Date: 19-May-2020

ENHERTU® Granted Breakthrough Therapy Designation In The U.S. For HER2 Mutant Metastatic Non-Small Cell Lung Cancer

Daiichi Sankyo Company, Limited and AstraZeneca’s ENHERTU® has been granted Breakthrough Therapy Designation (BTD) in the U.S. for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a HER2 mutation and with disease progression on or after platinum-based therapy.

NSCLC is the most common type of lung cancer, and prognosis is particularly poor for patients with metastatic disease as only about six to 10 percent will be alive five years after diagnosis.1,2 Approximately two to four percent of patients with NSCLC have a HER2 mutation.3,4

The U.S. Food and Drug Administration’s (FDA) BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The new medicine needs to have shown encouraging preliminary clinical results that demonstrate substantial improvement on a clinically significant endpoint over available medicines.

“We are encouraged by the promising evidence of activity seen with ENHERTU in patients with advanced lung cancer and a HER2 mutation,” said Gilles Gallant, BPharm, PhD, FOPQ, Senior Vice President, Global Head, Oncology Development, Oncology R&D, Daiichi Sankyo. “We look forward to working closely with the FDA on the potential for ENHERTU to become the first HER2 directed therapy approved for non-small cell lung cancer.”

“Today’s news is very welcome as we continue to evaluate the potential of ENHERTU to help patients with this devastating type of lung cancer,” said José Baselga, MD, PhD, Executive Vice President, Oncology R&D, AstraZeneca. “Targeted treatments and immunotherapies are demonstrating tremendous advancements, but there remains an unmet medical need for patients with HER2 mutations who are not benefiting from such therapies or for those whose cancer continues to progress.”

The FDA granted BTD based on data from ongoing phase 2 DESTINY-Lung01 trial currently assessing ENHERTU, a HER2 directed antibody drug conjugate (ADC), in patients with HER2 mutant metastatic NSCLC and data from the phase 1 trial published in Cancer Discovery. An interim analysis from DESTINY-Lung01 will be presented at the 2020 American Society of Clinical Oncology (ASCO20) Virtual Scientific Program.

The overall safety and tolerability profile of ENHERTU in the ongoing DESTINY-Lung01 trial is consistent with that seen in the phase 1 trial. The most common adverse events to date (n=42) are gastrointestinal and hematological including nausea, alopecia, anemia, decreased appetite and decreased neutrophil count. There have been five cases of drug-related interstitial lung disease (ILD) and pneumonitis in patients with HER2 mutant NSCLC, all of which were grade 2. There have been no ILD-related deaths.

This is the third BTD granted for ENHERTU in the U.S., and the fourth expedited regulatory designation received globally. Last week, ENHERTU received BTD in patients with HER2 positive unresectable gastric or gastroesophageal junction adenocarcinoma who have received two or more prior regimens including trastuzumab.

ENHERTU recently received accelerated approval in the U.S. and approval in Japan under the conditional early approval system for the treatment of adult patients with unresectable or metastatic HER2 positive metastatic breast cancer who have received two or more prior anti-HER2 based regimens.