IP Library Granted Patent US 10,787,484
Granted Patent B2
US 10,787,484 · App. 16/229,226 · Granted Sep 29, 2020

Compositions and methods for treating cardiovascular disease

Inventors: Maureen Beresini (South San Francisco, CA); Daniel Burdick (Burlingame, CA); Charles Eigenbrot, Jr. (Burlingame, CA); Daniel Kirchhofer (Los Altos, CA); Robert Lazarus (Millbrae, CA); Wei Li (Palo Alto, CA); John Quinn (South San Francisco, CA); Nicholas Skelton (San Mateo, CA); Mark Ultsch (Mill Valley, CA); Yingnan Zhang (South San Francisco, CA)
Assignee: Genentech, Inc.
C07K7/02A61K47/62A61P3/06C07K14/00C12N9/64C12N9/6424C12Q1/37C40B40/10G01N33/573G01N33/92A61K38/00G01N2500/00
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Quick Facts
Patent No.
US 10,787,484
App. No.
16/229,226
Granted
Sep 29, 2020
Kind
B2
Abstract

The invention provides PCSK9 inhibitors, compositions comprising the PCSK9 inhibitors, and methods of identifying and using the PCSK9 inhibitors.

Claims (481)

1. A PCSK9 inhibitor that binds an epitope of SEQ ID NO:1, wherein the epitope comprises at least one residue selected from the group consisting of V241, T339, D343, P364, A442, A443, and L444 of SEQ ID NO: 1 selected from compounds of Formula I:

Formula I

(SEQ ID NO: 2)

R 1 -X 1 -X 2 -X 3 -Trp-Asn-Leu-X 4 -X 5 -Ile-Gly-X 6 -R 2 ,

and pharmaceutically acceptable salts thereof; wherein,

R 1 is C 1 -C 4 acyl; or R 1 is absent;

X 1 is an amino acid sequence selected from: TVFTSWEEYLDWV (SEQ ID NO:3) and TAFTSWEEYLDWV (SEQ ID NO: 4); or X 1 is absent;

X 2 is an amino acid sequence comprising 1 to 4 amino acid residues selected from: glycine and serine; or X 2 is an amino acid residue selected from: 5-aminopentanoic acid, 6-aminohexanoic acid, 7-aminoheptanoic acid, 8-aminooctanoic acid, 9-aminonoanoic acid, 10-aminodecanoic acid, and 11-aminoundecanoic acid; or X 2 is absent;

X 3 is an amino acid sequence comprising 1 to 4 amino acid residues selected from: alanine, 2-aminocyclohexane-1-carboxylic acid, arginine, aspartic acid, cysteine, glycine, 3-hydroxyproline, isoleucine, leucine, methionine, phenylalanine, proline, and tryptophan;

X 4 is an amino acid residue selected from: alanine, 2,3-diaminopropionic acid, glutamine, glycine, lysine, and valine;

X 5 is an amino acid residue selected from: arginine and homo-arginine;

X 6 is an amino acid sequence comprising 1 to 5 amino acid residues selected from:

alanine, arginine, cysteine, glutamic acid, glutamine, glycine, leucine, lysine, phenylalanine, proline, serine, threonine, and tryptophan; or X 6 is absent; and

R 2 is amino; or R 2 is absent.

2. The PCSK9 inhibitor of claim 1 , wherein R 1 is C 1 -C 4 acyl.

3. The PCSK9 inhibitor of claim 2 , wherein R 1 is acetyl.

4. The PCSK9 inhibitor of claim 2 , wherein R 1 is valeryl.

5. The PCSK9 inhibitor of claim 1 , wherein R 1 is absent.

6. The PCSK9 inhibitor of claim 1 , wherein X 1 is an amino acid sequence selected from: TVFTSWEEYLDWV (SEQ ID NO: 3) and TAFTSWEEYLDWV (SEQ ID NO: 4).

7. The PCSK9 inhibitor of claim 6 , wherein X 1 is the amino acid sequence TVFTSWEEYLDWV (SEQ ID NO: 3).

8. The PCSK9 inhibitor of claim 6 , wherein X 1 is the amino acid sequence TAFTSWEEYLDWV (SEQ ID NO: 4).

9. The PCSK9 inhibitor of claim 1 , wherein X 1 is absent.

10. The PCSK9 inhibitor of claim 1 , wherein X 2 is an amino acid sequence comprising 1 to 4 amino acid residues selected from: glycine and serine.

11. The PCSK9 inhibitor of claim 10 , wherein X 2 is an amino acid sequence selected from: SG, GSG, GGSG (SEQ ID NO: 11), GSGG (SEQ ID NO: 12), and SGSG (SEQ ID NO: 13).

12. The PCSK9 inhibitor of claim 1 , wherein X 2 is an amino acid residue selected from: 5-aminopentanoic acid, 6-aminohexanoic acid, 7-aminoheptanoic acid, 8-aminooctanoic acid, 9-aminonoanoic acid, 10-aminodecanoic acid, and 11-aminoundecanoic acid.

