Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VX-581 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Part A: Single Ascending Dose | EXPERIMENTAL | Participants will be randomized to receive a single dose of VX-581. |
| Placebo: Part A | PLACEBO_COMPARATOR | Participants will be randomized to receive a single dose of placebo matched to VX-581. |
| Part B: Multiple Ascending Dose | EXPERIMENTAL | Participants will be randomized to receive multiple doses of VX-581. |
| Placebo: Part B | PLACEBO_COMPARATOR | Participants will be randomized to receive multiple doses of placebo matched to VX-581. |
| Part C: VX-581 with TEZ/D-IVA or D-IVA | EXPERIMENTAL | Participants will be randomized to receive multiple doses VX-581 in combination with TEZ/D-IVA or D-IVA. |
| Placebo: Part C | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-581 in combination with TEZ/D-IVA or D-IVA. |
| Name | Type | Description |
|---|---|---|
| VX-581 | DRUG | Suspension for Oral Administration. |
| Placebo | DRUG | Suspension for Oral Administration. |
| D-IVA | DRUG | Tablet for Oral Administration. |
| TEZ | DRUG | Tablet for Oral Administration. |
Key Inclusion Criteria: * 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 * Male and Female participants of non-childbearing potential Key Exclusion Criteria: * History of febrile illness or other acute illness that has not f...
| 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-581 is an investigational small molecule being developed for the treatment of cystic fibrosis. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities. The drug is being studied in a clinical trial that includes healthy participants as well as those with cystic fibrosis.
VX-581 targets the CFTR protein, which is the molecular target for the drug. CFTR is a protein involved in cystic fibrosis, and the drug is designed to interact with this protein as part of its therapeutic approach. The drug is a small molecule modality.
VX-581 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company. Vertex Pharmaceuticals is listed on the stock exchange under the ticker symbol VRTX. The company is conducting clinical trials to evaluate the safety and pharmacokinetics of VX-581.
VX-581 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The drug is currently being studied in an early-stage clinical trial to evaluate its safety and pharmacokinetics in healthy participants.
VX-581 is being studied in a Phase 1 clinical trial with the identifier NCT07283770. This trial is a dose escalation study evaluating the safety and pharmacokinetics of VX-581 in healthy participants. The study is currently recruiting and has an enrollment target of 136 participants. It is a randomized, double-blind, placebo-controlled trial conducted in the United States.
VX-581 is a distinct investigational drug candidate developed by Vertex Pharmaceuticals. It is not known to be the same as any other approved or marketed medication. The drug is being studied specifically for its potential role in treating cystic fibrosis, and it is currently in early-stage clinical development.