FDA clears Zymeworks’ IND for liver cancer drug candidate ZW251

Published 28/07/2025, 11:06
FDA clears Zymeworks’ IND for liver cancer drug candidate ZW251

VANCOUVER - The U.S. Food and Drug Administration has cleared the investigational new drug application for ZW251, Zymeworks Inc.’s (NASDAQ:ZYME) novel antibody-drug conjugate targeting hepatocellular carcinoma (HCC), the company announced Monday. The clinical-stage biopharmaceutical company, currently valued at approximately $998 million, maintains a strong financial position with more cash than debt on its balance sheet and a healthy current ratio of 4.71, according to InvestingPro data.

ZW251 is designed to target glypican-3 (GPC3), a protein expressed in over 75% of HCC cases, which is the most common type of primary liver cancer. The drug candidate combines a humanized IgG1 antibody with Zymeworks’ proprietary topoisomerase 1 inhibitor payload using a peptide cleavable linker.

In preclinical studies, ZW251 demonstrated strong anti-tumor activity across various HCC models, including patient-derived xenografts with different GPC3 expression levels. The company reported favorable tolerability in non-human primate studies at doses up to 120 mg/kg.

"This advancement marks the second ADC from our wholly-owned pipeline, utilizing our proprietary TOPO1i payload, to progress into clinical development, reinforcing confidence in our approach," said Paul Moore, Chief Scientific Officer of Zymeworks. The company’s strategic direction has garnered attention from Wall Street, with InvestingPro reporting that two analysts have recently revised their earnings expectations upward for the upcoming period.

The drug features a drug-antibody ratio of four, which the company believes could potentially allow for a broader range of dose levels - a potential benefit for HCC patients who often have impaired liver function due to chronic liver disease and cirrhosis.

Zymeworks plans to initiate Phase 1 clinical trials for ZW251 later this year. The company stated that the drug could potentially improve upon current standard of care for HCC either as a monotherapy or in combination treatment.

This announcement follows Zymeworks’ ongoing development of zanidatamab, its HER2-targeted bispecific antibody that has received regulatory approvals in the U.S., Europe, and China for treating HER2-positive biliary tract cancer.

The information in this article is based on a press release statement from Zymeworks Inc.

In other recent news, Zymeworks has received significant attention following the European Commission’s marketing authorization for its zanidatamab therapy, which is being commercialized by Jazz Pharmaceuticals for treating HER2-positive biliary tract cancer. Additionally, China’s National Medical Products Administration has granted conditional approval for zanidatamab, marking the first dual HER2-targeted bispecific antibody approved in China for HER2-high expression. These approvals highlight the potential impact of zanidatamab in addressing unmet medical needs in cancer treatment.

In the realm of analyst ratings, H.C. Wainwright has maintained a Neutral rating with a $13.00 price target, while Stifel has reiterated a Buy rating with a $28.00 price target for Zymeworks stock. Stifel’s confidence is bolstered by updated results from a Phase 2 trial that evaluated zanidatamab combined with chemotherapy for HER2-positive gastric and esophageal adenocarcinoma. The analysts noted the impressive median overall survival observed in the study and anticipate further validation from the ongoing Phase 3 HERIZON-GEA-01 trial. These developments underscore the growing interest and potential of Zymeworks’ therapeutic offerings in the oncology space.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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