Article Information
Group Information: The Zilucoplan MG Study Group authors appear at the end of the article.
Accepted for Publication: December 12, 2019.
Published Online: February 17, 2020. doi:10.1001/jamaneurol.2019.5125
Zilucoplan MG Study Group Authors: Richard Barohn, MD; Mazen Dimachkie, MD; Mamatha Pasnoor, MD; Constantine Farmakidis,
MD; Tina Liu, MBA, BS; Samantha Colgan, BS; Michael G. Benatar, MD, PhD; Tulio Bertorini,
MD; Rekha Pillai, MD; Robert Henegar, LPN; Mark Bromberg, MD, PhD; Summer Gibson,
MD; Teresa Janecki, BS; Miriam Freimer, MD; Bakri Elsheikh, MD, MMBS/FRCP; Paige Matisak,
BS; Angela Genge, MD, FRCPC, eMBA; Amanda Guidon, MD; William David, MD, PhD; Ali
A. Habib, MD; Veena Mathew, MSc; Tahseen Mozaffar, MD; John L. Hinton, MD; William
Hewitt, MD; Deborah Barnett, CCRC; Patricia Sullivan; Doreen Ho, MD; James F. Howard
Jr, MD; Rebecca E. Traub, MD; Manisha Chopra, MD, MBBS; Henry J. Kaminski, MD; Radwa
Aly, MSc; Elham Bayat, MD; Mohammad Abu-Rub, MD; Shaida Khan, DO; Dale Lange, MD;
Shara Holzberg, MS, CCC-SLP; Bhupendra Khatri, MD; Emily Lindman, BS; Tayo Olapo,
BS; Lisa M. Sershon, MMS, PA-C, MSCS; Robert P. Lisak, MD; Evanthia Bernitsas, MD;
Kelly Jia, MD; Rabia Malik, MD; Tiffany D. Lewis-Collins, BA, CMA; Michael Nicolle,
MD, FRCPC, DPhil; Richard J. Nowak, MD; Aditi Sharma, MBBS; Bhaskar Roy, MD; Joan
Nye, BS; Michael Pulley, MD, PhD; Alan Berger, MD; Yasmeen Shabbir, MBBS; Amit Sachdev,
MD, MS; Kimberly Patterson, BAS, R EEG T; Zaeem Siddiqi, MD, PhD; Mark Sivak, MD;
Joan Bratton, BA; George Small, MD; Anem Kohli, MBBS, BS; Mary Fetter, BS; Tuan Vu,
MD; Lucy Lam, MS, BS; Brittany Harvey, BA; Gil I. Wolfe, MD; Nicholas Silvestri, MD;
Kara Patrick, MS; Karen Zakalik, MBA, BA; Petra W. Duda, MD, PhD; James MacDougall,
PhD; Ramin Farzaneh-Far, MD; Angela Pontius, BA; Michelle Hoarty, MS, BA.
Affiliations of Zilucoplan MG Study Group Authors: University of North Carolina, Chapel Hill (Howard Jr, Traub, Chopra); Yale School
of Medicine, New Haven, Connecticut (Nowak, Sharma, Roy, Nye); University at Buffalo,
Buffalo, New York (Wolfe, Silvestri, Patrick, Zakalik); Ohio State University, Columbus
(Freimer, Elsheikh, Matisak); University of South Florida, Tampa (Vu, Lam, Harvey);
Infirmary Health, Mobile, Alabama (Hinton, Hewitt, Barnett, Sullivan); University
of Miami, Miami, Florida (Benatar); Ra Pharmaceuticals Inc, Cambridge, Massachusetts
(Duda, MacDougall, Farzaneh-Far, Pontius, Hoarty); George Washington University, Washington,
DC (Kaminski, Aly, Bayat, Abu-Rub); University of Kansas, Fairway (Barohn, Dimachkie,
Pasnoor, Farmakidis, Liu, Colgan); Wesley Neurology Clinic, Cordova, Tennessee (Bertorini,
Pillai, Henegar); University of Utah, Salt Lake City (Bromberg, Gibson, Janecki);
Montreal Neurological Institute, Montreal, Quebec, Canada (Genge); Massachusetts General
Hospital, Boston (Guidon, David); University of California, Irvine, Orange (Habib,
Mathew, Mozaffar); Lahey Hospital, Burlington, Massachusetts (Ho); University of Texas
Southwestern, Dallas, Irving (Khan); Hospital for Special Surgery, New York, New York
(Lange, Holzberg); Center for Neurological Disorders, St Francis Hospital at Ascension,
Milwaukee, Wisconsin (Khatri, Lindman, Olapo, Sershon); Wayne State University, Detroit,
Michigan (Lisak, Bernitsas, Jia); Rush University, Chicago, Illinois (Malik, Lewis-Collins);
London Health Sciences Center, London, Ontario, Canada (Nicolle); University of Florida,
Jacksonville (Pulley, Berger, Shabbir); Michigan State University, East Lansing (Sachdev,
Patterson); University of Alberta, Edmonton, Alberta, Canada (Siddiqi); Mount Sinai
Hospital, New York, New York (Sivak, Bratton); Allegheny Neurological Associates,
Pittsburgh, Pennsylvania (Small, Kohli, Fetter).
