US2009325903A1PendingUtilityA1

Boronate ester compounds and pharmaceutical compositions thereof

70
Assignee: MILLENNIUM PHARM INCPriority: Jun 17, 2008Filed: Jun 16, 2009Published: Dec 31, 2009
Est. expiryJun 17, 2028(~1.9 yrs left)· nominal 20-yr term from priority
A61P 37/06A61P 43/00A61P 9/00A61P 7/06A61P 9/10A61P 37/02A61P 35/04A61P 25/14A61P 25/28A61P 25/00A61P 31/00A61P 27/02A61P 25/04A61P 25/16A61P 29/00A61P 31/18A61P 35/00A61P 35/02A61P 1/16A61P 19/02A61P 1/04A61P 11/00A61P 1/00A61P 17/00A61P 11/06A61P 17/02A61P 17/06C07F 5/025A61K 31/454A61K 31/69C07K 5/06139C07F 5/04A61K 31/198A61K 47/183A61K 9/19A61K 9/0019C07C 233/83C07F 5/05A61K 38/00C07K 5/06191C07F 5/02
70
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Claims

Abstract

The present invention provides novel compounds useful as proteasome inhibitors. The invention also provides pharmaceutical compositions comprising the compounds of the invention and methods of using the compositions in the treatment of various diseases.

Claims

exact text as granted — not AI-modified
1 . A compound of formula (I): 
     
       
         
         
             
             
         
       
     
     or a pharmaceutically acceptable salt thereof, wherein:
 A is 0, 1, or 2; 
 P is hydrogen or an amino-group-blocking moiety; 
 R a  is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , —(CH 2 ) m —CH(R 4 )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
 R a1  is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , —(CH 2 ) m —CH(R 5a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
 each R a2  independently is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R 3 , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , —(CH 2 ) m —CH(R 5a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
 each R B  independently is a substituted or unsubstituted mono- or bicyclic ring system; 
 each R 4  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; or two R 4  on the same nitrogen atom, taken together with the nitrogen atom, form a substituted or unsubstituted 4- to 8-membered heterocyclyl ring having, in addition to the nitrogen atom, 0-2 ring heteroatoms independently selected from N, O, and S; 
 each R 5  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
 each R 5a  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
 each R 5b  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
 each R 6  independently is a substituted or unsubstituted aliphatic, aryl, or heteroaryl group; 
 Y is hydrogen, —CN, or —NO 2 ; 
 m is 0, 1, or 2; and 
 Z 1  and Z 2  together form a moiety derived from an alpha-hydroxy carboxylic acid, wherein the atom attached to boron in each case is an oxygen atom; or Z 1  and Z 2  together form a moiety derived from a beta-hydroxy carboxylic acid, wherein the atom attached to boron in each case is an oxygen atom. 
 
   
   
       2 . The compound of  claim 1 , characterized by formula (II): 
     
       
         
         
             
             
         
       
       or a pharmaceutically acceptable salt thereof, wherein: 
       each of R b1  and R b2  independently is hydrogen, —CO 2 H, —OH or a substituted or unsubstituted aliphatic, aryl, heteroaryl or heterocyclyl group; 
       each of R b3  and R b4  independently is hydrogen, —CO 2 H, or a substituted or unsubstituted aliphatic, aryl, heteroaryl or heterocyclyl group; 
       or R b2  and R b4  are each independently hydrogen, and R b1  and R b3 , taken together with the carbon atoms to which they are attached, form an unsubstituted or substituted fused 4- to 8-membered non-aromatic ring having 0-3 ring heteroatoms selected from the group consisting of O, N, and S, wherein said ring may be optionally fused to an unsubstituted or substituted 4- to 8-membered non-aromatic ring, or 5- to 6-membered aromatic ring having 0-3 ring heteroatoms selected from the group consisting of O, N, and S; 
       or R b2  and R b4  are absent, and R b1  and R b3 , taken together with the carbon atoms to which they are attached, form an unsubstituted or substituted fused 5- to 6-membered aromatic ring having 0-3 ring heteroatoms selected from the group consisting of O, N, and S, wherein said ring may be optionally fused to an unsubstituted or substituted 4- to 8-membered non-aromatic ring, or 5- to 6-membered aromatic ring having 0-3 ring heteroatoms selected from the group consisting of O, N, and S; and 
       n is 0 or 1. 
     
