Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07283770 | Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy Participants | PHASE1 | RECRUITING | 128 | — | — | Dec 9, 2025 | Nov 19, 2026 | May 18, 2026 | 1 | United States |
| 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. |
| Name | Type | Description |
|---|---|---|
| VX-581 | DRUG | Suspension for Oral Administration. |
| Placebo | DRUG | Suspension 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 | 9 | PHASE3 | VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, VNZ/TEZ/D-IVA, VX-522 mRNA therapy |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719, SION-451, SION-2222, SION-109 |
| BiomX Ltd | 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 |