公开(公告)号：WO2021050885A1

公开(公告)日：2021-03-18

申请号：PCT/US2020/050422

申请日：2020-09-11

申请人： RA PHARMACEUTICALS, INC.

发明人： READ, Simon J. ,  RICARDO, Alonso ,  SAYEGH, Camil ,  XU, Xiangyang ,  TANG, Yalan ,  TANG, Guo-Qing

IPC分类号： A61P21/00 ,  A61K38/12 ,  A61P21/04

摘要： The present disclosure provides methods of treating inflammatory indications with complement inhibitor compounds and compositions. Included are compounds and methods for treating amyotrophic lateral sclerosis (ALS).

公开(公告)号：WO2020086506A1

公开(公告)日：2020-04-30

申请号：PCT/US2019/057316

申请日：2019-10-22

申请人： RA PHARMACEUTICALS, INC.

发明人： DUDA, Petra ,  FARZANEH-FAR, Ramin ,  MA, Zhong ,  ZHU, Nanqun ,  THACKABERRY, Evan ,  RICARDO, Alonso

IPC分类号： A61K39/395 ,  A61K31/506 ,  A61P7/00 ,  C07K16/18

摘要： The present disclosure relates methods of treating myasthenia gravis with zilucoplan (RA101495), including generalized myasthenia gravis. The present dislosure also claims devices and kits for zilucoplan administration and methods of evaluating complement inhibitor treatment efficacy.

公开(公告)号：WO2020009805A3

公开(公告)日：2020-01-09

申请号：PCT/US2019/038247

申请日：2019-06-20

申请人： MERCK SHARP & DOHME CORP. ,  RA PHARMACEUTICALS, INC.

发明人： RICARDO, Alonso ,  TUCKER, Thomas, Joseph ,  BOYER, Nicolas, Cedric ,  DHAMNASKAR, Ketki, Ashok ,  MA, Zhong ,  KEREKES, Angela, Dawn ,  WU, Chengwei ,  HA, Sookhee, Nicole ,  YOUM, Hyewon ,  BIANCHI, Elisabetta ,  BRANCA, Danila ,  INGENITO, Raffaele ,  COSTANTINI, Willy ,  SHAHRIPOUR, Aurash ,  XIONG, Yusheng

IPC分类号： A61K38/12 ,  A61K38/10 ,  A61K31/175 ,  C07K7/50 ,  A61P3/06

摘要： Provided herein are cyclic polypeptide compounds that can, e.g., bind specifically to human proprotein convertase subtilisin/kexin type 9 (PCSK9) and optionally also inhibit interaction between human PCSK9 and human low density lipoprotein receptor (LDLR), and pharmaceutical compositions comprising one or more of these compounds. Also provided are methods of reducing LDL cholesterol level in a subject in need thereof that include administering to the subject one or more of the cyclic polypeptide compounds or a pharmaceutical composition provided herein.

公开(公告)号：WO2017181061A1

公开(公告)日：2017-10-19

申请号：PCT/US2017/027697

申请日：2017-04-14

申请人： RA PHARMACEUTICALS, INC.

发明人： JOSEPHSON, Kristopher ,  MA, Zhong ,  SUN, Yili ,  NIMS, Nathan Ezekiel ,  SEYB, Kathleen ,  TOBE, Sylvia ,  YE, Ping ,  VADYSIRISACK, Douangsone D. ,  TANG, Guo-Qing ,  TRECO, Douglas A.

IPC分类号： C07K7/08 ,  C08G65/48 ,  A61K38/00

摘要： The present invention provides Ras modulators including inhibitors and/or antagonists of Ras, Ras binding, and Ras-dependent cell signaling activity. Also provided are methods of utilizing the Ras modulators as therapeutics.

摘要翻译： 本发明提供了Ras调节剂，包括Ras，Ras结合和Ras依赖性细胞信号传导活性的抑制剂和/或拮抗剂。 还提供了利用Ras调节剂作为治疗剂的方法。

公开(公告)号：WO2022177635A2

公开(公告)日：2022-08-25

申请号：PCT/US2021/064552

申请日：2021-12-21

申请人： RA PHARMACEUTICALS, INC. ,  OKLAHOMA MEDICAL RESEARCH FOUNDATION

发明人： RICARDO, Alonso ,  LUPU, Florea ,  COGGESHALL, Mark

IPC分类号： A61K38/10 ,  A61K38/12 ,  A61P37/02 ,  C07K5/12 ,  C07K7/08 ,  C12N15/09 ,  A61K38/00

摘要： The present disclosure provides compounds, compositions, and methods for modulating complement activity. Included are C5 inhibitor compounds and related methods of use in treating complement-related indications, including those resulting from microbial infection.

公开(公告)号：WO2020185541A3

公开(公告)日：2020-09-17

申请号：PCT/US2020/021330

申请日：2020-03-06

申请人： RA PHARMACEUTICALS, INC.

