Described herein are potassium channel opener compounds, compositions, and related uses and preparations thereof for the treatment of a disease or disorder, such as congenital hyperinsulinism.
C07D 471/02 - Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups in which the condensed system contains two hetero rings
The present disclosure relates to the use of a compound of chemical Formula 1 or a pharmaceutically acceptable salt thereof for the purpose of preventing, improving, or treating a genetic obesity disease associated with the melanocortin-4 receptor (MC4R) pathway, particularly a genetic obesity disease associated with SH2B1 deficiency or a LEPR deficiency.
A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
C07D 207/02 - Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
A61K 31/535 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and at least one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
The disclosure is related to a method of treating a disorder, such as Prader Willi Syndrome (PWS), obesity or hyperphagia, in a subject using a melanocortin-4 receptor (MC4R) agonist. Also described is method of treating a subject having a deficiency in the pro-opiomelanocortin (POMC)-MC4R pathway, such as a POMC-null or a PCSK-null subject, using a MC4R agonist.
This disclosure provides, at least in part, a pharmaceutical product comprising: setmelanotide (also known as RM493); setmelanotide as sole active pharmaceutical ingredient formulated for injection; setmelanotide as the sole active ingredient; setmelanotide as the active pharmaceutical ingredient; setmelanotide as the active pharmaceutical ingredient for injection; or another pharmaceutical composition that has its primary mechanism of action at the MC4 receptor as an agonist (referred to herein as an MC4RAP), e.g., a lipid excipient, and/or a pharmaceutically acceptable carrier. The pharmaceutical product described herein provides a sustained release of setmelanotide or another pharmaceutical composition, which may result in a more desirable pharmacokinetic and pharmacodynamic profile upon administration.
The present invention relates to polypeptide compounds that are modulators (e.g., agonists and antagonists) of the melanocortin-4 receptor (MC4R) and pharmaceutical compositions comprising same. The compounds described herein are polypeptide of the following structural Formula (I):
The present invention relates to polypeptide compounds that are modulators (e.g., agonists and antagonists) of the melanocortin-4 receptor (MC4R) and pharmaceutical compositions comprising same. The compounds described herein are polypeptide of the following structural Formula (I):
The present invention relates to polypeptide compounds that are modulators (e.g., agonists and antagonists) of the melanocortin-4 receptor (MC4R) and pharmaceutical compositions comprising same. The compounds described herein are polypeptide of the following structural Formula (I):
or a pharmaceutically acceptable salt thereof. Values and preferred values of the variables in structural Formula (I) are described herein.
The disclosure relates to a method of treating a disease or disorder in a subject using a combination of: (i) a potassium channel activator (e.g., a SURx/Kirx channel activator compound) or a pharmaceutically acceptable salt thereof; and (ii) a melanocortin-4 receptor (MC4R) agonist.
A61K 31/513 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
A61K 31/549 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and at least one sulfur as the ring hetero atoms, e.g. sulthiame having two or more nitrogen atoms in the same ring, e.g. hydrochlorothiazide
45 - Legal and security services; personal services for individuals.
16 - Paper, cardboard and goods made from these materials
41 - Education, entertainment, sporting and cultural services
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Providing supportive personal stories in the field of obesity and obesity disorders via a website Printed pamphlets in the field of obesity and obesity disorders; Printed brochures about obesity and obesity disorders; Printed leaflets about obesity and obesity disorders; Printed informational flyers featuring obesity and obesity disorders; Printed informational sheets about obesity and obesity disorders On-line electronic newsletters delivered by e-mail in the field of obesity and obesity disorders Providing medical information via a website
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Pharmaceuticals and medicines for the treatment of metabolic
diseases and disorders, including obesity. Research and product development services in the fields of
metabolic diseases and disorders, including obesity.
Institute National De La Santé Et De La Recherche Mêdicale (France)
UNIVERSITÉ DE STRASBOURG (France)
Inventor
Van Der Ploeg, Leonardus H.T.
