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 being developed for Alpha 1-Antitrypsin Deficiency, a rare genetic condition. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is being studied in a first-in-human trial to evaluate its safety and tolerability.
VX-634 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. Vertex is conducting clinical research on this investigational drug for the treatment of Alpha 1-Antitrypsin Deficiency.
VX-634 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The Phase 1 trial has been completed, and the drug remains under evaluation for safety and tolerability in humans.
VX-634 has one completed Phase 1 clinical trial registered as NCT05579431, titled 'A Phase 1, First-in-human Study of VX-634'. The trial enrolled 127 participants in the United States, including healthy volunteers aged 18 years and older, and was randomized, double-blind, and placebo-controlled.
VX-634 is the primary name for this investigational drug candidate. No alternative names have been reported for this compound. It is a distinct small molecule being studied by Vertex Pharmaceuticals for Alpha 1-Antitrypsin Deficiency.