Ever Supreme's allogeneic CAR-T greenlighted for phase I/II trial

Source in Chinese: Ever Supreme Bio Technology

Ever Supreme Bio Technology yesterday (21 September) announced a major breakthrough in cell therapy. The U.S. FDA has recently approved the company's CAR001 (CAR-T) to undergo Phase I/IIa clinical trial, which is one of the few cases in the world where allogeneic CAR-T cells have been used to target solid tumors. In the future, Ever Supreme will have the opportunity to seize the global tens of billions of dollars of cell therapy business opportunities. 

According to the company, there are currently seven CAR-T cell products approved and launched in the market, all of which use autologous cells to fight against blood tumours. There are two major drawbacks to these cell therapies: first, the indications have not yet been widely applied to the treatment of solid tumours, but 80% of human cancers belong to solid tumours; furthermore, these marketed CAR-T cell products must be modified using autologous cells, which takes about one to two months to prepare. In addition, these marketed CAR-T cell products must be modified using autologous cells. In addition to time for preparation and quality issues, a single treatment is priced at US$400,000 (approximately NT$12 million).

Nevertheless, the world's best-selling autologous CAR-T product, YESCARTA, still amounted to US$1.16 billion in 2022, and the global CAR-T market saw US$18.61 billion in the same year. It is estimated the market will hit US$93.78 billion in 2030, with a compound annual growth rate of 22.4%.

Ever Supreme is in partnership with a team led by the Superintendent of China Medical University Hospital (CMUH), Dr. Der-Yang Cho, on developing CAR001. After years of research and development, CAR001 is the world's fourth allogeneic CAR-T cell therapy developed for the treatment of solid tumours using Gamma Delta-T cells as the carrier, and it is the world's first of its kind to use HLA-G as the target.

Ever Supreme explained that CAR001 leverages AI to screen for key human leukocyte antigen G (HLA-G) targets in solid tumours and uses mRNA-modified "heterologous non-viral gene-edited multi-targeted nanoantibody-CAR.BiTE-γδT immune cells". It has shown to eliminate more than 90% of solid tumours in preclinical experiments in animals. The research results had been published in the Journal for ImmunoTherapy of Cancer (JITC, IF 13.751) in 2021 and Advanced Science (IF 17.521) in 2023.

The company pointed out that during the 30-day process of applying for clinical trials in the U.S., some challenges were encountered, but the research team worked together to respond to FDA requests within 48 hours. It succeeded in pushing forth its new drug into the Phase I/IIa clinical trial in less than 30 days. A number of pharmaceutical companies around the world are currently approaching Ever Supreme for the licensing of CAR001.

As the first allogeneic HLA-G-CAR-BiTE cell therapy approved for human clinical trials, which opens up a new era of cancer treatment for advanced and difficult solid tumours, including colorectal, breast, lung and brain cancers, and that CAR001 has the advantage of being available off-the-shelf, providing patients with a convenient and effective treatment option.

Unlike other biotech companies, Ever Supreme has comprehensive production capabilities, which include the complete process from healthy donor cell harvesting, electro-transfection, to mass production. This one-stop development and production capability enables the company to comply with regulatory units to control the quality of the CAR001 products and increase production capacity. The company has not only been approved by the US FDA, but has also become the fourth company in the world to develop an allogeneic CAR-γδT therapy for the treatment of solid tumours.