Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VX-661 · 5 trials · 1 indication
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
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. 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, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent. Baseline was defined as Day 1 of PC Phase.
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. 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, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent. Baseline was defined as Day 1 of the OLE Phase.
An AE is defined as 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) is any AE that results in any of the following: death; life-threatening condition; inpatient hospitalization or prolongation of hospitalization; persistent or significant disability or incapacity; congenital anomaly or birth defect; or other important medical event. Treatment-emergent adverse events are defined as adverse events that were reported or worsened on or after start of study drug through the Follow-up Visit (28 days after last dose of study drug) or premature discontinuation.
Sweat samples were collected using an approved collection device. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug.
Sweat samples were collected using an approved collection device. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug. As per planned analysis, participants who received placebo in Group 4 and 6 were combined and compared with Group 6.
Sweat samples were collected using an approved collection device. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug.
| Arm | Type | Description |
|---|---|---|
| VX-661/IVA | EXPERIMENTAL | VX-661 100 milligram (mg) plus IVA 150 mg fixed dose combination (FDC) tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12. |
| Placebo | PLACEBO_COMPARATOR | Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12. |
| VX-661/Ivacaftor combination | EXPERIMENTAL | - |
| Ivacaftor monotherapy | EXPERIMENTAL | - |
| PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h | EXPERIMENTAL | Participants received VX-661 50 milligram (mg) tablet plus Ivacaftor (IVA) 150 mg tablet every 12 hours (q12h) for 12 weeks. |
| PC Phase: VX 661 placebo q12h + IVA placebo q12h | PLACEBO_COMPARATOR | Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks. |
| PC Phase: VX-661 100 mg qd + IVA 150 mg q12h | EXPERIMENTAL | Participants received two VX-661 50 mg tablets once daily (qd) plus IVA 150 mg tablet q12h for 12 weeks. |
| PC Phase: VX -661 placebo qd + IVA placebo q12h | PLACEBO_COMPARATOR | Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks. |
| OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h | EXPERIMENTAL | Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase. |
| Group 1-6d Combined: Placebo | PLACEBO_COMPARATOR | All participants in group 1, 2a, 2b, 3a, 3b, 4, 5a, 5b, 6a and 6d who received placebo matched to VX-661 tablet and/or placebo matched to ivacaftor tablet for up to 28 days. |
| Group 1: VX-661 10 mg qd | EXPERIMENTAL | All participants in group 1 who received VX-661 10 milligram (mg) tablet orally once daily (qd) for up to 28 days. |
| Group 2a: VX-661 30 mg qd | EXPERIMENTAL | All participants in group 2a who received VX-661 30 mg tablet orally qd and placebo matched to Ivacaftor tablet every 12 hours (q12h) for up to 28 days. |
| Group 2b: VX-661 10 mg qd/Ivacaftor 150 mg q12h | EXPERIMENTAL | All participants in group 2b who received VX-661 10 mg tablet qd and Ivacaftor 150 mg tablet q12h orally for up to 28 days. |
| Group 3a: VX-661 100 mg qd | EXPERIMENTAL | All participants in group 3a who received VX-661 100 mg tablet orally qd and placebo matched to Ivacaftor tablet q12h for up to 28 days. |
| Group 3b: VX-661 30 mg qd/Ivacaftor 150 mg q12h | EXPERIMENTAL | All participants in group 3b who received VX-661 30 mg tablet qd and Ivacaftor 150 mg tablet q12h orally for up to 28 days. |
| Group 4: VX-661 100 mg qd/Ivacaftor 150 mg q12h | EXPERIMENTAL | All participants in group 4 who received VX-661 100 mg tablet qd and Ivacaftor 150 mg tablet q12h orally for up to 28 days. |
| Group 5a: VX-661 150 mg qd | EXPERIMENTAL | All participants in group 5a who received VX-661 150 mg tablet orally qd for up to 28 days. |
| Group 5b: VX-661 150 mg qd/Ivacaftor 150 mg q12h | EXPERIMENTAL | All participants in group 5b who received VX-661 150 mg tablet qd and Ivacaftor 150 mg tablet q12h orally for up to 28 days. |
| Group 6a: VX-661 100 mg qd/Ivacaftor 50 mg q12h | EXPERIMENTAL | All participants in group 6a who received VX-661 100 mg tablet qd and Ivacaftor 50 mg tablet q12h orally for up to 28 days. |
| Group 6d: VX-661 50 mg q12h/Ivacaftor 150 mg q12h | EXPERIMENTAL | All participants in group 6d who received VX-661 50 mg tablet and Ivacaftor 150 mg tablet q12h orally for up to 28 days. |
| Group 7: Placebo | PLACEBO_COMPARATOR | All participants in group 7 who received placebo matched to VX-661 tablet orally qd in combination with physician-prescribed Kalydeco (Ivacaftor) for up to 28 days. |
| Group 7: VX-661 100 mg qd | EXPERIMENTAL | All participants in group 7 who received VX-661 100 mg tablet orally qd in combination with physician-prescribed Kalydeco (Ivacaftor) for up to 28 days. |
| Name | Type | Description |
|---|---|---|
| VX-661 plus ivacaftor combination | DRUG | - |
| Ivacaftor | DRUG | - |
| Placebo (matched to VX-661 plus ivacaftor combination) | DRUG | - |
| Placebo (matched to ivacaftor) | DRUG | - |
| VX-661/Ivacaftor | DRUG | Fixed dose combination tablet, oral use |
| Placebo matched to VX-661/ ivacaftor | DRUG | Fixed dose combination tablet, oral use |
| Placebo matched to Ivacaftor | DRUG | Tablet, oral use |
| VX-661 Plus Ivacaftor Combination Placebo | DRUG | FDC tablet, oral use |
| Ivacaftor placebo | DRUG | Tablet, oral use |
| VX-661 | DRUG | Tablet, oral use |
| Placebo matched to VX-661 | DRUG | Tablet, oral use |
Inclusion Criteria: * Confirmed diagnosis of CF defined as a sweat chloride value greater than or equal to (\>=)60 millimole per liter (mmol/L) by quantitative pilocarpine iontophoresis. * Heterozygous for the F508del-CFTR mutation and with a second CFTR mutation that is not likely to respond to VX...
| 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-661 is an investigational small molecule being developed for cystic fibrosis. It is studied in combination with ivacaftor in patients who carry the F508del-CFTR mutation, either homozygous or heterozygous. The drug is intended to address the underlying defect in the CFTR protein.
VX-661 targets the cystic fibrosis transmembrane conductance regulator (CFTR) protein. It is designed to correct the folding and trafficking defect caused by the F508del mutation, thereby improving the function of the CFTR channel. It is used in combination with ivacaftor, which potentiates the channel.
VX-661 is being developed by Vertex Pharmaceuticals Incorporated, traded on the NASDAQ under the ticker VRTX. The company is conducting clinical trials of VX-661 in combination with ivacaftor for cystic fibrosis.
VX-661 is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. Vertex Pharmaceuticals has completed five clinical trials, including Phase 2 and Phase 3 studies, evaluating VX-661 in combination with ivacaftor for cystic fibrosis.
VX-661 has been studied in five completed clinical trials, including NCT01531673, NCT02070744, NCT02392234, and NCT02516410. These trials evaluated VX-661 alone and in combination with ivacaftor in patients with cystic fibrosis who are homozygous or heterozygous for the F508del-CFTR mutation.
VX-661 is also known as tezacaftor. It is a CFTR corrector developed by Vertex Pharmaceuticals. In clinical trials, it is studied in combination with ivacaftor, and the combination is sometimes referred to as tezacaftor/ivacaftor.