- RATIONALE 306 trial met primary endpoint at interim analysis, demonstrating tislelizumab plus chemotherapy significantly improved overall survival compared to chemotherapy alone in patients with previously untreated advanced esophageal squamous cell carcinoma (ESCC)1
- Positive readout in first-line ESCC adds to clinical evidence for tislelizumab in esophageal cancer and follows FDA and EMA filing acceptances for second-line setting
- Results support development of tislelizumab in potentially synergistic combinations across Novartis advanced therapeutic platforms for treatment of array of solid tumors
Basel, April 27, 2022 — Novartis today announced positive topline results from an interim analysis of the Phase III RATIONALE 306 study, which showed anti-PD-1 immune checkpoint inhibitor tislelizumab plus chemotherapy significantly improved overall survival (OS) compared to chemotherapy in patients with previously untreated unresectable, locally advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC), regardless of PD-L1 expression. Novartis intends to submit these data to regulatory authorities, and will collaborate with BeiGene to present them at an upcoming medical meeting.
"People living with esophageal cancer experience painful everyday challenges and typically have a poor prognosis, with a five-year survival rate of around five percent for metastatic cases, underscoring the urgency for more immunotherapy options,” said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development. “We plan to discuss these data with health authorities, and we will continue to expand our tislelizumab clinical development program in pursuit of novel, synergistic combinations with the ultimate goal of extending survival for more patients.”
ESCC is the most common type of esophageal cancer globally, with an estimated 604,000 new cases and 544,000 deaths from esophageal cancer internationally in 2020.2 In the United States, it is estimated there will be more than 20,000 new diagnoses and more than 16,000 deaths from esophageal cancers.3
RATIONALE 306 (NCT03783442) is a multi-regional Phase III, randomized, placebo-controlled, double-blind study of tislelizumab in combination with chemotherapy versus chemotherapy alone in patients with unresectable, locally advanced recurrent or metastatic ESCC. Approximately 649 study participants were randomized 1:1 to receive either tislelizumab plus chemotherapy or chemotherapy alone. The primary endpoint is OS. Secondary endpoints include progression-free survival, objective response rate, duration of response, health-related quality of life measures and safety.
Tislelizumab is currently under review by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for advanced or metastatic ESCC after prior chemotherapy. The EMA is also reviewing tislelizumab for advanced or metastatic non-small cell lung cancer (NSCLC) after prior chemotherapy, and in combination with chemotherapy for previously untreated advanced or metastatic NSCLC.
Novartis is evaluating tislelizumab, a uniquely designed anti-PD-1 monoclonal antibody, in a global clinical development program consisting of 14 pivotal clinical trials across a broad array of solid tumors, with more than 8,800 patients enrolled to date in 35 countries. Novartis four distinct therapeutic platforms (immunotherapy, radioligand therapy, cell and gene therapy, targeted therapy) offer a unique opportunity to study tislelizumab in differentiated, potentially synergistic combinations across our pipeline and portfolio of market compounds.
Novartis has the rights to develop, manufacture and commercialize tislelizumab in North America, Europe and Japan through a collaboration and license agreement with BeiGene.
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “seek,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 108,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.
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- Novartis Data on File
- Sung H, Ferlay J, Siegel RL, et al. Global cancer statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA: a cancer journal for clinicians. 2021;71(3):209-249.
- American Cancer Society. Key statistics for esophageal cancer. Accessed March 1, 2022. https://www.cancer.org/cancer/esophagus-cancer/about/key-statistics.html
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