Share

June 04, 2019; 92 (23) Article

Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis

James F. Howard, Vera Bril, Ted M. Burns, Renato Mantegazza, Malgorzata Bilinska, Andrzej Szczudlik, Said Beydoun, Francisco Javier Rodriguez De Rivera Garrido, Fredrik Piehl, Mariarosa Rottoli, Philip Van Damme, Tuan Vu, Amelia Evoli, Miriam Freimer, Tahseen Mozaffar, E. Sally Ward, Torsten Dreier, Peter Ulrichts, Katrien Verschueren, Antonio Guglietta, Hans de Haard, Nicolas Leupin, Jan J.G.M. Verschuuren, on behalf of the Efgartigimod MG Study Group
First published May 22, 2019, DOI: https://doi.org/10.1212/WNL.0000000000007600
Full PDF
Short Form
Citation
Permissions

Make Comment

See Comments

Downloads
1551

Share

Article Information

vol. 92 no. 23 e2661-e2673
DOI: 
https://doi.org/10.1212/WNL.0000000000007600
PubMed: 
31118245
Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Print ISSN: 
0028-3878
Online ISSN: 
1526-632X
History: 
  • Received May 28, 2018
  • Accepted in final form January 31, 2019
  • First Published May 22, 2019.

Article Versions

Copyright & Usage: 
© 2019 American Academy of Neurology

Author Disclosures

    1. James F. Howard Jr, MD,
    2. Vera Bril, MD,
    3. Ted M. Burns, MD,
    4. Renato Mantegazza, MD,
    5. Malgorzata Bilinska, MD,
    6. Andrzej Szczudlik, MD,
    7. Said Beydoun, MD,
    8. Francisco Javier Rodriguez De Rivera Garrido, MD,
    9. Fredrik Piehl, MD, PhD,
    10. Mariarosa Rottoli, MD,
    11. Philip Van Damme, MD, PhD,
    12. Tuan Vu, MD,
    13. Amelia Evoli, MD,
    14. Miriam Freimer, MD,
    15. Tahseen Mozaffar, MD,
    16. E. Sally Ward, PhD,
    17. Torsten Dreier, PhD,
    18. Peter Ulrichts, PhD,
    19. Katrien Verschueren, MSc,
    20. Antonio Guglietta, MD,
    21. Hans de Haard, PhD,
    22. Nicolas Leupin, MD,
    23. Jan J.G.M. Verschuuren, MD, PhD;
    24. on behalf of the Efgartigimod MG Study Group
  1. James F. Howard Jr, MD,
    2. Scientific Advisory Boards:

    1. (1) SAB (non-remunerative), Alexion Pharmaceuticals (2) SAB (non-remunerative), argenx BVBA (3) SAB (non-remunerative, Ra Pharmaceuticals (4) SAB (non-remunerative), Toleranzia AB

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. (1) Alexion Pharmaceuticals, travel for investigator meetings, SAB meetings; honoraria for advisory training sessions (2) argenx BVBA, travel for investigator meeting, SAB meetings (3) Ra Pharmaceuticals, travel for investigator meeting, SAB meetings

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. (1) Consultant (non-remunerative), Alexion Pharmaceuticals (2) Consultant (non-remunerative), argenx BVBA (3) Consultant (non-remunerative), Ra Pharmaceuticals (4) Consultant (non-remunerative), Toleranzia AB

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. (1) Alexion Pharmaceuticals (2) argenx BVBA (3) Ra Pharmaceuticals

    Research Support, Government Entities:

    1. (1) PCORI Award #1609-35953/Duke Clinical Research Institute, Principal Site Investigator, 2018 ? 2021 (2) Centers for Disease Control, and Prevention (CDC) U01DD001116,Co-Investigator, 2014 ? 2019. (3) NIH U01-NS061799-01A2, Principal Site Investigator, 2013 ? 2021. (4) NIH NS079683-01A1, Inveseatigator, 2013 ? 2018. (5) NIH R01 NS074057-01A1, Co-Investigator, 2011 ? 2017

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  2. Vera Bril, MD,
    3. Scientific Advisory Boards:

