SpliSense Ltd.

Cystic Fibrosis Foundation funding follows positive SPL84 Phase 2a clinical results demonstrating the first-ever clinical proof-of-concept for an inhaled antisense oligonucleotide therapy in a lung disease Funding supports the ongoing global Phase 2b study evaluating SPL84 in people with cystic fibrosis carrying the 3849+10kb C→T mutation who are receiving standard-of-care CFTR modulators; topline results expected in H2 2027 JERUSALEM, June 16, 2026 /PRNewswire/ -- SpliSense, a clinical-stage biotechnology company developing transformative RNA-based therapies for pulmonary diseases, today announced that it has entered into a funding agreement with the Cystic Fibrosis Foundation, under which the Foundation will provide up to $13 million to support the continued clinical development of SPL84, SpliSense's lead inhaled antisense oligonucleotide (ASO) therapy for cystic fibrosis (CF). The funding follows SpliSense's positive Phase 2a clinical results for SPL84, which demonstrated favorable safety and encouraging efficacy, including improvement in lung function in up to 70% of treated participants and an estimated mean absolute improvement of 10 percentage points in ppFEV1 versus placebo. These results represented the first-ever clinical proof-of-concept for an inhaled ASO therapy in a pulmonary disease. "SPL84 has the potential to address a significant medical need for people living with cystic fibrosis who carry the 3849+10kb C→T mutation," said Gili Hart, Ph.D., Chief Executive Officer of SpliSense. "We are honored to receive this investment from the Cystic Fibrosis Foundation, one of the world's leading organizations advancing innovative therapies for people with CF. We believe this commitment reflects both the strength of our Phase 2 clinical data and the potential of SPL84 to become a transformative treatment option for patients, paving the way also for earlier candidates in our pipeline developed for additional lung diseases to advance into the clinic." Dr. Hart continued, "We appreciate the Foundation's long-standing support of SpliSense. Their continued support of SPL84 and our RNA-based platform enables us to accelerate development toward later-stage clinical studies and potential registration." The ongoing Phase 2b study is designed to evaluate the safety, tolerability, and efficacy of SPL84 in people with cystic fibrosis carrying the 3849+10kb C→T mutation who are receiving standard-of-care CFTR modulators. The randomized, placebo-controlled study is expected to enroll approximately 40 participants across sites in the United States, Europe and Israel. Topline results are anticipated in H2 2027 (NCT06429176). The positive Phase 2 results and the initiation of the Phase 2b program further validate SpliSense's proprietary inhaled ASO platform and support advancement of the Company's broader pipeline, including SPL5AC for muco-obstructive diseases such as chronic obstructive pulmonary disease (COPD), non-cystic fibrosis bronchiectasis (NCFB), asthma and cystic fibrosis, as well as SPL5B for idiopathic pulmonary fibrosis (IPF). About SPL84 SPL84 is an inhaled antisense oligonucleotide designed to correct the splicing defect caused by the 3849+10kb C→T mutation in the CFTR gene. The 3849+10kb C→T mutation represents a clinically meaningful CF population with continued unmet need despite available CFTR modulator therapies. By targeting the mutant CFTR RNA, SPL84 promotes production of functional CFTR protein. The therapy is administered directly to the lungs through inhalation, enabling targeted delivery to the primary site of disease. SPL84 has received Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration and was recently granted EMA PRIME designation as well. About Cystic Fibrosis (CF) CF is a genetic multisystem disorder that originates from various mutations in the CFTR gene, which is responsible for the production of the CFTR protein, a chloride channel expressed in the lungs as well as in other tissues. The past decade has seen a dramatic change in CF care with the approval of new CFTR modulators. However, approved CFTR modulators do not support all people with CF and do not offer a cure for the disease. Thus, new strategies of therapeutic development are essential to address partially responsive and non-responsive people with CF, specifically those carrying the 3849+10 Kb C->T splicing mutation. About SpliSense SpliSense is a clinical-stage biotechnology company developing ASO-based therapies for pulmonary diseases. The Company's proprietary inhaled ASO platform is designed to address the root causes of severe pulmonary disorders through modulation of gene expression and RNA processing. SpliSense is advancing a pipeline of programs in cystic fibrosis, muco-obstructive lung diseases (COPD, Asthma and NCFB) and idiopathic pulmonary fibrosis. For additional information, please visit . Company Contact: Tsipi Haitovsky Global Media Liaison +972-52-5989-892 [email protected] SOURCE SpliSense 21% more press release views with Request a Demo

