HRP20211561T1 - Modulacija aktivnosti komplementa - Google Patents

Modulacija aktivnosti komplementa Download PDF

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Publication number
HRP20211561T1
HRP20211561T1 HRP20211561TT HRP20211561T HRP20211561T1 HR P20211561 T1 HRP20211561 T1 HR P20211561T1 HR P20211561T T HRP20211561T T HR P20211561TT HR P20211561 T HRP20211561 T HR P20211561T HR P20211561 T1 HRP20211561 T1 HR P20211561T1
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Croatia
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group
absent
polypeptide
acid
methyl
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HRP20211561TT
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English (en)
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Michelle Denise HOARTY
Ketki Ashok DHAMNASKAR
Daniel Elbaum
Kristopher JOSEPHSON
Kelley Cronin LARSON
Zhong MA
Nathan Ezekiel Nims
Alonso RICARDO
Kathleen Seyb
Guo-Qing Tang
Douglas A. Treco
Zhaolin Wang
Ping Ye
Hong Zheng
Sarah Jacqueline PERLMUTTER
Robert Paul Hammer
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Ra Pharmaceuticals, Inc.
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Application filed by Ra Pharmaceuticals, Inc. filed Critical Ra Pharmaceuticals, Inc.
Publication of HRP20211561T1 publication Critical patent/HRP20211561T1/hr
Publication of HRP20211561T8 publication Critical patent/HRP20211561T8/hr

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/04Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/08Peptides having 5 to 11 amino acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/04Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/10Peptides having 12 to 20 amino acids
    • 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
    • A61K47/62Medicinal 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 a protein, peptide or polyamino acid
    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • A61K47/62Medicinal 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 a protein, peptide or polyamino acid
    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/02Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/02Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/472Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04Linear peptides containing only normal peptide links
    • C07K7/06Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04Linear peptides containing only normal peptide links
    • C07K7/08Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/64Cyclic peptides containing only normal peptide links
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Claims (15)

