IP Library Granted Patent US 10,478,446
Granted Patent B2
US 10,478,446 · App. 14/413,280 · Granted Nov 19, 2019

Compounds for use in polycystic kidney disease

Inventors: Alessandra Boletta (Milan, IT); Marco Chiaravalli (Milan, IT); Isaline Severine Marie-Josephe Rowe (Milan, IT)
Assignees: FONDAZIONE CENTRO SAN RAFFAELE OFFICE OF BIOTECHNOLOGY TRANSFER; FONDAZIONE TELETHON
A61K31/7008A61K31/155A61K31/352A61K31/436A61K31/437A61K31/44A61K31/7004A61K45/06
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Quick Facts
Patent No.
US 10,478,446
App. No.
14/413,280
Granted
Nov 19, 2019
Kind
B2
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 (11)

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).

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

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

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

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

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

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

8. 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.

9. 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.

10. 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.

11. 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.

Assignments (2)
CHANGE OF NAME Recorded Nov 28, 2023
From: FONDAZIONE TELETHON
To: FONDAZIONE TELETHON ETS
Reel/Frame 065694/0135 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded May 7, 2015
From: BOLETTA, ALESSANDRA; CHIARAVALLI, MARCO; ROWE, ISALINE SEVERINE MARIE-JOSEPHE
To: FONDAZIONE CENTRO SAN RAFFAELE OFFICE OF BIOTECHNOLOGY TRANSFER; FONDAZIONE TELETHON
Reel/Frame 035589/0988 →
Continuity (2)
Provisional Application 61667615 · Jul 3, 2012
Related Publication 20150297620A1 · Oct 22, 2015