PI3K-δ inhibitor for use in treatment regimens
Inventors
PICKERING, Catherine • van der VEEN, Lars • LAHN, Michael • ZORRILLA, Rebeca • JOHNSON, Zoë
Assignees
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Publication Number
US-12605389-B2
Publication Date
2026-04-21
Expiration Date
Abstract
A compound or a pharmaceutically acceptable salt thereof for use in a method of treatment of a disease or condition in which signalling through the PI3Kδ pathway is pathologically implicated in patients, for example cancer and inflammatory or autoimmune diseases. The compound is provided at a specified dose and has been found to have a favourable safety profile in humans, in particular with regard to hepatotoxicity, diarrhoea/colitis, respiratory infections, and hematologic toxicities.
Core Innovation
The invention relates to a PI3Kδ pathway-inhibiting compound for use in treating disease. The compound is provided as a pharmaceutically acceptable salt, preferably anhydrous hemifumarate, corresponding to a compound of Formula I or a pharmaceutically acceptable salt thereof.
The disclosed approach is directed to subjects in need of treatment for cancer and specifically includes uveal melanoma and mesothelioma. The problem addressed is the need for treating uveal melanoma or mesothelioma and for treating inflammatory/autoimmune conditions using a PI3Kδ pathway-inhibiting compound.
The disclosure further characterizes the treatment through pharmacodynamic and safety findings. It reports dose-dependent reduction of CD63+ basophils as a PI3Kδ inhibition marker and a favorable human safety profile versus typical PI3Kδ class toxicities, including lack of clinically significant ALT/AST elevations, reduced/no grade 3 diarrhoea/colitis, reduced/no clinically significant neutropenia, and longer tolerability for continued treatment.
Claims Coverage
The relevant portion provides one independent claim covering treatment of uveal melanoma or mesothelioma using a compound of Formula I or a pharmaceutically acceptable salt. The independent claim is supported by two inventive features that specify the targeted diseases and the hemifumarate salt of Formula Ia.
Treating uveal melanoma or mesothelioma by administering a compound of Formula I
A method of treating uveal melanoma or mesothelioma in a subject in need thereof, comprising administering to the subject a compound of Formula I or a pharmaceutically acceptable salt thereof.
Using the hemifumarate salt of Formula Ia
The method is characterized by administering a compound that is provided as a hemifumarate salt according to Formula Ia.
In the provided claim set, the core inventive coverage is a method for treating uveal melanoma and/or mesothelioma by administering a PI3Kδ pathway-inhibiting compound of Formula I, including as a pharmaceutically acceptable salt, with particular emphasis on the hemifumarate salt of Formula Ia.
Stated Advantages
Favourable human safety profile versus typical PI3Kδ class toxicities.
Lack of clinically significant ALT/AST elevations.
Reduced/no grade 3 diarrhoea/colitis.
Reduced/no clinically significant neutropenia.
Longer tolerability for continued treatment.
Dose-dependent reduction of CD63+ basophils as a pharmacodynamic indication of PI3Kδ inhibition.
Documented Applications
Treating uveal melanoma.
Treating mesothelioma.
Treating inflammatory/autoimmune conditions.
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