News & Events zu Vertex Pharmaceuticals Inc (VRTX)VRTXVertex Pharmaceuticals Inc

Vertex Pharmaceuticals Announces New Data from ALYFTREK Study

Vertex Pharmaceuticals announced new interim data from the ALYFTREK 2 to 5 years old open-label extension study, highlighting recovery of exocrine pancreatic function in some children in that age group, allowing those children to discontinue pancreatic enzyme replacement therapy Pancreatic exocrine insufficiency in children with cystic fibrosis was previously believed to be irreversible. These data were presented at the North American Cystic Fibrosis Conference. Presented as a late breaker poster at NACFC, the new interim data from 48 children who enrolled in a PERT discontinuation substudy VX22-121-106 (Study 106) Cohort 2, an ongoing open-label extension study of vanzacaftor/tezacaftor/deutivacaftor in children 2 to 5 years of age, demonstrate that early intervention with vanzacaftor/tezacaftor/deutivacaftor has a positive impact on exocrine pancreatic insufficiency and can help achieve PERT independence. EPI is one of the earliest and most severe manifestations of CF, occurring in ~90% of children during the first year of life, requiring lifelong treatment, and driving serious downstream comorbidities. Specifically, data from the interim analysis conducted after children received at least 48 weeks of vanzacaftor/tezacaftor/deutivacaftor treatment showed: Meaningful improvement in fecal elastase-1 levels demonstrates restored pancreatic function: Mean FE-1, a well-established endpoint of exocrine pancreatic function, was 239.7 undefined/g, representing a mean increase of 110.2 microgram/g from baseline, with nearly half (48.6%) of children reaching the pancreatic sufficiency threshold of greater than or equal to200 microgram/g. Of the 48 children eligible for the PERT discontinuation substudy, 18 (37.5%) successfully discontinued PERT for a mean duration of 8.6 weeks (range: 1.9 to 17.6 weeks) at the time of the interim data analysis. Additionally, 7 participants had discontinued PERT prior to the substudy eligibility. Across the full enrolled cohort of 66 children, 50% were not on PERT at the time of the data cut. The ability to discontinue PERT suggests reversal of prior pancreatic exocrine organ failure for those participants. Vertex also presented the following abstracts on clinical and real-world evidence on CFTR modulators as listed below. These abstracts are published in the Journal of Cystic Fibrosis: "Long-term benefits of VNZ/TEZ/D-IVA in reducing IV antibiotic use in people with CF aged 12 years and older: Post-hoc analyses of 96-week open-label extension analysis"; "Natural history study of exocrine pancreatic function in infants with cystic fibrosis less than 12 months of Age"; "Lifetime treatment burden and cost of PERT among people with cystic fibrosis."

Maze Stock Jumps 25% to $27.99 After Vertex Kidney Disease Data

Maze jumps 25% to $27.99 after Vertex kidney disease data

Vertex Pharmaceuticals Reports Positive Phase 2b Results for Inaxaplin

The company states: "Vertex Pharmaceuticals announced positive results from the Phase 2b AMPLIFIED study of inaxaplin in people with APOL1-mediated kidney disease - AMKD - and modest proteinuria and in people with AMKD and type 2 diabetes. In this study, inaxaplin was evaluated as a 45 mg once-daily dose on top of optimized standard of care. Forty-one people were enrolled and dosed in two cohorts: 1) people with AMKD with modest proteinuria (UACR greater than or equal to0.1 g/g to less than0.42 g/g) were eligible for cohort 1 (N=23), and 2) people with AMKD, type 2 diabetes and proteinuria were eligible for cohort 2 (N=18). The primary endpoint in the study was mean percent change in UACR at Week 13 compared to baseline, evaluated separately for each cohort. The other endpoints included mean percent change in urine protein to creatinine ratio - UPCR - at Week 13 compared to baseline and safety. In cohort 1, treatment with inaxaplin administered on top of optimized standard of care led to a reduction of -42.7% (95% CI -58.3%, -21.1%) in UACR at Week 13 compared to baseline. UPCR decreased by -44.7% from baseline at Week 13. These results are in line with the treatment effect previously reported in the Phase 2a study of inaxaplin in AMKD with focal segmental glomerulosclerosis (FSGS), which demonstrated a reduction in UACR of -43.4% and a reduction in UPCR of -47.6% at Week 13 compared to baseline. In this modest proteinuria cohort, most people were not diagnosed with FSGS. In cohort 2, treatment with inaxaplin administered on top of optimized standard of care led to a reduction in UACR at Week 13 of -17.3% (95% CI -36.3%, 7.2%) compared to baseline. UPCR decreased by -25.4% from baseline at Week 13. Inaxaplin was generally safe and well tolerated across both cohorts. There were no serious adverse events related to inaxaplin. All adverse events were mild or moderate in severity."