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...
| 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-828 is an investigational small molecule being developed for the treatment of cystic fibrosis. It is currently in Phase 1 clinical development and has been evaluated in healthy participants and in participants with cystic fibrosis in clinical trials conducted in the United States.
VX-828 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. The company is conducting Phase 1 clinical trials to evaluate the drug's safety, tolerability, and pharmacokinetics in healthy volunteers and in patients with cystic fibrosis.
VX-828 is in Phase 1 clinical development. Two Phase 1 trials have been completed, with a total enrollment of 233 participants. The drug is investigational and has not been approved by regulatory authorities. It is being studied for the treatment of cystic fibrosis.
VX-828 has been studied in two completed Phase 1 clinical trials. The first trial, NCT06154447, evaluated VX-828 in healthy participants and in participants with cystic fibrosis, enrolling 165 individuals. The second trial, NCT06861413, assessed the relative bioavailability of a VX-828 tablet formulation and enrolled 68 participants.
VX-828 is a distinct investigational drug candidate developed by Vertex Pharmaceuticals. No alternative names have been reported for this compound. It is a small molecule being studied for cystic fibrosis and is not known to be identical to any other marketed or investigational medication.