12 July 2018 - - Taipei, Taiwan-based biopharma company OBI Pharma, Inc. (TPEx: 4174) has been granted orphan drug designation by the US FDA for OBI-3424 for the treatment of hepatocellular carcinoma (HCC), the company said.
OBI-3424 is a first-in-class novel small-molecule prodrug that selectively targets cancers overexpressing the enzyme aldo-keto reductase 1C3 (AKR1C3), and selectively releases a potent DNA alkylating agent in the presence of the AKR1C3 enzyme.
This selective mode of activation distinguishes OBI-3424 from traditional alkylating agents, such as cyclophosphamide and ifosfamide, which are non-selective.
A phase 1/2 study of OBI-3424 in patients with solid tumors, including hepatocellular carcinoma and castrate-resistant prostate cancer, has commenced enrollment at the University of Texas M.D. Anderson Cancer Center.
OBI Pharma seeks to develop and license novel therapeutic agents for unmet medical needs, including cancer by targeting the Globo Series antigens, the AKR1C3 enzyme, and other promising targets.
The company's leading compound is Adagloxad Simolenin (formerly OBI-822), a late-stage novel active immune-oncology Globo Series-based therapeutic vaccine for the treatment of epithelial cancers, such as breast, ovarian, lung, prostate, colon, gastric, and pancreatic cancer.