IP Library › Granted Patent US 11,065,230
Granted Patent B2
US 11,065,230 · App. 16/330,956 · Granted Jul 20, 2021

Methods and pharmaceutical compositions for the treatment of systemic mastocytosis

Inventors: Olivier Hermine (Paris, FR); Flavia Guillem (Paris, FR); Gandhi Damaj (Paris, FR); Sophia Ladraa (Paris, FR)
Assignees: INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE); FONDATION IMAGINE; UNIVERSITE PARIS DESCARTES; ASSISTANCE PUBLIQUE—HOPITAUX DE PARIS (APHP); CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS); UNIVERSITE DE CAEN NORMANDIE; CENTRE HOSPITALIER REGIONAL UNIVERSITAIRE DE CAEN
A61K31/4196A61K31/397A61K31/4245A61K31/506A61P37/00A61K31/497A61K31/7105A61P35/04C12N2310/14C12N2310/531
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Quick Facts
Patent No.
US 11,065,230
App. No.
16/330,956
Granted
Jul 20, 2021
Kind
B2
Abstract

The present invention relates to methods and pharmaceutical compositions for the treatment of systemic mastocytosis. The inventors showed the effect of KPT-251 treatment on SCF-dependent human Mast cell (MC) line without KIT mutation (WT ROSA) and on two factor-independent MC lines with KIT mutations : ROSA Δ 417-419 insY and ROSA D816V. KPT is a Selective Inhibitor of Nuclear Export (SINE) that specifically inhibits the activity of the exportin-1 (XPO1). KPT-251 treatment induces minimal toxicity in non-cancerous hematopoietic cells both in vitro and in vivo. In particular, the present invention relates a method of treating systemic mastocytosis in patient in need thereof comprising administering to the patient a therapeutically effective amount of a XPO1 inhibitor.

Claims (4)

1. A method of treating systemic mastocytosis in a patient in need thereof comprising administering to the patient a therapeutically effective amount of a XPO1 inhibitor, wherein the XPO1 inhibitor is KPT-330, KPT-276, or KPT-251.

2. The method of claim 1 wherein the XPO1 inhibitor is KPT-330.

3. The method of claim 1 wherein the XPO1 inhibitor is KPT-276.

4. The method of claim 1 wherein the XPO1 inhibitor is KPT-251.

Assignments (3)
CHANGE OF NAME Recorded May 12, 2022
From: UNIVERSITE DE PARIS
To: UNIVERSITÉ PARIS CITÉ
Reel/Frame 059988/0388 →
MERGER Recorded May 12, 2022
From: UNIVERSITE PARIS DESCARTES
To: UNIVERSITE DE PARIS
Reel/Frame 060044/0856 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 24, 2021
From: HERMINE, OLIVIER; GUILLEM, FLAVIA; DAMAJ, GANDHI; LADRAA, SOPHIA
To: INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE); FONDATION IMAGINE; UNIVERSITE PARIS DESCARTES; ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (APHP); CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS); UNIVERSITE DE CAEN NORMANDIE; CENTRE HOSPITALIER REGIONAL UNIVERSITAIRE DE CAEN
Reel/Frame 055700/0488 →
Priority Claims (1)
EP 16306181 · Sep 16, 2016 · regional
Continuity (1)
Related Publication 20190247416A1 · Aug 15, 2019