SUZHOU, China and ROCKVILLE, Md., July 15, 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-2575, a novel Bcl-2 inhibitor being developed by the company, an Orphan Drug Designation (ODD) for the treatment of Waldenström macroglobulinemia (WM). This is the first ODD granted to APG-2575, and the second ODD Ascentage Pharma received from the FDA, following the previous ODD granted to the company’s third-generation BCR-ABL inhibitor HQP1351 in May 2020 for the treatment of chronic myeloid leukemia.
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-2575 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 WM.
WM is a type of lymphocytic lymphoma characterized by the bone marrow infiltration of lymphoplasmacytic cells with elevated monoclonal immunoglobulin M (IgM). WM is a rare disease that accounts for less than 2% of all non-Hodgkin’s lymphoma (NHL) patients in the US.
Treatment recommendations for WM from current NCCN guidelines suggest an objective response rate (ORR) of ~80% with current therapies; however, these therapies deliver a very low rate of very good partial response (VGPR) or deeper responses (~20% or lower), with most patients experiencing disease progression after initial response. Furthermore, patients are diagnosed with WM at a median age of 70, when many of them are intolerant of existing therapies because of poor health conditions, representing a significant unmet medical need for more effective and safer therapies.
APG-2575 is a novel, orally administered Bcl-2 selective inhibitor being developed by Ascentage Pharma. APG-2575 is designed to treat a variety of hematologic malignancies by selectively blocking Bcl-2 to restore the normal apoptosis process in cancer cells. Beside VENCLEXTA®(venetoclax), APG-2575 is one of the few Bcl-2 selective inhibitors currently in active clinical development worldwide and the first China-developed Bcl-2 selective inhibitor having entered clinical trials in China. APG-2575 has received clearances and approvals for multiple Phase Ib/II clinical studies in China, Australia, and the US in a range of hematologic malignancies globally, including a global multicenter Phase Ib/II study designed to evaluate the safety, tolerability, and efficacy of APG-2575 as a single agent or in combination with ibrutinib/rituximab for the treatment of patients with WM.
“WM represents an unmet medical need globally. APG-2575 is a key drug candidate in Ascentage Pharma’s pipeline targeting apoptosis. This ODD by the US FDA for the treatment of WM marks an important milestone in the global development and commercialization of APG-2575,” said Dr. Dajun Yang, Chairman & CEO 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-2575, which we hope will soon transform to benefit more patients.”
 Datamonitor, Market Spotlight: WM published on April 17, 2020.
APG-2575 is a novel, orally administered Bcl-2‒selective inhibitor being developed by Ascentage Pharma. APG-2575 is designed to treat a variety of hematologic malignancies by selectively blocking Bcl-2 to restore the normal apoptosis process in cancer cells. Ascentage Pharma has previously commenced Phase I studies of AGP-2575 single agent in China, Australia, and the United States. Since March 2020, the company has received approvals and clearances for several Phase Ib/II studies of APG-2575 in China, Australia, and the US, and is advancing clinical development of APG-2575 for a variety of hematologic malignancy indications, including relapsed/refractory chronic lymphocytic leukemia or small lymphocytic lymphoma, Waldenström macroglobulinemia, relapsed/refractory multiple myeloma, and relapsed/refractory acute myeloid leukemia.
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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