Vertex Pharmaceuticals Incorporated announced the presentation of multiple abstracts demonstrating the clinical benefits of treatment with CFTR modulators, including the Company’s most recently approved medicine, ALYFTREK, at the North American Cystic Fibrosis Conference held from October 22-25 in Seattle, Washington.

Several of the abstracts that will be presented at this year’s conference demonstrate that improvement in CFTR function, as measured by reduction in sweat chloride (SwCl), is associated with improved outcomes in people with cystic fibrosis (CF). ALYFTREK has shown greater reductions in sweat chloride compared to TRIKAFTA in Phase 3 trials, suggesting that ALYFTREK has the potential for even greater improvements in quality of life and other health-related outcomes.

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Data being presented on ALYFTREK and on correlation between lower SwCl and improved clinical outcomes with CFTR modulator treatment:

  • “Decreased Pulmonary Exacerbations and Lower IV Antibiotic Usage Following Vanzacaftor/Tezacaftor/Deutivacaftor Treatment in People with CF with F508del-Minimal Function Genotypes”: Post-hoc analyses of two pivotal Phase 3 studies (SKYLINE 102 and SKYLINE 103) will be presented, showing that F580del/minimal function mutation(F/MF)subjects treated with ALYFTREK had lower rates of pulmonary exacerbations (PEx), PEx requiring hospitalization or IV antibiotics, and IV antibiotic use vs. F/MF subjects treated with TRIKAFTA. Friday, October 24,1:15–2:15 p.m. PDT.
  • “Maximizing benefits with early CFTR modulator treatment: Lower sweat chloride is associated with improved clinical outcomes in children aged 6 to 11 years”: Data will be presented from a pooled analysis of data from participants in Phase 3 studies and open-label extension studies of CFTR modulators, including ALYFTREK. An analysis of three age cohorts (≥18 years, 12-17 years, and 6-11 years) with F/MF or F508del/F508del (F/F) mutations shows that participants who initiated therapy at a younger age achieved greater levels of CFTR function, as demonstrated by lower levels of SwCl. Among study participants in the 6-11 years cohorts, those that achieved lower SwCl levels experienced broad clinical benefits — as indicated by greater numerical improvements in lung function, respiratory symptoms, nutritional status and annual rates of PEx. Friday, October 24, 12:15–1:15 p.m. PDT.
  • “Changes in Sweat Chloride Concentrations Following CFTR Modulator Treatment and Association with Antibiotic Usage in Adolescents and Adults with Cystic Fibrosis”: Data will be presented from a pooled analysis of over 2,000 people with CF 12 years and older with F/MForF/F genotypeswho took part in Phase 3 clinical trials of CFTR modulators, including ALYFTREK. These data demonstrate that, following the initiation of treatment with CFTR modulators, achieving sweat chloride levels below the diagnostic threshold (<60 mmol/L) and normal levels (<30 mmol/L) was associated with reduced annual rates of IV and/or oral antibiotic usage compared to trial participants that reached higher levels of sweat chloride. Friday, October 24 at 1:15–2:15 p.m. PDT.

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Additional data being presented on Vertex’s other CFTR modulators, including three oral presentations:

  • “Clinical characteristics and outcomes of people with cystic fibrosis with homozygous minimal function genotypes: Comparison to those with F508del/minimal function genotypes” (Oral & Poster #728), on Thursday, October 23 from 10:15 a.m. to 12:15 p.m. PDT and poster presentation on Friday, October 24, at 1:15–2:15 p.m. PDT.
  • “The lived experience & unmet needs of people with CF with genotypes not responsive to CFTR modulators: A mixed-methods study” (Oral & Poster #429), on Saturday, October 25 from 10:15 a.m. to 12:15 p.m. PDT and poster presentation on Friday, October 24, at 12:15–1:15 p.m. PDT.
  • “Impact of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on healthcare resource utilization (HCRU) over three years in LONGITUDE – a UK CF Registry observational study” (Oral & Poster #729), on Friday, October 24, from 10:15 a.m. to 12:15 p.m. PDT and poster presentation on Friday, October 24, at 12:15–1:15 p.m. PDT.
  • “Baseline results from the NEMO Study: Psychometric evaluation of the Preschool Cystic Fibrosis Questionnaire-Revised in children with cystic fibrosis and caregivers in the United States and Australia” (Poster #529), will be presented at a Rapid Fire Poster Talk on Thursday, October 23, at 1:30–2:20 p.m. PDT in Theater C Exhibit Hall, and poster presentation onFriday, October 24, at 12:15–1:15 p.m. PDT.
  • “Real-world outcomes in people with cystic fibrosis treated with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) with up to four years of follow-up” (Poster #731), on Friday, October 24, at 12:15–1:15 p.m. PDT.
  • “Long-term effects of elexacaftor/tezacaftor/ivacaftor on clinical outcomes and quality of life among adolescents and adults aged 12+ with cystic fibrosis: Interim results from the TRAJECTORY Study” (Poster #739), on Friday, October 24, at 12:15–1:15 p.m. PDT.
  • “Impact of elexacaftor/tezacaftor/ivacaftor on clinical outcomes and quality of life in children with cystic fibrosis aged 6-11 years in the real-world setting” (Poster #734), on Friday, October 24, at 1:15–2:15 p.m. PDT.

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Source- businesswire