Jazz Pharmaceuticals plc (Nasdaq: JAZZ) has released pivotal new data from its Phase 2b HERIZON-BTC-01 clinical trial assessing zanidatamab in previously treated HER2-positive biliary tract cancer (BTC). The study’s findings were prominently featured at the American Society of Clinical Oncology (ASCO) Annual Meeting today, highlighting significant improvements in overall survival rates and continued positive responses in patients.

Trial Highlights

Zanidatamab, a bispecific antibody targeting the human epidermal growth factor receptor 2 (HER2), has shown sustained efficacy in treating unresectable, locally advanced, or metastatic HER2-positive BTC. The trial’s primary endpoint revealed a confirmed objective response rate (cORR) of 41.3% by independent central review, marking a notable achievement given the disease’s typically poor prognosis.

The latest analysis also demonstrated a median overall survival (OS) of 15.5 months across all participants, with a remarkable 18.1 months in patients exhibiting higher HER2 expression (IHC 3+). These outcomes significantly surpass the historical 6-9 months survival typically expected with second-line chemotherapy treatments.

Medical Expert Insights

Dr. Shubham Pant, a professor at The University of Texas MD Anderson Cancer Center, emphasized the urgent need for effective treatments for advanced BTC, noting the “deep and durable responses” achieved with zanidatamab. This treatment offers a chemotherapy-free option, which is crucial for improving patient quality of life and survival outcomes.

Regulatory and Future Steps

The robust results from the HERIZON-BTC-01 trial have been instrumental in Jazz Pharmaceuticals’ recent submissions for regulatory approval. The company announced that the U.S. Food and Drug Administration (FDA) granted a Priority Review for zanidatamab earlier this week. Similarly, a Marketing Authorization Application was submitted to the European Medicines Agency.

Moreover, the ongoing global, randomized Phase 3 trial, HERIZON-BTC-302, aims to further evaluate zanidatamab in combination with standard-of-care therapies, marking a significant step towards broadening treatment options for HER2-positive BTC.

The enduring positive outcomes of the HERIZON-BTC-01 trial reinforce zanidatamab’s potential as a transformative treatment for patients with advanced HER2-positive BTC. Jazz Pharmaceuticals continues to lead in the clinical development of targeted therapies, setting a new standard in oncological care.

  ASCO 2024, biliary tract cancer, clinical trials, HER2-positive, Jazz Pharmaceuticals, zanidatamab