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VX-864 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Placebo | PLACEBO_COMPARATOR | Participants received placebo matched to VX-864 in the treatment period for 28 days. |
| VX-864 100 mg | EXPERIMENTAL | Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days. |
| VX-864 300 mg | EXPERIMENTAL | Participants received VX-864 300 mg q12h in the treatment period for 28 days. |
| VX-864 500 mg | EXPERIMENTAL | Participants received VX-864 500 mg q12h in the treatment period for 28 days. |
| Name | Type | Description |
|---|---|---|
| VX-864 | DRUG | Tablets for oral administration. |
| Placebo | DRUG | Placebo matched to VX-864 for oral administration. |
Key Inclusion Criteria: * Subjects must have a PiZZ genotype confirmed at screening * Plasma AAT levels indicating severe deficiency at screening Key Exclusion Criteria: * History of a medical condition that could negatively impact the ability to complete the study * Solid organ, or hematological...
VX-864 is an investigational small molecule developed by Vertex Pharmaceuticals for Alpha1-Antitrypsin Deficiency, a rare genetic disease. It was studied in a Phase 2 clinical trial and is not approved for any use. It is designed as an oral therapy rather than an infused protein replacement.
VX-864 was being developed to treat Alpha1-Antitrypsin Deficiency, a rare inherited condition. The goal was to address the underlying protein deficiency rather than only manage symptoms. It was evaluated in adults with the PiZZ genotype, the form of the disease associated with the most severe protein deficiency.
VX-864 targets alpha1-antitrypsin (AAT), the protein that is deficient in Alpha1-Antitrypsin Deficiency. It is a small molecule designed to act on AAT, a protein target, rather than replacing the missing protein through infusion. This oral approach aims to correct the underlying deficiency at its source.
VX-864 is made by Vertex Pharmaceuticals Incorporated, which trades on the Nasdaq under the ticker VRTX. Vertex is the developer of the compound and sponsored its clinical evaluation in Alpha1-Antitrypsin Deficiency. The program is part of the company's rare disease portfolio.
VX-864 is in Phase 2 development and is investigational, meaning it is not approved by the FDA or any other regulator. One Phase 2 trial has been completed, and no trials are currently active. Vertex Pharmaceuticals has not announced a regulatory submission for this asset.
VX-864 was studied in one completed Phase 2 trial, NCT04474197, titled Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype. The trial enrolled 44 adults with Alpha1-Antitrypsin Deficiency across the United States, Canada, Germany, Ireland, Sweden, and the United Kingdom. It was randomized, double-blind, and placebo-controlled.