FDA Approved Utidelone Injectable (UTD1) Phase 2/3 Multi-National Clinical Trial for Non-Small Cell Lung Cancer

On June 15, 2023 Biostar Pharma, Inc. (hereinafter referred to as "Biostar"), the US subsidiary of Beijing Biostar Pharmaceuticals Co., Ltd. which is a synthetic biology driven biopharma company focusing on the discovery, development and commercialization of innovative oncology drugs, reported that, after having in-depth discussions with the FDA through pre-IND meetings, the FDA has approved the IND of a phase 2/3 clinical trial with seamless protocol design for the company’s core product utidelone injectable (UTD1) in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) (Press release, Biostar, JUN 15, 2023, View Source [SID1234632760]).

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Designated as BG01-2202, this trial is a multi-national, phase 2/3, open-label, randomized, controlled clinical study of utidelone injectable (UTD1) versus docetaxel. The study is going to be conducted at about 50 sites in about 10 countries and regions across US, Europe and Asia -Pacific. About 760 patients are planned to be enrolled, 90 for phase 2 with ORR (objective response rate) as the primary endpoint and 670 for phase 3 with OS (overall survival) as the primary endpoint and PFS (progression free survival), ORR, etc. as the secondary endpoints.

Dr. Li Tang, Chairman of Biostar Pharma commented: "This is an important milestone for global development of utidelone injectable — an innovative anti-cancer drug and new generation epothilone analogue. We are fully committed to advancing this pivotal multi-national study and also willing to partner with global pharmaceutical companies to bring this product to market around the world to benefit more patients".

This study was warranted by a previous open-label, multi-center phase 2 trial conducted in China (NCT03693547) of utidelone as single-agent to treat patients with advanced NSCLC who had received at least two prior systemic regimens including platinum-containing chemotherapy or targeted therapy, which demonstrated promising clinical efficacy and manageable safety profile with outcomes of 19.0% ORR, 81.0% DCR (disease control rate), 4.37 months of median PFS and 71% of 12-month OS rate. The median OS was not reached by the cut-off date. The results were selected by 2022 ELCC (European Lung Cancer Congress) as poster presentation. A phase 3 study to evaluate utidelone versus docetaxel in locally advanced or metastatic NSCLC patients that have failed chemotherapy with a platinum-containing regimen has also been initiated and being in progress now in China.

Unmet Medical Need

Lung cancer remains the leading cause of cancer death worldwide [1]. NSCLC accounts for about 85% of lung cancer cases [2,3] and more than 75% of patients with lung cancer are diagnosed with locally advanced or stage IV disease [4]. Although currently targeted therapies and/or immunotherapies are commonly used for advanced NSCLC, limitations are obvious. Many patients either do not have a targetable driver gene alteration or are not suitable for immunotherapy [5,6]. Besides, acquired resistance to targeted- and immune-therapy is a substantial problem. In this case, chemotherapy, such as platinum- and taxane-based regimens, remains the fundamental treatment. However, acquired resistance to standard chemotherapies still presents challenges, which may limit their efficacy [7,8]. There is a huge unmet clinical need for alternative chemotherapeutic agents that can overcome, or are less susceptible to, drug resistance and have clinical efficacy in heavily pretreated patients.

About Utidelone

Being derived from Biostar’s synthetic biology technology platform, utidelone is a genetically engineered best-in-class epothilone analog, which is a class of microtubule-targeting agent that has differential mechanisms of action from taxanes and is developed to overcome drug resistance. Utidelone injectable has been launched in China in 2021 for the treatment of metastatic breast cancer (MBC). It is the only approved new molecular of microtubule inhibitor in the past 10 years around the world. Among all single agent or combination regimen of non-taxane chemotherapies, utidelone injectable is the only one to achieve both PFS and OS benefits for heavily pretreated MBC patients. It demonstrated several advantageous features, such as overcoming taxane-resistant, low hematological toxicity suitable for longer term exposure, ability to cross the blood-brain barrier that is able to prevent and treat tumor brain metastasis. Relevant study results were twice selected for oral presentations at ASCO (Free ASCO Whitepaper) annual meetings and invited to publish in Lancet Oncology and Annals of Oncology.