Abata Therapeutics Receives FDA Clearance for ABA-101 in Progressive Multiple Sclerosis

Abata Therapeutics, based in Watertown, Massachusetts, has received clearance from the U.S. Food and Drug Administration (FDA) to conduct a first-in-human (FIH) Phase 1 study of their novel cell therapy, ABA-101, for progressive multiple sclerosis (MS). This marks a significant milestone for Abata, which focuses on developing regulatory T cell (Treg) therapies to treat severe autoimmune and inflammatory diseases.

ABA-101 is a tissue-specific T cell receptor (TCR)-Treg cell therapy designed for progressive MS, a disease characterized by central nervous system (CNS) inflammation and tissue injury. Samantha Singer, CEO of Abata, expressed excitement over the IND clearance, highlighting the therapy’s potential to address the critical unmet needs of MS patients. Singer noted that preclinical studies indicated ABA-101's safety and efficacy, showing promising results in tissue-specific trafficking, persistence, and suppression of inflammation.

Richard M. Ransohoff, co-founder and advisor of Abata, emphasized the unique position of ABA-101 in the MS treatment landscape. Current therapies do not effectively target the CNS-compartmentalized inflammation that drives progressive MS. ABA-101, by leveraging the natural role of Tregs, aims to directly reduce this inflammation and promote repair, potentially offering life-changing benefits to patients.

ABA-101 is particularly designed for MS patients who have imaging evidence of ongoing inflammatory tissue injury and are HLA-DRB1*15:01 positive, a group estimated to include around 45,000 individuals in the U.S. The therapy involves engineering a patient’s own Tregs to express a T cell receptor that specifically recognizes immunogenic myelin fragments in the CNS. This targeted approach aims to provide a localized anti-inflammatory effect at the site of disease. Preclinical studies have shown ABA-101 to be well-tolerated and effective in suppressing inflammatory cytokine production and promoting therapeutic effects.

Abata Therapeutics was founded by experts in Treg biology, TCR and antigen discovery, disease pathogenesis, and molecular and imaging biomarkers. The company aims to create tissue-specific, robust, and durable Treg cell therapies. Besides their lead program in progressive MS, Abata is also working on a second program targeting type 1 diabetes, which is currently in IND-enabling studies. Both diseases are characterized by substantial unmet medical needs, supporting the rationale for Abata’s Treg-based therapeutic approach.

Launched in 2021 by Third Rock Ventures, Abata has garnered support from a diverse group of investors, including ElevateBio, Lightspeed Venture Partners, Invus, Samsara BioCapital, Eurofarma, the JDRF T1D Fund, and Biogen. The company is committed to advancing its innovative therapies to provide transformative treatments for patients with severe autoimmune and inflammatory conditions.

Abata's announcement of the IND clearance and their plans to initiate the Phase 1 study by the end of the year signal a promising step forward in the development of new treatments for progressive multiple sclerosis. By leveraging the unique properties of Tregs, Abata aims to bring forward solutions that address the significant challenges faced by patients with this debilitating disease.