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AstraZeneca: Enhertu granted Orphan Drug Designation in the US for gastric cancer

Designation follows recent US Breakthrough Therapy Designations for
Enhertu for HER2-positive metastatic gastric cancer and HER2-mutant
metastatic non-small cell lung cancer

AstraZeneca and Daiichi Sankyo Company, Limited (Daiichi Sankyo)'s
Enhertu (trastuzumab deruxtecan) has been granted Orphan Drug
Designation (ODD) in the US for the treatment of patients with
gastric cancer, including gastroesophageal junction cancer.

An estimated 27,600 new cases of gastric cancer will be diagnosed this
year and the disease could lead to more than 11,000 deaths in the US
in 2020.[1 ]

The Food and Drug Administration (FDA) grants ODD to medicines
intended for the treatment, diagnosis or prevention of rare diseases
or disorders that affect fewer than 200,000 people in the US.

In the Phase II DESTINY-Gastric01 trial
patients with HER2-positive metastatic gastric or gastroesophageal
cancer who were treated with Enhertu, a HER2-directed antibody drug
conjugate (ADC), demonstrated a statistically significant and
clinically meaningful improvement in objective response rate (ORR),
the primary endpoint, and overall survival (OS), a key secondary
endpoint, versus patients treated with investigator's choice of
chemotherapy (irinotecan or paclitaxel monotherapy).

The overall safety and tolerability profile of Enhertu (6.4mg/kg) in
DESTINY-Gastric01 was consistent with that seen in the Phase I
gastric cancer trial. The most common adverse events were
haematologic and gastrointestinal including neutrophil count
decrease, anaemia, nausea and decreased appetite. There were cases of
drug-related interstitial lung disease (ILD) and pneumonitis, the
majority of which were Grade 1 and 2, with two Grade 3 and one Grade
4. No Grade 5 events (ILD-related deaths) occurred in patients with
gastric cancer in the Phase I trial or in the Phase II
DESTINY-Gastric01 trial.

The full results of DESTINY-Gastric-01 will be presented during the
2020 American Society of Clinical Oncology ASCO20 Virtual Scientific
Program, 29 to 31 May 2020.

Earlier this month, Enhertu received two Breakthrough Therapy
Designations for its potential use in HER2-positive unresectable or
metastatic gastric cancer
and HER2-mutant metastatic non-small cell lung cancer
Enhertu also received SAKIGAKE designation from Japan's Ministry of
Health Labour and Welfare (MHLW) for potential use in HER2-positive
gastric cancer in March 2018. A supplemental New Drug Application was
recently submitted to the MHLW.

Gastric cancer

Gastric (stomach) cancer is the fifth most common cancer worldwide and
the third leading cause of cancer mortality with a five-year survival
rate of 5% for metastatic disease. There were approximately one
million new cases reported in 2018 and 783,000 deaths.[2,3] In the
US, it is estimated that 27,600 new cases of gastric cancer will be
diagnosed in 2020 and more than 11,000 people will die from the

Approximately one in five gastric cancers are HER2 positive.[4,5] HER2
is a tyrosine kinase receptor growth-promoting protein expressed on
the surface of many types of tumours including gastric, breast and
lung cancers. Gastric cancer is usually diagnosed in the advanced
stage in the US, but even when diagnosed in earlier stages of the
disease the survival rate remains modest.[6] Recommended 1st-line
treatment for HER2-positive advanced or metastatic gastric cancer is
combination chemotherapy plus trastuzumab, an anti-HER2 medicine,
which has been shown to improve survival outcomes when added to
chemotherapy. For gastric cancer that progresses on 1st-line
treatment, there are no other approved HER2-targeted medicines.[7]


DESTINY-Gastric01 is a Phase II, open-label, multi-centre trial
testing the safety and efficacy of Enhertu in a primary cohort of 188
patients from Japan and South Korea with HER2-expressing, advanced
gastric or gastroesophageal junction adenocarcinoma (defined as IHC3+
or IHC2+/ISH+) who have progressed on two or more prior treatment
regimens including fluoropyrimidine and platinum chemotherapy and
trastuzumab. Patients were randomised 2:1 to receive Enhertu or
investigator's choice of chemotherapy (paclitaxel or irinotecan
monotherapy). Patients were treated with Enhertu 6.4mg/kg once every
three weeks or chemotherapy. The primary endpoint of the trial is
ORR, as assessed by an independent review committee. Secondary
endpoints include OS, progression-free survival, duration of
response, disease control rate and time to treatment failure as well
as pharmacokinetic and safety endpoints.[8]


