Compal's cell therapy subsidiary, ARCE Biotech, has made significant strides with its treatment for rare disease acute myeloid leukemia (AML). The company’s CAR-T cell therapy, ARD103, has successfully passed the U.S. FDA's Investigational New Drug (IND) review and has been approved to begin Phase 1/2 clinical trials. In addition, the team has formally submitted an Orphan Drug Designation (ODD) application to the U.S. FDA in Q3, which is expected to accelerate the clinical development process.
To date, ARCE Biotech has developed three proprietary platforms for chimeric antigen receptor T-cell (CAR-T) and chimeric antigen receptor natural killer (CAR-NK) cell technology: DashCAR, MaxCAR, and OneCAR. ARD103, the company's leading product, uses the DashCAR rapid production technology to generate autologous CAR-T cells that specifically target the CLL-1 antigen on AML cells. It is designed to treat relapsed/refractory AML (r/r AML) patients, a subgroup with few effective therapeutic options.
ARD103 incorporates a second-generation CAR gene structure, which has been screened and optimized. Using DashCAR technology, high-quality CAR-T cells can be produced within just three days, preserving the stem cell-like properties of the CAR-T cells. This results in a more durable and persistent immune response, critical for the long-term effectiveness of CAR-T therapy. DashCAR's rapid production process significantly reduces the manufacturing time, from the typical 2-3 weeks for existing cell therapies to just 3-5 days. This is particularly advantageous for patients with relapsed or refractory cancers, whose conditions cannot afford prolonged waits.
AML is a rare and aggressive cancer of the blood and bone marrow, characterized by the rapid proliferation of abnormal cells, which disrupt normal hematopoiesis. Without treatment, AML can be fatal within weeks or months due to the fast progression of the disease. In response, the ARCE team is dedicated to developing next-generation CAR immune cell therapies. Senior project manager and deputy director of ARCE Biotech, Li-Ming Lu, with decades of experience in drug development, emphasized, “ARD103 CAR-T will be the first step in treating r/r AML patients.”
ARCE plans to officially begin its Phase 1/2 trials by the first quarter of 2025, with patient recruitment already underway. Additionally, the company has applied to the U.S. FDA for Orphan Drug Designation for ARD103 in treating r/r AML, positioning it as a potential first-in-class cell therapy for this indication. The team highlights four key advantages of ARD103: rapid DashCAR® biomanufacturing within 3-5 days, high production yield, high specificity with no off-target effects, and proven efficacy and safety, which give strong prospects for the future development of this treatment.
Resource (mandarin): 仁寶「細胞治療」小金雞新藥邁大步 已向美FDA申請ODD
