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 - Composés hétérocycliques contenant des atomes d'azote comme uniques hétéro-atomes du système condensé, au moins un cycle étant un cycle à six chaînons avec un atome d'azote, non prévus dans les groupes dans lesquels le système condensé contient deux hétérocycles
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 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. guanéthidine ou rifamycines
C07D 207/02 - Composés hétérocycliques contenant des cycles à cinq chaînons, non condensés avec d'autres cycles, ne comportant qu'un atome d'azote comme unique hétéro-atome du cycle avec uniquement des atomes d'hydrogène ou de carbone liés directement à l'atome d'azote du cycle
A61K 31/535 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. guanéthidine ou rifamycines ayant des cycles à six chaînons avec au moins un azote et au moins un oxygène comme hétéro-atomes d'un cycle, p. ex. 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 - PyrimidinesPyrimidines hydrogénées, p. ex. triméthoprime ayant des groupes oxo liés directement à l'hétérocycle, p. ex. cytosine
A61K 31/549 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. guanéthidine ou rifamycines ayant des cycles à six chaînons avec au moins un azote et au moins un soufre comme hétéro-atomes d'un cycle, p. ex. sulthiame ayant plusieurs atomes d'azote dans le même cycle, p. ex. hydrochlorothiazide
A61P 3/00 - Médicaments pour le traitement des troubles du métabolisme
45 - Services juridiques; services de sécurité; services personnels pour individus
16 - Papier, carton et produits en ces matières
41 - Éducation, divertissements, activités sportives et culturelles
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et 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)
Inventeur(s)
Van Der Ploeg, Leonardus H.T.
Garfield, Alastair
Marion, Vincent
Abrégé
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 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et 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 - Thiadiazines-1, 2, 4Thiadiazines-1, 2, 4 hydrogénées condensées avec des carbocycles ou avec des systèmes carbocycliques condensées avec un cycle à six chaînons avec des atomes d'oxygène liés directement à l'atome de soufre du cycle substituées en position 6 ou 7 par des radicaux sulfamoyle ou sulfamoyle substitués avec uniquement des atomes d'hydrogène ou des radicaux ne contenant que des atomes de carbone, liés directement en position 3
C07D 417/04 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes de soufre et d'azote comme uniques hétéro-atomes du cycle, non prévus par le groupe contenant deux hétérocycles liés par une liaison directe de chaînon cyclique à chaînon cyclique
A61K 31/54 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. guanéthidine ou rifamycines ayant des cycles à six chaînons avec au moins un azote et au moins un soufre comme hétéro-atomes d'un cycle, p. ex. sulthiame
A61P 3/10 - Médicaments pour le traitement des troubles du métabolisme de l'homéostase du glucose de l'hyperglycémie, p. ex. antidiabétiques
A61P 5/50 - Médicaments pour le traitement des troubles du système endocrinien des hormones pancréatiques pour augmenter ou potentialiser l'activité de l'insuline
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 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et 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 9/14 - Préparations médicinales caractérisées par un aspect particulier à l'état particulaire, p. ex. poudres
A61K 38/08 - Peptides ayant de 5 à 11 amino-acides
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
A61K 47/10 - AlcoolsPhénolsLeurs sels, p. ex. glycérolPolyéthylène glycols [PEG]PoloxamèresAlkyléthers de PEG/POE
A61K 47/12 - Acides carboxyliquesLeurs sels ou anhydrides
A61K 47/24 - Composés organiques, p. ex. hydrocarbures naturels ou synthétiques, polyoléfines, huile minérale, gelée de pétrole ou ozocérite contenant des atomes autres que des atomes de carbone, d'hydrogène, d'oxygène, d'halogènes, d'azote ou de soufre, p. ex. cyclométhicone ou phospholipides
A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
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 - Appareils et instruments scientifiques et électriques
16 - Papier, carton et produits en ces matières
41 - Éducation, divertissements, activités sportives et culturelles
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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 - Éducation, divertissements, activités sportives et culturelles
Produits et 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 - Éducation, divertissements, activités sportives et culturelles
Produits et 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 - Produits pharmaceutiques, vétérinaires et hygièniques
16 - Papier, carton et produits en ces matières
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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 - Produits pharmaceutiques, vétérinaires et hygièniques
16 - Papier, carton et produits en ces matières
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et 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 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et 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)
Inventeur(s)
Van Der Ploeg, Leonardus, H.T.
