It’s only fair to share… Tambiciclib CAS 2247481-08-7 MF C25H35ClN6O2S, 519.10 4-[[[4-[5-chloro-2-[[4-[[(2R)-1-methoxypropan-2-yl]amino]cyclohexyl]amino]-4-pyridinyl]-1,3-thiazol-2-yl]amino]methyl]oxane-4-carbonitrile 2H-PYRAN-4-CARBONITRILE, 4-(((4-(5-CHLORO-2-((TRANS-4-(((1R)-2-METHOXY-1-METHYLETHYL)AMINO)CYCLOHEXYL)AMINO)-4-PYRIDINYL)-2-THIAZOLYL)AMINO)METHYL)TETRAHYDRO- cyclin-dependent kinase inhibitor, antineoplastic, GFH 009, JSH 009, XDZ7VK8CXC, Orphan Drug , Acute myeloid leukaemia, Peripheral T-cell lymphoma Tambiciclib (GFH009, JSH-009) is an orally active, highly potent and selective CDK9 inhibitor (IC50 = 1 nM), demonstrating >200-fold selectivity over other CDKs, >100-fold selectivity over DYRK1A/B, and excellent …
Tag «orphan drug»
Privosegtor
It’s only fair to share… Privosegtor CAS 1361200-34-1 MF C25H38FN5O4, MW 491.6 g/mol GLYCINAMIDE, N-(2-(2-FLUOROPHENYL)ETHYL)GLYCYL-N-(2-METHYLPROPYL)GLYCYL-N2-(3-(2-OXO-1-PYRROLIDINYL)PROPYL)-N-(2-(2-FLUOROPHENYL)ETHYL)GLYCYL-N-(2-METHYLPROPYL)GLYCYL-N2-(3-(2-OXO-1-PYRROLIDINYL)PROPYL)GLYCINAMIDEN-(2-(2-FLUOROPHENYL)ETHYL)GLYCYL-N-(2-METHYLPROPYL)GLYCYL-N2-(3-(2-OXOPYRROLIDIN-1-YL)PROPYL)GLYCINAMIDE N-[2-(2-fluorophenyl)ethyl]glycyl-N-(2-methylpropyl)glycyl-N2[3-(2-oxopyrrolidin-1-yl)propyl]glycinamideserum/ glucocorticoid-regulated kinase 2 (Sgk2) activator, Phase 2, Optic neuritis, orphan drug, BN-201, BN 201, G-79, G 79, KCN37L7EIH OCS-05 in Patients With Optic Neuritis CTID: NCT04762017 Phase: Phase 2 Status: Completed Date: 2025-09-22 N-[2-[(2-amino-2-oxoethyl)-[3-(2-oxopyrrolidin-1-yl)propyl]amino]-2-oxoethyl]-2-[2-(2-fluorophenyl)ethylamino]-N-(2-methylpropyl)acetamide (BN201) is a small peptide molecule, a first-in-class neuroprotective compound. …
Nuvisertib
It’s only fair to share… Nuvisertib CAS 1361951-15-6 MF C22H26ClF3N4O MW418.5 g/mol 2-[(1r,4r)-4-({3-[3-(trifluoromethyl)phenyl]imidazo[1,2-b]pyridazin-6-yl}amino)cyclohexyl]propan-2-olserine/ threonine kinase inhibitor, antineoplastic, Orphan Drug, myelofibrosis, SGI-9481, SGI 9481, TP-3654, TP 3654, EOB0N7BOY4 The chemical structure for nuvisertib was obtained from proposed INN list 130 (Feb. 2024), in which the compound is described as a serine/ threonine kinase inhibitor with antineoplastic action. …
Lirodegimod
It’s only fair to share… Lirodegimod CAS 2502186-79-8 MF C60H74ClN10O14PS, MW 1257.79 [2-[[(5S,8S,10aR)-3-acetyl-8-[[(2S)-5-amino-1-[2-chloro-3-[4-[[(2S)-1-[(2S,4R)-4-hydroxy-2-[[(1S)-1-[4-(4-methyl-1,3-thiazol-5-yl)phenyl]ethyl]carbamoyl]pyrrolidin-1-yl]-3,3-dimethyl-1-oxobutan-2-yl]amino]-4-oxobutyl]phenoxy]-5-oxopentan-2-yl]carbamoyl]-6-oxo-1,2,4,5,8,9,10,10a-octahydropyrrolo[1,2-a][1,5]diazocin-5-yl]carbamoyl]-1H-indole-5-carbonyl]phosphonic acid L-Prolinamide, N-[4-[3-[[(2S)-2-[[[(5S,8S,10aR)-3-acetyldecahydro-5-[[[5-(phosphonocarbonyl)-1H-indol-2-yl]carbonyl]amino]pyrrolo[1,2-a][1,5]diazocin-8-yl]carbonyl]amino]-5-amino-5-oxopentyl]oxy]-2-chlorophenyl]-1-oxobutyl]-3-methyl-L-valyl-4-hydroxy-N-[(1S)-1-[4-(4-methyl-5-thiazolyl)phenyl]ethyl]-, (4R)- KT 333, KT333, ANTINEOPLASTIC, Fast Track (United States), Orphan Drug (United States), 4Q6ZHJ2MNALirodegimod is a small molecule drug. The usage of the INN stem ‘-imod’ in the name indicates that Lirodegimod is a immunomodulator, both stimulant/suppressive and stimulant. Lirodegimod …
Gildeuretinol
It’s only fair to share… Gildeuretinol Retin-20,20,20-d3-ol CAS118139-35-8 MF C20H272H3O, MW 289.5 g/mol (2E,4E,6E,8E)-3-(2H3)methyl-7-methyl-9-(2,6,6-trimethylcyclohex-1-en-1-yl)nona-2,4,6,8-tetraen-1-ol; (20,20,20-2H3)retinol (2E,4E,6E,8E)-7-methyl-3-(trideuteriomethyl)-9-(2,6,6-trimethylcyclohexen-1-yl)nona-2,4,6,8-tetraen-1-olvitamin A analogue, Orphan Drug, Stargardt disease, breakthrough therapy, Pediatric Rare Disease designations, ALK-001, KL-49, ALK 001, KL 49 Gildeuretinol is an investigational new drug being developed by Alkeus Pharmaceuticals, Inc. for the treatment of retinal diseases, particularly Stargardt disease and geographic atrophy secondary to age-related macular degeneration (AMD). Stargardt disease is …
