AU2021296421A1 - ActRII proteins for the treatment of pulmonary arterial hypertension (PAH) - Google Patents

ActRII proteins for the treatment of pulmonary arterial hypertension (PAH) Download PDF

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Publication number
AU2021296421A1
AU2021296421A1 AU2021296421A AU2021296421A AU2021296421A1 AU 2021296421 A1 AU2021296421 A1 AU 2021296421A1 AU 2021296421 A AU2021296421 A AU 2021296421A AU 2021296421 A AU2021296421 A AU 2021296421A AU 2021296421 A1 AU2021296421 A1 AU 2021296421A1
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Australia
Prior art keywords
patient
polypeptide
kit
amino acid
seq
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Pending
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AU2021296421A
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English (en)
Inventor
Patrick Andre
Janethe De Oliveira Pena
Sachindra Joshi
Ravindra Kumar
Gang Li
Peter Linde
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Acceleron Pharma Inc
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Acceleron Pharma Inc
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Publication of AU2021296421A1 publication Critical patent/AU2021296421A1/en
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/179—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1796—Receptors; Cell surface antigens; Cell surface determinants for hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
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    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
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    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/68—Medicinal 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
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    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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    • A61K9/0056—Mouth soluble or dispersible forms; Suckable, eatable, chewable coherent forms; Forms rapidly disintegrating in the mouth; Lozenges; Lollipops; Bite capsules; Baked products; Baits or other oral forms for animals
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    • A61K9/06—Ointments; Bases therefor; Other semi-solid forms, e.g. creams, sticks, gels
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate 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
    • 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
    • A61K9/20—Pills, tablets, discs, rods
    • A61K9/2004—Excipients; Inactive ingredients
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/4841—Filling excipients; Inactive ingredients
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • Y—GENERAL 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
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
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  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Dermatology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
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  • Toxicology (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Endocrinology (AREA)
  • Neurology (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
AU2021296421A 2020-06-23 2021-06-22 ActRII proteins for the treatment of pulmonary arterial hypertension (PAH) Pending AU2021296421A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202063042722P 2020-06-23 2020-06-23
US63/042,722 2020-06-23
US202063084409P 2020-09-28 2020-09-28
US63/084,409 2020-09-28
US202063112513P 2020-11-11 2020-11-11
US63/112,513 2020-11-11
US202163188141P 2021-05-13 2021-05-13
US63/188,141 2021-05-13
PCT/US2021/038482 WO2021262718A1 (en) 2020-06-23 2021-06-22 Actrii proteins for the treatment of pulmonary arterial hypertension (pah)

Publications (1)

Publication Number Publication Date
AU2021296421A1 true AU2021296421A1 (en) 2023-01-19

Family

ID=79281768

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Application Number Title Priority Date Filing Date
AU2021296421A Pending AU2021296421A1 (en) 2020-06-23 2021-06-22 ActRII proteins for the treatment of pulmonary arterial hypertension (PAH)

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US (3) US20230226146A1 (enExample)
EP (1) EP4168037A4 (enExample)
JP (2) JP7854402B2 (enExample)
KR (1) KR20230026435A (enExample)
CN (1) CN115989035A (enExample)
AU (1) AU2021296421A1 (enExample)
BR (1) BR112022025644A2 (enExample)
CA (1) CA3182838A1 (enExample)
CL (1) CL2022003686A1 (enExample)
CO (1) CO2022018719A2 (enExample)
EC (1) ECSP23004827A (enExample)
IL (1) IL299189A (enExample)
MX (1) MX2022016237A (enExample)
PE (1) PE20230783A1 (enExample)
WO (1) WO2021262718A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4121088A4 (en) 2020-03-20 2024-07-03 Keros Therapeutics, Inc. Methods of using activin receptor type iib variants
EP4121078A4 (en) 2020-03-20 2024-08-14 Keros Therapeutics, Inc. ACTIVIN RECEPTOR TYPE II CHIMERAS AND METHODS OF USE THEREOF
US20230174620A1 (en) 2020-04-28 2023-06-08 Acceleron Pharma Inc. Actrii proteins and use in treating post-capillary pulmonary hypertension
KR20230026435A (ko) * 2020-06-23 2023-02-24 악셀레론 파마 인코포레이티드 Actrii 단백질 및 이의 용도
EP4568692A1 (en) * 2022-08-10 2025-06-18 GB002, Inc. Tyrosine kinase inhibitor and activin type 2 receptor antagonist combination therapy for treating pulmonary arterial hypertension (pah)
WO2024054985A2 (en) * 2022-09-09 2024-03-14 Keros Therapeutics, Inc. Methods of administering an activin type iib variant
WO2024238263A1 (en) * 2023-05-12 2024-11-21 Merck Sharp & Dohme Llc Methods of improving lung diffusion capacity in a patient with pulmonary arterial hypertension
CN121127502A (zh) * 2023-05-15 2025-12-12 宜明凯尔生物医药技术(上海)有限公司 用于治疗肺高压的重组融合蛋白
IL328834A (en) * 2023-12-07 2026-08-01 Merck Sharp & Dohme Llc Methods for treating pulmonary arterial hypertension using activin receptor type IIA (ACTRIIA) proteins and/or variants thereof
WO2025237307A1 (zh) * 2024-05-14 2025-11-20 苏州康宁杰瑞生物科技有限公司 凝血因子FXI/FXIa抑制剂用于治疗肺动脉高压和/或高血压
CN120888004B (zh) * 2025-09-30 2026-04-03 齐鲁制药有限公司 包含索特西普和其变体的组合物以及相关方法和应用

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA41052A (fr) 2014-10-09 2017-08-15 Celgene Corp Traitement d'une maladie cardiovasculaire à l'aide de pièges de ligands d'actrii
EP3298034A4 (en) 2015-05-20 2019-02-13 Celgene Corporation IN VITRO CELL CULTURE PROCEDURE FOR BETA THALASSEMIA BY MEANS OF ACTIVIN TYPE II RECEPTOR LIGANDS
AU2017296040C1 (en) * 2016-07-15 2023-06-22 Acceleron Pharma Inc. Compositions and methods for treating pulmonary hypertension
IL320014A (en) * 2018-01-12 2025-06-01 Keros Therapeutics Inc Activin receptor type iib variants and methods of use thereof
IL278546B2 (en) * 2018-05-09 2026-03-01 Keros Therapeutics Inc Activin receptor type IIA variants and methods of using them
KR20230026435A (ko) * 2020-06-23 2023-02-24 악셀레론 파마 인코포레이티드 Actrii 단백질 및 이의 용도

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US20230226146A1 (en) 2023-07-20
CO2022018719A2 (es) 2023-03-07
US20250249070A1 (en) 2025-08-07
US20250082722A1 (en) 2025-03-13
CA3182838A1 (en) 2021-12-30
CL2022003686A1 (es) 2023-05-26
EP4168037A4 (en) 2024-07-17
JP7854402B2 (ja) 2026-05-01
EP4168037A1 (en) 2023-04-26
MX2022016237A (es) 2023-03-08
JP2026123100A (ja) 2026-07-29
IL299189A (en) 2023-02-01
KR20230026435A (ko) 2023-02-24
BR112022025644A2 (pt) 2023-03-07
JP2023534143A (ja) 2023-08-08
PE20230783A1 (es) 2023-05-11
ECSP23004827A (es) 2023-03-31
WO2021262718A1 (en) 2021-12-30
CN115989035A (zh) 2023-04-18

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