Company: SION
Filing Date: 2025-01-17
Form Type: S-1
Source: 0001193125-25-008474
Chunk: 162

Company: Sionna Therapeutics, Inc.
Filing Date: 2025-01-17
Form: S-1
Chunk 162
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summary of product characteristics for Kaftrio, the brand name for Trikafta in Europe. Patients who discontinue use of Trikafta or experience tolerability challenges have limited or no alternative treatments available to improve their clinical
outcomes or quality of life. Currently, the alternatives for these patients are limited to less efficacious combination products that include one or more components of Trikafta, or Alyftrek, which demonstrated non-inferiority to Trikafta in the
primary endpoint of two Phase 3 clinical trials, providing patients with similar FEV as Trikafta and sweat chloride level improvements of 3 to 8 mmol/L. Approximately 69% of Alyftrek patients
in two Phase 3 clinical trials did not achieve normal CFTR function, defined as sweat chloride levels below 30 mmol/L. Our research with key opinion leaders has indicated the desire for more treatment options for CF patients, support for a new
mechanism of action that could provide clinically meaningful benefit for people living with CF, and need for an alternative for those patients who experience tolerability issues on Trikafta. We aim to expand the current treatment paradigm through a
proprietary dual combination or as an add-on to the standard of care.

Worldwide revenue for approved CFTR
modulators was approximately $10 billion in 2023, and it is expected to grow to $15 billion by 2029. Vertex, which markets all five of the currently approved CFTR modulators, reported revenues in 2023 from global sales of its then-approved
CFTR modulators of approximately $9.9 billion, more than $8.9 billion of which the company attributed to Trikafta. Vertex’s approved CFTR modulators target approximately 92,000 CF patients in North America, Europe and Australia, and
more than 20% of eligible patients are currently not on CFTR modulators. CF screening of newborn infants has served to identify CF patients as early as possible in their lives. For example, newborn screening for CF has been required in the U.S.
since 2010, and in 2021, 64.4% of newly diagnosed people with CF in the U.S. were identified by newborn screening, based on CFF registry data. The availability of CFTR modulators has also increased the use of genetic testing to determine eligibility
for treatment.

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Current Standard of Care and its Limitations

The approved CFTR modulators are oral small