Garfield, Alastair
Marion, Vincent
Abstract
The disclosure is related to a method of treating chronic kidney disease in a subject with a melanocortin-4 receptor (MC4R) agonist, e.g., a compound of any one of Formulas (I), (II), (III), (IV), (V), (VI), (VII), (VIII), (X), or (XI), or a pharmaceutically acceptable salt thereof (e.g., as described herein).
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Pharmaceuticals and medicines for the treatment of metabolic
diseases and disorders, namely, obesity. Research and product development services in the fields of
metabolic diseases and disorders, namely, obesity.
16.
METHOD OF TREATING MELANOCORTIN-4 RECEPTOR-ASSOCIATED DISORDERS IN HETEROZYGOUS CARRIERS
A method of treating a disorder in a subject. The method comprises administering to said subject an effective amount of an agonist of the melanocortin-4 receptor (MC4R). The subject is a heterozygous carrier of an MC4R mutation, and the disorder results from an attenuated response of MC4R to α-melanocortin stimulating hormone (α-MSH).
The disclosure is related to a method of treating a disease, disorder, or condition (e.g., obesity, such as hypothalamic obesity) in a subject using a melanocortin-4 receptor (MC4R) agonist.
The present disclosure relates to compounds of general formula (I) or a pharmaceutically acceptable salt, solvate and/or hydrate thereof, methods for their preparation, the use of said compounds for use as a medicament, and their use in the treatment and/or prevention of a disease or disorder, such as a metabolic disorder (e.g., hyperinsulinism, e.g., congenital hyperinsulinism). The present disclosure also relates to a pharmaceutical composition comprising a compound of general formula (I) and a pharmaceutically acceptable carrier and its use as a medicament, in particular its use in the treatment of a disease or disorder, such as a metabolic disorder (e.g., hyperinsulinism, e.g., congenital hyperinsulinism).
C07D 285/28 - 1,2,4-ThiadiazinesHydrogenated 1,2,4-thiadiazines condensed with carbocyclic rings or ring systems condensed with one six-membered ring with oxygen atoms directly attached to the ring sulfur atom substituted in position 6 or 7 by sulfamoyl or substituted sulfamoyl radicals with only hydrogen atoms or radicals containing only hydrogen and carbon atoms, directly attached in position 3
C07D 417/04 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing two hetero rings directly linked by a ring-member-to-ring- member bond
A61K 31/54 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and at least one sulfur as the ring hetero atoms, e.g. sulthiame
A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
A61P 5/50 - Drugs for disorders of the endocrine system of the pancreatic hormones for increasing or potentiating the activity of insulin
19.
METHODS OF TREATING MELANOCORTIN-4 RECEPTOR PATHWAY-ASSOCIATED DISORDERS
The disclosure is related to a method of treating a disorder, disorder, or condition associated with an MC4R pathway agonizable gene, such as obesity or hyperphagia, in a subject using a melanocortin-4 receptor (MC4R) agonist.
The disclosure is related to a method of treating a non-genetic obesity (e.g., hypothalamic obesity) in a subject using a melanocortin-4 receptor (MC4R) agonist.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Pharmaceuticals and medicines for the treatment of metabolic diseases and disorders, namely, obesity Research and product development services in the fields of metabolic diseases and disorders, namely, obesity
23.
METHOD OF TREATING MELANOCORTIN-4 RECEPTOR PATHWAY-ASSOCIATED DISORDERS
The disclosure is related to a method of treating a disorder, such as obesity or a condition associated with obesity, such as hyperphagia, in a subject using a melanocortin-4 receptor (MC4R) agonist.
The present invention relates to an ionic complex comprising a cationic polypeptide and an anionic excipient selected from: a PEG-carboxylic acid; a fatty acid having 10 or more carbon atoms; an anionic phospholipid; and a combination thereof. The invention also relates to a pharmaceutical composition comprising the ionic complex of the invention and a pharmaceutically acceptable carrier. The cationic polypeptide of the ionic complex has pharmacological activity and the complex can provide a more desirable pharmacokinetic profile for the cationic polypeptide of the complex as compared to the cationic polypeptide alone following administration. As such, the invention also relates to the use of the ionic complex and pharmaceutical composition comprising same to treat a subject suffering from a disease or disorder that is responsive to the cationic polypeptide of the ionic complex.
