Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Voxelotor · 5 trials · 1 indication
Number of participants with increase in Hb \>1 g/dL from Baseline to Week 24
AUCt was calculated using the linear/log trapezoidal rule.
AUCt was calculated using the linear/log trapezoid rule.
AUCt was calculated using the linear/log trapezoid rule.
AUCt was calculated using the linear/log trapezoid rule.
AUCt was calculated using the linear/log trapezoid rule.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCt was calculated using the linear/log trapezoid rule.
AUCt was calculated using the linear/log trapezoid rule.
AUCt was calculated using the linear/log trapezoid rule.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
AUCinf was calculated as AUClast + Clast/lamda z, where AUClast: area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration. Clast is the last measurable concentration and lamda z: apparent terminal elimination rate constant.
| Arm | Type | Description |
|---|---|---|
| Dose 1 | ACTIVE_COMPARATOR | voxelotor |
| Dose 2 | ACTIVE_COMPARATOR | voxelotor |
| Placebo | PLACEBO_COMPARATOR | Placebo |
| Part A | EXPERIMENTAL | To evaluate the effect of multiple doses of voxelotor on the plasma pharmacokinetics (PK) of a single dose of bupropion, repaglinide, flurbiprofen, omeprazole, and midazolam |
| Part B | EXPERIMENTAL | To evaluate the effect of multiple doses of voxelotor on the plasma PK of a single dose of metformin, furosemide, and rosuvastatin |
| Name | Type | Description |
|---|---|---|
| voxelotor | DRUG | - |
| Placebo | OTHER | - |
| GBT440 | DRUG | Oral drug |
| Bupropion | DRUG | Drug drug interaction |
| Repaglinide | DRUG | Drug drug interaction |
| Flurbiprofen | DRUG | Drug drug interaction |
| Omeprazole | DRUG | Drug drug interaction |
| Midazolam | DRUG | Drug drug interaction |
| Metformin | DRUG | Drug drug interaction |
| Furosemide | DRUG | Drug drug interaction |
| Rosuvastatin | DRUG | Drug drug interaction |
Inclusion Criteria: 1. Male or female study participants with sickle cell disease 2. Participants have had at least 1 episode of vaso-occlusive crisis (VOC) in the past 12 months. 3. Age 12 to 65 years 4. Hemoglobin (Hb) ≥5.5 and ≤10.5 g/dL during screening 5. For participants taking hydroxyurea (H...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 5 | PHASE3 | Etavopivat Low dose |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, 20vPCV |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Agios Pharmaceuticals, Inc. | AGIO | 2 | PHASE2 | Mitapivat |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470, Famotidine, Pantoprazole |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
GBT440 is an investigational small molecule being studied for sickle cell disease, anemia of sickle cell disease, and idiopathic pulmonary fibrosis with hypoxemia. It has also been evaluated in healthy subjects for pharmacokinetic studies. The drug is in clinical development and is not approved by the FDA.
GBT440 is being developed by Pfizer, Inc., which trades on the New York Stock Exchange under the ticker PFE. Pfizer is the sponsor of the clinical trials for this investigational drug.
GBT440 has been studied in Phase 1 and Phase 2 clinical trials. The Phase 2 trial evaluated the drug in patients with idiopathic pulmonary fibrosis and hypoxemia. All five clinical trials for GBT440 have been completed, and the drug remains investigational.
GBT440 has been studied in five completed clinical trials. These include NCT02497924, a study of absorption, metabolism, and excretion in healthy subjects; NCT02567695, a bioavailability study in healthy subjects; NCT02846324, a safety and tolerability study in patients with idiopathic pulmonary fibrosis; and NCT05981365, a drug interaction study in healthy subjects.
GBT440 is also known as voxelotor. One of its clinical trials, NCT05981365, is titled "Voxelotor CYP and Transporter Cocktail Interaction Study," confirming that GBT440 and voxelotor refer to the same drug.