BENZYLIDENEGUANIDINE DERIVATIVES AND THERAPEUTIC USE THEREOF FOR TREATMENT OF PROTEIN MISFOLDING RELATED DISEASES Russian patent published in 2018 - IPC C07C281/18 C07D213/61 C07D253/75 A61K31/155 A61K31/165 A61K31/216 A61K31/44 A61K31/53 A61P25/00 

FIELD: chemistry.

SUBSTANCE: invention relates to the use of compounds of formula (I) or pharmaceutically acceptable salts or tautomeric forms thereof for the treatment of disorders associated with stress caused by misfolding of proteins, in particular accumulation of misfolded proteins. Disorders are selected from Charcot-Marie-Tooth neuropathy; severe Dejerine-Sott syndrome; retinal diseases, preferably retinitis pigmentosa, retinal ciliopathy, macular degeneration or diabetic retinopathy or amyotrophic lateral sclerosis (ALS). In formula (I), R₁ is alkyl, Cl, F or Br; R₂ is H or F; R₃ is selected from H and alkyl; R₄ is selected from H, CO alkyl and COOalkyl; R₅ is H; or R₄ and R₅ are joined to form an oxodihydro triazine group which is optionally substituted with one or more alkyl groups R₁₀; X and Z, independently of one another, are CR₁₁, Y is selected from CR₁₁ and N; R₁₁ is H or F, where alkyl is C_1-20-alkyl. (I).

EFFECT: invention also relates to different variants of compounds of formula (I) and a pharmaceutical composition for use in the treatment of disorders associated with stress caused by misfolding of proteins, in particular accumulation of misfolded proteins.

14 cl, 6 dwg, 2 tbl, 16 ex