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

Publication ,  Journal Article
Werth, VP; Culton, DA; Concha, JSS; Graydon, JS; Blumberg, LJ; Okawa, J; Pyzik, M; Blumberg, RS; Hall, RP
Published in: J Invest Dermatol
December 2021

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.

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Published In

J Invest Dermatol

DOI

EISSN

1523-1747

Publication Date

December 2021

Volume

141

Issue

12

Start / End Page

2858 / 2865.e4

Location

United States

Related Subject Headings

  • Young Adult
  • Receptors, Fc
  • Pemphigus
  • Middle Aged
  • Male
  • Immunoglobulin G
  • Humans
  • Histocompatibility Antigens Class I
  • Female
  • Dermatology & Venereal Diseases
 

Citation

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Werth, V. P., Culton, D. A., Concha, J. S. S., Graydon, J. S., Blumberg, L. J., Okawa, J., … Hall, R. P. (2021). Safety, Tolerability, and Activity of ALXN1830 Targeting the Neonatal Fc Receptor in Chronic Pemphigus. J Invest Dermatol, 141(12), 2858-2865.e4. https://doi.org/10.1016/j.jid.2021.04.031
Werth, Victoria P., Donna A. Culton, Josef S. S. Concha, James S. Graydon, Laurence J. Blumberg, Joyce Okawa, Michal Pyzik, Richard S. Blumberg, and Russell P. Hall. “Safety, Tolerability, and Activity of ALXN1830 Targeting the Neonatal Fc Receptor in Chronic Pemphigus.J Invest Dermatol 141, no. 12 (December 2021): 2858-2865.e4. https://doi.org/10.1016/j.jid.2021.04.031.
Werth VP, Culton DA, Concha JSS, Graydon JS, Blumberg LJ, Okawa J, et al. Safety, Tolerability, and Activity of ALXN1830 Targeting the Neonatal Fc Receptor in Chronic Pemphigus. J Invest Dermatol. 2021 Dec;141(12):2858-2865.e4.
Werth, Victoria P., et al. “Safety, Tolerability, and Activity of ALXN1830 Targeting the Neonatal Fc Receptor in Chronic Pemphigus.J Invest Dermatol, vol. 141, no. 12, Dec. 2021, pp. 2858-2865.e4. Pubmed, doi: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.
Journal cover image

Published In

J Invest Dermatol

DOI

EISSN

1523-1747

Publication Date

December 2021

Volume

141

Issue

12

Start / End Page

2858 / 2865.e4

Location

United States

Related Subject Headings

  • Young Adult
  • Receptors, Fc
  • Pemphigus
  • Middle Aged
  • Male
  • Immunoglobulin G
  • Humans
  • Histocompatibility Antigens Class I
  • Female
  • Dermatology & Venereal Diseases