IP Library Granted Patent US 10,736,857
Granted Patent B2
US 10,736,857 · App. 16/165,985 · Granted Aug 11, 2020

Treatment of pervasive developmental disorders with redox-active therapeutics

Inventors: Guy M. Miller (Monte Sereno, CA); Viktoria Kheifets (Mountain View, CA)
Assignee: PTC THERAPEUTICS, INC.
A61K31/122A23L33/10A23L33/40A61K31/05C07C39/08C07C50/02C07C50/06A23V2002/00
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Quick Facts
Patent No.
US 10,736,857
App. No.
16/165,985
Granted
Aug 11, 2020
Kind
B2
Abstract

Methods of treating or suppressing pervasive developmental disorders (PDDs) including; autistic disorder, Asperger's syndrome, childhood disintegrative disorder (CDD), Rett's disorder, and PDD-not otherwise specified (PDD-NOS) or attention deficit/hyperactivity disorder (ADHD) comprising administering to a subject in need thereof a therapeutically effective amount of one or more compounds as disclosed herein.

Claims (41)

1. A method of reducing one or more symptoms associated with, or of treating or suppressing a pervasive developmental disorder (PDD) or attention deficit/hyperactivity disorder (ADHD), in a patient in need thereof comprising administering a therapeutically effective amount or physiologically effective amount of one or more compounds selected from the group consisting of Formula I, Formula II, and mixtures thereof,

wherein,

the bonds indicated with a dashed line are all single bonds or are all double bonds;

R 1 , R 2 , and R 3 are independently selected from the group consisting of H, (C 1 -C 6 )alkyl, (C 1 -C 6 )haloalkyl, (C 1 -C 6 )alkoxy, halogen, and CN;

or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

2. The method according to claim 1 , comprising administering a therapeutically effective amount or physiologically effective amount of one or more compounds selected from the group consisting of Formula Ia, Formula IIa, and mixtures thereof,

or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

3. The method according to claim 2 , wherein the one or more compounds are selected from the group consisting of 2-(3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trienyl)-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione (Ib); Ib-R; Ib-S; 2-(3 -hydroxy-3,7,11,15-tetramethylhexadecyl)-3 ,5,6-trimethylcyclohexa-2,5-diene- 1,4-dione (Ic); Ic-RRR; Ic-SRR; Ic-RSR; Ic-RRS; Ic-SSR; Ic-SRS; Ic-RSS; and Ic-SSS; or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

4. The method according to claim 2 , wherein the method is for reducing one or more symptoms associated with, or for treating or suppressing a pervasive developmental disorder wherein the pervasive developmental disorder is selected from the group consisting of autism, Asperger's syndrome, childhood disintegrative disorder, and PDD-not otherwise specified.

5. The method according to claim 4 , wherein the one or more compounds are selected from the group consisting of 2-(3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trienyl)-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione (Ib); lb-R; Ib-S; 2-(3-hydroxy-3,7,11,15-tetramethylhexadecyl)-3 ,5,6-trimethylcyclohexa-2,5-diene-1,4-dione (Ic); Ic-RRR; Ic-SRR; Ic-RSR; Ic-RRS; Ic-SSR; Ic-SRS; Ic-RSS; and Ic-SSS; or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

6. The method according to claim 2 , wherein the method is for reducing one or more symptoms associated with, or for treating or suppressing attention deficit/hyperactivity disorder (ADHD).

7. The method according to claim 6 , wherein the one or more compounds are selected from the group consisting of 2-(3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trienyl)-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione (Ib); Ib-R; Ib-S; 2-(3-hydroxy-3,7,11,15-tetramethylhexadecyl)-3 ,5,6-trimethylcyclohexa-2,5-diene-1,4-dione (Ic); Ic-RRR; Ic-SRR; Ic-RSR; Ic-RRS; Ic-SSR; Ic-SRS; Ic-RSS; and Ic-SSS; or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

8. The method of claim 1 , wherein the one or more compounds is selected from the group consisting of Formula I, Formula II, and mixtures thereof,

wherein,

the bonds indicated with a dashed line are all single bonds or are all double bonds;

R 1 , R 2 , and R 3 are independently selected from the group consisting of H, (C 1 -C 6 )alkyl, (C 1 -C 6 )haloalkyl, (C 1 -C 6 )alkoxy, halogen, and CN;

or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof;

and wherein the one or more compounds are administered as a medical food, functional food, food supplement, or dietary supplement comprising at least one of the group consisting of a physiologically or nutritionally acceptable carrier, adjuvant, excipient, buffer, and diluent.

