNA therapy to significantly enhance treatment outcomes. As Gili Hart, CEO of SpliSense, articulated, this designation marks a notable regulatory achievement for the company and validates the strengths of their mechanistic data from both non-clinical and emerging clinical studies.

Advancements Towards Phase 2b Study

Currently, SpliSense is progressing towards a global Phase 2b clinical trial for SPL84, which will take place in the U.S. and EU. This study aims to assess both the efficacy and safety of SPL84 in patients with the specified mutation—including those who are already on CFTR modulator treatments. Alongside SPL84, SpliSense is developing additional programs targeting other pulmonary conditions, such as SPL5AC for muco-obstructive diseases like Chronic Obstructive Pulmonary Disease (COPD), asthma, and Idiopathic Pulmonary Fibrosis (IPF).

Positive Results from Previous Studies

SpliSense's commitment to advancing SPL84 is grounded in encouraging results reported from previous studies. In September 2025, outcomes from the Phase 2 (SPL84-002) study showed a promising safety profile with no serious adverse events linked to the treatment, while revealing a clinically notable enhancement in lung function for up to 70% of patients receiving SPL84. The mean absolute improvement in lung function was recorded at 10 compared to a placebo. These findings signify a groundbreaking development in the application of inhaled antisense oligonucleotide therapy for pulmonary ailments.

Understanding the PRIME Designation

The PRIME designation is awarded to therapies indicating a major therapeutic advantage over existing treatments or providing solutions for patients lacking effective treatment options. This designation not only fast-tracks scientific and regulatory support from the EMA but also aims to expedite the overall development journey for products like SPL84, enhancing its potential impact on patient care.

The Mechanism of Action of SPL84

Specifically engineered to mend the splicing defect caused by the 3849+10Kb C>T mutation, SPL84 acts as an inhaled antisense oligonucleotide. This therapy directly addresses the fundamental pathology by facilitating the production of functional CFTR protein in the lungs. Administered weekly, it targets the very site where the disease manifests, ideally improving the patient's condition significantly.

Future Prospects and SpliSense's Broader Goals

In addition to the e-PRIME designation, SPL84 has also earned Orphan Drug and Fast Track recognition from the U.S. FDA. These endorsements support SpliSense's larger vision of utilizing its RNA-based platform not only for cystic fibrosis but also for various other pulmonary indications. As the company gears up for first-in-human trials for SPL5AC and SPL5B, set to kick off in 2026, it is evident that SpliSense is committed to innovating solutions that address critical gaps in pulmonary disease management.

To learn more about SpliSense and its groundbreaking therapies, visit SpliSense.