    1. (2) CSL Behring (2) Baxalta (2) Grifols (2) Argenyx (2) Octapharma (2) Alpha Technologies (3) Powell Mansfield Inc (2) Shire (2) Akcea (2) UCB (2) Alnylam

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. (2) CSL Behring

    Editorial Boards:

    1. PLoS One, editorial board member starting 2016

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. (2) CSL Behring, Grifols, Bionevia, Octapharma, Powell Mansfield Inc, Argenyx, Alpha Technologies, Baxalta, Akcea, UCB, Alnylam, Pfizer

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. (2) CSL Behring, Grifols, Bionevia, Octapharma, Baxalta, ArgenX, UCB, Shire

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Toronto General/Toronto Western Hospital Foundation GBS/CIDP Foundation International

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  3. Ted M. Burns, MD,
    4. Scientific Advisory Boards:

    1. Argenx, Momenta

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Argenx, Alexion Pharmaceuticals

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. received support for consulting activities from UCB Pharma, CSL Behring, Momenta, Argenx

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  4. Renato Mantegazza, MD,
    5. Scientific Advisory Boards:

    1. Dr. Mantegazza serves on a scientific advisory board for Alexion Pharmaceuticals and Biomarin

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. (1) Alexion Pharmaceuticals Advisory Board (2) Biomarin Advisory Board (3) Argenx Advisory Board

    Editorial Boards:

    1. NONE

    Patents:

    1. Italian Patent n.MI2011A 002106

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. (1) Annual Funding for Research from the Italian Ministry of Health

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. (1) AIM (Associazione Italiana per la lotta alla Miastenia)

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  5. Malgorzata Bilinska, MD,
    6. Scientific Advisory Boards:

    1. Argenix, Novartis, Griffols, Beringer, Baxter.

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Argenix, Novartis, Griffols - funding for travel to Investigator Mitting; Beringer -EFNS 2018 and 2017.Baxter - Neurological congress in Krakow

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Argenix, Griffols, Novartis Signature: Małgorzata Bilinska Date: 29 Jan 2019

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  6. Andrzej Szczudlik, MD,
    7. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. Neurologia i Neurochirurgia Polska Editor in Chief, 2005 - present

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  7. Said Beydoun, MD,
    8. Scientific Advisory Boards:

    1. Akcea, Alnylam, Alexion Pharmaceuticals, CSL, Grifols, Mitsubishi Tanabe, Shire

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. US Neurology; Editor-in Chief, 2017-

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. CSL, Grifols, Mitsubishi Tanabe, Alexion Pharmaceuticals, Shire

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Mallinkrodt, Pfizer, Catalyst, Argenx, UCB Pharma

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  8. Francisco Javier Rodriguez De Rivera Garrido, MD,
    9. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  9. Fredrik Piehl, MD, PhD,
    10. Scientific Advisory Boards:

    1. data safety monitoring committee Parexel/Chugai, outside of this study

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. 1. Biogen - unrestricted academic research projects outside of this study 2. Novartis - unrestricted academic research projects outside of this study 3. Genzyme - unrestricted academic research projects outside of this study 4. Merck KGaA - unrestricted academic research projects outside of this study

    Research Support, Government Entities:

    1. Swedish Medical Research Council (grant 2017-03054) PI, 2018- ongoing. Pcori, Washington DC (grant MS-1511-33196) PI, 2017- ongoing

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  10. Mariarosa Rottoli, MD,
    11. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  11. Philip Van Damme, MD, PhD,
    12. Scientific Advisory Boards:

    1. Served on the scientific advisory board of Biogen, Cytokinetcs, Sanofi, Pfizer, CSL Behring, Alexion Pharmaceuticals.

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. Clinical research fellowship from FWO-Vlaanderen

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  12. Tuan Vu, MD,
    13. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Allergan-Speaker bureau CSL Behring-Speaker bureau Alexion Pharmaceuticals-Speaker bureau MT Pharma-Speaker bureau

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Ipsen, commercial entity, consulting

    Speakers' Bureaus:

