Abata Therapeutics Announces First Development Candidate, ABA-101, a Novel Treg Cell Therapy for the Treatment of Progressive Multiple Sclerosis

ABA-101 aims to benefit a subset of progressive MS patients with no treatment options

ABA-101 is currently in IND-enabling studies and on track to enter the clinic in 2024

BOSTON, December 14, 2022 – 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 that it has chosen its first development candidate, ABA-101, which has the potential to benefit 45,000 progressive multiple sclerosis (MS) patients in the U.S. who have no treatment options today.

“The progressive pathology of MS has not been effectively treated because existing therapies don’t address the CNS-compartmentalized inflammation that drives that aspect of the disease,” said Richard M. Ransohoff, M.D., co-founder and chief medical officer at Abata and venture partner at Third Rock Ventures. “Our approach of leveraging the natural role of Tregs in the immune system enables us to resolve complex inflammatory mechanisms, offering new hope for patients with no treatment options. Our work to date developing ABA-101 validates our approach of using TCR-targeted Treg therapies to treat autoimmune diseases. We look forward to working with our new CSO, Ellen Cahir-McFarland, to translate the science behind ABA-101 into clinical studies in the first half of 2024.” 

“Since our inception, we’ve been acutely focused on advancing transformative therapies for people with autoimmune diseases. We believe that our Treg product engine has yielded a candidate in ABA-101 that is truly differentiated and can have significant benefit for people living with progressive MS,” said Samantha Singer, M.S., M.B.A., president and chief executive officer of Abata. “ABA-101 utilizes a TCR to target our product specifically to the site of ongoing pathology and to offer the potential for a robust, highly durable effect. Working with our partners at ElevateBio BaseCamp, we’ve rapidly established a foundational manufacturing process in less than one year, showing we can successfully engineer and expand Tregs for use as therapies. Overall, this work gives us great confidence not only in our ABA-101 program, but also in our additional programs in type 1 diabetes and inclusion body myositis.”

“At ElevateBio, we enjoy collaborating with scientific innovators like Abata, helping power their novel cell and gene therapies,” said Michael Paglia, M.S., chief operating officer, ElevateBio BaseCamp. “It’s been a pleasure working with the Abata team creating and refining a manufacturing process that enables them to expand Tregs engineered with an antigen-specific TCR at yields that will support clinical development of ABA-101 and serves as a template for their future programs. We look forward to the continued partnership.”

The hypothesis for ABA-101 as a Treg treatment for progressive MS is firmly rooted in the scientific literature. Single-cell data link chronic active lesions in patients to the adaptive immune system, reinforcing the possibility that a Treg can have a therapeutic effect in progressive MS. Additionally, EAE (experimental autoimmune encephalomyelitis) mouse studies demonstrateproof of biology, including TCR-Treg trafficking, persistence, biological activity, and suppression of inflammation – all critical elements for a successful treatment. Additional non-clinical studies establish Tregs’role in myelin repair, a further potential benefit of the approach. Abata will use MRI to select patients for inclusion as clinical trial participants, as chronic active lesions (identifiable with MRI) correlate with worsening diseasein progressive MS natural history studies.

About ABA-101 and Progressive Multiple Sclerosis

ABA-101 is Abata’s autologous Treg therapy in development for the treatment of progressive MS. It targets MS patients with non-relapsing progressive disease who have a DRB1*15:01 genetic haplotype and for whom imaging evidence of ongoing inflammatory tissue injury has been observed – an estimated patient group of about 45,000 in the U.S. today. ABA-101 is created by minimally engineering a patient’s own Tregs to express a TCR that specifically recognizes immunogenic myelin fragments in the CNS – an approach that offers a potentially strong safety profile and a highly localized anti-inflammatory effect at the site of disease. In vitro, ABA-101 has demonstrated antigen-specific, dose-dependent Treg functionality, anti-inflammatory cytokine production, and suppression of the production of inflammatory factors (T cell-derived cytokines). ABA-101 also retains its Treg phenotype under inflammatory conditions.

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. In addition to its lead program in progressive MS, Abata has programs in type 1 diabetes and inclusion body myositis – two other tissue-specific autoimmune diseases with substantial unmet need and 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 Cambridge, Mass. Please visit abatatx.com or follow us on Twitter or LinkedIn for more information.

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