2024 Annual Meeting

Overview of Lupus ABC’s Year One Accomplishments and Second Annual Meeting

The Lupus Accelerating Breakthroughs Consortium (Lupus ABC) is pleased to report on the progress made during its inaugural year, culminating in the successful second annual meeting held on April 15-16 in Bethesda, Maryland. Lupus ABC is a first-of-its-kind public-private partnership (PPP) uniting individuals with lupus and their advocates, industry, clinicians, researchers, and government stakeholders to collaboratively identify and pursue the most effective ways to accelerate the development of urgently needed personalized treatments for people with lupus.

Second Annual Meeting Highlights

The second annual meeting of Lupus ABC convened all stakeholders to review progress and chart future directions. In addition to an open plenary session held on the morning of April 16, meetings of the two governing bodies of the Consortium—the Lupus Voices Council (LVC) and the Research Committee (RC)—were held.

Plenary Session

The meeting commenced with opening remarks by Lupus Research Alliance (LRA) President and Chief Executive Officer Albert Roy and LRA Vice President and Chief Scientific Officer Teodora Staeva, Ph.D., who emphasized the power of the collective community through the strong partnerships cultivated and highlighted the Consortium’s ambitious goals, which were not only met but exceeded in its first year. Nikolay Nikolov, M.D., Acting Director of the Office of Immunology and Inflammation in the Center for Drug Evaluation and Research (CDER) at the Food and Drug Administration (FDA) expressed his enthusiasm to partner with Lupus ABC to advance drug development, emphasizing the critical role of collaboration in achieving breakthroughs. The opening remarks set the tone for an engaging meeting, emphasizing that people with lupus are at the center of and equal partners in this vital work.

A significant development highlighted at the meeting was the expansion of Lupus ABC’s partnership with the FDA to include the Center for Biologics Evaluation and Research (CBER). Keynote speaker Peter Marks, M.D., Ph.D., Director of CBER, presented on the promising potential of engineered cell therapies, such as CAR T cell therapies, for autoimmune diseases like lupus, reaffirming CBER’s commitment to collaborating with Lupus ABC to advance these treatments.

Updates were provided by RC co-chairs Nikolay Delev, M.D. (Bristol Myers Squibb) and Gary Koretzky, M.D., Ph.D. (Cornell University), who highlighted the rapid expansion of industry members in Lupus ABC’s first year as well as several year one activities including the establishment of the Executive Committee, convening of two exploratory meetings to refine the scope of future Working Groups, and the successful launch of four projects, described below. The RC co-chairs also outlined year two activities, including providing opportunities for broader community engagement, enacting new projects or Working Groups, and releasing Lupus ABC’s first Request For Proposals (RFP). LVC interim co-chair Anna Fisch, M.S. and co-chair Judith N. Mills, M.B.S. also presented year one accomplishments, including convening the first meeting of the fully constituted group and holding an ideation session on priority areas of focus for those living with lupus.

Chairs or members of Lupus ABC’s first four projects provided broad updates, highlighting their progress, goals, milestones, and future directions.

To conclude the plenary session, a dynamic panel discussion on lupus clinical trial design issues and inclusion of companion diagnostics was moderated by Eric Morand, M.D., Ph.D. (Monash University) and featured insights from experts across academia, industry, and the patient community, emphasizing the importance of patient perspectives in clinical trial design. Panelists included Richard Furie, M.D. (Northwell Health), Sandra Garces, M.D., Ph.D. (Bristol Myers Squibb), Joan Merrill, M.D. (Oklahoma Medical Research Foundation), Nikolay Nikolov, M.D. (CDER, FDA), and Narae Yun (LVC member).

Lupus Voices Council Meeting

The LVC convened to discuss several topics of interest to the lupus community, including the importance of trust and inclusiveness, support through patient advocacy and testimonials, and targeted educational resources. A Lupus ABC consultant presented five topics, which were discussed and prioritized by the group as potential projects that they could champion. LVC members expressed enthusiastic support for the development of a resource toolkit for patients and providers to guide them through the clinical trial process. Participants also emphasized the importance of support systems, partnering with those with lupus throughout the treatment development cycle, and a comprehensive approach to clinical trial engagement.

The LVC is responsible for providing the Lupus ABC with the perspectives, priorities, and needs of individuals with lupus, including the symptoms and experiences that matter most, as well as identifying mechanisms for ensuring that diverse community perspectives are reflected in the Lupus ABC’s efforts. The LVC will seek nominations from the community to fill openings in the future.

Research Committee Meeting

The RC convened in the afternoon of April 16 after the conclusion of the plenary session. Invited speaker Louis DeGennaro, Ph.D. (Cantlous Advisory Partners and previous President of the Leukemia and Lymphoma Society) shared insights from the Leukemia and Lymphoma Society’s BeatAML Master Trial, which utilized an innovative paradigm for conducting clinical trials. Assigning patients to sub-studies based on results from molecular, immunophenotypic, and/or biochemical studies demonstrated significant advances in precision medicine approaches for acute myeloid leukemia. In his presentation and the discussion that followed, Dr. DeGennaro emphasized the importance of genetic profiling and targeted therapies and how this approach could be applied to lupus.

