CA2913785C - Compounds for treating spinal muscular atrophy - Google Patents
Compounds for treating spinal muscular atrophy Download PDFInfo
- Publication number
- CA2913785C CA2913785C CA2913785A CA2913785A CA2913785C CA 2913785 C CA2913785 C CA 2913785C CA 2913785 A CA2913785 A CA 2913785A CA 2913785 A CA2913785 A CA 2913785A CA 2913785 C CA2913785 C CA 2913785C
- Authority
- CA
- Canada
- Prior art keywords
- fluoro
- pyridin
- methylimidazo
- methylpiperazin
- rac
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D471/00—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 C07D451/00 - C07D463/00
- C07D471/02—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 C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
- C07D471/04—Ortho-condensed systems
-
- 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
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
- A61K31/437—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
-
- 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
- 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
- A61K31/4985—Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D263/00—Heterocyclic compounds containing 1,3-oxazole or hydrogenated 1,3-oxazole rings
- C07D263/52—Heterocyclic compounds containing 1,3-oxazole or hydrogenated 1,3-oxazole rings condensed with carbocyclic rings or ring systems
- C07D263/54—Benzoxazoles; Hydrogenated benzoxazoles
- C07D263/56—Benzoxazoles; Hydrogenated benzoxazoles with only hydrogen atoms, hydrocarbon or substituted hydrocarbon radicals, directly attached in position 2
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
- C07D413/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings
- C07D413/12—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings linked by a chain containing hetero atoms as chain links
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
- C07D413/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
- C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
- C07D487/04—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
- C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
- C07D487/10—Spiro-condensed systems
Landscapes
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Life Sciences & Earth Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Epidemiology (AREA)
- Hospice & Palliative Care (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Psychiatry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
- Plural Heterocyclic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Low-Molecular Organic Synthesis Reactions Using Catalysts (AREA)
- Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
- Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP13173487.3 | 2013-06-25 | ||
| EP13173487 | 2013-06-25 | ||
| PCT/US2014/043577 WO2014209841A2 (en) | 2013-06-25 | 2014-06-23 | Compounds for treating spinal muscular atrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2913785A1 CA2913785A1 (en) | 2014-12-31 |
| CA2913785C true CA2913785C (en) | 2021-08-31 |
Family
ID=48669815
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2913785A Active CA2913785C (en) | 2013-06-25 | 2014-06-23 | Compounds for treating spinal muscular atrophy |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US20160145270A1 (enExample) |
| EP (1) | EP3013345B1 (enExample) |
| JP (1) | JP6492071B2 (enExample) |
| KR (1) | KR102314290B1 (enExample) |
| CN (1) | CN105358151B (enExample) |
| AR (1) | AR096684A1 (enExample) |
| BR (1) | BR112015030288B1 (enExample) |
| CA (1) | CA2913785C (enExample) |
| MX (1) | MX363456B (enExample) |
| RU (1) | RU2673542C2 (enExample) |
| TW (1) | TW201542543A (enExample) |
