Ascentage Pharma Announces 2021 Annual Results with Commercialization Programs Going Full Steam Ahead

SUZHOU, China and ROCKVILLE, Md., March. 21, 2022 /PRNewswire/ -- Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today released its annual results for full year 2021, during which the company achieved significant progress realizing its vision of global innovation strategy including key areas such as clinical development, strategic partnerships and intellectual property rights. During the same period, the company's lead asset, the third-generation BCR-ABL inhibitor olverembatinib (HQP1351) was granted marketing authorization in China, marking a transformative step in the company's transition from a biotech to a full-fledged biopharmaceutical company with commercialized products, officially opening the new chapter on commercialization.

During the reporting period, Ascentage Pharma achieved steady improvement to its cash flow. While commercializing its first approved product, the company raised RMB1,300 million through the issuance and placement of additional shares in 2021. The company's cash and bank balances on December 31, 21, increased by 70.3% from the same time last year, to RMB1,740 million. During the reporting period, the company recorded a total revenue of RMB27.91 million, which was increased by 123.2% from the same period last year. This increase in revenue is primarily attributable to the sales of the company's first pharmaceutical product, commercialization license fee income and patented IP license fee income from customers. It is also worth noting that since receiving the approval for olverembatinib till the end of February, 2022, the product realized an accumulated invoice amount of RMB50.41 million (unaudited, inclusive of value added tax).

The company's first marketing authorization officially opens the chapter on commercialization

In November 2021, olverembatinib, Ascentage Pharma's lead asset and the novel drug that was designated a National Major New Drug Development and Manufacturing Program, was approved in China for the treatment of adult patients with tyrosine kinase inhibitor (TKI)-resistant chronic phase chronic myeloid leukemia (CML-CP) or accelerated-phase CML (CML-AP) harboring the T315I mutation as confirmed by a validated diagnostic test. This approval marked a transformative step in Ascentage Pharma's transition from a biotech to a full-fledged biopharmaceutical company with approved products and commercialization capabilities. This approval for the first third-generation BCR-ABL inhibitor in China addresses an urgent unmet clinical need in the country, effectively ending the lack of treatment options for Chinese patients with drug-resistant CML harboring the T315I mutation. Since receiving the approval for olverembatinib till the end of February 2022, the product realized an accumulated invoice amount of RMB50.41 million (unaudited, inclusive of value added tax).

To date, Ascentage Pharma has built a commercialization team possessing a strong track record in the field of hematology and entered into a broad strategic partnership with Innovent Biologics ("Innovent") on the joint-commercialization of olverembatinib in China. During the reporting period, Ascentage Pharma steadily advanced the product's commercialization and access programs through strategic partnerships with multiple distribution service providers such as Sinopharm Group, Shanghai Pharmaceutical and CR Pharma. In the two months following its launch in China, olverembatinib was included into Hui Min Bao, a government-backed commercial supplemental insurance plan of 10 municipalities, with Huzhou being the first city to include olverembatinib in the first month after the drug was approved. Such inclusions are expected in many more cities in 2022.

Also in 2021, we officially inaugurated the company's global headquarters and R&D center, creating a strong base supporting the company's accelerating global expansion and its ongoing integration of internal and external resources, has extended Ascentage Pharma's competitive-edge in the innovative drug arena.

Remaining steadfastly committed to global innovation while rapidly advancing clinical development programs

In 2021, Ascentage Pharma continued to increase its investment in innovation, with the research and development expenses increased by 35.8% year on year, to RMB767 million. At present, Ascentage Pharma has built a rich and high-potential pipeline comprising a range of assets with first-in-class or best-in-class potentials globally.

The company has rapidly advanced its global clinical development programs, and is currently conducting over 50 Phase I/II clinical trials in the United States, Australia, Europe, and China. Meanwhile, the company has further strengthened its intellectual property portfolio, paving the way for its accelerating research and development. As of December 31, 2021, the company holds 178 issued patents and more than 600 patent applications globally, among of which, 135 patents were issued overseas.

Highlighting the company's progress in clinical development, olverembatinib became Ascentage Pharma's first drug candidate granted the Orphan Designation in the European Union (EU); meanwhile, the MDM2-p53 inhibitor alrizomadlin (APG-115) with "first-in-class" potential, was granted additional Orphan Drug Designations (ODDs) and its first Fast Track Designation (FTD) by the US Food and Drug Administration (FDA). To date, Ascentage Pharma has obtained a total of 2 FTDs and 15 ODDs from the FDA and 1 Orphan Designation from the EU, setting a record in the number of designations granted to any Chinese biopharmaceutical company and is a manifestation of the company's capabilities in global innovation.

