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VX-407 · 5 trials · 1 indication
| Arm | Type | Description |
|---|---|---|
| VX-407 | EXPERIMENTAL | Participants will receive VX-407 for up to 52 weeks. |
| Part A: VX-407 With Levonorgestrel/Ethinyl Estradiol (LNG/EE) | EXPERIMENTAL | Participants will receive a single dose of LNG/EE on Days 1 and 21 in fasted state. Participants will also receive VX-407 every 12 hours (q12h) from Days 8 through 26 in fasted state. |
| Part B (Optional): VX-407 With Norgestimate/Ethinyl Estradiol (NGM/EE) | EXPERIMENTAL | Participants will receive a single dose of NGM/EE on Days 1 and 23 in fasted state. Participants will also receive VX-407 q12h from Days 10 through 30 in fasted state. |
| Part C (Optional): VX-407 With Norethindrone/Ethinyl Estradiol (NET/EE) | EXPERIMENTAL | Participants will receive a single dose of NET/EE on Days 1 and 19 in fasted state. Participants will also receive VX-407 q12h from Days 6 through 22 in fasted state. |
| Part D (Optional): VX-407 With Drospirenone/Ethinyl Estradiol (DRSP/EE) | EXPERIMENTAL | Participants will receive a single dose of DRSP/EE on Days 1 and 21 in fasted state. Participants will also receive VX-407 q12h from Days 8 through 26 in fasted state. |
| Part A: Single Ascending Dose | EXPERIMENTAL | Participants will be randomized to receive a single dose of VX-407. |
| Placebo Part A | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-407. |
| Part B: Multiple Ascending Dose | EXPERIMENTAL | Participants will be randomized to receive multiple doses of VX-407. |
| Placebo Part B | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-407. |
| Part A: Single Ascending Dose (SAD) | EXPERIMENTAL | Participants will be randomized to receive a single dose of different dose levels of VX-407. |
| Part A: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-407. |
| Part B: Multiple Ascending Dose (MAD) | EXPERIMENTAL | Participants will be randomized to receive multiple doses of different dose levels of VX-407. The dose levels will be determined based on the data from Part A. |
| Part B: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive multiple doses of placebo matched to VX-407. |
| Part C: Drug-Drug Interaction | EXPERIMENTAL | Participants will be administered Midazolam (MDZ) in the presence or absence of VX-407. The dose levels will be determined based on the data from Part B. |
| Part D | EXPERIMENTAL | Participants will be randomized to receive VX-407 in 1 of 3 treatment sequences with 3 dosing periods to assess the relative bioavailability of VX-407 formulations and the effect of food on the pharmacokinetics of VX-407. |
| Name | Type | Description |
|---|---|---|
| VX-407 | DRUG | Tablets for oral administration. |
| LNG/EE | DRUG | Combination Tablets for Oral Administration. |
| NGM/EE | DRUG | Combination Tablets for Oral Administration. |
| NET/EE | DRUG | Combination Tablets for Oral Administration. |
| DRSP/EE | DRUG | Combination Tablets for Oral Administration. |
| Placebo | DRUG | Suspension for oral administration. |
| Midazolam | DRUG | Syrup for oral administration. |
Key Inclusion Criteria: * A pre-existing diagnosis of ADPKD as defined in the protocol * Willing and able to comply with scheduled visits and other study procedures * Participants with ADPKD with Mayo imaging classification (MIC) status of 1B (with htTKV ≥250 mL/m), 1C, 1D, or 1E confirmed by abdom...
VX-407 is an investigational small molecule being developed for Autosomal Dominant Polycystic Kidney Disease (ADPKD), a genetic disorder characterized by cyst growth in the kidneys. It is currently in Phase 2 clinical development and is being studied in patients with a subset of PKD1 gene variants.
VX-407 targets PC1, a protein encoded by the PKD1 gene. Mutations in PKD1 are a common cause of ADPKD, and VX-407 is designed to address the underlying protein defect. The drug is being studied specifically in patients with certain PKD1 gene variants.
VX-407 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. Vertex is conducting clinical trials of VX-407 in healthy volunteers and in patients with ADPKD.
VX-407 is in Phase 2 clinical development. It has completed three Phase 1 trials in healthy participants and is currently in an active Phase 2a study called AGLOW, which is enrolling patients with ADPKD who have a subset of PKD1 gene variants.
VX-407 has been studied in several clinical trials, including NCT06345755, NCT07022119, and NCT07074327, all Phase 1 studies in healthy participants. The active Phase 2a trial is NCT07161037, known as AGLOW, which is evaluating VX-407 in patients with ADPKD who have specific PKD1 gene variants.
VX-407 is the sole name provided for this investigational drug. It is not known to have any alternative names or aliases. The drug is being developed exclusively by Vertex Pharmaceuticals for the treatment of Autosomal Dominant Polycystic Kidney Disease.