| WO (1) | WO2014209841A2 (enExample) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN105392790B (zh) | 2013-08-19 | 2019-04-19 | 豪夫迈·罗氏有限公司 | 4H-吡啶并[1,2-a]嘧啶-4-酮化合物制备用于预防或治疗癌症的药物的用途 |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| RU2725979C2 (ru) * | 2014-06-25 | 2020-07-08 | Ф. Хоффманн-Ля Рош Аг | ИМИДАЗО[1,2-а]ПИРАЗИН-1-ИЛ-БЕНЗАМИДЫ ДЛЯ ЛЕЧЕНИЯ СПИНАЛЬНОЙ МЫШЕЧНОЙ АТРОФИИ |
| AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| WO2016161572A1 (en) * | 2015-04-08 | 2016-10-13 | Merck Sharp & Dohme Corp. | TrkA KINASE INHIBITORS, COMPOSITIONS AND METHODS THEREOF |
| EP3359685A1 (en) | 2015-10-09 | 2018-08-15 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| KR102162062B1 (ko) | 2015-11-12 | 2020-10-07 | 에프. 호프만-라 로슈 아게 | 척수성 근위축증의 치료용 조성물 |
| CN113750101A (zh) | 2015-12-10 | 2021-12-07 | Ptc医疗公司 | 用于治疗亨廷顿病的方法 |
| EP3386978B1 (en) | 2015-12-10 | 2021-01-27 | H. Hoffnabb-La Roche Ag | Bridged piperidine derivatives |
| ES2882500T3 (es) | 2015-12-14 | 2021-12-02 | Cold Spring Harbor Laboratory | Oligómeros antisentido para el tratamiento del síndrome de Dravet |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| US11407753B2 (en) | 2017-06-05 | 2022-08-09 | Ptc Therapeutics, Inc. | Compounds for treating Huntington's disease |
| CA3067591A1 (en) | 2017-06-28 | 2019-01-03 | Ptc Therapeutics, Inc. | Methods for treating huntington's disease |
| EP3645121B8 (en) | 2017-06-28 | 2025-06-18 | PTC Therapeutics, Inc. | Methods for treating huntington's disease |
| SG10202002990XA (en) | 2017-08-04 | 2020-05-28 | Skyhawk Therapeutics Inc | Methods and compositions for modulating splicing |
| RS65031B1 (sr) | 2017-08-25 | 2024-02-29 | Stoke Therapeutics Inc | Antisens oligomeri za lečenje stanja i bolesti |
| PL3700570T3 (pl) | 2017-10-23 | 2025-05-05 | Stoke Therapeutics, Inc. | Oligomery antysensowne w leczeniu schorzeń oraz chorób wynikających z degradacji rna zgodnie z nmd |
| KR20210005559A (ko) | 2018-03-27 | 2021-01-14 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 화합물 |
| JP2021523227A (ja) | 2018-05-04 | 2021-09-02 | ストーク セラピューティクス，インク． | コレステリルエステル蓄積症の処置のための方法及び組成物 |
| WO2020005877A1 (en) | 2018-06-27 | 2020-01-02 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating huntington's disease |
| CN112469707B (zh) | 2018-06-27 | 2024-06-21 | 利伯纳生物科学株式会社 | 脊髓性肌萎缩症的预防剂或治疗剂 |
| LT3814357T (lt) | 2018-06-27 | 2024-06-25 | Ptc Therapeutics, Inc. | Heterocikliniai ir heteroarilų junginiai, skirti hantingtono ligos gydymui |
| US11685746B2 (en) | 2018-06-27 | 2023-06-27 | Ptc Therapeutics, Inc. | Heteroaryl compounds for treating Huntington's disease |
| MA54076A (fr) | 2018-11-02 | 2022-02-09 | Merck Sharp & Dohme | 2-amino-n-hétéroaryl-nicotinamides utilisés en tant qu'inhibiteurs de nav1.8 |
| US20220089592A1 (en) * | 2019-01-18 | 2022-03-24 | Biogen Ma Inc. | Imidazo[1,2-a]pyridinyl derivatives as irak4 inhibitors |
| KR20210135241A (ko) | 2019-02-05 | 2021-11-12 | 스카이호크 테라퓨틱스, 인코포레이티드 | 스플라이싱을 조절하는 방법 및 조성물 |
| KR20210135507A (ko) | 2019-02-06 | 2021-11-15 | 스카이호크 테라퓨틱스, 인코포레이티드 | 스플라이싱을 조절하는 방법 및 조성물 |
| MX2021010199A (es) * | 2019-02-25 | 2021-09-21 | Beijing Innocare Pharma Tech Co Ltd | Proceso para la preparacion de 6-(1-acriloilpiperidin-4-il)-2-(4-f enoxifenil)nicotinamida. |
| WO2021007378A1 (en) | 2019-07-11 | 2021-01-14 | Ptc Therapeutics, Inc. | Compounds for use in treating huntington's disease |
| EP4110464A1 (en) * | 2020-02-28 | 2023-01-04 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| AU2021270720A1 (en) | 2020-05-11 | 2022-12-08 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
| JP7720864B2 (ja) | 2020-05-13 | 2025-08-08 | シーエイチディーアイ ファウンデーション，インコーポレーテッド | ハンチントン病を処置するためのｈｔｔモジュレータ |
| WO2022169230A1 (ko) * | 2021-02-03 | 2022-08-11 | 단국대학교 천안캠퍼스 산학협력단 | 6-메톡시니코틴아미드를 유효성분으로 포함하는 근감소증의 예방 또는 치료용 조성물 |
| IL312891A (en) | 2021-11-17 | 2024-07-01 | Chdi Foundation Inc | Htt modulators for treating huntington's disease |
