Abata Therapeutics Announces FDA Clearance of Investigational New Drug Application for Clinical Evaluation of ABA-101 in Progressive Multiple Sclerosis

First open IND for Abata’s best-in-class autologous T cell receptor (TCR)-Treg cell therapy platform for autoimmune disease

Expected initiation of first-in-human Phase 1 study in patients by end of year

WATERTOWN, MA, July 9, 2024 – 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 the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application to initiate the first-in-human (FIH) Phase 1 study of ABA-101 in patients with progressive multiple sclerosis (MS).

“We are excited to have reached IND clearance for our first product candidate, ABA-101, a tissue-specific TCR-Treg cell therapy for progressive MS. This represents not only an important milestone for Abata but also a significant advancement in the field as one of the first tissue-targeted Treg cell therapies for autoimmune disease,” said Samantha Singer, M.S., M.B.A., president and chief executive officer of Abata. “In preclinical studies, ABA-101 was shown to be safe and demonstrated tissue-specific trafficking, persistence, and robust suppression of inflammation, supporting its potential therapeutic effect. The quality of the data package, including a cutting-edge precision medicine trial design for MS, supported rapid clearance of our IND in less than a month. We look forward to initiating our Phase 1 study later this year in patients and evaluating the potential impact of ABA-101 on this progressive, devastating disease.”

“Across the MS treatment landscape, there are no effective therapies that address the CNS-compartmentalized inflammation driving progressive multiple sclerosis. ABA-101 is uniquely positioned to directly ameliorate this progressive pathology as well as promote repair and clinical improvement, offering life-changing benefits to those living with this disease today,” said Richard M. Ransohoff, M.D., co-founder and advisor of Abata and venture partner at Third Rock Ventures. “Receiving IND clearance for ABA-101 validates our unique approach of leveraging the natural role of Tregs in the immune system to treat autoimmune diseases where current treatments are either insufficient or non-existent.”

About ABA-101 and Progressive Multiple Sclerosis

ABA-101 is Abata’s autologous Treg therapy in development for the treatment of progressive multiple sclerosis. It was specifically designed for MS patients with progressive disease who have imaging evidence of ongoing inflammatory tissue injury and are HLA-DRB1*15:01 positive – an estimated patient group of about 45,000 in the U.S. today. ABA-101 is created by engineering a patient’s own Tregs to express a T cell receptor (TCR) that specifically recognizes immunogenic myelin fragments in the CNS. This approach was designed to offer a strong safety profile and a highly localized anti-inflammatory effect at the site of disease. In in vivo preclinical studies, ABA-101 was well-tolerated and demonstrated antigen-dependent Treg functionality, anti-inflammatory cytokine production, suppression of the production of inflammatory cytokines and therapeutic effect. 

About Abata Therapeutics

Abata Therapeutics is dedicated to translating the biology of 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’s lead program in progressive MS is on track to initiate a Phase 1 study by the end of 2024 and its second program in type 1 diabetes is in IND-enabling studies. Both indications are tissue-specific autoimmune diseases with substantial unmet need, supporting a strong rationale for Abata’s Treg approach. The company was launched in 2021 by Third Rock Ventures, and today is supported by a diverse syndicate of investors, including ElevateBio, Lightspeed Venture Partners, Invus, Samsara BioCapital, Eurofarma, the JDRF T1D Fund, and Biogen. Abata is based in Watertown, Mass. Please visit abatatx.com or follow us on X (formerly known as Twitter) or LinkedIn for more information.

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