13. The PCSK9 inhibitor of claim 12 , wherein X 2 is the amino acid residue 6-aminohexanoic acid.

14. The PCSK9 inhibitor of claim 12 , wherein X 2 is the amino acid residue 7-aminoheptanoic acid.

15. The PCSK9 inhibitor of claim 12 , wherein X 2 is the amino acid residue 8-aminooctanoic acid.

16. The PCSK9 inhibitor of claim 1 , wherein X 2 is absent.

17. The PCSK9 inhibitor of claim 1 , wherein X 3 is an amino acid sequence selected from: P, MP, LMP, ALMP (SEQ ID NO: 14), CALP (SEQ ID NO: 15), CFIP (SEQ ID NO: 16), CFLP (SEQ ID NO: 17), CRAP (SEQ ID NO: 18), CRL(Hyp) (SEQ ID NO: 19), CRLP (SEQ ID NO: 20), DAMP (SEQ ID NO: 21), DLAP (SEQ ID NO: 22), and DLMP (SEQ ID NO: 23).

18. The PCSK9 inhibitor of claim 1 , wherein X 4 is the amino acid residue alanine.

19. The PCSK9 inhibitor of claim 1 , wherein X 4 is the amino acid residue 2,3-diaminopropionic acid.

20. The PCSK9 inhibitor of claim 1 , wherein X 4 is the amino acid residue glutamine.

21. The PCSK9 inhibitor of claim 1 , wherein X 4 is the amino acid residue glycine.

22. The PCSK9 inhibitor of claim 1 , wherein X 4 is the amino acid residue lysine.

23. The PCSK9 inhibitor of claim 1 , wherein X 4 is the amino acid residue valine.

24. The PCSK9 inhibitor of claim 1 , wherein X 5 is the amino acid residue arginine.

25. The PCSK9 inhibitor of claim 1 , wherein X 5 is the amino acid residue homo-arginine.

26. The PCSK9 inhibitor of claim 1 , wherein X 6 is an amino acid sequence selected from: L, S, LL, LAR, LGC, LLA, LLC, LLR, LPC, LTR, SQGC (SEQ ID NO: 24), SQCWF (SEQ ID NO: 26), and SQGCW (SEQ ID NO: 27).

27. The PCSK9 inhibitor of claim 1 , wherein X 6 is absent.

28. The PCSK9 inhibitor of claim 1 , wherein R 2 is amino.

29. The PCSK9 inhibitor of claim 1 , wherein R 2 is absent.

30. The PCSK9 inhibitor of claim 1 , selected from the following compounds and pharmaceutically acceptable salts thereof:

(SEQ ID NO: 29)

Ac-TVFTSWEEYLDWV-GSG-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 38)

Ac-TVFTSWEEYLDWV-GSG-CWNLGRIGSQGC-NH2;

(SEQ ID NO: 40)

Ac-TVFTSWEEYLDWV-GGSG-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 41)

Ac-TVFTSWEEYLDWV-GSGG-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 42)

Ac-TVFTSWEEYLDWV-(Aoc)-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 44)

Ac-TVFTSWEEYLDWV-(Ahx)-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 46)

Ac-TVFTSWEEYLDWV-GSG-CRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 47)

Ac-TVFTSWEEYLDWV-GSG-DLMPWNLVRIGLLR-NH2;

(SEQ ID NO: 48)

Ac-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 49)

Ac-GSG-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 51)

Ac-TVFTSWEEYLDWV-(Ahp)-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 149)

Ac-TVFTSWEEYLDWV-SGG-CRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 52)

Ac-TVFTSWEEYLDWV-GSGG-CRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 53)

SG-CFIPWNLQRIGLLC-NH2;

(SEQ ID NO: 54)

SG-CRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 55)

SG-DLMPWNLVRIGLLR

(SEQ ID NO: 56)

Ac-SG-CFIPWNLQRIGLLC-NH2;

(SEQ ID NO: 57)

Ac-SG-CRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 58)

Ac-CFIPWNLQRIGLLC-NH2;

(SEQ ID NO: 59)

Ac-CRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 62)

Ac-DLMPWNLVRIGLLR-NH2;

(SEQ ID NO: 64)

Ac-SG-CRL(Hyp)WNLQRIGLPC-NH2;

(SEQ ID NO: 65)

Ac-TVFTSWEEYLDWV-GSGG-CFLPWNLQRIGLLC-NH2;

(SEQ ID NO: 66)

Ac-CWNLKRIGSQGCW-NH2;

(SEQ ID NO: 67)

Ac-GSG-CWNLKRIGSQGCW-NH2;

(SEQ ID NO: 69)

Ac-DLMAWNLVRIGLLR-NH2;

(SEQ ID NO: 70)

Ac-DLAPWNLVRIGLLR-NH2;

(SEQ ID NO: 71)

Ac-DAMPWNLVRIGLLR-NH2;

(SEQ ID NO: 72)

Ac-ALMPWNLVRIGLLR-NH2;

(SEQ ID NO: 73)

Ac-DLMPWNLVRIGLL-NH2;

(SEQ ID NO: 74)

Ac-DLMPWNLVRIGL-NH2;

