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...
| 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-659 is an investigational small molecule being developed for the treatment of cystic fibrosis. It is being studied in combination therapy for patients with cystic fibrosis, including those who are heterozygous for the F508del mutation and a minimal function mutation, and those who are homozygous for F508del.
VX-659 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. Vertex is conducting clinical trials to evaluate the safety and efficacy of VX-659 in patients with cystic fibrosis.
VX-659 is in Phase 3 clinical development. It has completed Phase 1, Phase 2, and Phase 3 trials, with all four trials completed. The drug is investigational and has not been approved by regulatory authorities.
VX-659 has been studied in four completed clinical trials. These include NCT03029455, a Phase 1 study in healthy subjects and adults with cystic fibrosis; NCT03224351, a Phase 2 study of combination therapy; NCT03447249, a Phase 3 study in patients heterozygous for F508del and a minimal function mutation; and NCT03460990, a Phase 3 study in patients homozygous for F508del.
VX-659 is a distinct investigational compound developed by Vertex Pharmaceuticals. It is being studied as part of combination therapy for cystic fibrosis, but it is not the same as other approved Vertex drugs. No alternative names for VX-659 have been reported.