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VX-770 · 5 trials · 2 indications
Subjects inhaled hyperpolarized helium-3 (3He) gas mixed with nitrogen to make a total volume of approximately one-third forced vital capacity (FVC) to a maximum of 1 liter and hold their breath for 20 seconds or less. Rapid magnetic resonance imaging (MRI) was performed during inhalation/exhalation and/or breath-hold. Areas of decreased ventilation were observed as ventilation defects that are visualized as decreased (and/or absent) 3He intensity in 3He-MRI. The total ventilation defect was defined as the ratio of total ventilation defect volume (L) to total lung volume (L), expressed as a percentage. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in VX-770 treatment phase (Day 15 to 42).
Subjects were asked to inhale hyperpolarized 3 He gas mixed with nitrogen to make a total volume of approximately one-third forced vital capacity (FVC) to a maximum of 1 liter and hold their breath for 20 seconds or less. Rapid MRI was performed during inhalation/exhalation and/or breath-hold. Areas of decreased ventilation were observed as ventilation defects that are visualized as decreased (and/or absent) 3He intensity in 3He MRI. The total ventilation defect was defined as the ratio of total ventilation defect volume (L) to total lung volume (L), expressed as a percentage. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in Part B (48 weeks).
| Arm | Type | Description |
|---|---|---|
| VX-770 | EXPERIMENTAL | Part A: Subjects received placebo tablets matched to VX-770 150 milligram (mg) orally twice daily from Day 1 to 14 (Placebo run-in period), followed by VX-770 150 mg tablets orally twice daily from Day 15 to 42 (VX-770 treatment period), and then placebo tablets matched to VX-770 150 mg orally twice daily from Day 43 to 57 (Placebo washout period) during Part A of the study. Part B: Subjects received VX-770 150 mg tablets orally twice daily for 48 weeks during Part B of the study. Part B included subjects from Part A and newly enrolled subjects. |
| Group A | EXPERIMENTAL | approximately 12 male and female subjects with moderate hepatic impairment |
| Group B | EXPERIMENTAL | approximately 12 healthy male and female subjects |
| desipramine | EXPERIMENTAL | - |
| Midazolam | EXPERIMENTAL | - |
| Rosiglitazone | EXPERIMENTAL | - |
| Fluconazole | EXPERIMENTAL | - |
| Rifampin | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| VX-770 | DRUG | Tablet. |
| Placebo | DRUG | Tablet. |
| Rifampin | DRUG | In Period 2, subjects will receive a daily oral dose of rifampin 600 mg on Days 1 through 10 |
Inclusion Criteria: * Male or female with Cystic Fibrosis * Must have the G551D-CFTR mutation on at least 1 allele * FEV1 ≥40% of predicted normal for age, gender, and height at Screening * 12 years of age or older * Must be able to swallow tablets Exclusion Criteria: * History of solid organ or ...
| 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-770 is an investigational small molecule being developed by Vertex Pharmaceuticals for the treatment of cystic fibrosis. It has been studied in patients with cystic fibrosis who have the G551D mutation, as well as in healthy volunteers for drug interaction and safety studies.
VX-770 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. The company has conducted clinical trials of VX-770 in the United States, Czechia, and Slovakia.
VX-770 is in Phase 2 clinical development for cystic fibrosis. It has completed Phase 1 and Phase 2 trials, with the Phase 2 study evaluating its effect on hyperpolarized helium-3 magnetic resonance imaging in subjects with cystic fibrosis and the G551D mutation.
VX-770 has been studied in four completed clinical trials. These include NCT01018368, a Phase 1 study of VX-770 and rifampin in healthy male subjects; NCT01060566, a Phase 1 study of VX-770 on midazolam and rosiglitazone and the effect of fluconazole on VX-770; NCT01161537, a Phase 2 study in cystic fibrosis patients with the G551D mutation; and NCT01208285, a Phase 1 study in subjects with moderate hepatic impairment.
VX-770 is an investigational drug and is not FDA approved. It is still in clinical development, with completed Phase 1 and Phase 2 trials. The drug has not yet reached the stage of regulatory approval.