New 4-amino piperidine derivative used in pharmaceutical composition for inhibiting serine palmitoyltransferase in a subject, reducing elevated ceramide levels, treating a subject with a disease characterized by elevated ceramide levels, the disease is a muscular dystrophy, Neuropathy, ataxia
2024-10-21
专利权人INTONATION RES LAB PRIVATE LTD (INTO-Non-standard)
申请日期2024-10-21
专利号IN202441079852-A
成果简介NOVELTY - 4-Amino piperidine derivative (I) or its salt, is new. USE - 4-Amino piperidine derivative (I) used in pharmaceutical composition for inhibiting serine palmitoyltransferase in a subject, reducing elevated ceramide levels, treating a subject with a disease characterized by elevated ceramide levels, or a disease or condition mediated by serine palmitoyltransferase, the disease or condition is a muscular dystrophy, an inflammatory myopathy, a metabolic myopathy or a myofibrillar myopathy, the muscular dystrophy is myotonic muscular dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Facioscapulohumeral Dystrophy, Congenital Muscular Dystrophy, Oculopharyngeal Muscular Dystrophy, Distal Muscular Dystrophy or Emery-Dreifuss Muscular Dystrophy; the inflammatory myopathy is polymyositis, dermatomyositis, inclusion body myositis, necrotizing autoimmune myopathy and inclusion body myopathy (IBM) associated with Paget disease of the bone (PDB) or frontotemporal dementia (FTD); the metabolic myopathy is Kearns-Sayre syndrome (KSS), Leigh syndrome, Mitochondrial DNA (mtDNA) depletion syndrome, Mitochondrial encephalopathy, lactic acidosis and stroke-like episodes (MELAS) syndrome, Mitochondrial neurogastrointestinal encephalopathy (MNGIE), Myoclonic epilepsy with ragged red fibers (MERRF), Neuropathy, ataxia and retinitis pigmentosa (NARP) syndrome, Pearson syndrome, Progressive external ophthalmoplegia (PEO) or sarcopenia; and the myofibrillar myopathy is plectinopathy, desminopathy, α-β-crystallinopathy, myotilinopathy, filaminopathy, zaspopathy or nemaline myopathy, amyloidosis, Facioscapulohumeral Dystrophy, myotonic dystrophy, Oculopharyngeal Muscular Dystrophy, mitochondrial myopathy, ICU sarcopenia, age related sarcopenia, αβ-crystallinopathy, nemaline myopathy, a myofibrillar myopathy and amyloidosis, sarcopenia, such as age related sarcopenia, lysosomal storage disease, Aspartylglucosaminuria, Batten Disease, Cystinosis, Fabry Disease, Gaucher Disease Types I, II, or III, Glycogen Storage Disease II (Pompe Disease), GM2-Gangliosidosis Type I (Tay Sachs Disease), GM2- Gangliosidosis Type II (Sandhoff Disease), Metachromatic Leukodystrophy, Mucolipidosis Types I, II/III or IV, Mucopolysaccharide Storage Diseases (Hurler Disease and variants, Hunter syndrome, Sanfilippo Types A, B, C or D, Morquio Types A or B, Maroteaux-Lamy or Sly diseases), Niemann-Pick Disease Types A/B, C1 or C2 or Schindler disease Types I or II (all claimed). No biological data given. ADVANTAGE - None given. DETAILED DESCRIPTION - 4-Amino piperidine derivative of formula (I) or its salt, is new. X, Z=C(O) and S(O)2; Y, Y1=bond and NH; Ar1=optionally substituted 6-10 membered aromatic carbocyclylene, an optionally substituted 5-10 membered heteroarylene or an optionally substituted 3-10 membered heterocyclylene; Ar2=optionally substituted 6-10 membered aromatic carbocyclylene or optionally substituted 5-10 membered heteroarylene; and R1=1-6C alkyl, (CH2)x3-7C cycloalkyl, (CH2)xphenyl, (CH2)x(3-7 membered heterocyclyl) or (CH2)x(5-6 membered heteroaryl); where the 1-6C alkyl, (CH2)x3-7C cycloalkyl, (CH2)xphenyl, (CH2)x(3-7 membered heterocyclyl) and the (CH2)x(5-6 membered heteroaryl) are optionally substituted with one or more groups selected from halo, ORa , NRaR b, S(O)iR a , NRaS(O)iR a , S(O)iNRaR b, C(=O)ORa , OC(=O)ORa , C(=S)ORa , O(C=S)Ra , C(=O)NRaR b, NRaC(=O)Rb, C(=S)NRaR b, NRaC(=S)Rb, NRa (C=O)ORb, O(C=O)NRaR b, NRa (C=S)ORb, O(C=S)NRaR b, NRa (C=O)NRbR c , NRa (C=S)NRbR c , C(=S)Ra , C(=O)Ra and 1-6C alkyl; L1, L3=O-, -N-, -S-, -SO2-, -SO3-, -SO2NH-, - NHSO2-, -(CH2)1-4, -O(CH2)1-4-, -(CH2)1-4-O-, -O(CH2)2-4O-, -NH(CH2)1-4-, -(CH2)1-4-NH-, and - NH(CH2)2-4NH-; L2=2-6C alkenylene, 2-6C alkenylene oxide, 1-6C alkylene, 3-7C cycloalkylene, phenylene, 3-7 membered heterocyclylene or 5-6 membered heteroarylene; where the 1-6C alkylene, 2-6C alkenylene, 3-7C cycloalkylene, phenylene, 3-7 membered heterocyclylene, and the 5-6 membered heteroarylene are optionally substituted with one or more groups selected from halo, ORa , NRaR b, S(O)iR a , NRaS(O)iR a , S(O)iNRaR b, C(=O)ORa , OC(=O)ORa , C(=S)ORa , O(C=S)Ra , C(=O)NRaR b, NRaC(=O)Rb, C(=S)NRaR b, NRaC(=S)Rb, NRa (C=O)ORb, O(C=O)NRaR b, NRa (C=S)ORb, O(C=S)NRaR b, NRa (C=O)NRbR c , NRa (C=S)NRbR c , C(=S)Ra , C(=O)Ra and 1-6C alkyl; Ra, Rb, Rc=H and 1-3C alkyl; i=0, 1, 2, 3 or 4 ; and x=0 or 1.
IPC 分类号A61K-031/133 ; A61K-031/195 ; A61K-031/198 ; A61P-035/00
国家印度
专业领域医药卫生
语种英语
成果类型专利
文献类型科技成果
条目标识符http://119.78.100.226:8889/handle/3KE4DYBR/14820
专题中国科学院新疆生态与地理研究所
作者单位
INTONATION RES LAB PRIVATE LTD (INTO-Non-standard)
推荐引用方式
GB/T 7714
SAMEER,LSPVV L,POTLURI V,et al. New 4-amino piperidine derivative used in pharmaceutical composition for inhibiting serine palmitoyltransferase in a subject, reducing elevated ceramide levels, treating a subject with a disease characterized by elevated ceramide levels, the disease is a muscular dystrophy, Neuropathy, ataxia. IN202441079852-A[P]. 2024.
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