US2017333435A1PendingUtilityA1

Substituted imidazo[1,5-a]pyrimidines and their use in the treatment of medical disorders

49
Assignee: LYSOSOMAL THERAPEUTICS INCPriority: Nov 6, 2014Filed: Nov 6, 2015Published: Nov 23, 2017
Est. expiryNov 6, 2034(~8.3 yrs left)· nominal 20-yr term from priority
A61P 43/00A61K 31/16A61P 25/18A61P 25/24A61K 31/519C07D 487/04A61P 25/08A61P 25/28A61P 25/16A61K 31/13C07D 519/00A61K 2300/00
49
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Claims

Abstract

The invention provides substituted imidazo┌1,5-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 imidazo[1,5-a]pyrimidine compounds described herein include substituted 2,4-dimethyl-N-phenylimidazo[1,5-a]pyrimidine-8-carbox-amide 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  represents independently for each occurrence hydrogen, C 1-4 alkyl, C 1-4 haloalkyl, C 1-4 alkoxyl, —(C 1-4 alkylene)-(C 1-4 alkoxyl), cyclopropyl, cyano, chloro, or fluoro; 
         R 2  is hydrogen, C 1-4 alkyl, C 1-4 haloalkyl, C 1-4 alkoxyl, cyclopropyl, cyano, chloro, or fluoro; 
         R 3  represents independently for each occurrence hydrogen or C 1-4 alkyl; 
         R 4  represents independently for each occurrence hydrogen, C 1-4 alkyl, or —C(O)R 3 ; 
         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, 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 ; and 
 a bicyclic carbocyclyl that is partially unsaturated or a mono-cyclic or bicyclic 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 ; 
 
         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, 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 
 halogen or cyano; 
 
         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; and 
         n is 1, 2, or 3. 
       
     
     
         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 alkoxyl, cyclopropyl, cyano, chloro, or fluoro. 
     
     
         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 imidazo[1,5-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  claim 1 - 14 ,  17 , or  18 , wherein Y 1  is a 2-8 membered heteroalkyl optionally substituted by a 6-10 membered aryl or a 3-10 membered heterocyclyl. 
     
     
         20 . The compound of any one of  claim 1 - 14 ,  17 , or  18 , wherein Y 1  is —O—(C 1-7  alkyl). 
     
     
         21 . The compound of any one of  claim 1 - 14 ,  17 , or  18 , wherein Y 1  is —O-butyl, —O-pentyl, or —O-hexyl. 
     
     
         22 . The compound of any one of  claim 1 - 14 ,  17 , or  18 , wherein Y 1  is —(C 1-3 alkylene)-O-(5-6 membered heteroaryl). 
     
     
         23 . The compound of any one of  claim 1 - 14 ,  17 , or  18 , wherein Y 1  is a 3-10 membered heterocyclyl, 6-10 membered aryl, C 3-7 -cycloalkyl, —O-(3-6 membered heterocyclyl), —O-(6-10 membered aryl), or —O—(C 2-6 alkenyl). 
     
     
         24 . The compound of any one of  claim 1 - 14 ,  17 , or  18 , 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. 
     
     
         25 . The compound of any one of  claim 1 - 14 ,  17 , or  18 , wherein Y 1  is 5-membered heteroaryl. 
     
     
         26 . The compound of any one of  claim 1 - 14 ,  17 , or  18 , wherein Y 1  is furanyl, pyrrolyl, thiophenyl, imidazolyl, pyrazolyl, oxazolyl, or thiazolyl. 
     
     
         27 . The compound of any one of  claim 1 - 14 ,  17 , or  18 , 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 24 alkynylene)-(5-6 membered heteroaryl), or C 2-6 alkenyl. 
     
     
         28 . The compound of any one of  claim 1 - 14 ,  17 , or  18 , wherein Y 1  is C 2-6 alkynyl. 
     
     
         29 . The compound of any one of  claim 1 - 14 ,  17 , or  18 , wherein Y 1  is —C≡CH. 
     
