Yimufeng Announces FDA Orphan Drug Designation for EpCAM-Targeted CAR-T Therapy IMC001 in Gastric Cancer

On September 17, Immunofoco announced that its self-developed autologous CAR-T cell injection product targeting EpCAM (IMC001) has recently received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of gastric cancer patients.

- The first product to use EpCAM as a CAR-T target and publish clinical trial data

- The granting of U.S. orphan drug designation recognizes the significant impact of IMC001 in the field of gastric cancer treatment.

IMC001 is an autologous CAR-T product targeting EpCAM. EpCAM is a biomarker of circulating tumor cells (CTC). It is highly expressed in both metastatic and primary sites of digestive system tumors, while its expression is low in normal tissues. In normal tissues, EpCAM is expressed on the basal lateral side of cells, which is less accessible to immune cells. During malignant transformation of tumors, the expression pattern of EpCAM changes to a uniform distribution on the cell membrane. Based on the differences in expression levels and patterns between normal and tumor tissues, IMC001 can distinguish tumor tissues from normal tissues, demonstrating a favorable dosing window and showing highly promising anti-tumor activity in preclinical studies.

To evaluate the safety and preliminary efficacy of IMC001 in advanced EpCAM+ digestive system tumors, Immunofoco has conducted two IIT clinical studies. In the clinical trial for advanced gastric cancer, two out of six patients in the medium- and low-dose groups were assessed as having partial responses (PR). The survival time of all three patients in the medium-dose group has exceeded 10 months, including one PR patient who successfully underwent radical gastrectomy 28 weeks after a single infusion of IMC001 monotherapy, with good safety demonstrated.

The U.S. Orphan Drug Designation recognizes the significant role of IMC001 in the field of gastric cancer treatment. The Orphan Drug Designation is a special drug recognition system for drugs targeting rare diseases or diseases with a total patient population not exceeding 200,000. This designation will bring many advantages to the further development and commercialization of IMC001, as well as a series of preferential policies and support provided by the U.S. FDA, offering strong assurance for the implementation of IMC001.

Editor: Mu Mian

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