# US 10,478,446 B2 · Compounds for use in polycystic kidney disease

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- Number: US 10,478,446 B2
- Document ID: US10478446B2
- Status: Granted
- Kind: B2
- Application number: 14/413,280
- Filed: 2013-07-03
- Published: 2015-10-22
- Granted: 2019-11-19
- Earliest claimed: 2012-07-03
- Assignees: Fondazione Centro San Raffaele Office of Biotechnology Transfer; Fondazione Telethon
- Applicants: Fondazione Centro San Raffaele Office of Biotechnology Transfer; Fondazione Telethon
- Inventors: Alessandra Boletta; Marco Chiaravalli; Isaline Severine Marie-Josephe Rowe
- Web page: https://patentgrep.com/patent/US10478446B2

## CPC

- A61K 31/7008: Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
- A61K 31/155: Amidines ([IMAGE cpc-sch-A61K-1029.gif]), e.g. guanidine (H2N—C(=NH)—NH2), isourea (N=C(OH)—NH2), isothiourea (—N=C(SH)—NH2)
- A61K 31/352: condensed with carbocyclic rings, e.g. methantheline {(cannabinoids A61K31/658)}
- A61K 31/436: the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
- A61K 31/437: the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
- A61K 31/44: Non condensed pyridines; Hydrogenated derivatives thereof
- A61K 31/7004: Monosaccharides having only carbon, hydrogen and oxygen atoms
- A61K 45/06: Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
- A61P 13/12: of the kidneys
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

Polycystic kidney disease is a cystic genetic disorder of the kidneys. Recent identification of signaling cascades de-regulated in PKD has led to the initiation of several clinical trials, but an effective therapy is still lacking. A new therapeutic paradigm is capable of improving the proliferation rate and cystic kidney volume in PKD.

## Claims

### Claim 1

A method of treating autosomal dominant polycystic kidney disease (ADPKD) in a subject in need thereof, comprising administering an effective amount of a pharmaceutical composition comprising 2-deoxyglucose (2DG).

### Claim 2 (depends on 1)

A method of treatment according to claim 1, wherein said 2DG is administered at a dosage in the range 20-250 mg/kg.

### Claim 3 (depends on 1)

A method of treatment according to claim 1, wherein said 2DG is administered at a dosage in the range 30-200 mg/kg.

### Claim 4 (depends on 1)

A method of treatment according to claim 1, wherein said 2DG is administered at a dosage in the range 40-150 mg/kg.

### Claim 5 (depends on 1)

A method of treatment according to claim 1, wherein said 2DG is administered at a dosage in the range 45-100 mg/kg.

### Claim 6 (depends on 1)

A method of treatment according to claim 1, wherein said 2DG is administered at a dosage in the range 50-65 mg/kg.

### Claim 7 (depends on 1)

A method of treatment according to claim 1, wherein said 2DG is administered at a dosage of about 60 mg/kg.

### Claim 8 (depends on 1)

The method of treatment according to claim 1, wherein said pharmaceutical composition further comprises at least one extracellular signal-regulated kinases (ERK) inhibitor selected from 2-(2-Amino-3-methoxyphenyl)-4H-1-benzopyran-4-one (PD98059), 1,4-Diamino-2,3-dicyano-1,4-bis[2-aminophenylthio]butadiene (UO126) or combinations thereof.

### Claim 9 (depends on 1)

The method of treatment according to claim 1, wherein said pharmaceutical composition further comprises at least one Serine/threonine-protein kinase (B-raf) inhibitor selected from Vemurafenib (RG7204), (E)-2,3-Dihydro-5-[1-(2-hydroxyethyl)-3-(4-pyridinyl)-IH-pyrazol-4-yl]-IH-inden-1-one oxime (GDC-0879), N-[3-[(5-chloro-1H-pyrrolo[2,3 b]pyridin-3-yl)carbonyl]-2,4-difluorophenyl]-1 propanesulfonamide (PLX-4720), Sorafenib or combinations thereof.

### Claim 10 (depends on 1)

The method of treatment according to claim 1, wherein said pharmaceutical composition further comprises at least one mTOR inhibitor selected from rapamycin, metformin, 5-aminoimidazole-4-6-carboxamide ribunocleoside (AICAR) or combinations thereof.

### Claim 11 (depends on 1)

The method of treatment according to claim 1, wherein said pharmaceutical composition further comprises at least one compound selected from the group comprising 2-(2-Amino-3-methoxyphenyl)-4H-1-benzopyran-4-one (PD98059), 1,4-Diamino-2,3-dicyano-1,4-bis[2-aminophenylthio]butadiene (UO126), Vemurafenib (RG7204), E)-2,3-Dihydro-5-[1-(2-hydroxyethyl)-3-(4-pyridinyl)-IH-pyrazol-4-yl]-IH-inden-1-one oxime (GDC-0879), N-[3-[(5-chloro-1H-pyrrolo[2,3 b]pyridin-3-yl)carbonyl]-2,4-difluorophenyl]-1 propanesulfonamide (PLX-4720), Sorafenib, rapamycin, metformin, 5-aminoimidazole-4-6-carboxamide ribonucleoside (AICAR) or combinations thereof.