• Pursuant to the terms of the licensing agreement between Zymeworks and BeiGene, Zymeworks is entitled to receive an $8 million milestone payment from BeiGene and remains eligible to receive up to $164 million based on additional milestones plus royalties on product sales
• A Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for zanidatamab as a treatment for previously-treated, unresectable, locally advanced, or metastatic HER2-positive biliary tract cancer (BTC) has also been recently accepted for Priority Review by the FDA with target action date of November 29, 2024

 Zymeworks Inc, a clinical-stage biotechnology company developing a diverse pipeline of novel, multifunctional biotherapeutics to improve the standard of care for difficult-to-treat diseases, today announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China has accepted the BLA for zanidatamab for second-line treatment of HER2-positive BTC. Under the terms of Zymeworks’ Asia Pacific license and collaboration agreement with BeiGene for the development and commercialization of zanidatamab, Zymeworks is entitled to receive an $8 million milestone payment. Zymeworks also remains eligible to receive up to $164 million based on additional milestones plus royalties on product sales in the Asia Pacific region.

This BLA is based on the data from the HERIZON-BTC-01 (NCT04466891, CTR20202607) clinical trial, which was published in Lancet Oncology in June 2023¹. The trial is an open-label phase 2b study that evaluated the efficacy and safety of zanidatamab in previously treated patients with unresectable, locally advanced, or metastatic HER2-positive BTC. Zanidatamab demonstrated clinically meaningful anticancer activity and durable responses in subjects with previously treated HER2-positive BTC. As of October 10, 2022, the objective response rate (ORR), median duration of response (DOR), and median progression-free survival (PFS) assessed by independent central review (ICR) were 41.3% (95% CI: 30.4–52.8), 12.9 months (95% CI: 6.0–not estimable), and 5.5 months (95% CI: 3.7–7.2), respectively.

“Acceptance of this BLA in China represents a significant milestone in the global effort to bring effective, targeted treatment options to those affected by locally advanced or metastatic HER2-positive BTC,” said Kenneth Galbraith, Chair and Chief Executive Officer of Zymeworks. “We are grateful to all of the stakeholders who have worked tirelessly to help us reach this milestone, including our dedicated teams in manufacturing, quality control, regulatory affairs, and clinical research at Zymeworks, and our collaboration partner in Asia Pacific, BeiGene, as well as the study investigators, patients, and families who have supported this development program for zanidatamab. We remain confident about zanidatamab’s potential as a new treatment option for multiple HER2-expressing cancers, with ongoing Phase 3 trials in first-line advanced or metastatic HER2-positive BTC (HERIZON-BTC-302) and first-line HER2-positive gastroesophageal adenocarcinoma (HERIZON-GEA-01).”

“We further our mission by collaborating with other innovative companies to advance the development and delivery of impactful cancer medicines to more patients around the world,” said Clare Fisher, Senior Vice President of Business Development at BeiGene. “We thank our partners at Zymeworks for their dedication and contributions toward achievement of this important milestone for zanidatamab. We look forward to continued partnership as we work to reach more patients in the Asia-Pacific region.” 

Under the terms of its collaboration with BeiGene regarding zanidatamab, Zymeworks has received $53 million in upfront and milestone payments as well as certain co-development funding for zanidatamab clinical studies, not including the $8 million milestone payment Zymeworks is eligible to receive in connection with the NMPA’s acceptance of the BLA for zanidatamab for second-line treatment of HER2-positive BTC. Through our collaboration with BeiGene on zanidatamab, we remain eligible to receive up to $164 million in additional development and commercial milestones together with tiered royalties of up to 19.5% of net sales in BeiGene territories.