Taiwanese biotech company OBI Pharma announced on the 7th that its new antibody-drug conjugate (ADC) drug, OBI-992, has been granted Orphan Drug Designation (ODD) by the U.S. FDA for the treatment of gastric cancer, including gastroesophageal junction adenocarcinoma.
OBI Pharma's CEO, Heidi Wang, stated that the FDA grants Orphan Drug Designation to drugs that show potential in treating rare diseases, in order to encourage more organizations to develop treatments for such conditions. In the U.S., a disease is classified as rare if it affects fewer than 200,000 people. Drugs that receive this designation can benefit from various incentives provided by the U.S. drug regulatory authorities, such as market exclusivity and additional regulatory support.
OBI-992 is an ADC designed by OBI Pharma that targets TROP2, a protein highly expressed in various solid tumors, including gastric cancer (GC) and other potential cancer types. In June, OBI-992 entered Phase I/II clinical trials in the U.S., where it is being tested on patients with advanced solid tumors to evaluate its safety, pharmacokinetics, and preliminary efficacy.
Chief Medical Officer M. Wayne Saville noted that preclinical studies have shown OBI-992 to have several key advantages over other TROP2-targeting ADCs in development. These advantages include high stability in the bloodstream, a "bystander effect" that effectively targets cancer cells even without TROP2 expression, and the ability to overcome potential drug resistance. OBI-992 demonstrated outstanding efficacy in both animal studies and organoid cancer models. "We look forward to further proving through clinical research that OBI-992 has the potential to be best-in-class," Saville remarked.
OBI-992 is an ADC that specifically targets TROP2, a protein highly expressed in multiple solid tumors, including lung, breast, ovarian, and gastric cancers, making it a promising target for cancer therapy.
The drug works by linking an antibody to a potent topoisomerase I (TOP1) inhibitor through a hydrophilic linker. This linker maintains stability in the bloodstream and, upon binding to TROP2 on the surface of cancer cells, is internalized into the cell. Once inside, the small molecule drug is released, killing the cancer cells. Various animal models have shown that OBI-992 has high anti-tumor activity, excellent pharmacokinetics, and a good safety profile.
OBI Pharma acquired the rights to the TROP2 antibody from Biosion in December 2021, and has since been responsible for the ADC's development and construction, with commercial rights outside of China.
Resource (mandarin): 浩鼎ADC新藥 獲美國FDA核准治療胃癌「孤兒藥」資格