A61K 47/12 - Carboxylic acidsSalts or anhydrides thereof
A61K 47/24 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
25.
METHODS OF TREATING MELANOCORTIN-4 RECEPTOR PATHWAY-ASSOCIATED DISORDERS
The disclosure is related to a method of treating a disorder, disorder, or condition associated with an MC4R pathway agonizable gene, such as obesity or hyperphagia, in a subject using a melanocortin-4 receptor (MC4R) agonist.
09 - Scientific and electric apparatus and instruments
16 - Paper, cardboard and goods made from these materials
41 - Education, entertainment, sporting and cultural services
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Downloadable electronic publications in the nature of flyers and brochures in the field of genetic testing for medical purposes. Printed flyers and brochures in the field of genetic testing for medical purposes. Providing on-line publications in the nature of news articles and videos in the field of genetic testing for medical purposes. Genetic testing for medical purposes.
This disclosure provides, at least in part, a pharmaceutical product comprising: setmelanotide (also known as RM493); setmelanotide as sole active pharmaceutical ingredient formulated for injection; setmelanotide as the sole active ingredient; setmelanotide as the active pharmaceutical ingredient; setmelanotide as the active pharmaceutical ingredient for injection; or another pharmaceutical composition that has its primary mechanism of action at the MC4 receptor as an agonist (referred to herein as an MC4RAp), e.g., a lipid excipient, and/or a pharmaceutically acceptable carrier. The pharmaceutical product described herein provides a sustained prelease of setmelanotide or another pharmaceutical composition, which may result in a more desirable pharmacokinetic and pharmacodynamic profile upon administration.
The disclosure is related to a method of treating a disorder, such as Prader Willi Syndrome (PWS), obesity or hyperphagia, in a subject using a melanocortin-4 receptor (MC4R) agonist. Also described is method of treating a subject having a deficiency in the pro-opiomelanocortin (POMC)-MC4R pathway, such as a POMC-null or a PCSK-null subject, using a MC4R agonist.
A method of treating a disorder in a subject. The method comprises administering to said subject an effective amount of an agonist of the melanocortin-4 receptor (MC4R). The subject is a heterozygous carrier of an MC4R mutation, and the disorder results from an attenuated response of MC4R to α-melanocortin stimulating hormone (α-MSH).
The present invention relates to polypeptide compounds that are modulators (e.g., agonists and antagonists) of the melanocortin-4 receptor (MC4R) and pharmaceutical compositions comprising same. The compounds described herein are polypeptide of the following structural Formula (I):
or a pharmaceutically acceptable salt thereof. Values and preferred values of the variables in structural Formula (I) are described herein.
41 - Education, entertainment, sporting and cultural services
Goods & Services
Conducting seminars in the field of obesity and obesity disorders; educational services, namely, providing non-downloadable webinars in the field of obesity and obesity disorders
41 - Education, entertainment, sporting and cultural services
Goods & Services
Conducting seminars in the field of genetic obesity disorders; educational services, namely, providing non-downloadable webinars in the field of genetic obesity disorders
05 - Pharmaceutical, veterinary and sanitary products
16 - Paper, cardboard and goods made from these materials
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in genetic factor disease detection, namely, rare obesity Printed instructional materials in the field of genetic testing relevant to obesity Genetic testing for medical purposes in the field of obesity; providing medical information in the field of genetic testing relevant to obesity; medical counseling provided to patients in the fields of obesity, genetic testing and genetics; consulting services in the field of healthcare provided to healthcare professionals in the fields of obesity, genetic testing and genetics
05 - Pharmaceutical, veterinary and sanitary products
16 - Paper, cardboard and goods made from these materials
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Diagnostic kits comprised of medical diagnostic reagents and assays for testing of bodily fluids for use in genetic factor disease detection, namely, rare obesity Printed instructional materials in the field of genetic testing relevant to obesity Genetic testing for medical purposes in the field of obesity; providing medical information in the field of genetic testing relevant to obesity; medical counseling provided to patients in the fields of obesity, genetic testing and genetics; consulting services in the field of healthcare provided to healthcare professionals in the fields of obesity, genetic testing and genetics
38.