    1. Allergan (commercial) - nothing related to the study CSL Behring-Speaker bureau - Nothing related to the study Alexion Pharmaceuticals-Speaker bureau - nothing related to the study MT Pharma-Speaker bureau - nothing related to the study

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. I participated in multicenter clinical trials as a site PI for Alexion Pharmaceuticals, Ra, UCB, Biogen, Flex, Grifols, Octapharma, Mallinkrodt, Orion, Cytokinetics, and ARGX

    Research Support, Government Entities:

    1. I participated in multicenter trials sponsored by NIH as a site PI: 5 R01 NS090083-05, 1 R21 HDO75327

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  13. Amelia Evoli, MD,
    14. Scientific Advisory Boards:

    1. scientific advisory board - Alexion Pharmaceuticals data safety monitoring board - UCB

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other activities:

    1. Scientific award jury member for Grifols

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  14. Miriam Freimer, MD,
    15. Scientific Advisory Boards:

    1. 1.Advisory board- Alexion Pharmaceuticals 2. Advisory board- CSL Behring

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. Alexinon- Have not given any talks

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. 1. Alexion Pharmaceuticals 2. UCB 3.Argenx 4. Immunovant 5. Alnylum 6.Amicus 7.Ra 8.Orphozyme 9.Acceleron 10. Catalyst 11. Grifols

    Research Support, Government Entities:

    1. NIH- neuronext

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Muscular Dystrophy Association clinic grant

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  15. Tahseen Mozaffar, MD,
    16. Scientific Advisory Boards:

    1. On Medical and Scientific Advisory Board for the Myositis Association, Amicus, Biomarin, Genzyme, Idera Pharmaceuticals, and Ultragenyx

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Received travel reimbursement for advisory board meeting for 1) Genzyme Corporation; 2) Biomarin; and 3) Amicus

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. 1) Commercial or non-profit entity: Amicus, 2) Commercial or non-profit entity: Biomarin, 3) Commercial or non-profit entity: Genzyme, 4) Commercial or non-profit entity: Sarepta and 5) Commercial or non-profit entity: Ultragenyx 6) Commercial or non-profit entity: OptionCare 7) Commercial or non-profit entity: Spark Therapeutics

    Speakers' Bureaus:

    1. 1) Commercial or non-profit entity: Genzyme Corporation 2) Commercial or non-profit entity: Grifols

    Other activities:

    1. received financial support from NuFACTOR, Inc. during the course of this study.

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. 1) Commercial or non-profit entity: Alnylam 2) Commercial or non-profit entity: Alexion Pharmaceuticals 3) Commercial or non-profit entity: Amicus 4) Commercial or non-profit entity: Biomarin 5) Commercial or non-profit entity: CSL Behring 6) Commercial or non-profit entity: Genzyme 7) Commercial or non-profit entity: Ionis 8) Commercial or non-profit entity: GSK 9) Commercial or non-profit entity: Novartis 10) Commercial or non-profit entity: Ultragenyx 11) Commercial or non-profit entity: Grifols 12) Commercial or non-profit entity: Octapharma

    Research Support, Government Entities:

    1. NIH Funding (primary investigator NS107210)

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  16. E. Sally Ward, PhD,
    17. Scientific Advisory Boards:

    1. (1) Non-Profit - Therapeutic Antibody Core Scientific Advisory Board, Cancer Prevention and Research Institute of Texas (2015 - present) (2) Non-Profit - Scientific Advisory Board for Antibody Biology and Engineering, Gordon Research Conference (2017 - present)