   
   
       3 . The compound of  claim 2 , wherein each of R b3  and R b4  independently is hydrogen, C 1-6  aliphatic, or —(CH 2 ) p —CO 2 H;
 and p is 0, 1, or 2.   
   
   
       4 . The compound of  claim 3  wherein P is R c —C(O)—, R c —O—C(O)—, R c —N(R 4c )—C(O)—, R c —S(O) 2 —, or R c —N(R 4c )—S(O) 2 —;
 R c  is selected from the group consisting of C 1-6  aliphatic, C 1-6  fluoroaliphatic, R D , T 1 -R D , and -T 1 —R 2c ;   T 1  is a C 1-6  alkylene chain substituted with 0-2 independently selected R 3a  or R 3b , wherein the alkylene chain optionally is interrupted by C(R 5 )—C(R 5 )—, —C≡C—, or —O—;   R D  is a substituted or unsubstituted mono- or bicyclic ring system;   R 2c  is halo, —OR 5 , —SR 6 , —S(O)R 6 , —SO 2 R 6 , —SO 2 N(R 4 ) 2 , —N(R 4 ) 2 , —NR 4 C(O)R 5 , —NR 4 C(O)—N(R 4 ) 2 , —NR 4 CO 2 R 6 , —N(R 4 )SO 2 R 6 , —N(R 4 )SO 2 N(R 4 ) 2 , —O—C(O)R 5 , —OC(O)N(R 4 ) 2 , —C(O)—R 5 , —CO 2 R 5 , or —C(O)N(R 4 ) 2 ;   each R 3a  independently is selected from the group consisting of —F, —OH, —O(C 1-4  alkyl), —CN, —N(R 4 ) 2 , —C(O)(C 1-4  alkyl), —CO 2 H, —CO 2 (C 1-4  alkyl), —C(O)NH 2 , and —C(O)—NH(C 1-4  alkyl);   each R 3b  independently is a C 1-3  aliphatic substituted or unsubstituted with R 3a  or R 7 ; or two substituents R 3b  on the same carbon atom, taken together with the carbon atom to which they are attached, form a 3- to 6-membered cycloaliphatic ring;   each R 7  is a substituted or unsubstituted aromatic group; and   R 4c  is hydrogen, C 1-4  alkyl, C 1-4  fluoroalkyl, or C 6-10  ar(C 1-4 )alkyl, the aryl portion of which is substituted or unsubstituted.   
   
   
       5 . The compound of  claim 3 , wherein A is 0;
 R a1  is hydrogen, —(CH 2 ) m —CH 2 —R B , or —(CH 2 ) m —CH(R 5a )—OR 5b ; and   m is 0.   
   
   
       6 . The compound of  claim 3 , wherein P is R c —C(O)— or R c —S(O) 2 — and R c  is —R D . 
   
   
       7 . The compound of  claim 6 , wherein R D  is a substituted or unsubstituted mono- or bicyclic ring system selected from the group consisting of phenyl, pyridinyl, pyrimidinyl, pyrazinyl, naphthyl, benzimidazolyl, quinolinyl, isoquinolinyl, quinoxalinyl, tetrahydroquinolinyl, tetrahydroisoquinolinyl, tetrahydroquinoxalinyl, and dihydrodrobenzoxazinyl. 
   
   
       8 . The compound of  claim 7 , wherein R D  is substituted on substitutable carbon atoms with 0-1 R d  and 0-2 R 8d ;
 each R d  independently is C 1-6  aliphatic, C 1-6  fluoroaliphatic or halo; and   each R 8d  independently is C 1-4  aliphatic, C 1-4  fluoroaliphatic or halo.   
   
   
       9 . The compound of  claim 4 , wherein A is 0;
 R a  is C 1-6  aliphatic, or —(CH 2 ) m —C 1-2 —R B ;   R a1  is hydrogen, —(CH 2 ) m —CH 2 —R B , or —(CH 2 ) m —CH(R 5a )—OR 5b ;   P is R c —C(O)— or R c —S(O) 2 —;   R c  is R D ; and   m is 0 or 1.   
   
   
       10 . The compound of  claim 9 , wherein R a  is C 1-6  aliphatic. 
   