(SEQ ID NO: 75)

Ac-DLMPWNLVRIG-NH2;

(SEQ ID NO: 77)

Ac-LMPWNLVRIGLLR-NH2;

(SEQ ID NO: 78)

Ac-MPWNLVRIGLLR-NH2;

(SEQ ID NO: 79)

Ac-PWNLVRIGLLR-NH2;

(SEQ ID NO: 81)

Ac-DLMPWNLVRIGLLA-NH2;

(SEQ ID NO: 82)

Ac-DLMPWNLVRIGLAR-NH2;

(SEQ ID NO: 83)

Ac-DLMPWNLVRIGALR-NH2;

(SEQ ID NO: 87)

Ac-DLMPWNLARIGLLR-NH2;

(SEQ ID NO: 150)

GCLWNLKRIGSQCWF

(SEQ ID NO: 94)

Ac-CRLPWNLKRIGLPC-NH2;

(SEQ ID NO: 95)

Ac-CRLPWNLQRIGLAc-NH2;

(SEQ ID NO: 96)

Ac-CRLPWNLQRIGAPC-NH2;

(SEQ ID NO: 100)

Ac-CRLPWNLARIGLPC-NH2;

(SEQ ID NO: 104)

Ac-CRLAWNLQRIGLPC-NH2;

(SEQ ID NO: 105)

Ac-CRAPWNLQRIGLPC-NH2;

(SEQ ID NO: 106)

Ac-CALPWNLQRIGLPC-NH2;

(SEQ ID NO: 107)

Ac-CRLPWNLQRIGLGC-NH2;

(SEQ ID NO: 108)

Ac-CRLPWNLQRIGGGC-NH2;

(SEQ ID NO: 109)

Ac-CRL(Ach)WNLQRIGLPC-NH2;

(SEQ ID NO: 114)

Ac-GCLWNLKRIGSQCWF-NH2;

(SEQ ID NO: 151)

Ac-GCLWNLARIGSQCWF-NH2;

(SEQ ID NO: 115)

Ac-FCLWNLARIGSQCWF-NH2;

(SEQ ID NO: 152)

GCLWNLKRIGSQCWF-NH2;

(SEQ ID NO: 116)

WCLWNLKRIGSQCWF-NH2;

(SEQ ID NO: 153)

Ac-GCLWNLKRIGSQCWF

(SEQ ID NO: 117)

Ac-WCLWNLKRIGSQCWF

(SEQ ID NO: 118)

Ac-TVFTSWEEYLDWV-(Ahx)-AWNLKRIGS-NH2;

(SEQ ID NO: 119)

Ac-TVFTSWEEYLDWV-(Ahx)-AWNLKRIG-NH2;

(SEQ ID NO: 123)

Ac-FCLWNLKRIGAQCWF-NH2;

(SEQ ID NO: 124)

Ac-GSG-CWNL(Dpr)RIGSQGC-NH2;

(SEQ ID NO: 126)

Ac-CRLPWNLQRIGLpC-NH2;

(SEQ ID NO: 127)

Ac-CRLPWNLQRIGIPC-NH2;

(SEQ ID NO: 130)

Ac-CRLPWNLqRIGLPC-NH2;

(SEQ ID NO: 134)

Ac-CRLpWNLQRIGLPC-NH2;

(SEQ ID NO: 135)

Ac-CR1PWNLQRIGLPC-NH2;

(SEQ ID NO: 136)

Ac-CrLPWNLQRIGLPC-NH2;

(SEQ ID NO: 137)

Ac-cRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 144)

Ac-CRLPWNLQ(homoR)IGLPC-NH2;

and

(SEQ ID NO: 146)

Ac-PWNLVRIGL-NH2.

31. A PCSK9 inhibitor that binds an epitope of SEQ ID NO:1, wherein the epitope comprises at least one residue selected from the group consisting of V241, T339, D343, P364, A442, A443, and L444 of SEQ ID NO: 1 selected from compounds of Formula II:

Formula II

 (SEQ ID NO: 154)

R 1 -X 1 -X 2 -X 3 -X 7 -Asn-Leu-X 4 -X 5 -Ile-Gly-X6-R 2 ,

and pharmaceutically acceptable salts thereof;

wherein,

R 1 is selected from: C 1 -C 4 acyl, arylcarbonyl, C 3 -C 7 cycloalkylcarbonyl, and heteroarylcarbonyl; each optionally substituted with one or two substituents each independently selected from:

C 1 -C 4 alkyl;

amino;

aryl, optionally substituted with one or two substituents each independently selected from: C 1 -C 4 alkoxy, amino, halo, and hydroxy;

aryl-C 1 -C 4 alkyl;

arylcarbonyl, optionally substituted with one substituent selected from: C 1 -C 4 alkyl and C 1 -C 4 alkylsulfonyl;

aryloxy, optionally substituted with one or two substituents each independently selected from: C 1 -C 4 acyl, C 1 -C 4 alkoxy, C 1 -C 4 alkyl, halo, and hydroxy-C 1 -C 4 alkyl;

carboxyamino, optionally substituted with one substituent selected from: aryl-C 1 -C 4 alkyl;

