WO2024008904A3 - Composés isvd hautement puissants capables de remplacer fviii(a) - Google Patents

Composés isvd hautement puissants capables de remplacer fviii(a) Download PDF

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Publication number
WO2024008904A3
WO2024008904A3 PCT/EP2023/068805 EP2023068805W WO2024008904A3 WO 2024008904 A3 WO2024008904 A3 WO 2024008904A3 EP 2023068805 W EP2023068805 W EP 2023068805W WO 2024008904 A3 WO2024008904 A3 WO 2024008904A3
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WO
WIPO (PCT)
Prior art keywords
isvd
highly potent
fviii
substituting
haemophilia
Prior art date
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PCT/EP2023/068805
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English (en)
Other versions
WO2024008904A2 (fr
Inventor
Andreas VEGGE
Daniele GRANATA
Eva Johansson
Jais Rose Bjelke
Jacob LUND
Philip Jonas Sassene
Per J. GREISEN
Thomas Egebjerg
Frantisek Hubalek
Simone FULLE
Mathias Norrman
Marie-Ange Buyse
Evelyn De Tavernier
Soren Steffensen
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Novo Nordisk A/S
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Application filed by Novo Nordisk A/S filed Critical Novo Nordisk A/S
Publication of WO2024008904A2 publication Critical patent/WO2024008904A2/fr
Publication of WO2024008904A3 publication Critical patent/WO2024008904A3/fr

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/36Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against blood coagulation factors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/185Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19Carboxylic acids, e.g. valproic acid
    • A61K31/20Carboxylic acids, e.g. valproic acid having a carboxyl group bound to a chain of seven or more carbon atoms, e.g. stearic, palmitic, arachidic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/455Nicotinic acids, e.g. niacin; Derivatives thereof, e.g. esters, amides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/395Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/395Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39591Stabilisation, fragmentation
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/505Medicinal preparations containing antigens or antibodies comprising antibodies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/54Medicinal preparations containing antigens or antibodies characterised by the route of administration
    • A61K2039/541Mucosal route
    • A61K2039/542Mucosal route oral/gastrointestinal
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/20Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/22Immunoglobulins specific features characterized by taxonomic origin from camelids, e.g. camel, llama or dromedary
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/30Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/31Immunoglobulins specific features characterized by aspects of specificity or valency multispecific
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/40Immunoglobulins specific features characterized by post-translational modification
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/50Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/56Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/50Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/56Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
    • C07K2317/569Single domain, e.g. dAb, sdAb, VHH, VNAR or nanobody®
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/90Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/94Stability, e.g. half-life, pH, temperature or enzyme-resistance

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Organic Chemistry (AREA)
  • Microbiology (AREA)
  • Mycology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La présente invention concerne des dérivés de polypeptides ISVD capables de se lier au facteur IX(a) et au facteur X(a) de coagulation qui sont très puissants et fournissent une demi-vie suffisamment longue pour permettre une administration sous-cutanée et perorale efficace. Les dérivés de polypeptides ISVD de l'invention sont ainsi appropriés pour le traitement de l'hémophilie A, de l'hémophilie A avec des inhibiteurs et de l'hémophilie A acquise par diverses voies d'administration comprenant une administration sous-cutanée et perorale.
PCT/EP2023/068805 2022-07-08 2023-07-07 Composés isvd hautement puissants capables de remplacer fviii(a) WO2024008904A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP22183825 2022-07-08
EP22183825.3 2022-07-08

Publications (2)

Publication Number Publication Date
WO2024008904A2 WO2024008904A2 (fr) 2024-01-11
WO2024008904A3 true WO2024008904A3 (fr) 2024-02-22

Family

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PCT/EP2023/068805 WO2024008904A2 (fr) 2022-07-08 2023-07-07 Composés isvd hautement puissants capables de remplacer fviii(a)

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Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7459354B2 (ja) * 2022-07-08 2024-04-01 ノヴォ ノルディスク アー/エス FVIII(a)の代わりとなることができる非常に効力があるISVD化合物

Citations (6)

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US20180140673A1 (en) * 2014-12-23 2018-05-24 Novo Nordisk A/S FGF21 Derivatives and Uses Thereof
WO2019096874A1 (fr) * 2017-11-15 2019-05-23 Novo Nordisk A/S Liants de facteur x renforçant l'activation fx
CN110343181A (zh) * 2018-04-08 2019-10-18 苏州康宁杰瑞生物科技有限公司 针对凝血因子ix(fix)的单域抗体
WO2019215063A1 (fr) * 2018-05-07 2019-11-14 Novo Nordisk A/S Compositions solides comprenant un agoniste de glp-1 et un sel d'acide n-(8-(2-hydroxybenzoyl)amino)caprylique
US20210284715A1 (en) * 2017-02-06 2021-09-16 Applied Stemcell, Inc. Coagulation factor viii mimetic protein and uses thereof
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EP2192131A1 (fr) 1992-08-21 2010-06-02 Vrije Universiteit Brussel Immunoglobulines dépourvus de chaînes légères
DK0937140T3 (da) 1996-06-27 2008-01-28 Vlaams Interuniv Inst Biotech Antistofmolekyler, som interagerer specifikt med et målmolekyles aktive sted eller klöft
CA2390691C (fr) 1999-12-24 2016-05-10 Genentech, Inc. Methodes et compositions permettant de prolonger les demi-vies d'elimination de composes bioactifs
ES2352697T3 (es) 2003-11-07 2011-02-22 Ablynx N.V. Anticuerpos de dominio único de camelidae vhh dirigidos contra el receptor del factor de crecimiento epidérmico y usos de los mismos.
JP2008511286A (ja) 2004-06-02 2008-04-17 ダイアテック・ピーティワイ・リミテッド サメ類のIgNARドメインに基づく結合成分
WO2007121318A2 (fr) 2006-04-12 2007-10-25 Emisphere Technologies, Inc. Préparations destinées à l'administration d'insuline
RU2620071C2 (ru) 2010-11-17 2017-05-22 Чугаи Сеияку Кабушики Каиша Мультиспецифическая связывающая антиген молекула, которая обладает альтернативной функцией к функции фактора свертывания крови viii
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UA128389C2 (uk) 2017-09-29 2024-07-03 Чугаі Сейяку Кабусікі Кайся Мультиспецифічна антигензв'язувальна молекула, яка має активність заміщувати кофакторну функцію фактора коагуляції крові viii (fviii), та фармацевтичний склад, який містить згадану молекулу як активний інгредієнт
PE20211399A1 (es) 2018-08-01 2021-07-27 Novo Nordisk As Anticuerpos procoagulantes mejorados
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US20210284715A1 (en) * 2017-02-06 2021-09-16 Applied Stemcell, Inc. Coagulation factor viii mimetic protein and uses thereof
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