SpliSense Granted EMA EARLY PRIME Designation for SPL84 in Cystic Fibrosis

This is a paid press release. Contact the press release distributor directly with any inquiries. SpliSense Granted EMA EARLY PRIME Designation for SPL84 in Cystic Fibrosis PR Newswire March 3, 2026 3 min read 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. Story Continues 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] Cision View original content: https://www.prnewswire.com/news-releases/splisense-granted-ema-early-prime-designation-for-spl84-in-cystic-fibrosis-302702182.html Terms and Privacy Policy Privacy & Cookie Settings More Info BTC-USD Bitcoin USD 84,928.86 +688.00 (+0.82%) VST Vistra Corp. 140.02 +0.27 (+0.19%) ETH-USD Ethereum USD 2,684.18 +19.77 (+0.74%) XRP-USD XRP USD 1.49 +0.03 (+1.73%) LEN Lennar Corporation 79.81 -2.30 (-2.80%) ADVERTISEMENT Portfolios Sign in to access your portfolio Sign in MXL MaxLinear, Inc. 105.93 +13.81 (+14.99%) IMOS ChipMOS TECHNOLOGIES INC. 83.30 +10.62 (+14.61%) SYNA Synaptics Incorporated 121.10 +14.95 (+14.08%) PENG Penguin Solutions, Inc. 61.36 +6.38 (+11.60%) VSH Vishay Intertechnology, Inc. 38.18 +3.91 (+11.41%) MAAS Maase Inc. 13.70 -2.11 (-13.35%) WDC Western Digital Corporation 415.29 -47.27 (-10.22%) STX Seagate Technology Holdings plc 848.99 -96.58 (-10.21%) UCTT Ultra Clean Holdings, Inc. 72.69 -5.95 (-7.57%) GDS GDS Holdings Limited 27.57 -2.23 (-7.48%) NKE NIKE, Inc. 33.87 -1.28 (-3.64%) SPCX Space Exploration Technologies Corp. 158.96 +10.89 (+7.35%) NVDA NVIDIA Corporation 233.95 +3.09 (+1.34%) INTC Intel Corporation 119.33 -0.67 (-0.56%) NU Nu Holdings Ltd. 13.43 +0.14 (+1.05%) Top economic events United Kingdom There are no important events for this country at this time. Select "All" to see top events in other countries or view all events. View All Events Edit your Dock Terms and Privacy Policy Privacy & Cookie Settings Ad Terms Feedback More Info

The release as fetched from its publisher. splisense.com ↗