C 3 -C 7 cycloalkyl;

heteroaryl, optionally substituted with one or two substituents each independently selected from: C 1 -C 4 alkyl and amino;

heterocyclyl, optionally substituted with one or two substituents each independently selected from: C 1 -C 4 alkyl, C 1 -C 4 alkylsulfonyl, carboxamide, heteroaryl-C 1 -C 4 alkyl, and oxo;

heterocyclylcarbonyl; and

ureido;

or R 1 is absent;

X 1 is an amino acid sequence selected from: TVFTSWEEYLDWV (SEQ ID NO: 3), TVFTS(W6fl)EEYLDWV (SEQ ID NO: 30), and TAFTSWEEYLDWV (SEQ ID NO: 4); or X 1 is absent;

X 2 is an amino acid sequence comprising 1 to 4 amino acid residues selected from: glycine and serine; or X 2 is an amino acid residue selected from: 5-aminopentanoic acid, 6-aminohexanoic acid, 7-aminoheptanoic acid, 8-aminooctanoic acid, 9-aminonoanoic acid, 10-aminodecanoic acid, and 11-aminoundecanoic acid; or X 2 is absent;

X 3 is an amino acid sequence comprising 1 to 6 amino acid residues selected from: alanine, 2-aminocyclohexane-1-carboxylic acid, arginine, aspartic acid, cysteine, glutamic acid, glycine, 3-hydroxyproline, isoleucine, leucine, methionine, phenylalanine, proline, serine, tryptophan, and tyrosine;

X 4 is an amino acid residue selected from: alanine, 2,3-diaminopropionic acid, glutamine, glycine, lysine, and valine;

X 5 is an amino acid residue selected from: arginine and homo-arginine;

X 6 is an amino acid sequence comprising 1 to 5 amino acid residues selected from: alanine, arginine, aspartic acid, cysteine, glutamic acid, glutamine, glycine, leucine, lysine, phenylalanine, proline, serine, threonine, and tryptophan; or X 6 is absent;

X 7 is an amino acid residue selected from: tryptophan, 6-fluorotryptophan, 6-chlorotryptophan, 6-bromotryptophan, and 6-methyltryptophan; and

R 2 is amino; or R 2 is absent.

32. The PCSK9 inhibitor of claim 31 , wherein R 1 is C 1 -C 4 acyl.

33. The PCSK9 inhibitor of claim 32 , wherein R 1 is acetyl.

34. The PCSK9 inhibitor of claim 32 , wherein R 1 is valeryl.

35. The PCSK9 inhibitor of claim 31 , wherein R 1 is absent.

36. The PCSK9 inhibitor of claim 31 , wherein R 1 is selected from: C 1 -C 4 acyl, arylcarbonyl, C 3 -C 7 cycloalkylcarbonyl, and heteroarylcarbonyl; each optionally substituted with one or two substituents each independently selected from:

C 1 -C 4 alkyl;

amino;

aryl, optionally substituted with one or two substituents each independently selected from: methoxy, amino, fluoro, chloro, and hydroxy;

aryl-C 1 -C 4 alkyl;

arylcarbonyl, optionally substituted with one substituent selected from: methyl and methylsulfonyl;

aryloxy, optionally substituted with one or two substituents each independently selected from: acetyl, methoxy, methyl, ethyl, bromo, and hydroxymethyl;

carboxyamino, optionally substituted with one substituent selected from: phenylmethyl;

C 3 -C 7 cycloalkyl;

heteroaryl, optionally substituted with one or two substituents each independently selected from: methyl and amino;

heterocyclyl, optionally substituted with one or two substituents each independently selected from: methyl, methylsulfonyl, carboxamide, pyridinylmethyl, and oxo;

heterocyclylcarbonyl; and

ureido.

37. The PCSK9 inhibitor of claim 31 , wherein R 1 is selected from: C 1 -C 4 acyl, arylcarbonyl, C 3 -C 7 cycloalkylcarbonyl, and heteroarylcarbonyl; each optionally substituted with one or two substituents each independently selected from:

C 1 -C 4 alkyl selected from: methyl and t-butyl;

amino;

phenyl, optionally substituted with one or two substituents each independently selected from: methoxy, amino, fluoro, chloro, and hydroxy;

phenylmethyl;

benzoyl, optionally substituted with one substituent selected from: methyl and methylsulfonyl;

phenoxy, optionally substituted with one or two substituents each independently selected from: acetyl, methoxy, methyl, ethyl, bromo, and hydroxymethyl;

carboxyamino, optionally substituted with one substituent selected from: phenylmethyl;

cyclohexyl;

heteroaryl selected from: imidazolyl, indolyl, pyrazolyl, pyridinyl, and triazolyl, each optionally substituted with one or two substituents each independently selected from: methyl and amino;

heterocyclyl selected from: morpholinyl, piperidinyl, piperazinyl, pyrrolidinyl, tetrahydrothiophenyl, tetrahydropyrimidinyl, thiomorpholinyl, each optionally substituted with one or two substituents each independently selected from: methyl, methylsulfonyl, carboxamide, pyridinylmethyl, and oxo;

morpholinylcarbonyl; and

ureido.

