A61K31/4245

CRYSTALLINE FORM OF THE COMPOUND (S)-3-{4-[5-(2-CYCLOPENTYL-6-METHOXY-PYRIDIN-4-YL)-[1,2,4]OXADIAZOL-3-YL]-2-ETHYL-6-METHYL-PHENOXY}-PROPANE-1,2-DIOL
20230002365 · 2023-01-05 ·

The present invention relates to a crystalline form of the compound (S)-3-{4-[5-(2-cyclopentyl-6-methoxy-pyridin-4-yl)-[1,2,4]oxadiazol-3-yl]-2-ethyl-6-methyl-phenoxy}-propane-1,2-diol.

SUBSTITUTED BENZAMIDES AS MODULATORS OF TREX1

Provided are compounds of Formula (I): (I) and pharmaceutically acceptable salts and compositions thereof, which are useful for treating a variety of conditions associated with TREX1.

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SUBSTITUTED BENZAMIDES AS MODULATORS OF TREX1

Provided are compounds of Formula (I): (I) and pharmaceutically acceptable salts and compositions thereof, which are useful for treating a variety of conditions associated with TREX1.

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SPHINGOSINE-1-PHOSPHATE RECEPTOR 1 AGONIST AND LIPOSOMAL FORMULATIONS THEREOF

The present disclosure is directed to a compound or compositions comprising the same for decreasing vascular permeability and the prevention or inhibition of metastasis in a cancer.

SPHINGOSINE-1-PHOSPHATE RECEPTOR 1 AGONIST AND LIPOSOMAL FORMULATIONS THEREOF

The present disclosure is directed to a compound or compositions comprising the same for decreasing vascular permeability and the prevention or inhibition of metastasis in a cancer.

THERAPEUTIC COMBINATIONS OF DRUGS AND METHODS OF USING THEM
20230002377 · 2023-01-05 ·

Provided are therapeutic combinations or formulations of drugs comprising triple monoamine reuptake inhibitors, melanin concentrating hormone receptor 1 (MCHRT) antagonists and diazoxide or its formulations and various combinations thereof, these in combination with other drugs or active agents. Provided are methods for the treatment of various conditions, including genetic confirmed syndromes, and diseases, using therapeutic combinations and formulations of drugs as provided herein. Provided are methods for administering triple monoamine reuptake inhibitors (TRIs), melanin concentrating hormone receptor 1 (MCHRT) antagonists and diazoxide or diazoxide or its formulations, whose dosages are determined using a method as provided herein including empirical methods for safe and predictable titration and to determine the initial therapeutic dose; model-based methods for safe and predictable titration and to determine the initial therapeutic dose and to determine the lowest therapeutic dose or to determine an optimal effective dose, including use of Bayesian pharmacometric models.

THERAPEUTIC COMBINATIONS OF DRUGS AND METHODS OF USING THEM
20230002377 · 2023-01-05 ·

Provided are therapeutic combinations or formulations of drugs comprising triple monoamine reuptake inhibitors, melanin concentrating hormone receptor 1 (MCHRT) antagonists and diazoxide or its formulations and various combinations thereof, these in combination with other drugs or active agents. Provided are methods for the treatment of various conditions, including genetic confirmed syndromes, and diseases, using therapeutic combinations and formulations of drugs as provided herein. Provided are methods for administering triple monoamine reuptake inhibitors (TRIs), melanin concentrating hormone receptor 1 (MCHRT) antagonists and diazoxide or diazoxide or its formulations, whose dosages are determined using a method as provided herein including empirical methods for safe and predictable titration and to determine the initial therapeutic dose; model-based methods for safe and predictable titration and to determine the initial therapeutic dose and to determine the lowest therapeutic dose or to determine an optimal effective dose, including use of Bayesian pharmacometric models.

SUBSTITUTED HYDANTOINAMIDES AS ADAMTS7 ANTAGONISTS

The application relates to substituted hydantoinamides of formula (I) as ADAMTS7 antagonists, to processes for their preparation, their use alone or in combination for the treatment or prophylaxis of diseases, in particular of cardiovascular diseases, including atherosclerosis, coronary artery disease (CAD), peripheral vascular disease (PAD), arterial occlusive disease or restenosis after angioplasty. R.sup.1 is hydrogen, alkyl, cycloalkyl, heterocycloalkyl, heteroaryl or phenyl; R.sup.2 is hydrogen, cyano, halogen, alkylsulfonyl, alkyl, cycloalkyl or alkoxy; R.sup.3, R.sup.4, R.sup.5, R.sup.6, R.sup.7 and R.sup.8 are independently hydrogen, halogen, alkyl or alkoxy; most groups being optionally substituted; with the proviso that at least one of R.sup.2, R.sup.3, R.sup.4 is H; X.sup.1, X.sup.2, X.sup.3, X.sup.4, X.sup.5 and X.sup.6 are independently N or C; with the proviso that in each ring maximal one X is N.

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SUBSTITUTED HYDANTOINAMIDES AS ADAMTS7 ANTAGONISTS

The application relates to substituted hydantoinamides of formula (I) as ADAMTS7 antagonists, to processes for their preparation, their use alone or in combination for the treatment or prophylaxis of diseases, in particular of cardiovascular diseases, including atherosclerosis, coronary artery disease (CAD), peripheral vascular disease (PAD), arterial occlusive disease or restenosis after angioplasty. R.sup.1 is hydrogen, alkyl, cycloalkyl, heterocycloalkyl, heteroaryl or phenyl; R.sup.2 is hydrogen, cyano, halogen, alkylsulfonyl, alkyl, cycloalkyl or alkoxy; R.sup.3, R.sup.4, R.sup.5, R.sup.6, R.sup.7 and R.sup.8 are independently hydrogen, halogen, alkyl or alkoxy; most groups being optionally substituted; with the proviso that at least one of R.sup.2, R.sup.3, R.sup.4 is H; X.sup.1, X.sup.2, X.sup.3, X.sup.4, X.sup.5 and X.sup.6 are independently N or C; with the proviso that in each ring maximal one X is N.

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COMPOSITIONS FOR AN ORALLY ACTIVE 1,2,4-OXADIAZOLE FOR THE TREATMENT OF DISEASE
20230218587 · 2023-07-13 ·

The present invention relates to specific doses of and dosing regimens for using a 1,2,4-oxadiazole benzoic acid compound in treating or preventing diseases associated with nonsense mutations. In particular, the invention relates to specific doses and dosing regimens for the use of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid in mammals having diseases associated with nonsense mutations.