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. (1) Non-Profit - SELECTBIO, reimbursement (travel, hotel, meals) for March 2017 Advances in Drug Discovery conference (keynote speaker) (2) Non-Profit - GTCbio, reimbursement (travel, hotel, meals) for July 2017 5th Antibody and Protein Therapeutics conference (keynote speaker) (3) Commercial - argenx BVBA, reimbursement (travel, hotel, meals) for October 2017 meeting/presentation (speaker) (4) Non-Profit - Cancer Prevention and Research Institute of Texas, reimbursement (travel, hotel, meals) for November 2017 Therapeutic Antibody Core Symposium (keynote speaker) (5) Commercial - Cambridge Healthtech Institute, reimbursement (travel, hotel, meals) for January 2018 17th Annual PepTalk - Protein Science Week (invited speaker) (6) Non-Profit - Lupus Research Alliance, reimbursement (travel, hotel, meals) for January 2018 Human Immunology- Technology Initiative in Lupus (invited speaker) (7) Non-Profit - The Histochemical Society, reimbursement (travel, hotel, meals) for April 2018 Annual Meeting at Experimental Biology (invited speaker) (8) Commercial - Cambridge Healthtech Institute, reimbursement (travel, hotel, meals) for May 2018 PEGS 19th Annual Engineering Antibodies conference (keynote speaker) (9) Non-Profit - SELECTBIO, reimbursement (travel, hotel, meals) for May 2018 Stem Cells and Antibodies in Drug Discovery (invited speaker) (10) Non-Profit - Federation of American Societies for Experimental Biology, reimbursement (travel, hotel, meals) for June 2018 Immunoreceptors and Immunotherapy conference (primary organizer and speaker) (11) Non-Profit - Cancer Prevention and Research Institute of Texas, reimbursement (travel, hotel, meals) for November 2018 Therapeutic Antibody Core Symposium (invited speaker) (12) Non-Profit - The Antibody Society, reimbursement (travel, hotel, meals) for December 2018 Antibody Engineering & Therapeutics conference (keynote speaker) (13) Commercial - argenx BVBA, reimbursement (travel, hotel, meals) for January 2019 meeting

    Editorial Boards:

    1. (1) Protein Engineering, Design and Selection (formerly Protein Engineering), Editorial Board member, 2003-present (2) The Antibody Society, Board of Distinguished Advisors member, 2009-present (3) mAbs, Editorial Board member, 2009-present

    Patents:

    1. (1) (owned by UT Southwestern Medical Center) filed May 2006, Immunoglobulin Molecules With Improved Characteristics (2) (owned by UT Southwestern Medical Center and argenx BVBA) filed December 2014, FcRn Antagonists and Methods of Use E.S.W. is also a (co-)inventor on various patents that are not study-related from which she receives royalty payments through her previous employers (UT Southwestern Medical Center, U.K. Medical Research Council). E.S.W. has only partial knowledge of the licensing arrangements for these patents.

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. E.S.W. has served as a consultant for various parties in the previous two years (contractual obligations do not permit public disclosure without prior consent from various parties).

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. (1) Commercial - argenx BVBA Scientific Research Contract (2) Commercial - Bioverativ Scientific Research Contract (3) Commercial - Shire Pharmaceuticals Scientific Research Contract

    Research Support, Government Entities:

    1. (1) National Institutes of Health, R01 AR056478, Principal Investigator, August 2008 - May 2015 (2) National Institutes of Health, R01 GM085575, Co- Principal Investigator, August 2013 - present (3) Cancer Prevention and Research Institute of Texas, RP110441, Principal Investigator/Program Leader, July 2011 - December 2017 (4) Cancer Prevention and Research Institute of Texas, RP140141, Principal Investigator, August 2014 - present (5) Cancer Prevention and Research Institute of Texas, RP160051, Principal Investigator, March 2016 ? present

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. (1) National Multiple Sclerosis Society (RG 2411) (2) Wellcome Trust (U.K.) Research grant

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. E.S.W. is a (co-)inventor on various patents from which she receives royalty payments through her previous employers (UT Southwestern Medical Center, U.K. Medical Research Council). E.S.W. has only partial knowledge of the licensing arrangements for these patents.

    Stock/Stock Options, Research Sponsor:

    1. (1) argenx BVBA

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. E.S.W. has served as a consultant/expert witness in the previous two years (contractual obligations do not permit public disclosure without prior consent from various parties).