1. Polipeptid s formulom R1-Xaa0-Xaa1-Xaa2-Xaa3-Xaa4-Xaa5-Xaa6-Xaa7-Xaa8-Xaa9-Xaa10-Xaa11-Xaa12-Xaa13-Xaa14-Xaa15-Xaa16-Xaa17-Xaa18-R2, naznačen time što: (a) R1 je odabran iz skupine koju čine H, acetilna skupina, acilna skupina koja sadrži linearni ili razgranati, zasićeni ili nezasićeni lanac ugljikovodika od 1 do 20 ugljikovih atoma, skupinu heptanoil, amid, karbamat, urea, PEG, hidroksialkil škrob i polipeptid; (b) Xaa0 je odsutan, ili aminokiselina odabrana iz skupine koju čine Met i norvalin; (c) Xaa1 je odsutan ili je odabran iz skupine koju čine (S)-2-amino-5-azidopentanska kiselina, klorooctena kiselina, (S)-2-aminohept-6-enojska kiselina, 4-aminomaslačna kiselina, 5-aminopentanska kiselina, 5-aminoheksanska kiselina, ornitin, Lys, homolizin, Glu, Asp, 3-tiopropionska kiselina i Cys; (d) Xaa2 je odsutan ili je odabran iz skupine koju čine Ala, D-Ala, tertbutil-glicin, Lys, Ser, Cys i Val; (e) Xaa3 je odsutan ili je odabran iz skupine koju čine Ala, norvalin, Val i Glu; (f) Xaa4 je odsutan ili je odabran iz skupine koju čine Ala, Cys, Arg, Ser, Glu, fenilglicin i norvalin; (g) Xaa5 je odsutan ili je odabran iz skupine koju čine Tyr, Arg, Cys, Phe, N-metil-tirozin i Ala; (h) Xaa6 je odsutan ili je odabran iz skupine koju čine Glu, N-metil-glutaminska kiselina, cikloheksilglicin, Lys, Tyr, Pro, N-metil-serin, tertbutil-glicin, Val, norleucin, norvalin, 7-azatriptofan, Asn, Asp, (S)-2-aminopent-4-inska kiselina, (S)-2-aminopent-4-enska kiselina, Cys i Ala; (i) Xaa7 je odsutan ili je odabran iz skupine koju čine Asn, N-metil-asparagin, N-metil-glicin, N-metil-serin, homocistein, Thr, Tyr, fenilglicin, tert-butilglicin, alfa-metil L-asparaginska kiselina, (S)-2-amino-3-(1H-tetrazol-5-il)propanska kiselina, N-metilasparaginska kiselina, cikloleucin, 4-amino-tetrahidro-piran-4-karboksilna kiselina, Arg, Glu, Asp, Cys i Ala; (j) Xaa8 je tert-butilglicin; (k) Xaa9 je Tyr; (l) Xaa10 je 7-azatriptofan ili Trp; (m) Xaa11 je Glu; (n) Xaal2 je Tyr; (o) Xaal3 je odsutan ili je odabran iz skupine koju čine propargil-glicin, Pro, Ala, N-metil-glicin, Ser, N-metil-serin, N-metil-alanin, norvalin, Cys i Tbg; (p) Xaal4 je odsutan ili je odabran iz skupine koju čine amino izomaslačna kiselina, tert-butilglicin, Cys, Pro, Asn, fenilglicin, D-fenilglicin, N-metil-fenilglicin, norvalin, His, Ala, D-Ala, i cikloheksilglicin; (q) Xaal15 je odsutan ili je odabran iz skupine koju čine norvalin, Lys, N-ε-kaprilni lizin, N-ε-kapril lizin, N-ε-lauril lizin, N-ε-palmitoil lizin, Pro, Cys, Tyr, Gly, propargil-glicin, homoCys, N-metil-serin i tert-butilglicin; (r) Xaa16 je odsutan ili je odabran iz skupine koju čine norvalin, Cys, Lys i Ala; (s) Xaa17 je odsutan ili je odabran iz skupine koju čine Pro, Glu i Nvl; (t) Xaa18 je odsutan ili je norvalin; i (u) R2 je odsutan ili je odabran iz skupine koju čine B20, B28, K14, -NH2 i -N(CH3)2; pri čemu je polipeptid inhibitor C5; i veličina polipeptida je u rasponu od 11 do 50 aminokiselina.
2. Polipeptid prema patentnom zahtjevu 1, naznačen time što Xaa10 je 7-azatriptofan.
3. Polipeptid prema patentnom zahtjevu 1 ili 2, naznačen time što: (i) Xaa1 je Cys ili odsutan; (ii) Xaa2 je odsutan ili je odabran iz skupine koju čine tertbutil-glicin i Val; (iii) Xaa3 je odsutan ili je odabran iz skupine koju čine Glu i Nvl; (iv) Xaa4 je odsutan ili je odabran iz skupine koju čine Arg, Cys i Ser; (v) Xaa5 je Phe ili Tyr; (vi) Xaa6 je Cys ili Glu; i/ili (vii) Xaa7 je Asp ili Asn.
4. Polipeptid prema bilo kojem od patentnih zahtjeva 1-3, naznačen time što: (i) Xaal3 je odsutan ili je odabran iz skupine koju čine Pro, N-metil-glicin i Ala; (ii) Xaal4 je odsutan ili je odabran iz skupine koju čine cikloheksilglicin i norvalin; i/ili (iii) Xaa15 je odsutan ili je odabran iz skupine koju čine norvalin i N-metil-serin.
5. Polipeptid prema patentnom zahtjevu 1, naznačen time što sadrži premošćujući dio između dvije aminokiseline.
