Onconix Therapeutics (476060) announced on the 24th that its dual-target anticancer drug candidate "Nesuparyb" has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of small cell lung cancer. With FDA orphan drug designation, conditional approval can be obtained based solely on Phase 2 clinical trial results.
Nesuparyb previously received FDA orphan drug designation for pancreatic cancer treatment in 2021 and gastric cancer treatment last year. The addition of small cell lung cancer is seen as reaffirming its potential as a multi-cancer anticancer drug.
Small cell lung cancer is classified as a representative intractable cancer due to its rapid proliferation, early metastasis, and high recurrence rate. After first-line treatment, therapeutic options upon relapse are limited, and improving long-term survival rates remains a challenge, making this an area with high medical demand for drugs with new mechanisms of action.
Nesuparyb is a dual-mechanism anticancer drug that simultaneously inhibits PARP, an enzyme involved in DNA damage repair in cancer cells, and Tankyrase (TNKS), which plays a crucial role in cancer cell proliferation, differentiation plasticity, metastasis, and acquisition of treatment resistance.
Small cell lung cancer in particular is considered a cancer type with high validity for treatment strategies targeting DNA damage response. However, despite new treatments such as immune checkpoint inhibitor combination therapies, chemotherapy remains the primary treatment, and there is a lack of treatment options with sustained efficacy after relapse.
An Onconix Therapeutics official stated, "With the safety and tolerability of Nesuparyb confirmed through Phase 1 clinical trials, we have simultaneously entered Phase 2 clinical trials for four indications, attracting significant attention from the global market," adding, "We will continue to explore indication expansion, focusing on cancer types with high dependence on replication stress and DNA damage response."