SpliSense Receives $13 Million to Propel SPL84 Clinical Trials for Cystic Fibrosis Treatment

SpliSense Secures $13 Million Funding for SPL84 Trials

SpliSense, a pioneering biotechnology firm specialized in RNA-based therapies for pulmonary diseases, is set to advance its promising investigational drug, SPL84, thanks to a significant funding agreement with the Cystic Fibrosis Foundation. Under this new arrangement, SpliSense will receive up to $13 million, aimed at furthering the Phase 2b clinical trials crucial for addressing cystic fibrosis (CF).

The recent investment comes on the heels of successful Phase 2a clinical outcomes that showcased not only favorable safety profiles but also significant efficacy improvements in lung function. Remarkably, the study revealed a 10 percentage point improvement in lung function (ppFEV1) in nearly 70% of patients compared to those on a placebo, marking a historical breakthrough as the first clinical proof-of-concept for an inhaled antisense oligonucleotide therapy in a respiratory condition.

Dr. Gili Hart, CEO of SpliSense, expressed gratitude for the funding from the Cystic Fibrosis Foundation, noting, "This investment underscores the strength of our Phase 2 clinical data and highlights the potential of SPL84 to transform treatment options for cystic fibrosis patients, especially those with the 3849+10kb C→T mutation, which is not adequately addressed by existing therapies."

The SPL84 Phase 2b Clinical Study

The ongoing Phase 2b study is structured to evaluate the safety, tolerability, and efficacy of SPL84 in a broader patient cohort, specifically targeting individuals who carry the 3849+10kb C→T mutation while undergoing standard care with CFTR modulators. This randomized, placebo-controlled trial is expected to include approximately 40 participants across various sites in the United States, Europe, and Israel, with topline results projected for the second half of 2027. This strategic initiative aims to enhance the therapeutic landscape for cystic fibrosis, a life-threatening genetic disorder that has historically been challenging to treat.

The promising results from the previous Phase 2 trials solidify SpliSense’s commitment to its proprietary inhaled ASO platform, driving momentum not only for SPL84 but also for other pipeline therapies. This includes SPL5AC, which targets muco-obstructive diseases like chronic obstructive pulmonary disease (COPD), asthma, and non-cystic fibrosis bronchiectasis; as well as SPL5B for idiopathic pulmonary fibrosis (IPF).

Understanding SpliSense and Cystic Fibrosis

Founded with the mission to revolutionize pulmonary disease treatment, SpliSense emphasizes the importance of directly addressing root causes through innovative RNA-based therapies. Their lead candidate, SPL84, aims to correct the underlying splicing defect associated with the 3849+10kb C→T mutation in the CFTR gene, which has represented a substantial unmet medical need in the CF patient population.

The CFTR gene is critical for producing the CFTR protein, a chloride channel pivotal for maintaining fluid balance in various tissues, including the lungs. While recent breakthroughs in CF care, such as new CFTR modulators, have positively impacted many patients, there remains a significant population that either does not respond to these therapies or continues to struggle with the disease. As SpliSense pushes forward with SPL84, it brings renewed hope to these patients and the CF community at large.

As advancements in clinical trials signal potential progress, stakeholders and observers in the biotechnology field will keenly watch how SPL84 performs in upcoming phases. SpliSense's strategic partnership with the Cystic Fibrosis Foundation not only highlights the potential of innovative therapies but also showcases a shared commitment to improving the lives of those affected by this challenging disease.

For more details about SpliSense and its transformative therapies, visit www.splisense.com.