IP Library Granted Patent US 11,938,101
Granted Patent B2
US 11,938,101 · App. 17/692,133 · Granted Mar 26, 2024

Polymorphic forms of (R)-2-hydroxy-2-methyl-4-(2,4,5-trimethyl-3,6-dioxocyclohexa-1,4-dienyl)butanamide

Inventors: Paul Mollard (Mountain View, CA); Peter Giannousis (Mountain View, CA); Shazad Suchit (Devens, MA); Mahmoud Mirmehrabi (Halifax, CA); Christopher R. Cornell (Mountain View, CA); Kieron E. Wesson (Mountain View, CA)
Assignee: PTC THERAPEUTICS, INC.
A61K31/122A61K31/164C07B57/00C07C215/08C07C215/28C07C215/30C07C231/12C07C235/78C07B2200/13C07C2601/16
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Quick Facts
Patent No.
US 11,938,101
App. No.
17/692,133
Granted
Mar 26, 2024
Kind
B2
Abstract

Disclosed herein are polymorphic and amorphous forms of anhydrate, hydrate, and solvates of (R)-2-hydroxy-2-methyl-4-(2,4,5-trimethyl-3,6-dioxocyclohexa-1,4-dienyl)butanamide and methods of using such compositions for treating or suppressing oxidative stress disorders, including mitochondrial disorders, impaired energy processing disorders, neurodegenerative diseases and diseases of aging. Further disclosed are methods of making such polymorphic and amorphous forms.

Claims (20)

1. A composition comprising a polymorph of an anhydrate, a hydrate, or a solvate of (R)-2-hydroxy-2-methyl-4-(2,4,5-trimethyl-3,6-dioxocyclohexa-1,4-dienyl)butanamide, wherein the composition comprises a polymorph Form I and at least one polymorph selected from the group consisting of Form II, Form III, Form IV, Form V, and Form VI;

wherein a powder X-ray diffraction pattern for polymorph Form I comprises characteristic peaks at least at the following angular positions, wherein the angular positions may vary by ±0.2: 12.06, 15.33, 17.03, and 17.26;

wherein a powder X-ray diffraction pattern for polymorph Form V comprises characteristic peaks at least at the following angular positions, wherein the angular positions may vary by ±0.2: 9.61, 11.49, 12.93, and 15.45;

wherein a powder X-ray diffraction pattern for polymorph Form III comprises characteristic peaks at least at the following angular positions, wherein the angular positions may vary by ±0.2: 9.16, 14.02, 15.23, and 21.10;

wherein a powder X-ray diffraction pattern for polymorph Form II comprises characteristic peaks at least at the following angular positions, wherein the angular positions may vary by ±0.2: 9.63, 10.85, 11.33, and 19.33;

wherein a powder X-ray diffraction pattern for polymorph Form IV comprises characteristic peaks at least at the following angular positions, wherein the angular positions may vary by ±0.2: 4.31, 8.76, 12.97, and 13.20; and

wherein a powder X-ray diffraction pattern for polymorph Form VI comprises characteristic peaks at least at the following angular positions, wherein the angular positions may vary by ±0.2: 6.27, 9.91, 12.94, and 15.71.

2. The composition of claim 1 , wherein the composition comprises polymorph Form I and Form II.

3. The composition of claim 1 , wherein the composition comprises polymorph Form I and Form III.

4. The composition of claim 1 , wherein the composition comprises polymorph Form I and Form IV.

5. The composition of claim 1 , wherein the composition comprises polymorph Form I and Form V.

6. The composition of claim 1 , wherein the composition comprises polymorph Form I and Form VI.

7. A pharmaceutical composition comprising the composition of claim 1 and a pharmaceutically acceptable carrier.

8. A method of treating or suppressing an oxidative stress disorder, modulating one or more energy biomarkers, normalizing one or more energy biomarkers, or enhancing one or more energy biomarkers, comprising

administering to an individual in need thereof a therapeutically effective amount or effective amount of the composition of claim 1 .

