US 11,964,022 B2
Small molecule binders of the oncogenic fusion transcription factor PAX3-FOXO1
Angela Nicole Koehler, Belmont, MA (US); Shelby Doyle, Cambridge, MA (US); Becky Leifer, Malden, MA (US); Madeleine Henley, Brighton, MA (US); Beat W. Schaefer, Zürich (CH); and Marco Wachtel, Zürich (CH)
Assigned to Massachusetts Institute of Technology, Cambridge, MA (US); and Universität Zürich, Zürich (CH)
Filed by Massachusetts Institute of Technology, Cambridge, MA (US); and Universität Zürich, Zürich (CH)
Filed on Sep. 16, 2022, as Appl. No. 17/946,898.
Claims priority of provisional application 63/280,091, filed on Nov. 16, 2021.
Prior Publication US 2023/0149552 A1, May 18, 2023
Int. Cl. A61K 47/55 (2017.01)
CPC A61K 47/55 (2017.08) 5 Claims
 
1. A compound of formula (I):
P—L—U (I)
or a pharmaceutically acceptable salt thereof, wherein
P is a FOXO1 fusion protein binding moiety and is:

OG Complex Work Unit Chemistry

OG Complex Work Unit Chemistry

OG Complex Work Unit Chemistry
L is a bivalent linker and is

OG Complex Work Unit Chemistry
and
U is an ubiquitin ligase binding moiety and is Cereblon (CRBN) E3 ligase moiety having the structure:

OG Complex Work Unit Chemistry
((2-(2,6-dioxopiperidin-3-yl)-1,3-dioxoisoindolin-4-yl) amino).