38. The PCSK9 inhibitor of claim 31 , wherein R 1 is selected from: acetyl, n-propionyl, n-butanoyl, isovaleryl, valeryl, benzoyl, cyclopropylcarbonyl, cyclobutylcarbonyl, cyclohexylcarbonyl, indolylcarbonyl, pyrazolylcarbonyl, and pyridinylcarbonyl; each optionally substituted with one or two substituents each independently selected from:

C 1 -C 4 alkyl selected from: methyl and t-butyl;

amino;

phenyl, optionally substituted with one or two substituents each independently selected from: methoxy, amino, fluoro, chloro, and hydroxy;

phenylmethyl;

benzoyl, optionally substituted with one substituent selected from: methyl and methylsulfonyl;

phenoxy, optionally substituted with one or two substituents each independently selected from: acetyl, methoxy, methyl, ethyl, bromo, and hydroxymethyl;

carboxyamino, optionally substituted with one substituent selected from: phenylmethyl;

cyclohexyl;

heteroaryl selected from: imidazolyl, indolyl, pyrazolyl, pyridinyl, and triazolyl, each optionally substituted with one or two substituents each independently selected from: methyl and amino;

heterocyclyl selected from: morpholinyl, piperidinyl, piperazinyl, pyrrolidinyl, tetrahydrothiophenyl, tetrahydropyrimidinyl, thiomorpholinyl, each optionally substituted with one or two substituents each independently selected from: methyl, methylsulfonyl, carboxamide, pyridinylmethyl, and oxo;

morpholinylcarbonyl; and

ureido.

39. The PCSK9 inhibitor of claim 31 , wherein R 1 is selected from: acetyl, n-butanoyl, dioxidothiomorpholino)propanoyl, 2-(1-methyl-1H-pyrazol-4-yl)cyclopropane-1-carbonyl, 3-(2-oxopyrrolidin-1-yl)propanoyl, 1-(1-methylpyrrolidin-3-yl)-1H-pyrazole-4-carbonyl, 2-(1-(pyridin-2-ylmethyl)piperidin-4-yl)acetyl, 3-(4-carbamoylpiperazin-1-yl)propanoyl, 3-(1H-imidazol-4-yl)propanoyl, 2-(1-(methylsulfonyl)piperidin-4-yl)acetyl, 6-morpholinonicotinoyl, 3-(3,5-dimethyl-1H-pyrazol-1-yl)propanoyl, 3-morpholinopropanoyl, 3-(pyridin-3-yl)propanoyl, 2-(1,1-dioxidotetrahydrothiophen-3-yl)acetyl, 3-(4-methylpiperazin-1-yl)propanoyl, 3-ureidopropanoyl, 3-(1H-1,2,4-triazol-1-yl)propanoyl, 2-(2,4-dioxo-3,4-dihydropyrimidin-1(2H)-yl)acetyl, 3-benzylcyclobutane-1-carbonyl, [1,1′-biphenyl]-4-carbonyl, 5-phenylpentanoyl, 4-phenylcyclohexane-1-carbonyl, 5,5-dimethylhexanoyl, 3,5,5-trimethylhexanoyl, 5-(pyridin-4-yl)-1H-pyrazole-3-carbonyl, 3-cyclohexylpropanoyl, 3-(4-aminophenyl)propanoyl, 4-morpholinobutanoyl, 4-(4-(methylsulfonyl)phenyl)-4-oxobutanoyl, 3-(3-aminophenyl)propanoyl, 4-oxo-4-(p-tolyl)butanoyl, 3-(4-methoxyphenyl)propanoyl, 4-morpholino-4-oxobutanoyl, 3-(3-hydroxyphenyl)propanoyl, 4-(((benzyloxy)carbonyl)amino)butanoyl, 3-(4-hydroxyphenyl)propanoyl, 3-(6-aminopyridin-3-yl)propanoyl, 4-(4-bromophenoxy)butanoyl, 4-(4-acetylphenoxy)butanoyl, 3-(pyridin-4-yl)-1H-pyrazole-5-carbonyl, 3-(1H-indol-3-yl)propanoyl, 4-(1H-indol-3-yl)butanoyl, 4-(4-fluorophenyl)butanoyl, 3-(4-fluorophenyl)propanoyl, 3-(4-chlorophenyl)propanoyl, 3-(3-methoxyphenyl)propanoyl, 3-(4-methoxyphenoxy)propanoyl, 4-(4-(hydroxymethyl)-3-methoxyphenoxy)butanoyl, 3-(4-acetylphenoxy)propanoyl, 4-(p-tolyloxy)butanoyl, 4-phenoxybutanoyl, 4-(4-ethylphenoxy)butanoyl, 3-(p-tolyloxy)propanoyl, 4-phenylbutanoyl, 3-(4-hydroxy-3-methoxyphenyl)propanoyl, 4-(4-hydroxyphenyl)butanoyl, 2-(pyridin-3-yl)cyclopropane-1-carbonyl, 1H-indole-6-carbonyl, 2-amino-3-(4-hydroxyphenyl)propanoyl, and 3-(3,4-dihydroxyphenyl)propanoyl.

