Therapy Areas: Hereditary Disorders
SpliSense completes SPL84 first-in-human, Phase one clinical trial for cystic fibrosis treatment
7 September 2023 -

SpliSense, an Israel-based biopharmaceutical company, announced on Wednesday that it has completed a first-in-human, Phase one clinical trial of SPL84, the company's lead inhaled anti sense oligonucleotide (ASO) product intended for the treatment of patients with cystic fibrosis (CF) carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the transmembrane conductance regulator (CFTR) gene.

During the Phase one study, 32 healthy male volunteers were administered with a single dose of the product or placebo and four doses were tested: 20, 40, 80, and 160 mg (active: placebo 3:1). SPL84 was shown to be safe and well tolerated, and no serious adverse events or significant related adverse events were identified. There were no significant changes from baseline in vital signs, clinical laboratory values, ECG, physical examination, or pulmonary function. Systemic exposure to SPL84 was very low and dose dependent. The first-in-human Phase 1 placebo-controlled, double–blind, randomised study evaluating the safety, tolerability, and pharmacokinetics of SPL84 was a single ascending dose study in healthy volunteers, administered via inhalation.



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