Benzinga | Oct 19, 2021 08:12AM EDT

Vertex Pharma Reports 5 Scientific Abstracts About Co.'s Cystic Fibrosis Portfolio Will Be Presented At North American Cystic Fibrosis Conference Nov. 2-5, 2021

- 96-week interim results of TRIKAFTA(r) (elexacaftor/tezacaftor/ivacaftor and ivacaftor) study show no loss of pulmonary function in people with at least one F508del allele, a first for any CFTR modulator --

- Real-world data from people treated with KALYDECO(r) (ivacaftor) over approximately 6 years show lower rates of mortality, lung transplant and pulmonary exacerbations than comparator cohort -

- Additional presentations highlight safety and efficacy profile of TRIKAFTA(r) -

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that five scientific abstracts about the company's portfolio of cystic fibrosis (CF) medicines will be presented at the 2021 North American Cystic Fibrosis Conference (NACFC) taking place virtually November 2-5, 2021.

Key data being presented include 96-week interim results from an ongoing TRIKAFTA(r) open-label extension study in people with CF ages 12 years and older with F508del/Minimal Function (F/MF) or F508del/F508del (F/F) genotypes, showing that the favorable safety profile and clinically meaningful improvements in lung function, respiratory symptoms and CFTR function as measured by sweat chloride observed in the Phase 3 pivotal studies were maintained through an additional 96 weeks of treatment (Poster #681). Additionally, a post hoc analysis of the annualized mean rate of change in percent predicted forced expiratory volume in 1 second (ppFEV1) showed there was no loss of pulmonary function over 96 weeks in this CF population, which is a first for any CFTR modulator to date.

Also presented at this year's conference are data on results from a retrospective study of patients with gating mutations ages 6 years or older treated with KALYDECO(r) showing that people treated with KALYDECO(r) over approximately six years of follow up had significantly lower rates of mortality, lung transplant and pulmonary exacerbations (PEx) compared to a cohort of patients that were not eligible for and not receiving KALYDECO treatment (Poster #178).

"The data we're presenting this year clearly demonstrate that our portfolio of CFTR modulators has truly transformed the CF treatment landscape," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. "The long-term follow up data from TRIKAFTA in particular demonstrates the unprecedented treatment effect of this medicine and reinforces the high bar it sets for safety and efficacy. We're committed to continuing to serially innovate in our CF program until we reach our goal of bringing transformative medicines to everyone with this disease."

Additional Presentations

In addition to the studies noted above, other presentations at NACFC include:

* INTERIM RESULTS FROM THE HELIO STUDY: Interim analysis of a study of the real-world clinical effectiveness of TRIKAFTA(r) in people with CF age 12 years and older with at least one F508del allele who were ineligible for another CFTR modulator, demonstrating clinically meaningful improvements in lung function and nutritional status at 6 months. In addition, the annualized PEx rate was lower with TRIKAFTA treatment. These results are consistent with findings from pivotal clinical trials (Poster #56).

* QUALITATIVE STUDY OF PATIENTS TREATED WITH TRIKAFTA AND CAREGIVERS: Results from an ongoing qualitative study to evaluate (1) the real-world patient experience of TRIKAFTA(r) treatment from the perspective of people with CF and caregivers and (2) the impact of TRIKAFTA(r) on the caregiver experience. The impact of the SARS-CoV-2 pandemic was also included in the assessment of the patient and caregiver experience. Results from this study demonstrate that TRIKAFTA(r) has a meaningful and substantial impact on the daily lives of people with CF and caregivers, including the ability to cope with living through the SARS-CoV-2 pandemic (Poster #285).

* INTERIM RESULTS FROM A PHASE 3 OPEN-LABEL EXTENSION STUDY OF CHILDREN WITH CF AGES 6 YEARS AND OLDER: An interim analysis at week 24 of an ongoing, 96-week, Phase 3, open-label extension study designed to assess the long-term safety and efficacy of TRIKAFTA(r) in children 6 years of age and older with at least one F508del allele. Results were consistent with the previously established safety profile of TRIKAFTA(r) in this age group. Results also showed robust and clinically meaningful improvements in lung function, respiratory symptoms, and CFTR activity as measured by sweat chloride and indicate TRIKAFTA(r) provides long-term benefit in this younger patient population (Poster #562).