     
         30 . The compound of any one of  claim 1 - 14 ,  17 , or  18 , wherein Y 1  is —C≡C—(C 1-6 alkylene)-OR 4 . 
     
     
         31 . The compound of any one of  claim 1 - 14 ,  17 , or  18 , wherein Y 1  is —C≡C—CH 2 —O—CH 3 . 
     
     
         32 . 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, cyclopropyl, isopropyl, or —(C 1-4 alkylene)-(C 1-4 alkoxyl); 
         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. 
       
     
     
         33 . The compound of  claim 32 , wherein A 1  is C 5-10  cycloalkyl substituted once by Y 1  and 0-1 occurrences of Y 2 . 
     
     
         34 . The compound of  claim 32 , wherein A 1  is phenyl substituted once by Y 1  and 0-1 occurrences of Y 2 . 
     
     
         35 . The compound of any one of  claims 32 - 34 , wherein any occurrence of Y 2  is independently C 1-3 alkyl, halogen, or C 1-3 haloalkyl. 
     
     
         36 . The compound of any one of  claims 32 - 35 , wherein Y 1  is a 2-8 membered heteroalkyl optionally substituted by a 6-10 membered aryl or a 3-10 membered heterocyclyl. 
     
     
         37 . The compound of any one of  claims 32 - 35 , wherein Y 1  is —O—(C 1-7 alkyl). 
     
     
         38 . The compound of any one of  claims 32 - 35 , wherein Y 1  is —O-butyl, —O-pentyl, or —O-hexyl. 
     
     
         39 . The compound of any one of  claims 32 - 35 , wherein Y 1  is —(C 1-3 alkylene)-O-(5-6 membered heteroaryl). 
     
     
         40 . The compound of any one of  claims 32 - 35 , wherein Y 1  is a 5-membered heteroaryl. 
     
     
         41 . The compound of any one of  claims 32 - 35 , wherein Y 1  is furanyl, pyrrolyl, thiophenyl, imidazolyl, pyrazolyl, oxazolyl, or thiazolyl. 
     
     
         42 . The compound of any one of  claims 32 - 35 , wherein Y 1  is pyridinyl, pyrimidinyl, pyrazinyl, isooxazolyl, isothiazolyl, imidazolyl, oxadiazolyl, thiadiazolyl, imidazolinyl, oxazolinyl, pyrazolinyl, or thiazolinyl. 
     
     
         43 . The compound of any one of  claims 32 - 35 , 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. 
     
     
         44 . The compound of any one of  claims 32 - 35 , wherein Y 1  is C 2-6 alkynyl. 
     
     
         45 . The compound of any one of  claims 32 - 35 , wherein Y 1  is —C≡C—(C 1-6 alkylene)-O—(C 1-2 alkyl). 
     
     
         46 . The compound of any one of  claims 32 - 35 , wherein Y 1  is —C≡C—CH 2 —O—CH 3 . 
     
     
         47 . The compound of  claim 32 , 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 . 
     
     
         48 . The compound of  claim 32 , 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. 
     
     
         49 . 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. 
     
     
         50 . A pharmaceutical composition, comprising a compound of any one of  claims 1 - 49  and a pharmaceutically acceptable carrier. 
     
     
         51 . 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 - 49  to treat the disorder. 
     
     
         52 . The method of  claim 51 , wherein the disorder is Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy. 
     
     
         53 . The method of  claim 51 , wherein the disorder is Gaucher disease. 
     
     
         54 . The method of  claim 51 , wherein the disorder is Parkinson's disease. 
     
     
         55 . The method of  claim 51 , wherein the disorder is Lewy body disease. 
     
     
         56 . The method of  claim 51 , wherein the disorder is dementia. 
     
     
         57 . The method of  claim 51 , wherein the disorder is multiple system atrophy. 
     
     
         58 . The method of any one of  claims 51 - 57 , wherein the patient is a human.

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