HXYT's STAR-T Therapy YTS104 Granted FDA Orphan Drug Designation for Relapsed/Refractory Acute Myeloid Leukemia

On December 26, 2022, Huaxia Yingtai (Beijing) Biotechnology Co., Ltd. announced that it had received a written response from the Office of Orphan Products Development (OOPD) of the U.S. Food and Drug Administration (FDA).The synthetic T-cell receptor antigen receptor (STAR-T) injection (R&D code: YTS104) independently developed by the company has been granted Orphan Drug Designation (ODD) for the treatment of relapsed/refractory acute myeloid leukemia (R/R AML).

FDA Orphan Drug Designation (ODD) can help accelerate the clinical development and registration process of drugs in the United States. After YTS104 received the FDA’s ODD, it is entitled to several preferential policies, including FDA’s clinical research guidance support for the candidate product, special fee reductions, and seven years of market exclusivity in the U.S. after approval for marketing.

Huaxia Yingtai(Beijing)Biotechnology Co., Ltd. CEO Dr. Guohua Pan stated:

"I am very proud of our team. Following the completion of the Phase I clinical trial initiation and the enrollment of the first patient for the company’s dual-target STAR-T product in early December, the YTS104 product has been granted FDA Orphan Drug Designation. This not only demonstrates the recognition from both China's NMPA and the U.S. FDA review departments for our product but also marks that Huaxia Yingtai(Beijing)Biotechnology Co., Ltd. has moved from new drug discovery into a new phase of clinical development. The FDA Orphan Drug Designation is an important milestone in the development process of YTS104, playing a positive role in accelerating its clinical development in both China and the U.S., as well as future registration and market launch. We are confident that through relentless exploration and effort, we will soon bring a new treatment option to patients with acute myeloid leukemia worldwide."

About YTS104

YTS104 Cell Injection is an autologous cell therapy drug based on the innovative STAR-T platform developed by Huaxia Yingtai (Beijing) Biotechnology Co., Ltd. Its primary target indication is relapsed/refractory acute myeloid leukemia (R/R AML). This product is manufactured by using a lentiviral gene transduction method to introduce specific STAR genes into autologous T cells, followed by in vitro cell expansion, and subjected to functional and quality control testing to produce the cell therapy drug. The product's advantage lies in the use of two independently screened nanobodies targeting different epitopes, which are linked to the α and β chains of the STAR structure, respectively, with the expectation of enhanced antigen recognition and cytotoxic effects.

About FDA Orphan Drug Designation (ODD)

Orphan Drug Designation (ODD) is a status granted by the FDA's Office of Orphan Products Development (OOPD) to drugs (including biologics) that meet the criteria for prevention, treatment, and diagnosis of rare diseases. Orphan drugs, also known as rare disease drugs, refer to medicines used for the prevention, treatment, and diagnosis of rare diseases. The FDA has clear defining criteria for rare diseases, specifically those affecting fewer than 200,000 people in the United States. Due to the small patient population, limited market demand, and high research and development costs, few pharmaceutical companies focus on the development of treatments for these conditions, hence the term "orphan drugs." In 1983, the United States enacted the Orphan Drug Act (ODA), which stipulates that any drug candidate granted orphan drug designation may be eligible for a series of supportive policies.

About Acute Myeloid Leukemia (AML)

Acute Myeloid Leukemia (AML) is a clonal malignant proliferation disease of myeloid progenitor cells in the hematopoietic system, characterized by abnormal proliferation of primitive and immature myeloid cells in the bone marrow and peripheral blood. Clinically, it manifests as anemia, bleeding, infection, fever, organ infiltration, metabolic abnormalities, etc., with strong heterogeneity, limited treatment options, and generally poor prognosis. In 2017, the global incidence of AML was 119,570 cases, with China ranking among the top three globally in both incidence and mortality, at 13,200 cases and 7,100 deaths, respectively. Compared to non-monocytic subtypes, monocytic AML (M4 and M5) carries a higher risk of marrow and extramedullary relapse after stem cell transplantation, with incidences of 34% and 50%, respectively. For patients with relapsed or refractory AML, there are no definitive treatments that significantly extend survival. In elderly patients with relapsed or refractory AML treated with low-dose chemotherapy, the median overall survival (OS) is 5-9 months, while for those with ≥2 relapses, the median OS is only 3 months.

About Huaxia Yingtai

Huaxia Yingtai (Beijing) Biotechnology Co., Ltd. is an innovative pharmaceutical company specializing in the development of T-cell immunotherapy products, committed to addressing unmet clinical needs such as advanced cancer, with a global perspective. It was founded in March 2018 by renowned immunologists. The company has established two innovative technology platforms, STAR-T and enTCR-T, along with a mature industrialization platform for process development and quality control. It has developed a rich pipeline of products targeting hematologic tumors, solid tumors, and viral infections. Over 10 registered "First-in-Human" clinical studies have been initiated, yielding positive clinical safety and efficacy data, which have been successively published in international academic conferences and journals. Multiple programs are actively advancing into pivotal clinical studies. The company's mission is "Dedicated to science, pursuing excellence, relieving cancer patients from pain, and becoming a pioneer and leader in globally innovative cellular immunotherapy products based in China for the benefit of patients worldwide."

Editor: Dada Xiwa

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