HRP20210207T1 - Uporaba protutijela anti-sklerostin u liječenju osteogenesis imperfecta - Google Patents

Uporaba protutijela anti-sklerostin u liječenju osteogenesis imperfecta Download PDF

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HRP20210207T1
HRP20210207T1 HRP20210207TT HRP20210207T HRP20210207T1 HR P20210207 T1 HRP20210207 T1 HR P20210207T1 HR P20210207T T HRP20210207T T HR P20210207TT HR P20210207 T HRP20210207 T HR P20210207T HR P20210207 T1 HRP20210207 T1 HR P20210207T1
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sclerostin antibody
amino acid
administered
basis
human patient
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HRP20210207TT
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Uwe Junker
Michaela Kneissel
Anthony Kent HALL
Rena Joy EUDY
Matthew Manning RIGGS
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Mereo Biopharma 3 Limited
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    • C07ORGANIC CHEMISTRY
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    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • 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
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/08Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/545Medicinal preparations containing antigens or antibodies characterised by the dose, timing or administration schedule
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/55Medicinal preparations containing antigens or antibodies characterised by the host/recipient, e.g. newborn with maternal antibodies
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/20Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/21Immunoglobulins specific features characterized by taxonomic origin from primates, e.g. man
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/20Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • 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
    • C07K2317/565Complementarity determining region [CDR]
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/70Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/76Antagonist effect on antigen, e.g. neutralization or inhibition of binding
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
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    • C07K2317/00Immunoglobulins specific features
    • C07K2317/90Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/92Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
    • 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)
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Claims (15)

