Company: SION
Filing Date: 2025-03-20
Form Type: 10-K
Source: 0002036042-25-000005
Chunk: 4

Company: Sionna Therapeutics, Inc.
Filing Date: 2025-03-20
Form: 10-K
Item: Item 1
Chunk 4
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 clinical trial in healthy volunteers. SION-109 was generally well tolerated at all dose levels administered in all parts of this Phase 1 trial. The target exposure for SION-109 as part of a dual combination with SION-451 or SION-719 was achieved with single and multiple doses.

•Navocaftor (SION-3067), a potentiator, has been evaluated in Phase 2 trials, where it demonstrated potential as a combination therapy.

Figure 3 below captures the targeted binding locations within the CFTR structure for our pipeline of NBD1 stabilizers and complementary modulators.

Figure 3. Our Multi-Prong Approach to Potentially Improving CFTR Function

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Our current portfolio of programs is summarized in Figure 4 below:

Figure 4. Our Proprietary Pipeline of Product Candidates for the Treatment of CF

Clinical trials for galicaftor, SION-2851, and navocaftor were conducted by AbbVie or Galapagos NV.

Our Strategy

Our mission is to revolutionize the current treatment paradigm for CF patients by developing novel medicines that normalize the function of the CFTR protein to deliver clinically meaningful benefit to CF patients. The key pillars of our strategy are:

•Advance our novel NBD1 stabilizers. Topline data from our Phase 1 clinical trials of SION-719 and SION-451 are expected in the first half of 2025. After completion of a drug-drug interaction trial, we plan to initiate a Phase 2a proof-of-concept trial in the second half of 2025 to evaluate an NBD1 stabilizer in combination with the current standard of care in CF patients, as well as combination MAD trial(s) evaluating dual combinations of an NBD1 stabilizer with galifactor (SION-2222) and/or SION-109 in healthy subjects. In preclinical studies using the CFHBE model, the combination of SION-719 or SION-451 with one of our complementary modulators or ETI showed increased chloride transport, which indicates improvement in in vitro CFTR activity. Given the clinically predictive nature of the CFHBE model, we believe these data indicate the potential for an NBD1-anchored dual combination to improve CFTR activity, which we believe will deliver clinically meaningful benefit to CF patients. 

•Develop and advance our pipeline of complementary modulators for proprietary combination product development. We are developing a proprietary pipeline of complementary CFTR modulators designed to work synergistically