HRP20231247T1 - Postupci uporabe aktivatora piruvat kinaze - Google Patents
Postupci uporabe aktivatora piruvat kinaze Download PDFInfo
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- HRP20231247T1 HRP20231247T1 HRP20231247TT HRP20231247T HRP20231247T1 HR P20231247 T1 HRP20231247 T1 HR P20231247T1 HR P20231247T T HRP20231247T T HR P20231247TT HR P20231247 T HRP20231247 T HR P20231247T HR P20231247 T1 HRP20231247 T1 HR P20231247T1
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- 238000000034 method Methods 0.000 title claims 41
- 108020005115 Pyruvate Kinase Proteins 0.000 title claims 2
- 102000013009 Pyruvate Kinase Human genes 0.000 title claims 2
- 239000012190 activator Substances 0.000 title 1
- 229940125904 compound 1 Drugs 0.000 claims 48
- XAYGBKHKBBXDAK-UHFFFAOYSA-N n-[4-[4-(cyclopropylmethyl)piperazine-1-carbonyl]phenyl]quinoline-8-sulfonamide Chemical compound C=1C=C(NS(=O)(=O)C=2C3=NC=CC=C3C=CC=2)C=CC=1C(=O)N(CC1)CCN1CC1CC1 XAYGBKHKBBXDAK-UHFFFAOYSA-N 0.000 claims 8
- 150000003839 salts Chemical class 0.000 claims 8
- 108700014121 Pyruvate Kinase Deficiency of Red Cells Proteins 0.000 claims 6
- 208000002903 Thalassemia Diseases 0.000 claims 5
- 208000007475 hemolytic anemia Diseases 0.000 claims 5
- AICOOMRHRUFYCM-ZRRPKQBOSA-N oxazine, 1 Chemical compound C([C@@H]1[C@H](C(C[C@]2(C)[C@@H]([C@H](C)N(C)C)[C@H](O)C[C@]21C)=O)CC1=CC2)C[C@H]1[C@@]1(C)[C@H]2N=C(C(C)C)OC1 AICOOMRHRUFYCM-ZRRPKQBOSA-N 0.000 claims 4
- 208000007056 sickle cell anemia Diseases 0.000 claims 4
- 108010044467 Isoenzymes Proteins 0.000 claims 2
- 230000004913 activation Effects 0.000 claims 2
- 230000003213 activating effect Effects 0.000 claims 1
- 208000005980 beta thalassemia Diseases 0.000 claims 1
- 201000003922 congenital nonspherocytic hemolytic anemia Diseases 0.000 claims 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/496—Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
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- Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Life Sciences & Earth Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Other Investigation Or Analysis Of Materials By Electrical Means (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Enzymes And Modification Thereof (AREA)
Claims (24)
1. Spoj 1 za uporabu u postupku za liječenje nedostatka piruvat kinaze (PKD) kod subjekta kojem je to potrebno, koji se sastoji od oralne primjene subjektu doze od oko 10 mg do oko 60 mg ili oko 60 mg do oko 200 mg spoja 1 ili njegova farmaceutski prihvatljiva sol ili hidrat jednom ili dva puta dnevno da se na taj način liječi PKD kod subjekta, naznačen time što je spoj 1 N-(4-(4-(ciklopropilmetil)piperazin-1-karbonil)fenil)kinolin-8-sulfonamid.
2. Spoj 1 za uporabu u postupku za liječenje nedostatka piruvat kinaze (PKD) kod subjekta kojem je to potrebno, koji sadrži oralno davanje subjektu doze od oko 50 mg do oko 300 mg spoja 1 ili njegove farmaceutski prihvatljive soli ili hidrata jednom ili dva puta dnevno da se na taj način liječi PKD kod subjekta naznačen time što je spoj 1 N-(4-(4-(ciklopropilmetil)piperazin-1-karbonil)fenil)kinolin-8-sulfonamid.
3. Spoj 1 za uporabu u postupku prema patentnom zahtjevu 1 ili 2, naznačen time što postupak sadrži aktiviranje piruvat kinaze R, ili
pri čemu postupak sadrži aktiviranje mutantnog PKR izozima.
4. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1 do 3, naznačen time što postupak sadrži aktivaciju mutantnog PKR izozima.
5. Spoj 1 za uporabu u postupku za liječenje hemolitičke anemije koji sadrži oralno davanje subjektu kojem je to potrebno doze od oko 10 mg do oko 60 mg ili oko 60 mg do oko 200 mg spoja 1 ili njegove farmaceutski prihvatljive soli ili hidrata jednom ili dva puta dnevno da se na taj način liječi hemolitička anemija kod subjekta naznačen time što je spoj 1 N-(4-(4-(ciklopropilmetil)piperazin-1-karbonil)fenil)kinolin-8-sulfonamid.