   
       11 . The compound of  claim 10 , wherein R 8  is CH 2 —R B ;
 R B  is phenyl; and   R D  is 2-pyrazinyl.   
   
   
       12 . The compound of  claim 10 , wherein R a1  is —CH(R 5a )—OR 5b ;
 R 5a  is C 1-6  aliphatic;   R 5b  is hydrogen; and   R D  is 6-phenyl-2-pyridinyl-.   
   
   
       13 . The compound of  claim 10 , wherein R a1  is hydrogen, and R D  is 2,5-dichlorophenyl. 
   
   
       14 . The compound of  claim 3 , wherein Z 1  and Z 2  together form a moiety derived from citric acid. 
   
   
       15 . The compound of  claim 14 , in a substantially crystalline form. 
   
   
       16 . The compound of  claim 14 , characterized by formula (III), (IIIa), (IV), (IVa): 
     
       
         
         
             
             
         
       
       or a mixture thereof. 
     
   
   
       17 . The compound of  claim 16 , wherein R a  is C 1-6  aliphatic. 
   
   
       18 . The compound of  claim 16 , wherein A is 0; R a1  is hydrogen, —(CH 2 ) m —CH 2 —R B , or —(CH 2 ) m —CH(R 5 )—OR 5b ; P is R c —C(O)—; and R c  is —R D . 
   
   
       19 . The compound of  claim 18 , wherein R D  is a substituted or unsubstituted mono- or bicyclic ring system selected from the group consisting of phenyl, pyridinyl, pyrimidinyl, pyrazinyl, naphthyl, benzimidazolyl, quinolinyl, isoquinolinyl, quinoxalinyl, tetrahydroquinolinyl, tetrahydroisoquinolinyl, tetrahydroquinoxalinyl, and dihydrobenzoxazinyl. 
   
   
       20 . The compound of  claim 18 , wherein R a  is isobutyl. 
   
   
       21 . The compound of  claim 20 , wherein R a1  is —CH 2 —R B ;
 R B  is phenyl; and   R D  is 2-pyrazinyl.   
   
   
       22 . The compound of  claim 20 , wherein R a1  is hydrogen, and
 R D  is 2,5-dichlorophenyl.   
   
   
       23 . The compound of  claim 20 , wherein R a1  is —CH(R 5a )—OR 5b ;
 R 5a  is C 1-6  aliphatic;   R 5b  is hydrogen; and   R D  is 6-phenyl-2-pyridinyl-.   
   
   
       24 . A pharmaceutical composition comprising the compound of formula (I): 
     
       
         
         
             
             
         
       
       or a crystalline form thereof, a filler, and optionally a lubricant; 
       wherein: 
       A is 0, 1, or 2; 
       P is hydrogen or an amino-group-blocking moiety; 
       R a  is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , —(CH 2 ) m —CH(R 5a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
       R a1  is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , —(CH 2 ) m —CH(R 5a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
       each R a2  independently is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , —(CH 2 ) m —CH(R 5a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
       each R B  independently is a substituted or unsubstituted mono- or bicyclic ring system; 
       each R 4  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; or two R 4  on the same nitrogen atom, taken together with the nitrogen atom, form a substituted or unsubstituted 4- to 8-membered heterocyclyl ring having, in addition to the nitrogen atom, 0-2 ring heteroatoms independently selected from N, O, and S; 
       each R 5  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
       each R 5a  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
       each R 5b  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
       each R 6  independently is a substituted or unsubstituted aliphatic, aryl, or heteroaryl group; 
       Y is hydrogen, —CN, or —NO 2 ; 
       m is 0, 1, or 2; and 
       Z 1  and Z 2  together form a moiety derived from an alpha-hydroxy carboxylic acid, wherein the atom attached to boron in each case is an oxygen atom; or Z 1  and Z 2  together form a moiety derived from a beta-hydroxy carboxylic acid, wherein the atom attached to boron in each case is an oxygen atom. 
     
   
   
       25 . The pharmaceutical composition of  claim 24 , wherein the pharmaceutical composition optionally further comprises a flow-aid, and optionally further comprises a buffer. 
   
