Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VX-659 · 4 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-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...
Top 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | Alyftrek, Trikafta, Ivacaftor, VX-581, VX-272 |
| 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 |
| Roivant Sciences Ltd. | ROIV | 1 | PHASE1 | SP-101 |
VX-659 is an investigational small molecule developed for the treatment of cystic fibrosis. It was studied as part of a combination regimen in people with specific CFTR mutations, including those homozygous for F508del and those heterozygous for F508del with a minimal function mutation. It is not an approved therapy.
VX-659 was developed by Vertex Pharmaceuticals Incorporated, which trades on the Nasdaq under the ticker VRTX. Vertex sponsored the clinical program evaluating VX-659 as part of a combination cystic fibrosis treatment regimen.
VX-659 reached Phase 3 clinical development. Four trials are recorded for the program, spanning Phase 1 through Phase 3, and all four are completed. No trials are currently active, and VX-659 is an investigational agent rather than an approved medicine.
VX-659 was evaluated in four completed trials: NCT03029455, a Phase 1 study in healthy subjects and adults with cystic fibrosis; NCT03224351, a Phase 2 study; NCT03460990, a Phase 3 study in patients homozygous for F508del; and NCT03447249, a Phase 3 study in patients heterozygous for F508del with a minimal function mutation.
The VX-659 clinical program enrolled 788 participants across four completed trials. Enrollment ranged from 116 participants in the Phase 3 F508del homozygous study to 385 in the Phase 3 F508del minimal function study. The trials were randomized, double blind, and controlled, and included sites in North America, Europe, Israel, and Australia.