China NMPA Accepts Zymeworks' Application for Zanidatamab in Second-Line Biliary Tract Cancer Treatment

Zymeworks Inc., a biotechnology company focused on the development of innovative biotherapeutics, recently received acceptance for its Biologics License Application (BLA) for zanidatamab by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China. This acceptance marks a significant step forward in the treatment of HER2-positive biliary tract cancer (BTC) for patients who have undergone previous treatments and have unresectable, locally advanced, or metastatic disease.

Under the licensing agreement with BeiGene, Zymeworks is set to receive an $8 million milestone payment and remains eligible for up to $164 million based on further milestones in addition to royalties on product sales in the Asia Pacific region. This collaboration has already brought Zymeworks $53 million in upfront and milestone payments.

The BLA is grounded on data from the HERIZON-BTC-01 clinical trial, which was published in the Lancet Oncology in June 2023. This open-label phase 2b study assessed the efficacy and safety of zanidatamab in previously treated patients with HER2-positive BTC. The results were promising, showing an objective response rate (ORR) of 41.3%, a median duration of response (DOR) of 12.9 months, and a median progression-free survival (PFS) of 5.5 months.

Kenneth Galbraith, Chair and CEO of Zymeworks, expressed the significance of this BLA acceptance, highlighting the global effort to provide targeted treatment options for advanced or metastatic HER2-positive BTC. He thanked the dedicated teams at Zymeworks, BeiGene, and the study investigators, patients, and families involved in the development program for zanidatamab. Galbraith reiterated zanidatamab's potential as a new treatment for various HER2-expressing cancers, with ongoing Phase 3 trials for advanced or metastatic HER2-positive BTC and HER2-positive gastroesophageal adenocarcinoma.

Clare Fisher, Senior Vice President of Business Development at BeiGene, emphasized the collaborative effort in advancing cancer medicines to a global patient base. She acknowledged Zymeworks' role in achieving this milestone and expressed eagerness for continued partnership.

Zanidatamab is a bispecific antibody that targets the HER2 receptor through biparatopic binding. This design allows it to engage multiple mechanisms of action, including dual HER2 signal blockade, removal of HER2 protein from the cell surface, and immune-mediated cytotoxicity. Developed initially by Zymeworks, zanidatamab is now being advanced by Jazz Pharmaceuticals and BeiGene under licensing agreements.

The U.S. Food and Drug Administration (FDA) has also accepted and granted Priority Review to zanidatamab’s BLA, setting a target action date of November 29, 2024. The FDA has given zanidatamab several designations, including Breakthrough Therapy for previously treated HER2 gene-amplified BTC, and Fast Track for refractory BTC and first-line gastroesophageal adenocarcinoma. Additionally, zanidatamab has received Orphan Drug designations from both the FDA and the European Medicines Agency for BTC and gastric cancer.

The HERIZON-BTC-01 trial was a global, multicenter, open-label, single-arm phase 2b study that evaluated the efficacy and safety of zanidatamab in patients with previously treated advanced or metastatic HER2-amplified BTC. The primary endpoint was the objective response rate (ORR) assessed by independent central review, with secondary endpoints including the duration of response (DOR), progression-free survival (PFS), and overall survival (OS).

Biliary tract cancer, including gallbladder cancer and cholangiocarcinoma, is relatively rare and often diagnosed at an advanced stage, resulting in a poor prognosis. Approximately 12,000 people across the U.S., Europe, and Japan are diagnosed with BTC annually, with most cases being inoperable.

Zymeworks continues to drive forward its mission to develop novel biotherapeutics for difficult-to-treat cancers and other diseases. The company’s innovative platforms and drug development engine enable the precise engineering of highly differentiated antibody-based therapies. Through collaborations and strategic partnerships, Zymeworks aims to address significant unmet medical needs with its advanced pipeline of product candidates.

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