Onconic Therapeutics, a subsidiary of Jeil Pharmaceutical specializing in new drug development, announced on February 18 that its next-generation dual-target synthetic lethal cancer drug candidate, Nesuparib, has been granted Orphan Drug Designation (ODD) by the U.S. 

Food and Drug Administration (FDA) for the treatment of gastric cancer and gastroesophageal junction cancer.

Previously, Nesuparib received ODD status from both the FDA and the Korean Ministry of Food and Drug Safety (MFDS) in 2021 for pancreatic cancer. This latest designation further validates the drug’s therapeutic potential in additional oncology indications, according to the company.

Nesuparib is a next-generation synthetic lethal targeted therapy that simultaneously inhibits PARP (Poly ADP-ribose polymerase) and Tankyrase. PARP plays a key role in DNA damage repair, which helps cancer cells survive, while Tankyrase regulates the Wnt/β-catenin signaling pathway, crucial for tumor growth and metastasis. By blocking both enzymes, Nesuparib is expected to more effectively suppress cancer cell proliferation compared to conventional PARP inhibitors.

Onconic Therapeutics stated, “This dual-inhibition mechanism enhances anti-cancer efficacy beyond existing PARP inhibitors and has the potential to be effective in indications where conventional PARP inhibitors have shown limited success.”

The company added, “Having the same drug candidate designated as an orphan drug for two different cancer types underscores the strong technological foundation and development potential of Nesuparib as an anti-cancer therapy. We are committed to advancing research and development to provide new treatment options for gastric cancer patients with limited alternatives.”

Onconic plans to present its latest research findings on Nesuparib at the upcoming American Association for Cancer Research (AACR) 2025 in April.