40. The PCSK9 inhibitor claim 31 , wherein X 1 is an amino acid sequence selected from: TVFTSWEEYLDWV (SEQ ID NO: 3), TVFTS(W6fl)EEYLDWV (SEQ ID NO: 30), and TAFTSWEEYLDWV (SEQ ID NO: 4).

41. The PCSK9 inhibitor of claim 40 , wherein X 1 is the amino acid sequence TVFTSWEEYLDWV (SEQ ID NO: 3).

42. The PCSK9 inhibitor of claim 40 , wherein X 1 is the amino acid sequence TVFTS(W6fl)EEYLDWV (SEQ ID NO: 30).

43. The PCSK9 inhibitor of claim 40 , wherein X 1 is the amino acid sequence TAFTSWEEYLDWV (SEQ ID NO: 4).

44. The PCSK9 inhibitor of claim 31 , wherein X 1 is absent.

45. The PCSK9 inhibitor of claim 31 , wherein X 2 is an amino acid sequence comprising 1 to 4 amino acid residues selected from: glycine and serine.

46. The PCSK9 inhibitor of claim 45 , wherein X 2 is an amino acid sequence selected from: G, GG, SG, GSG, GGSG (SEQ ID NO: 11), GSGG (SEQ ID NO: 12), and SGSG (SEQ ID NO: 13).

47. The PCSK9 inhibitor of claim 31 , wherein X 2 is an amino acid residue selected from: 5-aminopentanoic acid, 6-aminohexanoic acid, 7-aminoheptanoic acid, 8-aminooctanoic acid, 9-aminonoanoic acid, 10-aminodecanoic acid, and 11-aminoundecanoic acid.

48. The PCSK9 inhibitor of claim 47 , wherein X 2 is the amino acid residue 6-aminohexanoic acid.

49. The PCSK9 inhibitor of claim 47 , wherein X 2 is the amino acid residue 7-aminoheptanoic acid.

50. The PCSK9 inhibitor of claim 47 , wherein X 2 is the amino acid residue 8-aminooctanoic acid.

51. The PCSK9 inhibitor of claim 31 , wherein X 2 is absent.

52. The PCSK9 inhibitor of claim 31 , wherein X 3 is an amino acid sequence selected from: P, MP, LMP, ALMP (SEQ ID NO: 14), CALP (SEQ ID NO: 15), CFI(Hyp) (SEQ ID NO: 155), CFIP (SEQ ID NO: 16), CFLP (SEQ ID NO: 17), CRAP (SEQ ID NO: 18), CRL(Hyp) (SEQ ID NO: 19), CRLP (SEQ ID NO: 20), DAMP (SEQ ID NO: 21), DLAP (SEQ ID NO: 22), DLMP (SEQ ID NO: 23), DSYPG (SEQ ID NO: 156), ESFPG (SEQ ID NO: 157), ESYPG (SEQ ID NO: 158), MDSFPG (SEQ ID NO: 159), MESFPG (SEQ ID NO: 160), and SFAFPG (SEQ ID NO: 161).

53. The PCSK9 inhibitor of claim 31 , wherein X 4 is the amino acid residue alanine.

54. The PCSK9 inhibitor of claim 31 , wherein X 4 is the amino acid residue 2,3-diaminopropionic acid.

55. The PCSK9 inhibitor of claim 31 , wherein X 4 is the amino acid residue glutamine.

56. The PCSK9 inhibitor of claim 31 , wherein X 4 is the amino acid residue glycine.

57. The PCSK9 inhibitor of claim 31 , wherein X 4 is the amino acid residue lysine.

58. The PCSK9 inhibitor of claim 31 , wherein X 4 is the amino acid residue valine.

59. The PCSK9 inhibitor of claim 31 , wherein X 5 is the amino acid residue arginine.

60. The PCSK9 inhibitor of claim 31 , wherein X 5 is the amino acid residue homo-arginine.

61. The PCSK9 inhibitor of claim 31 , wherein X 6 is an amino acid sequence selected from: L, S, LL, LAR, LDR, LER, LGC, LGR, LLA, LLC, LLQ, LLR, LPC, LPR, LSR, LTR, SQGC (SEQ ID NO: 24), SQCEY (SEQ ID NO: 25), SQCWF (SEQ ID NO: 26), and SQGCW (SEQ ID NO: 27).

