Sionna Therapeutics Announces Advancement of Cystic Fibrosis Pipeline Programs and Presentation at J.P. Morgan 41st Annual Healthcare Conference

- Enrolling Phase 1 study of lead NBD1 modulator, SION-638, following IND clearance from the FDA; data expected in the second half of 2023 -

- SION-109, targeting complementary mechanism ICL4, on-track for IND submission in the first half of 2023 -

- Three additional development candidates nominated for progression including two Series 2 NBD1 modulators -

BOSTON, Jan. 5, 2023 /PRNewswire/ -- Sionna Therapeutics, a life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF), today announced advancement of the company's preclinical and clinical development programs for novel small molecules designed to target the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein and complementary mechanisms. Mike Cloonan, President and Chief Executive Officer of Sionna, will provide an overview of the Company's progress and pipeline updates at the J.P. Morgan 41^st Annual Healthcare Conference on January 10, 2023, at 8:00 AM PST in San Francisco.

"We formed Sionna to deliver therapies that could be transformational for people living with CF, and data continue to reinforce the opportunity to improve CFTR function for modulator-eligible patients," said Mr. Cloonan. "Modulators that provide normal CFTR function could have a significant impact on clinical outcomes for people with CF. We are progressing with our lead candidate and multiple Series 2 modulators targeting NBD1, as well as complementary mechanisms, to target multiple domains of CFTR. This strategy will support future development of proprietary combination treatments with the potential to achieve full CFTR correction."

As previously announced, the Investigational New Drug application (IND) for SION-638, the company's lead novel small molecule targeting the NBD1 domain, has cleared. The first subjects have been dosed in a Phase 1 study to evaluate the safety and pharmacokinetics of SION-638. Sionna expects data from this study in the second half of 2023. The company is also advancing the development of compounds targeting complementary mechanisms and plans to file an IND for SION-109, targeting NBD1's interface with the intracellular loop 4 (ICL4) region, in the first half of 2023.

In addition, Sionna announced today the nomination of three compounds as development candidates (DCs). Two compounds target the NBD1 domain, SION-719 and SION-451, and one compound, SION-676, targets the transmembrane domain 1 (TMD1) of CFTR. Both NBD1 directed compounds come from the company's second Series of NBD1 directed modulators and have demonstrated high potency with the potential to achieve full CFTR correction when combined with a complementary modulator targeting either ICL4 or TMD1. The DC nominations are supported by in vitro results in the cystic fibrosis human bronchial epithelial cell (CFHBE) assay and robust preclinical experiments including 14-day exploratory safety studies in two species.

CF is caused by genetic mutations in the CFTR protein, 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, ΔF508, causes NBD1 to unfold at body temperature and severely impairs CFTR function. CF is a serious, potentially fatal genetic disease that can have a significant and often severe impact on health and life expectancy. There are over 100,000 people living with CF around the world. It is estimated that 90 percent of people living with CF have the genetic mutation ΔF508.

Sionna Therapeutics is a life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF) by normalizing the function of CFTR, the key protein associated with disease progression in CF. Building on over a decade of extensive research on the genetic mutations associated with CF and founded in 2019, Sionna is advancing a pipeline of small molecules engineered to correct the protein defects caused by ΔF508, the most common mutation that affects the CFTR protein. The company has a first-in-class portfolio of programs targeting correction of NBD1, the key and unique mechanism to enable full restoration of ΔF508-CFTR function, and complementary programs targeting ICL4 and TMD1. Sionna's pipeline has the potential to deliver best-in-class efficacy and reach previously unachievable levels of long-term benefit for people with CF. For information about Sionna visit https://www.sionnatx.com/.

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SOURCE Sionna Therapeutics