The trials will evaluate SION-719 and SION-451, two novel NBD1 stabilizers aimed at improving CFTR protein function in cystic fibrosis patients.
RT’s Three Key Takeaways:
- Novel Approach: The phase 1 trials are evaluating two new NBD1 stabilizers, SION-719 and SION-451, which are designed to improve CFTR protein function in cystic fibrosis patients.
- Focus on Safety and Efficacy: The trials will assess the safety, tolerability, and pharmacokinetics of these treatments in healthy volunteers, with the goal of determining their potential for advancing to further clinical development.
- Stabilizing CFTR Protein: Preclinical data shows the NBD1 stabilizers, when combined with complementary modulators, significantly improve in vitro CFTR protein activity and demonstrate the potential to achieve superior clinical outcomes.
Sionna Therapeutics, a clinical-stage life sciences company developing treatments for cystic fibrosis (CF), announced that the first subjects have been dosed in two separate phase 1 clinical trials evaluating the safety, tolerability, and pharmacokinetics of SION-719 and SION-451 in healthy volunteers.
SION-719 and SION-451 are product candidates from the company’s next-generation series of highly potent nucleotide-binding domain 1 (NBD1) stabilizers. The trials are being conducted in Australia under the Clinical Trial Notification process.
“We are pleased with the company’s strong execution and the progress these first-in-class programs have made in entering the clinic,” says Mike Cloonan, president and chief executive officer of Sionna, in a release. “We have a novel ability to target NBD1, which we believe could be the key to raising the efficacy bar and unlocking improved clinical benefits for CF patients. We expect that the data from these single and multiple ascending dose trials will inform our decision about a lead NBD1 stabilizer to bring forward into the next stages of clinical development.”
NBD1 Stabilizers Show Promise
CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which codes for an epithelial ion channel that is essential for producing healthy, freely flowing mucus in the airways, digestive system, and other organs. The most common mutation in CFTR, F508del, causes NBD1 to unfold at body temperature and severely impairs CFTR function.
Sionna has presented preclinical data, including data from the clinically predictive human bronchial epithelial cell model, that show its NBD1 stabilizers, SION-719 and SION-451, when combined with complementary modulators, significantly improve in vitro CFTR protein activity and demonstrate the potential to achieve superior clinical outcomes.
Sionna is developing the first-ever clinical-stage NBD1 stabilizers and is also developing complementary modulators with the goal of advancing differentiated combination therapies for CF.
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