62. The PCSK9 inhibitor of claim 31 , wherein X 6 is absent.

63. The PCSK9 inhibitor of claim 31 , wherein X 7 is tryptophan.

64. The PCSK9 inhibitor of claim 31 , wherein X 7 is 6-fluorotryptophan.

65. The PCSK9 inhibitor of claim 31 , wherein X 7 is 6-chlorotryptophan.

66. The PCSK9 inhibitor of claim 31 , wherein X 7 is 6-bromotryptophan.

67. The PCSK9 inhibitor of claim 31 , wherein X 7 is 6-methyltryptophan.

68. The PCSK9 inhibitor of claim 31 , wherein R 2 is amino.

69. The PCSK9 inhibitor of claim 31 , wherein R 2 is absent.

70. The PCSK9 inhibitor of claim 31 , selected from the following compounds and pharmaceutically acceptable salts thereof:

(SEQ ID NO: 29)

Ac-TVFTSWEEYLDWV-GSG-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 38) 

Ac-TVFTSWEEYLDWV-GSG-CWNLGRIGSQGC-NH2;

(SEQ ID NO: 40) 

Ac-TVFTSWEEYLDWV-GGSG-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 41) 

Ac-TVFTSWEEYLDWV-GSGG-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 42) 

Ac-TVFTSWEEYLDWV-(Aoc)-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 44) 

Ac-TVFTSWEEYLDWV-(Ahx)-CWNLKRIGSQGC-NH2

(SEQ ID NO: 46) 

Ac-TVFTSWEEYLDWV-GSG-CRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 47) 

Ac-TVFTSWEEYLDWV-GSG-DLMPWNLVRIGLLR-NH2;

(SEQ ID NO: 48) 

Ac-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 49) 

Ac-GSG-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 51) 

Ac-TVFTSWEEYLDWV-(Ahp)-CWNLKRIGSQGC-NH2;

(SEQ ID NO: 149) 

Ac-TVFTSWEEYLDWV-SGG-CRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 52) 

Ac-TVFTSWEEYLDWV-GSGG-CRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 53) 

SG-CFIPWNLQRIGLLC-NH2;

(SEQ ID NO: 54) 

SG-CRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 55) 

SG-DLMPWNLVRIGLLR;

(SEQ ID NO: 56) 

Ac-SG-CFIPWNLQRIGLLC-NH2;

(SEQ ID NO: 57)  

Ac-SG-CRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 58) 

Ac-CFIPWNLQRIGLLC-NH2;

(SEQ ID NO: 59) 

Ac-CRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 62) 

Ac-DLMPWNLVRIGLLR-NH2;

(SEQ ID NO: 64) 

Ac-SG-CRL(Hyp)WNLQRIGLPC-NH2;

(SEQ ID NO: 65) 

Ac-TVFTSWEEYLDWV-GSGG-CFLPWNLQRIGLLC-NH2;

(SEQ ID NO: 66) 

Ac-CWNLKRIGSQGCW-NH2;

(SEQ ID NO: 67) 

Ac-GSG-CWNLKRIGSQGCW-NH2;

(SEQ ID NO: 69) 

Ac-DLMAWNLVRIGLLR-NH2;

(SEQ ID NO: 70) 

Ac-DLAPWNLVRIGLLR-NH2;

(SEQ ID NO: 71) 

Ac-DAMPWNLVRIGLLR-NH2;

(SEQ ID NO: 72) 

Ac-ALMPWNLVRIGLLR-NH2;

(SEQ ID NO: 73) 

Ac-DLMPWNLVRIGLL-NH2;

(SEQ ID NO: 74) 

Ac-DLMPWNLVRIGL-NH2;

(SEQ ID NO: 75) 

Ac-DLMPWNLVRIG-NH2;

(SEQ ID NO: 77) 

Ac-LMPWNLVRIGLLR-NH2;

(SEQ ID NO: 78) 

Ac-MPWNLVRIGLLR-NH2;

(SEQ ID NO: 79) 

Ac-PWNLVRIGLLR-NH2;

(SEQ ID NO: 81) 

Ac-DLMPWNLVRIGLLA-NH2;

(SEQ ID NO: 82) 

Ac-DLMPWNLVRIGLAR-NH2;

(SEQ ID NO: 83) 

Ac-DLMPWNLVRIGALR-NH2;

(SEQ ID NO: 87) 

Ac-DLMPWNLARIGLLR-NH2;

(SEQ ID NO: 150) 

GCLWNLKRIGSQCWF;

(SEQ ID NO: 94) 

Ac-CRLPWNLKRIGLPC-NH2;

(SEQ ID NO: 95) 

Ac-CRLPWNLQRIGLAC-NH2;

(SEQ ID NO: 96) 

Ac-CRLPWNLQRIGAPC-NH2;

(SEQ ID NO: 100) 

Ac-CRLPWNLARIGLPC-NH2;

(SEQ ID NO: 101) 

Ac-CRLAWNLQRIGLPC-NH2;

(SEQ ID NO: 102) 

Ac-CRAPWNLQRIGLPC-NH2;

(SEQ ID NO: 103) 

Ac-CALPWNLQRIGLPC-NH2;

(SEQ ID NO: 107) 

Ac-CRLPWNLQRIGLGC-NH2;

(SEQ ID NO: 108) 

Ac-CRLPWNLQRIGGGC-NH2;

(SEQ ID NO: 109) 

Ac-CRL(Ach)WNLQRIGLPC-NH2;

(SEQ ID NO: 114) 

Ac-GCLWNLKRIGSQCWF-NH2;

(SEQ ID NO: 151) 

Ac-GCLWNLARIGSQCWF-NH2;