1. Protutijelo anti-sklerostin, naznačeno time, da je za uporabu u liječenju osteogenesis imperfecta (OI) kod ljudskog pacijenta, što uključuje davanje ljudskom pacijentu terapijski učinkovite količine protutijela anti-sklerostin, pri čemu protutijelo anti-sklerostin sadrži: (a) varijabilnu regiju CDR1 teškog lanca, koja sadrži aminokiselinski slijed naveden u SEQ ID NO: 4; (b) varijabilnu regiju CDR2 teškog lanca, koja sadrži aminokiselinski slijed naveden u SEQ ID NO: 15; (c) varijabilnu regiju CDR3 teškog lanca, koja sadrži aminokiselinski slijed naveden u SEQ ID NO: 26; (d) varijabilnu regiju CDR1 lakog lanca, koja sadrži aminokiselinski slijed naveden u SEQ ID NO: 37; (e) varijabilnu regiju CDR2 lakog lanca, koja sadrži aminokiselinski slijed naveden u SEQ ID NO: 48; i (f) varijabilnu regiju CDR3 lakog lanca, koja sadrži aminokiselinski slijed naveden u SEQ ID NO: 59.
2. Protutijelo anti-sklerostin za uporabu prema patentnom zahtjevu 1, naznačeno time, da OI jest OI tipa I, OI tipa III ili OI tipa IV.
3. Protutijelo anti-sklerostin za uporabu prema patentnom zahtjevu 1 ili zahtjevu 2, naznačeno time, da ljudski pacijent ima jednu ili više mutacija u genima COL1A1 i/ili COL1A2.
4. Protutijelo anti-sklerostin za uporabu prema bilo kojem od prethodnih patentnih zahtjeva, naznačeno time, da protutijelo anti-sklerostin sadrži: a) VH polipeptidni slijed koji je najmanje 90-posto istovjetan slijedu u odnosu na aminokiselinske sljedove navedene kao SEQ ID NO: 70; i/ili b) VL polipeptidni slijed koji je najmanje 90-posto istovjetan slijedu u odnosu na aminokiselinske sljedove navedene kao SEQ ID NO: 81.
5. Protutijelo anti-sklerostin za uporabu prema bilo kojem od prethodnih patentnih zahtjeva, naznačeno time, da protutijelo anti-sklerostin sadrži: a) aminokiselinski slijed teškog lanca pune duljine koji je najmanje 90-posto istovjetan slijedu u odnosu na aminokiselinske sljedove navedene kao SEQ ID NO: 172; i/ili b) aminokiselinski slijed lakog lanca pune duljine koji je najmanje 90-posto istovjetan slijedu u odnosu na aminokiselinske sljedove navedene kao SEQ ID NO: 173.
6. Protutijelo anti-sklerostin za uporabu prema bilo kojem od prethodnih patentnih zahtjeva, naznačeno time, da protutijelo anti-sklerostin sadrži VL polipeptidni slijed koji sadrži aminokiselinski slijed naveden kao SEQ ID NO: 81 i VH polipeptidni slijed koji sadrži aminokiselinski slijed naveden kao SEQ ID NO: 70.
7. Protutijelo anti-sklerostin za uporabu prema bilo kojem od prethodnih patentnih zahtjeva, naznačeno time, da protutijelo anti-sklerostin sadrži aminokiselinski slijed lakog lanca pune duljine koji sadrži aminokiselinski slijed naveden kao SEQ ID NO: 173 i aminokiselinski slijed teškog lanca pune duljine koji sadrži aminokiselinski slijed naveden kao SEQ ID NO: 172.
8. Protutijelo anti-sklerostin za uporabu prema bilo kojem od prethodnih patentnih zahtjeva, naznačeno time, da se protutijelo anti-sklerostin daje u dozi od 1 do 50 mg po kilogramu tjelesne težine ljudskog pacijenta.
9. Protutijelo anti-sklerostin za uporabu prema bilo kojem od prethodnih patentnih zahtjeva, naznačeno time, da se protutijelo anti-sklerostin daje: (a) u dozi od 1 do 30 mg po kilogramu tjelesne težine ljudskog pacijenta, primjerice u dozi od 1 do 20 mg po kilogramu tjelesne težine ljudskog pacijenta; ili (b) u dozi od 10 do 30 mg po kilogramu tjelesne težine ljudskog pacijenta; i/ili (c) u dozi od 10 do 5000 mg, primjerice u dozi od 100 do 3000 mg; i/ili (d) ljudskom pacijentu na dnevnoj bazi, tjednoj bazi, dvotjednoj bazi, mjesečnoj bazi, dvomjesečnoj bazi ili kvartalnoj bazi.
10. Protutijelo anti-sklerostin za uporabu prema bilo kojem od prethodnih patentnih zahtjeva, naznačeno time, da program liječenja obuhvaća prvi režim doziranja nakon kojega opcionalno slijedi drugi režim doziranja.
11. Protutijelo anti-sklerostin za uporabu prema patentnom zahtjevu 10, naznačeno time, da: (a) prvi režim doziranja iznosi od 1 do 50 mg po kilogramu tjelesne težine ljudskog pacijenta, koji se primjenjuje na mjesečnoj bazi, primjerice prvi režim doziranja iznosi 20 mg po kilogramu tjelesne težine ljudskog pacijenta, koji se primjenjuje na mjesečnoj bazi; (b) drugi režim doziranja iznosi od 1 do 50 mg po kilogramu tjelesne težine ljudskog pacijenta, koji se primjenjuje na dvomjesečnoj bazi ili na kvartalnoj bazi, primjerice drugi režim doziranja iznosi 20 mg po kilogramu tjelesne težine ljudskog pacijenta, koji se primjenjuje na dvomjesečnoj bazi ili na kvartalnoj bazi; i/ili (c) prvi režim doziranja iznosi 20 mg po kilogramu tjelesne težine ljudskog pacijenta, koji se primjenjuje na mjesečnoj bazi i drugi režim doziranja iznosi 20 mg po kilogramu tjelesne težine ljudskog pacijenta, koji se primjenjuje na dvomjesečnoj ili kvartalnoj bazi, primjerice prvi režim doziranja iznosi 20 mg po kilogramu tjelesne težine ljudskog pacijenta, koji se primjenjuje na mjesečnoj bazi u vremenskom periodu od jedne godine; i drugi režim doziranja iznosi 20 mg po kilogramu tjelesne težine ljudskog pacijenta, koji se primjenjuje na dvomjesečnoj ili kvartalnoj bazi u vremenskom periodu od najmanje jedne godine.
12. Protutijelo anti-sklerostin za uporabu prema patentnom zahtjevu 10, naznačeno time, da: (a) prvi režim doziranja iznosi od 10 do 5000 mg koji se primjenjuje na mjesečnoj bazi; (b) drugi režim doziranja iznosi od 10 do 5000 mg koji se primjenjuje na dvomjesečnoj ili kvartalnoj bazi; i/ili (c) prvi režim doziranja iznosi od 10 do 5000 mg koji se primjenjuje na mjesečnoj bazi i drugi režim doziranja iznosi od 10 do 5000 mg koji se primjenjuje na dvomjesečnoj ili kvartalnoj bazi, primjerice prvi režim doziranja iznosi od 10 do 5000 mg koji se primjenjuje na mjesečnoj bazi u vremenskom periodu od jedne godine, a drugi režim doziranja iznosi od 10 do 5000 mg koji se primjenjuje na dvomjesečnoj ili kvartalnoj bazi u vremenskom periodu od najmanje jedne godine.
13. Protutijelo anti-sklerostin za uporabu prema bilo kojem od prethodnih patentnih zahtjeva, naznačeno time, da se protutijelo anti-sklerostin daje intravenozno.
14. Protutijelo anti-sklerostin za uporabu prema bilo kojem od prethodnih patentnih zahtjeva, naznačeno time, da obuhvaća davanje dodatne terapijske tvari, kao što su bifosfonat, paratiroidni hormon, kalcilitici, kalcimimetici (primjerice cinakalcet), statini, anabolički steroidi, lantanijeve i stroncijeve soli, i/ili natrijev fluorid.
15. Protutijelo anti-sklerostin za uporabu prema bilo kojem od prethodnih patentnih zahtjeva, naznačeno time, da se protutijelo formulira u farmaceutskom pripravku.
HRP20210207TT 2016-12-21 2021-02-05 Uporaba protutijela anti-sklerostin u liječenju osteogenesis imperfecta HRP20210207T1 (hr)

