Chugai Files for Additional Tumor-Agnostic Indication of Alecensa for ALK Fusion / Rearrangement Gene-Positive Solid Tumors Including Pediatric Patients

• Application based on an investigator-initiated Japanese Phase II clinical study evaluating the efficacy and safety of Alecensa in rare cancers with ALK gene abnormalities in unresectable advanced or recurrent settings
• If approved, Alecensa is expected to become the world’s first tumor-agnostic treatment for ALK fusion / rearrangement gene-positive solid tumors

TOKYO, June 26, 2025 -- Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) today announced that it has filed a regulatory application with the Ministry of Health, Labour and Welfare (MHLW) for an additional indication of its anti-cancer agent/ALK inhibitor Alecensa^® (generic name: alectinib) for ALK fusion / rearrangement gene-positive unresectable advanced or recurrent solid tumors, including pediatric patients.

“I am very pleased that Alecensa has reached the regulatory application stage as a tumor-agnostic treatment for ALK fusion / rearrangement gene-positive solid tumors. This application represents an important step in expanding the treatment potential of Alecensa beyond non-small cell lung cancer and anaplastic large cell lymphoma, a hematological malignancy, for which it has already been approved, to various solid tumors with ALK fusion / rearrangement genes. We will work toward obtaining approval to deliver Alecensa as a new treatment option to patients with various cancer types as quickly as possible.” said Chugai’s President and CEO, Dr. Osamu Okuda.

The application for this additional indication is based on the results of the TACKLE study, an investigator initiated Japanese Phase II clinical study evaluating the efficacy and safety of Alecensa in pediatric and adult patients with rare cancers harboring ALK gene abnormalities (fusion / rearrangement genes, activating mutations, and gene copy number amplification) in unresectable advanced or recurrent settings. In this study, the objective response rate as assessed by the central review committee, which was the primary endpoint, was 76.5% (95% CI: 50.1%-93.2%) (13/17 patients) in the ALK fusion / rearrangement gene-positive subpopulation. The incidence of adverse events was 73.1% (19/26 patients), with the main adverse reactions being lymphocyte count decreased, and neutrophil count decreased at 23.1% each (6/26 patients), anemia at 19.2% (5/26 patients), and blood creatinine increased at 15.4% (4/26 patients). The safety profile observed in this study was similar to the previously established safety profile of Alecensa, with no new safety signals identified.

Chugai Pharmaceutical, a leading company in the oncology field, remains committed to addressing unmet medical needs in cancer treatment with innovative medicines for patients and healthcare professionals.

About the TACKLE study

The TACKLE study (NCCH1712/MK003, jRCT2091220364) is an investigator initiated, multicenter, open-label, single-arm, Japanese Phase II clinical study evaluating the efficacy and safety of Alecensa in pediatric and adult patients with unresectable rare cancers harboring ALK gene abnormalities (fusion / rearrangement genes, activating mutations, gene copy number amplification). In this study, safety and efficacy were evaluated in 26 patients. The primary endpoint was objective response rate, and secondary endpoints included progression-free survival, overall survival, and safety. The TACKLE study is being conducted as a substudy of the MASTER KEY project,¹ which promotes the development of treatments for rare cancers through industry-academia collaboration with the National Cancer Center Hospital.

About ALK fusion / rearrangement gene-positive solid tumors
ALK fusion / rearrangement genes are abnormal genes created when the ALK (anaplastic lymphoma kinase) gene fuses with other genes (such as EML4, NPM) as a result of chromosomal translocation.^2,3 ALK fusion / rearrangement proteins produced from these fusion / rearrangement genes are thought to promote cancer cell proliferation. ALK fusion / rearrangement genes have been identified in patients with inflammatory myofibroblastic tumors, lung cancer, breast cancer, colorectal cancer, and other cancers.^2,4,5

About Alecensa
Alecensa is a highly selective, central nervous system-active, oral medicine created at Chugai Pharmaceutical Co., Ltd. for people with non-small cell lung cancer (NSCLC) whose tumors are identified as anaplastic lymphoma kinase (ALK) positive. ALK fusion / rearrangement gene-positive lung cancer is found in approximately 3-5% of NSCLC cases.⁴ Alecensa is already approved in over 100 countries as an initial (first-line) and second-line treatment for ALK fusion / rearrangement gene-positive metastatic NSCLC, including in the United States, Europe, Japan, China, and Taiwan. For adjuvant therapy of ALK fusion / rearrangement gene-positive NSCLC, Alecensa received approval in the United States in April 2024, followed by Europe in June 2024, and Japan in August 2024. In Japan, Alecensa has also been approved for the treatment of recurrent or refractory ALK fusion / rearrangement gene-positive anaplastic large cell lymphoma.

Trademarks used or mentioned in this release are protected by law.

Sources:

1. National Cancer Center Research Institute. MASTER KEY Project Overview [Internet; cited May 2025]. Available from: https://www.ncc.go.jp/jp/ncch/masterkeyproject/outline/overview/master_key_project_overview.html.
2. Morris, SW. et al.: Fusion of a kinase gene, ALK, to a nucleolar protein gene, NPM, in non-Hodgkin’s lymphoma. Science 267(5196): 316-317, 1995.
3. Soda, M. et al.: Identification of the transforming EML4-ALK fusion gene in non-small-cell lung cancer. Nature 448(7153): 561-566, 2007.
4. Biomarker Committee of the Japan Lung Cancer Society. Guidance for ALK Gene Testing in Lung Cancer Patients Version 4.0 (Japanese only)
5. Lin, E. et al.: Exon array profiling detects EML4-ALK fusion in breast, colorectal, and non-small cell lung cancers. Mol. Cancer Res. 7: 1466-1476, 2009.