Richard Furie, M.D. (Northwell Health) facilitated a discussion around lupus trial design, noting challenges such as the low number of patients eligible to enter trials and high placebo response. Dr. Furie also emphasized the necessity of addressing broader patient needs, including flare prevention and steroid-sparing, and led a robust discussion amongst RC members on these critical needs. Dr. Furie concluded by providing the committee with a list of topics to be addressed for improving clinical trial design. The RC suggested that these topics be explored further outside of Lupus ABC. Once more developed, they can be submitted for consideration in response to an RFP for new projects that will be released in Q4 of 2024.

Year One Project Updates

Since its inception, Lupus ABC has exceeded its initial target of establishing one to two projects during the first 12-18 months of operation by advancing four projects in just 12 months. During the RC session, co-chairs of three of the Lupus ABC Working Groups/projects provided updates on their challenges, opportunities, and 12-24-month goals, with input solicited from Innovator Members, to reach alignment on timelines and deliverables and formalize milestones. Below are summaries of the projects and updates provided at the annual meeting:

Treatment Response Measure for Systemic Lupus Erythematosus (TRM-SLE):

Led by Eric Morand, M.D., Ph.D. (Monash University), this project aims to develop a new measurement tool that captures meaningful treatment response for use in SLE clinical trials. The project was already in progress with strong support from the global community. Because of its promising potential, Lupus ABC became a partner to provide further support to bolster its success. A representative from CDER has been appointed to the steering committee to offer regulatory insights. Additionally, members of the LVC serve on both the Steering Committee and the Patient Advisory Board to provide additional patient perspectives.

The developed tool will be compared to the primary outcome measures currently used, SRI and BICLA. Dr. Morand noted that, while challenges they face include scale, complexity of the project, and operationalization, with support from academic collaborators and industry partners, the group aims to deliver a final product and manuscript in mid-late 2025.

Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Working Group:

Established in January 2024 and co-chaired by Benjamin Chong, M.D. (University of Texas Southwestern Medical Center) and Denesh Chitkara, M.D. (EMD Serono), this Working Group aims to refine CLASI to align with FDA standards for use as an outcome measure for cutaneous lupus in clinical trials. Comprised of members from the LVC, leading figures from academia, industry, and the FDA/CDER, the Working Group has identified the main concerns with the use of CLASI and is compiling published and unpublished data to address them.

Dr. Chitkara noted that, despite the limitations of the CLASI instrument, this Working Group provides the unprecedented opportunity to have an open dialogue and identify areas of agreement and disagreement with academics, industry, patients, and the FDA. The group outlined five points of concern with CLASI from the FDA and compiled data and responses to address them.

Chimeric Antigen Receptor (CAR) T Cell Working Group:

Launched in March 2024 and co-chaired by Elena Hsieh, M.D. (University of Colorado) and James Chung, M.D., Ph.D. (Kyverna Therapeutics), this Working Group was established to address outstanding questions on the use of CAR T cell therapy in lupus. The near-term goal of the project is to develop a position paper on the key considerations to advance CAR T cell therapies for lupus. The longer-term goal is to develop and launch a biomarker-based project for lupus CAR T therapy. The Working Group comprises experts from CBER/FDA, academia, industry, and members of the LVC.

Dr. Chung noted that while drafting the position paper presents challenges, including integrating perspectives from a large number of participants, there is a significant opportunity to standardize clinical trial design for CAR T in lupus and identify key areas of focus to accelerate regulatory approval.

Patient Reported Outcomes (PRO) for Lupus:

Before a Working Group is launched on this topic, a planning committee chaired by Patti Katz, Ph.D. (UCSF) is organizing a meeting on lupus PROs. Scheduled for October 2024, the meeting aims to bring together PRO experts to discuss the state of lupus PROs and establish a strategic roadmap for future working group efforts.

Additional Year One Accomplishments

In year one, Lupus ABC expanded its collaboration with the FDA, extending its partnership beyond the initial liaison with CDER to include CBER in February 2024. Lupus ABC has broadened the participation of other key stakeholders, including a 59% increase in industry participation (from 17 to 28 members as of April 2024), and welcomed the Lupus Foundation of America in October 2023, underscoring the Consortium’s collaborative approach toward advancing therapeutics for individuals with lupus.

Looking Ahead

As Lupus ABC enters its second year, the Consortium remains committed to advancing its mission through ongoing stakeholder engagement, project development, and partnership expansion. The RC and LVC will continue to meet quarterly, while Working Groups will meet monthly to advance the goals of the Lupus ABC projects. The dates and location for the third annual meeting in 2025 will be released shortly.

Plans for future initiatives, including an in-person meeting on PROs, are underway. An RFP for new projects will be released to the broader community in late 2024 and posted on the Lupus ABC website when available. The lupus community will be kept abreast of Lupus ABC developments via its website; visit LupusABC.org for more information and updates.

In its first year, Lupus ABC has made considerable progress. By leveraging the expertise and resources of its diverse stakeholders and expanding critical partnerships, the Consortium succeeded in advancing multiple projects, illustrating the power of a unified approach to tackling complex challenges in therapeutics development for lupus.