6. Spoj 1 za uporabu u postupku za liječenje hemolitičke anemije kod subjekta kojem je to potrebno, koji sadrži oralno davanje subjektu doze od oko 50 mg do oko 300 mg spoja 1 ili njegove farmaceutski prihvatljive soli ili hidrata jednom ili dva puta dnevno da se na taj način liječi hemolitička anemija kod subjekta naznačen time što je spoj 1 N-(4-(4-(ciklopropilmetil)piperazin-1-karbonil)fenil)kinoline-8-sulfonamid.
7. Spoj 1 za uporabu u postupku prema zahtjevu 5 ili 6, naznačen time što je hemolitička anemija nasljedna ne-sferocitna hemolitička anemija.
8. Spoj 1 za uporabu u postupku za liječenje talasemije koji sadrži oralno davanje subjektu kojem je to potrebno doze od oko 10 mg do oko 60 mg ili oko 60 mg do oko 200 mg spoja 1 ili njegove farmaceutski prihvatljive soli ili hidrata jednom ili dva puta dnevno da se na taj način liječi talasemija kod subjekta naznačen time što je spoj 1 N-(4-(4-(ciklopropilmetil)piperazin-1-karbonil)fenil)kinolin-8-sulfonamid.
9. Spoj 1 za uporabu u postupku za liječenje talasemije kod subjekta kojem je to potrebno, koji sadrži oralno davanje subjektu doze od oko 50 mg do oko 300 mg spoja 1 ili njegove farmaceutski prihvatljive soli ili hidrata jednom ili dva puta dnevno da se na taj način liječi talasemija kod subjekta naznačen time što je spoj 1 N-(4-(4-(ciklopropilmetil)piperazin-1-karbonil)fenil)kinolin-8-sulfonamid.
10. Spoj 1 za uporabu u postupku prema zahtjevu 7 ili 8, naznačen time što je talasemija beta-talasemija.
11. Spoj 1 za uporabu u postupku za liječenje anemije srpastih stanica koji sadrži oralno davanje subjektu kojem je to potrebno doze od oko 10 mg do oko 60 mg ili oko 60 mg do oko 200 mg spoja 1 ili farmaceutski prihvatljive soli ili hidrata od toga jednom ili dva puta dnevno da se na taj način liječi anemija srpastih stanica naznačen time što je spoj 1 N-(4-(4-(ciklopropilmetil)piperazin-1-karbonil)fenil)kinolin-8-sulfonamid.
12. Spoj 1 za uporabu u postupku za liječenje anemije srpastih stanica kod subjekta kojem je to potrebno, koji sadrži oralno davanje subjektu doze od oko 50 mg do oko 300 mg spoja 1 ili njegove farmaceutski prihvatljive soli ili hidrata jednom ili dva puta dnevno da se na taj način liječi anemija srpastih stanica kod subjekta naznačen time što je spoj 1 N-(4-(4-(ciklopropilmetil)piperazin-1-karbonil)fenil)kinolin-8-sulfonamid.
13. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1, 5, 8, i 11, naznačen time što postupak sadrži oralno davanje subjektu jednom ili dva puta dnevno doze od oko 10 mg do oko 60 mg spoja 1.
14. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1, 5, 8, i 11, naznačen time što postupak sadrži oralno davanje subjektu jednom ili dva puta dnevno doze od oko 60 mg do oko 200 mg spoja 1.
15. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1 do 14, naznačen time što postupak sadrži oralno davanje subjektu jednom ili dva puta dnevno doze od oko 30 mg, ili oko 120 mg spoja 1.
16. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1 do 14, naznačen time što postupak sadrži davanje subjektu jednom ili dva puta dnevno doze od oko 50 mg, oko 75 mg, oko 100 mg, oko 125 mg, oko 150 mg, oko 175 mg, oko 200 mg, oko 225 mg, oko 250 mg, oko 275 mg ili oko 300 mg spoja 1.
17. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1 do 14, naznačen time što postupak sadrži davanje spoja 1 jednom dnevno.
18. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1 do 14, naznačen time što postupak sadrži davanje spoja 1 dva puta dnevno.
19. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1 do 14, naznačen time što postupak sadrži davanje spoja 1 u dozi od oko 50 mg dva puta dnevno.
20. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1 do 14, naznačen time što postupak sadrži davanje spoja 1 u dozi od oko 100 mg dva puta dnevno.
21. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1 do 14, naznačen time što postupak sadrži davanje subjektu doze spoja 1 od oko 15 mg oko svakih 12 sati, oko 60 mg oko svakih 12 sati, oko 120 mg otprilike svakih 12 sati.
22. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1 do 14, naznačen time što postupak sadrži davanje subjektu doze spoja 1 od oko 10 mg do oko 60 mg svakih 12 sati ili oko 60 mg do oko 200 mg otprilike svakih 12 sati.
23. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1 do 14, naznačen time što postupak sadrži davanje subjektu doze spoja 1 od oko 60 mg do oko 200 mg oko svaka 24 sata.
