Vertex Pharmaceuticals recently presented data from several clinical trials of cystic fibrosis treatments showing positive outcomes for CF patients. Data focused on the approved therapies Orkambi and Kalydeco, and investigative treatments like tezacaftor/ivacaftor and several triple regimens.
These findings were presented at the North American Cystic Fibrosis Conference (NACFC) that ran Nov. 2-4 in Indianapolis.
Orkambi (ivacaftor/lumacaftor) data were shown in a poster “Safety and Efficacy of Lumacaftor/Ivacaftor (LUM/IVA) in Patients aged ≥6 years with CF Homozygous for F508del-CFTR (Phase 3 Extension Study).”
It involved an analysis of an ongoing 96-week extension study (NCT03125395) of Orkambi in 240 children, age 6 to 11, with two copies of the F508del-CFTR mutation (a mutation that causes CF).
An interim analysis at 48 weeks of treatment showed that Orkambi improved the patients’ lung function, sweat chloride and body mass index.
Concerning safety, the analysis revealed a similar safety profile to that observed in previous Phase 3 studies in children. Most adverse events reported were mild or moderate, and the most common ones were cough, pulmonary exacerbation, pyrexia (fever), headache, nasal congestion, and upper respiratory tract infection.
“Cystic fibrosis is a progressive disease where the damage begins at birth,” Mark Chilvers, MD, lead investigator for the extension study, and a clinical associate professor in the division of respiratory medicine, Department of Pediatrics, at the University of British Columbia, said in a press release.
“Because of this, it is critical to begin treating the disease as early as possible. These data are important because they demonstrate that in children as young as 6 years of age, Orkambi is well tolerated and that the respiratory and nutritional changes are sustained over time” Chilvers added.
Other data presented at the meeting also showed that Orkambi improved the extent and severity of structural lung damage in CF patients ages 6 to 11. More detailed information about Orkambi’s data presented at NACFC can be found here.
Real-world data on another Vertex’s therapy, Kalydeco (ivacaftor), was provided in two poster presentations.
This data resulted from an ongoing, five-year, post-approval, observational study evaluating Kalydeco’s long-term effects. This real-world data, including 2,159 patients, showed that those given Kalydeco had improved lung function and nutritional measurements, plus a lower risk of pulmonary exacerbations, hospitalization, transplantation and death, compared to patients not receiving the drug.
No new safety concerns were reported.
At NACFC 2017, data on the efficacy and safety of Vertex’s investigative combination of tezacaftor/ivacaftor was also presented.
Results from the EXTEND Phase 3 open-label clinical trial (NCT02565914) — in patients who had completed the Phase 3 EVOLVE (NCT02347657) and EXPAND (NCT02392234) studies — showed tezacaftor/ivacaftor treatment led to a sustained improvement in lung function for up to 48 total weeks of treatment, consistent with results from the earlier Phase 3 trials.
The combo therapy was also shown to be generally well-tolerated and to have a consistent safety profile.
More detailed information about tezacaftor/ivacaftor’s data presented at NACFC can be found here.
Data from Phase 1 and 2 studies (NCT03227471, NCT02951195 and NCT03029455) showed that such triple combinations improved lung function in CF patients. Triple combination regimens have the potential to treat CF patients who have mutations that are not responsive to tezacaftor/ivacaftor.
All three combinations tested were generally well-tolerated, with the majority of adverse events being mild to moderate in severity.
Data from ongoing Phase 2 trials are expected in the beginning of 2018.
“The breadth of data presented at this meeting demonstrate significant progress toward our key goals of bringing disease-modifying medicines to all people with CF and increasing the benefits for patients on our current medicines,” said Jeffrey Chodakewitz, MD, executive vice president and chief medical officer at Vertex.
“We also know that many people with CF are still waiting for a medicine to treat the cause of their disease and we are continuing our efforts to develop additional new medicines, such as triple combination regimens, for these people with a sense of urgency” Chodakewitz added.
Vertex plans to possibly advance two of its triple combination regimens into Phase 3 testing by mid-2018.
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