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Also known as CTP-656, Deutivacaftor (D-IVA), Deutivacaftor, D-IVA
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...
Top 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | Alyftrek, Trikafta, Ivacaftor, VX-581, VX-272 |
| 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 |
| Roivant Sciences Ltd. | ROIV | 1 | PHASE1 | SP-101 |
VX-561 is an investigational small molecule being studied for the treatment of cystic fibrosis in adults aged 18 years and older. It is a Phase 2 candidate developed by Vertex Pharmaceuticals. It has not been approved for any indication and remains in clinical development.
VX-561 targets CFTR, the cystic fibrosis transmembrane conductance regulator protein. CFTR dysfunction underlies cystic fibrosis, and VX-561 is designed as a small molecule that acts on this protein target. The Phase 2 program enrolled biomarker-selected participants with cystic fibrosis.
VX-561 is developed by Vertex Pharmaceuticals Incorporated, which trades on the Nasdaq under the ticker VRTX. Vertex is the sponsor of the clinical program evaluating VX-561 in cystic fibrosis.
VX-561 is in Phase 2 development for cystic fibrosis. The completed Phase 2 study evaluated efficacy and safety in adults aged 18 years and older. VX-561 is investigational and has not been approved by the FDA for any indication.
VX-561 has been studied in NCT03911713, a Phase 2 trial titled "A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis." The trial is completed, enrolled 77 participants, and was randomized, double-blind, and active-controlled across the United States, Australia, Belgium, Germany, Ireland, the Netherlands, and the United Kingdom.