24. Spoj 1 za uporabu u postupku prema bilo kojem od zahtjeva 1 do 14, pri čemu postupak sadrži davanje subjektu doze spoja 1 od oko 90 mg oko svaka 24 sata, oko 120 mg oko svaka 24 sata, oko 150 mg otprilike svaka 24 sata, oko 180 mg otprilike svaka 24 sata, ili oko 200 mg otprilike svaka 24 sata.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562174216P | 2015-06-11 | 2015-06-11 | |
| PCT/US2016/036893 WO2016201227A1 (en) | 2015-06-11 | 2016-06-10 | Methods of using pyruvate kinase activators |
| EP16808374.9A EP3307271B1 (en) | 2015-06-11 | 2016-06-10 | Methods of using pyruvate kinase activators |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20231247T1 true HRP20231247T1 (hr) | 2024-02-02 |
Family
ID=57504271
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20231247TT HRP20231247T1 (hr) | 2015-06-11 | 2016-06-10 | Postupci uporabe aktivatora piruvat kinaze |
Country Status (20)
| Country | Link |
|---|---|
| US (2) | US11234976B2 (hr) |
| EP (2) | EP3307271B1 (hr) |
| JP (3) | JP7320339B2 (hr) |
| AU (2) | AU2016276951B2 (hr) |
| CA (1) | CA2989111C (hr) |
| DK (1) | DK3307271T3 (hr) |
| ES (1) | ES2959690T3 (hr) |
| FI (1) | FI3307271T3 (hr) |
| HR (1) | HRP20231247T1 (hr) |
| HU (1) | HUE063541T2 (hr) |
| LT (1) | LT3307271T (hr) |
| MA (1) | MA44392B1 (hr) |
| MD (1) | MD3307271T2 (hr) |
| MX (1) | MX384883B (hr) |
| PL (1) | PL3307271T3 (hr) |
| PT (1) | PT3307271T (hr) |
| RS (1) | RS64777B1 (hr) |
| SI (1) | SI3307271T1 (hr) |
| SM (1) | SMT202300344T1 (hr) |
| WO (1) | WO2016201227A1 (hr) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3708169A1 (en) | 2009-06-29 | 2020-09-16 | Agios Pharmaceuticals, Inc. | 2,3-dihydrobenzo[b][1,4]dioxine-6-sulfonamide derivatives having an anticancer activity |
| ES2970938T3 (es) | 2011-05-03 | 2024-05-31 | Agios Pharmaceuticals Inc | Activadores de piruvato quinasa para uso en terapia |
| TN2020000045A1 (en) | 2017-03-20 | 2021-10-04 | Forma Therapeutics Inc | Pyrrolopyrrole compositions as pyruvate kinase (pkr) |
| WO2019099651A1 (en) * | 2017-11-16 | 2019-05-23 | Agios Pharmaceuticals, Inc. | Methods of using deuterated pyruvate kinase activators |
| SMT202300327T1 (it) * | 2017-11-22 | 2023-11-13 | Agios Pharmaceuticals Inc | Forme cristalline di n-(4-(4-(ciclopropilmetil) piperazin-1-carbonil)fenil)chinolina-8-solfonammide |
| CN108169370B (zh) * | 2017-12-19 | 2020-09-04 | 万舒（北京）医药科技有限公司 | 测定生物样品中丙酮酸乙酯及其代谢物丙酮酸的方法 |
| US12053458B2 (en) | 2018-09-19 | 2024-08-06 | Novo Nordisk Health Care Ag | Treating sickle cell disease with a pyruvate kinase R activating compound |
| WO2020061255A1 (en) | 2018-09-19 | 2020-03-26 | Forma Therapeutics, Inc. | Activating pyruvate kinase r |
| CN118206485A (zh) | 2019-05-22 | 2024-06-18 | 安吉奥斯医药品有限公司 | N-(4-(4-(环丙基甲基)哌嗪-1-羰基)苯基)喹啉-8-磺酰胺的结晶盐形式 |
| US11878049B1 (en) * | 2019-06-14 | 2024-01-23 | Agios Pharmaceuticals, Inc. | Mitapivat therapy and modulators of cytochrome P450 |
| EP4031133A4 (en) | 2019-09-19 | 2023-12-27 | Novo Nordisk Health Care AG | Pyruvate kinase r (pkr) activating compositions |
| WO2022067039A1 (en) | 2020-09-25 | 2022-03-31 | Agios Pharmaceuticals, Inc. | Pharmaceutical formulation |
| US12128035B2 (en) | 2021-03-19 | 2024-10-29 | Novo Nordisk Health Care Ag | Activating pyruvate kinase R |
| WO2022231627A1 (en) | 2021-04-30 | 2022-11-03 | Agios Pharmaceuticals, Inc. | Methods for titrating mitapivat for use in treating thalassemia |
| US20240207254A1 (en) | 2021-04-30 | 2024-06-27 | Agios Pharmaceuticals, Inc. | Methods for titrating mitapivat |
| WO2023154036A1 (en) | 2022-02-08 | 2023-08-17 | Agios Pharmaceuticals, Inc. | Methods for titrating mitapivat |
| MX2024005852A (es) | 2021-11-16 | 2024-05-28 | Agios Pharmaceuticals Inc | Compuestos para tratar anemias asociadas a mds y otras afecciones. |
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