Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VX-828 · 2 trials · 1 indication
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | ACTIVE_COMPARATOR | Participants will be randomized to receive a single dose of VX-828 suspension. |
| Cohort 2 | EXPERIMENTAL | Participants will be randomized to receive a single dose of VX-828 tablets. |
| Part A: Single Ascending Dose (SAD) | EXPERIMENTAL | Participants will be randomized to receive a single dose of different dose levels of VX-828. |
| Part A: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-828. |
| Part B: Multiple Ascending Dose (MAD) | EXPERIMENTAL | Participants will be randomized to receive multiple doses of different dose levels of VX-828. The dose levels will be determined based on the data from Part A. |
| Part B: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-828. |
| Part C: Drug Drug Interaction | EXPERIMENTAL | Participants will receive a single dose of VX-828, followed by a washout period, Itraconazole administration, and concomitant administration of itraconazole and VX-828; or participants will receive Midazolam followed by VX-828/TEZ/D-IVA administration, and concomitant administration of VX-828/TEZ/D-IVA and Midazolam. Part C will be an open-label optional cohort. |
| Part D: VX-828 in combination with TEZ/VX-118 ,TEZ/D-IVA or D-IVA | EXPERIMENTAL | Participants will be randomized to receive VX-828 in combination with TEZ/VX-118, TEZ/D-IVA, or D-IVA. |
| Part D: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-828/TEZ/VX-118 or placebo matched to VX-828 in combination with TEZ/D-IVA or D-IVA |
| Part E: VX-828 in Combination with D-IVA with or without TEZ in CF | EXPERIMENTAL | Participants with cystic fibrosis will receive VX-828 in combination with D-IVA with or without TEZ. |
| Name | Type | Description |
|---|---|---|
| VX-828 | DRUG | Tablets for Oral Administration. |
| Placebo | DRUG | Suspension for Oral Administration |
| Itraconazole | DRUG | Solution for Oral Administration |
| Midazolam | DRUG | Syrup for Oral Administration |
| Tezacaftor | DRUG | Tablets for Oral Administration |
| VX-118 | DRUG | Tablets for Oral Administration |
| Deutivacaftor | DRUG | Tablets for Oral Administration |
Key Inclusion Criteria: * Body mass index (BMI) of 18.0 to 32.0 kilogram per square meter (kg/m\^2) * A total body weight of more than (\>) 50 Kg * Nonsmoker or ex-smoker for at least 3 months before screening * Participants of non-childbearing potential Key Exclusion Criteria: * History of febri...
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-828 is an investigational small molecule developed by Vertex Pharmaceuticals for cystic fibrosis, a respiratory disease. It is a Phase 1 clinical-stage candidate and has not been approved. Two Phase 1 trials of VX-828 have been completed, enrolling a total of 233 participants.
VX-828 targets CFTR, the cystic fibrosis transmembrane conductance regulator protein. CFTR dysfunction underlies cystic fibrosis, and VX-828 is designed as a small molecule directed at this protein target. The trials of VX-828 were biomarker selected, meaning participants were enrolled based on a specified biomarker.
VX-828 is developed by Vertex Pharmaceuticals Incorporated, which trades under the ticker VRTX. Vertex is the sponsor of the clinical program evaluating VX-828 in cystic fibrosis.
VX-828 is in Phase 1 clinical development. It is investigational and has not been approved by the FDA. Both Phase 1 trials of VX-828 have been completed, and no active trials are currently listed for the program.
Two completed Phase 1 trials have evaluated VX-828. NCT06154447 studied VX-828 in healthy participants and in participants with cystic fibrosis, enrolling 165 people. NCT06861413 evaluated the relative bioavailability of a VX-828 tablet formulation, enrolling 68 people. Both were conducted in the United States.
Both VX-828 Phase 1 trials were randomized, double blind, and active controlled, and both enrolled adults aged 18 years and older, including healthy volunteers. NCT06154447 enrolled 165 participants with and without cystic fibrosis, while NCT06861413 enrolled 68 participants to assess the relative bioavailability of a VX-828 tablet formulation.