   
       26 . The pharmaceutical composition of  claim 25 , wherein the pharmaceutical composition comprises about 0.2% to about 3% of the compound of formula (I), or a crystalline form thereof, about 86.5% to about 99.8% of a filler, optionally up to about 1.5% of a lubricant, optionally up to about 5% of a flow-aid, and optionally up to about 5% of a buffer, by weight as a percentage of total weight. 
   
   
       27 . The pharmaceutical composition of  claim 25 , wherein the pharmaceutical composition comprises about 0.2% to about 3% of the compound of formula (I), or a crystalline form thereof; about 97% to about 99.8% of a filler; and optionally up to about 1.5% of a lubricant, by weight as a percentage of total weight. 
   
   
       28 . The pharmaceutical composition of  claim 25 , wherein the pharmaceutical composition comprises about 0.25% to about 2% of the compound of formula (I), or a crystalline form thereof; and about 98% to about 99.75% of a filler, by weight as a percentage of total weight. 
   
   
       29 . The pharmaceutical composition of  claim 25 , wherein the pharmaceutical composition is an oral pharmaceutical dosage form. 
   
   
       30 . The pharmaceutical composition of  claim 29 , wherein the oral pharmaceutical dosage form is a capsule. 
   
   
       31 . The pharmaceutical composition of  claim 25 , wherein the compound of formula (I), or a crystalline form thereof, is present in an amount of about 0.2% to about 3%, by weight as a percentage of total weight. 
   
   
       32 . The pharmaceutical composition of  claim 25 , wherein the compound of formula (I), or a crystalline form thereof, is present in an amount of about 0.25% to about 2%, by weight as a percentage of total weight. 
   
   
       33 . The pharmaceutical composition of  claim 25 , wherein the filler is present in an amount of about 97% to about 99.8%, by weight as a percentage of total weight. 
   
   
       34 . The pharmaceutical composition of  claim 25 , wherein the filler is present in an amount of about 98% to about 99.75%, by weight as a percentage of total weight. 
   
   
       35 . The pharmaceutical composition of  claim 25 , wherein the filler is present in an amount of about 86.5% to about 99.8%, by weight as a percentage of total weight. 
   
   
       36 . The pharmaceutical composition of  claim 25 , wherein the filler is selected from the group consisting of powdered cellulose, microcrystalline cellulose, silicified microcrystalline cellulose, low-moisture microcrystalline cellulose, sodium starch glycolate, pregelatinized starch, and mixtures thereof. 
   
   
       37 . The pharmaceutical composition of  claim 25 , wherein the filler is selected from the group consisting of low-moisture microcrystalline cellulose, sodium starch glycolate, pregelatinized starch, and mixtures thereof. 
   
   
       38 . The pharmaceutical composition of  claim 25 , wherein the lubricant, when present, is present in an amount of up to about 1.5%, by weight as a percentage of total weight. 
   
   
       39 . The pharmaceutical composition of  claim 25 , wherein the lubricant, when present, is present in an amount of about 1%, by weight as a percentage of total weight. 
   
   
       40 . The pharmaceutical composition of  claim 25 , wherein the lubricant, when present, is selected from the group consisting of magnesium stearate, glyceryl bebenate, hydrogenated vegetable oil, talc, zinc stearate, calcium stearate, sucrose stearate, sodium stearyl fumarate, and mixtures thereof. 
   
   
       41 . The pharmaceutical composition of  claim 25 , wherein the lubricant is magnesium stearate. 
   
   
       42 . The pharmaceutical composition of  claim 25 , wherein the flow-aid, when present, is present in an amount of up to about 5%, by weight as a percentage of total weight. 
   
   
       43 . The pharmaceutical composition of  claim 25 , wherein the flow-aid, when present is present in an amount of about 1%, by weight as a percentage of total weight. 
   
   
       44 . The pharmaceutical composition of  claim 25 , wherein the flow-aid, when present, is talc. 
   
   
       45 . The pharmaceutical composition of  claim 25 , wherein the buffer, when present, is present in an amount of up to about 5%, by weight as a percentage of total weight. 
   
   
       46 . The pharmaceutical composition of  claim 25 , wherein the buffer, when present, is present in an amount of about 2%, by weight as a percentage of total weight. 
   
   
       47 . The pharmaceutical composition of  claim 25 , wherein the buffer, when present, is sodium citrate. 
   
