Pharma Focus Asia

Biostar Pharma Secures FDA Approval for Phase 2 Trial of Utidelone Injection (UTD1) in HER2-Negative Breast Cancer Brain Metastases

Friday, July 05, 2024

Biostar Pharma, Inc., the U.S. subsidiary of Beijing Biostar Pharmaceuticals Co., Ltd. (Biostar), which is a synthetic biology-driven biopharmaceutical company focused on developing and commercializing innovative oncology drugs, announced today that their core pipeline product, Utidelone Injection (UTD1), has received US 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, depending on the molecular classification of the cancer. The current standard treatment for BCBM mainly includes local therapies such as surgery and radiation, along with drug therapy. However, due to the blood-brain barrier (BBB) and blood-tumor barrier (BTB), many drugs effective for extracranial breast cancer metastases have limited intracranial permeability, resulting in poor outcomes for BCBM patients, particularly those with HER2-negative BCBM. The median progression-free survival (mPFS) for HR+/HER2-negative BCBM is about 4-6 months, while it is only 2.8 months for TNBC brain metastasis.

While recent developments in small-molecule tyrosine kinase inhibitors (TKIs) and antibody-drug conjugates (ADCs) have improved survival for HER2-positive BCBM patients, there remains a lack of effective drug treatments for HER2-negative BCBM. No drugs have been approved for this condition globally, highlighting a significant unmet medical need.

Utidelone can cross the BBB due to its unique physicochemical properties and resistance to P-glycoprotein-mediated efflux. This has been demonstrated in preclinical studies and several clinical trials. A phase 2 study presented at ASCO 2024 showed that combining utidelone with bevacizumab for HER2-negative BCBM treatment yielded a CNS-ORR of 42.6%, an mPFS of 7.7 months, and a 12-month overall survival (OS) rate of 74.4% among 47 patients. In the HR-/HER2- subgroup, the CNS-ORR was 55% and the mPFS was 8.4 months. The safety profile was manageable, with most adverse events being Grade 1 or 2.

In March 2024, Utidelone was granted orphan drug designation by the US FDA for the treatment of BCBM. Subsequently, Biostar filed an Investigational New Drug (IND) application for this phase 2 study for HER2-negative BCBM. The US FDA's approval of this application marks a significant step in Biostar's global development strategy.

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. The study follows a Simon's 2-stage design and will be conducted at 10-15 sites in the US, targeting the enrollment of 120 patients. The primary endpoint is CNS-ORR, with secondary endpoints including progression-free survival (PFS), duration of response (DOR), and overall survival (OS).



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