세계의 두경부암 파이프라인 인사이트

Executive Summary

Head and neck cancer refers to a group of cancers that develop in the tissues and structures of the head and neck, including the mouth, throat, larynx (voice box), sinuses, nasal cavity, salivary glands and lymph nodes in the neck. These cancers typically arise from the squamous cells that line these areas, known as squamous cell carcinomas, although other types of cancer can occur. Early detection and treatment can lead to a good prognosis, but survival rates decrease when cancer is diagnosed at later stages or if it has spread extensively.

Clinical trials play a vital role in advancing the treatment and management of head and neck cancer by testing new therapies, drugs and approaches to improve patient outcomes. These trials provide crucial data on the safety, effectiveness and potential side effects of new treatments, helping healthcare professionals make informed decisions. Clinical trials are essential for improving treatment options for head and neck cancer, leading to advancements in chemotherapy, immunotherapy, targeted therapies, radiation techniques and personalized medicine. These trials not only improve survival rates but also enhance patients' quality of life and shape the future of cancer care in the head and neck cancer market.

For instance, in January 2025, GORTEC, known as Head and Neck Radiation Oncology Group, announced that the randomized Phase 3 NIVOPOSTOP GORTEC 2018-01 trial evaluating nivolumab, Bristol Myers Squibb's anti-PD-1 therapy, as a post-operative treatment component for resected patients with locally advanced squamous cell carcinoma of head and neck (LA-SCCHN), with high risk of relapse met its primary endpoint of Disease-Free survival (DFS) across all comers.

List of Key Companies

The major and key players in the head and neck cancer pipeline include

• Merck & Co., Inc.
• GlaxoSmithKline
• Rakuten Medical, Inc.
• Incyte Corporation
• Adlai Nortye Biopharma Co., Ltd.
• Merus N.V.
• Akeso Biopharma Co., Ltd.
• Coordination Pharmaceuticals, Inc.
• QBiotics Group Limited
• Psivac Ltd
• BioNTech SE
• Pfizer Inc.
• Ascendis Pharma A/S
• Shanghai Henlius Biotech
• Fulgent Pharma LLC.

Regulatory Designations

• In February 2025, Merck announced that the U.S. Food and Drug Administration (FDA) accepted for priority review a new supplemental Biologics License Application (sBLA) seeking approval for KEYTRUDA (pembrolizumab), Merck's anti-PD-1 therapy, for the treatment of patients with resectable locally advanced head and neck squamous cell carcinoma (LA-HNSCC) as neoadjuvant treatment, then continued as adjuvant treatment in combination with standard of care radiotherapy with or without cisplatin and then as a single agent. The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of June 23, 2025.
• In February 2025, Pyxis Oncology, Inc. announced that the FDA granted Fast Track Designation for its drug PYX-201, aimed at treating adult patients with recurrent or metastatic head and neck squamous cell carcinoma who have not responded to previous treatments. PYX-201 is a first-in-concept antibody-drug conjugate that targets a specific structural component of the tumor extracellular matrix. PYX-201 is currently being evaluated in two active trials, including a study in combination with Merck's KEYTRUDA. The FDA's Fast Track program is designed to expedite the development of drugs addressing serious conditions and unmet medical needs.

Future Perspectives and Conclusion

The head and neck cancer pipeline has been undergoing significant transformation in recent years, driven by advances in research, new therapeutic strategies, and growing demand for more effective and personalized treatments. In the near future, we can expect continued innovation in treatment approaches, improved patient outcomes, and an expansion of therapeutic options.

One of the most promising areas for future development in the head and neck cancer pipeline is personalized medicine. The genetic and molecular landscape of head and neck cancers is diverse and new research is focusing on developing treatments tailored to individual genetic profiles. Advances in genomic sequencing and biomarker identification are enabling more precise treatment approaches.

For instance, the KEYNOTE-048 trial, which evaluated pembrolizumab (Keytruda) for recurrent or metastatic squamous cell carcinoma of the head and neck (R/M HNSCC), exemplified the potential for immunotherapy to be used as a personalized treatment for patients with high PD-L1 expression. The trial showed that pembrolizumab significantly improved survival in patients with high PD-L1 expression, paving the way for personalized immune checkpoint inhibitor therapies in HNC.

Immunotherapy is increasingly becoming a central pillar of head and neck cancer treatment, particularly in the context of checkpoint inhibitors. These drugs work by stimulating the immune system to attack cancer cells, and their use has shown significant promise in treating head and neck cancers, especially in recurrent and metastatic settings. Another exciting development in the pipeline is the use of combination therapies, which involve combining different modalities, such as immunotherapy, chemotherapy, radiation, and targeted therapies, to improve outcomes for patients with head and neck cancer.

The head and neck cancer pipeline is poised for continued growth and innovation. As the understanding of the molecular and genetic underpinnings of these cancers improves, the treatment landscape will become more personalized, with targeted therapies and immunotherapies playing an increasingly central role. Combination therapies, including the pairing of immunotherapies with chemotherapy or radiation, will likely become a standard of care, providing more effective options for patients at different stages of the disease.

The development of precision medicine and the use of genetic biomarkers will enable clinicians to tailor treatments to individual patients, improving the chances of successful outcomes. Immunotherapy will continue to be a major focus, expanding to earlier stages of cancer and offering promising alternatives to traditional chemotherapy. Furthermore, innovations in diagnostic tools, such as liquid biopsies and AI-based imaging, will improve early detection and help personalize treatment plans more effectively.

Table of Contents

1. Executive Summary

• Introduction

2. Head and Neck Cancer: Overview

• Introduction
• Causes
• Pathophysiology
• Signs and Symptoms
• Diagnosis
• Treatment

3. Pipeline Analysis

• Assessment by Phase
  • Phase I Clinical Trials
  • Phase II Clinical Trials
  • Phase III Clinical Trials
• Assessment by Mechanism of Action
  • Immunotherapy
  • Targeted Therapies
  • Chemotherapy
  • Radiation Therapy
  • Combination Therapy
  • Others
• Assessment by Route of Administration
  • Intravenous
  • Oral
  • Topical

4. List of Key Companies Involved

• Merck & Co., Inc.*
  • Detailed Drug Profile
• GlaxoSmithKline
• Rakuten Medical, Inc.
• Incyte Corporation
• Adlai Nortye Biopharma Co., Ltd.
• Merus N.V.
• Akeso Biopharma Co., Ltd.
• Coordination Pharmaceuticals, Inc.
• QBiotics Group Limited
• Psivac Ltd
• BioNTech SE
• Pfizer Inc.
• Ascendis Pharma A/S
• Shanghai Henlius Biotech
• Fulgent Pharma LLC.

5. Regulatory Designations

• Orphan Drug Designation
• Fastrack Designation
• Breakthrough Therapy Designation
• Priority Review
• PRIME Designation
• RMAT Designation
• ATMP Designation

6. Clinical Data Readouts

• Trial Initiation
• Trial Completion
• Safety/Efficacy- Data Readouts
• Discontinued Products

7. Estimated Approvals

8. Merger and Acquisitions

9. Future Perspectives and Conclusion