Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VX-634 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Part A | EXPERIMENTAL | Participants grouped in different cohorts will receive a single ascending dose of VX-634. |
| Placebo Part A | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-634. |
| Part B | EXPERIMENTAL | Participants grouped in different cohorts will receive multiple doses of VX-634.The dose levels will be determined based on the data from Part A. |
| Placebo Part B | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-634. |
| Name | Type | Description |
|---|---|---|
| VX-634 | DRUG | Suspension for oral administration. |
| Placebo | DRUG | Suspension for oral administration. |
Key Inclusion Criteria: * Participants of age between 18 to 55 years (inclusive) * Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (kg/m\^2) * A total body weight of more than (\>)50 kg * Participants of non childbearing potential * Non smoker or ex-smoker for at least 3 months befo...
VX-634 is an investigational small molecule developed by Vertex Pharmaceuticals for Alpha 1-Antitrypsin Deficiency, a rare genetic disease. It is designed to target the AAT protein. The drug has completed a Phase 1 first-in-human clinical trial and remains in clinical development.
VX-634 is being developed as a treatment for Alpha 1-Antitrypsin Deficiency, a rare inherited condition. It is a small molecule that targets the AAT protein. The program is in Phase 1 development, and the drug has not been approved for any indication.
VX-634 targets AAT, the alpha 1-antitrypsin protein. It is a small molecule designed to act on this protein target. Vertex Pharmaceuticals is developing it for Alpha 1-Antitrypsin Deficiency, where the AAT protein is the underlying disease driver.
VX-634 is developed by Vertex Pharmaceuticals Incorporated, which trades on the Nasdaq under the ticker VRTX. Vertex is the sponsor of the VX-634 program for Alpha 1-Antitrypsin Deficiency.
VX-634 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. Its single Phase 1 first-in-human trial has been completed, and no active trials are currently listed for the program.
VX-634 has been studied in one Phase 1 first-in-human trial, NCT05579431, titled A Phase 1, First-in-human Study of VX-634. The trial enrolled 127 participants in the United States, was randomized, double-blind, and placebo-controlled, and has been completed.