On July 5, 2024 Biostar Pharma, Inc., the U.S. subsidiary of Beijing Biostar Pharmaceuticals Co., Ltd. (Biostar), which is a synthetic biology-driven biopharma company focusing on the development and commercialization of innovative oncology drugs, reported that their core pipeline product Utidelone Injection (UTD1) had been granted to conduct a phase 2 study (BG01-2402) for HER2- breast cancer brain metastasis (BCBM) by the US FDA (Press release, Beijing Biostar Technologies, JUL 5, 2024, View Source;breast-cancer-brain-metastasis-302189908.html [SID1234644691]).

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Depending on the molecular classification of breast cancer, approximately 20~50% of metastatic breast cancer patients develop brain metastases.[1] The current standard of care for BCBM is primarily local treatment with surgery and radiation therapy, supplemented by drug therapy. Due to blood-brain barrier (BBB) and blood-tumor barrier (BTB), many drugs that are effective for extracranial metastasis of breast cancer have very limited intracranial permeability, leading to poor prognosis for BCBM patients, especially with HER2- BCBM.[2-3] The mPFS for HR+/HER2- BCBM is about 4-6 months,[4-8] while the mFPS for TNBC brain metastasis is only 2.8 months.[9]

In recent years, multiple small-molecule TKIs and ADC drugs have brought survival benefits to HER2+ BCBM patients. However, there is still a lack of drug treatment regimens with proven efficacy for HER2- BCBM, and no drug has been approved for HER2- BCBM worldwide, suggesting a significant unmet medical need.

Utidelone can penetrate BBB due to its unique physicochemical characteristics and insusceptibility to P-glycoprotein-mediated efflux, which has been confirmed by preclinical drug tissue distribution studies and several clinical trials. A phase 2 study of utidelone in combination with bevacizumab for the treatment of HER2- BCBM presented at the ASCO (Free ASCO Whitepaper) 2024 demonstrated efficacy outcomes of 42.6% of CNS-ORR, 7.7 months of mPFS, and 74.4% of 12-month OS rate with total 47 eligible patients being enrolled. The efficacy was even better in HR-/HER2- subgroup, showing 55% of CNS-ORR and 8.4 months of mPFS. The safety was manageable with majority of the AEs being Grade 1~2, suggesting a promising efficacy and manageable safety profile of utidelone for HER2- BCBM, and the potential of utidelone to become a new treatment option in the space.

Utidelone has been granted an "orphan drug designation" by the US FDA for the treatment of BCBM in Mar 2024. Biostar then filed an IND application for this phase 2 study for HER2- BCBM. The US FDA clearance of the application marks an important milestone of Biostar’s globalization development strategy.

About BG01-2402 Study

BG01-2402 study refers to "A Pivotal Phase II Clinical Trial of Utidelone Injection (UTD1) Plus Capecitabine (CAP) in HER2-negative Breast Cancer Patients with Brain Metastases". The purpose of the study is to evaluate the intracranial and systemic efficacy of Utidelone Injection in combination with capecitabine in HER2- BCBM patients. This study follows a Simon’s 2-stage design, and is planned to be conducted at 10-15 sites in the US with enrollment target of 120 patients. The primary endpoint is CNS-ORR; the secondary endpoints include PFS, DOR and OS, etc.