Lisaftoclax

ROCKVILLE, Md. and SUZHOU, China, Aug. 22, 2024 /PRNewswire/ -- Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in discovering, developing and commercializing both first-in-class and best-in-class therapies for malignancies, today announced its 2024 interim results. During the reporting period, Ascentage Pharma continued to execute its global innovation strategy, having achieved breakthroughs across various aspects of its business including external collaborations, commercialization, and clinical development. In particular, the company has set a new record with its revenue for the first half of 2024 and reported profit for the first time. During the reporting period, Ascentage Pharma has set a new record for its revenue that grew by 477% from the same period last year to RMB824 million, with most of the revenue generated from sales of pharmaceutical products and license income. For the very first time in its history, Ascentage Pharma reached profitability, having reported a net profit of RMB163 million. Ascentage Pharma made rapid progress with the commercialization of its first approved product, olverembatinib (HQP1351). During the reporting period, olverembatinib generated a sales revenue of RMB113 million, which grew by 120% from the second half of 2023 and 5% year-over-year. Ascentage Pharma and Takeda, a multinational pharmaceutical company, have entered into an exclusive option agreement that granted Takeda an exclusive option to enter into an exclusive license agreement for olverembatinib. Once exercised, the Option would allow Takeda to license global rights to develop and commercialize olverembatinib in all territories outside of, among others, the mainland China, Hong Kong China, Macau China, and Taiwan China. Ascentage Pharma has received US$100 million in option payment and a US$75 million equity investment. The company's cash flows continued to improve. As of June 30, 2024, the company's cash balances were RMB1.1 billion and rose to RMB1.8 billion by early July, 2024. Entered into a landmark global collaboration that is the largest BD deal among small molecule oncology drugs in China Ascentage Pharma constantly seeks global partnerships. During the reporting period, the company and Takeda, a multinational pharmaceutical company, entered into an exclusive option agreement for olverembatinib. This collaboration, with a total value reaching US$1.3 billion, includes an option payment of US$100 million and an option exercise fee and potential milestone payments totaling US$1.2 billion. Additionally, Ascentage Pharma is eligible for tiered double-digit royalties on annual net sales. To date, this is the largest out-licensing deal for any small molecule oncology drug in China. Furthermore, Ascentage Pharma also received an equity investment from Takeda. This collaboration highlights the significant clinical and commercial potential of olverembatinib in the global market and laid a solid foundation for the future global commercialization of the drug. Ascentage Pharma has already received the US$100 million option payment and US$75 million equity investment from Takeda. Commercialization of olverembatinib accelerated and strengthened the company ' s revenue generating ability Following the inclusion of the first approved indication of Ascentage Pharma's novel drug candidate olverembatinib into the China National Reimbursement Drug List (NRDL) in January 2023 and the regulatory approval for its new indication in November 2023, olverembatinib continued to realize its growth potential while boosting company's ability to generate revenue through product sales. During the reporting period, the sales revenue of olverembatinib grew by 120% from the second half of 2023 and by 5% year-over-year, to RMB113 million. Ascentage Pharma is expeditiously expanding the commercialization of its approved product. As of June 30, 2024, olverembatinib has entered 670 hospitals and direct-to-patient (DTP) pharmacies nationwide and its number of listed hospitals has grown by 79% compared to the end of last year. Since the approval of its new indication in November 2023 till June 30, 2024, olverembatinib has been included into 114 supplemental insurance for major diseases and Huimin insurance plans of 20 provinces and 83 municipalities. Additionally, olverembatinib was included into the special drugs catalog of the Huimin insurance plan of 20 provinces and municipalities such as Hebei, Hainan, Inner Mongolia Autonomous Region, Wuxi, Huzhou, Shenzhen, and Yantai, which greatly improved the drug's accessibility and alleviated the burden on patients. As the first China-approved third-generation BCR-ABL inhibitor, olverembatinib's clinical value and therapeutic potential continued to received high profile recognition during the reporting period, both in China and globally. In January 2024, clinical trial results of olverembatinib were included into