Corresponding Author: James F. Howard Jr, MD, University of North Carolina, 170 Manning Dr, 2200 Physician
Office Bldg, CB No. 7025, Chapel Hill, NC 27599-7026 (
howardj@
123456neurology.unc.edu
).
Open Access: This is an open access article distributed under the terms of the
CC-BY-NC-ND License. © 2020 Howard JF Jr et al.
JAMA Neurology.
Author Contributions
: Dr Howard had full access to all of the data in the study and takes responsibility
for the integrity of the data and the accuracy of the data analysis.
Concept and design: Howard, Nowak, Benatar, Duda, Farzaneh-Far, Kaminski, Barohn, Mozaffar, Sullivan,
Olapo, Lisak, Pontius, Hoarty.
Acquisition, analysis, or interpretation of data: Howard, Nowak, Wolfe, Freimer, Vu, Hinton, Duda, MacDougall, Farzaneh-Far, Kaminski,
Barohn, Dimachkie, Pasnoor, Farmakidis, Liu, Colgan, Bertorini, Pillai, Henegar, Bromberg,
Gibson, Janecki, Elsheikh, Matisak, Genge, Guidon, David, Habib, Mathew, Mozaffar,
Hewitt, Barnett, Sullivan, Ho, Traub, Chopra, Aly, Bayat, Abu-Rub, Khan, Lange, Holzberg,
Khatri, Lindman, Sershon, Lisak, Bernitsas, Jia, Malik, Lewis-Collins, Nicolle, Sharma,
Roy, Nye, Pulley, Berger, Shabbir, Sachdev, Patterson, Siddiqi, Sivak, Bratton, Small,
Kohli, Fetter, Lam, Harvey, Silvestri, Patrick, Zakalik, Pontius, Hoarty.
Drafting of the manuscript: Howard, Nowak, Duda, Farzaneh-Far, Kaminski, Barohn, Lewis-Collins, Nye, Sivak, Fetter,
Lam, Pontius.
Critical revision of the manuscript for important intellectual content: Howard, Nowak, Wolfe, Freimer, Vu, Hinton, Benatar, Duda, MacDougall, Farzaneh-Far,
Kaminski, Barohn, Dimachkie, Pasnoor, Farmakidis, Liu, Colgan, Bertorini, Pillai,
Henegar, Bromberg, Gibson, Janecki, Elsheikh, Matisak, Genge, Guidon, David, Habib,
Mathew, Mozaffar, Hewitt, Barnett, Sullivan, Ho, Traub, Chopra, Aly, Bayat, Abu-Rub,
Khan, Lange, Holzberg, Khatri, Lindman, Olapo, Sershon, Lisak, Bernitsas, Jia, Malik,
Nicolle, Sharma, Roy, Pulley, Berger, Shabbir, Sachdev, Patterson, Siddiqi, Bratton,
Small, Kohli, Harvey, Silvestri, Patrick, Zakalik, Pontius, Hoarty.
Statistical analysis: MacDougall, Farzaneh-Far, Barohn, Sivak, Hoarty.
Obtained funding: Farzaneh-Far, Barohn.
Administrative, technical, or material support: Vu, Hinton, Barohn, Henegar, Matisak, Genge, Guidon, Barnett, Sullivan, Chopra, Holzberg,
Bernitsas, Jia, Lewis-Collins, Nicolle, Roy, Nye, Shabbir, Patterson, Small, Fetter,
Lam, Pontius.
Supervision: Freimer, Duda, Farzaneh-Far, Kaminski, Barohn, Mozaffar, Khatri, Sershon, Shabbir,
Sachdev.
