CA3102105A1 - Compositions and methods for increasing tetrahydrobiopterin plasma exposure - Google Patents

Compositions and methods for increasing tetrahydrobiopterin plasma exposure Download PDF

Info

Publication number
CA3102105A1
CA3102105A1 CA3102105A CA3102105A CA3102105A1 CA 3102105 A1 CA3102105 A1 CA 3102105A1 CA 3102105 A CA3102105 A CA 3102105A CA 3102105 A CA3102105 A CA 3102105A CA 3102105 A1 CA3102105 A1 CA 3102105A1
Authority
CA
Canada
Prior art keywords
sepiapterin
subject
food
administration
effective amount
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.)
Pending
Application number
CA3102105A
Other languages
English (en)
French (fr)
Inventor
Neil Smith
Jonathan Reis
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
PTC Therapeutics MP Inc
Original Assignee
PTC Therapeutics MP Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by PTC Therapeutics MP Inc filed Critical PTC Therapeutics MP Inc
Publication of CA3102105A1 publication Critical patent/CA3102105A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • A61K9/0065—Forms with gastric retention, e.g. floating on gastric juice, adhering to gastric mucosa, expanding to prevent passage through the pylorus

Landscapes

  • Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Epidemiology (AREA)
  • Physiology (AREA)
  • Nutrition Science (AREA)
  • Obesity (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
CA3102105A 2018-05-30 2019-05-30 Compositions and methods for increasing tetrahydrobiopterin plasma exposure Pending CA3102105A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862677926P 2018-05-30 2018-05-30
US62/677,926 2018-05-30
US201862771397P 2018-11-26 2018-11-26
US62/771,397 2018-11-26
US201962822365P 2019-03-22 2019-03-22
US62/822,365 2019-03-22
GC201937662 2019-05-28
GC2019/37662 2019-05-28
PCT/US2019/034515 WO2019232126A1 (en) 2018-05-30 2019-05-30 Compositions and methods for increasing tetrahydrobiopterin plasma exposure

Publications (1)

Publication Number Publication Date
CA3102105A1 true CA3102105A1 (en) 2019-12-05

Family

ID=68699016

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3102105A Pending CA3102105A1 (en) 2018-05-30 2019-05-30 Compositions and methods for increasing tetrahydrobiopterin plasma exposure

Country Status (10)

Country Link
US (2) US12257252B2 (enExample)
EP (1) EP3801534B1 (enExample)
JP (1) JP7547215B2 (enExample)
CN (1) CN112654356A (enExample)
BR (1) BR112020024329A2 (enExample)
CA (1) CA3102105A1 (enExample)
DK (1) DK3801534T3 (enExample)
FI (1) FI3801534T3 (enExample)
MX (1) MX2020012980A (enExample)
WO (1) WO2019232126A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA46973A (fr) 2016-11-29 2019-10-09 Censa Pharmaceuticals Inc Polymorphes de sépiaptérine et leurs sels
MA46972A (fr) 2016-11-29 2019-10-09 Censa Pharmaceuticals Inc Forme polymorphe de sépiaptérine
JP7811085B2 (ja) 2017-09-01 2026-02-04 ピーティーシー セラピューティクス エムピー，インコーポレイテッド セピアプテリンを含む医薬組成物及びその使用
FI3801536T3 (fi) * 2018-05-30 2024-08-29 Ptc Therapeutics Mp Inc Sepiapteriinin antaminen ilman ruokaa käytettäväksi menetelmässä sepiapteriinin plasma-altistuksen lisäämiseksi
EP3801534B1 (en) 2018-05-30 2026-02-18 PTC Therapeutics MP, Inc. Treatment of phenylketonuria with sepiapterin
EP4093405A1 (en) * 2020-01-24 2022-11-30 PTC Therapeutics MP, Inc. Methods for treating parkinson's disease with sepiapterin

