OBI Pharma’s small-molecule prodrug OBI-3424 has secured orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of acute lymphoblastic leukemia (ALL).
OBI-3424 is a DNA alkylating cancer therapeutic agent that targets aldo-keto reductase 1C3 (AKR1C3) overexpressing cancers.
AKR1C3 overexpression has been known to be found in various treatment-resistant and difficult-to-treat cancers like hepatocellular carcinomas (HCC), ALL and castrate-resistant prostate cancer (CRPC).
OBI-3424 has been designed to selectively release a DNA alkylating agent in the presence of the AKR1C3 enzyme. OBI Pharma said that the selective mode of activation of the small-molecule prodrug makes it different from conventional alkylating agents like cyclophosphamide and ifosfamide, which are non-selective.
The orphan drug designation in the US for OBI-3424 is its second in the country. In July, it was granted similar status for the treatment of HCC.
OBI Pharma general manager Amy Huang said: “This additional orphan drug designation for OBI-3424 by the FDA is a significant step in the development of this drug candidate in ALL, including T-ALL, an unmet medical need disease with limited treatment 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 ALL.”
Currently, a phase 1/2 trial of OBI-3424 in patients with solid tumors has started enrollment at the University of Texas M.D. Anderson Cancer Center. The trial will assess the drug for the treatment of HCC and castrate-resistant prostate cancer (CRPC) among other cancers.
The patient enrollment follows the clearance given by the FDA in April for the investigational new drug (IND) application for the early-stage clinical trial.
OBI Pharma acquired global rights for OBI-3424 from Threshold Pharmaceuticals in mid-2017 with the exception of certain Asia countries including China, India and Japan where the rights are held by Ascenta Pharma.
Founded in 2002, OBI Pharma is engaged in development and licensing of novel therapeutic agents for unmet medical needs against cancer targets like Globo Series, AKR1C3, and other potential targets.