Method of treating melanocortin-4 receptor-associated disorders in heterozygous carriers
A method of treating a disorder in a subject. The method comprises administering to said subject an effective amount of an agonist of the melanocortin-4 receptor (MC4R). The subject is a heterozygous carrier of an MC4R mutation, and the disorder results from an attenuated response of MC4R to α-melanocortin stimulating hormone (α-MSH).
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Pharmaceuticals and medicines for the treatment of metabolic
diseases and disorders, including obesity, diabetes,
metabolic syndrome and cachexia, and gastrointestinal
diseases and disorders, including gastroparesis,
post-operative ileus, chronic constipation, and inflammatory
bowel disease. Research and product development services in the fields of
metabolic diseases and disorders, including obesity,
diabetes, metabolic syndrome and cachexia, and
gastrointestinal diseases and disorders, including
gastroparesis, post-operative ileus, chronic constipation,
and inflammatory bowel disease.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
(1) Pharmaceuticals and medicines for the treatment of metabolic diseases and disorders, namely, obesity, diabetes, metabolic syndrome and cachexia, and gastrointestinal diseases and disorders, namely, gastroparesis, post-operative ileus, chronic constipation, and inflammatory bowel disease. (1) Research and product development services in the fields of metabolic diseases and disorders, namely, obesity, diabetes, metabolic syndrome and cachexia, and gastrointestinal diseases and disorders, namely, gastroparesis, post-operative ileus, chronic constipation, and inflammatory bowel disease.
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (France)
Inventor
Van Der Ploeg, Leonardus, H.T.
Garfield, Alastair
Marion, Vincent
Abstract
The disclosure is related to a method of treating chronic kidney disease in a subject with a melanocortin-4 receptor (MC4R) agonist, e.g., a compound of any one of Formulas (I), (II), (III), (IV), (V), (VI), (VII), (VIII), (X), or (XI), or a pharmaceutically acceptable salt thereof (e.g., as described herein).
A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
C12Q 1/00 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions
G01N 33/74 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving hormones
This disclosure provides, at least in part, a pharmaceutical product comprising: setmelanotide (also known as RM493); setmelanotide as sole active pharmaceutical ingredient formulated for injection; setmelanotide as the sole active ingredient; setmelanotide as the active pharmaceutical ingredient; setmelanotide as the active pharmaceutical ingredient for injection; or another pharmaceutical composition that has its primary mechanism of action at the MC4 receptor as an agonist (referred to herein as an MC4RAp), e.g., a lipid excipient, and/or a pharmaceutically acceptable carrier. The pharmaceutical product described herein provides a sustained release of setmelanotide or another pharmaceutical composition, which may result in a more desirable pharmacokinetic and pharmacodynamic profile upon administration.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 47/26 - Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharidesDerivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
A61K 47/32 - Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers
05 - Pharmaceutical, veterinary and sanitary products
Goods & Services
Pharmaceuticals and medicines for the treatment of metabolic diseases and disorders, including obesity [, diabetes, metabolic syndrome and cachexia, and gastrointestinal diseases and disorders, including gastroparesis, post-operative ileus, chronic constipation, and inflammatory bowel disease ]
42 - Scientific, technological and industrial services, research and design
Goods & Services
Research and product development services in the fields of metabolic diseases and disorders, including obesity [, diabetes, metabolic syndrome and cachexia, and gastrointestinal diseases and disorders, including gastroparesis, post-operative ileus, chronic constipation, and inflammatory bowel disease ]
16 - Paper, cardboard and goods made from these materials
41 - Education, entertainment, sporting and cultural services
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
45 - Legal and security services; personal services for individuals.
