Akeso Announces FDA Approval for Penpulimab-kcqx in Two BLA Indications for Comprehensive Treatment of Advanced Nasopharyngeal Carcinoma

HONG KONG, April 24, 2025 /PRNewswire/ -- Akeso, Inc. (9926.HK) ("Akeso" or the "Company") is excited to announce that the U.S. Food and Drug Administration (FDA) has approved its differentiated PD-1 monoclonal antibody, penpulimab-kcqx, in combination with cisplatin or carboplatin and gemcitabine for the first-line treatment of adult recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC). FDA also approved penpulimab-kcqx as a single agent for adults with metastatic non-keratinizing NPC with disease progression on or after platinum-based chemotherapy and with least one other prior line of therapy. Penpulimab-kcqx was developed independently by Akeso, with further development and commercialization managed through a joint venture with Chia Tai-Tianqing Pharmaceutical Group.

This milestone marks penpulimab-kcqx as Akeso's first internally developed innovative biologic to receive FDA approval. The approval underscores the robust clinical research behind penpulimab-kcqx and marks Akeso's successful entry into the United States regulatory system for the first time. This achievement highlights the company's innovative drug development capabilities and its commitment to adhering to the highest international standards in pharmaceutical quality management.

The FDA's approval of penpulimab-kcqx validates Akeso's international drug development strategy and expansion capabilities. This approval lays a strong foundation for Akeso's continued clinical development efforts in the global therapeutics markets.

Penpulimab-kcqx has been approved in China for two indications: 1. first-line treatment of advanced NPC, and 2. second or later line treatment of advanced NPC. The recent FDA approval of penpulimab-kcqx offers a new, immunotherapy option for advanced NPC patients in the US.

The FDA approval is based on the international Phase III clinical trial AK105-304 and the pivotal AK105-202 study, which supported the two Biologics License Application (BLA) for penpulimab-kcqx. These studies demonstrated the drug's clinical benefits and favorable safety profile across  two stages of treatment for metastatic NPC.  AK105-304 is a randomized, double-blind, international Phase III trial that enrolled NPC patients of diverse ethnicities. The data will be presented at the 2025 American Association for Cancer Research (AACR) Annual Meeting. Previously, the FDA granted penpulimab-kcqx Breakthrough Therapy Designation (BTD), Orphan Drug Designation (ODD), and Fast Track Designation (FTD) for NPC treatment, highlighting the critical unmet need for this therapy.

According to the WHO 2020 Global Cancer Statistics, over 133,000 new NPC cases are diagnosed annually worldwide, with more than 70% of the patients presented with locally advanced disease. Recurrent or metastatic NPC has a poor prognosis and limited survival. Penpulimab-kcqx's FDA approval will expand the number of NPC patients that can benefit from its treatment.

Prof. Chaosu Hu, Principal Investigator of penpulimab-kcqx from Fudan University Shanghai Cancer Center, commented: "This milestone enhances international treatment guidelines for advanced NPC and extends the benefits of China's innovations to global patients, ultimately reshaping the treatment landscape for metastatic NPC worldwide."

Prof. Xiaozhong Chen, Investigator of penpulimab-kcqx from Zhejiang Cancer Hospital, added: "The FDA approval of penpulimab-kcqx confirms its high efficacy and low toxicity, positioning China's innovative drug development in alignment with international standards."

Dr. Yu Xia, Founder, Chairwoman, President & CEO of Akeso, expressed: "We are very excited by the approval of penpulimab-kcqx's approval in the US FDA for first line and later line NPC.  Beyond reaching our first international regulatory milestone, this approval also provides an important immunotherapy treatment option for patients with NPC in the United States. The FDA approval of penpulimab-kcqx not only highlights the quality of our innovation but also underscores Akeso's focus on delivering treatments for difficult to treat cancers for patients around the world. We are deeply grateful to all the researchers, participants, and patients who have contributed to this success. Akeso will continue to advance first and best in class therapies, including bispecific antibodies and CD47 inhibitors, challenge global standards of care and unlocking the full potential of our pipeline for cancer patients everywhere."

Forward-Looking Statement of Akeso, Inc.

This announcement by Akeso, Inc. ( 9926. HK) contains "forward-looking statements". These statements reflect the current beliefs and expectations of Akeso's management and are subject to significant risks and uncertainties. There can be no assurance that penpulimab-kcqx or other pipeline candidates will obtain the required regulatory approvals or achieve commercial success. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements.

Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company's ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company's patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions.

Akeso does not undertake any obligation to publicly revise these forward-looking statements to reflect events or circumstances after the date hereof, except as required by law.

Please see Prescribing Information for penpulimab-kcqx at https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm when it is posted.

About Akeso
Akeso (HKEX: 9926.HK) is a leading biopharmaceutical company committed to the research, development, manufacturing and commercialization of the world's first or best-in-class innovative biological medicines. Founded in 2012, the company has created a unique integrated R&D innovation system with the comprehensive end-to-end drug development platform (ACE Platform) and bi-specific antibody drug development technology (Tetrabody) as the core, a GMP-compliant manufacturing system and a commercialization system with an advanced operation mode, and has gradually developed into a globally competitive biopharmaceutical company focused on innovative solutions. With fully integrated multi-functional platform, Akeso is internally working on a robust pipeline of over 50 innovative assets in the fields of cancer, autoimmune disease, inflammation, metabolic disease and other major diseases. Among them, 24 candidates have entered clinical trials (including 15 bispecific/multispecific antibodies and bispecific ADCs. Additionally, 7 new drugs are commercially available, and 4 new drugs with 4 new indications are under regulatory review for approval. Through efficient and breakthrough R&D innovation, Akeso always integrates superior global resources, develops the first-in-class and best-in-class new drugs, provides affordable therapeutic antibodies for patients worldwide, and continuously creates more commercial and social values to become a global leading biopharmaceutical enterprise.

For more information, please visit https://www.akesobio.com/en/about-us/corporate-profile/ and follow us on Linkedin.

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