| Size | Price | |
|---|---|---|
| 500mg | ||
| 1g | ||
| 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].
|
| References | |
| Additional Infomation |
Pharmacological correction of the defective ion channel with cystic fibrosis transmembrane conductance regulator (CFTR) has become an attractive approach to therapy directed at the root cause of the life-limiting disease cystic fibrosis (CF). CFTR defects range from absence, misfolding, and resulting degradation to functional defects of the CFTR protein. The discovery and development of the CFTR potentiator ivacaftor was a major break-through in CF therapy and has triggered an enormous incentive for seeking effective modulators such as lumacaftor, tezacaftor or elexacaftor for all patients with CF. A number of emerging CFTR modulators are currently in the development pipeline, and rescue levels of CFTR protein approach a cure for cystic fibrosis. In this review, we identify and characterize all preclinical and clinical emerging CFTR modulators and discuss the in vitro pharmacology, looking at CFTR protein expression and chloride transport and the translation to the clinic. The new emerging CFTR modulators could offer new therapeutic solutions for CF patients.[1]
|
| Molecular Formula |
C28H31F3KN5O4S
|
|---|---|
| Molecular Weight |
629.73535656929
|
| Exact Mass |
629.168
|
| Elemental Analysis |
C, 53.40; H, 4.96; F, 9.05; K, 6.21; N, 11.12; O, 10.16; S, 5.09
|
| CAS # |
2204245-47-4
|
| Related CAS # |
2204245-48-5; 2204245-47-4 (potassium);
|
| PubChem CID |
134687598
|
| Appearance |
Typically exists as solid at room temperature
|
| Hydrogen Bond Donor Count |
0
|
| Hydrogen Bond Acceptor Count |
11
|
| Rotatable Bond Count |
9
|
| Heavy Atom Count |
42
|
| Complexity |
1050
|
| Defined Atom Stereocenter Count |
1
|
| SMILES |
[K+].S(C1C=CC=CC=1)([N-]C(C1=CC=C(N2C=CC(=N2)OCCC2(C(F)(F)F)CC2)N=C1N1C[C@@H](C)CC1(C)C)=O)(=O)=O
|
| InChi Key |
BWMSVLSYIQHCQP-FYZYNONXSA-M
|
| InChi Code |
InChI=1S/C28H32F3N5O4S.K/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);/q;+1/p-1/t19-;/m0./s1
|
| Chemical Name |
potassium;benzenesulfonyl-[6-[3-[2-[1-(trifluoromethyl)cyclopropyl]ethoxy]pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carbonyl]azanide
|
| Synonyms |
Bamocaftor potassium; VX-659 potassium; VX-659 potassium salt; Bamocaftor potassium [USAN]; 2204245-47-4; UNII-VY7D8MTV72; VY7D8MTV72; Bamocaftor potassium (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) |
Typically soluble in DMSO (e.g. 10 mM)
|
|---|---|
| Solubility (In Vivo) |
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.
Injection Formulations
Injection Formulation 1: DMSO : Tween 80： Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution. Injection Formulation 2: DMSO : PEG300 ：Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO → 400 μLPEG300 → 50 μL Tween 80 → 450 μL Saline) Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO → 900 μL Corn oil) Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals). View More
Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO → 900 μL (20% SBE-β-CD in saline)] Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium) Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals). View More
Oral Formulation 3: Dissolved in PEG400 (Please use freshly prepared in vivo formulations for optimal results.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 1.5880 mL | 7.9398 mL | 15.8796 mL | |
| 5 mM | 0.3176 mL | 1.5880 mL | 3.1759 mL | |
| 10 mM | 0.1588 mL | 0.7940 mL | 1.5880 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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