On July 9, 2018 OBI Pharma, Inc., a Taiwan biopharma company (TPEx: 4174), reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for OBI-3424 for the Treatment of Hepatocellular Carcinoma (HCC) (Press release, OBI Pharma, JUL 9, 2018, View Source [SID1234527610]). OBI-3424 is a first in class DNA alkylating cancer therapeutic agent targeting aldo-keto reductase 1C3 (AKR1C3) overexpressing cancers.
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A Phase 1/2 study of OBI-3424 in patients with solid tumors, including hepatocellular carcinoma (HCC) and castrate-resistant prostate cancer (CRPC), has commenced enrollment at the University of Texas M.D. Anderson Cancer Center.
Amy Huang, General Manager of OBI Pharma, noted, "The orphan drug designation for OBI-3424 by the FDA is a significant step in the development of this drug candidate. OBI-3424 is intended to treat a devastating form of Liver Cancer with limited therapeutic options. We are excited that the FDA has recognized the need to develop novel targeted therapeutic agents such as OBI-3424 in the fight against this disease".
About Hepatocellular Cancer (HCC)
Hepatocellular Carcinoma (HCC) is a form of liver cancer associated with various stages of malignant growth in the liver. It is the sixth most common cancer worldwide, but is rare in the United States with a prevalence of 61,483 cases in 2018. HCC is considered a lethal cancer, with a survival rate of around 12.2% for five years, and the third major leading cause of cancer-related deaths worldwide. While most liver cancers are preventable, the incidence of HCC has recently increased in the United States, possibly due to the prevalence of common risk factors such as chronic liver disease, viral liver infections such as hepatitis, and cirrhosis. HCC patients also have a high risk of developing drug resistance to standard of care (SoC) treatments, creating a need for alternative treatment.
About Orphan Drug Designation (ODD)
The orphan drug designation provides OBI Pharma with potential benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees, and tax credits for qualified clinical trials. The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the U.S.
About OBI-3424
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.
AKR1C3 overexpression has been documented in a number of treatment-resistant and difficult-to-treat cancers including: hepatocellular carcinomas (HCC), castrate-resistant prostate cancer (CRPC), and T-cell acute lymphoblastic leukemia (T-ALL). AKR1C3 is highly expressed in up to 15 solid and liquid tumors.
OBI Pharma holds worldwide rights for OBI-3424 with the exception of the following countries, whose rights are held by Ascenta Pharma: China, Hong Kong, Macao, Taiwan, Japan, South Korea, Singapore, Malaysia, Thailand, Turkey, and India.