9. The method of claim 8 , wherein the method is a method of treating or suppressing an oxidative stress disorder selected from the group consisting of: a mitochondrial disorder; an inherited mitochondrial disease; Alpers Disease; Barth syndrome; a Beta-oxidation Defect; Carnitine-Acyl-Carnitine Deficiency; Carnitine Deficiency; a Creatine Deficiency Syndrome; Co-Enzyme Q10 Deficiency; Complex I Deficiency; Complex II Deficiency; Complex III Deficiency; Complex IV Deficiency; Complex V Deficiency; COX Deficiency; chronic progressive external ophthalmoplegia (CPEO); CPT I Deficiency; CPT II Deficiency; Friedreich's Ataxia (FA); Glutaric Aciduria Type II; Kearns-Sayre Syndrome (KSS); Lactic Acidosis; Long-Chain Acyl-CoA Dehydrogenase Deficiency (LCAD); LCHAD; Leigh Syndrome; Leigh-like Syndrome; Leber's Hereditary Optic Neuropathy (LHON); Lethal Infantile Cardiomyopathy (LIC); Luft Disease; Multiple Acyl-CoA Dehydrogenase Deficiency (MAD); Medium-Chain Acyl-CoA Dehydrogenase Deficiency (MCAD); Mitochondrial Myopathy, Encephalopathy, Lactacidosis, Stroke (MELAS); Myoclonic Epilepsy with Ragged Red Fibers (MERRF); Mitochondrial Recessive Ataxia Syndrome (MIRAS); Mitochondrial Cytopathy, Mitochondrial DNA Depletion; Mitochondrial Encephalopathy; Mitochondrial Myopathy; Myoneurogastrointestinal Disorder and Encephalopathy (MNGIE); Neuropathy, Ataxia, and Retinitis Pigmentosa (NARP); Pearson Syndrome; Pyruvate Carboxylase Deficiency; Pyruvate Dehydrogenase Deficiency; a Respiratory Chain Disorder; Short-Chain Acyl-CoA Dehydrogenase Deficiency (SCAD); SCHAD; Very Long-Chain Acyl-CoA Dehydrogenase Deficiency (VLCAD); a myopathy; cardiomyopathy; encephalomyopathy; a neurodegenerative disease; Parkinson's disease; Alzheimer's disease; amyotrophic lateral sclerosis (ALS); a motor neuron disease; a neurological disease; epilepsy; an age-associated disease; macular degeneration; diabetes; metabolic syndrome; cancer; brain cancer; a genetic disease; Huntington's Disease; a mood disorder; schizophrenia; bipolar disorder; a pervasive developmental disorder; autistic disorder; Asperger's syndrome; childhood disintegrative disorder (CDD); Rett's disorder; PDD-not otherwise specified (PDD-NOS); a cerebrovascular accident; stroke; a vision impairment; optic neuropathy; dominant inherited juvenile optic atrophy; optic neuropathy caused by a toxic agent; glaucoma; Stargardt's macular dystrophy; diabetic retinopathy; diabetic maculopathy; retinopathy of prematurity; ischemic reperfusion-related retinal injury; oxygen poisoning; a haemoglobinopathy; thalassemia; sickle cell anemia; seizures; ischemia; renal tubular acidosis; attention deficit/hyperactivity disorder (ADHD); a neurodegenerative disorder resulting in hearing or balance impairment; Dominant Optic Atrophy (DOA); Maternally inherited diabetes and deafness (MIDD); chronic fatigue; contrast-induced kidney damage; contrast-induced retinopathy damage; Abetalipoproteinemia; retinitis pigmentosum; Wolfram's disease; Tourette syndrome; cobalamin c defect; methylmalonic aciduria; glioblastoma; Down's syndrome; acute tubular necrosis; a muscular dystrophy; a leukodystrophy; Progressive Supranuclear Palsy; spinal muscular atrophy; hearing loss; noise induced hearing loss; traumatic brain injury; Juvenile Huntington's Disease; Multiple Sclerosis; NGLY1; Multiple System Atrophy; Adrenoleukodystrophy; and Adrenomyeloneuropathy.

10. The method of claim 9 , wherein the oxidative stress disorder is cancer.

11. The method of claim 9 , wherein the oxidative stress disorder is an age-associated disease.

12. The method of claim 9 , wherein the oxidative stress disorder is a muscular dystrophy.

13. The method of claim 9 , wherein the oxidative stress disorder is amyotrophic lateral sclerosis (ALS).

Assignments (5)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 11, 2022
From: CORNELL, CHRISTOPHER R.; GIANNOUSIS, PETER; MOLLARD, PAUL; WESSON, KIERON E.
To: EDISON PHARMACEUTIALS, INC.
Reel/Frame 059245/0578 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 11, 2022
From: MIRMEHRABI, MAHMOUND; SUCHIT, SHAZAD
To: JOHNSON MATTHEY PHARMACEUTICAL MATERIALS, INC.
Reel/Frame 059245/0668 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 11, 2022
From: JOHNSON MATTHEY PHARMACEUTICAL MATERIALS, INC.
To: EDISON PHARMACEUTIALS, INC.
Reel/Frame 059245/0764 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 11, 2022
From: BIOELECTRON TECHNOLOGY CORPORATION
To: PTC THERAPEUTICS, INC.
Reel/Frame 059361/0927 →
CHANGE OF NAME Recorded Mar 11, 2022
From: EDISON PHARMACEUTIALS, INC.
To: BIOELECTRON TECHNOLOGY CORPORATION
Reel/Frame 059362/0747 →
Continuity (6)
Continuation 16919044 · Jul 1, 2020
Continuation 16354070 · Mar 14, 2019
Continuation 15536603
Provisional Application 62133276 · Mar 13, 2015
Provisional Application 62092743 · Dec 16, 2014
Related Publication 20220265578A1 · Aug 25, 2022