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5925323A (ja) 1982-03-03 1984-02-09 鐘淵化学工業株式会社 プテリン誘導体からなる脳内神経伝達物質の関わる疾病の治療剤
US5910304A (en) 1982-12-13 1999-06-08 Texas A&M University System Low-dose oral administration of interferons
CA1320905C (en) 1986-11-06 1993-08-03 Joseph M. Cummins Treatment of immuno-resistant disease
JP2002515965A (ja) 1995-08-18 2002-05-28 ダブリュー． ランドリ，ドナルド 抗体触媒反応の調節による有機化合物の検出
US6036949A (en) 1998-03-05 2000-03-14 Amarillo Biosciences, Inc. Treatment of fibromyalgia with low doses of interferon
US8188043B2 (en) 1999-07-28 2012-05-29 The Board Of Trustees Of The Leland Stanford Jr. University Nicotine in therapeutic angiogenesis and vasculogenesis
FR2859996B1 (fr) 2003-09-19 2006-02-03 Aventis Pharma Sa Solvat acetonique du dimethoxy docetaxel et son procede de preparation
WO2005048926A2 (en) 2003-11-13 2005-06-02 The General Hospital Corporation Methods for treating pain
PL1708690T3 (pl) 2003-11-17 2017-01-31 Biomarin Pharmaceutical Inc. Leczenie fenyloketonurii za pomocą BH4
JP5184783B2 (ja) 2003-11-17 2013-04-17 バイオマリン ファーマシューティカル インコーポレイテッド テトラヒドロビオプテリン、及びテトラヒドロビオプテリン類似体の製造方法
EP1687308B1 (en) 2003-11-17 2019-06-12 Merck & Cie Process for preparating (6R)-L-erythrotetrahydrobiopterin hydrochloride crystalline form B from other crystalline forms
KR20070084270A (ko) 2004-11-17 2007-08-24 바이오마린 파머수티컬 인크. 안정한 정제 제형물
JP2008523090A (ja) 2004-12-08 2008-07-03 バイオマリン ファーマシューティカル インコーポレイテッド 新生児肺高血圧症の治療のための方法および組成物
BRPI0609421B1 (pt) 2005-04-28 2021-06-01 Shiratori Pharmaceutical Co., Ltd. Processos para produção de um composto 1,1-bis-alquil sulfonila, para produção de um composto aldeído, e para produção de um derivado de hidrazona
US20080075666A1 (en) 2006-08-25 2008-03-27 Dudley Samuel C Jr Methods and compositions for treating diastolic dysfunction
LT2545939T (lt) 2007-04-11 2021-03-10 Biomarin Pharmaceutical Inc. Tetrahidrobiopterinas, skirtas gydyti ligas, susijusias supadidėjusiu fenilalanino kiekiu
WO2010017570A2 (de) 2008-08-12 2010-02-18 Orpha Swiss Gmbh Pharmazeutische darreichungsform enthaltend tetrahydrobiopterin
EP2562176B1 (en) 2010-04-22 2018-08-15 Nihon University Medicinal agent and beverage/food for preventing cerebral dysfunction and improving same
US9884909B2 (en) 2010-12-01 2018-02-06 Alderbio Holdings Llc Anti-NGF compositions and use thereof
CN103458900A (zh) * 2011-03-01 2013-12-18 鲁必康研究私人有限公司 四氢生物蝶呤的稳定组合物
US9216178B2 (en) * 2011-11-02 2015-12-22 Biomarin Pharmaceutical Inc. Dry blend formulation of tetrahydrobiopterin
CN104736539B (zh) 2012-05-07 2017-09-01 白鸟制药株式会社 墨蝶呤和四氢乳酰蝶呤的制造法
EP3213062B1 (en) 2014-10-31 2020-02-12 Children's Medical Center Corporation Methods and assays relating to sepiapterin reductase inhibition
MA46972A (fr) 2016-11-29 2019-10-09 Censa Pharmaceuticals Inc Forme polymorphe de sépiaptérine
MA46973A (fr) 2016-11-29 2019-10-09 Censa Pharmaceuticals Inc Polymorphes de sépiaptérine et leurs sels
JP7811085B2 (ja) * 2017-09-01 2026-02-04 ピーティーシー セラピューティクス エムピー，インコーポレイテッド セピアプテリンを含む医薬組成物及びその使用
EP3801534B1 (en) 2018-05-30 2026-02-18 PTC Therapeutics MP, Inc. Treatment of phenylketonuria with sepiapterin
FI3801536T3 (fi) 2018-05-30 2024-08-29 Ptc Therapeutics Mp Inc Sepiapteriinin antaminen ilman ruokaa käytettäväksi menetelmässä sepiapteriinin plasma-altistuksen lisäämiseksi
AU2019277372B2 (en) * 2018-05-30 2024-12-19 Ptc Therapeutics Mp, Inc. Pharmaceutically acceptable salts of sepiapterin
IL290321B1 (en) 2019-08-05 2026-04-01 Ptc Therapeutics Inc Use of spiapterin and its metabolites for radiation exposure treatment
IL291565B1 (en) * 2019-09-25 2026-04-01 Ptc Therapeutics Inc Method for treating phenylalanine irregularity

