Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VX-371+ HS · 1 trial · 1 indication
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs included abnormal clinically significant findings for spirometry, clinical laboratory parameters, standard 12-lead electrocardiograms (ECGs), vital signs and ophthalmologic examinations. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent were events between first dose of study drug and up to 112 days (up to 28 days after last dose) that were absent before treatment or that worsened relative to pretreatment state. TEAEs included both serious and non-serious TEAEs.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Study Baseline was defined as the most recent non-missing measurement before the first dose of inhaled study drug in the study. Day 28 measurements after treatment discontinuation from the treatment period in which discontinuation occurred were included in the analysis.
| Arm | Type | Description |
|---|---|---|
| Sequence 1: VX-371 + Hypertonic Saline (HS), then HS | EXPERIMENTAL | Participants received 85 microgram (mcg) VX-371 diluted in 3 milliliter (mL) 4.2 percent (%) HS through oral nebulized inhalation twice daily for 28 days (Day 1 to Day 28) in treatment period 1 followed by a 28 days washout period (Day 29 to Day 56) and then received 3 mL 4.2% HS through oral nebulized inhalation twice daily for 28 days (Day 57 to Day 84) in treatment period 2. Participants also received a stable dose of 2 tablets of Orkambi (lumacaftor 400 milligram (mg)/ivacaftor 250 mg) orally every 12 hours with fat-containing food throughout the study as part of their cystic fibrosis (CF) standard of care. |
| Sequence 2: HS, then VX-371 + HS | EXPERIMENTAL | Participants received 3 mL 4.2% HS through oral nebulized inhalation twice daily for 28 days (Day 1 to Day 28) in treatment period 1 followed by a 28 days washout period (Day 29 to Day 56) and then received 85 mcg VX-371 diluted in 3 mL 4.2% HS through oral nebulized inhalation twice daily for 28 days (Day 57 to Day 84) in treatment period 2. Participants also received a stable dose of 2 tablets of Orkambi (lumacaftor 400 mg/ivacaftor 250 mg) orally every 12 hours with fat-containing food throughout the study as part of their CF standard of care. |
| Sequence 3: VX-371 + Placebo, then Placebo | EXPERIMENTAL | Participants received 85 mcg VX-371 diluted in 3 mL 0.17% Saline (placebo) through oral nebulized inhalation twice daily for 28 days (Day 1 to Day 28) in treatment period 1 followed by a 28 days washout period (Day 29 to Day 56) and then received 3 mL 0.17% saline (placebo) through oral nebulized inhalation twice daily for 28 days (Day 57 to Day 84) in treatment period 2. Participants also received a stable dose of 2 tablets of Orkambi (lumacaftor 400 mg/ivacaftor 250 mg) orally every 12 hours with fat-containing food throughout the study as part of their CF standard of care. |
| Sequence 4: Placebo, then VX-371 + Placebo | EXPERIMENTAL | Participants received 3 mL 0.17% saline (placebo) through oral nebulized inhalation twice daily for 28 days (Day 1 to Day 28) in treatment period 1 followed by a 28 days washout period (Day 29 to Day 56) and then received 85 mcg VX-371 diluted in 3 mL 0.17% saline (placebo) through oral nebulized inhalation twice daily for 28 days (Day 57 to Day 84) in treatment period 2. Participants also received a stable dose of 2 tablets of Orkambi (lumacaftor 400 mg/ivacaftor 250 mg) orally every 12 hours with fat-containing food throughout the study as part of their CF standard of care. |
| Name | Type | Description |
|---|---|---|
| VX-371 + HS | DRUG | - |
| Hypertonic saline | DRUG | - |
| Placebo | DRUG | - |
| Orkambi | DRUG | - |
| VX-371 | DRUG | - |
Inclusion Criteria: * Willing and able to use the delivery device as directed by the study manual. * Confirmed diagnosis of CF, defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis. * Homozygous for the F508del CFTR mutation. If the CFTR screening genotype result i...
| 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-371 is an investigational small molecule being studied for cystic fibrosis and primary ciliary dyskinesia. It has been evaluated in Phase 2 clinical trials for these conditions. The drug is not approved and remains in clinical development.
VX-371 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker VRTX. Vertex has sponsored Phase 2 clinical trials of VX-371 in cystic fibrosis and primary ciliary dyskinesia.
VX-371 is in Phase 2 clinical development. Two Phase 2 trials have been completed, one in cystic fibrosis and one in primary ciliary dyskinesia. VX-371 is an investigational drug and has not been approved by regulatory authorities.
VX-371 has been studied in two completed Phase 2 trials. NCT02709109 evaluated VX-371 in 147 patients with cystic fibrosis who are homozygous for the F508del-CFTR mutation. NCT02871778 evaluated VX-371 in 123 patients with primary ciliary dyskinesia. Both trials were randomized, double-blind, and placebo-controlled.
VX-371 is also known by other names, though the specific alternative names are not publicly listed in the trial records. Researchers and clinicians may refer to the drug by its code name VX-371 in publications and clinical trial registries.