Conflict of Interest Disclosures: Dr Howard has received research support from Alexion Pharmaceuticals, argenx BVBA,
the US Centers for Disease Control and Prevention, the Muscular Dystrophy Association
(MDA), the National Institutes of Health (NIH; including the National Institute of
Neurological Disorders and Stroke and the National Institute of Arthritis and Musculoskeletal
and Skin Diseases), argenx and Ra Pharmaceuticals; honoraria/consulting fees from
Alexion Pharmaceuticals, argenx, Ra Pharmaceuiticals, and Viela Bio; and nonfinancial
support from Alexion Pharmaceuticals, argenx BVBA, Ra Pharmaceuticals, and Toleranzia.
Dr Nowak has received research support from the NIH, Genentech, Alexion Pharmaceuticals,
Ra Pharmaceuticals, Myasthenia Gravis Foundation of America, Momenta, and Grifols
and has served as consultant/advisor for Alexion Pharmaceuticals, CSL Behring, Grifols,
Ra Pharmaceuticals, Roivant Sciences, and Momenta. Dr Wolfe is a consultant for Grifols,
Takeda, argenx, and Momenta; is on speaker’s bureaus for Grifols and Alexion Pharmaceuiticals;
and has received grant/research support from Alexion Pharmaceuticals, argenx, Ra Pharmaceuticals,
and Immunovant. Dr Freimer has received honoraria from argenx and Alexion and research
support from Catalyst Pharmaceuticals, Ra Pharmaceuticals, Amicus Therapeutics, Alexion
Pharmaceuticals, Momenta, Alnylam Pharmaceuticals, UCB, Orphazyme, and NIH (NeuroNExt).
Dr Vu serves as site principal investigator for MG clinical trials sponsored by Alexion
Pharmaceuticals, argenx, NIH, Grifols, Ra Pharmaceuticals, and UCB and is on speaker’s
bureaus for Alexion Pharmaceuticals, Mitsubishi Tanabe Pharma America, CSL Behring,
and Allergan. Dr Benatar was site investigator for MG trials sponsored by UCB, Alexion
Pharmaceuticals, and an NIH-funded study of rituximab; has reported personal fees
from AveXis, Biogen, Genentech, Mitsubishi Tanabe Pharma, and Ra Pharmaceuticals;
has provided consulting services to UCB; and has a patent pending for determining
the onset of amyotrophic lateral sclerosis. Drs Duda and Farzaneh-Far and Mss Pontius
and Hoarty are employees of Ra Pharmaceuticals. Dr Kaminski has received grant 508240
from the MDA; is a consultant for Alnylam Pharmaceuticals, Ra Pharmaceuticals, and
UCB; and is CEO and CMO of ARC Biotechnology, LLC, based on US patent 8961981. Dr
MacDougall is a consultant for Ra Pharmaceuticals. Dr Barohn served as a consultant
for NuFactor and Momenta and receives research support from PTC Therapeutics, Ra Pharmaceuticals,
Orphazyme, Sanofi Genzyme, the US Food and Drug Administration (FDA) Office of Orphan
Products Development (OOPD), NIH, and Patient-Centered Outcomes Research Institute
(PCORI). Dr Dimachkie recently served as a consultant or on speaker’s bureaus for
Alnylam, Audentes, CSL Behring, Sanofi Genzyme, Momenta, NuFactor, RMS Medical, Shire
Takeda and Terumo and received grants from Alexion Pharmaceuticals, Alnylam Pharmaceuticals,
Amicus Therapeutics, Biomarin, Bristol-Myers Squibb, Catalyst, CSL Behring, FDA/OPD,
GlaxoSmithKline, Genentech, Grifols, MDA, NIH, Novartis, Genzyme, Octapharma, Orphazyme,
UCB, ViroMed Laboratories, and TMA Pharma. Dr Pasnoor has served as a medical advisor
or consultant for CSL Behring, Momenta, Alexion Pharmaceuticals, and Terumo BCT. Dr
Farmakidis has served as a consultant for Momenta and participated in an advisory
board for Terumo BCT. Dr Elsheikh received research support from Biogen, Alexion Pharmaceuticals,
and UCB and served as a consultant for Biogen and Bio-Stealth. Dr Gibson has received
travel subsidies and honoraria for time spent on advisory boards for Cytokinetics
and CSL Behring. Dr Genge is a consultant for Biogen, Sanofi Genzyme, CSL Behring,
Mitsubishi Tanabe Pharma America, AL-S Pharma, AB Sciences, Novartis, Wave Life Sciences,
argenx, Alexion Pharmaceuticals, AveXis, Akcea Therapeutics, and Cytokinetics. Dr
Guidon has been a consultant for Momenta; participated on a medical advisory board
with Alexion; has received royalties from Oakstone Publishing; and received clinical
trial support from Momenta and PCORI. Dr David has received royalties from Oakstone
Publishing. Dr Habib serves on advisory boards for Alexion Pharmaceuticals, and argenx;
