Biostar Pharma, Inc., the U.S. subsidiary of Beijing Biostar Pharmaceuticals Co., Ltd., a synthetic biology-driven biopharma company specializing in the development and commercialization of innovative oncology drugs, announced that their key pipeline product, Utidelone injection (UTD1), has received FDA approval to conduct a phase 2 study (BG01-2402) for HER2-negative breast cancer brain metastasis (BCBM).
Approximately 20-50% of metastatic breast cancer patients develop brain metastases based on the molecular classification of their breast cancer. The current standard care for BCBM involves local treatments such as surgery and radiation, along with drug therapy. However, due to the blood-brain barrier (BBB) and blood-tumor barrier (BTB), many drugs that are effective for extracranial breast cancer metastases have limited intracranial permeability, resulting in poor prognosis for BCBM patients, especially those with HER2-negative BCBM.
The median progression-free survival (mPFS) for hormone receptor-positive (HR+)/HER2-negative BCBM is about 4-6 months, while for triple-negative breast cancer (TNBC) brain metastasis it is only 2.8 months.
In recent years, several small-molecule TKIs and ADC drugs have improved survival rates for HER2-positive BCBM patients. However, there remains a significant unmet medical need for HER2-negative BCBM, as no drug has been approved for this indication worldwide.
Utidelone’s ability to penetrate the BBB, due to its unique physicochemical properties and resistance to P-glycoprotein-mediated efflux, has been confirmed by preclinical drug tissue distribution studies and various clinical trials. A phase 2 study presented at ASCO 2024 demonstrated that utidelone, in combination with bevacizumab, showed promising efficacy outcomes in HER2-negative BCBM patients, with a CNS-ORR of 42.6%, mPFS of 7.7 months, and a 12-month overall survival (OS) rate of 74.4% among 47 eligible patients. Particularly, in the HR-negative/HER2-negative subgroup, the study showed a CNS-ORR of 55% and mPFS of 8.4 months. The safety profile was manageable, with most adverse events being Grade 1-2, indicating utidelone’s potential as a new treatment option for HER2-negative BCBM.
In March 2024, Utidelone was granted “orphan drug designation” by the FDA for the treatment of BCBM. Following this, Biostar submitted an IND application for the phase 2 study for HER2-negative BCBM, which has now been cleared by the FDA, marking a significant milestone in Biostar’s global development strategy.
As reported by pharmabiz.com, the BG01-2402 study, titled “A Pivotal Phase II Clinical Trial of Utidelone Injection (UTD1) Plus Capecitabine (CAP) in HER2-negative Breast Cancer Patients with Brain Metastases,” aims to evaluate the intracranial and systemic efficacy of Utidelone Injection combined with capecitabine in HER2-negative BCBM patients. This study will follow a Simon’s two-stage design and is planned to be conducted at 10-15 sites across the U.S., targeting an enrollment of 120 patients. The primary endpoint is CNS-ORR, while secondary endpoints include PFS, duration of response (DOR), and OS.