Enhertu (trastuzumab deruxtecan; fam-trastuzumab deruxtecan-nxki in
the US) is a HER2-directed ADC and is the lead ADC in the oncology
portfolio of Daiichi Sankyo and the most advanced programme in
AstraZeneca's ADC scientific platform. ADCs are targeted cancer
medicines that deliver cytotoxic chemotherapy ("payload") to cancer
cells via a linker attached to a monoclonal antibody that binds to a
specific target expressed on cancer cells.

Enhertu (5.4mg/kg) is approved in the US and Japan for the treatment
of adult patients with unresectable or metastatic HER2-positive
breast cancer who have received two or more prior anti-HER2-based
regimens based on the DESTINY-Breast01 trial.

Enhertu clinical development

A comprehensive development programme is underway globally with six
registrational trials evaluating the efficacy and safety of Enhertu
monotherapy across multiple HER2-targetable cancers including breast,
gastric and lung cancers. Trials in combination with other anticancer
treatments, such as immunotherapy, are also underway.

Collaboration between AstraZeneca and Daiichi Sankyo

In March 2019, AstraZeneca and Daiichi Sankyo entered into a global
collaboration to jointly develop and commercialise Enhertu worldwide,
except in Japan where Daiichi Sankyo maintains exclusive rights.
Daiichi Sankyo is solely responsible for manufacturing and supply.

AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a
quickly growing portfolio of new medicines that has the potential to
transform patients' lives and the Company's future. With six new
medicines launched between 2014 and 2020, and a broad pipeline of
small molecules and biologics in development, the Company is
committed to advance oncology as a key growth driver for AstraZeneca
focused on lung, ovarian, breast and blood cancers. In addition to
AstraZeneca's main capabilities, the Company is actively pursuing
innovative partnerships and investment that accelerate the delivery
of our strategy, as illustrated by the investment in Acerta Pharma in

By harnessing the power of four scientific platforms -
Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response
and ADCs - and by championing the development of personalised
combinations, AstraZeneca has the vision to redefine cancer treatment
and, one day, eliminate cancer as a cause of death.


AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led
biopharmaceutical company that focuses on the discovery, development
and commercialisation of prescription medicines, primarily for the
treatment of diseases in three therapy areas - Oncology,
Cardiovascular, Renal & Metabolism, and Respiratory & Immunology.
Based in Cambridge, UK, AstraZeneca operates in over 100 countries
and its innovative medicines are used by millions of patients
worldwide. Please visit (
and follow the Company on Twitter @AstraZeneca


For details on how to contact the Investor Relations Team, please
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1. American Cancer Society. Stomach Cancer: About Stomach Cancer.
Available at:

2. Bray F, et al. Global cancer statistics 2018: GLOBOCAN estimates
of incidence and mortality worldwide for 36 cancers in 185 countries.
CA Cancer J. Clin. 2018; 68:394-424.

3. American Cancer Society. Stomach Cancer: Early Detection,
Diagnosis, and Staging. Available at:

4. American Cancer Society. Stomach Cancer: Treating Stomach Cancer.
Available at:

5. Iqbal N, Iqbal N. Human Epidermal Growth Factor Receptor 2 (HER2)
in Cancers: Overexpression and Therapeutic Implications. Mol Biol
Int. 2014;2014:852748. doi:10.1155/2014/852748.

6. Curea F.G, et al. Current Targeted Therapies in HER2-Positive
Gastric Adenocarcinoma. Cancer Biotherapy & Radiopharmaceuticals.
2017;32 (10).

7. NCCN Guidelines® Gastric Cancer. Version 4.2019. December 20,
2019: MS-22-36.

8. ClinicalTrials.Gov. NCT03329690. Available at:

Christina Malmberg HägerstrandPresschef AstraZeneca AB och Norden
Baltikum+ 46 8 552 53 106


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