IP Library Granted Patent US 10,751,302
Granted Patent B2
US 10,751,302 · App. 16/354,070 · Granted Aug 25, 2020

Polymorphic and amorphous 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)
Assignee: PTC Therapeutics, Inc.
A61K31/122A61K31/164C07B57/00C07C215/08C07C215/28C07C215/30C07C231/12C07C235/78C07B2200/13C07C2601/16
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Quick Facts
Patent No.
US 10,751,302
App. No.
16/354,070
Granted
Aug 25, 2020
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 polymorph of an anhydrateor a hydrate of (R)-2-hydroxy-2-methyl-4-(2,4,5-trimethyl-3,6-dioxocyclohexa-1,4-dienyl)butanamide, wherein the polymorph is selected from Form III (hydrate), or Form II (anhydrate); 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: 14.02, 15.23, and 21.10; and 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, 11.33, and 19.33.

2. The polymorph of claim 1 , wherein the polymorph is Form III, wherein a powder X-ray diffraction pattern for the polymorph comprises characteristic peaks at least at the following angular positions, wherein the angular positions may vary by ±0.2: 14.02, 15.23, and 21.10.

3. The polymorph of claim 1 , wherein the polymorph is Form III, wherein a powder X-ray diffraction pattern for the polymorph 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, 21.10, and 22.69.

4. The polymorph of claim 1 , wherein the polymorph is Form III, wherein the polymorph has a powder x-ray diffraction pattern substantially as shown in a) orb) of FIG. 20 .

5. A composition comprising the polymorph of claim 2 , wherein at least about 95% by mole of the composition is the polymorph Form III, exclusive of any solvents, carriers or excipients.

6. The polymorph of claim 1 , wherein the polymorph is Form III, having a differential scanning calorimetry (DSC) thermogram substantially as shown in FIG. 22 .

7. The polymorph of claim 6 , wherein the DSC thermogram comprises two endothermic peaks at about 72.0 and 150.7° C.

8. The polymorph of claim 1 , wherein the polymorph is Form III, having a thermogravimetric analysis (TGA) thermogram substantially as shown in FIG. 23 .

9. The polymorph of claim 1 , wherein the polymorph is Form III, having a 1 H NMR spectrum substantially as shown in FIG. 42 .

10. A pharmaceutical composition comprising the polymorph of claim 2 , and a pharmaceutically acceptable carrier.

11. The polymorph of claim 1 , wherein the polymorph is Form II, wherein a powder X-ray diffraction pattern for the polymorph comprises characteristic peaks at least at the following angular positions, wherein the angular positions may vary by ±0.2: 9.63, 11.33, and 19.33.

12. The polymorph of claim 1 , wherein the polymorph is Form II, wherein a powder X-ray diffraction pattern for the polymorph 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, 13.47, and 19.33.

13. The polymorph of claim 1 , wherein the polymorph is Form II, wherein the polymorph has a powder x-ray diffraction pattern substantially as shown in a) of FIG. 15 .

14. A composition comprising the polymorph of claim 11 , wherein at least about 95% by mole of the composition is the polymorph Form II, exclusive of any solvents, carriers or excipients.

15. The polymorph of claim 1 , wherein the polymorph is Form II, having a differential scanning calorimetry (DSC) thermogram substantially as shown in FIG. 17 .

16. The polymorph of claim 15 , wherein the DSC thermogram comprises two endothermic peaks at about 133.9 and 151.3° C.

17. The polymorph of claim 1 , wherein the polymorph is Form II, having a thermogravimetric analysis (TGA) thermogram substantially as shown in FIG. 18 .

18. The polymorph of claim 1 , wherein the polymorph is Form II, having a 1 H NMR spectrum substantially as shown in FIG. 44 .

19. A pharmaceutical composition comprising the polymorph of claim 11 , and a pharmaceutically acceptable carrier.

20. A method of treating or suppressing an oxidative stress disorder, comprising administering to an individual in need thereof a therapeutically effective amount of the polymorph of claim 1 , wherein the oxidative stress disorder is 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 POLG Mutation; 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.

Assignments (5)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Nov 15, 2019
From: BIOELECTRON TECHNOLOGY CORPORATION
To: PTC THERAPEUTICS, INC.
Reel/Frame 051041/0478 →
CHANGE OF NAME Recorded Aug 22, 2019
From: EDISON PHARMACEUTICALS, INC.
To: BIOELECTRON TECHNOLOGY CORPORATION
Reel/Frame 050133/0037 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 20, 2019
From: CORNELL, CHRISTOPHER R.; GIANNOUSIS, PETER; MOLLARD, PAUL; WESSON, KIERON E.
To: EDISON PHARMACEUTICALS, INC.
Reel/Frame 050109/0111 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 19, 2019
From: JOHNSON MATTHEY PHARMACEUTICAL MATERIALS, INC., D/B/A JOHNSON MATTHEY PHARMA SERVICES
To: EDISON PHARMACEUTICALS, INC.
Reel/Frame 050092/0489 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 16, 2019
From: MIRMEHRABI, MAHMOUD; SUCHIT, SHAZAD
To: JOHNSON MATTHEY PHARMACEUTICAL MATERIALS, INC., D/B/A JOHNSON MATTHEY PHARMA SERVICES
Reel/Frame 050080/0890 →