Takeda's Investigational Drug Mobocertinib (TAK-788) Granted Breakthrough Therapy Designation in China for EGFR Exon20 Insertion-Positive NSCLC

Shanghai, October 15, 2020 /PRNewswire/ -- Takeda China today announced that its investigational innovative drug Mobocertinib (TAK-788) has been approved by the Center for Drug Evaluation (CDE) of the National Medical Products AdministrationInclusion in the "Breakthrough Therapy Drug List", for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring epidermal growth factor receptor (EGFR) exon 20 insertion mutations who have received at least one prior systemic chemotherapy. Currently, there are no approved treatment regimens worldwide for this specific subtype of NSCLC.

Since July 2020, when the National Medical Products Administration issued the "Procedures for the Review of Breakthrough Therapy Drugs (Trial)"^[1]Subsequently, Takeda China submitted the Breakthrough Therapy designation application for Mobocertinib on the very first day the submission channel opened. The inclusion of Mobocertinib in the “Breakthrough Therapy Drug” program is expected to accelerate its research, development, and regulatory approval process in China, thereby enabling earlier access for Chinese patients. Mobocertinib is the first innovative drug from a foreign pharmaceutical company to receive the “Breakthrough Therapy Drug” designation in China.

Professor Zhou Caicun, Director of the Department of Oncology at Shanghai Pulmonary Hospital affiliated with Tongji University, Director of the Institute of Lung Tumors at Tongji University School of Medicine, and lead investigator of the mobocertinib clinical trials, stated: “In the field of lung cancer, patients with non-small cell lung cancer (NSCLC) harboring EGFR exon 20 insertion mutations have a poorer prognosis compared to those with common EGFR mutations, T790M resistance mutations, or rare mutations. Currently, no drugs targeting this specific mutation have been approved globally, resulting in an urgent unmet clinical need among Chinese patients. Based on current research data, mobocertinib shows promise in delivering significant benefits to NSCLC patients with EGFR exon 20 insertion mutations. We sincerely hope that its inclusion in the Breakthrough Therapy designation will facilitate earlier access to mobocertinib for patients in China.”

Non-Small Cell Lung Cancer (NSCLC) is the most common type of lung cancer.One ofAccording to statistics from the World Health Organization (WHO), there are approximately 1.8 million new cases of lung cancer globally each year, with non-small cell lung cancer (NSCLC) accounting for about 85% of these cases.^[2,3]. EGFR exon 20 insertion mutations are a rare type of mutation in non-small cell lung cancer (NSCLC), accounting for approximately 2.1% of all NSCLC cases.^[4,5]. In China, the incidence of EGFR exon 20 insertion mutations accounts for approximately 2.3% of all NSCLC cases.^[6,7]Currently, no therapeutic agents targeting EGFR exon 20 have been approved globally. Meanwhile, existing EGFR-TKIs, chemotherapy, and immunotherapy offer limited clinical benefit for patients with non-small cell lung cancer (NSCLC) harboring EGFR exon 20 insertion mutations. These patients suffer significantly from the disease, highlighting an urgent unmet clinical need for effective treatments.

Mobocertinib is a small-molecule tyrosine kinase inhibitor (TKI) developed by Takeda that selectively targets EGFR and human epidermal growth factor receptor 2 (HER2) exon 20 insertion mutations. It is also the first drug from Takeda to achieve global synchronous development with participation from China, and it is predicted to deliver breakthrough therapeutic benefits for patients with non-small cell lung cancer (NSCLC) harboring EGFR exon 20 insertion mutations. Preliminary efficacy and safety data from a Phase 1/2 clinical trial of mobocertinib (AP32788-15-101) demonstrated that, in patients with refractory NSCLC carrying EGFR exon 20 insertion mutations, treatment with mobocertinib resulted in a median progression-free survival (mPFS) of 7.3 months and an objective response rate (ORR) of 43%, with a manageable safety profile.^[8]. In December 2019, mobocertinib received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA), and in April 2020, it was granted Breakthrough Therapy Designation by the FDA.

References:

1. Announcement of the National Medical Products Administration on Issuing Three Documents, Including the “Procedures for the Review of Breakthrough Therapy Drugs (Trial)” (No. 82 of 2020).https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20200708151701834.html

2. World Health Organization. Latest Global Cancer Data. https://www.who.int/cancer/PRGlobocanFinal.pdf . Accessed May 11, 2019.

3. 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.

4. Leduc C, et al. Ann Oncol. 2017;28:2715- -2724.

5. Kobayashi Y, Mitsudomi T. Cancer Sci. 2016;107:1179-1186

6. OncoTargets and Therapy 2018:11 7323–7331

7. Mol Oncol  . 2020 Aug;14(8):1695-1704.

8. Doebele RC, Riely GJ, Spira AI, Horn L, Piotrowska Z, Costa DB, et al. First report of safety, PK, and preliminary antitumor activity of the oral EGFR/HER2 exon 20 inhibitor TAK-788

(AP32788) in non-small cell lung cancer (NSCLC).. Journal of Clinical Oncology. 2018/5/20;36(15_suppl): 9015-9015.