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Werth VP, Culton DA, Concha JSS, et al. Safety, Tolerability, and Activity of ALXN1830 Targeting the Neonatal Fc Receptor in Chronic Pemphigus. J Invest Dermatol. 2021;141(12):2858-2865.e4doi: 10.1016/j.jid.2021.04.031.
Werth, V. P., Culton, D. A., Concha, J. S. S., Graydon, J. S., Blumberg, L. J., Okawa, J., Pyzik, M., Blumberg, R. S., & Hall, R. P. (2021). Safety, Tolerability, and Activity of ALXN1830 Targeting the Neonatal Fc Receptor in Chronic Pemphigus. The Journal of investigative dermatology, 141(12), 2858-2865.e4. https://doi.org/10.1016/j.jid.2021.04.031
Werth, Victoria P, et al. "Safety, Tolerability, and Activity of ALXN1830 Targeting the Neonatal Fc Receptor in Chronic Pemphigus." The Journal of investigative dermatology vol. 141,12 (2021): 2858-2865.e4. doi: https://doi.org/10.1016/j.jid.2021.04.031
Werth VP, Culton DA, Concha JSS, Graydon JS, Blumberg LJ, Okawa J, Pyzik M, Blumberg RS, Hall RP. Safety, Tolerability, and Activity of ALXN1830 Targeting the Neonatal Fc Receptor in Chronic Pemphigus. J Invest Dermatol. 2021 Dec;141(12):2858-2865.e4. doi: 10.1016/j.jid.2021.04.031. Epub 2021 Jun 12. PMID: 34126109.
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J Invest Dermatol. 2021 Dec;141(12):2858-2865.e4. doi: 10.1016/j.jid.2021.04.031. Epub 2021 Jun 12.

Safety, Tolerability, and Activity of ALXN1830 Targeting the Neonatal Fc Receptor in Chronic Pemphigus.

The Journal of investigative dermatology

Victoria P Werth, Donna A Culton, Josef S S Concha, James S Graydon, Laurence J Blumberg, Joyce Okawa, Michal Pyzik, Richard S Blumberg, Russell P Hall

Affiliations

  1. Department of Dermatology, Penn Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA; Dermatology Division, Corporal Michael J. Crescenz VAMC, Philadelphia, Pennsylvania, USA. Electronic address: [email protected].
  2. Department of Dermatology, School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.
  3. Department of Dermatology, Penn Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA; Dermatology Division, Corporal Michael J. Crescenz VAMC, Philadelphia, Pennsylvania, USA.
  4. Alexion Pharmaceuticals, Inc, Boston, Massachusetts, USA; Syntimmune, Boston, Massachusetts, USA.
  5. Syntimmune, Boston, Massachusetts, USA.
  6. Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
  7. Department of Dermatology, Duke University Medical Center, Durham, North Carolina, USA.

PMID: 34126109 DOI: 10.1016/j.jid.2021.04.031

Abstract

Pemphigus is a debilitating IgG-mediated autoimmune disease requiring better tolerated, more targeted, and rapid onset therapies. ALXN1830 is a humanized IgG4 antibody that blocks neonatal Fc receptor interactions with IgG. A multicenter, open-label safety and tolerability phase 1b/2 trial (NCT03075904) was conducted in North America from July 2017 to January 2019 and included patients aged ≥18 years with a confirmed diagnosis of pemphigus (vulgaris or foliaceus) and active disease. Dosing included five weekly intravenous doses of ALXN1830 (10 mg/kg) and follow-up through day 112 (study termination). Pharmacokinetics, pharmacodynamics, safety, and efficacy, as evaluated by determining the change in the median pemphigus disease area index, were determined. In this pilot study of eight patients, five weekly infusions of ALXN1830 produced a rapid improvement in the pemphigus disease area index score within 14 days of the first dose. Pemphigus disease area index improvement increased further together with reductions in IgG, circulating immune complexes of IgG, and anti-desmoglein antibodies without affecting albumin, IgM, IgA, or C-reactive protein levels. ALXN1830 was well-tolerated, with headache as the most common adverse event. This study reveals the importance of neonatal Fc receptor in the biology of pemphigus and the potential for use of ALXN1830 in pemphigus treatment.

Published by Elsevier Inc.

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