DUBLIN - Jazz Pharmaceuticals plc (NASDAQ:JAZZ) has concluded its rolling submission for a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for zanidatamab, aimed at treating HER2-positive biliary tract cancer (BTC).
The application is based on the Phase 2b HERIZON-BTC-01 trial, which met its primary endpoint, demonstrating a confirmed objective response rate of 41.3% in patients who had previously undergone treatment.
Zanidatamab, a HER2-targeted bispecific antibody, has been designed to bind two separate parts of the HER2 protein, potentially leading to improved treatment outcomes. The drug has shown tolerable safety profiles, with the most common adverse events being diarrhea and infusion-related reactions, both of which were generally low-grade and manageable.
The Phase 2b trial data, as of October 10, 2022, indicated a median duration of response of 12.9 months in patients with previously treated HER2-positive BTC. This compares favorably to historical response rates of 5 to 15% for second-line standard-of-care chemotherapy in BTC patients. Additionally, the trial reported a median progression-free survival of 5.5 months.
The company has also initiated the HERIZON-BTC-302 Phase 3 trial, which is currently enrolling patients. This global study will evaluate the efficacy and safety of zanidatamab in combination with standard-of-care therapy for first-line treatment of advanced or metastatic HER2-positive BTC.
If approved, zanidatamab would be the first HER2-targeted therapy specifically sanctioned for BTC in the United States. The FDA has already granted zanidatamab Breakthrough Therapy designation for previously treated HER2-amplified BTC and Fast Track designations for its use as a single agent in refractory BTC and in combination with chemotherapy for first-line gastroesophageal adenocarcinoma.
Zanidatamab is being developed by Jazz Pharmaceuticals and BeiGene (NASDAQ:BGNE), Ltd. under license agreements from Zymeworks (NASDAQ:ZYME). The drug has also received Orphan Drug designations from the FDA and the European Medicines Agency for the treatment of BTC and gastric cancer.
Biliary tract cancers are rare and often have a poor prognosis, with most patients diagnosed at a stage where surgical removal of the tumor is not possible. Jazz Pharmaceuticals, headquartered in Dublin, Ireland, is focused on developing innovative treatments for serious diseases with limited therapeutic options.
The information in this article is based on a press release statement from Jazz Pharmaceuticals.
InvestingPro Insights
As Jazz Pharmaceuticals (NASDAQ:JAZZ) moves forward with its application for zanidatamab, investors are closely monitoring the company's financial health and market performance. According to InvestingPro data, Jazz Pharmaceuticals boasts a strong market capitalization of $7.42 billion, reflecting investor confidence in the company's potential. The company's gross profit margin stands impressively high at 92.59% for the last twelve months as of Q4 2023, indicating efficient management and a solid competitive edge in its operational execution.
Key InvestingPro Tips highlight that the company's management has been actively engaged in share buybacks, which can signal confidence in the firm's future and often provide support for the stock price. Furthermore, Jazz Pharmaceuticals is anticipated to experience net income growth this year, which is a positive sign for investors looking for companies with an upward trajectory in earnings.
InvestingPro data also reveals that Jazz Pharmaceuticals trades with a price-to-earnings (P/E) ratio of 18.18, and an adjusted P/E ratio of 12.3 for the last twelve months as of Q4 2023, suggesting that the stock may be reasonably valued compared to its earnings. Additionally, the company is trading near its 52-week low, which might present a potential entry point for investors seeking to capitalize on the company's future growth prospects.
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