   
       48 . The pharmaceutical composition of  claim 25 , wherein:
 the alpha-hydroxy carboxylic acid or beta-hydroxy carboxylic acid is citric acid;   A is 0;   R a  is isobutyl;   R a1  is hydrogen, C 1-6  aliphatic, —(CH 2 ) m —CH 2 —R B , or —(CH 2 ) m —CH(R 5a )—OR 5b ;   P is R c —C(O)—;   R c  is —R D ;   m is 0 or 1;   the filler is selected from the group consisting of low-moisture microcrystalline cellulose, sodium starch glycolate, pregeletanized starch, and mixtures thereof; and   the lubricant, when present, is magnesium stearate.   
   
   
       49 . The pharmaceutical composition of  claim 25 , wherein
 the compound of formula (I) is represented by compounds (I-1), (I-15), or (I-18):   
     
       
         
         
             
             
         
       
       the filler is selected from the group consisting of low-moisture microcrystalline cellulose, sodium starch glycolate, pregeletanized starch, and mixtures thereof; and 
       the lubricant, when present, is magnesium stearate. 
     
   
   
       50 . The pharmaceutical composition of  claim 25 , wherein:
 the alpha-hydroxy carboxylic acid or beta-hydroxy carboxylic acid is citric acid;   A is 0;   R a  is isobutyl;   R a1  is hydrogen, C 1-6  aliphatic, —(CH 2 ) m —CH 2 —R B , or —(CH 2 ) m —CH(R 5a )—OR 5b ;   P is R c —C(O)—;   R c  is —R D ;   m is 0 or 1;   the filler is selected from the group consisting of low-moisture microcrystalline cellulose, sodium starch glycolate, pregeletanized starch, and mixtures thereof;   the lubricant, when present, is magnesium stearate;   the flow-aid, when present, is talc; and   the buffer, when present, is sodium citrate.   
   
   
       51 . The pharmaceutical composition of  claim 25 , wherein
 the compound of formula (I) is represented by compounds (I-1), (I-15) or (I-18):   
     
       
         
         
             
             
         
       
       the filler is selected from the group consisting of low-moisture microcrystalline cellulose, sodium starch glycolate, pregeletanized starch, and mixtures thereof; 
       the lubricant, when present, is magnesium stearate; 
       the flow-aid, when present, is talc; and 
       the buffer, when present, is sodium citrate. 
     
   
   
       52 . A pharmaceutical composition comprising the compound of formula (I-1): 
     
       
         
         
             
             
         
       
     
     or a crystalline form thereof, a filler, and optionally a lubricant. 
   
   
       53 . The pharmaceutical composition of  claim 52 , wherein:
 the filler is selected from the group consisting of low-moisture microcrystalline cellulose, sodium starch glycolate, pregeletanized starch, and mixtures thereof; and   the lubricant, when present, is magnesium stearate.   
   
   
       54 . The pharmaceutical composition of  claim 52 , wherein the crystalline form is Form 2. 
   
   
       55 . The pharmaceutical composition of  claim 52 , wherein the pharmaceutical composition optionally further comprises a flow-aid, and optionally further comprises a buffer. 
   
   
       56 . The pharmaceutical composition of  claim 55 , wherein:
 the filler is selected from the group consisting of low-moisture microcrystalline cellulose, sodium starch glycolate, pregeletanized starch, and mixtures thereof;   the lubricant, when present, is magnesium stearate;   the flow-aid, when present, is talc; and   the buffer, when present, is sodium citrate.   
   
   
       57 . A unit dose pharmaceutical composition comprising the compound of formula (I-1), or a crystalline form thereof, wherein the compound of formula (I-1) is present in an amount equivalent on a molar weight basis of about 0.1 mg to about 3.0 mg of the compound of formula (VIII-1). 
   
   
       58 . A unit dose pharmaceutical composition comprising about 0.143 mg to about 4.3 mg of the compound of formula (I-1), or a crystalline form thereof, measured as about 0.1 mg to about 3.0 mg of the compound of formula (VIII-1), on a weight for weight basis. 
   