(SEQ ID NO: 115) 

Ac-FCLWNLARIGSQCWF-NH2;

(SEQ ID NO: 152) 

GCLWNLKRIGSQCWF-NH2;

(SEQ ID NO: 116) 

WCLWNLKRIGSQCWF-NH2;

(SEQ ID NO: 153) 

Ac-GCLWNLKRIGSQCWF;

(SEQ ID NO: 117) 

Ac-WCLWNLKRIGSQCWF;

(SEQ ID NO: 118) 

Ac-TVFTSWEEYLDWV-(Ahx)-AWNLKRIGS-NH2;

(SEQ ID NO: 119) 

Ac-TVFTSWEEYLDWV-(Ahx)-AWNLKRIG-NH2;

(SEQ ID NO: 123) 

Ac-FCLWNLKRIGAQCWF-NH2;

(SEQ ID NO: 124) 

Ac-GSG-CWNL(Dpr)RIGSQGC-NH2;

(SEQ ID NO: 126) 

Ac-CRLPWNLQRIGLpC-NH2;

(SEQ ID NO: 127) 

Ac-CRLPWNLQRIGIPC-NH2;

(SEQ ID NO: 128)

Ac-CRLPWNLQRiGLPC-NH2;

(SEQ ID NO: 129) 

Ac-CRLPWNLQrIGLPC-NH2;

(SEQ ID NO: 130) 

Ac-CRLPWNLqRIGLPC-NH2;

(SEQ ID NO: 131) 

Ac-CRLPWNIQRIGLPC-NH2;

(SEQ ID NO: 132) 

Ac-CRLPWnLQRIGLPC-NH2;

(SEQ ID NO: 133) 

Ac-CRLPRwNLQRIGLPC-NH2;

(SEQ ID NO: 134) 

Ac-CRLpWNLQRIGLPC-NH2;

(SEQ ID NO: 135) 

Ac-CRlPWNLQRIGLPC-NH2;

(SEQ ID NO: 136) 

Ac-CrLPWNLQRIGLPC-NH2;

(SEQ ID NO: 137) 

Ac-cRLPWNLQRIGLPC-NH2;

(SEQ ID NO: 144) 

Ac-CRLPWNLQ(homoR)IGLPC-NH2;

(SEQ ID NO: 146)

Ac-PWNLVRIGL-NH2;

(SEQ ID NO: 162) 

Ac-SG-DLMPWNLVRIGLLR-NH2;

(SEQ ID NO: 163) 

n-PrC(O)-WNLVRIGLLR-NH2;

(SEQ ID NO: 164) 

Ac-G-LMPWNLVRIGLLR-NH2;

(SEQ ID NO: 165) 

Ac-GG-MPWNLVRIGLLR-NH2;

(SEQ ID NO: 193)

Ac-MDSFPGWNLVRIGLLR-NH2;

(SEQ ID NO: 194) 

Ac-SFAFPGWNLVRIGLLR-NH2;

(SEQ ID NO: 195) 

Ac-DSYPGWNLVRIGLLR-NH2;

(SEQ ID NO: 196) 

Ac-ESYPGWNLVRIGLLR-NH2;

(SEQ ID NO: 197) 

Ac-ESFPGWNLVRIGLLR-NH2;

(SEQ ID NO: 198) 

Ac-DLMPWNLKRIGLLR-NH2;

(SEQ ID NO: 200)

Ac-DLMPWNLVRIGLPR-NH2;

(SEQ ID NO: 267) 

Ac-ESFPGWNLV(homoR)IGLLR-NH 2 ;

(SEQ ID NO: 268) 

Ac-SFAFPGWNLV(homoR)IGLLR-NH 2 ;

(SEQ ID NO: 269) 

Ac-MESFPGWNLV(homoR)IGLLR-NH 2 ;

(SEQ ID NO: 270) 

Ac-DSYPGWNLV(homoR)IGLLR-NH 2 ;

(SEQ ID NO: 271) 

Ac-ESYPGWNLV(homoR)IGLLR-NH 2 ;

(SEQ ID NO: 272) 

Ac-SFAFPGWNLK(homoR)IGLLR-NH 2 ;

(SEQ ID NO: 273) 

Ac-MESFPGWNLK(homoR)IGLLR-NH 2 ;

(SEQ ID NO: 281)

Ac-TVFTS(W6fl)EEYLDWV-GSG-CRLPWNLQRIGLPC-NH2;

and

(SEQ ID NO: 282) 

Ac-SG-CFI(Hyp)WNLQRIGLLC-NH2.

71. An inhibited PCSK9 comprising a PCSK9 inhibitor of claim 1 bound to PCSK9.

72. A pharmaceutical composition comprising a PCSK9 inhibitor of claim 1 and a pharmaceutically acceptable carrier.

73. A PCSK9 inhibitor, or pharmaceutically acceptable salts thereof selected from the group consisting of:

Continuity (3)
Continuation PCTEP2017065324 · Jun 21, 2017
Provisional Application 62354631 · Jun 24, 2016
Related Publication 20190177366A1 · Jun 13, 2019