is on a speaker’s bureau for Alexion Pharmaceuticals; and has received research support
from Alexion Pharmaceuticals, Ra Pharmaceuticals, Immunovant, UCB, Acceleron Pharma,
and Catalyst Pharmaceuticals. Ms Mathew served as the primary coordinator and evaluator
for myasthenia gravis clinical trials sponsored by Alexion Pharmaceuticals, argenx,
Grifols, Ra Pharmaceuticals, and UCB and is a consultant for Ra Pharmaceuticals. Dr
Mozaffar has received travel subsidies and honoraria for time spent on advisory boards
for aTyr Pharma, Alnylam Pharmaceuticals, Alexion Pharmaceuticals, Amicus Therapeutics,
argenx, Audentes Therapeutics, Sanofi Genzyme, Sarepta Therapeutics, Spark Therapeutics,
Mitsubishi Tanabe Pharma America, and Ultragenyx; served on speaker’s bureaus for
Alexion Pharmaceuticals, CSL Behring, Grifols, and Sanofi-Genzyme; received research
funding from the Myositis Association, the MDA, the NIH, Alexion Pharmaceuticals,
Amicus Therapeutics, argenx, aTyr Pharma, Bristol-Myers Squibb, Idera, Ionis Pharmaceuticals,
Grifols, Momenta, Ra Pharmaceuticals, Sanofi-Genzyme, Spark Therapeutics, UCB, Ultragenyx,
and Valerion Therapeutics; and serves on the data safety monitoring board for Acceleron
and AveXis. Dr Traub has received research support from Alnylam Pharmaceuticals, Akcea
Therapeutics, and Vertex Pharmaceuticals and received honoraria/consulting fees from
Alnylam Pharmaceuticals, Akcea Therapeutics, and CSL Behring. Dr Chopra has received
honoraria from Alexion Pharmaceuticals, and Ra Pharmaceuticals. Dr Khan has participated
in advisory boards for Alexion and received a Wellstone trainee clinical fellowship
award and NIH research grant 1U54HD08735-01 (2016-2018). Dr Lange has received research
support from Cytokinetics, Orion, Orphazyme, Northeast ALS Consortium (NEALS), Lilly/ALS
Pharma, ALS Association of Greater New York, Alexion Pharmaceuticals, Acorda Therapeutics,
Momenta, Immunovant, UCB, Ra Pharmaceuticals, Baxalta, CSL Behring, and the MDA. Ms
Holzberg has received research support from Cytokinetics, Orion, Orphazyme, NEALS,
Lilly/ALS Pharma, ALS Association of Greater New York, Alexion Pharmaceuticals, Acorda
Therapeutics, Momenta, Immunovant, UCB, Ra Pharmaceuticals, Baxalta, CSL Behring,
and the MDA. Dr Khatri has received financial compensation for speaking and research
from Teva, Bayer, Biogen, Sanofi Genzyme, Serono EMD, Novartis, Genentech, Acorda,
Mallinckrodt, Terumo, and Celgene. Mrs Sershon has received financial compensation
for speaking, advisory boards, and consulting from Biogen, Sanofi Genzyme, Serono
EMD, Novartis, Genentech, Celgene, and Alexion. Dr Lisak has received research support
from the National Multiple Sclerosis Society, the NIH, and Mallinckrodt; participated
in clinical trials for Teva, Chugai, MedImmune, Novartis, Genentech (Roche), Alexion
Pharmaceuticals, argenx, Ra Pharmaceuticals, and Catalyst Pharmaceuticals; has consulted
for and received honoraria from Novartis, argenx, Mallinckrodt, GLG Consulting, Slingshot
Consulting, Guidepoint Consulting, Insights Consulting, Schlesinger Group, Clearview
Consulting, Alpha Sites Consulting, Putnam Group Consulting, Health Advances, Haven
Consulting, Decision Resources Group, and ck2 Medical Communications; has received
honoraria from MedDay Pharmaceuticals; has participated in a speaker’s bureau for
Teva Pharmaceuticals; and receives publishing royalties from Blackwell Publishers
(
International Neurology, 2nd edition) and Oxford University Press (
Neuroimmunology). Dr Bernitsas has received research support from the NIH, Merck/EMD Serono, Genentech
(Roche), Sanofi Genzyme, Mallinckrodt, Chugai, MedImmune, and Novartis and participated
in speaker’s bureaus and/or consulted for Celgene, Biogen, Merck/EMD Serono, Bayer,
and Sanofi Genzyme. Dr Malik has received research support from Catalyst Pharmaceuticals
and PCORI and serves as the director of the MDA-funded clinic at Rush University Medical
Center. Ms Lewis-Collins has served as a site clinical research coordinator for Catalyst
Pharmaceuticals, PCORI, and Ra Pharmaceuticals. Dr Nicolle has received honoraria
from Alexion Pharmaceuticals. Dr Roy has received honoraria from Alexion Pharmaceuticals.