6. Polipeptid prema patentnom zahtjevu 5, naznačen time što premošćujući dio sadrži strukturu odabranu iz skupine koju čine strukture I-XIX; [image] pri čemu je svaki X neovisno N ili CH, tako da nijedan prsten ne sadrži više od 2 N; svaki Z je neovisno veza, NR, O, S, CH2, C(O)NR, NRC(O), S(O)vNR, NRS(O)v; svaki m je neovisno odabran od 0, 1, 2, i 3; svaki v je neovisno odabran od 1 i 2; svaki R je neovisno odabran od H i C1-C6; a svaki premošćujući dio je povezan s polipeptidom pomoću neovisno odabranih C0-C6 razmaknica.
7. Polipeptid prema patentnom zahtjevu 5, naznačen time što premošćujući dio sadrži značajku odabranu iz skupine koju čine disulfidna veza, amidna veza (laktam), tioeterska veza, aromatski prsten, nezasićeni alifatski ugljikovodični lanac, zasićeni alifatski ugljikovodični lanac i prsten triazola.
8. Polipeptid prema patentnom zahtjevu 5, naznačen time što premošćujući dio sadrži aromatski prsten koji je nastao reakcijom s poli(bromometil)benzenom.
9. Polipeptid prema patentnom zahtjevu 1, naznačen time što je navedeni polipeptid konjugiran s hidrofilnim polimerom.
10. Polipeptid prema patentnom zahtjevu 9, naznačen time što: (a) hidrofilni polimer je odabran iz skupine koju čine homopolimeri polialkilen oksida, polipropilen glikoli, polioksietilenirani polioli i njihovi kopolimeri; ili (b) hidrofilni polimer sadrži polietilen glikol (PEG).
11. Polipeptid prema patentnom zahtjevu 1, naznačen time što: (a) navedeni polipeptid sadrži najmanje jedan lipidni dio; (b) navedeni polipeptid je konjugiran s polipeptidom koji veže albumin, pri čemu polipeptid koji veže albumin sadrži aminokiselinsku sekvencu odabranu iz skupine koju čine SEQ ID NO: 202-204; ili (c) navedeni polipeptid je konjugiran s polipeptidom koji prodire u stanicu, pri čemu polipeptid koji prodire u stanicu sadrži aminokiselinsku sekvencu odabranu iz skupine koju čine SEQ ID NO: 205-210.
12. Pripravak, naznačen time što sadrži polipeptid prema bilo kojem od patentnih zahtjeva 1-11 i prihvatljiv nosač ili pomoćnu tvar.
13. Pripravak prema patentnom zahtjevu 12, za uporabu u postupku terapije inhibiranja cijepanja C5 u staničnom sustavu, te navedeni postupak obuhvaća dovođenje u kontakt navedenog staničnog sustava s pripravkom.
14. Pripravak za uporabu prema patentnom zahtjevu 13, naznačen time što je navedeni stanični sustav ljudski subjekt.
15. Pripravak za uporabu prema patentnom zahtjevu 14, naznačen time što navedeni ljudski subjekt sadrži bolest, poremećaj i/ili stanje povezano s komplementom, pri čemu, po izboru, navedena bolest, poremećaj i/ili stanje povezano s komplementom odabrana iz skupine koju čine upalna indikacija, rana, ozljeda, autoimuna bolest, vaskularna indikacija, neurološka indikacija, indikacija povezana s bubrezima, očna bolest, paroksizmalna noćna hemoglobinurija, i atipični hemolitički uremijski sindrom.
HRP20211561TT 2014-06-12 2015-06-12 Modulacija aktivnosti komplementa HRP20211561T8 (hr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201462011368P 2014-06-12 2014-06-12
US201462077460P 2014-11-10 2014-11-10
US201562108772P 2015-01-28 2015-01-28
EP19194070.9A EP3628680B1 (en) 2014-06-12 2015-06-12 Modulation of complement activity

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HRP20211561T1 true HRP20211561T1 (hr) 2021-12-24
HRP20211561T8 HRP20211561T8 (hr) 2022-03-04

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HRP20191763TT HRP20191763T1 (hr) 2014-06-12 2019-09-27 Modulacija komplementarne aktivnosti

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EP (4) EP3973994A1 (hr)
JP (4) JP6432954B2 (hr)
KR (3) KR102346228B1 (hr)
CN (2) CN111187338A (hr)
AP (1) AP2016009612A0 (hr)
AU (3) AU2015274482B2 (hr)
BR (3) BR112016029076B1 (hr)
CA (2) CA2949985C (hr)
CY (2) CY1122227T1 (hr)
DK (2) DK3154561T3 (hr)
ES (2) ES2895029T3 (hr)
HR (2) HRP20211561T8 (hr)
HU (2) HUE045646T2 (hr)
IL (3) IL249093B (hr)
LT (2) LT3154561T (hr)
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NZ (3) NZ727420A (hr)
PL (2) PL3154561T3 (hr)
PT (2) PT3154561T (hr)
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RU (1) RU2670988C2 (hr)
SG (1) SG11201610222UA (hr)
SI (2) SI3628680T1 (hr)
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