ROCKVILLE, Md. and SUZHOU, China, Oct. 7, 2024 /PRNewswire/ -- Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in discovering, developing and commercializing therapies to address global unmet medical needs primarily for malignancies, today announced that APG-2449, a FAK/ALK/ROS1 tyrosine kinase inhibitor (TKI), has been cleared by the Center for Drug Evaluation (CDE) of China's National Medical Product Administration (NMPA) to enter two registrational Phase III studies that will separately evaluate APG-2449 in patients with non-small cell lung cancer (NSCLC) who are resistant to or intolerant of second-generation anaplastic lymphoma kinase (ALK) TKIs; and treatment-naïve patients with ALK-positive advanced or locally advanced NSCLC.

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These two pivotal studies will be multicenter, open-label, randomized, registrational Phase III studies: first one is to evaluate the efficacy and safety of APG-2449 versus platinum-based chemotherapies in patients with NSCLC who are resistant to or intolerant of second-generation ALK TKIs. The second registrational Phase III study is designed to evaluate the efficacy and safety of APG-2449 versus crizotinib as frontline therapies for treatment-naïve patients with ALK-positive advanced or locally advanced NSCLC. These two studies are the studies of an investigational drug not yet approved by the FDA in the US.

ALK-positive NSCLC is a type of lung cancer with a specific molecular profile characterized by the abnormal arrangement or the fusion of the ALK gene which occurs in approximately 3%-5% of all lung cancer cases. Most patients with ALK-positive NSCLC are relatively young, non-smoking or only have a light smoking history, and have a higher risk of brain metastasis.

Despite that multiple ALK-targeted therapies have already been approved, more than half of patients with NSCLC treated with second-generation ALK TKIs would develop acquired resistance, thus the Chinese Society of Clinical Oncology (CSCO) guidelines' recommendation of platinum-based chemotherapies as a treatment option for patients who had failed on second-generation ALK-targeted therapies. It is widely acknowledged that chemotherapies are commonly associated with strong side effects and there is a growing general preference for chemotherapy-free regimens for the treatment of advanced tumors. Therefore, patients with resistance to second-generation ALK TKIs have an enormous unmet clinical need for new therapies that are effective and safe.

APG-2449, developed by Ascentage Pharma, is an orally-active small molecule FAK inhibitor and a third-generation ALK/ROS1 TKI, and the first FAK inhibitor cleared by the CDE to enter clinical study in China. In the first-in-human trial, APG-2449 demonstrated preliminary clinical benefit and favorable tolerability in patients with NSCLC who were either second-generation ALK TKI resistant or treatment-naïve. APG-2449 also showed potential inhibitory effect on brain metastases, with its ability to cross the blood-brain barrier confirmed through pharmacokinetics (PK) analysis on cerebrospinal fluid. Biomarker analysis found that the phosphorylated FAK (pFAK) expression in tumor tissues at baseline in patients with NSCLC who were second-generation ALK TKI-resistant, were positively correlated with the progress-free survival (PFS) after treatment with APG-2449, indicating that elevated phosphorylated FAK could be associated with drug resistance to second-generation ALK TKIs.

Prof. Li Zhang, the principal investigator of these two registrational Phase III studies from Sun Yat-sen University Cancer Center, commented, "APG-2449 is an effective multitargeted inhibitor that acts on FAK/ALK/ROS1. In previously released clinical data, APG-2449 consistently showed manageable safety and favorable antitumor activity in patients with NSCLC. We are particularly encouraged by the preliminary efficacy observed in patients with resistance to second-generation ALK TKIs, as it suggests that multitargeted inhibition on FAK and ALK may offer a new strategy for the management of patients with NSCLC resistant to second-generation ALK TKIs. We look forward to initiating the two registrational Phase III studies of APG-2449 in order to further validate the drug candidate and allow more patients to benefit from this novel therapeutic agent as soon as possible."

"There is considerable unmet clinical need in the field of NSCLC. APG-2449, a FAK/ALK/ROS1 TKI, has already showed its therapeutic potential in the released clinical data," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "The CDE's approvals for the two registrational Phase III studies of APG-2449 are very encouraging as they mark a major milestone in the drug candidate's clinical development. To fulfill our mission of addressing unmet clinical needs in China and around the world, we will expeditiously advance these clinical development programs for the benefit of more patients."

References:

  1. https://ascopubs.org/doi/10.1200/JCO.2022.40.16_suppl.9071
  2. https://ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.3124

About Ascentage Pharma

Ascentage Pharma (6855.HK) is a global, integrated biopharmaceutical company engaged in discovering, developing and commercializing therapies to address global unmet medical needs primarily in malignancies. On October 28, 2019, Ascentage Pharma was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code 6855.HK.

The company has built a rich pipeline of innovative drug candidates that includes novel, highly potent Bcl-2 and dual Bcl-2/Bcl-xL inhibitors, as well as candidates aimed at IAP and MDM2-p53 pathways, and next-generation TKIs. Ascentage Pharma is also the only company in the world with active clinical programs targeting all three known classes of key apoptosis regulators. The company has conducted more than 40 clinical trials in the US, Australia, Europe, and China, including 11 registrational studies (completed/ ongoing/planned).

Olverembatinib, the company's first lead asset developed for the treatment of drug-resistant chronic myeloid leukemia (CML) and the company's first approved product in China, has been granted Priority Review Designations and Breakthrough Therapy Designations by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA). To date, the drug had been included into the China National Reimbursement Drug List (NRDL). Furthermore, olverembatinib has been granted Orphan Drug Designations (ODDs) and a Fast Track Designation (FTD) by the US FDA, and an Orphan Designation by the EMA of the EU.

To date, Ascentage Pharma has obtained a total of 16 ODDs from the US FDA and 1 Orphan Designation from the EMA of the EU for 4 of the company's investigational drug candidates. Leveraging its robust R&D capabilities, Ascentage Pharma has built a portfolio of global intellectual property rights and entered into global partnerships and other relationships with numerous leading biotechnology and pharmaceutical companies such as Takeda, AstraZeneca, Merck, Pfizer and Innovent; and research and development relationships with leading research institutions such as Dana-Farber Cancer Institute, Mayo Clinic, MD Anderson Cancer Center, National Cancer Institute and the University of Michigan.

The company has built a talented team with a wealth of global experience in the discovery and development of innovative drugs and fully functional commercial manufacturing and Sales & Marketing teams. One pivotal aim of Ascentage Pharma is to continuously strengthen its R&D capabilities and accelerate its clinical development programs, in order to fulfil its mission of addressing unmet clinical needs in China and around the world for the benefit of more patients.

Forward-Looking Statements

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, Ascentage Pharma undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events, or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions and expectations or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions and expectations may alter in light of future development.

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