   
       59 . A pharmaceutical composition comprising the compound of formula (I): 
     
       
         
         
             
             
         
       
       or a crystalline form thereof, a bulking agent; and a buffer; 
       wherein: 
       A is 0, 1, or 2; 
       P is hydrogen or an amino-group-blocking moiety; 
       R a  is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 —(CH 2 ) m —CH(R 5a )—OR b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
       R a1  is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , —(CH 2 ) m —CH(R 5a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
       each R a2  independently is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , —(CH 2 ) m —CH(R 5a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
       each R B  independently is a substituted or unsubstituted mono- or bicyclic ring system; 
       each R 4  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; or two R 4  on the same nitrogen atom, taken together with the nitrogen atom, form a substituted or unsubstituted 4- to 8-membered heterocyclyl ring having, in addition to the nitrogen atom, 0-2 ring heteroatoms independently selected from N, O, and S; 
       each R 5  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
       each R 5a  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
       each R 5b  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
       each R 6  independently is a substituted or unsubstituted aliphatic, aryl, or heteroaryl group; 
       Y is hydrogen, —CN, or —NO 2 ; 
       m is 0, 1, or 2; and 
       Z 1  and Z 2  together form a moiety derived from an alpha-hydroxy carboxylic acid, wherein the atom attached to boron in each case is an oxygen atom; or Z 1  and Z 2  together form a moiety derived from a beta-hydroxy carboxylic acid, wherein the atom attached to boron in each case is an oxygen atom. 
     
   
   
       60 . The pharmaceutical composition of  claim 59 , wherein the bulking agent is present in an amount of about 1% w/v to about 5% w/v. 
   
   
       61 . The pharmaceutical composition of  claim 59 , wherein the bulking agent is present in an amount of about 3% w/v. 
   
   
       62 . The pharmaceutical composition of  claim 59 , wherein the bulking agent is glycine. 
   
   
       63 . The pharmaceutical composition of  claim 59 , wherein the buffer is sodium citrate, and citric acid. 
   
   
       64 . The pharmaceutical composition of  claim 59 , wherein
 the alpha-hydroxy carboxylic acid or beta-hydroxy carboxylic acid is citric acid;   A is 0;   R a  is isobutyl;   R a1  is hydrogen, C 1-6  aliphatic, —(CH 2 ) m —CH 2 —R B , or —(CH 2 ) m —CH(R 5a )—OR 5b ;   P is R c —C(O)—);   R c  is —R D ;   m is 0 or 1;   the bulking agent is glycine; and   the buffer is sodium citrate, and citric acid.   
   
   
       65 . The pharmaceutical composition of  claim 59  wherein
 the compound of formula (I) is represented by compounds (I-1), (I-15) or (I-1×):   
     
       
         
         
             
             
         
       
       the bulking agent is glycine; and 
       the buffer is sodium citrate, and citric acid. 
     
   
   
       66 . A pharmaceutical composition comprising the compound of formula (I) in a lyophilized powder: 
     
       
         
         
             
             
         
       
       wherein: 
       A is 0, 1, or 2; 
       P is hydrogen or an amino-group-blocking moiety; 
       R a  is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , —(CH 2 ) m —CH(R 5a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
       R a1  is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , —(CH 2 ) m —CH(R 5a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
       each R a2  independently is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —-CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , —(CH 2 ) m —CH(R 5a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
       each R B  independently is a substituted or unsubstituted mono- or bicyclic ring system; 
       each R 4  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; or two R 4  on the same nitrogen atom, taken together with the nitrogen atom, form a substituted or unsubstituted 4- to 8-membered heterocyclyl ring having, in addition to the nitrogen atom, 0-2 ring heteroatoms independently selected from N, O, and S; 
       each R 5  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
       each R 5a  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
       each R 5b  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
       each R 6  independently is a substituted or unsubstituted aliphatic, aryl, or heteroaryl group; 
       Y is hydrogen, —CN, or —NO 2 ; 
       m is 0, 1, or 2; and 
       Z 1  and Z 2  together form a moiety derived from an alpha-hydroxy carboxylic acid, wherein the atom attached to boron in each case is an oxygen atom; or Z 1  and Z 2  together form a moiety derived from a beta-hydroxy carboxylic acid, wherein the atom attached to boron in each case is an oxygen atom. 
     
   
   
       67 . The pharmaceutical composition of  claim 66 , further comprising a bulking agent) and a buffer. 
   