Garfield, Alastair
Marion, Vincent
Abrégé
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 3/00 - Médicaments pour le traitement des troubles du métabolisme
A61K 38/08 - Peptides ayant de 5 à 11 amino-acides
A61P 13/12 - Médicaments pour le traitement des troubles du système urinaire des reins
C12Q 1/00 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions
G01N 33/74 - Analyse chimique de matériau biologique, p. ex. de sang ou d'urineTest par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligandsTest immunologique faisant intervenir des 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 ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
A61K 47/26 - Hydrates de carbone, p. ex. polyols ou sucres alcoolisés, sucres aminés, acides nucléiques, mono-, di- ou oligosaccharidesLeurs dérivés, p. ex. polysorbates, esters d’acide gras de sorbitan ou glycyrrhizine
A61K 47/32 - Composés macromoléculaires obtenus par des réactions faisant intervenir uniquement des liaisons non saturées carbone-carbone, p. ex. carbomères
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et 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 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et 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 ]
41 - Éducation, divertissements, activités sportives et culturelles
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
45 - Services juridiques; services de sécurité; services personnels pour individus
Produits et 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
41 - Éducation, divertissements, activités sportives et culturelles
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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
41 - Éducation, divertissements, activités sportives et culturelles
44 - Services médicaux, services vétérinaires, soins d'hygiène et de beauté; services d'agriculture, d'horticulture et de sylviculture.
Produits et 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 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et 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 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et 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 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et 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 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et 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 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
A61K 9/14 - Préparations médicinales caractérisées par un aspect particulier à l'état particulaire, p. ex. poudres
A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
A61K 47/12 - Acides carboxyliquesLeurs sels ou anhydrides
A61K 47/24 - Composés organiques, p. ex. hydrocarbures naturels ou synthétiques, polyoléfines, huile minérale, gelée de pétrole ou ozocérite contenant des atomes autres que des atomes de carbone, d'hydrogène, d'oxygène, d'halogènes, d'azote ou de soufre, p. ex. cyclométhicone ou phospholipides
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 - Produits pharmaceutiques, vétérinaires et hygièniques
42 - Services scientifiques, technologiques et industriels, recherche et conception
Produits et 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 ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
A61K 47/26 - Hydrates de carbone, p. ex. polyols ou sucres alcoolisés, sucres aminés, acides nucléiques, mono-, di- ou oligosaccharidesLeurs dérivés, p. ex. polysorbates, esters d’acide gras de sorbitan ou glycyrrhizine
A61K 47/32 - Composés macromoléculaires obtenus par des réactions faisant intervenir uniquement des liaisons non saturées carbone-carbone, p. ex. carbomères
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (France)
RHYTHM PHARMACEUTICALS, INC. (USA)
Inventeur(s)
Van Der Ploeg, Leonardus H.T.
Garfield, Alastair
Marion, Vincent
Abrégé
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).
A61K 38/08 - Peptides ayant de 5 à 11 amino-acides
A61P 3/00 - Médicaments pour le traitement des troubles du métabolisme
A61P 13/12 - Médicaments pour le traitement des troubles du système urinaire des reins
C07K 7/06 - Peptides linéaires ne contenant que des liaisons peptidiques normales ayant de 5 à 11 amino-acides
G01N 33/74 - Analyse chimique de matériau biologique, p. ex. de sang ou d'urineTest par des méthodes faisant intervenir la formation de liaisons biospécifiques par ligandsTest immunologique faisant intervenir des hormones
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 - Composés hétérocycliques ayant l'azote comme hétéro-atome d'un cycle, p. ex. guanéthidine ou rifamycines ayant des cycles à six chaînons avec au moins un azote et au moins un soufre comme hétéro-atomes d'un cycle, p. ex. sulthiame
A61P 3/10 - Médicaments pour le traitement des troubles du métabolisme de l'homéostase du glucose de l'hyperglycémie, p. ex. antidiabétiques
A61P 5/50 - Médicaments pour le traitement des troubles du système endocrinien des hormones pancréatiques pour augmenter ou potentialiser l'activité de l'insuline
C07D 285/28 - Thiadiazines-1, 2, 4Thiadiazines-1, 2, 4 hydrogénées condensées avec des carbocycles ou avec des systèmes carbocycliques condensées avec un cycle à six chaînons avec des atomes d'oxygène liés directement à l'atome de soufre du cycle substituées en position 6 ou 7 par des radicaux sulfamoyle ou sulfamoyle substitués avec uniquement des atomes d'hydrogène ou des radicaux ne contenant que des atomes de carbone, liés directement en position 3
C07D 417/04 - Composés hétérocycliques contenant plusieurs hétérocycles, au moins un cycle comportant des atomes de soufre et d'azote comme uniques hétéro-atomes du cycle, non prévus par le groupe contenant deux hétérocycles liés par une liaison directe de chaînon cyclique à chaînon cyclique