the US National Comprehensive Cancer Network® (NCCN) Guidelines for the Management of Chronic Myeloid Leukemia (CML). Moreover, olverembatinib has received upgraded recommendations from the 2024 Chinese Society of Clinical Oncology (CSCO) Guidelines for the Diagnosis and Treatment of Hematologic Malignancies in CML and Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL), plus a number of additional Level I recommendations from the guideline. In July 2024, olverembatinib was approved in Macau China for the treatment of adult patients with tyrosine kinase inhibitor (TKI)-resistant chronic-phase CML (CML-CP) or accelerated-phase CML (CML-AP) harboring the T315I mutation; and adult patients with CML-CP resistant to and/or intolerant of first- and second-generation TKIs, marking another major milestone for olverembatinib following initial approvals it received in the Chinese mainland. Global innovation strategy yielding more encouraging results with eight registrational studies being simultaneously advanced Guided by its global innovation strategy, Ascentage Pharma delivered multiple milestone achievements and swiftly advanced its global clinical development programs during the reporting period. To date, two of the company's key drug candidates, olverembatinib and lisaftoclax (APG-2575), have been cleared to enter 11 registrational studies, including 3 registrational studies that have been completed and 8 registrational studies currently ongoing. In February 2024, olverembatinib was cleared by the US Food and Drug Administration (FDA) to enter a global registrational Phase III study in previously treated adult patients with CML-CP. This is the first FDA-cleared registrational Phase III study of olverembatinib and it represents a big step forward for Ascentage Pharma's global clinical development in hematologic malignancies. Outside hematology, the Center for Drug Evaluation (CDE) of China's National Medical Product Administration (NMPA) cleared a global registrational Phase III study of olverembatinib in patients with succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumor (GIST) who had failed prior systemic treatment. This clearance represents a crucial step in Ascentage Pharma's clinical development in solid tumors. Meanwhile, Ascentage Pharma also made notable strides with the clinical development of lisaftoclax in hematologic malignancies. During the reporting period, the China CDE cleared a Phase III study of lisaftoclax in combination with azacitidine (AZA) for the first-line treatment of newly-diagnosed patients with higher-risk myelodysplastic syndrome (MDS). This is the fourth cleared registrational Phase III study of lisaftoclax. Winning recognition from around the world with robust clinical data spanning multiple indications As an innovative biopharmaceutical company focused on global innovation, Ascentage Pharma has built a high value pipeline composed of assets with first- and best-in-class potentials and conducted more than 40 clinical trials in China, the US, Australia, Europe, and Canada. In this process, Ascentage Pharma made steady progress exploring and validating a number of its drug candidates across multiple indications and presented updated results from its clinical programs at a number of international congresses, showcasing its prowess in R&D and clinical development. During the reporting period, results from four clinical studies of olverembatinib, lisaftoclax, and APG-2449, the company's three key drug candidates, were selected for presentations at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. This is the seventh consecutive year for Ascentage Pharma to present at the meeting. Among these results, the data of olverembatinib in patients with TKI-resistant SDH-deficient GIST were selected for an Oral Report. In the data, olverembatinib showed promising clinical benefit including a clinical benefit rate (CBR) of 92.3% and a median progression-free survival (PFS) of 25.7 months in patients with SDH-deficient GIST, an indication that currently lacks standard of care treatment. At the same meeting, Ascentage Pharma presented the latest data that further demonstrated the therapeutic potential of its FAK/ALK/ROS1 TKI APG-2449 for the treatment of non-small cell lung cancer (NSCLC). At the 2024 European Hematology Association Hybrid Congress (EHA 2024) which took place in June this year, Ascentage Pharma released update results from five studies of olverembatinib, lisaftoclax, and APG-5918, three of the company's key drug candidates. These data highlighted the company's competitiveness in the field of hematologic malignancies. The three studies of olverembatinib presented at EHA 2024 include one that reported the latest median 1-year follow-up data of olverembatinib in patients with CML and Ph+ ALL, updated from the data released at the 2023 American Society of Hematology (ASH) Annual Meeting. In the