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US201662437353P 2016-12-21 2016-12-21
EP17823190.8A EP3478719B1 (en) 2016-12-21 2017-12-21 Use of anti-sclerostin antibodies in the treatment of osteogenesis imperfecta
PCT/GB2017/053850 WO2018115880A1 (en) 2016-12-21 2017-12-21 Use of anti-sclerostin antibodies in the treatment of osteogenesis imperfecta

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EP (2) EP3478719B1 (hr)
JP (2) JP7050335B2 (hr)
KR (3) KR20190096409A (hr)
CN (2) CN117442720A (hr)
AU (1) AU2017381433A1 (hr)
BR (1) BR112019012731A2 (hr)
CA (1) CA3047221A1 (hr)
CL (1) CL2019001749A1 (hr)
CY (1) CY1124238T1 (hr)
DK (1) DK3478719T3 (hr)
ES (1) ES2862922T3 (hr)
HR (1) HRP20210207T1 (hr)
HU (1) HUE053436T2 (hr)
IL (1) IL267430A (hr)
LT (1) LT3478719T (hr)
MX (1) MX2019007161A (hr)
NZ (1) NZ754676A (hr)
PL (1) PL3478719T3 (hr)
PT (1) PT3478719T (hr)
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WO (1) WO2018115880A1 (hr)

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RU2019118719A (ru) 2021-01-22
JP2020502219A (ja) 2020-01-23
RU2019118719A3 (hr) 2021-06-01
EP3478719B1 (en) 2021-01-20
SI3478719T1 (sl) 2021-09-30
EP3868780A1 (en) 2021-08-25
JP7050335B2 (ja) 2022-04-08
CN110325548B (zh) 2023-11-17
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AU2017381433A1 (en) 2019-07-04
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