2025 Annual Meeting

Overview of Lupus ABC’s Third Annual Meeting and Year Two Accomplishments

The Lupus Accelerating Breakthroughs Consortium (Lupus ABC) convened its third annual meeting on March 25-26, 2025, in Washington, D.C. The milestone event marked two years since the consortium’s establishment as the public-private partnership (PPP) with the U.S. Food and Drug Administration (FDA) dedicated to accelerating the development of safer and more effective therapeutics for lupus. Lupus ABC brings together people living with lupus and their advocates, along with researchers, clinicians, industry members, and regulators, in a collaborative effort to overcome barriers to drug development, placing the needs of those living with lupus at the center of its mission.

Meeting Highlights

This year’s meeting welcomed 113 Lupus ABC members, including members from the Lupus Voices Council (LVC), comprised of patient advocates living with lupus and/or their care partners, and the Research Committee (RC), comprised of representatives from the patient community, academia, industry, medical community, and government agencies (including the FDA). Attendance from every group surpassed last year’s numbers, reflecting broad and growing engagement across the consortium.

A keynote presentation by Janet Woodcock, MD, former Acting Commissioner of the FDA, on the unique power of public private partnerships set the stage for a dynamic meeting including the synthesis of the tremendous progress made by Lupus ABC in the past year by its Working groups, an insightful panel discussion on cognitive effect of lupus, and a highly stimulating discussion on immune resetting treatments for lupus.

Plenary Session

The plenary session opened with remarks from U.S. Food and Drug Administration (FDA) officials. Nikolay Nikolov, MD, from the Center for Drug Evaluation and Research (CDER), reaffirmed the FDA’s commitment to engaging people living with lupus and the broader lupus community to accelerate the development of safer and more effective treatments. Nicole Verdun, MD, from the Center for Biologics Evaluation and Research (CBER), echoed these sentiments. She acknowledged the promising potential of cell and gene therapy for lupus but highlighted the continued need for innovative approaches that can deliver safe treatments that are durable and accessible.

A moving performance titled “A Portrait of a Life with Lupus” wasdelivered by Shanelle Gabriel, MEd—an artist, consultant, and LVC member.She shared her personal journey navigating lupus symptoms and concluded with a powerful call to action: “Please do not let Lupus ABC be the only reason that the patient voice is in the room.” Her moving words on the struggles of living with lupus and the strength to persevere drew a standing ovation. It was a powerful reminder of the human impact and the urgency of Lupus ABC’s mission.

Dr. Janet Woodcock spoke on the transformative power of PPPs to overcome drug development barriers by enabling collaborative innovation. PPPs provide operational support for efforts such as tool development that require resources and collaboration that individual entities cannot achieve on their own. She emphasized the unique challenges of lupus heterogeneity and asserted that precision medicine in lupus will require novel regulatory, development, and statistical paradigms as the field advances.

Key updates followed from Nikolay Delev, MD (RC Co-Chair), Judith Mills, MBS, and Veronica Vargas Lupo, MBA (LVC Co-Chairs), along with co-chairs of individual working groups and projects (described in detail below).

The plenary session concluded with a panel discussion moderated by Meggan Mackay, MD, (Feinstein Institutes for Medical Research) on the cognitive impact of lupus. Panelists included Alvina Chu, MD (AbbVie), Raj Nair, MD (CDER/FDA), Erika Reyna, MAA (LVC member and person living with lupus), Zahi Touma, MD, PhD (University of Toronto), and Narae Yun (LVC member and person living with lupus). LVC members shared personal accounts of cognitive challenges like brain fog, including difficulty in finding the right words, memorizing, and organizing thoughts. Other panelists discussed the complexity of measuring cognitive impairments in lupus and the variability of symptoms across patients. This session emphasized the need to assess cognitive impacts— one of key focus of Lupus ABC’s upcoming patient-reported outcomes (PRO) project led by Dr. Touma.

Research Committee Meeting

The RC held a closed meeting moderated by RC Co-Chairs Dr. Delev and Gary Koretzky, MD, PhD (Weill Cornell Medicine). Maximilian Konig, MD (Johns Hopkins University School of Medicine), presented on “Immune Effector Cell Therapies in SLE: Opportunities, Challenges, and Next Steps.” Dr. Konig spoke of the immense potential of these therapies to achieve durable remission and “immune reset” in systemic lupus erythematosus (SLE). He outlined their challenges including toxicity, manufacturing failure risks, management of flares during treatment, and the need for conditioning chemotherapy prior to treatment. He also highlighted promising modalities such as T cell engagers, as well as cell therapy products manufactured from a single donor (allogeneic) or inside the patient’s body (in vivo). In particular, he discussed the unique advantages of bispecific T cell engagers in lupus, including the lack of conditioning therapy, scalability in manufacturing, and reduced risks of secondary infections.

Following Dr. Konig’s presentation, Barbara Mittleman, MD (AbelZeta) and Dr. Delev facilitated a discussion on expanding the chimeric antigen receptor (CAR) T Cell Therapy Working Group to include a broader therapeutic focus. They encouraged the RC to consider novel, impactful project ideas that can leverage the PPP model to accelerate the development of these immune cell engaging therapies. Members agreed to refine project ideas for the next phase of the working group in a future exploratory meeting.

The session concluded with discussions on the next phase of several key Lupus ABC projects (detailed below).

Lupus Voices Council Meeting

The LVC also convened a closed session as part of the Lupus ABC annual meeting, beginning with a workshop to develop an abstract for the American College of Rheumatology Annual Convergence Meeting. The abstract focuses on the innovative patient-centered governance structure of Lupus ABC and its potential to be a model for PPPs in other disease areas.

