SUZHOU, China and ROCKVILLE, MD., Sept. 14, 2020 — Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced that the US Food and Drug Administration (FDA) has granted APG-115, a novel MDM2-p53 inhibitor being developed by the company, an Orphan Drug Designation (ODD) for the treatment of gastric cancer (GC). This is the first ODD granted for APG-115.
The term “orphan drugs” refers to pharmaceutical products developed for the prevention, diagnosis, and treatment of rare diseases or conditions. In the United States, an orphan disease is defined as a disease or condition with a prevalence of less than 200,000 patients in the country. Since the Orphan Drug Act was passed in 1983, the US government has provided incentives and policy support to encourage development of orphan drugs. This ODD from the FDA qualifies APG-115 for various development incentives, including a tax credit on expenditures incurred in clinical studies, a waiver of the New Drug Application (NDA) fee, research grant awarded by the FDA, and most importantly, 7 years of US market exclusivity upon approval for the treatment of GC.
The global incidence of GC is significantly different between the eastern and western countries. According to the National Cancer Institute’s Surveillance, Epidemiology, and End Results Program, in 2017, there were an estimated 116,525 people living with GC in the US1. GC is currently considered as a rare disease in US. Asian countries such as China and Japan have a much higher incidence. GC is the third leading cause of cancer deaths worldwide, followed only by lung and colorectal cancer in overall mortality. About 1 in 12 of all oncological deaths are attributable to GC2.
The National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for GC recommends a multidisciplinary approach for treatment of unresectable, advanced, metastatic GC patients. For patients with disease progression receiving second-line therapy, there are not many treatment options and the prognosis remains poor. Thus, effective treatment options are urgently needed3 to improve the disease outcome and reduce the mortality.
APG-115 is an orally administered, selective, small-molecule inhibitor of the MDM2-p53 protein-protein interaction (PPI). APG-115 has strong binding affinity to MDM2 and is designed to activate tumor suppression activity of p53 by blocking the MDM2-p53 PPI. APG-115 is the first MDM2-p53 inhibitor entering clinical development in China, with multiple ongoing clinical studies in solid tumors and hematologic malignancies in China and the US. APG-115 has shown promising results in preclinical studies for the treatment of GC.
“At present, the treatment of GC represents an urgent unmet clinical need globally. This ODD by the US FDA for the treatment of GC marks an important milestone in the global development and commercialization of APG-115,” said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. “All the policy support and incentives as a result of this ODD will help us accelerate the global clinical development of APG-115, which we hope will soon transform to benefit more patients.”
APG-115 is an orally administered, selective, small-molecule inhibitor of the MDM2-p53 PPI. APG-115 has strong binding affinity to MDM2 and is designed to activate p53 tumor suppression activity by blocking the MDM2-p53 PPI. Ascentage Pharma has previously commenced three clinical trials of APG-115 in the US, including a Phase I study as single agent, a Phase Ib/II study in combination with pembrolizumab for treatment of metastatic melanoma and other advanced solid tumors, and a Phase I/II study as a single agent or in combination with chemotherapy for treatment of salivary gland cancer. APG-115 is the first MDM2-p53 inhibitor to enter clinical studies in China. A Phase I study as a single agent, and a Phase Ib study as a single agent or in combination with chemotherapy for treatment of AML (acute myeloid leukemia) or MDS (myelodysplastic syndrome) are ongoing in China.
About Ascentage Pharma
Ascentage Pharma (6855.HK) is a globally, clinical-stage biotechnology company engaged in developing novel therapies for cancers, CHB, and senesce diseases. 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.
Ascentage Pharma focuses on developing therapeutics that inhibit protein-protein interactions to restore apoptosis, or programmed cell death. The company has built a pipeline of eight clinical drug candidates, including 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 tyrosine kinase inhibitors. 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 is conducting more than 30 Phase I/II clinical trials in the US, Australia, and China. The company’s core drug candidate HQP1351 was recently granted orphan drug and fast-track designations by the US Food and Drug Administration (FDA), and a New Drug Application for HQP1351 has been submitted in China. APG-2575, another key drug candidate of the company, was recently granted orphan drug designation by the FDA.
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