Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Vanzacaftor · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Group 1 | EXPERIMENTAL | Participants will receive VNZ-matching placebo, moxifloxacin-matching placebo and VNZ at different time points. |
| Group 2A | ACTIVE_COMPARATOR | Participants will receive VNZ-matching placebo, moxifloxacin, and moxifloxacin-matching placebo at different time points. |
| Group 2B | ACTIVE_COMPARATOR | Participants will receive VNZ-matching placebo, moxifloxacin-matching placebo and moxifloxacin at different time points. |
| Name | Type | Description |
|---|---|---|
| Vanzacaftor | DRUG | Tablets for oral administration. |
| Vanzacaftor Placebo | DRUG | Placebo for oral administration. |
| Moxifloxacin | DRUG | Tablets for oral administration. |
| Moxifloxacin Placebo | DRUG | Tablets for oral administration. |
Key Inclusion Criteria: * Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (Kg/m\^2), both inclusive * Male and female participants of age 18 to 45 years, both inclusive * Serum potassium, calcium, and magnesium values within normal ranges Key Exclusion Criteria: * Median QTcF\>450...
| 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 |
Vanzacaftor is an investigational small molecule being developed for the treatment of cystic fibrosis. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is being studied for its effects on the QT/QTc interval in healthy participants.
Vanzacaftor is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. The company is conducting clinical trials to evaluate the safety and effects of this investigational drug for cystic fibrosis.
Vanzacaftor is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. The completed Phase 1 trial evaluated the drug's effect on the QT/QTc interval in healthy participants, and the drug remains in early-stage clinical testing.
Vanzacaftor has one completed clinical trial, NCT05867147, which was a Phase 1 electrocardiogram study to evaluate the effect of the drug on the QT/QTc interval in healthy participants. The trial was conducted in the United States and enrolled 56 participants aged 18 years and older.
Yes, Vanzacaftor is a small molecule drug. It is being developed as a potential treatment for cystic fibrosis and is currently in Phase 1 clinical trials. The drug is administered orally and is being studied for its cardiac safety profile in healthy volunteers.