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3-(5-methoxy-1-oxoisoindoline-2-yl) piperidine-2, 6-diketone derivative and application thereof
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A C1-C6, alkoxy technology, applied in the field of treatment of hereditary blood diseases, can solve the problems of low response rate and genotoxicity
Pending Publication Date: 2022-07-29
NOVARTIS AG
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Problems solved by technology
Hydroxyurea is the current standard of care for SCD and is thought to provide benefit via induction of HbF, but is genotoxic, causes dose-limiting neutropenia, and has a response rate of less than 40%
Method used
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Embodiment 1
[0175] Embodiment 1. A compound of formula (I') or a pharmaceutically acceptable salt, hydrate, solvate, prodrug, stereoisomer, or tautomer thereof,
[0176]
[0177] in:
[0178] Y is selected from O, CH 2 , CF 2 , and CHF;
[0179] z is an integer from 0 to 2;
[0180] R X1 and R X2 each independently selected from hydrogen and C 1 -C 6 alkyl;
[0181] R Y1 and R Y2 each independently selected from hydrogen and C 1 -C 6 alkyl;
[0184] R Z1 and R Z2 1 of the and R Y1 and R Y2 1 of them together form C 1 -C 2 an alkylene bridging group, and R Z1 and R Z2 and R Y1 and R Y2 The other one is hydrogen;
[0185] R 1 selected from hydrogen and C 1 -C 6 alkyl;
[0186] R 2 selected from hydrogen, -C(=O)-R 3 , C 3 -C 8 Cycloalkyl, C 1 -C 6 haloalkyl, and C 1 -C 10 Alkyl, wherein the alkyl is substituted with 0-1 substituents independently selected from: C 6 -C 10 Aryl, 5- to 10-membered hetero...
Embodiment 2
[0208] Embodiment 2. The compound of formula (I') according to claim 1, or a pharmaceutically acceptable salt, hydrate, solvate, prodrug, stereoisomer, or tautomer thereof, wherein : z is 1; and R Z1 and R Z2 1 of the and R Y1 and R Y2 1 of them together form C 1 -C 2 an alkylene bridging group, and R Z1 and R Z2 and R Y1 and R Y2 The other one is all hydrogen.
Embodiment 3
[0209] Embodiment 3. The compound of formula (I') according to claim 1 or 2 or a pharmaceutically acceptable salt, hydrate, solvate, prodrug, stereoisomer, or tautomer thereof , where: z is 1; and R Z1 and R Z2 1 of the and R Y1 and R Y2 1 of them together form C 1 an alkylene bridging group, and R Z1 and R Z2 and R Y1 and R Y2 The other one is all hydrogen.
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Abstract
The present disclosure relates to compounds of Formula (I ') and pharmaceutical compositions and their use in reducing the expression levels of Wide Interval Zinc Finger Motif (WIZ) or inducing the expression of fetal hemoglobin (HbF) and in the treatment of hereditary hematological disorders (e.g., hemoglobinopathy, e.g., beta-hemoglobinopathy) such as sickle celldisease and beta-thalassemia.
Description
[0001] priority claim [0002] This application claims the benefit of priority to US Provisional Application No. 62 / 950,048, filed on December 18, 2019, the disclosure of which is incorporated herein by reference in its entirety. technical field [0003] The present disclosure relates to 3-(5-methoxy-1-oxoisoindolin-2-yl)piperidine-2,6-dione compounds and pharmaceutical compositions and their reduction in broad spaced zinc finger motifs (WIZ) protein expression levels, and / or induced fetal hemoglobin (HbF) protein expression levels, and in the treatment of inherited blood disorders (hemoglobinopathies such as beta-hemoglobinopathies) such as sickle celldisease and beta-thalassemia use. Background technique [0004] Sickle celldisease (SCD) is a group of serious inherited blood disorders that cause red blood cells to twist into a sickle shape. These cells can cause blockages in blood flow, which can lead to severe pain, organ damage and premature death. Beta thalassemia...
Claims
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