Zymeworks Announces China NMPA Acceptance of Biologics License Application for Zanidatamab for Second-Line Treatment of Biliary Tract Cancer
Zymeworks announced the acceptance of the Biologics License Application (BLA) for zanidatamab by the National Medical Products Administration (NMPA) in China for second-line treatment of HER2-positive biliary tract cancer (BTC). This acceptance triggers an $8 million milestone payment from BeiGene, with potential for up to $164 million in future milestones and royalties on product sales. The application is based on data from the HERIZON-BTC-01 trial, showing a 41.3% objective response rate, a 12.9-month median duration of response, and a 5.5-month median progression-free survival. Additionally, the U.S. FDA recently accepted a BLA for zanidatamab for priority review, with a target action date of November 29, 2024. Zymeworks has already received $53 million in payments under its collaboration with BeiGene, not including the recent $8 million milestone.
- Acceptance of BLA by NMPA in China for zanidatamab.
- Triggers $8 million milestone payment from BeiGene.
- Potential to receive up to $164 million in additional milestones.
- FDA accepted BLA for zanidatamab for priority review.
- HERIZON-BTC-01 trial showed 41.3% objective response rate.
- 12.9 months median duration of response.
- 5.5 months median progression-free survival.
- Zymeworks already received $53 million in payments from BeiGene.
- Eligible for tiered royalties of up to 19.5% on net sales.
- Median progression-free survival of only 5.5 months.
- Uncertainty around achieving the full $164 million milestone potential.
- Dependence on successful commercialization and sales performance in the Asia Pacific region.
Insights
Zymeworks stands to receive an
From a financial perspective, the
Investors should also consider the
The acceptance of the Biologics License Application (BLA) by the National Medical Products Administration (NMPA) for zanidatamab is significant for the medical field, especially for treating HER2-positive biliary tract cancer (BTC). The clinical trial data, published in Lancet Oncology, showed promising results. The objective response rate (ORR) was
These clinical outcomes indicate that zanidatamab could become an important therapeutic option for patients who have limited treatments available. This potentially transforms the therapeutic landscape for a difficult-to-treat cancer type. However, investors should be aware of the risks associated with drug approvals and market adoption. The efficacy and safety profiles demonstrated in the trials are strong indicators, but broader market acceptance will depend on further phase 3 trial results and real-world data.
The acceptance of the BLA in China and the potential market launch of zanidatamab in the Asia Pacific region is a strategic move by Zymeworks to capture a significant market share in the oncology sector. China represents a large and growing market for cancer treatments. With the NMPA's acceptance, Zymeworks and BeiGene are well-positioned to penetrate this market, especially considering the high incidence of biliary tract cancer in Asia.
Furthermore, the collaboration with BeiGene, a well-established player in the region, could facilitate smoother market entry and commercialization. The strategic partnership enhances Zymeworks' ability to navigate regulatory landscapes, distribution networks and market dynamics in the Asia Pacific region. This can potentially lead to high adoption rates and substantial sales, benefiting both companies. However, investors should consider competitive dynamics, as other companies are also developing treatments for HER2-positive cancers. Market share and revenue will depend on how zanidatamab performs relative to competitors.
- 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
VANCOUVER, British Columbia, June 10, 2024 (GLOBE NEWSWIRE) -- Zymeworks Inc. (Nasdaq: ZYME), 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
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
“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
About Zanidatamab
Zanidatamab is an investigational HER2-targeted bispecific antibody that can simultaneously bind two non-overlapping epitopes of the HER2 receptor, known as biparatopic binding. This unique design and increased binding results in multiple mechanisms of action, including dual HER2 signal blockade, removal of HER2 protein from the cell surface, and immune-mediated cytotoxicity leading to encouraging antitumor activity in patients. Zanidatamab is being developed in multiple clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is being developed by Jazz Pharmaceuticals and BeiGene under license agreements from Zymeworks, which first developed the molecule.
The U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review of the Biologics License Application (BLA) for zanidatamab with a Prescription Drug User Fee Act (PDUFA) action date of November 29, 2024. Zanidatamab was also granted Breakthrough Therapy designation in patients with previously treated HER2 gene-amplified BTC, and given two Fast Track designations: one as a single agent for refractory BTC and one in combination with standard of care chemotherapy for first-line gastroesophageal adenocarcinoma (GEA). Additionally, zanidatamab has received Orphan Drug designations from FDA for the treatment of BTC and GEA, as well as Orphan Drug designation from the European Medicines Agency for the treatment of BTC and gastric cancer. Zanidatamab was also granted Breakthrough Therapy designation from the Center for Drug Evaluation (CDE) in China.
About HERIZON-BTC-01
HERIZON-BTC-01 (NCT04466891, CTR20202607) is a global, multicenter, open-label, single-arm phase 2b trial to evaluate the efficacy and safety of zanidatamab in patients with previously treated advanced or metastatic HER2-amplified BTC. The primary endpoint was objective response rate (ORR) assessed by independent central review (ICR) in HER2-positive patients. Secondary endpoints included ICR-assessed duration of response (DOR), progression-free survival (PFS), and overall survival (OS).
About Biliary Tract Cancer
Biliary tract cancer (BTC), including gallbladder cancer and intrahepatic and extrahepatic cholangiocarcinoma, account for <
About Zymeworks Inc.
