Biostar Pharma Secures FDA Approval for Phase 2 Trial of Utidelone Injection (UTD1) in HER2-Negative Breast Cancer Brain Metastases
Oerview
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).
Metastatic Breast Cancer
- 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.
Small-Molecule Tyrosine Kinase Inhibitors
- 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: Pharmacokinetic Properties
- 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.
ODD for Utidelone
- 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.
BG01-2402 Study
- 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).
