ACR Meeting Abstracts

ACR Meeting Abstracts

  • Meetings
    • ACR Convergence 2024
    • ACR Convergence 2023
    • 2023 ACR/ARP PRSYM
    • ACR Convergence 2022
    • ACR Convergence 2021
    • ACR Convergence 2020
    • 2020 ACR/ARP PRSYM
    • 2019 ACR/ARP Annual Meeting
    • 2018-2009 Meetings
    • Download Abstracts
  • Keyword Index
  • Advanced Search
  • Your Favorites
    • Favorites
    • Login
    • View and print all favorites
    • Clear all your favorites
  • ACR Meetings

Abstract Number: 3033

Safety and Efficacy of Single Dose VAY736 (anti-BAFF-R mAb) in Patients with Primary Sjögren’s Syndrome (pSS)

Thomas Doerner1, Maximilian Posch2, Frank Wagner2, Andreas Hueser2, Thomas Fischer3, Louise Mooney4, Olivier Petricoul4, Paul Maguire4, Parasar Pal5, Julie Doucet4, Maciej Cabanski4, Esther Kamphausen4, Remi Kazma4 and Stephen Oliver4, 1Rheumatology and Clinical Immunology, Charité – Universitätsmedizin, Berlin, Germany, 2Charité Research Organisation GmbH, Berlin, Germany, 3Institut für Radiologie und Kinderradiologie, Charité - Universitätsmedizin, Berlin, Germany, 4Novartis Pharma AG, Basel, Switzerland, 5Novartis Pharma AG, Hyderabad, India

Meeting: 2016 ACR/ARHP Annual Meeting

Date of first publication: September 28, 2016

Keywords: B cell targeting, BAFF and Sjogren's syndrome

  • Tweet
  • Click to email a link to a friend (Opens in new window) Email
  • Click to print (Opens in new window) Print
Session Information

Date: Tuesday, November 15, 2016

Title: Sjögren's Syndrome I: Clinical Insights

Session Type: ACR Concurrent Abstract Session

Session Time: 2:30PM-4:00PM

Safety and efficacy of single dose VAY736 (anti-BAFFR mAb) in patients with primary Sjögren’s syndrome (pSS) T Dörner1, M Posch2, F. Wagner2, A Hüser2, T Fischer1, L Mooney3, O Petricoul3, P Maguire3, P Pal3, J Doucet3, M Cabanski3, E Kamphausen3, R Kazma3, S Oliver3 1Charité Hospital, Berlin; 2Charité Research Organisation, Berlin; 3Novartis Institutes for Biomedical Research, Basel

Background/Purpose: VAY736 is a novel, defucosylated, human IgG1 mAb targeting the receptor for B cell activating Factor of the TNF family (BAFF-R), providing both enhanced antibody-dependent cellular cytotoxicity-mediated depletion of B cells and blockade of BAFF:BAFF-R signaling that drives B cell differentiation, proliferation and survival. We evaluate here safety and efficacy of the dual mechanisms of action of VAY736 in patients with pSS, a highly BAFF-driven, systemic autoimmune disease involving lymphocytic infiltration and progressive dysfunction of exocrine glands along with various extra-glandular manifestations.

Methods: A single center, randomized, parallel group, double-blind, placebo-controlled trial recruited 27  seropositive pSS patients with EULAR Sjögren’s Syndrome Disease Activity Index (ESSDAI) ≥6 over a 10-month period for treatment with intravenous VAY736 at either a single high dose, (n=12), a single lower dose (n=6), or with placebo (n=9). Outcomes were measured at baseline and at weeks (w)6, 12 and 24. The primary outcome was change in ESSDAI at w12. Secondary outcomes included the EULAR Sjögren‘s Syndrome Patient Reported Index (ESSPRI), Short Form-36 (SF-36), Multidimensional Fatigue Inventory (MFI), physician/patient global VAS assessments, salivary flow rate, Ocular Staining Score (OSS), high resolution salivary gland ultrasound (US) de Vita scores/perfusion and elastography, serum markers of B cell hyperactivity and flow cytometry-determined lymphocyte subsets.