Zymeworks is a global biotechnology company committed to the discovery, development, and commercialization of novel, multifunctional biotherapeutics. Zymeworks’ mission is to make a meaningful difference in the lives of people impacted by difficult-to-treat cancers and other diseases. The Company’s complementary therapeutic platforms and fully integrated drug development engine provide the flexibility and compatibility to precisely engineer and develop highly differentiated antibody-based therapeutic candidates. Zymeworks engineered and developed zanidatamab, a HER2-targeted bispecific antibody using the Company’s proprietary Azymetric™ technology. Zymeworks has entered into separate agreements with BeiGene, Ltd. (BeiGene) and Jazz Pharmaceuticals Ireland Limited (Jazz), granting each exclusive rights to develop and commercialize zanidatamab in different territories. Zanidatamab is currently being evaluated in multiple global clinical trials as a potential best-in-class treatment for patients with HER2-expressing cancers. A Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for zanidatamab as a treatment for previously-treated, unresectable, locally advanced, or metastatic HER2-positive biliary tract cancer (BTC) has been accepted and granted Priority Review. A BLA has also been accepted for review by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China. If approved, zanidatamab would be the first HER2-targeted treatment specifically approved for BTC in the U.S. and China. Zymeworks is rapidly advancing a deep pipeline of product candidates based on its experience and capabilities in both antibody-drug conjugates and multispecific antibody therapeutics across multiple novel targets in indications that represent areas of significant unmet medical need. In addition to Zymeworks’ wholly owned pipeline, its therapeutic platforms have been further leveraged through strategic partnerships with global biopharmaceutical companies. For information about Zymeworks, visit www.zymeworks.com and follow @ZymeworksInc on X.
Cautionary Note Regarding Forward-Looking Statements
This press release includes “forward-looking statements” or information within the meaning of the applicable securities legislation, including Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements in this press release include, but are not limited to, statements that relate to Zymeworks’ expectations regarding implementation of its strategic priorities; the anticipated benefits of the licensing agreement with BeiGene and agreements with other partners, including Zymeworks’ ability to receive any future milestone payments and royalties thereunder; the potential addressable market of zanidatamab; the timing of and results of interactions with regulators; Zymeworks’ clinical development of its product candidates and enrollment in its clinical trials; the timing and status of ongoing and future studies and the related data; expectations regarding future regulatory filings and approvals; the timing of and results of interactions with regulators; potential safety profile and therapeutic effects of zanidatamab and Zymeworks’ other product candidates; the commercial potential of technology platforms and product candidates and other information that is not historical information. When used herein, words such as “plan”, “believe”, “expect”, “may”, “continue”, “anticipate”, “potential”, “will”, “progress”, and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon Zymeworks’ current expectations and various assumptions. Zymeworks believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. Zymeworks may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various factors, including, without limitation: any of Zymeworks’ or its partners’ product candidates may fail in development, may not receive required regulatory approvals, or may be delayed to a point where they are not commercially viable; Zymeworks may not achieve milestones or receive additional payments under its collaborations; regulatory agencies may impose additional requirements or delay the initiation of clinical trials; the impact of new or changing laws and regulations; market conditions; the impact of pandemics and other health crises on Zymeworks’ business, research and clinical development plans and timelines and results of operations, including impact on its clinical trial sites, collaborators, and contractors who act for or on Zymeworks’ behalf; clinical trials may not demonstrate safety and efficacy of any of Zymeworks’ or its collaborators’ product candidates; Zymeworks’ assumptions and estimates regarding its financial condition, future financial performance and estimated cash runway may be incorrect; inability to maintain or enter into new partnerships or strategic collaborations; and the factors described under “Risk Factors” in Zymeworks’ quarterly and annual reports filed with the Securities and Exchange Commission (copies of which may be obtained at www.sec.gov and www.sedar.com).
Although Zymeworks believes that such forward-looking statements are reasonable, there can be no assurance they will prove to be correct. Investors should not place undue reliance on forward-looking statements. The above assumptions, risks and uncertainties are not exhaustive. Forward-looking statements are made as of the date hereof and, except as may be required by law, Zymeworks undertakes no obligation to update, republish, or revise any forward-looking statements to reflect new information, future events or circumstances, or to reflect the occurrences of unanticipated events.
Contacts:
Investor Inquiries:
Shrinal Inamdar
Director, Investor Relations
(604) 678-1388
ir@zymeworks.com
Media Inquiries:
Diana Papove
Senior Director, Corporate Communications
(604) 678-1388
media@zymeworks.com
1 Harding JJ, Fan J, Oh D-Y, et al. Zanidatamab for HER2-amplified, unresectable, locally advanced or metastatic biliary tract cancer (HERIZON-BTC-01): a multicentre, single-arm, phase 2b study. Lancet Oncol. 2023;24(7):772-782.
2 Valle JW, et al. Lancet 2021; 397:428-44
3 Siegel RL, et al. CA Cancer J Clin 2022; 72;7-33
4 BTC overall diagnosed patients as per SEER 22;
5 Assumes anatomic subsites intrahepatic CCA, extrahepatic CCA, gallbladder cancer, and BTC unspecified;
6 Assumes HER2 positivity rates per anatomical subsite from: Galdy, S., Lamarca, A., McNamara, M.G. et al. Cancer Metastasis Rev 36, 141–157 (2017), Nobuyoshi Hiraoka, et al. Human Pathology, Volume 105, 2020, Pages 9-19
7 Major markets: U.K, France, Germany, Spain, Italy. Note: HER2+ BTC patients in Jazz-controlled commercial territories, which includes Japan, and excludes other certain Asia Pacific countries licensed to BeiGene, Ltd
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