US2018185368A1PendingUtilityA1

Substituted pyrrolo[1,2-a]pyrimidines and their use in the treatment of medical disorders

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Assignee: LYSOSOMAL THERAPEUTICS INCPriority: Nov 6, 2014Filed: Nov 6, 2015Published: Jul 5, 2018
Est. expiryNov 6, 2034(~8.3 yrs left)· nominal 20-yr term from priority
A61K 31/519A61P 25/18A61K 31/165C07D 519/00A61P 25/16C07D 487/04
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Claims

Abstract

The invention provides substituted pyrrolo[1,2-a]pyrimidines and related organic compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary substituted pyrrolo[1,2-a]pyrimidines compounds described herein include substituted 2,4-dimethyl-N-phenylpyrrolo[1,2-a]pyrimidine-8-carboxamide compounds and variants thereof.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . A compound of Formula I: 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof, wherein: 
         R 1  and R 2  each represent independently for each occurrence hydrogen, deuterium, C 1-4  alkyl, C 1-4  haloalkyl, C 1-4  deuteroalkyl, C 1-4  alkoxyl, —(C 1-4  alkylene)-(2-6 membered heteroalkyl), cyclopropyl, cyano, halogen, hydroxyl, or —N(R 4 ) 2 ; 
         R 3  represents independently for each occurrence hydrogen, C 1-6  alkyl, or C 3-6  cycloalkyl; 
         R 4  represents independently for each occurrence hydrogen, C 1-4  alkyl, cyclopropyl, or —C(O)R 3 ; 
         R 5  represents independently for each occurrence C 1-4  alkyl or C 3-6  cycloalkyl; 
         X 1  is one of the following:
 (a) a carbonyl-containing linker selected from —C(O)N(H)-ψ, —C(O)N(H)(C 1-6  alkylene)-ψ, and —C(O)-(3-6 membered heterocycloalkylene containing at least one ring —N(H)— group)-ψ; where ψ is a bond to A 1 ; or 
 (b) an amine-containing linker selected from —(C 1-4  alkylene)-N(H)-ψ and —(C 1-4  alkylene)-N(H)—(C 1-4  alkylene)-ψ; 
 
         A 1  is a cyclic group selected from:
 C 3-10  cycloalkyl, phenyl, naphthyl, or 5-6 membered heteroaryl, each of which is substituted by 1 or 2 occurrences of Y 1  and 0, 1, 2, or 3 occurrences of Y 2 ; 
 a 5-14 membered partially unsaturated carbocyclyl, or a 3-16 membered heterocyclyl, each of which is substituted by 0, 1, or 2 occurrences of Y 1  and 0, 1, 2, or 3 occurrences of Y 2 ; and 
 phenyl substituted with 0, 1, 2, or 3 occurrences of Y 2 ; 
 
         Y 1  represents, independently for each occurrence, one of the following:
 2-8 membered heteroalkyl optionally substituted by a 6-10 membered aryl, a 3-membered heterocyclyl, or C 3-6  halocycloalkyl; 
 3-10 membered heterocyclyl, 6-10 membered aryl, C 3-7  cycloalkyl, —O—C 3-7  cycloalkyl, —O-(3-6 membered heterocyclyl), —O-(6-10 membered aryl), or —O—(C 2-6  alkynyl); 
 C 2-6  alkynyl, —C≡C—(C 1-6  alkylene)-OR 4 , —C≡C—(C 1-6  alkylene)-N(R 3 ) 2 , —(C 2-4  alkynylene)-(5-6 membered heteroaryl), or C 2-6  alkenyl; or 
 C 1-6  haloalkyl, C 1-6  alkyl, halogen, cyano, —CO 2 R 3 , —C(O)R 5 , —S(O) 2 R 5 , —C(O)N(R 5 ) 2 , —C(O)N(R 3 ) 2 , —N(R 3 )C(O)R 5 , or —O—(C 1-8  haloalkyl); 
 
         Y 2  represents, independently for each occurrence, deuterium, C 1-6  alkyl, C 3-6  cycloalkyl, halogen, C 1-6  haloalkyl, C 1-6  hydroxyalkyl, hydroxyl, C 1-6  alkoxyl, cyano, azido, —N(R 3 ) 2 , —(C 1-6  alkylene)-(5-6 membered heterocyclyl), —(C 1-6  alkylene)-CO 2 R 3 , or C 1-6  haloalkyl-substituted C 3-6  cycloalkyl; 
         m is 1 or 2; 
         n is 1, 2, or 3; and 
         provided that at least one occurrence of R 1  or R 2  is other than hydrogen when (i) A 1  is an unsubstituted heterocyclyl, (ii) A 1  is an unsubstituted phenyl or a phenyl substituted only by halogen, or (iii) Y 2  is halogen. 
       