9. The method according to claim 1 , wherein the one or more compounds is 2-(3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trienyl)-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione (lb), or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

10. The method according to claim 4 , wherein the one or more compounds is 2-(3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trienyl)-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione (lb), or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

11. A method of reducing one or more symptoms associated with, or of treating or suppressing Rett's disorder or Rett syndrome, in a patient in need thereof comprising administering a therapeutically effective amount or physiologically effective amount of one or more compounds selected from the group consisting of Formula I, Formula II, and mixtures thereof,

wherein,

the bonds indicated with a dashed line are all single bonds or are all double bonds;

R 1 , R 2 , and R 3 are independently selected from the group consisting of H, (C 1 -C 6 )alkyl, (C 1 -C 6 )haloalkyl, (C 1 -C 6 )alkoxy, halogen, and CN;

or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

12. The method according to claim 11 , comprising administering a therapeutically effective amount or physiologically effective amount of one or more compounds selected from the group consisting of Formula Ia, Formula IIa, and mixtures thereof,

or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

13. The method according to claim 11 , comprising administering a therapeutically effective amount or physiologically effective amount of one or more compounds selected from the group consisting of Formula (Ia) and mixtures thereof,

or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

14. The method according to claim 11 , comprising administering a therapeutically effective amount or physiologically effective amount of one or more compounds selected from the group consisting of Formula (IIa) and mixtures thereof,

or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

15. The method according to claim 11 wherein the one or more compounds is 2-(3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trienyl)-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione (lb), or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

16. The method of claim 11 , wherein the one of more compounds is 2-((3R,6E,10E)-3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10-dienyl)-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione (Ib-R), or a solvate, or a hydrate thereof.

17. A method of reducing one or more symptoms associated with, or of treating or suppressing autistic spectrum disorder, in a patient in need thereof comprising administering a therapeutically effective amount or physiologically effective amount of one or more compounds selected from the group consisting of Formula I, Formula II, and mixtures thereof,

wherein,

the bonds indicated with a dashed line are all single bonds or are all double bonds;

R 1 , R 2 , and R 3 are independently selected from the group consisting of H, (C 1 -C 6 )alkyl, (C 1 -C 6 )haloalkyl, (C 1 -C 6 )alkoxy, halogen, and CN;

or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

18. The method according to claim 17 , wherein the autistic spectrum disorder is autism.

19. The method according to claim 17 wherein the one or more compounds is 2-(3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trienyl)-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione (lb), or a stereoisomer, a mixture of stereoisomers, a solvate, or a hydrate thereof.

20. The method of claim 17 , wherein the one of more compounds is 2-((3R, 6E,10E)-3-hydroxy-3 ,7, 11,15-tetramethylhexadeca-6,10-dienyl)-3 ,5,6-trimethylcyclohexa-2,5-diene-1,4-dione (Ib-R), or a solvate, or a hydrate thereof.

Assignments (3)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Nov 15, 2019
From: BIOELECTRON TECHNOLOGY CORPORATION
To: PTC THERAPEUTICS, INC.
Reel/Frame 051041/0478 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 2, 2019
From: MILLER, GUY M.; KHEIFETS, VIKTORIA
To: EDISON PHARMACEUTICALS, INC.
Reel/Frame 049947/0690 →
CHANGE OF NAME Recorded Aug 2, 2019
From: EDISON PHARMACEUTICALS, INC.
To: BIOELECTRON TECHNOLOGY CORPORATION
Reel/Frame 049951/0401 →