Novartis announces anti-PD-1 tislelizumab accepted by EMA for regulatory review in esophageal and lung cancers

• European Medicines Agency validated MAAs for tislelizumab in multiple non-small cell lung cancer and esophageal squamous cell carcinoma indications
  
  
• Submissions based on data from Phase III RATIONALE 302, 303, 304 and 307 trials, in which tislelizumab monotherapy and in combination with chemotherapy demonstrated significant clinical benefits versus chemotherapy in multiple cancer types and lines of therapy
  
  
• EMA filings follow US FDA filing acceptance for tislelizumab in esophageal cancer
  
  
• Novartis is evaluating tislelizumab in 14 pivotal clinical trials in a broad array of solid tumors, with more than 8,800 patients enrolled to date in 35 countries 

Basel, April 6, 2022 — Novartis today announced that the European Medicines Agency (EMA) validated Marketing Authorization Applications (MAAs) for the immune checkpoint inhibitor tislelizumab for adults with:

• Locally advanced or metastatic, squamous or non-squamous non-small cell lung cancer (NSCLC) as first-line treatment in combination with chemotherapy,
• Locally advanced or metastatic NSCLC as monotherapy after prior chemotherapy, and
• Unresectable, recurrent, locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) as monotherapy after prior chemotherapy.

Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody being developed both as a monotherapy and in combination with other therapies.¹

"This is an important step toward expanding treatment options for cancer patients in Europe, and builds on the US FDA filing acceptance for tislelizumab in esophageal cancer,” said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development. “We look forward to working with the EMA to make tislelizumab available to people with these aggressive cancers, while continuing to expand our development program to investigate the potential of novel, synergistic combinations.”

The MAAs include data from the pivotal Phase III RATIONALE 302 trial, in which tislelizumab demonstrated a significant improvement in overall survival versus chemotherapy as treatment for people with ESCC who had received prior chemotherapy. The submissions also include data from the pivotal Phase III RATIONALE 303 trial, which showed tislelizumab significantly improved overall survival versus chemotherapy in people with NSCLC after treatment with chemotherapy, and from RATIONALE 304 and 307, which showed tislelizumab plus chemotherapy significantly improved progression-free survival versus chemotherapy in people with untreated squamous and non-squamous NSCLC.

Lung cancer is one of the most common cancers worldwide, accounting for more than 2 million new cases diagnosed each year.² More people die of lung cancer every year than any other cancer.³ 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 2020.³

About RATIONALE Trials
RATIONALE 302 (NCT03430843) is a multi-regional, open-label, randomized Phase III study of tislelizumab versus chemotherapy in patients with advanced unresectable/metastatic esophageal squamous cell carcinoma who had received prior systemic therapy. Approximately 513 patients were randomized 1:1 to receive tislelizumab or investigator chosen chemotherapy. The primary endpoint is overall survival (OS); the key secondary endpoint was OS in the PD-L1 positive population. Other secondary endpoints include progression-free survival (PFS), objective response rate (ORR), duration of response (DoR), health-related quality of life measures and safety.

RATIONALE 303 (NCT03358875) is a multi-regional, open-label, multicenter, randomized Phase III study of tislelizumab versus chemotherapy in patients with locally advanced or metastatic NSCLC who have progressed on a prior platinum-containing regimen. Approximately 805 patients were randomized 1:1 to receive tislelizumab or chemotherapy. The co-primary endpoints are OS in all patients and OS in PD-L1 positive patients. Secondary endpoints include PFS, ORR, DoR, health-related quality of life measures and safety.

RATIONALE 304 (NCT03663205) is an open-label, multicenter, randomized Phase III study of tislelizumab plus chemotherapy versus chemotherapy alone in patients with untreated advanced non-squamous NSCLC. Approximately 334 patients were randomized 1:1 to receive either tislelizumab plus chemotherapy or chemotherapy. The primary endpoint is PFS. Secondary endpoints include OS, ORR, DoR, health-related quality of life measures and safety.

RATIONALE 307 (NCT03594747) is an open-label, multicenter, randomized Phase III study of tislelizumab plus chemotherapy versus chemotherapy in patients with untreated advanced squamous NSCLC. Approximately 360 patients were randomized 1:1:1 to receive tislelizumab plus paclitaxel, tislelizumab plus nab-paclitaxel, or chemotherapy alone. The primary endpoint is PFS. Secondary endpoints include OS, ORR, DoR, health-related quality of life measures and safety.

About Tislelizumab
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.

Disclaimer
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About Novartis
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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References

1. Zhang T, Song X, Xu L, et al. The binding of an anti-PD-1 antibody to FcγRΙ has a profound impact on its biological functions. Cancer Immunol Immunother. 2018;67(7):1079-1090.
2. World Health Organization. Cancer. Available at: https://www.who.int/news-room/fact-sheets/detail/cancer. Accessed November 1, 2021.
3. 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.

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