It’s only fair to share… Amezalpat CAS 1616372-41-8 MF C34H41N3O4 MW555.7 g/mol 2-[5-[3-[3-[1-[(4-tert-butylphenyl)methyl]-4-ethyl-5-oxo-1,2,4-triazol-3-yl]propyl]phenyl]-2-ethoxyphenyl]acetic acid peroxisome proliferator-activated receptor alpha (PPARα) antagonist, antineoplastic, TPST 1120, FDA Fast Track, Orphan Drug, 1EQ4LQN9N3 Amezalpat (formerly TPST-1120) is an investigational, oral, small-molecule inhibitor targeting peroxisome proliferator-activated receptor alpha (PPAR being developed by Tempest Therapeutics. It works by directly targeting tumor cells and reducing immune …
Tag «orphan drug»
Repinatrabit
It’s only fair to share… Repinatrabit CAS 2837993-05-0 MF C18H22F4N4O3 MW 418.4 g/mol (3R)-3-[cyclopropyl-[[2-fluoro-4-(trifluoromethoxy)phenyl]methylcarbamoyl]amino]piperidine-1-carboxamide (3R)-3-[cyclopropyl({[2-fluoro-4-(trifluoromethoxy)phenyl]methyl}carbamoyl)amino]piperidine-1-carboxamidesolute carrier family 6 member 19 (SLC6A19) inhibitor(phenylketonuria), JNT-517, JNT 517, orphan drug, rare pediatric disease designations, Jnana Therapeutics, 5P44NDU6AC, JN 11804, JN-11804 Repinatrabit (JNT-517) is an investigational, oral, small-molecule drug developed by Jnana Therapeutics (now part of Otsuka Pharmaceutical) to treat Phenylketonuria …
Cambritaxestat
It’s only fair to share… Cambritaxestat CAS 1979939-16-6 MFC25H22ClF3N4O2 MW502.9 g/mol N-[(1S)-1-(4-chlorophenyl)ethyl]-3-[3-[[4-(trifluoromethoxy)phenyl]methyl]imidazo[4,5-b]pyridin-2-yl]propanamide N-[(1S)-1-(4-chlorophenyl)ethyl]-3-(3-{[4-(trifluoromethoxy)phenyl]methyl}-3H-imidazo[4,5-b]pyridin-2-yl)propanamideautotaxin inhibitor, antineoplastic, Orphan Drug, IOA 289, IOA-289, IOA289, LYY3P2KA27, CRT 0273750 Cambritaxestat is an autotaxin inhibitor. Cambritaxestat is an orally bioavailable small molecule inhibitor of autotaxin (ATX; ectonucleotide pyrophosphatase/phosphodiesterase family member 2; ENPP2), with potential antifibrotic and antineoplastic activities. Upon oral administration, cambritaxestat …
Tambiciclib
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 …
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 …