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NZ738016B2 - Amide-linked ep4 agonist-bisphosphonate compounds and uses thereof - Google Patents

Amide-linked ep4 agonist-bisphosphonate compounds and uses thereof Download PDF

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Publication number
NZ738016B2
NZ738016B2 NZ738016A NZ73801616A NZ738016B2 NZ 738016 B2 NZ738016 B2 NZ 738016B2 NZ 738016 A NZ738016 A NZ 738016A NZ 73801616 A NZ73801616 A NZ 73801616A NZ 738016 B2 NZ738016 B2 NZ 738016B2
Authority
NZ
New Zealand
Prior art keywords
compound
bone
fracture
group
bis
Prior art date
Application number
NZ738016A
Other versions
NZ738016A (en
Inventor
Gang Chen
Marion Thevenin
Robert N Young
Original Assignee
Simon Fraser University
Filing date
Publication date
Application filed by Simon Fraser University filed Critical Simon Fraser University
Priority claimed from PCT/IB2016/053482 external-priority patent/WO2016199111A1/en
Publication of NZ738016A publication Critical patent/NZ738016A/en
Publication of NZ738016B2 publication Critical patent/NZ738016B2/en

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/548Phosphates or phosphonates, e.g. bone-seeking
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/28Phosphorus compounds with one or more P—C bonds
    • C07F9/38Phosphonic acids [RP(=O)(OH)2]; Thiophosphonic acids ; [RP(=X1)(X2H)2(X1, X2 are each independently O, S or Se)]
    • C07F9/3804Phosphonic acids [RP(=O)(OH)2]; Thiophosphonic acids ; [RP(=X1)(X2H)2(X1, X2 are each independently O, S or Se)] not used, see subgroups
    • C07F9/3839Polyphosphonic acids
    • C07F9/3873Polyphosphonic acids containing nitrogen substituent, e.g. N.....H or N-hydrocarbon group which can be substituted by halogen or nitro(so), N.....O, N.....S, N.....C(=X)- (X =O, S), N.....N, N...C(=X)...N (X =O, S)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/553Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having one nitrogen atom as the only ring hetero atom
    • C07F9/572Five-membered rings

Abstract

The present invention relates to EP4 agonist-bisphosphonate conjugates or related compounds and uses thereof. Said conjugates or related compounds may be used to provide delivery of an EP4 agonist or related compound to a desired site of action, such as a bone. Bisphosphonate moieties, linked to the EP4 agonists via amide linkers, have been implicated in the inhibition of bone resorption and bone targeting.

Claims (29)

