US 12,440,500 B2
Phytoecdysones and the derivatives thereof for use in the treatment of neuromuscular diseases
Mathilde Latil, Paris (FR); Pierre Dilda, Paris (FR); René Lafont, Paris (FR); and Stanislas Veillet, Savigny sur Orge (FR)
Assigned to BIOPHYTIS, Paris (FR); and SORBONNE UNIVERSITE, Paris (FR)
Appl. No. 17/439,681
Filed by BIOPHYTIS, Paris (FR); and SORBONNE UNIVERSITE, Paris (FR)
PCT Filed Mar. 12, 2020, PCT No. PCT/EP2020/056590
§ 371(c)(1), (2) Date Sep. 15, 2021,
PCT Pub. No. WO2020/187678, PCT Pub. Date Sep. 24, 2020.
Claims priority of application No. 1902726 (FR), filed on Mar. 15, 2019.
Prior Publication US 2022/0160732 A1, May 26, 2022
Int. Cl. A61K 31/575 (2006.01); A61K 31/58 (2006.01); A61P 21/00 (2006.01)
CPC A61K 31/575 (2013.01) [A61K 31/58 (2013.01); A61P 21/00 (2018.01)] 18 Claims
 
1. A method of treatment of a specific disorder of the motor neurons in mammals suffering from a neuromuscular disease including an alteration of the muscular function due to the specific disorder of the motor neurons, wherein said neuromuscular disease is spinal muscular atrophy (SMA), said method comprising the step of administering to a subject in need thereof an effective dose of a composition comprising at least 20-hydroxyecdysone and/or at least one semi-synthetic derivative of 20-hydroxyecdysone of the general formula (I):

OG Complex Work Unit Chemistry
wherein:
V—U is a carbon-carbon single bond and Y is a hydroxyl group or a hydrogen, or V—U is an ethylenic C═C bond;
X is an oxygen;
Q is a carbonyl group;
R1 is chosen from:
a (C1-C6)W(C1-C6) group;
a (C1-C6)W(C1-C6)W(C1-C6) group;
a (C1-C6)W(C1-C6)CO2(C1-C6) group;
a (C1-C6)A group, A representing a heterocycle optionally substituted with a group of the type OH, OMe, (C1-C6), N(C1-C6), CO2(C1-C6);
a CH2Br group;
W being a heteroatom chosen from N, O and S.