Patti Katz, PhD (University of California, San Francisco) presented updates on the PRO project and discussed one component of the proposed PRO project: analyzing how PROs from completed clinical trials correspond with improvements in clinical disease measures, to determine which PRO measures are best suited for measuring lupus outcomes.

Benjamin Chong, MD (University of Texas Southwestern), also presented an update on the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) working group. Dr. Chong emphasized the connection between CLASI scores and patient quality of life and discussed defining thresholds for clinically meaningful changes in CLASI score, which can help define appropriate endpoints.

Year Two Project Updates

During the plenary session, Dr. Delev and the co-chairs of the Lupus ABC working groups/projects provided updates on their progress in Year 2. In the closed RC and LVC meetings, the project leaders discussed next steps and future goals.

Treatment Response Measures for Systemic Lupus Erythematosus (TRM-SLE) Project

Eric Morand, MD, PhD (Monash University), provided an update on the TRM-SLE project, which aims to develop and validate a new clinical outcome assessment for use in clinical trials as a measure of treatment response. The group undertook a multi-step process to address the limitations of existing treatment response measures, including a literature review to define the unmet need and approach, a consensus process for what domains to include within the measurement, followed by a series of consensus processes within each measurement domain to identify best existing measurements, potential gaps, and response definitions. Based on these efforts, the group recently published a paper (https://lupus.bmj.com/content/lupusscimed/12/1/e001484.full.pdf) on the domains that met consensus for inclusion in the TRM-SLE assessment.

The final stages of TRM-SLE development will address outstanding questions from these efforts and integrate the domains into a finalized measurement instrument.

CLASI Working Group

Dr. Chong discussed the major barrier that the CLASI working group aims to solve—the lack of consensus around appropriate clinical trial outcome measures for evaluating cutaneous lupus erythematosus (CLE), which delays and prevents drug development for CLE. The working group focused on CLASI as the most promising CLE outcome measure and compiled evidence to address the FDA concerns with its use in lupus clinical trials. Data and written responses were compiled and shared with FDA representatives in August 2024, and the working group achieved consensus that CLASI is a suitable outcome measure for assessment of CLE response in clinical trials.

This milestone acknowledgement by the FDA opens the door for drug development in cutaneous lupus. Future working group efforts (led by Dr. Chong) will specifically address what threshold of CLASI activity improvement is clinically meaningful and acceptable as a primary endpoint and what minimum CLASI activity score is needed for inclusion in clinical trials.

CAR T-cell Therapy Working Group

Dr. Delev presented an update on the CAR T-cell therapy working group, co-chaired by Elena Hsieh, M.D. (University of Colorado), and James Chung, M.D., Ph.D. (formerly at Kyverna, currently at Amgen). In 2024, the working group met for six discussion-based meetings focused on considerations for eligibility for lupus patients enrolling in CAR T-cell therapy trials, clinical development considerations in the early and late phases, considerations on lymphodepletion pre-conditioning regimens in lupus, lupus-specific immune monitoring, and pre-clinical lupus models for studying cell therapy. These discussions were incorporated into a position paper that was in the final stages of revision prior to journal submission at the time of the meeting. Dr. Delev also summarized the key call to action statements in the manuscript to set the stage for a potential next phase of the working group.

Patient Reported Outcomes (PROs) for Lupus Project

Dr. Katz presented an update on Lupus ABC’s PRO-focused efforts. On October 16-17, 2024, Lupus ABC convened a meeting focused on PROs to establish the current state of PROs in lupus and to develop a roadmap to advance lupus PROs for use as endpoints in clinical trials. The meeting comprised 70 attendees from academia, industry, patient advocacy organizations, FDA, other government representatives, as well as people with lupus and their care partners. It began with a panel discussion on the importance of PROs to the lupus community, moderated by and composed of individuals living with lupus and their care partners. It was followed by presentations by key experts on lupus PROs. Breakout discussions focused on specific measurement domains and their rationale, selection of PROs for specific trials, appropriate questionnaire burden, and potential next steps to move the effort forward. The meeting discussions and outcomes are being synthesized into a position paper for publication.

The meeting led to the proposal to develop two PRO-focused projects, which were presented to the RC. Dr. Touma outlined a plan to identify and select measurement domains that are clinically meaningful to patients and to identify relevant PRO measures best suited to each domain. Dr. Katz outlined a proposal to analyze existing PRO measurement data from clinical trials to determine which PRO measures have the strongest supporting evidence for use as endpoints in clinical trials. Results from both projects will support best-practice recommendations for PROMs.

Additional Year Two Accomplishments and Looking Ahead

Overall, Lupus ABC delivered significant advancements during its second year of operations and will continue to build on this momentum during Year 3. Major accomplishments from Year 2 include:

The FDA’s acknowledgement of CLASI as a reasonable outcome measurement for assessing treatment response in CLE.
The successful convening of an in-person meeting on PROs that led to the approval of two projects to advance the development and incorporation of PROs as outcome measures in lupus clinical trials.
Preparation of 5 manuscripts (2 published, 3 in final stages) on working group activities and Lupus ABC in general.

Looking ahead to Year 3, the Lupus ABC will finalize and publish the remaining three manuscripts: a report on the PRO meeting outcomes, a perspective on the CAR T–cell Therapy working group’s considerations for advancing cell therapy in lupus, and the CLASI working group’s recommendation for the use of CLASI to evaluate treatment response in CLE trials (manuscript provisionally accepted at Nature Reviews Rheumatology). The CLASI working group will initiate and launch a new project focused on defining a clinically meaningful threshold of change for the outcome measure. Lupus ABC will also harness the momentum of the PRO meeting into two new PRO-focused projects. Lupus ABC will also hold strategic planning discussions on the next phase and scope of the CAR T cell therapy working group. A new Request for Proposals for additional projects will be released in 2025.