Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VX-809 · 4 trials · 1 indication
Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. Serious adverse event (SAE) (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. An AE that started at or after initial dosing of study drug, or increased in severity after initial dosing of study drug visit is considered treatment-emergent.
Blood samples drawn during the study will be analyzed to measure the PK parameters, such as concentration, exposure and half-life of VX-809 and its metabolite.
Blood samples drawn during the study will be analyzed to measure the PK parameters, such as concentration, exposure and half-life of VX-770 and its metabolites.
| Arm | Type | Description |
|---|---|---|
| LUM/IVA | EXPERIMENTAL | Fixed-dose combination with lumacaftor (LUM) 200 mg every 12 hours (q12h)/ ivacaftor (IVA) 250 mg q12h |
| Placebo | PLACEBO_COMPARATOR | Matching placebo q12h |
| VX-809, 25 mg | EXPERIMENTAL | VX-809, 25 milligram (mg) capsule orally once daily for 28 days. |
| VX-809, 50 mg | EXPERIMENTAL | VX-809, 50 mg capsule orally once daily for 28 days. |
| VX-809, 100 mg | EXPERIMENTAL | VX-809, 100 mg capsule orally once daily for 28 days. |
| VX-809, 200 mg | EXPERIMENTAL | VX-809, 200 mg capsule orally once daily for 28 days. |
| Treatment Arm | EXPERIMENTAL | Subjects randomized to study drug will take VX-809 for 14 days followed by a 14 day washout. Next subjects will take VX-770 for 14 days followed by a 14 day washout. Lastly, subjects will take both VX-809 and VX-770 for 14 days. |
| Placebo Arm | PLACEBO_COMPARATOR | Subjects randomized to placebo will take VX-809 placebo for 14 days followed by a 14 day washout. Next subjects will take VX-770 placebo for 14 days followed by a 14 day washout. Lastly, subjects will take both VX-809 and VX-770 placebo for 14 days. |
| Treatment Period 1 | EXPERIMENTAL | - |
| Treatment Period 2 | EXPERIMENTAL | - |
| Treatment Period 3 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| VX-809 | DRUG | - |
| Placebo | DRUG | - |
| VX-770 | DRUG | - |
| VX-809 placebo | DRUG | capsule, taken once daily |
| VX-770 placebo | DRUG | tablet, taken once every 12 hours |
| VX-809 & VX-770 | DRUG | VX-809 capsule, once daily, and VX-770 tablets, once every 12 hours, for 14 days |
Inclusion Criteria: * Subjects who weigh ≥15 kg without shoes a the Screening Visit * Subjects with confirmed diagnosis of CF at the Screening Visit. * Subjects who are homozygous for the F508del CFTR mutation * Subjects with ppFEV1 of ≥70 percentage points adjusted for age, sex, and height * Subje...
| 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-809 is an investigational small molecule being developed for cystic fibrosis. It is a corrector of the F508del-CFTR protein mutation, which is the most common cause of the disease. The drug is designed to improve the function of the defective CFTR protein in patients with this specific genetic mutation.
VX-809 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. The company has conducted multiple clinical trials of VX-809, both alone and in combination with other drugs, for the treatment of cystic fibrosis.
VX-809 has completed Phase 3 clinical trials for cystic fibrosis. The drug is investigational and has not been approved by regulatory authorities. It has been studied in combination with ivacaftor (VX-770) in patients homozygous for the F508del-CFTR mutation, with trials completed in multiple countries.
VX-809 has been studied in four completed clinical trials. NCT00865904 was a Phase 2 study in cystic fibrosis patients with the F508del-CFTR mutation. NCT00966602 and NCT01216046 were Phase 1 drug-drug interaction studies with VX-770. NCT02514473 was a Phase 3 study of lumacaftor (VX-809) combined with ivacaftor in homozygous F508del patients.
Yes, VX-809 is the same drug as lumacaftor. The compound was originally designated VX-809 during early development and later received the generic name lumacaftor. It is being developed by Vertex Pharmaceuticals for cystic fibrosis, typically in combination with ivacaftor.