| Size | Price | Stock | Qty |
|---|---|---|---|
| 1mg |
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| Other Sizes |
|
| Targets |
CFTR/cystic fibrosis transmembrane conductance regulator
|
|---|---|
| ln Vitro |
Bamocaftor is a CFTR corrector intended to repair the function of the F508del-CFTR protein [1].
|
| ln Vivo |
Presently, elexacaftor (VX-445) and bamocaftor (VX-659) are two new compounds being tested in phase 3 trials. Both drugs are next-generation CFTR correctors designed to restore F508del-CFTR protein function in patients with CF when administered with tezacaftor and ivacaftor. Furthermore, positive data has been reported in CF patients who are heterozygous for the F508del CFTR mutation or have one minimal function (MF) mutation (F508del-MF). In the F508del-MF group, treatment with either ivacaftor + tezacaftor + elexacaftor or bamocaftor resulted in mean absolute improvements in ppFEV1 of 13.8 and 13.3 percentage points, respectively (P < 0.001). In the F508del cohort, patients received standard ivacaftor–tezacaftor treatment. In this cohort the addition of elexacaftor and bamocaftor resulted in an 11.0-point and 9.7-point rise in the percentage of predicted FEV1 (P < 0.001), respectively[1].
|
| Toxicity/Toxicokinetics |
The side effects most commonly observed were increased sputum production, pulmonary exacerbations, hemoptysis, and pyrexia. Generally speaking, the double or triple combination regimens were well tolerated across all tezacaftor trials. On the basis of these results, the triple combination of elexacaftor + tezacaftor + ivacaftor was submitted for global regulatory approval in 2019. The submission is a major step toward the goal of bringing treatments to the largest remaining group of people with CF that still do not have an approved medicine yet, as well as toward providing significantly enhanced benefits to patients that are homozygous for the F508del CFTR mutation.
|
| References | |
| Additional Infomation |
Bamocaftor (VX-659) is under investigation in clinical trial NCT03224351 (A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis).
|
| Molecular Formula |
C28H32F3N5O4S
|
|---|---|
| Molecular Weight |
591.6450
|
| Exact Mass |
591.212
|
| Elemental Analysis |
C, 56.84; H, 5.45; F, 9.63; N, 11.84; O, 10.82; S, 5.42
|
| CAS # |
2204245-48-5
|
| Related CAS # |
2204245-47-4 (potassium);2204245-48-5;
|
| PubChem CID |
134393443
|
| Appearance |
White to off-white solid powder
|
| Density |
1.4±0.1 g/cm3
|
| Index of Refraction |
1.622
|
| LogP |
5.71
|
| Hydrogen Bond Donor Count |
1
|
| Hydrogen Bond Acceptor Count |
10
|
| Rotatable Bond Count |
9
|
| Heavy Atom Count |
41
|
| Complexity |
1040
|
| Defined Atom Stereocenter Count |
1
|
| SMILES |
C[C@H]1CC(N(C1)C2=C(C=CC(=N2)N3C=CC(=N3)OCCC4(CC4)C(F)(F)F)C(=O)NS(=O)(=O)C5=CC=CC=C5)(C)C
|
| InChi Key |
IGEOJNMYRZUKIK-IBGZPJMESA-N
|
| InChi Code |
InChI=1S/C28H32F3N5O4S/c1-19-17-26(2,3)35(18-19)24-21(25(37)34-41(38,39)20-7-5-4-6-8-20)9-10-22(32-24)36-15-11-23(33-36)40-16-14-27(12-13-27)28(29,30)31/h4-11,15,19H,12-14,16-18H2,1-3H3,(H,34,37)/t19-/m0/s1
|
| Chemical Name |
N-(benzenesulfonyl)-6-[3-[2-[1-(trifluoromethyl)cyclopropyl]ethoxy]pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide
|
| Synonyms |
Bamocaftor; VX-659; 2204245-48-5; Bamocaftor [USAN]; VX659; UNII-8C7XEW3K7S; 8C7XEW3K7S; Bamocaftor (USAN);
|
| HS Tariff Code |
2934.99.9001
|
| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
|
| Solubility (In Vitro) |
DMSO : ~100 mg/mL (~169.02 mM)
|
|---|---|
| Solubility (In Vivo) |
Solubility in Formulation 1: ≥ 2.5 mg/mL (4.23 mM) (saturation unknown) in 10% DMSO + 90% (20% SBE-β-CD in Saline) (add these co-solvents sequentially from left to right, and one by one), clear solution.
For example, if 1 mL of working solution is to be prepared, you can add 100 μL of 25.0 mg/mL clear DMSO stock solution to 900 μL of 20% SBE-β-CD physiological saline solution and mix evenly. Preparation of 20% SBE-β-CD in Saline (4°C，1 week): Dissolve 2 g SBE-β-CD in 10 mL saline to obtain a clear solution. Solubility in Formulation 2: ≥ 2.5 mg/mL (4.23 mM) (saturation unknown) in 10% DMSO + 90% Corn Oil (add these co-solvents sequentially from left to right, and one by one), clear solution. For example, if 1 mL of working solution is to be prepared, you can add 100 μL of 25.0 mg/mL clear DMSO stock solution to 900 μL of corn oil and mix evenly. (Please use freshly prepared in vivo formulations for optimal results.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 1.6902 mL | 8.4509 mL | 16.9019 mL | |
| 5 mM | 0.3380 mL | 1.6902 mL | 3.3804 mL | |
| 10 mM | 0.1690 mL | 0.8451 mL | 1.6902 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation:：Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
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