During the reporting period, Ascentage Pharma rapidly advanced its drug candidates in clinical development. The third pivotal Phase II study of the company's lead drug candidate, olverembatinib, in patients with CML resistant/intolerant to first- and second-generation TKIs, has already completed its patient enrollment. In March 2021, a Breakthrough Therapy Designation for the indication was granted to olverembatinib by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA), accelerating the future development and regulatory approval of the product.

The pivotal Phase II study of lisaftoclax (APG-2575), the first China-developed Bcl-2 selective inhibitor entering clinical development in China, for the treatment of relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (R/R CLL/SLL) was approved by the CDE of the NMPA in China by the end of 2021, and subsequently dosed its first patient in March 2022, making lisaftoclax the second Bcl-2 selective inhibitor entering pivotal trials globally. Moreover, lisaftoclax was cleared by the US FDA in June 2021 to enter a clinical study evaluating the drug candidate as a single agent or in combination with other antitumor therapies for the treatment of patients with advanced estrogen receptor-positive (ER+) breast cancer or other solid tumors, making its first move in indication-expansion to solid tumors. Lisaftoclax is currently being investigated in 18 clinical studies globally in a range of hematologic malignancies and solid tumors, and has thus far showed enormous therapeutic potential.

Validating best-in-class and first-in-class potentials in clinical development focused delivering meaningful clinical benefits

As a global leader in the development of apoptosis-targeting therapies, Ascentage Pharma has continued to demonstrate its capabilities in global innovation by attracting widespread interest at major scientific events and delivering impressive progress with the clinical development of its apoptosis-targeting drug candidates.

Results from three clinical studies of olverembatinib were selected as abstracts, including one oral presentation, at the 2021 American Society of Hematology (ASH) Annual Meeting. This was the fourth consecutive year in which data of olverembatinib were selected for oral presentation at the ASH Annual Meeting, an indication of the widespread interest in the drug's efficacy and safety from the international hematology community. Results of olverembatinib reported in the oral presentation included the long-term follow-up data (at a median follow-up duration of 39 months) further demonstrated a favorable tolerability, potent and durable efficacy, and clear best-in-class potential in the treatment of patients with TKI-resistant CML. In addition, the two pivotal studies in patients with T315I-mutant CML also showed promising results.

The clinical data of the Bcl-2 inhibitor lisaftoclax, a key candidate of the company's apoptosis-targeted pipeline, were also presented at the 2021 ASH Annual Meeting. These results included the first data readout on lisaftoclax in Chinese patients with hematologic malignances, which demonstrated favorable tolerability and great therapeutic potential, in absence of any tumor lysis syndrome (TLS). The six patients with CLL who received lisaftoclax at 200 mg or higher doses achieved an overall response rate (ORR) of 100% and one complete response. Prior to the ASH Annual Meeting, Ascentage Pharma had released the most updated data from the first-in-human study of lisaftoclax in patients with R/R CLL/SLL and other hematologic malignancies at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting, including preliminary data demonstrating an objective response rate (ORR) of 80% and a manageable safety profile, in absence of any TLS despite daily dose ramp-up. All these data collectively further validated lisaftoclax as a drug candidate with global best-in-class potential.

In an oral presentation at the 2021 ASCO Annual Meeting, Ascentage Pharma reported the latest results from the Phase II study of alrizomadlin, a MDM2-p53 inhibitor and another key candidate of the company's apoptosis-targeted pipeline, in combination with pembrolizumab in patients with unresectable or metastatic melanoma or advanced solid tumors that have progressed on prior immuno-oncologic drugs. These results provided the clinical evidence supporting the potential synergy between a MDM2-p53 inhibitor and an immune checkpoint inhibitor, and the first-in-class potential of alrizomadlin. Furthermore, the preclinical results of alrizomadlin were published in the renowned scientific journal, Nature Immunology, in March 2021 has demonstrated MDM2's pivotal biological role in T-cell stability, survival, and antitumor immunity, potentially laying a foundation for synergistic effects between MDM2-targeted agents such as alrizomadlin and cancer immunotherapy.