     
     
         2 . The compound of  claim 1 , wherein R 1  represents independently for each occurrence C 1-4  alkyl, C 1-4  haloalkyl, —(C 1-4  alkylene)-(2-6 membered heteroalkyl), cyclopropyl, halogen, or —N(R 4 ) 2 . 
     
     
         3 . The compound of  claim 1 , wherein R 1  is methyl. 
     
     
         4 . The compound of any one of  claims 1 - 3 , wherein n is 2. 
     
     
         5 . The compound of  claim 4 , wherein the R 1  groups are located at the 2 and 4 positions of the pyrrolo[1,2-a]pyrimidinyl. 
     
     
         6 . The compound of any one of  claims 1 - 5 , wherein R 2  is hydrogen. 
     
     
         7 . The compound of any one of  claims 1 - 6 , wherein R 3  and R 4  each represent independently for each occurrence hydrogen, methyl, or ethyl. 
     
     
         8 . The compound of any one of  claims 1 - 7 , wherein X 1  is —C(O)N(H)-ψ. 
     
     
         9 . The compound of any one of  claims 1 - 7 , wherein X 1  is —C(O)N(H)(C 1-6  alkylene)-ψ or —C(O)-(3-6 membered heterocycloalkylene containing at least one ring —N(H)— group)-ψ. 
     
     
         10 . The compound of any one of  claims 1 - 9 , wherein A 1  is C 5-10  cycloalkyl substituted once by Y 1  and 0-1 occurrences of Y 2 . 
     
     
         11 . The compound of any one of  claims 1 - 9 , wherein A 1  is C 3-7  cycloalkyl substituted once by Y 1  and 0-1 occurrences of Y 2 . 
     
     
         12 . The compound of any one of  claims 1 - 9 , wherein A 1  is a bicyclic carbocyclyl that is partially unsaturated or a bicyclic heterocyclyl, each of which is substituted by 0 or 1 occurrence of Y 1  and 0, 1, or 2 occurrences of Y 2 . 
     
     
         13 . The compound of any one of  claims 1 - 9 , wherein A 1  is phenyl substituted once by Y 1  and 0-1 occurrences of Y 2 . 
     
     
         14 . The compound of any one of  claims 1 - 9 , wherein A 1  is a 5-6 membered heteroaryl substituted once by Y 1  and 0-1 occurrences of Y 2 . 
     
     
         15 . The compound of any one of  claims 1 - 9 , wherein A 1  is a bicyclic carbocyclyl that is partially unsaturated or a bicyclic heterocyclyl, each of which is substituted by 0 or 1 occurrence of Y 2  selected from the group consisting of C 1-6  alkyl, C 3-6  cycloalkyl, halogen, C 1-6  haloalkyl, hydroxyl, and C 1-6  alkoxyl. 
     
     
         16 . The compound of any one of  claims 1 - 9 , wherein A 1  is 
       
         
           
           
               
               
           
         
       
       wherein m is 0, 1, or 2; and Y 2  represents independently for each occurrence C 1-6  alkyl, C 3-6  cycloalkyl, halogen, C 1-6  haloalkyl, hydroxyl, or C 1-6  alkoxyl. 
     
     
         17 . The compound of any one of  claims 1 - 14 , wherein any occurrence of Y 2  is independently C 1-6  alkyl, C 3-6  cycloalkyl, halogen, C 1-6  haloalkyl, or hydroxyl. 
     
     
         18 . The compound of any one of  claims 1 - 14 , wherein any occurrence of Y 2  is independently C 1-3  alkyl. 
     
     
         19 . The compound of any one of  claims 1 - 14 , wherein Y 2  is C 1-6  haloalkyl-substituted C 3-6  cycloalkyl. 
     
     
         20 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is a 2-8 membered heteroalkyl optionally substituted by a 6-10 membered aryl or a 3-10 membered heterocyclyl. 
     
