Pharma Focus Asia

Zymeworks Announces NMPA Acceptance of Zanidatamab Biologics License Application for Second-Line Treatment of Biliary Tract Cancer in China

Tuesday, June 11, 2024

Zymeworks Inc. (Nasdaq: ZYME), a clinical-stage biotechnology firm focused on developing innovative biotherapeutics, announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China has accepted the Biologics License Application (BLA) for zanidatamab. This BLA pertains to the use of zanidatamab for the second-line treatment of HER2-positive biliary tract cancer (BTC). As a result of this milestone, Zymeworks will receive an $8 million payment from its Asia Pacific partner, BeiGene. The company also stands to gain up to $164 million from future milestones and will earn royalties on sales in the Asia Pacific region.

This application is based on findings from the HERIZON-BTC-01 clinical trial (NCT04466891, CTR20202607), which were published in Lancet Oncology in June 2023. This phase 2b study assessed the effectiveness and safety of zanidatamab in patients with unresectable, locally advanced, or metastatic HER2-positive BTC who had previously undergone treatment. The results showed 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, all determined by independent central review (ICR).

"Acceptance of this BLA in China is a significant step towards providing targeted treatment options for those with advanced or metastatic HER2-positive BTC," stated Kenneth Galbraith, Chair and CEO of Zymeworks. He acknowledged the efforts of various teams involved in the process and emphasized zanidatamab’s potential as a treatment for multiple HER2-expressing cancers, citing ongoing Phase 3 trials for HER2-positive BTC and gastroesophageal adenocarcinoma.

Clare Fisher, Senior Vice President of Business Development at BeiGene, highlighted the importance of global collaboration in advancing cancer treatments. She praised the partnership with Zymeworks and expressed a commitment to bringing impactful cancer therapies to patients across the Asia-Pacific region.

Zymeworks has already received $53 million in payments and co-development funding from BeiGene, not including the recent $8 million milestone. The company remains eligible for up to $164 million in additional development and commercial milestones, along with tiered royalties of up to 19.5% of net sales in BeiGene territories.

Zanidatamab is a bispecific antibody targeting HER2, capable of binding two different HER2 epitopes simultaneously (biparatopic binding). This results in enhanced HER2 signal blockade, removal of HER2 from cell surfaces, and induction of immune-mediated cytotoxicity, showing promising antitumor activity. Developed by Zymeworks, zanidatamab is being tested in various clinical trials for HER2-expressing solid tumors. It is licensed to Jazz Pharmaceuticals and BeiGene for development and commercialization.

The U.S. FDA has granted Priority Review to the BLA for zanidatamab, with a Prescription Drug User Fee Act (PDUFA) action date of November 29, 2024. Additionally, zanidatamab has received Breakthrough Therapy designation for HER2-positive BTC, Fast Track designation for refractory BTC and first-line GEA in combination with chemotherapy, and Orphan Drug designations for BTC and gastric cancer from both the FDA and EMA.

HERIZON-BTC-01 (NCT04466891, CTR20202607) is a global, multicenter, open-label phase 2b trial evaluating the efficacy and safety of zanidatamab in patients with advanced or metastatic HER2-amplified BTC. The primary endpoint is the ORR by ICR in HER2-positive patients, with secondary endpoints including DOR, PFS, and overall survival (OS).

Biliary tract cancer (BTC), which includes gallbladder cancer and intrahepatic and extrahepatic cholangiocarcinoma, accounts for less than 1% of all adult cancers and generally has a poor prognosis. HER2 is a validated target for cancer therapy. Annually, around 12,000 people in the U.S., Europe, and Japan are diagnosed with BTC, with over 65% presenting with tumors that cannot be surgically removed.



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