Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VX-445/TEZ/IVA · 2 trials · 1 indication
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Arm | Type | Description |
|---|---|---|
| TEZ/IVA | ACTIVE_COMPARATOR | Following a run-in period of 4 weeks with Tezacaftor (TEZ)/Ivacaftor (IVA), participants received TEZ 100 milligram (mg)/IVA 150 mg as fixed-dose combination (FDC) tablet in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the triple combination (TC) treatment period. |
| VX-445/TEZ/IVA TC | EXPERIMENTAL | Following a run-in period of 4 weeks with TEZ/IVA, participants received VX-445 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the TC treatment period. |
| Placebo | PLACEBO_COMPARATOR | Participants who received placebo matched to VX-445/TEZ/IVA for 24 weeks in the TC treatment period. |
| Name | Type | Description |
|---|---|---|
| VX-445/TEZ/IVA | DRUG | Participants received VX-445/TEZ/IVA orally once daily in the morning. |
| TEZ/IVA | DRUG | Participants received TEZ/IVA orally once daily in the morning. |
| IVA | DRUG | Participants received IVA orally once daily in the evening. |
| Placebo | DRUG | Participants received placebo matched to VX-445/TEZ/IVA orally once daily in the morning. |
Key Inclusion Criteria: * Homozygous for the F508del mutation (F/F) * Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height Key Exclusion Criteria: * Clinically significant cirrhosis with or without portal hypertension * Lung infection with org...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | VX-121/TEZ/D-IVA |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719 |
| BiomX Inc. | PHGE | 1 | PHASE2 | BX004 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
VX-445/TEZ/IVA is an investigational small molecule combination therapy being developed for cystic fibrosis. It is being studied in patients who are heterozygous for the F508del mutation and a minimal function mutation, as well as in patients homozygous for F508del. The drug is currently in Phase 3 clinical development.
VX-445/TEZ/IVA is a combination therapy that includes VX-445, tezacaftor, and ivacaftor. It targets the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is defective in people with cystic fibrosis. The combination aims to correct and potentiate the CFTR protein function.
VX-445/TEZ/IVA is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company. Vertex is focused on developing therapies for serious diseases, including cystic fibrosis. The company is publicly traded under the ticker symbol VRTX.
VX-445/TEZ/IVA is in Phase 3 clinical development. It has completed two Phase 3 trials, with a total enrollment of 518 participants. The drug is not yet approved and remains investigational, as it is still undergoing clinical evaluation for safety and efficacy.
VX-445/TEZ/IVA has been studied in two completed Phase 3 trials. NCT03525444 enrolled 405 patients with cystic fibrosis heterozygous for F508del and a minimal function mutation. NCT03525548 enrolled 113 patients homozygous for F508del. Both trials were randomized, double-blind, and active-controlled.
VX-445/TEZ/IVA is a combination of VX-445, tezacaftor, and ivacaftor. VX-445 is also known as elexacaftor, so the drug is commonly referred to as elexacaftor/tezacaftor/ivacaftor. This triple combination is being developed for cystic fibrosis.