Takeda Announces China NMPA Acceptance of New Drug Application for Mobocertinib (TAK-788) as a Treatment for Adult Patients with EGFR Exon20 Insertion+ Metastatic Non-Small Cell Lung Cancer

OSAKA, Japan, July 07, 2021--Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) (“Takeda”) today announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration of China (NMPA) has accepted the company’s New Drug Application (NDA) for mobocertinib (TAK-788) and granted priority review for this Class-1 innovative drug, for the treatment of adult patients with non-small cell lung cancer(NSCLC) with epidermal growth factor receptor (EGFR) Exon20 insertion mutations. Mobocertinib is the first oral therapy specifically designed to target EGFR Exon20 insertion mutations. The U.S. Food and Drug Administration (FDA) recently granted priority review for the company’s New Drug Application for mobocertinib with a Prescription Drug User Fee Act (PDUFA) target action date set for October 26, 2021.

“Patients with EGFR Exon20 insertion+ mNSCLC face critical unmet needs in China,” said Sean Shan, president of Takeda in China. “The acceptance by CDE marks an important step to accelerate the delivery of innovative treatments to patients with limited therapeutic options. The Chinese government’s healthcare reforms are rewarding innovation which also present an opportunity for Takeda’s current portfolio and pipeline of innovative and life transforming medicines.”

The EGFR Exon20 insertion mutation is a rare mutation in non-small cell lung cancer (NSCLC). In China, the incidence of NSCLC with the EGFR Exon20 insertion mutations accounts for about 2.3 percent of all NSCLC cases[1]. Currently, there are no approved therapies targeting EGFR Exon20 insertion mutations, and the current EGFR TKIs and chemotherapy provide limited benefits for these patients whose clinical needs are very urgent[2].

“Takeda is committed to bringing established innovative products as well as exciting, new molecular entities like mobocertinib to patients in China. And, when possible, ensuring submission and approval occurs in a similar timeframe as in other regions,” said Lin Wang, Head of the Takeda Development Center Asia. “We’re preparing for an exciting period of growth and expansion in fiscal year 2021, with the potential for up to 12 submissions and six approvals, which will contribute significantly toward our goal of delivering more than 15 innovative medicines to patients in China by fiscal year 2024.”

The NDA for mobocertinib is primarily based on results from the Phase 1/2 trial, which is evaluating the safety and efficacy of oral mobocertinib in patients with mNSCLC.

About Mobocertinib (TAK-788)

Mobocertinib is an investigational, first-in-class, oral tyrosine kinase inhibitor (TKI) specifically designed to selectively target epidermal growth factor receptor (EGFR) Exon20 insertion mutations. In 2019, the U.S. FDA granted mobocertinib Orphan Drug Designation for the treatment of lung cancer with HER2 mutations or EGFR mutations including Exon20 insertion mutations. In April 2020, mobocertinib received Breakthrough Therapy Designation from the FDA for patients with EGFR Exon20 insertion+ metastatic non-small cell lung cancer (mNSCLC) whose disease has progressed on or after platinum-based chemotherapy. In October 2020, mobocertinib was designated as a Breakthrough Therapy in China by the Center for Drug Evaluation (CDE) for locally advanced or metastatic NSCLC patients with EGFR Exon20 insertion mutations who have been previously treated with at least one prior systemic chemotherapy.

About the Phase 1/2 Trial

The Phase 1/2 trial aims to evaluate the safety, pharmacokinetics and anti-tumor activity of oral mobocertinib in patients with non-small cell lung cancer (NSCLC). The trial is comprised of a Phase 1 dose-escalation, evaluating mobocertinib as a monotherapy and in combination with chemotherapy, several expansion cohorts and an extension cohort in patients with epidermal growth factor receptor (EGFR) Exon20 insertion+ metastatic NSCLC (mNSCLC).

The platinum-pretreated population efficacy analysis investigated 114 patients with EGFR Exon20 insertion+ mNSCLC who received prior platinum-based therapy in the Phase 1/2 trial and were treated with mobocertinib at the 160mg once daily dose.

About EGFR Exon20 Insertion+ mNSCLC

Non-small cell lung cancer (NSCLC) is the most common form of lung cancer, accounting for approximately 85 percent of the estimated 1.8 million new cases of lung cancer diagnosed each year worldwide, according to the World Health Organization.1,2 Patients with epidermal growth factor receptor (EGFR) Exon20 insertion+ metastatic NSCLC (mNSCLC) make up approximately 1-2 percent of patients with NSCLC, and the disease is more common in Asian populations compared to Western populations.3-7 This disease carries a worse prognosis than other EGFR mutations because there are currently no U.S. FDA-approved therapies that target EGFR Exon20 insertions, and current EGFR TKIs and chemotherapy provide limited benefit for these patients.

Takeda is committed to continuing research and development in EGFR Exon20 insertion+ mNSCLC with the hope of introducing a targeted treatment option for the approximately 30,000 patients diagnosed with the disease worldwide each year, including 3,000 in the U.S. alone.3,4

Takeda’s Commitment to Oncology

Our core R&D mission is to deliver novel medicines to patients with cancer worldwide through our commitment to science, breakthrough innovation and passion for improving the lives of patients. Whether it’s with our hematology therapies, our robust pipeline, or solid tumor medicines, we aim to stay both innovative and competitive to bring patients the treatments they need. For more information, visit www.takedaoncology.com.

Media Contacts:

Chinese Media

YunQing Yao
Yunqing.yao@takeda.com

Media Outside China

Lauren Padovan
Lauren.Padovan@Takeda.com
+1 (617) 444-1419

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people’s lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries. For more information, visit https://www.takeda.com.

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  1. World Health Organization. Latest Global Cancer Data. https://www.who.int/cancer/PRGlobocanFinal.pdf. Accessed May 11, 2019.
  2. American Cancer Society. What is Non-Small Cell Lung Cancer? https://www.cancer.org/cancer/non-small-cell-lung-cancer/about/what-is-non-small-cell-lung-cancer.html.
  3. Riess, Jonathan W. Diverse EGFR Exon 20 Insertions and Co-Occurring Molecular Alterations Identified by Comprehensive Genomic Profiling of NSCLC. https://www.jto.org/article/S1556-0864(18)30770-6/fulltext. Accessed April 7, 2020.
  4. Fang, Wenfeng. BMC Cancer. EGFR exon 20 insertion mutations and response to osimertinib in non-small-cell lung cancer. https://bmccancer.biomedcentral.com/articles/10.1186/s12885-019-5820-0. Accessed April 7, 2020.
  5. Kobayashi Y, Mitsudomi T. Not all epidermal growth factor receptor mutations in lung cancer are created equal: Perspectives for individualized treatment strategy. Cancer Sci. 2016;107(9):1179-1186. doi:10.1111/cas.12996
  6. Yatabe Y, Kerr KM, Utomo A, et al. EGFR mutation testing practices within the Asia Pacific region: results of a multicenter diagnostic survey. J Thorac Oncol. 2015;10(3):438-445. doi:10.1097/JTO.0000000000000422
  7. Kris MG, Johnson BE, Berry LD, et al. Using multiplexed assays of oncogenic drivers in lung cancers to select targeted drugs. JAMA. 2014;311(19):1998-2006. doi:10.1001/jama.2014.3741