WATERTOWN, Mass.--(BUSINESS WIRE)-- Abata Therapeutics, a company focused on translating the biology of regulatory T cells (Tregs) into transformational medicines for patients living with severe autoimmune and inflammatory diseases, today announced its second development candidate, ABA-201, which has the potential to be a disease-modifying Treg cell therapy for patients with type 1 diabetes (T1D) who have remaining beta (β) cell function.

“The team has been incredibly successful building out our Treg cell therapy discovery engine, generating in only two years, two distinct therapeutic candidates for the treatment of two serious autoimmune diseases with high unmet need,” said Samantha Singer, M.S., M.B.A., president and chief executive officer of Abata. “We are rapidly developing ABA-201 for T1D and expect to begin clinical studies in 2025. This success builds on the scientific and operational infrastructure of our lead program, ABA-101, which is being developed to treat progressive multiple sclerosis and will enter clinical studies next year. We’re also thrilled to announce the extension of our partnership with ElevateBio. The successful partnership on our lead program affords the advantage of a ‘plug-and-play’ process that will greatly accelerate the advancement of the ABA-201 program.”

The hypothesis for ABA-201 as a Treg treatment for T1D is firmly rooted in the scientific literature. Results from preclinical models of insulitis, such as the nonobese diabetic (NOD) mouse, and clinical trials testing either immunomodulators or polyclonal Tregs in patients with at-risk or new-onset T1D, reinforce the possibility that autoantigen-specific Tregs can have a highly potent therapeutic effect in T1D. Additional non-clinical studies have established the role of Tregs in suppressing β-cell injury, a further potential benefit of the approach.

“ABA-201 is a Treg cell therapy that uses a TCR to target the pancreas and draining lymph nodes in patients with T1D to limit ongoing β-cell destruction and preserve residual β-cell mass, with the added potential to promote repair of damaged tissue and establish long-lived tissue residency. Suppressing T cell function has been shown to delay T1D onset. However, no therapeutic exists to truly halt the autoimmunity that drives T1D and durably recalibrate the immune response. We believe our targeted approach to suppressing pancreatic islet-associated inflammation could offer significant and long-lasting clinical benefit to patients,” said Leonard Dragone, M.D., Ph.D., chief medical officer of Abata. “This second program expands the scope of our pioneering Treg cell-based approach to patients with T1D. As someone who has spent much of my career caring for patients and working to treat autoimmune diseases, I’m truly excited to see what these candidates can do for patients.”

“A disease-modifying therapy for the treatment of T1D has been a goal of the JDRF T1D Fund since its inception. Approximately 64,000 patients are newly diagnosed with T1D each year in the U.S., and there are currently no approved therapies that reset immune tolerance and halt the loss of beta cell function in those patients,” said Steven St. Peter, M.D., managing director of JDRF T1D Fund, a part of JDRF International, the leading T1D research foundation. “We believe there is great potential in Treg-based therapeutic approaches to treat T1D by preserving beta cell mass, and thus improving clinical outcomes. The JDRF T1D Fund has been an investor in Abata from the beginning, and we will be supporting Abata and ABA-201 with great anticipation in the coming years.”

Abata continues to build upon its significant experience and leadership in autoimmune diseases. In addition to Gerald Nepom, M.D., Ph.D., senior advisor, Immune Tolerance Network (ITN), and emeritus director of the Benaroya Research Institute, Abata is announcing a distinguished set of clinical advisors to support the ABA-201 program. This group brings deep expertise in T1D pathogenesis and the clinical development of novel therapeutics. The T1D advisory team includes:

• Kevan Herold, M.D., C.N.H. Long Professor of Immunobiology and of Medicine (Endocrinology), Yale School of Medicine
• Linda DiMeglio, M.D., M.P.H., Professor of Pediatrics at Indiana University School of Medicine; Division Chief Pediatric Endocrinology and Diabetology
• Stephen Gitelman, M.D., Mary B. Olney, MD/KAK Distinguished Professor, Pediatric Diabetes and Clinical Research, University of California San Francisco
• Jason Gaglia, M.D., M.M.Sc., Section Head Clinical, Behavioral, and Outcomes Research, Joslin Diabetes Center; and Assistant Professor of Medicine, Harvard Medical School

About ABA-201 and Type 1 Diabetes
ABA-201 is Abata’s autologous Treg therapy in development for the treatment of T1D. It targets T1D patients who have remaining beta cell function and a specific HLA genetic haplotype. ABA-201 is created by minimally engineering a patient’s own Tregs to express a TCR that specifically recognizes immunogenic protein fragments in the pancreas and draining lymph nodes – an approach that offers a potentially strong safety profile and a highly localized anti-inflammatory effect at the site of disease. In vitro, ABA-201 has demonstrated antigen-specific, dose-dependent Treg functionality, anti-inflammatory cytokine production, and suppression of the production of inflammatory factors (T cell-derived cytokines). The number of new cases of T1D in the United States is estimated at 64,000 annually.

About Abata Therapeutics
Abata Therapeutics is focused on translating the biology of regulatory T cells (Tregs) into transformational medicines for patients with severe autoimmune and inflammatory diseases. Founded by pioneers in Treg biology, TCR and antigen discovery, disease pathogenesis, and molecular and imaging biomarkers, Abata has developed a differentiated product engine to create engineered Treg cell therapies that are tissue-specific, robust, and durable. Abata has two programs in IND-enabling stages - the lead program is in progressive multiple sclerosis (MS), and its second program is in type 1 diabetes. Both indications are tissue-specific autoimmune diseases with substantial unmet needs, supporting a strong rationale for Abata’s Treg approach. The company was launched in 2021 by Third Rock Ventures, with participation from a diverse syndicate of investors, including ElevateBio, Lightspeed Venture Partners, Invus, Samsara BioCapital, and the JDRF T1D Fund. Abata is based in Watertown, Mass. Please visit abatatx.com or follow us on Twitter or LinkedIn for more information.

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