     
         21 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is —O—(C 1-7  alkyl). 
     
     
         22 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is —O-butyl, —O-pentyl, or —O-hexyl. 
     
     
         23 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is —(C 1-3  alkylene)-O-(5-6 membered heteroaryl). 
     
     
         24 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is a 3-10 membered heterocyclyl, 6-10 membered aryl, C 3-7  cycloalkyl, —O-(3-6 membered heterocyclyl), —O-(6-membered aryl), or —O—(C 2-6  alkynyl). 
     
     
         25 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is a 3-10 membered heterocyclyl selected from the group consisting of a 5-6 membered heteroaryl and a 5-6 membered heterocycloalkyl. 
     
     
         26 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is 5-membered heteroaryl. 
     
     
         27 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is furanyl, pyrrolyl, thiophenyl, imidazolyl, pyrazolyl, oxazolyl, or thiazolyl. 
     
     
         28 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is C 2-6  alkynyl, —C≡C—(C 1-6  alkylene)-OR 4 , —C≡C—(C 1-6  alkylene)-N(R 3 ) 2 , —(C 2-4  alkynylene)-(5-6 membered heteroaryl), or C 2-6  alkenyl. 
     
     
         29 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is C 2-6  alkynyl. 
     
     
         30 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is —C≡CH. 
     
     
         31 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is —C≡C—(C 1-6  alkylene)-OR 4 . 
     
     
         32 . The compound of any one of  claims 1 - 14  or  17 - 19 , wherein Y 1  is —C≡C—CH 2 —O—CH 3 . 
     
     
         33 . The compound of  claim 1 , wherein the compound is represented by Formula I-A: 
       
         
           
           
               
               
           
         
         or a pharmaceutically acceptable salt thereof, wherein: 
         R 1  is independently methyl, isopropyl, cyclopropyl, C 1-2  haloalkyl, —(CH 2 ) 1-2 —O—(C 1-3  alkyl), chloro, fluoro, or —N(R 4 ) 2 ; 
         R 2  is hydrogen; 
         R 3  and R 4  each represent independently for each occurrence hydrogen or C 1-4  alkyl; 
         A 1  is a cyclic group selected from:
 C 3-10  cycloalkyl, phenyl, or 5-6 membered heteroaryl, each of which is substituted by 1 occurrence of Y 1  and 0, 1, or 2 occurrences of Y 2 ; and 
 a bicyclic carbocyclyl that is partially unsaturated or a bicyclic heterocyclyl, each of which is substituted by 0 or 1 occurrence of Y 1  and 0, 1, or 2 occurrences of Y 2 ; 
 
         Y 1  represents, independently for each occurrence, one of the following:
 2-8 membered heteroalkyl optionally substituted by a 6-10 membered aryl or a 3-10 membered heterocyclyl; 
 3-10 membered heterocyclyl, 6-10 membered aryl, —O-(3-6 membered heterocyclyl), —O-(6-10 membered aryl), or —O—(C 2-6  alkynyl); or 
 C 2-6  alkynyl, —C≡C—(C 1-6  alkylene)-OR 4 , —C≡C—(C 1-6  alkylene)-N(R 3 ) 2 , —(C 2-4  alkynylene)-(5-6 membered heteroaryl), or C 2-6  alkenyl; 
 
         Y 2  represents, independently for each occurrence, C 1-6  alkyl, C 3-6  cycloalkyl, halogen, C 1-6  haloalkyl, C 1-6  hydroxyalkyl, hydroxyl, C 1-6  alkoxyl, cyano, azido, —N(R 3 ) 2 , —(C 1-6  alkylene)-(5-6 membered heterocyclyl), —(C 1-6  alkylene)-CO 2 R 3 , or C 1-6  haloalkyl-substituted C 3-6  cycloalkyl. 
       
     
     
         34 . The compound of  claim 33 , wherein A 1  is C 3-7  cycloalkyl substituted once by Y 1  and 0-1 occurrences of Y 2 . 
     
     
         35 . The compound of  claim 33 , wherein A 1  is phenyl substituted once by Y 1  and 0-1 occurrences of Y 2 . 
     
     
         36 . The compound of any one of  claims 33 - 35 , wherein any occurrence of Y 2  is independently C 1-3  alkyl, halogen, or C 1-3  haloalkyl. 
     