WHAT IS CLAIMED IS:
1. A compound, or a pharmaceutically acceptable salt thereof, the compound comprising at least one EP4 agonist or related moiety linked to an amide linker through an ester bond and an amino bisphosphonate moiety linked to the amide linker through an amide bond, wherein the EP4 agonist or related compound is selected from the group consisting of: i) a compound according to the chemical structure wherein: X is -CH -, -S-, -O-, or -NH-; Y is COOR', tetrazole, or C(O)NHSO R; R is alkyl with one to ten carbon atoms or aryl; n is 1, 2, or 3; R is independently H or halogen; Ar is aryl, aryl substituted with halogen or alkyl with one to ten carbon atoms, or heteroaryl; R' is H or alkyl with one to ten carbon atoms, and is a double or single bond; or ii) a compound selected from Compound B, wherein, in Compound B, a dashed line indicates the presence or absence of a bond; A is 1 1 2 1 2 phenyl; X is CH , O or S; Y is OR or NR R and R and R are independently H or C 2 1-6 alkyl; wherein the amide linker has a structure selected from the group consisting of , and wherein R is each independently H, OR', halogen, CN, or C(O)R', R' is each independently H or alkyl with one to ten carbon atoms, or the two R's form a ring of up to 6 carbons, NHR'', if present, is the amino group of the amino bisphosphonate, q, if present, is 1 or 2, and n, if present, is 1, 2 or 3, wherein one or more of the aliphatic carboxylic acid groups of the amide linker can react with the C-15 or equivalent hydroxyl group of the EP4 agonist or related compound, to form the ester bond, and the remaining carboxylic acid group can react with the amino group of the amino bisphosphonate to form the amide bond, and wherein the amino bisphosphonate moiety has the following formula wherein m is 1, 2, 3, 4, 5 or 6.
2. The compound of claim 1 wherein the amide linker is 4-(carboxymethyl) benzoic acid or 3,5-bis-(carboxymethyl)benzoic acid.
3. The compound of claim 1 or claim 2 wherein the bisphosphonate is alendronic acid, 4-aminohydroxybutylidene-1,1-bisphosphonic acid, alendronate, 4-amino hydroxybutylidene-1,1-bisphosphonic acid monosodium trihydrate, 6-amino hydroxyhexylidene-1,1-bisphosphonic acid, or 3-aminohydroxypropylidene-1,1- bisphosphonic acid.
4. A compound according to Formula I, or a pharmaceutically acceptable salt thereof: Formula I wherein: X is -CH -, -S-, -O-, or -NH-; Y is COOR', tetrazole, or C(O)NHSO R; Z is OH or H; R is alkyl with one to ten carbon atoms or aryl; n is 1, 2, or 3; m is 0, 1, 2, 3, 4, 5, or 6; q is 1 or 2; R is independently H or halogen; Ar is aryl, aryl substituted with halogen or alkyl with one to ten carbon atoms, or heteroaryl; R is each independently H, OR', halogen, CN, or C(O)R'; R' is each independently H or alkyl with one to ten carbon atoms, or two R's may form a ring of up to 6 carbons; and is a double or single bond.
5. The compound of claim 1 or 4 selected from the group consisting of: Sodium (4-(4-(2-(((R,E)((R)(7-ethoxyoxoheptyl)oxopyrrolidinyl)-1,1-difluoro- 1-phenylbutenyl)oxy)oxoethyl)benzamido)hydroxybutane-1,1-diyl)bis(hydrogen phosphonate); Sodium (3-(4-(2-(((R,E)((R)(7-ethoxyoxoheptyl)oxopyrrolidinyl)-1,1-difluoro- 1-phenylbutenyl)oxy)oxoethyl)benzamido)hydroxypropane-1,1- diyl)bis(hydrogen phosphonate); Sodium (6-(4-(2-(((R,E)((R)(7-ethoxyoxoheptyl)oxopyrrolidinyl)-1,1-difluoro- 1-phenylbutenyl)oxy)oxoethyl)benzamido)hydroxyhexane-1,1- diyl)bis(hydrogen phosphonate); Sodium (4-(4-(2-(((R,E)((R)(6-carboxyhexyl)oxopyrrolidinyl)-1,1-difluoro phenylbutenyl)oxy)oxoethyl)benzamido)hydroxybutane-1,1-diyl)bis(hydrogen phosphonate); Sodium (3-(4-(2-(((R,E)((R)(6-carboxyhexyl)oxopyrrolidinyl)-1,1-difluoro phenylbutenyl)oxy)oxoethyl)benzamido)hydroxypropane-1,1-diyl)bis(hydrogen phosphonate); Sodium (6-(4-(2-(((R,E)((R)(6-carboxyhexyl)oxopyrrolidinyl)-1,1-difluoro phenylbutenyl)oxy)oxoethyl)benzamido)hydroxyhexane-1,1-diyl)bis(hydrogen phosphonate); and Sodium (4-(3,5-bis(2-(((R,E)((R)(6-carboxyhexyl)oxopyrrolidinyl)-1,1-difluoro phenylbutenyl)oxy)oxoethyl)benzamido)hydroxybutane-1,1-diyl)bis(hydrogen phosphonate).
6. The compound of any one of claims 1 to 5 wherein the compound is hydrolyzable in vivo.
7. The compound of claim 6 wherein the compound is inactive prior to hydrolyzation.
8. The compound of any one of claims 1 to 7 wherein the amide bond is resistant to hydrolysis in vivo.
9. A composition comprising the compound of any one of claims 1 to 8 in combination with a carrier.
10. A pharmaceutical composition comprising the compound of any one of claims 1 to 8, in combination with a pharmaceutically acceptable carrier.
