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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03460990 | A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F) | PHASE3 | COMPLETED | 116 | — | — | May 1, 2018 | Oct 8, 2018 | Oct 17, 2019 | 46 | United States, Australia +4 |
| NCT03447249 | A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) | PHASE3 | COMPLETED | 385 | — | — | Mar 7, 2018 | Feb 5, 2019 | Mar 13, 2020 | 101 | United States, Australia +9 |
| NCT03224351 | A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis | PHASE2 | COMPLETED | 124 | — | — | Aug 8, 2017 | Feb 28, 2018 | Apr 22, 2021 | 47 | United States, Ireland +2 |
| NCT03029455 | A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis | PHASE1 | COMPLETED | 163 | — | — | Nov 1, 2016 | Aug 1, 2017 | Sep 5, 2017 | 10 | United Kingdom |
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-659/TEZ/IVA TC | EXPERIMENTAL | Following a run-in period of 4 weeks with TEZ/IVA, participants received VX-659 240 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-659/TEZ/IVA for 24 weeks in the TC treatment period. |
| Part 1: Placebo | PLACEBO_COMPARATOR | Participants received placebo matched to VX-659/TEZ/IVA in TC treatment period for 4 weeks and placebo matched TEZ/IVA in washout period for 4 days. |
| Part 1: VX-659/TEZ/IVA TC - Low Dose | EXPERIMENTAL | Participants received VX-659 80 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 days. |
| Part 1: VX-659/TEZ/IVA TC - Medium Dose | EXPERIMENTAL | Participants received VX-659 240 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 days. |
| Part 1: VX-659/TEZ/IVA TC - High Dose | EXPERIMENTAL | Participants received VX-659 400 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 days. |
| Part 2: TEZ/IVA | ACTIVE_COMPARATOR | Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 weeks. |
| Part 2: VX-659/TEZ/IVA TC | EXPERIMENTAL | Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-659 400 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 weeks. |
| Part 3: Placebo | PLACEBO_COMPARATOR | Participants received placebo matched to VX-659/TEZ/VX-561 in TC treatment period for 4 weeks. |
| Part 3: VX-659/TEZ/VX-561 TC | EXPERIMENTAL | Participants received VX-659 400 mg qd/TEZ 100 mg qd/VX-561 200 mg qd in TC treatment period for 4 weeks. |
| Part A: VX-659 or Matching Placebo | EXPERIMENTAL | Part A includes single-dose escalation. |
| Part B: VX-659 or Matching Placebo | EXPERIMENTAL | Part B includes multiple-dose escalation. |
| Part C: VX-659 in TC with TEZ/IVA or Matching Triple Placebo | EXPERIMENTAL | Part C includes multiple dose escalation of VX-659 administered in Triple Combination (TC). |
| Part D: VX-659 in TC with TEZ/IVA or Matching Triple Placebo | EXPERIMENTAL | Part D includes subjects with CF. Participants will receive TC or matching placebos. |
| Name | Type | Description |
|---|---|---|
| VX-659/TEZ/IVA | DRUG | Participants received VX-659/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 TEZ/IVA orally once daily in the morning. |
| VX-659 | DRUG | Tablet for oral administration. |
| Placebo (matched to VX-659/TEZ/IVA) | DRUG | Placebo matched to VX-659 and TEZ/IVA. |
| TEZ | DRUG | Tablet for oral administration. |
| VX-561 | DRUG | Tablet for oral administration. |
| Placebo (matched to VX-659/TEZ/VX-561) | DRUG | Placebo matched to VX-659, TEZ and VX-561. |
| Tezacaftor | DRUG | - |
| Ivacaftor | DRUG | - |
| VX-659 Matching Placebo | DRUG | - |
| Triple Combination (TC) Matching Placebos | DRUG | - |
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 |