SpliSense Secures Up to $13 Million from Cystic Fibrosis Foundation to Advance Phase 2b Study of Inhaled ASO Therapy SPL84
核心洞察
SpliSense (搜索) entered a funding agreement with the Cystic Fibrosis Foundation for up to $13 million to support the Phase 2b clinical development of SPL84, an inhaled antisense oligonucleotide therapy for cystic fibrosis (搜索).
The funding follows positive Phase 2a results showing lung function improvement in up to 70% of treated participants and an estimated mean absolute improvement of 10 percentage points in ppFEV1 versus placebo.
The ongoing randomized, placebo-controlled Phase 2b study will enroll approximately 40 participants across the U.S., Europe, and Israel, with topline results expected in H2 2027.
SpliSense (搜索), a clinical-stage biotechnology company based in Jerusalem, 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, the company's lead inhaled antisense oligonucleotide (ASO) therapy for cystic fibrosis (搜索) (CF). The announcement, made on June 16, 2026, follows positive Phase 2a clinical results that demonstrated 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 (搜索) who carry the 3849+10kb C→T mutation," said Gili Hart, Ph.D., Chief Executive Officer of SpliSense (搜索). "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."
Phase 2a Results Validate Inhaled ASO Platform
The Foundation's investment was catalyzed by compelling Phase 2a clinical data for SPL84, which demonstrated favorable safety and encouraging efficacy. Notably, improvement in lung function was observed in up to 70% of treated participants, with an estimated mean absolute improvement of 10 percentage points in ppFEV1 versus placebo. These results represent the first-ever clinical proof-of-concept for an inhaled ASO therapy in a lung disease, marking a significant milestone for both SpliSense (搜索) and the broader field of RNA-based pulmonary therapeutics.
Dr. Hart emphasized that the 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."
Ongoing Phase 2b Study Design
The ongoing Phase 2b study (NCT06429176) is a randomized, placebo-controlled trial designed to evaluate the safety, tolerability, and efficacy of SPL84 in people with cystic fibrosis (搜索) carrying the 3849+10kb C→T mutation who are receiving standard-of-care CFTR (搜索) modulators. The study is expected to enroll approximately 40 participants across sites in the United States, Europe, and Israel. Topline results are anticipated in the second half of 2027.
Mechanism of Action and Regulatory Designations
SPL84 is an inhaled antisense oligonucleotide designed to correct the splicing defect caused by the 3849+10kb C→T mutation in the CFTR (搜索) gene. 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.
The 3849+10kb C→T mutation represents a clinically meaningful CF population with continued unmet need despite available CFTR (搜索) modulator therapies. While the past decade has seen dramatic changes in CF care with the approval of new CFTR modulators, these therapies do not support all people with CF and do not offer a cure. New therapeutic strategies remain essential for partially responsive and non-responsive individuals.
SPL84 has received Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration and was recently granted EMA PRIME designation, underscoring the regulatory recognition of the unmet need in this patient population.
Broader Pipeline Implications
The positive Phase 2 results and initiation of the Phase 2b program further validate SpliSense (搜索)'s proprietary inhaled ASO platform. The company is advancing a broader pipeline that includes SPL5AC for muco-obstructive diseases such as chronic obstructive pulmonary disease (搜索) (COPD), non-cystic fibrosis bronchiectasis (搜索) (NCFB), asthma (搜索), and cystic fibrosis (搜索), as well as SPL5B for idiopathic pulmonary fibrosis (搜索) (IPF).
The Cystic Fibrosis Foundation operates under a "philanthropic venture capital" model, investing directly in companies developing therapies in its field. This approach has precedent in the CF space, including support for the development of Kalydeco (ivacaftor) from Vertex, which received significant funding from the CF Foundation during its development and went on to reach peak annual revenues of $500 million.