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...
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 |
Vanzacaftor is an investigational small molecule being developed for cystic fibrosis, a respiratory disease. It is in Phase 1 clinical development and has not been approved for any indication. The only completed trial to date studied its cardiac safety in healthy volunteers rather than testing it as a cystic fibrosis treatment.
Vanzacaftor targets CFTR, the cystic fibrosis transmembrane conductance regulator protein, and is classified as a CFTR positive modulator. By acting on CFTR, it aims to improve the function of the defective ion channel that causes cystic fibrosis. This mechanism is the basis for its development as a potential therapy for the disease.
Vanzacaftor 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 the drug. The company is known for its work in cystic fibrosis therapeutics.
Vanzacaftor is in Phase 1 clinical development. It is an investigational agent and has not received FDA approval. The program has completed one Phase 1 trial, and no active trials are currently listed for the drug.
Vanzacaftor has been studied in one completed Phase 1 trial, NCT05867147, titled an electrocardiogram study to evaluate the effect of vanzacaftor on the QT/QTc interval in healthy participants. The trial enrolled 56 healthy volunteers in the United States and was randomized, double-blind, and active-controlled.