Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03525548 | A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F) | PHASE3 | COMPLETED | 113 | — | — | Aug 3, 2018 | Dec 28, 2018 | Jan 27, 2020 | 44 | United States, Belgium +2 |
| NCT03525444 | A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) | PHASE3 | COMPLETED | 405 | — | — | Jun 15, 2018 | Apr 24, 2019 | May 19, 2020 | 115 | United States, Australia +11 |
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 | 9 | PHASE3 | VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, VNZ/TEZ/D-IVA, VX-522 mRNA therapy |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719, SION-451, SION-2222, SION-109 |
| BiomX Ltd | 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 |