On March 27, 2024 Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported its annual results for the full year 2023 (Press release, Ascentage Pharma, MAR 27, 2024, View Source [SID1234641516]). During the reporting period, Ascentage Pharma made further strides in revenue generation through its commercialized product, achieved rapid progress with its patient-centric global innovation strategy, and delivered numerous milestones in clinical development and global expansion.
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Newly approved indication of olverembatinib expands the drug’s addressable market
In 2023, Ascentage Pharma recorded a revenue of RMB222 million, most of which was attributable to product sales and milestone payments, including a revenue of RMB194 million from product sales. For the first time, the company achieved commercial breakeven through topline growth while keeping selling and distribution expenses at a manageable level and significantly improving the efficiency of marketing activities.
Developed internally by Ascentage Pharma, olverembatinib is the first and only approved third-generation BCR-ABL inhibitor in China. In January 2023, as a Model Product of China-Developed Innovative Drugs, olverembatinib was included into the 2022 National Reimbursement Drug List (NRDL), a major step-forward that drastically improved the drug’s accessibility and paved the way for accelerated volume growth. Since its approval till December 31, 2023, olverembatinib generated an accumulated sales revenue of RMB362 million (audited, inclusive of value-added tax).
Meanwhile, Ascentage Pharma continued to strengthen its ability to drive revenue growth through product commercialization. On November 17, 2023, olverembatinib was approved for adult patients with chronic-phase chronic myeloid leukemia (CML-CP) resistant and/or intolerant of first-and second-generation tyrosine kinase inhibitors (TKIs). The indication expansion will allow a broader population of patients with CML to benefit from the drug and offer a new treatment option to drug-resistant patients, which will further olverembatinib’s commercial success. During the reporting period, the sales volume of olverembatinib increased 259%, the number of listed hospitals increased 567%, the total number of patients on treatment increased 123%. Looking ahead in 2024, with the approval of the new indication of olverembatinib, compounded by accelerated hospital listings, a growing number of patients on treatment, and ever longer duration of treatment, more patients will benefit from olverembatinib, driving the sales of the product.
Obtained clearances for five global registrational Phase III trials as the global innovation strategy began to bear fruit
Led by its global innovation strategy, Ascentage Pharma has delivered multiple milestones in clinical development as it effectively advanced global clinical development programs. Since the beginning of 2023, olverembatinib and the Bcl-2 inhibitor lisaftoclax (APG-2575) have been cleared to commence five global registrational Phase III studies, bolstering the company’s moat in the field of hematologic malignancies.
In February 2024, the US Food and Drug Administration (FDA) cleared a global registrational Phase III trial of olverembatinib in previously treated adult patients with CML-CP. This is the first global registrational Phase III trial of olverembatinib cleared by the US FDA and represents another step in Ascentage Phara’s growing presence in the global hematology arena. In January 2024, as a global best-in-class innovative drug from China, olverembatinib was included in the National Comprehensive Cancer Network (NCCN)’s latest guidelines for the management of CML. This inclusion highlights the therapeutic utility of olverembatinib and marks another major recognition from the global oncology community.
In addition to CML, olverembatinib has also made significant progress with its clinical development for the treatment of patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). In July 2023, the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) granted the clearance for the global registrational Phase III trial of olverembatinib in combination with chemotherapy versus imatinib in combination with chemotherapy for the treatment of newly diagnosed patients with Ph+ ALL, paving the way for olverembatinib to potentially become the first China-approved TKI for the treatment of patients with Ph+ ALL in the first-line setting.
Lisaftoclax obtained clearances for three global registrational Phase III studies. In August 2023, the US FDA cleared a global registrational Phase III study of lisaftoclax in previously treated patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). This regulatory clearance marked a major step-forward in the global clinical development of lisaftoclax as it can potentially accelerate the drug’s journey to market as the world’s second approved Bcl-2 inhibitor competing in the global market. Shortly after receiving this clearance, lisaftoclax was approved by the China CDE to enter a global registrational Phase III study of lisaftoclax combined with acalabrutinib, a Bruton’s tyrosine kinase (BTK) inhibitor, versus immunochemotherapy for the first-line treatment of patients with CLL/SLL. In December 2023, the China CDE approved another global registrational Phase III study of lisaftoclax for the first-line treatment of newly diagnosed elderly or unfit patients with acute myeloid leukemia (AML).
Promising clinical results underscore strong competitiveness in the field of hematologic malignancies
Pursuing its mission of addressing unmet clinical needs in China and around the world, Ascentage Pharma has built a rich pipeline composed of promising drug candidates with first-in-class and/or best-in-class potentials, and is conducting more than 40 clinical studies in China, the US, Australia, Europe, and Canada. The company’s innovative and clinical development capabilities have received growing recognition from the global research community as it continued to explore and validate the therapeutic potential of its drug candidates and frequently showcased new clinical data at major international congresses.