   
       68 . The pharmaceutical composition of  claim 67 , wherein the bulking agent is glycine. 
   
   
       69 . The pharmaceutical composition of  claim 67 , wherein the buffer is sodium citrate and citric acid. 
   
   
       70 . The pharmaceutical composition of  claim 67 , wherein the alpha-hydroxy carboxylic acid or beta-hydroxy carboxylic acid is citric acid;
 A is 0;   R a  is isobutyl;   R a1  is hydrogen, C 1-6  aliphatic, —(CH 2 ) m —CH 2 —R B , or —(CH 2 ) m —CH(R 5a )—OR 5b ;   P is R c —C(O)—;   R c  is —R D ;   m is 0 or 1;   the bulking agent is glycine; and   the buffer is sodium citrate, and citric acid.   
   
   
       71 . The pharmaceutical composition of  claim 67 , wherein
 the compound of formula (I) is represented by compounds (I-1), (I-15) or (I-18):   
     
       
         
         
             
             
         
       
       the bulking agent is glycine; and 
       the buffer is sodium citrate, and citric acid. 
     
   
   
       72 . A unit dose pharmaceutical composition comprising the compound of formula (I-1), in a lyophilized powder: 
     
       
         
         
             
             
         
       
       wherein: 
       the compound of formula (I-1) is present in an amount equivalent on a molar weight basis of about 1 mg to about 5 mg of the compound of formula (VIII-1). 
     
   
   
       73 . The unit dose pharmaceutical composition of  claim 72 , wherein the compound of formula (I-1) is present in an amount equivalent on a molar weight basis of about 3.5 mg of the compound of formula (VIII-1). 
   
   
       74 . The unit dose pharmaceutical composition of  claim 72 , wherein the pharmaceutical composition further comprises glycine, sodium citrate and citric acid. 
   
   
       75 . The unit dose pharmaceutical composition of  claim 74 , wherein the amount of glycine present is about 0.01 g to about 0.50 g. 
   
   
       76 . The unit dose pharmaceutical composition of  claim 74 , wherein the sodium citrate and citric acid is present in an amount equivalent to about 0.005 g to about 0.250 g of citrate ion. 
   
   
       77 . A unit dose pharmaceutical composition comprising the compound of formula (I-15), in a lyophilized powder: 
     
       
         
         
             
             
         
       
       wherein: 
       the compound of formula (I-15) is present in an amount equivalent on a molar weight basis of about 1 mg to about 5 mg of the compound of formula (VIII-15). 
     
   
   
       78 . The unit dose pharmaceutical composition of  claim 77 , wherein the compound of formula (I-15) is present in an amount equivalent on a molar weight basis of about 3.5 mg of the compound of formula (VIII-15). 
   
   
       79 . The unit dose pharmaceutical composition of  claim 78 , wherein the pharmaceutical composition further comprises glycine, sodium citrate and citric acid. 
   
   
       80 . The unit dose pharmaceutical composition of  claim 78 , wherein the amount of glycine present is about 0.01 g to about 0.50 g. 
   
   
       81 . The unit dose pharmaceutical composition of  claim 78 , wherein the sodium citrate and citric acid is present in an amount equivalent to about 0.005 g to about 0.250 g of citrate ion. 
   
   
       82 . A method for preparation of a pharmaceutical composition of the compound (I-1) as a lyophilized powder, the method comprising the steps:
 (f-1) combining:
 i. water; 
 ii. the compound (I-1); 
 iii. glycine; 
 iv. sodium citrate; and 
 v. citric acid; to form a mixture; and 
   (f-2) lyophilizing the mixture.   
   
   
       83 . A method for the preparation of a pharmaceutical composition of the compound (I-15) as a lyophilized powder, the method comprising the steps
 (g-1) combining:
 i. an aqueous solvent mixture comprising water and tert-butyl alcohol; 
 ii. the compound (VIII-15); 
 iii. glycine; 
 iv. sodium citrate; and 
 v. citric acid; to form a mixture; and 
   (g-2) lyophilizing the mixture.   
   
   
       84 . The method of  claim 83 , wherein the tert-butyl alcohol in the aqueous solvent mixture is present in an amount of about 3% v/v to about 6% v/v tert-butyl alcohol. 
   
