Biostar Pharma's Utidelone Injection Gets FDA Green Light For Phase 2 Study

Biostar Pharma, the U.S. branch of Beijing Biostar Pharmaceuticals Co., announced today that the U.S. FDA has granted approval for a phase 2 study (BG01-2402) of their leading pipeline product, Utidelone Injection (UTD1), for the treatment of HER2- breast cancer brain metastasis (BCBM). Biostar, a synthetic biology-driven biopharma company, focuses on developing and commercializing innovative oncology drugs.

Brain metastases develop in approximately 20-50% of metastatic breast cancer patients, varying with the molecular classification of the cancer. The standard treatment for BCBM typically involves local therapies such as surgery and radiation, supplemented by drug therapy. However, due to the blood-brain barrier (BBB) and blood-tumor barrier (BTB), many drugs that work for extracranial metastasis of breast cancer have limited effectiveness for intracranial metastasis, leading to poor outcomes for BCBM patients, especially those with HER2- BCBM. The median progression-free survival (mPFS) for HR+/HER2- BCBM is about 4-6 months, while for TNBC brain metastasis, it is only 2.8 months.

While recent advances with small-molecule TKIs and ADC drugs have improved survival for HER2+ BCBM patients, there remains a lack of effective drug treatments for HER2- BCBM, with no approved drugs worldwide, highlighting a significant unmet medical need. Utidelone's ability to penetrate the BBB, due to its unique physicochemical properties and resistance to P-glycoprotein-mediated efflux, has been demonstrated in preclinical drug distribution studies and several clinical trials. A phase 2 study presented at ASCO 2024 showed that utidelone, in combination with bevacizumab, achieved a CNS-ORR of 42.6%, mPFS of 7.7 months, and a 12-month OS rate of 74.4% in 47 enrolled patients.

The efficacy was even more pronounced in the HR-/HER2- subgroup, with a CNS-ORR of 55% and an mPFS of 8.4 months. Most adverse events were manageable and graded 1-2, indicating a promising efficacy and safety profile for utidelone in treating HER2- BCBM. In March 2024, the US FDA granted utidelone "orphan drug designation" for the treatment of BCBM. Following this, Biostar filed an IND application for the phase 2 study for HER2- BCBM. The FDA's approval of this application represents a significant milestone in Biostar's global development strategy.