results, olverembatinib showed excellent durable clinical benefits and favorable long-term tolerability in patients who had been treated with multiple TKIs (including those who were resistant to ponatinib and/or asciminib), regardless of whether they harbored the T315I mutation. The latest data from multiple studies of lisaftoclax were also selected for presentations at the 2024 ASCO Annual Meeting and EHA 2024. These results highlighted lisaftoclax' therapeutic potential in a range of hematologic malignancies. Data released at this year's ASCO Annual Meeting include the latest promising results from a Phase Ib/II study of lisaftoclax in combination with AZA in patients with treatment-naïve (TN) or relapsed/refractory (R/R) acute myeloid leukemia (AML), and a global, multicenter Phase Ib/II study of lisaftoclax alone or in combinations for the treatment of patients with Waldenström macroglobulinemia (WM). At EHA 2024, the company presented encouraging updated data of lisaftoclax in patients with R/R multiple myeloma (MM) or immunoglobulin light-chain (AL) amyloidosis. Furthermore, results from three preclinical studies of olverembatinib, alrizomadlin (APG-115), APG-2449, and APG-5918 were announced at the 2024 American Association of Cancer Research Annual Meeting (AACR 2024), providing important scientific rationale for the future clinical development of these assets. Deepening global innovation while opening a whole new chapter on growth and development To support its long-term strategy and growth, during the reporting period, Ascentage Pharma has confidentially submitted a draft registration statement on Form F-1 to the US Securities and Exchange Commission (SEC) relating to the proposed initial public offering of American depositary shares (ADSs) representing its ordinary shares. Ascentage Pharma is steadfastly committed to its global innovation strategy and strengthening its intellectual property portfolio. As of June 30, 2024, Ascentage Pharma holds 520 issued patents globally, among of which 367 patents were issued outside of China. Dr. Dajun Yang, Chairman & CEO of Ascentage Pharma, said, "In the first half of 2024, Ascentage Pharma has achieved multiple milestones in global business development, product commercialization, and global clinical development. These achievements highlight our global innovative capabilities and competitiveness in the field of hematologic malignancies. During the reporting period, we entered into a global collaboration valued at up to US$1.3 billion with Takeda, a multinational pharmaceutical company, for olverembatinib, in an agreement that also includes tiered double-digit royalties on annual net sales. This is the largest BD deal for any small molecule oncology drug in China. Moreover, we have already received US$75 million in equity investment from Takeda. This collaboration is another validation of our leadership in the industry and olverembatinib's value in the global market that will hopefully demonstrate clinical potential in more conditions that currently lack treatment options. In February 2024, the US FDA cleared a global registrational Phase III study of olverembatinib, marking yet another major milestone in the global clinical development of the drug. It is worth emphasizing that during the reporting period, Ascentage Pharma achieved profitability for the first time in its history. With olverembatinib included into the China NRDL and approved for a new indication in the country, we are empowered to further strengthen our commercialization capabilities. At the same time, we made broad progress with our global clinical development programs, expanded the potential indications of our key drug candidates, and reinforced our competitiveness in the field of hematologic malignancies. Pressing ahead with our patient-centric global innovation strategy, we will continue to build out our commercialization capabilities and accelerate the global clinical development of our investigational assets. To fulfill our mission of addressing unmet clinical needs in China and around the world, we aspire to bring our innovative drugs to the global market as soon as possible for the benefit of more patients around the world and create additional value for societies and our investors." About Ascentage Pharma Ascentage Pharma (6855.HK) is a global, integrated biopharmaceutical company engaged in discovering, developing and commercializing both first-in-class and best-in-class 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 pipeline of 9 clinical-stage 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 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. SOURCE Ascentage Pharma