11. Use of the compound of any one of claims 1 to 8, or the pharmaceutical composition of claim 10, in the manufacture of a medicament for treating or preventing a condition associated with abnormal or excessive bone loss, or with abnormal or reduced bone resorption, or with abnormal calcium metabolism, or that would be benefited by administration of an EP4 agonist or related compound, in a subject in need thereof.
12. The use of claim 11, wherein the condition is selected from the group consisting of osteoporosis, Paget's disease, abnormally increased bone turnover, bone graft, periodontal disease, alveolar bone loss, tooth loss, bone fracture, periprostheticosteolysis, osteogenesis imperfecta, metastatic bone disease, and irritable bowel syndrome.
13. Use of the compound of any one of claims 1 to 8, or the pharmaceutical composition of claim 10, in the manufacture of a medicament for selectively delivering a compound to bone, in a subject in need thereof.
14. The use of claim 13 wherein the bone is a bone in need of treatment.
15. The use of claim 14 wherein the bone in need of treatment is selected from the group consisting of a green stick fracture, compound fracture, lateral fracture, pathologic fracture resulting from an invasive tumor, compression fracture, and fracture requiring a surgical procedure for realignment of a bone.
16. The use of any one of claims 11 to 15 wherein the compound binds to bone.
17. The use of any one of claims 11 to 16 wherein the compound is hydrolyzed after binding to bone, is inactive prior to hydrolyzation or releases an active agent after hydrolyzation.
18. The use of any one of claims 11 to 17 wherein the amide bond of the compound is resistant to hydrolysis in vivo.
19. The use of any one of claims 16 to 18 wherein the bisphosphonate moiety remains attached to the bone.
20. A method of preparing a compound in accordance with any one of claims 1 to 8, comprising: i) providing at least one EP4 agonist or related moiety comprising a hydroxyl group, an amide linker comprising at least two carboxylic acid groups, and a bisphosphonate moiety comprising an amino group; ii) reacting one of the carboxylic acid groups of the amide linker with the hydroxyl group of the EP4 agonist or related moiety, to form an ester bond, and iii) reacting the other carboxylic acid of the amide linker group with the amino group of the bisphosphonate to form an amide bond.
21. A method of selectively delivering a compound to bone, the method comprising administering an effective amount of the compound of any one of claims 1 to 8 or the pharmaceutical composition of claim 10 to a non-human subject in need thereof.
22. The method of claim 21 wherein the bone is a bone in need of treatment.
23. The method of claim 22 wherein the bone in need of treatment is selected from the group consisting of a green stick fracture, compound fracture, lateral fracture, pathologic fracture resulting from an invasive tumor, compression fracture, and fracture requiring a surgical procedure for realignment of a bone.
24. A method of treating or preventing a condition associated with abnormal or excessive bone loss, or with abnormal or reduced bone resorption, or with abnormal calcium metabolism, or that would be benefited by administration of an EP4 agonist or related compound, comprising administering an effective amount of the compound of any one of claims 1 to 8, or the pharmaceutical composition of claim 10, to a non-human subject in need thereof.
25. The method of any one of claims 21 to 24 wherein the compound binds to bone.
26. The method of any one of claims 21 to 25 wherein the compound is hydrolyzed after binding to bone, is inactive prior to hydrolyzation or releases an active agent after hydrolyzation.
27. The method of any one of claims 21 to 26 wherein the amide bond of the compound is resistant to hydrolysis in vivo.
28. The method of any one of claims 25 to 27 wherein the bisphosphonate moiety remains attached to the bone.
29. The compound of claim 1 or 4, substantially as herein described with reference to any one of the Examples and/or
NZ738016A 2016-06-13 Amide-linked ep4 agonist-bisphosphonate compounds and uses thereof NZ738016B2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562175118P 2015-06-12 2015-06-12
PCT/IB2016/053482 WO2016199111A1 (en) 2015-06-12 2016-06-13 Amide-linked ep4 agonist-bisphosphonate compounds and uses thereof

Publications (2)

Publication Number Publication Date
NZ738016A NZ738016A (en) 2024-10-25
NZ738016B2 true NZ738016B2 (en) 2025-01-28

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