Expanded external collaborations through multiple new landmark partnerships

In the past year, Ascentage Pharma entered into a multifaceted strategic collaboration with Innovent on the joint-commercialization of olverembatinib in China, the joint clinical development of lisaftoclax, and equity investment in Ascentage Pharma. This strategic collaboration created a new partnering model for the development of innovative therapies in China, and the two companies will collaborate in every possible manner to accelerate the introduction of China-developed innovative therapies to the world and bring clinical benefit to more patients in need.

While building win-win partnerships in China, Ascentage Pharma has continuously sought new partners overseas. To accelerate its global innovation, the company entered into a Cooperative Research and Development Agreement (CRADA) with the world-leading research institute, the National Cancer Institute (NCI), to collaborate on the non-clinical and clinical development of Ascentage Pharma's drug compound pelcitoclax (APG-1252).

Moreover, Ascentage Pharma received another milestone payment from its global licensee, UNITY Biotechnology ("UNITY"), after UNITY reported positive data from a Phase I clinical study of the senolytic drug candidate UBX1325 and dosed the first patient in the subsequent Phase IIa clinical study. In the future, Ascentage Pharma will continue to work closely with UNITY in the development of anti-aging drugs that will bring hope to patients around the world.

During the same period, Ascentage Pharma entered into a clinical trial collaboration and supply agreement with Pfizer Inc. ("Pfizer) to evaluate the combination of Ascentage Pharma's Bcl-2 selective inhibitor, lisaftoclax, in combination with Pfizer's IBRANCE® (palbociclib), a CDK4/6 inhibitor, in patients with recurrent, locally advanced or metastatic estrogen receptor-positive (ER+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer. This collaboration with Pfizer can potentially further accelerate the clinical development of lisaftoclax.

Dr. Dajun Yang, Chairman & CEO of Ascentage Pharma, said, 'In 2021, Ascentage Pharma achieved remarkable progress in commercialization, clinical development and business development. These progress are highlighted by the approval of the third-generation BCR-ABL inhibitor, olverembatinib, as it is milestone signifying that Ascentage Pharma has officially entered the commercial-stage.

The launch of olverembatinib is a strong validation of our effort in fulfilling the founding mission of addressing unmet clinical needs in China and around the world, and our patient-focused global innovation strategy. Meanwhile, the data readouts of lisaftoclax and alrizomadlin at renowned international scientific meetings such as those of the ASCO and ASH once again highlighted these drug candidates' best-in-class and first-in-class potentials, as well as their potential therapeutic utility and clinical superiorities.

Furthermore, we have actively expanded our strategic partnerships through the comprehensive collaboration with Innovent that created a new partnering model for the development of innovative therapies in China. Globally, we have also entered into multiple landmark agreements with leading research institutions and pharmaceutical companies such as NCI and Pfizer.

As a company focused on original pharmaceutical innovation, we will continue to advance the indication expansion and global clinical development of olverembatinib, while pressing ahead with the development programs of other investigational assets. We will remain steadfastly committed to global innovation and the mission of addressing unmet clinical needs in China and around the world, to create additional value for our investors and bring much needed novel therapies to our patients as soon as possible.'

About Ascentage Pharma

Ascentage Pharma (6855.HK) is a globally focused biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B, and age-related 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 (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 is conducting more than 50 Phase I/II clinical trials in the US, Australia, Europe, and China. Olverembatinib, the company's core drug candidate developed for the treatment of drug-resistant chronic myeloid leukemia (CML), was granted Priority Review status and a Breakthrough Therapy Designation (BTD) by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA), and is already approved for the indication. In addition, the olverembatinib was also granted an Orphan Drug Designation (ODD) and a Fast Track Designation (FTD) by the US FDA, and an Orphan Designation by the EU. To date, Ascentage Pharma has obtained a total of 15 ODDs from the US FDA and 1 ODD from the EU for four of the company's investigational drug candidates. Ascentage Pharma has been designated for multiple Major National R&D Projects, including five National Major New Drug Discovery and Manufacturing projects, one New Drug Incubator status, four Innovative Drug Programs, and one Major Project for the Prevention and Treatment of Infectious Diseases.

Leveraging its robust R&D capabilities, Ascentage Pharma has built a portfolio of global intellectual property rights and entered into global partnerships with numerous renowned biotechnology and pharmaceutical companies and research institutes such as UNITY Biotechnology, MD Anderson Cancer Center, Mayo Clinic, Dana-Farber Cancer Institute, MSD, AstraZeneca, and Pfizer. The company has built a talented team with global experience in the discovery and development of innovative drugs and is setting up its world-class 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 or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.

SOURCE Ascentage Pharma