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1. (WO2017034990) COMPOSITION AND METHODS OF USE OF TETRAHYDROISOQUINOLINE SMALL MOLECULES TO BIND AND MODULATE PCSK9 PROTEIN ACTIVITY
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Pub. No.: WO/2017/034990 International Application No.: PCT/US2016/047798
Publication Date: 02.03.2017 International Filing Date: 19.08.2016
IPC:
A61P 3/06 (2006.01) ,A61K 31/472 (2006.01) ,C07D 217/02 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3
Drugs for disorders of the metabolism
06
Antihyperlipidemics
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
33
Heterocyclic compounds
395
having nitrogen as a ring hetero atom, e.g. guanethidine, rifamycins
435
having six-membered rings with one nitrogen as the only ring hetero atom
47
Quinolines; Isoquinolines
472
Non-condensed isoquinolines, e.g. papaverine
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
217
Heterocyclic compounds containing isoquinoline or hydrogenated isoquinoline ring systems
02
with only hydrogen atoms or radicals containing only carbon and hydrogen atoms, directly attached to carbon atoms of the nitrogen-containing ring; Alkylene-bis-isoquinolines
Applicants:
PORTOLA PHARMACEUTICALS, INC. [US/US]; 270 E. Grand Ave., Suite 22 South San Francisco, CA 94080, US
Inventors:
BARTA, Thomas, E.; US
BOURNE, Jonathan William; US
MONROE, Kyle, D.; US
MUEHLEMANN, Michael, M.; US
PANDEY, Anjali; US
BOWERS, Simeon; US
Agent:
SU, Ting; US
KELLY, Gilbert, Thomson; US
CARROLL, Peter, G.; US
HAMDAN, Maha, A.; US
Priority Data:
62/208,06821.08.2015US
Title (EN) COMPOSITION AND METHODS OF USE OF TETRAHYDROISOQUINOLINE SMALL MOLECULES TO BIND AND MODULATE PCSK9 PROTEIN ACTIVITY
(FR) COMPOSITION ET PROCÉDÉS D'UTILISATION DE PETITES MOLÉCULES DE TYPE TÉTRAHYDROISOQUINOLINE POUR SE LIER À PCSK9 ET MODULER L'ACTIVITÉ PROTÉIQUE DE PCSK9
Abstract:
(EN) This invention is related to the field of PCSK9 biology and the composition and methods of use of small organic compounds as ligands for modulation of PCSK9 biological activity. In particular, the invention provides compositions of small organic compounds that modulate circulating levels of low density lipoproteins by altering the conformation of the protein PCSK9. Binding these small organic compound ligands to PCSK9 alters the conformation of the protein, modifying the interaction between PCSK9 and an endogenous low density lipoprotein receptor, and can lead to reduced or increased levels of circulating LDL-cholesterol. High LDL- cholesterol levels are associated with increased risk for heart disease. Low LDL-cholesterol levels may be problematic in other conditions, such as liver dysfunction; thus, there is also utility for small organic compound ligands that can raise LDL levels.
(FR) La présente invention relève du domaine de la biologie de PCSK9 et concerne une composition et des procédés d'utilisation de petits composés organiques en tant que ligands pour moduler l'activité biologique de PCSK9. En particulier, l'invention concerne des compositions constituées de petits composés organiques qui modulent les taux circulants de lipoprotéines basse densité par modification de la conformation de la protéine PCSK9. La liaison de ces ligands de type petit composé organique à PCSK9 modifie la conformation de la protéine, modifiant ainsi l'interaction entre la PCSK9 et un récepteur endogène de lipoprotéine basse densité, et permet d'obtenir des taux réduits ou accrus de cholestérol LDL circulant. Des taux élevés de cholestérol LDL sont associés à un risque accru de maladie cardiaque. De bas taux de cholestérol LDL peuvent s'avérer problématiques dans d'autres affections, telles que la dysfonction hépatique; par conséquent, les ligands de type petit composé organique capables d'élever les taux LDL sont également utiles.
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Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)