ROCKVILLE, Md. and SUZHOU, China, Aug. 12, 2024 /PRNewswire/ -- Ascentage Pharma (6855.HK), a global, integrated biopharmaceutical company engaged in discovering, developing and commercializing both first- and best-in-class therapies for malignancies, announced today it has been cleared by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) to initiate a registrational Phase III study of lisaftoclax (APG-2575), one of the company's key drug candidates, in combination with azacitidine (AZA) for the first-line treatment of newly-diagnosed patients with higher-risk myelodysplastic syndrome (MDS). This clears the fourth registrational Phase III study of lisaftoclax, marking another major milestone in the clinical development of the drug. This study (GLORA-4) is a multi-center, randomized, double-blind, pivotal registrational Phase III trial to evaluate the efficacy of lisaftoclax in combination with AZA in newly-diagnosed adult patients with higher-risk MDS. As a heterogeneous myeloid clonal disease originating from hematopoietic stem cells, MDS commonly occurs in older population with a median age of onset of 70 years and an incidence rate that increases with age.1 MDS is characterized by the abnormal growth of myeloid cells. Its clinical manifestations include hematopoietic failure, refractory cytopenia, and the propensity for high-risk patients to progress to acute myeloid leukemia (AML). Data show that approximately 10% of low-risk patients and 50% of high-risk patients with MDS would progress to AML and thereafter face a dismal prognosis.2 At present, there are limited treatment options for patients with MDS and the treatment outcome for most patients remains relatively poor. Demethylation agents (AZA or decitabine) are the current standard first-line treatment for patients with higher-risk MDS. Studies showed that compared to conventional care regimens, monotherapy with AZA can improve the overall survival of patients with MDS.3 Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only available curative treatment for MDS. However, the complex characteristics of MDS and the old ages of patients have resulted in the intolerability of chemotherapies that limited the rate of transplantation, and the high rate of transplantation-associated mortality among patients with MDS. The low survival rate of patients with higher-risk MDS underscores an urgent unmet medical need for novel therapies and medicines that can offer higher efficacies. Lisaftoclax is a novel, orally administered Bcl-2 selective inhibitor being developed by Ascentage Pharma to treat the patients with malignancies by selectively blocking the antiapoptotic protein Bcl-2 and hence restoring the normal apoptosis process in cancer cells. Lisaftoclax is expected to be the first Bcl-2 inhibitor for which an NDA will be filed for chronic lymphocytic leukemia (CLL)/ small lymphocytic lymphoma (SLL) in China and the second anywhere globally that has demonstrated clinical activity for the treatment of patients with CLL and entered pivotal registrational studies. In published clinical results released at the 2023 American Society of Hematology (ASH) Annual Meeting, the drug candidate lisaftoclax has demonstrated clinical benefit and tolerability in patients with higher-risk MDS.4 "There is considerable unmet clinical need for patients with MDS. Lisaftoclax, a Bcl-2 inhibitor, has already shown promising clinical benefit and tolerability in early studies in patients with MDS," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "We are very encouraged by this approval for initiation of the registrational Phase III study in the first-line treatment of patients with higher-risk MDS, as it clears the way for the fourth registrational Phase III study of lisaftoclax. Fulfilling our mission of addressing unmet clinical needs in China and around the world, we will actively press ahead with the clinical trials of lisaftoclax for the benefit of more patients." *Lisaftoclax is an investigational drug that has not been approved in any country or region. References: Fenaux P, Haase D, Santini V, et al. Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2021 Feb;32(2):142-156. doi: 10.1016/j.annonc.2020.11.002. NCCN Guidelines® Version 1.2023 on Myelodysplastic Syndromes Fenaux P, Mufti GJ, Hellstrom-Lindberg E, et al. International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32. doi: 10.1016/S1470-2045(09)70003-8. Wang H, Wei X, Jiang Q, et al. Safety and Efficacy of Lisaftoclax (APG-2575), a Novel BCL-2 Inhibitor (BCL-2i), in Relapsed or Refractory (R/R) or Treatment-Naïve (TN) Patients (Pts) with Acute Myeloid leukemia (AML), Myelodysplastic Syndrome (MDS), or Other Myeloid Neoplasms. Blood (2023) 142 (Supplement 1): 2925. About Ascentage Pharma Ascentage Pharma (6855.HK) is a global, integrated biopharmaceutical company engaged in discovering, developing and commercializing both first-in-class and best-in-class 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 pipeline of 9 clinical-stage 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 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. SOURCE Ascentage Pharma