     
         37 . The compound of any one of  claims 33 - 36 , wherein Y 1  is a 2-8 membered heteroalkyl optionally substituted by a 6-10 membered aryl or a 3-10 membered heterocyclyl. 
     
     
         38 . The compound of any one of  claims 33 - 36 , wherein Y 1  is —O—(C 1-7  alkyl). 
     
     
         39 . The compound of any one of  claims 33 - 36 , wherein Y 1  is —O-butyl, —O-pentyl, or —O-hexyl. 
     
     
         40 . The compound of any one of  claims 33 - 36 , wherein Y 1  is —(C 1-3  alkylene)-O-(5-6 membered heteroaryl). 
     
     
         41 . The compound of any one of  claims 33 - 36 , wherein Y 1  is a 5-membered heteroaryl. 
     
     
         42 . The compound of any one of  claims 33 - 36 , wherein Y 1  is furanyl, pyrrolyl, thiophenyl, imidazolyl, pyrazolyl, oxazolyl, or thiazolyl. 
     
     
         43 . The compound of any one of  claims 33 - 36 , wherein Y 1  is pyridinyl, pyrimidinyl, pyrazinyl, isooxazolyl, isothiazolyl, imidazolyl, oxadiazolyl, thiadiazolyl, imidazolinyl, oxazolinyl, pyrazolinyl, or thiazolinyl. 
     
     
         44 . The compound of any one of  claims 33 - 36 , wherein Y 1  is C 2-6  alkynyl, —C≡C—(C 1-6  alkylene)-OR 4 , —C≡C—(C 1-6  alkylene)-N(R 3 ) 2 , —(C 2-4  alkynylene)-(5-6 membered heteroaryl), or C 2-6  alkenyl. 
     
     
         45 . The compound of any one of  claims 33 - 36 , wherein Y 1  is C 2-6  alkynyl. 
     
     
         46 . The compound of any one of  claims 33 - 36 , wherein Y 1  is —C≡C—(C 1-6  alkylene)-O—(C 1-2  alkyl). 
     
     
         47 . The compound of any one of  claims 33 - 36 , wherein Y 1  is —C≡C—CH 2 —O—CH 3 . 
     
     
         48 . The compound of  claim 33 , wherein A 1  is an 8-12 membered bicyclic carbocyclyl that is partially unsaturated or an 8-12 membered bicyclic heterocyclyl, each of which is substituted by 0 or 1 occurrence of Y 1  and 0, 1, or 2 occurrences of Y 2 . 
     
     
         49 . The compound of  claim 33 , wherein A 1  is 
       
         
           
           
               
               
           
         
       
       wherein m is 0, 1, or 2; and Y 2  represents independently for each occurrence C 1-6  alkyl, C 3-6  cycloalkyl, halogen, C 1-6  haloalkyl, hydroxyl, or C 1-6  alkoxyl. 
     
     
         50 . The compound of  claim 1 , wherein the compound is any one of the compounds in Tables 1, 2, or 3 herein, or a pharmaceutically acceptable salt thereof. 
     
     
         51 . A pharmaceutical composition, comprising a compound of any one of  claims 1 - 50  and a pharmaceutically acceptable carrier. 
     
     
         52 . A method of treating a disorder selected from the group consisting of Gaucher disease, Parkinson's disease, Lewy body disease, dementia, multiple system atrophy, epilepsy, bipolar disorder, schizophrenia, an anxiety disorder, major depression, polycystic kidney disease, type 2 diabetes, open angle glaucoma, multiple sclerosis, and multiple myeloma, comprising administering to a patient in need thereof a therapeutically effective amount of a compound of any one of  claims 1 - 50  to treat the disorder. 
     
     
         53 . The method of  claim 52 , wherein the disorder is Gaucher disease. 
     
     
         54 . The method of  claim 52 , wherein the disorder is Parkinson's disease. 
     
     
         55 . The method of  claim 52 , wherein the disorder is Lewy body disease. 
     
     
         56 . The method of  claim 52 , wherein the disorder is dementia. 
     
     
         57 . The method of  claim 52 , wherein the disorder is multiple system atrophy. 
     
     
         58 . The method of any one of  claims 52 - 57 , wherein the patient is a human.

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