Claziprotamide
It’s only fair to share… Claziprotamide CAS 2361124-03-8, BBP 671 MF C19H20ClFN4O MW374.8 g/mol 1-[4-(6-chloropyridazin-3-yl)piperazin-1-yl]-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-one 1-[4-(6-chloropyridazin-3-yl)piperazin-1-yl]-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-onepantothenate kinases 1 and 3 (PanK1 and PanK3) positive allosteric modulator Claziprotamide is an investigational new drug that is being evaluated for the treatment of rare metabolic disorders such as pantothenate kinase-associated neurodegeneration (PKAN) and neurodegeneration with brain iron accumulation (NBIA). It acts as a positive allosteric modulator …
Sparsentan
It’s only fair to share… Sparsentan (PS433540,RE-021) C32H40N4O5S Average mass592.749 FDA APPROVED 2023/2/17, Filspari 4′-((2-butyl-4-oxo-1,3-diazaspiro[4.4]non-1-en-3-yl)methyl)-N-(4,5-dimethylisoxazol-3-yl)-2′-(ethoxymethyl)-[1,1′-biphenyl]-2-sulfonamide 4′-[(2-Butyl-4-oxo-1.3-diazaspiro[4.41non-l-en-3-yl)methvn-N-(3,4- dimethyl-5-isoxazolyl)-2′-ethoxymethyl [ 1 , l’-biphenyll -2-sulfonamide SparsentanPS433540; RE-021, formerly known as DARACAS :254740-64-24-[(2-butyl-4-oxo-1,3-diazaspiro[4.4]non-1-en-3-yl)methyl]-N-(4,5- dimethylisoxazol-3-yl)-2-(ethoxymethyl)biphenyl-2-sulfonamideMechanism of Action:acting as both an Endothelin Receptor Antagonist (ERA) and Angiotensin Receptor Blocker (ARB).Indication: Focal Segmental Glomerulosclerosis (FSGS).Focal Segmental Glomerulosclerosis (FSGS) is a rare …
Tebapivat
It’s only fair to share… Tebapivat CAS 2283422-04-6 WeightAverage: 392.44Monoisotopic: 392.116778341 Chemical FormulaC18H16N8OS 10-[(6-aminopyridin-2-yl)methyl]-7-methyl-4-(1H-pyrazol-5-ylmethyl)-3-thia-5,7,10,11-tetrazatricyclo[6.4.0.02,6]dodeca-1(8),2(6),4,11-tetraen-9-one6-[(6-aminopyridin-2-yl)methyl]-4-methyl-2-[(1H-pyrazol-3-yl)methyl]-4,6-dihydro-5H-[1,3]thiazolo[5′,4′:4,5]pyrrolo[2,3-d]pyridazin-5-one 6-[(6-aminopyridin-2-yl)methyl]-4-methyl-2-[(1H-pyrazol-3-yl)methyl]-4,6-dihydro-5H-[1,3]thiazolo[5′,4′:4,5]pyrrolo[2,3-d]pyridazin-5-one Tebapivat is under investigation in clinical trial NCT05490446 (A Study of Tebapivat (AG-946) in Participants With Anemia Due to Lower-risk Myelodysplastic Syndromes (LR-MDS)). Tebapivat is an orally available activator of the red cell isoform of pyruvate kinase (PK-R; PKR), with potential to …
HEXASODIUM PHYTATE
It’s only fair to share… HEXASODIUM PHYTATE cas 34367-89-0 myo-Inositol, 1,2,3,4,5,6-hexakis(dihydrogen phosphate) sodium salt (1:6) hexasodium;[2,3,4,5,6-pentakis[[hydroxy(oxido)phosphoryl]oxy]cyclohexyl] hydrogen phosphate free form RN: 83-86-3 C6H12Na6O24P6, 791.93 x Na salt 14306-25-3 C6H18O24P6.xNa free form : 83-86-3 myo-Inositol, 1,2,3,4,5,6-hexakis(dihydrogen phosphate), sodium salt (1R,2R,3S,4S,5R,6S)-CYCLOHEXANE-1,2,3,4,5,6-HEXAYL-HEXAKIS(DIHYDROGEN PHOSPHATE) INOSITOL, HEXAKIS(DIHYDROGEN PHOSPHATE) HEXASODIUM SALT, MYO- Hexasodium phytate (also known as SNF472) is a compound being developed as …
Clofutriben
It’s only fair to share… Clofutriben Cas 1204178-50-6 HCL 1203941-88-1 Molecular Weight 424.80 Formula C19H16ClF3N4O2 Clofutriben (ASP3662) is a 11β-hydroxysteroid dehydrogenase type 1 inhibitor. Clofutriben is an orally bioavailable selective inhibitor of the enzyme 11beta-hydroxysteroid dehydrogenase type 1 (11b-HSD1; 11bHSD1; HSD11B1; HSD1; HSD-1), with potential protective activity for disorders of corticosteroid excess. Upon oral administration, …