On September 25, Beijing Huahaizhongtian Biopharmaceutical Co., Ltd. (hereinafter referred to as "Huahaizhongtian," stock code: 2563.HK) announced that its core product, Utidelone, has been granted another Orphan Drug Designation by the U.S. Food and Drug Administration (FDA), this time for the treatment of pancreatic cancer. This marks the third Orphan Drug Designation granted to Utidelone by the FDA, following those for breast cancer brain metastases and gastric cancer.

Pancreatic cancer is a highly malignant tumor, often referred to as the "king of cancers" due to its insidious early symptoms, difficulty in diagnosis, rapid progression, strong invasiveness, and poor prognosis. The five-year survival rate is only around 10%, which is significantly lower than that of other common cancers, making it the lowest among all malignant tumors. Currently, there is no clearly effective treatment regimen for pancreatic cancer. Gemcitabine-based combination therapies remain the primary clinical treatment; however, pancreatic cancer cells readily develop resistance to gemcitabine, leading to unsatisfactory therapeutic outcomes.

The potential of Utidelone for treating pancreatic cancer has been fully demonstrated in both non-clinical and clinical trials. Preclinical data show that Utidelone can significantly inhibit the proliferation and colony formation ability of pancreatic cancer cells and exhibits good anti-tumor activity in pancreatic cancer models. When combined with gemcitabine, Utidelone can significantly reduce the IC50 value of gemcitabine without diminishing its cytotoxic effect on tumor cells. Furthermore, the anti-tumor activity of the combination is superior to that of traditional paclitaxel combined with gemcitabine.

At the 2024 CSCO Annual Meeting, Huahaizhongtian presented preliminary data from a multicenter, single-arm Phase II clinical study of Utidelone combined with gemcitabine as a first-line treatment for unresectable advanced pancreatic cancer. As of the data cut-off, 20 patients with advanced pancreatic cancer that was surgically unresectable and unsuitable for local therapy had been enrolled, with 11 completing their first efficacy evaluation. Among these, 3 achieved partial remission (PR) and 5 had stable disease (SD). The objective response rate (ORR) was 27.27%, the disease control rate (DCR) reached 72.72%, and the median overall survival (mOS) was 9.57 months.

Utidelone holds the promise of bringing meaningful survival benefits and disease control to patients with advanced pancreatic cancer, potentially improving the current desert-like treatment landscape for pancreatic cancer and establishing a new therapeutic option.