Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VX-561 · 1 trial · 1 indication
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Arm | Type | Description |
|---|---|---|
| Ivacaftor | ACTIVE_COMPARATOR | Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks. |
| VX-561: 25 mg | EXPERIMENTAL | Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks. |
| VX-561: 50 mg | EXPERIMENTAL | Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks. |
| VX-561: 150 mg | EXPERIMENTAL | Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks. |
| VX-561: 250 mg | EXPERIMENTAL | Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks. |
| Name | Type | Description |
|---|---|---|
| VX-561 | DRUG | VX-561 tablets for oral administration. |
| IVA | DRUG | 150-mg film-coated tablet for oral administration. |
| Placebo | DRUG | Placebo matched to IVA. |
Key Inclusion Criteria: * Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D * On ivacaftor therapy * FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: * History of cli...
| 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-561 is an investigational small molecule being developed for the treatment of cystic fibrosis. It is being studied in patients aged 18 years and older with this condition. The drug is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.
VX-561 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company. Vertex is publicly traded on the Nasdaq stock exchange under the ticker symbol VRTX. The company is conducting clinical trials to evaluate the safety and efficacy of VX-561 in patients with cystic fibrosis.
VX-561 is in Phase 2 clinical development. A Phase 2 study has been completed to evaluate its efficacy and safety in adults with cystic fibrosis. The drug is investigational and has not received approval from the FDA or other regulatory agencies for commercial use.
VX-561 has been studied in a Phase 2 clinical trial registered as NCT03911713. This completed study enrolled 77 participants aged 18 years and older with cystic fibrosis. The trial was conducted in the United States, Australia, Belgium, Germany, Ireland, Netherlands, and the United Kingdom.
No alternative names for VX-561 have been disclosed. The drug is identified by its code name VX-561 in clinical trial registries and scientific literature. It is a distinct investigational compound being developed by Vertex Pharmaceuticals for cystic fibrosis.