| EP4441042A1 (en) * | 2021-12-03 | 2024-10-09 | Rgenta Therapeutics, Inc. | 5-pyrimidinecarboxamide derivatives and methods of using the same |
| WO2023196972A1 (en) * | 2022-04-08 | 2023-10-12 | John Phillip Adams | Improving brain function |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SE9801526D0 (sv) * | 1998-04-29 | 1998-04-29 | Astra Ab | New compounds |
| ES2359836T3 (es) * | 2004-04-23 | 2011-05-27 | Bristol-Myers Squibb Company | Heterociclos monociclicos como inhibidores de quinasa. |
| WO2006101455A1 (en) | 2005-03-21 | 2006-09-28 | S*Bio Pte Ltd | Imidazo[1,2-a]pyridine derivatives: preparation and pharmaceutical applications |
| US7666880B2 (en) * | 2005-03-21 | 2010-02-23 | S*Bio Pte Ltd. | Imidazo[1,2-A]pyridine derivatives: preparation and pharmaceutical applications |
| CA2626898A1 (en) * | 2005-11-03 | 2007-05-18 | Christopher Hamblett | Substituted nicotinamide compounds |
| WO2009042907A1 (en) * | 2007-09-27 | 2009-04-02 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isoindoline compounds for the treatment of spinal muscular atrophy and other uses |
| US8633019B2 (en) * | 2008-05-27 | 2014-01-21 | Ptc Therapeutics, Inc. | Methods for treating spinal muscular atrophy |
| ATE552255T1 (de) * | 2008-06-05 | 2012-04-15 | Glaxo Group Ltd | 4-aminoindazole |
| US8986935B2 (en) * | 2008-08-13 | 2015-03-24 | Ptc Therapeutics, Inc. | Methods for treating spinal muscular atrophy |
| UY32138A (es) * | 2008-09-25 | 2010-04-30 | Boehringer Ingelheim Int | Amidas sustituidas del ácido 2-(2,6-dicloro-fenilamino)-6-fluoro-1-metil-1h-bencimidazol-5-carboxílico y sus sales farmacéuticamente aceptables |
| WO2012142459A1 (en) * | 2011-04-14 | 2012-10-18 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isoindoline compounds for the treatment of spinal muscular atrophy and other uses |
-
2014
- 2014-06-23 EP EP14816651.5A patent/EP3013345B1/en active Active
- 2014-06-23 WO PCT/US2014/043577 patent/WO2014209841A2/en not_active Ceased
- 2014-06-23 JP JP2016523822A patent/JP6492071B2/ja active Active
- 2014-06-23 CA CA2913785A patent/CA2913785C/en active Active
- 2014-06-23 US US14/899,397 patent/US20160145270A1/en not_active Abandoned
- 2014-06-23 KR KR1020157036344A patent/KR102314290B1/ko active Active
- 2014-06-23 BR BR112015030288-2A patent/BR112015030288B1/pt active IP Right Grant
- 2014-06-23 RU RU2015154676A patent/RU2673542C2/ru active
- 2014-06-23 MX MX2015017345A patent/MX363456B/es unknown
- 2014-06-23 AR ARP140102349A patent/AR096684A1/es unknown
- 2014-06-23 CN CN201480035676.2A patent/CN105358151B/zh active Active
- 2014-06-24 TW TW103121679A patent/TW201542543A/zh unknown
-
2018
- 2018-04-09 US US15/948,444 patent/US10501482B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| WO2014209841A2 (en) | 2014-12-31 |
| MX2015017345A (es) | 2016-08-03 |
| MX363456B (es) | 2019-03-25 |
| US20160145270A1 (en) | 2016-05-26 |
| KR20160022312A (ko) | 2016-02-29 |
| CN105358151B (zh) | 2019-04-12 |
| KR102314290B1 (ko) | 2021-10-21 |
| JP2016530231A (ja) | 2016-09-29 |
| US10501482B2 (en) | 2019-12-10 |
| BR112015030288A2 (enExample) | 2017-08-22 |
| CA2913785A1 (en) | 2014-12-31 |
| AR096684A1 (es) | 2016-01-27 |
| RU2673542C2 (ru) | 2018-11-28 |
| TW201542543A (zh) | 2015-11-16 |
| US20180222925A1 (en) | 2018-08-09 |
| CN105358151A (zh) | 2016-02-24 |
| JP6492071B2 (ja) | 2019-03-27 |
| HK1215399A1 (zh) | 2016-08-26 |
| WO2014209841A3 (en) | 2015-10-29 |
| RU2015154676A (ru) | 2017-07-28 |
| RU2015154676A3 (enExample) | 2018-03-15 |
| BR112015030288B1 (pt) | 2022-10-18 |
| EP3013345A4 (en) | 2016-11-30 |
| EP3013345B1 (en) | 2017-11-22 |
| EP3013345A2 (en) | 2016-05-04 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20190620 |