Goods & Services
Printed pamphlets, brochures, leaflets, informational flyers, and informational sheets in the field of obesity and obesity disorders On-line electronic newsletters delivered by email in the field of obesity and obesity disorders Providing a web site that features medical information about obesity disorders; Providing links to the web sites of others in the field of or relevant to obesity and obesity disorders Providing a website via a global computer network featuring supportive personal stories on the subject of obesity and obesity disorders
16 - Paper, cardboard and goods made from these materials
41 - Education, entertainment, sporting and cultural services
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Printed leaflets, informational flyers, informational sheets and mailings in the nature of informational brochures, flyers and sheets in the field of obesity disorders On-line electronic newsletters delivered by e-mail in the field of obesity disorders Providing a web site that features medical information about obesity disorders; providing screening services for obesity disorders
16 - Paper, cardboard and goods made from these materials
41 - Education, entertainment, sporting and cultural services
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Printed leaflets, informational flyers, informational sheets and mailings in the nature of informational brochures, flyers and sheets in the field of obesity disorders On-line electronic newsletters delivered by e-mail in the field of obesity disorders Providing a web site that features medical information about obesity disorders; providing screening services for obesity disorders
The present invention relates to polypeptide compounds that are modulators (e.g., agonists and antagonists) of the melanocortin-4 receptor (MC4R) and pharmaceutical compositions comprising same. The compounds described herein are polypeptide of the following structural Formula (I):
or a pharmaceutically acceptable salt thereof. Values and preferred values of the variables in structural Formula (I) are described herein.
The disclosure is related to a method of treating a disorder, such as Prader Willi Syndrome (PWS), obesity or hyperphagia, in a subject using a melanocortin-4 receptor (MC4R) agonist. Also described is method of treating a subject having a deficiency in the pro-opiomelanocortin (POMC)-MC4R pathway, such as a POMC-null or a PCSK-null subject, using a MC4R agonist.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Pharmaceuticals and medicines for the treatment of metabolic
diseases and disorders, including obesity, diabetes,
metabolic syndrome and cachexia, and gastrointestinal
diseases and disorders, including gastroparesis,
post-operative ileus, chronic constipation, and inflammatory
bowel disease. Research and product development services in the fields of
metabolic diseases and disorders, including obesity,
diabetes, metabolic syndrome and cachexia, and
gastrointestinal diseases and disorders, including
gastroparesis, post-operative ileus, chronic constipation,
and inflammatory bowel disease.
54.
METHOD OF TREATING MELANOCORTIN-4 RECEPTOR PATHWAY-ASSOCIATED DISORDERS
The disclosure is related to a method of treating a disorder, such as obesity or a condition associated with obesity, such as hyperphagia, in a subject using a melanocortin-4 receptor (MC4R) agonist.
A method of treating a disorder in a subject. The method comprises administering to said subject an effective amount of an agonist of the melanocortin-4 receptor (MC4R). The subject is a heterozygous carrier of an MC4R mutation, and the disorder results from an attenuated response of MC4R to α-melanocortin stimulating hormone (α-MSH).
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
(1) Pharmaceuticals and medicines for the treatment of metabolic diseases and disorders, namely obesity, diabetes, metabolic syndrome and cachexia, and gastrointestinal diseases and disorders, namely gastroparesis, post-operative ileus, chronic constipation, and inflammatory bowel disease (1) Research and product development services in the fields of metabolic diseases and disorders, namely obesity, diabetes, metabolic syndrome and cachexia, and gastrointestinal diseases and disorders, namely gastroparesis, post-operative ileus, chronic constipation, and inflammatory bowel disease
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Pharmaceuticals and medicines for the treatment of metabolic diseases and disorders, including obesity, diabetes, metabolic syndrome and cachexia, and gastrointestinal diseases and disorders, including gastroparesis, post-operative ileus, chronic constipation, and inflammatory bowel disease. Research and product development services in the fields of metabolic diseases and disorders, including obesity, diabetes, metabolic syndrome and cachexia, and gastrointestinal diseases and disorders, including gastroparesis, post-operative ileus, chronic constipation, and inflammatory bowel disease.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Pharmaceuticals and medicines for the treatment of metabolic diseases and disorders, including obesity, diabetes, metabolic syndrome and cachexia, and gastrointestinal diseases and disorders, including gastroparesis, post-operative ileus, chronic constipation, and inflammatory bowel disease. Research and product development services in the fields of metabolic diseases and disorders, including obesity, diabetes, metabolic syndrome and cachexia, and gastrointestinal diseases and disorders, including gastroparesis, post-operative ileus, chronic constipation, and inflammatory bowel disease.