  17. Torsten Dreier, PhD,
    18. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. 1) WO 2015/100299 Al human IgG1 derived Fc-fragment with ABDEG mutations and efficacy in murine ITP model. 2) WO 2018/083122 Al PK/PD data in cynomolgus monkeys and human volunteers

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. CDO of argenx BVBA, the sponsor of the study described in manuscript

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  18. Peter Ulrichts, PhD,
    19. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. (1)FcRn antagonists and method of use (2) methods of reducing serum levels of fc-containing agents using fcrn antagonists (3)Chimeric human-llama antigens and methods of use (4)Antibodies to CD70 (5)Antibodies to complex targets

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. argenx bvba, research fellow, 2010-current

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  19. Katrien Verschueren, MSc,
    20. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. argenx BVBA Since Sep 2017 Sr. Biostatistician

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  20. Antonio Guglietta, MD,
    21. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. I have been an employee of Argenx since Oct 2016. currently I am the VP of Clinical Development in this company

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. As an employee of Argenx, I received stock options in the the following years: 2016, 2017 and 2018.

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  21. Hans de Haard, PhD,
    22. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. Named as inventor on patent 1) WO 2015/100299 A1 entitled

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. Chief Scientific Officer and one of the three founders of the biotech company argenx BVBA (sponsor of the study described in the manuscript); have an employment at the company for more than 10 years (since July 2008). Get compensation in the form of salary and stock.

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. As employee and Chief Scientific Officer of the sponsor argenx BVBA I was involved in writing the documents used for the interactions with FDA for the clinical study described in the manuscript.

  22. Nicolas Leupin, MD,
    23. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. argenx BVBA employee

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. argenx BVBA employee, Chief Medical Officer

  23. Jan J.G.M. Verschuuren, MD, PhD;
    24. Scientific Advisory Boards:

    1. Myasthenia Foundation of America (MGFA) medical/scientific advisory board. Study Steering Committee of Argenx. Advisory meeting for Rapharma. All reimbursements were received by the LUMC.

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. Journal of Neuromuscular Diseases, member editorial board, 2014-now

    Patents:

    1. Patents on MuSK myasthenia gravisE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. The Neurology department of the LUMC has received fees from Alexion Pharmaceuticals, Argen-X, and Rapharma because of consultancies

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Princes Beatrix Spierfonds, grant for MG research, Prinicpal investigator.

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. Development of MuSK diagnostic antibody test for TECAN/IBL.

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  24. on behalf of the Efgartigimod MG Study Group
    1. From the Department of Neurology (J.F.H.), University of North Carolina, Chapel Hill; Krembil Neuroscience Centre (V.B.), University Health Network, Toronto, Canada; Department of Neurology (T.M.B.), University of Virginia, Charlottesville; Department of Neuroimmunology and Neuromuscular Diseases (R.M.), Fondazione Istituto Neurologico Carlo Besta, Milan, Italy; Department of Neurology (B.M.), Wroclaw Medical University; Department of Neurology (A.S.), Jagiellonian University Medical College, Cracow, Poland; Department of Neurology (S.B.), University of Southern California, Keck School of Medicine, Los Angeles County Medical Center; Department of Neurology (F.J.R.D.R.G.), La Paz University Hospital, Neuroscience Area of IdiPAZ Health Research Institute, Autonoma University of Madrid, Spain; Neuroimmunology Unit, Department Clinical Neuroscience (F.P.), Karolinska Institutet, Karolinska University Hospital (Solna), Stockholm, Sweden; USC Neurologia (M.R.), USS Malattie Autoimmuni–Centro Sclerosi Multipla, Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII, Bergamo, Italy; Neurology Department (P.V.D.), University Hospitals Leuven; Laboratory of Neurobiology (P.V.D.), Department of Neuroscience, KU Leuven and Center for Brain & Disease Research, VIB, Leuven, Belgium; Department of Neurology (T.V.), University of South Florida, Morsani College of Medicine, Tampa; Institute of Neurology (A.E.), Fondazione Policlinico Universitario “A. Gemelli” IRCCS, Università Cattolica del Sacro Cuore, Rome, Italy; Neurology Department (M.F.), The Ohio State University, Columbus; Department of Neurology (T.M.), University of California, Irvine; Department of Molecular and Cellular Medicine (E.S.W.), Texas A&M University Health Science Center, College Station; argenx BVBA (T.D., P.U., K.V., A.G., H.d.H., N.L.), Zwijnaarde, Belgium; and Department of Neurology (J.J.G.M.V.), Leiden University Medical Center (LUMC), the Netherlands.
    1. Correspondence
      Dr. Leupin nleupin{at}argenx.com