Dr Pulley has participated in advisory board meetings for Alexion Pharmaceuticals,
Bio Products Laboratory, Stealth BioTherapeutics, CSL Behring, Mitsubishi Tanabe Pharma
America, and Catalyst Pharmaceuticals. Dr Berger has received consulting fees for
Best Doctors Inc. Dr Sachdev has participated in speaker’s bureaus for CSL Behring,
Alexion Pharmaceuticals, Strongbridge Biopharma, and Akcea Therapeutics; has acted
as a consultant for argenx and Alexion Pharmaceuticals; has participated as a principal
investigator for Catalyst Pharmaceuticals, Alexion Pharmaceuticals, Momenta, Ra Pharmaceuticals,
Eisai Corporation, and the MDA; and has been a grant recipient for MDA. Ms Harvey
serves as a consultant for Syneos and Ra Pharmaceuticals. Dr Silvestri has served
as a consultant for Alexion and argenx, received royalties from Springer Publishing,
and has been a speaker for Strongbridge Biopharma. Dr Howard reported grants and nonfinancial
support from Ra Pharmaceuticals during the conduct of the study; grants, personal
fees, and nonfinancial support from Alexion Pharmaceuticals, and argenx BVBA; and
grants from MDA, US Centers for Disease Control and Prevention, and the NIH outside
the submitted work. Dr Nowak reported grants and personal fees from Ra Pharmaceuticals
during the conduct of the study; grants and personal fees from Alexion Pharmaceuticals,
Momenta, Immunovant, argenx; and grants from Annexon Biosciences and Grifols outside
the submitted work. Dr Wolfe reported grants from Ra Pharmaceuticals during the conduct
of the study; grants from argenx and CSL Behring; and personal fees from Alexion outside
the submitted work. Dr Freimer reported personal fees and other support from Ra Pharmaceuticals
during the conduct of the study and the submitted work. Dr Vu reported serving as
a speaker for Alexion; consulting work for argenx and UCB; and participating in trials
in MG sponsored by Alexion Pharmaceuticals, argenx, UCB, and Grifols. Dr Hinton reported
grants, personal fees, and nonfinancial support from Ra Pharmaceuticals during the
conduct of the study and nonfinancial support from Immunovant outside the submitted
work. Dr Benatar reported personal fees from Ra Pharmaceuticals and Viela Bio during
the conduct of the study; in addition, Dr Benatar had a patent to Determining Onset
of ALS pending. Dr Duda reported other support from Ra Pharmaceuticals during the
conduct of the study; in addition, Dr Duda had a patent to listed as an inventor on
a patent application relevant to this work pending. Dr MacDougall reported personal
fees from Ra Pharmaceuticals during the conduct of the study. Dr Farzaneh-Far is an
employee of Ra Pharmaceuticals and holds equity in the company. Dr Kaminski reported
personal fees from Ra Pharmaceuticals during the conduct of the study; personal fees
from UCB outside the submitted work; and serving as principal investigator for Rare
Disease Clinical Research Network for Myasthenia Gravis (MGNet, U54 NS115054). Dr
Dimachkie reported grants from University of Kansas during the conduct of the study.