发明人： MA, Zhong ,  ZHU, Nanqun ,  THACKABERRY, Evan ,  FARZANEH-FAR, Ramin ,  RICARDO, Alonso

IPC分类号： A61K38/12 ,  A61K31/506 ,  A61P7/00 ,  A61K38/13 ,  C07K16/28 ,  A61K39/395

摘要： The disclosure relates to a method of treating a complement-related indication in a subject by inhibiting C5 activity within or beneath a tissue of the subject, the method comprising contacting the subject with a tissue-penetrating C5 inhibitor, wherein the tissue-penetrating C5 inhibitor comprises zilucoplan (RA101495) and wherein the tissue- penetrating C5 inhibitor permeates the tissue and/or diffuses across the tissue, thereby inhibiting C5 activity within or beneath the tissue.

公开(公告)号：WO2020009805A2

公开(公告)日：2020-01-09

申请号：PCT/US2019/038247

申请日：2019-06-20

申请人： MERCK SHARP & DOHME CORP. ,  RA PHARMACEUTICALS, INC.

发明人： RICARDO, Alonso ,  TUCKER, Thomas, Joseph ,  BOYER, Nicolas, Cedric ,  DHAMNASKAR, Ketki, Ashok ,  MA, Zhong ,  KEREKES, Angela, Dawn ,  WU, Chengwei ,  HA, Sookhee, Nicole ,  YOUM, Hyewon ,  BIANCHI, Elisabetta ,  BRANCA, Danila ,  INGENITO, Raffaele ,  COSTANTINI, Willy ,  SHAHRIPOUR, Aurash ,  XIONG, Yusheng

IPC分类号： A61K38/12

摘要： Provided herein are cyclic polypeptide compounds that can, e.g., bind specifically to human proprotein convertase subtilisin/kexin type 9 (PCSK9) and optionally also inhibit interaction between human PCSK9 and human low density lipoprotein receptor (LDLR), and pharmaceutical compositions comprising one or more of these compounds. Also provided are methods of reducing LDL cholesterol level in a subject in need thereof that include administering to the subject one or more of the cyclic polypeptide compounds or a pharmaceutical composition provided herein.

公开(公告)号：WO2019051436A1

公开(公告)日：2019-03-14

申请号：PCT/US2018/050317

申请日：2018-09-11

申请人： RA PHARMACEUTICALS, INC.

发明人： FAHRNER, Richard Lee ,  PHILLIPS, Christian Winthrop

IPC分类号： C12P7/64

CPC分类号： A61K9/0019 ,  A61K9/08 ,  A61K47/14

摘要： The present disclosure provides formulations for sustained release of therapeutic agents. Included are formulations incorporating a mixture of acylglycerols. Also included are methods of delivering therapeutic agents to subjects using sustained release formulations and methods of treating complement-related indications using formulations disclosed.

公开(公告)号：WO2017105939A1

公开(公告)日：2017-06-22

申请号：PCT/US2016/065228

申请日：2016-12-07

申请人： RA PHARMACEUTICALS, INC.

发明人： DEMARCO, Steven James ,  ARATA, Michelle Denise ,  PARKER, Grace Victoria ,  RICARDO, Alonso ,  TOBE, Sylvia ,  TRECO, Douglas A.

IPC分类号： A61K38/12 ,  A61P7/00 ,  A61P37/02 ,  A61M5/30

CPC分类号： A61K38/12 ,  A61K9/0019

摘要： The present invention relates to polypeptide modulators of complement activity, including cyclic polypeptide modulators. Included are methods of utilizing such modulators as therapeutics.

摘要翻译： 本发明涉及补体活性的多肽调节剂，包括环状多肽调节剂。 包括利用这种调节剂作为治疗剂的方法。

公开(公告)号：WO2016123371A1

公开(公告)日：2016-08-04

申请号：PCT/US2016/015412

申请日：2016-01-28

申请人： RA PHARMACEUTICALS, INC.

发明人： ARATA, Michelle, Denise ,  DHAMNASKAR, Ketki, Ashok ,  ELBAUM, Daniel ,  JOSEPHSON, Kristopher ,  LARSON, Kelley, Cronin ,  MA, Zhong ,  NIMS, Nathan, Ezekiel ,  RICARDO, Alonso ,  SEYB, Kathleen ,  TANG, Guo-Qing ,  TRECO, Douglas, A. ,  WANG, Zhaolin ,  YE, Ping ,  ZHENG, Hong ,  PERLMUTTER, Sarah, Jacqueline

IPC分类号： A61K39/00 ,  A61K39/395

CPC分类号： A61K38/10 ,  A61K39/00 ,  C07K16/36 ,  C07K2317/76

摘要： The present invention provides polypeptide modulators of complement activity, including cyclic polypeptide modulators. Also provided are methods of utilizing such modulators as therapeutics.

摘要翻译： 本发明提供了补体活性的多肽调节剂，包括环状多肽调节剂。 还提供了利用这种调节剂作为治疗剂的方法。