Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VX-445 · 2 trials · 1 indication
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Arm | Type | Description |
|---|---|---|
| TEZ/IVA | ACTIVE_COMPARATOR | Following a run-in period of 4 weeks with Tezacaftor (TEZ)/Ivacaftor (IVA), participants received TEZ 100 milligram (mg)/IVA 150 mg as fixed-dose combination (FDC) tablet in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the triple combination (TC) treatment period. |
| VX-445/TEZ/IVA TC | EXPERIMENTAL | Following a run-in period of 4 weeks with TEZ/IVA, participants received VX-445 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the TC treatment period. |
| Placebo | PLACEBO_COMPARATOR | Participants who received placebo matched to VX-445/TEZ/IVA for 24 weeks in the TC treatment period. |
| Name | Type | Description |
|---|---|---|
| VX-445/TEZ/IVA | DRUG | Participants received VX-445/TEZ/IVA orally once daily in the morning. |
| TEZ/IVA | DRUG | Participants received TEZ/IVA orally once daily in the morning. |
| IVA | DRUG | Participants received IVA orally once daily in the evening. |
| Placebo | DRUG | Participants received placebo matched to VX-445/TEZ/IVA orally once daily in the morning. |
Key Inclusion Criteria: * Homozygous for the F508del mutation (F/F) * Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height Key Exclusion Criteria: * Clinically significant cirrhosis with or without portal hypertension * Lung infection with org...
Top 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | Alyftrek, Trikafta, Ivacaftor, VX-272, Tezacaftor |
| 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-445 is an investigational small molecule developed for the treatment of cystic fibrosis. It is being studied in people with specific CFTR mutations, including those homozygous for F508del and those heterozygous for F508del with a minimal function mutation. It is not yet approved for any indication.
VX-445 is being developed by Vertex Pharmaceuticals Incorporated, which trades on the Nasdaq under the ticker VRTX. Vertex is the sponsor of the clinical program evaluating VX-445 in cystic fibrosis.
VX-445 is in Phase 3 clinical development. Two Phase 3 trials have been completed, and the drug remains investigational. It has not been approved by the FDA or any other regulatory agency for any indication.
VX-445 has been evaluated in two completed Phase 3 trials: NCT03525548, which studied VX-445 combination therapy in CF subjects homozygous for F508del (F/F) with 113 participants, and NCT03525444, which studied VX-445 combination therapy in subjects heterozygous for F508del and a minimal function mutation (F/MF) with 405 participants.