At the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) (ASH 2023), Ascentage Pharma presented the latest results from multiple studies of olverembatinib and lisaftoclax, demonstrating the clinical advantages of these products.
Among those studies, two studies of olverembatinib were selected for Oral Presentations, making 2023 the sixth consecutive year in which data of olverembatinib were selected for Oral Presentations at the ASH (Free ASH Whitepaper) Annual Meeting. One of these Oral Presentations featured a registrational Phase II study evaluating the efficacy and safety of olverembatinib versus the best available therapy (BAT) in patients with CML-CP resistant and/or intolerant to prior treatment with TKIs. Results of the study showed that compared to the BAT, olverembatinib significantly improved the prognosis of patients with CML. The other Oral Presentation featured the preliminary results from a Phase II study of olverembatinib. Selected for "Highlights of ASH (Free ASH Whitepaper)", these results showed encouraging clinical benefit and favorable tolerability of an olverembatinib regimen that could potentially usher in an era of chemotherapy-free treatment for Ph+ ALL.
After releasing preliminary results from the US study of olverembatinib in an Oral Report at the ASH (Free ASH Whitepaper) Annual Meeting in 2022, at ASH (Free ASH Whitepaper) 2023, Ascentage Pharma presented updated data from a larger patient sample that showed the favorable clinical benefit and tolerability of olverembatinib, as a monotherapy and in combinations, in heavily pretreated patients with CML or Ph+ ALL, particularly those who have failed prior treatment with the third-generation TKI ponatinib or the allosteric STAMP inhibitor asciminib. These results suggest that olverembatinib has global best-in-class potential as an effective new therapy for patients with CML or Ph+ ALL.
Also at the 2023 ASH (Free ASH Whitepaper) Annual Meeting, Ascentage Pharma released results from three clinical studies of lisaftoclax, for the treatment of CLL, AML, and multiple myeloma (MM), separately. Among them, the study in patients with relapsed/refractory (R/R) CLL once again showed the favorable tolerability and significant efficacy of lisaftoclax. In particular, the company released the first dataset of lisaftoclax in MM and AML that reaffirmed the global best-in-class potential and unique therapeutic utility of lisaftoclax and provided strong evidence of the drug’s therapeutic applications to indications beyond CLL.
Prior to that, Ascentage Pharma presented updated clinical data of olverembatinib, lisaftoclax, the MDM2-p53 inhibitor alrizomadlin (APG-115), and the FAK/ALK/ROS1 inhibitor APG-2449, four of the company’s lead drug candidates, at the 59th American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO 2023). In particular, the data of olverembatinib showed promising clinical benefit and favorable tolerability in patients with TKI-resistant succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumor (GIST), thus indicating a potential breakthrough for an indication that currently has no effective treatment. Also in 2023, olverembatinib was granted a Breakthrough Therapy Designation (BTD) by the China CDE for the treatment of patients with SDH-deficient GIST who had received first-line treatment.
Building vertically integrated industrialization capabilities to further accelerate global expansion
Upholding its global innovation strategy, Ascentage Pharma is committed to building a large portfolio of global intellectual property rights. As of December 31, 2023, Ascentage Pharma holds 498 issued patents globally, among which 352 patents were issued overseas.
In addition, Ascentage Pharma received a zero-deficiency report from the Good Manufacturing Practice (GMP) compliance audit by a Qualified Person (QP) of the European Union (EU) in April 2023, which indicates that the company’s Global Manufacturing Center and its quality management system are now fully compliant with the GMP standards of the EU, generating extra tailwind to Ascentage Pharma’s ongoing transition towards a global biopharmaceutical company.
"In 2023, Ascentage Pharma has achieved multiple milestones across product commercialization, global clinical development, and corporate development, while continuing to showcase its prowess in global innovation," said Dr. Dajun Yang, Chairman and CEO of Ascentage Pharma. "The NRDL inclusion and newly approved indication of olverembatinib has further boosted the company’s commercialization capabilities. Meanwhile, olverembatinib, supported by its excellent efficacy and safety, was reaffirmed as a best-in-class drug and begun to show encouraging therapeutic value in additional indications that currently have no treatment options."
Dr. Yang continued, "In the past year, we accelerated our global clinical development programs and have in the process obtained clearances, including two from FDA, for five global registrational Phase III trials of olverembatinib and lisaftoclax. At the same time, our lead drug candidates continued to show clinical potential in additional indications, thus further strengthening our position in the field of hematologic malignancies."
In conclusion, Dr. Yang said, "Implementing our global innovation strategy with a patient-centric approach, we have built a rich pipeline that holds enormous clinical potential, particularly in hematologic malignancies. Moving forward, we will further enhance our commercialization and clinical development capabilities, and accelerate our global clinical development programs. Fulfilling the mission of addressing unmet clinical needs in China and around the world, we aim to bring our innovative drugs to the global market in the future for the benefit more patients around the world and create greater value for our communities and shareholders."