    Article usage

    Article usage: May 2019 to October 2023

    AbstractFullPdfSource
    May 201913662142Highwire
    Jun 20191173101130Highwire
    Jul 20199376074Highwire
    Aug 20197726580Highwire
    Sep 20194986958Highwire
    Oct 20196405067Highwire
    Nov 20195642340Highwire
    Dec 20196803460Highwire
    Jan 20207114461Highwire
    Feb 20206175556Highwire
    Mar 20205953359Highwire
    Apr 20204472340Highwire
    May 20207043968Highwire
    Jun 20204142140Highwire
    Jul 20203071333Highwire
    Aug 2020281819Highwire
    Sep 20203101839Highwire
    Oct 20204952738Highwire
    Nov 20202421016Highwire
    Dec 2020214627Highwire
    Jan 2021303927Highwire
    Feb 20212832228Highwire
    Mar 20213532322Highwire
    Apr 20212961418Highwire
    May 20212681513Highwire
    Jun 20212601714Highwire
    Jul 20212291213Highwire
    Aug 2021206118Highwire
    Sep 20213112314Highwire
    Oct 20212852713Highwire
    Nov 202129763Highwire
    Dec 20213661716Highwire
    Jan 20223332626Highwire
    Feb 20224152319Highwire
    Mar 20225532113Highwire
    Apr 20223902516Highwire
    May 20223491913Highwire
    Jun 20223201712Highwire
    Jul 202234954Highwire
    Aug 2022370116Highwire
    Sep 202237078Highwire
    Oct 2022346910Highwire
    Nov 20223711010Highwire
    Dec 2022306264Highwire
    Jan 20233842410Highwire
    Feb 2023341117Highwire
    Mar 20233331815Highwire
    Apr 2023404147Highwire
    May 2023270103Highwire
    Jun 2023316159Highwire
    Jul 2023493134Highwire
    Aug 202328594Highwire
    Sep 20233362410Highwire
    Oct 2023382115Highwire

    Letters: Rapid online correspondence

    No comments have been published for this article.
    Comment

    REQUIREMENTS

    You must ensure that your Disclosures have been updated within the previous six months. Please go to our Submission Site to add or update your Disclosure information.

    Your co-authors must send a completed Publishing Agreement Form to Neurology Staff (not necessary for the lead/corresponding author as the form below will suffice) before you upload your comment.

    If you are responding to a comment that was written about an article you originally authored:
    You (and co-authors) do not need to fill out forms or check disclosures as author forms are still valid
    and apply to letter.

    Submission specifications:

    • Submissions must be < 200 words with < 5 references. Reference 1 must be the article on which you are commenting.
    • Submissions should not have more than 5 authors. (Exception: original author replies can include all original authors of the article)
    • Submit only on articles published within 6 months of issue date.
    • Do not be redundant. Read any comments already posted on the article prior to submission.
    • Submitted comments are subject to editing and editor review prior to posting.

    More guidelines and information on Disputes & Debates

    Compose Comment

    More information about text formats

    Plain text

    • No HTML tags allowed.
    • Web page addresses and e-mail addresses turn into links automatically.
    • Lines and paragraphs break automatically.
    Author Information
    NOTE: The first author must also be the corresponding author of the comment.
    First or given name, e.g. 'Peter'.
    Your last, or family, name, e.g. 'MacMoody'.
    Your email address, e.g. [email protected]
    Your role and/or occupation, e.g. 'Orthopedic Surgeon'.
    Your organization or institution (if applicable), e.g. 'Royal Free Hospital'.
    Publishing Agreement
    NOTE: All authors, besides the first/corresponding author, must complete a separate Publishing Agreement Form and provide via email to the editorial office before comments can be posted.
    CAPTCHA
    This question is for testing whether or not you are a human visitor and to prevent automated spam submissions.

    Vertical Tabs

    You May Also be Interested in

    Back to top
    Advertisement

    Related Articles

    Topics Discussed

    Alert Me

    Recommended articles