   
       85 . A method for the preparation of a pharmaceutical composition of the compound (I-1) as a liquid pharmaceutical dosage form, said process comprising the step of reconstituting a lyophilized powder of the compound (I-1) according to  claim 72  with water for injection. 
   
   
       86 . A method for the preparation of s pharmaceutical composition of the compound (I-15) as a liquid pharmaceutical dosage form, said method comprising the step of reconstituting a lyophilized powder of the compound (I-15) according to  claim 77  with water for injection. 
   
   
       87 . A liquid pharmaceutical composition comprising the compound of formula (I): 
     
       
         
         
             
             
         
       
       a buffer, and optionally a tonicity modifier; 
       wherein: 
       A is 0, 1, or 2; 
       P is hydrogen or an amino-group-blocking moiety; 
       R a  is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R 8 , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 —(CH 2 ) m —CH(R a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
       R a1  is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CONR 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , —(CH 2 ) m —CH(R 5a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
       each R a2  independently is hydrogen, C 1-6  aliphatic, C 1-6  fluoroaliphatic, —(CH 2 ) m —CH 2 —R B , —(CH 2 ) m —CH 2 —NHC(═NR 4 )NH—Y, —(CH 2 ) m —CH 2 —CON(R 4 ) 2 , —(CH 2 ) m —CH 2 —N(R 4 )CON(R 4 ) 2 , —(CH 2 ) m —CH(R 6 )N(R 4 ) 2 , (CH 2 ) m —CH(R 5a )—OR 5b , or —(CH 2 ) m —CH(R 5 )—SR 5 ; 
       each R B  independently is a substituted or unsubstituted mono- or bicyclic ring system; 
       each R 4  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; or two R 4  on the same nitrogen atom, taken together with the nitrogen atom, form a substituted or unsubstituted 4- to 8-membered heterocyclyl ring having, in addition to the nitrogen atom, 0-2 ring heteroatoms independently selected from N, O, and S; 
       each R 5  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
       each R 5a  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
       each R 5b  independently is hydrogen or a substituted or unsubstituted aliphatic, aryl, heteroaryl, or heterocyclyl group; 
       each R 6  independently is a substituted or unsubstituted aliphatic, aryl, or heteroaryl group; 
       Y is hydrogen, —CN, or —NO 2 ; 
       m is 0, 1, or 2; and 
       Z 1  and Z 2  together form a moiety derived from an alpha-hydroxy carboxylic acid, wherein the atom attached to boron in each case is an oxygen atom; or Z 1  and Z 2  together form a moiety derived from a beta-hydroxy carboxylic acid, wherein the atom attached to boron in each case is an oxygen atom. 
     
   
   
       88 . The liquid pharmaceutical composition of  claim 87 , wherein the tonicity modifier, when present, is sodium chloride. 
   
   
       89 . The liquid pharmaceutical composition of  claim 87 , wherein the buffer is sodium citrate, and citric acid. 
   
   
       90 . The liquid pharmaceutical composition of  claim 87  wherein:
 the alpha-hydroxy carboxylic acid or beta-hydroxy carboxylic acid is citric acid;   A is 0;   R a  is isobutyl;   R a1  is hydrogen, C 1-6  aliphatic, —(CH 2 ) m —CH 2 —R B , or —(CH 2 ) m —CH(R 5a )—OR 5b ;   P is R c —C(O)—;   R c  is —R;   m is 0 or 1;   the buffer is sodium citrate and citric acid; and   the tonicity modifier, when present, is sodium chloride.   
   
   
       91 . A unit dose liquid pharmaceutical composition comprising the compound of formula (I-1): 
     
       
         
         
             
             
         
       
       wherein: 
       the compound of formula (I-1) is present at a concentration of about 0.5 mg/ml to about 3 mg/ml of the compound of formula (VIII-1). 
     
   
   
       92 . The unit dose liquid pharmaceutical composition of  claim 91 , further comprising sodium citrate, citric acid and sodium chloride. 
   
   
       93 . A method for treating cancer, comprising the administration of a therapeutically effective amount of the pharmaceutical composition of any one of  claims 24 ,  52 ,  57 ,  58 ,  59 ,  72 ,  77 , and  87 .

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