62.
METHOD OF TREATING MELANOCORTIN-4 RECEPTOR PATHWAY-ASSOCIATED DISORDERS
The disclosure is related to a method of treating a disorder, such as obesity or a condition associated with obesity, such as hyperphagia, in a subject using a melanocortin-4 receptor (MC4R) agonist.
The disclosure is related to a method of treating a disorder, such as Prader Willi Syndrome (PWS), obesity or hyperphagia, in a subject using a melanocortin-4 receptor (MC4R) agonist. Also described is method of treating a subject having a deficiency in the pro-opiomelanocortin (POMC)-MC4R pathway, such as a POMC-null or a PCSK-null subject, using a MC4R agonist.
The present invention relates to an ionic complex comprising a cationic polypeptide and an anionic excipient selected from: a PEG-carboxylic acid; a fatty acid having 10 or more carbon atoms; an anionic phospholipid; and a combination thereof. The invention also relates to a pharmaceutical composition comprising the ionic complex of the invention and a pharmaceutically acceptable carrier. The cationic polypeptide of the ionic complex has pharmacological activity and the complex can provide a more desirable pharmacokinetic profile for the cationic polypeptide of the complex as compared to the cationic polypeptide alone following administration. As such, the invention also relates to the use of the ionic complex and pharmaceutical composition comprising same to treat a subject suffering from a disease or disorder that is responsive to the cationic polypeptide of the ionic complex.
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
A61K 47/12 - Carboxylic acidsSalts or anhydrides thereof
A61K 47/24 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
The present invention relates to polypeptide compounds that are modulators (e.g., agonists and antagonists) of the melanocortin-4 receptor (MC4R) and pharmaceutical compositions comprising same. The compounds described herein are polypeptide of the following structural Formula (I):
or a pharmaceutically acceptable salt thereof. Values and preferred values of the variables in structural Formula (I) are described herein.
A method of treating a disorder in a subject. The method comprises administering to said subject an effective amount of an agonist of the melanocortin-4 receptor (MC4R). The subject is a heterozygous carrier of an MC4R mutation, and the disorder results from an attenuated response of MC4R to a-melanocortin stimulating hormone (a-MSH).
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
Pharmaceuticals and medicines for the treatment of metabolic diseases and disorders, including obesity [, diabetes, metabolic syndrome and cachexia, and gastrointestinal diseases and disorders, including gastroparesis, post-operative ileus, chronic constipation, and inflammatory bowel disease ] Research and product development services in the fields of metabolic diseases and disorders, including obesity [, diabetes, metabolic syndrome and cachexia, and gastrointestinal diseases and disorders, including gastroparesis, post-operative ileus, chronic constipation, and inflammatory bowel disease ]
68.
METHOD OF TREATING MELANOCORTIN-4 RECEPTOR-ASSOCIATED DISORDERS IN HETEROZYGOUS CARRIERS
A method of treating a disorder in a subject. The method comprises administering to said subject an effective amount of an agonist of the melanocortin-4 receptor (MC4R). The subject is a heterozygous carrier of an MC4R mutation, and the disorder results from an attenuated response of MC4R to a-melanocortin stimulating hormone (a-MSH).
The present invention relates to polypeptide compounds that are modulators (e.g., agonists and antagonists) of the melanocortin-4 receptor (MC4R) and pharmaceutical compositions comprising same. The compounds described herein are polypeptide of the following structural Formula (I): or a pharmaceutically acceptable salt thereof. Values and preferred values of the variables in structural Formula (I) are described herein.
