A2 Bio receives FDA Orphan Drug Designation for novel cell therapy program in colorectal cancer

A2 Biotherapeutics, a clinical-stage cell therapy company developing first-in-class logic-gated cell therapies, has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for A2B530 for the treatment of germline heterozygous HLA-A*02(+) patients with colorectal cancer that expresses carcinoembryonic antigen (CEA) and has lost HLA-A*02 expression.

A2B530 is the first autologous logic-gated cell therapy developed from A2 Bio’s proprietary TmodTM platform. The Tmod platform utilizes a dual-receptor design consisting of an activator that targets tumor cells and a blocker that protects normal cells. This novel design is aimed at tackling the fundamental challenge in solid tumor cancer medicines – the ability to selectively kill tumor cells and protect normal cells. A2B530 consists of an activator that targets CEA and a blocker that targets HLA-A*02.

Enrollment is ongoing in EVEREST-1 (NCT05736731), a seamless Phase 1/2 study to evaluate safety and efficacy of A2B530 in colorectal, pancreatic and non-small cell lung cancers.

“The FDA granting Orphan Drug Designation validates the tremendous unmet need for improved therapies for patients with colorectal cancer,” said William Go, M.D., Ph.D, Chief Medical Officer of A2 Bio. “This designation supports our commitment to use our novel technology platform to develop new treatment options for patients with difficult-to-treat cancers.”

FDA Orphan Drug Designation incentivizes the development of innovative drugs and biologics for the safe and effective treatment of rare diseases and conditions that affect fewer than 200,000 people in the United States.

Orphan Drug Designation qualifies A2 Bio for certain development incentives related to the A2B530 clinical program, including tax credits for clinical trials, prescription drug user fee exemptions and potentially up to seven years of market exclusivity upon regulatory approval.

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