Israel-based SpliSense announced a funding agreement with the Cystic Fibrosis Foundation under which the Foundation will provide up to USD 13 million to advance the Phase IIb clinical development of SPL84, its inhaled antisense oligonucleotide therapy for cystic fibrosis. The capital is directed at a patient subpopulation carrying the 3849+10kb C→T mutation who continue to have unmet need despite access to approved CFTR modulators — a group for which no targeted therapy currently exists. The funding follows positive Phase II results reported in September 2025 showing lung function improvement in up to 70% of SPL84-treated participants and an estimated mean absolute improvement of 10 percentage points in ppFEV1 versus placebo. The agreement is structured as a philanthropic funding arrangement with The Cystic Fibrosis Foundation as the sole funder, with no dilutive securities, interest obligations, or repayment terms disclosed. The Foundation has a prior funding relationship with SpliSense, having previously provided up to USD 8.5 million to support the Phase II initiation of SPL84 in April 2024, making this its fourth known investment in the program. SpliSense is a clinical-stage biotechnology company headquartered in Jerusalem developing antisense oligonucleotide-based therapies for pulmonary diseases. Its proprietary inhaled ASO platform is designed to modulate RNA processing and gene expression directly in the lungs via inhalation, enabling targeted delivery to the primary site of disease. Its lead asset, SPL84, is an inhaled ASO designed to correct the splicing defect caused by the 3849+10kb C→T mutation in the CFTR gene. By binding to mutant CFTR RNA, SPL84 promotes production of functional CFTR protein. The therapy is administered weekly by inhalation. SPL84 holds FDA Fast Track and Orphan Drug designations, and in March 2026 received EMA Early PRIME designation, recognizing remaining unmet need and supportive clinical data in this mutation subgroup. The ongoing Phase IIb study is a randomized, placebo-controlled trial evaluating the safety, tolerability, and efficacy of SPL84 in patients carrying the 3849+10kb C→T mutation who are receiving standard-of-care CFTR modulators. The study is expected to enroll approximately 40 participants across sites in the US, Europe, and Israel, with topline results anticipated in H2 2027. Management has explicitly linked the current CFF funding to supporting this trial through to data readout. Beyond SPL84, SpliSense is advancing two earlier-stage programs: SPL5AC, targeting MUC5AC expression for muco-obstructive diseases including COPD, non-cystic fibrosis bronchiectasis, asthma, and CF; and SPL5B, targeting MUC5B expression for idiopathic pulmonary fibrosis. Both are expected to enter the clinic, with first-in-human studies previously guided for 2026. The company’s investors include OrbiMed, Israel Biotech Fund, Integra Holdings, and Biotel Limited, alongside the Cystic Fibrosis Foundation. The 3849+10kb C→T mutation represents a clinically meaningful but numerically smaller CF subpopulation. While approved CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (Trikafta) have substantially improved outcomes for patients with the F508del mutation — and Vertex Pharmaceuticals’ vanzacaftor/tezacaftor/deutivacaftor (Alyftrek) received EU authorization in June 2025 for patients with at least one non-Class I mutation — patients carrying splicing mutations such as 3849+10kb C→T may have limited or partial responsiveness to modulator therapy. SPL84’s mechanism operates at the RNA level to restore CFTR protein production rather than potentiating or correcting a defective protein, positioning it as a potential complement to or alternative for modulator-ineligible or partially responsive patients in this genotype. The competitive landscape for mutation-specific CF therapies beyond the dominant F508del population remains sparse. Gene therapy approaches, including Krystal Biotech’s KB407 and 4D Molecular Therapeutics’ 4D-710, are pursuing mutation-agnostic strategies via inhaled viral vectors, but both remain in Phase I/II development with no approved products. SpliSense’s ASO approach is mutation-specific and targets a defined splicing defect, which differentiates it mechanistically but also limits its addressable patient population. The CFF’s continued and deepening financial commitment to SpliSense — now totaling over USD 21 million across multiple tranches — reflects the Foundation’s established model of providing non-dilutive capital to advance programs targeting underserved CF subpopulations. The Foundation has previously funded programs at Krystal Biotech, 4D Molecular Therapeutics, and others using similar grant-style agreements. For SpliSense, which remains privately held with no disclosed public financing plans, CFF funding represents the primary mechanism for advancing SPL84 through late-stage clinical development without requiring additional equity dilution ahead of potential registration-enabling data in H2 2027. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. 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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: Company Contact: Tsipi Haitovsky Global Media Liaison +972-52-5989-892 [email protected] SOURCE Splisense 21% more press release views with Request a Demo