Also Published As

Publication number Publication date
WO2019232126A1 (en) 2019-12-05
JP7547215B2 (ja) 2024-09-09
US12257252B2 (en) 2025-03-25
EP3801534B1 (en) 2026-02-18
FI3801534T3 (fi) 2026-04-14
EP3801534A4 (en) 2022-03-16
EP3801534A1 (en) 2021-04-14
JP2024178188A (ja) 2024-12-24
US20250195529A1 (en) 2025-06-19
DK3801534T3 (da) 2026-03-30
US20210161901A1 (en) 2021-06-03
MX2020012980A (es) 2021-04-29
CN112654356A (zh) 2021-04-13
BR112020024329A2 (pt) 2021-02-23
JP2021525746A (ja) 2021-09-27

Similar Documents

Publication Publication Date Title
US12329757B2 (en) Methods for increasing sepiapterin plasma exposure
US20250195529A1 (en) Compositions and methods for increasing tetrahydrobiopterin plasma exposure
US12213982B2 (en) Pharmaceutical compositions comprising sepiapterin and uses thereof
KR20170037676A (ko) 테트라하이드로바이오프테린 투여 방법, 관련 조성물 및 측정 방법
KR20210038848A (ko) 세피아프테린의 제약상 허용되는 염
JP7855033B2 (ja) テトラヒドロビオプテリン血漿曝露を増加させるための組成物及び方法
HK40119071A (en) Methods for increasing sepiapterin plasma exposure
HK40051961A (en) Administration of sepiapterin without food for use in a method for increasing sepiapterin plasma exposure
HK40051961B (en) Administration of sepiapterin without food for use in a method for increasing sepiapterin plasma exposure
US20240122931A1 (en) Methods for treating covid-19 with sepiapterin
BR112020024346A2 (pt) métodos para aumentar a exposição de sepiapterina no plasma
CA3207811A1 (en) Methods for treating glioblastomas with sepiapterin

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220928

B12 Application deemed to be withdrawn, abandoned or lapsed

Free format text: ST27 STATUS EVENT CODE: N-2-6-B10-B12-B303 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO AN EXAMINER'S REQUISITION

Effective date: 20240729

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 6TH ANNIV.) - STANDARD

Year of fee payment: 6

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250424

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250424

C11 Application revival requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-C10-C11-C106 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REINSTATEMENT REQUEST RECEIVED

Effective date: 20250728

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20250728

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250731

C13 Application revived following an application revival request

Free format text: ST27 STATUS EVENT CODE: A-6-2-C10-C13-C102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REINSTATEMENT REQUIREMENTS DEEMED COMPLIANT FOR ALL ABANDONMENT REASONS

Effective date: 20260223

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: N-6-6-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20260223

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20260225

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20260312

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20260312

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 7TH ANNIV.) - STANDARD

Year of fee payment: 7

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20260423

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20260423