The present invention relates to an ionic complex comprising a cationic polypeptide and an anionic excipient selected from: a PEG-carboxylic acid; a fatty acid having 10 or more carbon atoms; an anionic phospholipid; and a combination thereof. The invention also relates to a pharmaceutical composition comprising the ionic complex of the invention and a pharmaceutically acceptable carrier. The cationic polypeptide of the ionic complex has pharmacological activity and the complex can provide a more desirable pharmacokinetic profile for the cationic polypeptide of the complex as compared to the cationic polypeptide alone following administration. As such, the invention also relates to the use of the the ionic complex and pharmaceuctical composition comprising same to treat a subject suffering from a disease or disorder that is responsive to the cationic polypeptide of the ionic complex.
The disclosure is related to a method of treating a disorder, disorder, or condition associated with an MC4R pathway agonizable gene, such as obesity or hyperphagia, in a subject using a melanocortin-4 receptor (MC4R) agonist.
The disclosure is related to a method of treating a disorder, such as obesity or a condition associated with obesity, such as hyperphagia, in a subject using a melanocortin-4 receptor (MC4R) agonist.
The disclosure is related to a method of treating a disorder, such as Prader Willi Syndrome (PWS), obesity or hyperphagia, in a subject using a melanocortin-4 receptor (MC4R) agonist. Also described is method of treating a subject having a deficiency in the pro-opiomelanocortin (POMC)-MC4R pathway, such as a POMC-null or a PCSK-null subject, using a MC4R agonist.
This disclosure provides, at least in part, a pharmaceutical product comprising: setmelanotide (also known as RM493); setmelanotide as sole active pharmaceutical ingredient formulated for injection; setmelanotide as the sole active ingredient; setmelanotide as the active pharmaceutical ingredient; setmelanotide as the active pharmaceutical ingredient for injection; or another pharmaceutical composition that has its primary mechanism of action at the MC4 receptor as an agonist (referred to herein as an MC4RAp), e.g., a lipid excipient, and/or a pharmaceutically acceptable carrier. The pharmaceutical product described herein provides a sustained release of setmelanotide or another pharmaceutical composition, which may result in a more desirable pharmacokinetic and pharmacodynamic profile upon administration.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 47/26 - Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharidesDerivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
A61K 47/32 - Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (France)
RHYTHM PHARMACEUTICALS, INC. (USA)
Inventor
Van Der Ploeg, Leonardus H.T.
Garfield, Alastair
Marion, Vincent
Abstract
The disclosure is related to a method of treating chronic kidney disease in a subject with a melanocortin-4 receptor (MC4R) agonist, e.g., a compound of any one of Formulas (I), (II), (III), (IV), (V), (VI), (VII), (VIII), (X), or (XI), or a pharmaceutically acceptable salt thereof (e.g., as described herein).
The present invention relates to an ionic complex comprising a cationic polypeptide and an anionic excipient selected from: a PEG-carboxylic acid; a fatty acid having 10 or more carbon atoms; an anionic phospholipid; and a combination thereof. The invention also relates to a pharmaceutical composition comprising the ionic complex of the invention and a pharmaceutically acceptable carrier. The cationic polypeptide of the ionic complex has pharmacological activity and the complex can provide a more desirable pharmacokinetic profile for the cationic polypeptide of the complex as compared to the cationic polypeptide alone following administration. As such, the invention also relates to the use of the ionic complex and pharmaceutical composition comprising same to treat a subject suffering from a disease or disorder that is responsive to the cationic polypeptide of the ionic complex.
A61K 31/54 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and at least one sulfur as the ring hetero atoms, e.g. sulthiame
A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
A61P 5/50 - Drugs for disorders of the endocrine system of the pancreatic hormones for increasing or potentiating the activity of insulin
C07D 285/28 - 1,2,4-ThiadiazinesHydrogenated 1,2,4-thiadiazines condensed with carbocyclic rings or ring systems condensed with one six-membered ring with oxygen atoms directly attached to the ring sulfur atom substituted in position 6 or 7 by sulfamoyl or substituted sulfamoyl radicals with only hydrogen atoms or radicals containing only hydrogen and carbon atoms, directly attached in position 3
C07D 417/04 - Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group containing two hetero rings directly linked by a ring-member-to-ring- member bond
