On May 19, 2021 Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") reported updated data from the Phase 1/2 trial of mobocertinib (TAK-788) orally administered in patients with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive (insertion+) metastatic non-small cell lung cancer (mNSCLC) who received prior platinum-based chemotherapy (Press release, Takeda, MAY 19, 2021, View Source [SID1234580308]). The results showed mobocertinib continued to demonstrate clinically meaningful benefit after over a year of follow up and will be presented at the virtual 57th American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on June 4.

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"Patients with EGFR Exon20 insertion+ mNSCLC have no proven targeted therapy options," said Suresh S. Ramalingam, MD, Deputy Director of Winship Cancer Institute of Emory University. "The updated results from the Phase 1/2 study of mobocertinib demonstrate an encouraging objective response rate, duration of response and overall survival in patients who have received prior platinum-based chemotherapy."

The analysis from the Phase 1/2 trial included patients with EGFR Exon20 insertion+ mNSCLC who received prior platinum-based chemotherapy. All patients were treated at the 160 mg once daily oral dose. Building on the findings presented in January at the International Association for the Study of Lung Cancer (IASLC) 2020 World Conference on Lung Cancer (WCLC), results showed a median overall survival (OS) of 24 months with a median follow up of 14 months, and responses were observed across diverse EGFR Exon20 insertion variants. Other key data points remained consistent with previously reported data, including a confirmed objective response rate (ORR) of 28%, a median duration of response (DoR) of 17.5 months and a disease control rate (DCR) of 78% per independent review committee (IRC).

The safety profile observed was manageable and consistent with previous findings. The most common treatment-related adverse events (TRAEs; ≥ 20%) in platinum-pretreated patients from the updated data were diarrhea (91%), rash (45%), paronychia (38%), decreased appetite (35%), nausea (34%), dry skin (31%) and vomiting (30%). The only Grade ≥3 TRAE (≥5%) was diarrhea (21%). AEs leading to discontinuation in >2% were diarrhea (4%) and nausea (4%).

"We are excited to add this promising overall survival data to the body of evidence demonstrating mobocertinib’s potential as an effective oral treatment option for platinum-pretreated patients with EGFR Exon20 insertion+ mNSCLC," said Christopher Arendt, PhD, Head, Oncology Therapeutic Area Unit, Takeda. "Mobocertinib is currently undergoing priority review with the U.S. FDA, and we look forward to continuing conversations with regulatory agencies around the world to introduce mobocertinib as a new treatment option for these patients."

The FDA previously granted mobocertinib Breakthrough Therapy Designation in April 2020 and priority review for the New Drug Application (NDA) in April 2021. If approved, mobocertinib will be the first oral therapy available that is specifically designed to selectively target EGFR Exon20 insertion mutations.

Takeda has established an Expanded Access Program (EAP) for patients who may be eligible to receive access to mobocertinib while this investigational therapy is under review by regulatory authorities. Additional information, including the specific conditions to qualify for Takeda’s EAP, is available here.

Learn more about Takeda Oncology’s presence at this year’s ASCO (Free ASCO Whitepaper) Annual Meeting. Takeda will host a webcast for analysts and investors on Tuesday, June 8, at 6:30 p.m. ET to discuss these and other data being presented at ASCO (Free ASCO Whitepaper) and to provide an update on the oncology pipeline. Please contact [email protected] for further details. Presentation slides and an archived replay of the webcast will be available at View Source

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 evaluated 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, which evaluated 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 160 mg 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% 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% 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 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.