Results: Analyses to w24 included all 27 patients. VAY736 was safe and well-tolerated with no drug-related SAE, drop outs or discontinuations. Mean age was 50.5 years with 4 males, 2 in placebo and 1 in each treated arm. Baseline mean ESSDAI scores (range) were 11.5 (6-18), 14.5 (6-31) and 11.1 (6-19) in the high dose, lower dose and placebo arms, respectively. Target engagement was confirmed by serum BAFF levels and rapid, profound depletion of circulating B cells in all VAY736-treated patients; up to 99% by 24h and remaining so to 12-24w and beyond. The primary endpoint of ESSDAI was reduced within 12w but did not reach clinical or statistical significance versus placebo. However, improvements for VAY736-treated subjects were seen across clinical secondary outcomes, particularly for patient and physician global assessments and SF-36 physical. Reductions in parotid gland stiffness by US suggest early signs of salivary gland improvement. B cell hyperactivity markers were also reduced in treated patients. There were no consistent changes in salivary flow rate or OSS.

Conclusion: Despite a limited, single infusion, VAY736 achieved in this early phase trial trends for improvement in the primary outcome and across all secondary outcomes. Thus, this treatment was safe and suggests a positive therapeutic effect for this dual mechanisms of action in pSS that warrant further evaluation.


Disclosure: T. Doerner, Novartis Pharmaceutical Corporation, 5,UCB, 5; M. Posch, None; F. Wagner, None; A. Hueser, None; T. Fischer, None; L. Mooney, Novartis Pharmaceutical Corporation, 3; O. Petricoul, Novartis Pharmaceutical Corporation, 3; P. Maguire, Novartis Pharmaceutical Corporation, 3; P. Pal, Novartis Pharmaceutical Corporation, 3; J. Doucet, Novartis Pharmaceutical Corporation, 3; M. Cabanski, Novartis Pharmaceutical Corporation, 3; E. Kamphausen, Novartis Pharmaceutical Corporation, 3; R. Kazma, Novartis Pharmaceutical Corporation, 3; S. Oliver, Novartis Pharmaceutical Corporation, 3.

To cite this abstract in AMA style:

Doerner T, Posch M, Wagner F, Hueser A, Fischer T, Mooney L, Petricoul O, Maguire P, Pal P, Doucet J, Cabanski M, Kamphausen E, Kazma R, Oliver S. Safety and Efficacy of Single Dose VAY736 (anti-BAFF-R mAb) in Patients with Primary Sjögren’s Syndrome (pSS) [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). https://acrabstracts.org/abstract/safety-and-efficacy-of-single-dose-vay736-anti-baff-r-mab-in-patients-with-primary-sjogrens-syndrome-pss/. Accessed .
  • Tweet
  • Click to email a link to a friend (Opens in new window) Email
  • Click to print (Opens in new window) Print

« Back to 2016 ACR/ARHP Annual Meeting

ACR Meeting Abstracts - https://acrabstracts.org/abstract/safety-and-efficacy-of-single-dose-vay736-anti-baff-r-mab-in-patients-with-primary-sjogrens-syndrome-pss/

Advanced Search

Your Favorites

You can save and print a list of your favorite abstracts during your browser session by clicking the “Favorite” button at the bottom of any abstract. View your favorites »

All abstracts accepted to ACR Convergence are under media embargo once the ACR has notified presenters of their abstract’s acceptance. They may be presented at other meetings or published as manuscripts after this time but should not be discussed in non-scholarly venues or outlets. The following embargo policies are strictly enforced by the ACR.

Accepted abstracts are made available to the public online in advance of the meeting and are published in a special online supplement of our scientific journal, Arthritis & Rheumatology. Information contained in those abstracts may not be released until the abstracts appear online. In an exception to the media embargo, academic institutions, private organizations, and companies with products whose value may be influenced by information contained in an abstract may issue a press release to coincide with the availability of an ACR abstract on the ACR website. However, the ACR continues to require that information that goes beyond that contained in the abstract (e.g., discussion of the abstract done as part of editorial news coverage) is under media embargo until 10:00 AM ET on November 14, 2024. Journalists with access to embargoed information cannot release articles or editorial news coverage before this time. Editorial news coverage is considered original articles/videos developed by employed journalists to report facts, commentary, and subject matter expert quotes in a narrative form using a variety of sources (e.g., research, announcements, press releases, events, etc.).

Violation of this policy may result in the abstract being withdrawn from the meeting and other measures deemed appropriate. Authors are responsible for notifying colleagues, institutions, communications firms, and all other stakeholders related to the development or promotion of the abstract about this policy. If you have questions about the ACR abstract embargo policy, please contact ACR abstracts staff at [email protected].

Wiley

  • Online Journal
  • Privacy Policy
  • Permissions Policies
  • Cookie Preferences

© Copyright 2025 American College of Rheumatology