Dr Farmakidis reported personal fees from Momenta and Terumo BCT outside the submitted
work. Dr Bromberg reported personal fees from Grifols, Alexion Pharmaceuticals, Accordant,
and Genzyme outside the submitted work. Dr Elsheikh reported grants and nonfinancial
support from Ra Pharmaceuticals during the conduct of the study; grants, personal
fees, and nonfinancial support from Biogen; and grants and nonfinancial support from
Alexion Pharmaceuticals, UCB, Momenta, and Catalyst Pharmaceuticals outside the submitted
work. Dr Genge reported being a site principal investigator for phase 2 study in MG.
Dr Guidon reported personal fees and other support from Momenta and other support
from Oakstone Publishing and Alexion outside the submitted work. Dr Habib reported
grants from Ra Pharmaceuticals during the conduct of the study; grants and other support
from Alexion Pharmaceuticals, and argenx; and grants from Immunovant outside the submitted
work. Dr Mathew reported personal fees from Ra Pharmaceuticals outside the submitted
work. Dr Chopra reported other support from Ra Pharmaceuticals and Alexion Pharmaceuticals,
and nonfinancial support from argenx during the conduct of the study. Dr Khan reported
personal fees from Alexion and Ra Pharmaceuticals and grants from NIH outside the
submitted work. Dr Lange reported grants from Ra Pharmaceuticals during the conduct
of the study and from Alexion Pharmaceuticals, Momenta, and Immunovant outside the
submitted work. Dr Holzberg reported grants from Ra Pharmaceuticals during the conduct
of the study and grants from NIH outside the submitted work. Dr Khatri reported personal
fees and nonfinancial support from Biogen, Alexion Pharmaceuticals, Genentech, Sanofi
Genzyme, Celgene, and Serono outside the submitted work. Dr Sershon reported personal
fees from Alexion Pharmaceuticals, Biogen, Sanofi Genzyme, Novartis, EMD Serono, and
Celgene outside the submitted work. Dr Lisak reported grants from Ra Pharmaceuticals
during the conduct of the study; grants from Alexion Pharmaceuticals, Genentech (Roche),
and MedImmune; grants and personal fees from argenx, Teva, and Catalyst Pharmaceuticals;
personal fees from Novartis, Mallinckrodt, GLG Consulting, Alpha Sites, Schelsinger
Group, Insights Consulting, Slingshots Consulting, Informa Pharmaceutical Consulting,
Health Choices, and Adivo Consulting; and other support from Anavex outside the submitted
work. Dr Bernitsas reported grants from Genentech (Roche), Chugai, and Novartis; and
personal fees from Biogen and EMD Serono outside the submitted work. Dr Malik reported
grants from Ra Pharmaceuticals during the conduct of the study and personal fees from
MDA and Conquer MG outside the submitted work. Dr Roy reported grants from Ra Pharmaceuticals
during the conduct of the study and personal fees from Alexion Pharmaceuticals, outside
the submitted work. Dr Pulley reported personal fees from Alexion Pharmaceuticals,
argenx, Bio Products Laboratory, Catalyst Pharmaceuticals, and CSL Behring outside
the submitted work. Dr Berger reported grants from University of Florida during the
conduct of the study. Dr Shabbir reported grants from Ra Pharmaceuticals during the
conduct of the study. Dr Sachdev reported grants from Ra Pharmaceuticals during the
conduct of the study; grants and personal fees from Alexion Pharmaceuticals; personal
fees from CSL Behring, Strongbridge Biopharma, Akcea Therapeutics, and argenx; and
grants from Catalyst Pharmaceuticals, Momenta, Eisai Corporation, and MDA outside
the submitted work. Dr Harvey reported personal fees from Alexion Pharmaceuticals,
Inc and Ra Pharmaceuticals outside the submitted work. Dr Silvestri reported personal
fees from Alexion Pharmaceuticals, and argenx outside the submitted work. Dr Pontius
is an employee and holds stock and stock options of Ra Pharmaceuticals outside the
submitted work. No other disclosures were reported.
Funding/Support: Editorial assistance and graphic support, provided by Jessica Deckman, PhD, of The
Lockwood Group (Stamford, Connecticut), was supported by funding from Ra Pharmaceuticals
Inc. This study was funded by Ra Pharmaceuticals Inc.
Role of the Funder/Sponsor: The funding source contributed to the design and conduct of the study; collection,
management, analysis, and interpretation of the data; preparation, review, or approval
of the manuscript; and the decision to submit the manuscript for publication.
Additional Contributions: We thank Andrea Bozoki, MD, Michigan State University, East Lansing, and Gilles Hoffman,
PhD, Rush University, Chicago, Illinois. No compensation was received from a funding
source.