Merck Announces EMA Orphan Drug Designation for Bifunctional Immunotherapy M7824 in Biliary Tract Cancer

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- European Medicine Agency (EMA) grants M7824, an investigational
bifunctional immunotherapy, orphan drug designation in biliary
tract cancer (BTC)
- EMA orphan drug designation for M7824 in BTC follows FDA orphan
drug designation weeks earlier
- BTC is a group of rare, aggressive gastrointestinal cancers
associated with limited treatment options and poor outcomes

Merck, a leading science and technology company, today announced
that the European Medicines Agency (EMA) has granted orphan drug
designation (ODD) to M7824, for the treatment of biliary tract cancer
(BTC). The EMA ODD follows the Food and Drug Administration (FDA)
also granting orphan drug designation to M7824 in BTC just weeks ago.
M7824 is an investigational bifunctional immunotherapy designed to
combine co-localized blocking of the transforming growth factor-?
(TGF-?) and PD-L1 immune escape mechanisms.

"EMA orphan drug designation is another recognition of Merck's
determination to bring innovative therapies to people suffering from
challenging cancers like biliary tract cancer," said Luciano
Rossetti, Head of Global Research & Development at the Biopharma
Business of Merck. "This is the second orphan drug designation for
M7824 in a matter of weeks and Merck is eager to further explore the
potential of this new class of immunotherapy to advance outcomes in a
number of difficult-to-treat tumors."

BTC is a collective term for a group of rare and aggressive
gastrointestinal cancers, including intrahepatic cholangiocarcinoma
(ICC), extrahepatic cholangiocarcinoma (ECC), and gallbladder
carcinoma (GBC).[1] Approximately 186,000 cases of BTC are estimated
to occur annually worldwide.[2] Treatment options are limited and the
median survival rate in the advanced setting is less than one year;
objective tumor response with commonly used chemotherapy is typically
less than 10% with short duration of response.[1],[3],[5]

EMA orphan designation is designed to encourage the development of
new treatments for life-threatening or chronically debilitating
conditions that are rare (affecting not more than five in 10,000
people in the European Union). Medicines that meet the EMA's orphan
designation criteria qualify for a number of incentives to help
support advancement.

The first clinical data for M7824 in BTC, presented at the
European Society of Medical Oncology (ESMO) congress in October,
demonstrated clinical activity in Asian patients who had progressed
after platinum-based first-line treatment. The overall response rate
(ORR) among the total of 30 patients was 20%, as assessed by an
independent review committee (IRC), and responses were observed
across PD-L1 levels with a duration of response ranging from 8.3
months to 13.9+ months. Grade 3 or higher treatment-related adverse
events (TRAEs) were experienced by 10 patients (33.3%) and the most
common Grade 3 TRAEs were rash (10%) and lipase increase (10%).

M7824 is an investigational bifunctional immunotherapy that
combines a TGF-? trap with the anti-PD-L1 mechanism in one fusion
protein. Designed to combine co-localized blocking of the two
immunosuppressive pathways, M7824 is thought to control tumor growth
by potentially restoring and enhancing anti-tumor responses. M7824 is
an important part of a novel combination approach that seeks to
harness the power of the immune system and address the tremendously
complex nature of difficult-to-treat tumors. To date, more than 670
patients with various types of solid tumors have been treated across
the program with M7824. In addition to BTC, M7824 is being studied in
solid tumor indications, including non-small cell lung cancer, HPV
associated tumors and gastrointestinal cancers, such as gastric
cancer, esophageal squamous cell carcinoma and esophageal
adenocarcinoma.

About M7824

M7824 is an investigational bifunctional immunotherapy that is
designed to combine a TGF-? trap with the anti-PD-L1 mechanism in one
fusion protein. M7824 is designed to combine co-localized blocking of
the two immunosuppressive pathways - targeting both pathways aims to
control tumor growth by potentially restoring and enhancing
anti-tumor responses. M7824 is currently in Phase I studies for solid
tumors, as well as a trial to investigate M7824 compared with
pembrolizumab as a first-line treatment in patients with
PD-L1-expressing advanced NSCLC. The multicenter, randomized,
open-label, controlled study is evaluating the safety and efficacy of
M7824 versus pembrolizumab as a monotherapy treatment.

About the EMA Orphan Designation

The EMA defines an orphan medicine as 'a medicine for the
diagnosis, prevention or treatment of a life-threatening or
chronically debilitating condition that is rare (affecting not more
than five in 10,000 people in the European Union) or where the
medicine is unlikely to generate sufficient profit to justify
research and development costs'. Orphan designation by the EMA
qualifies the sponsor for incentives provided for in Regulation (EC)
No 141/2000 (the Orphan Regulation), which can include protocol
assistance for clinical trials, access to the centralized
authorization procedure, reduced fees for regulatory activities and
ten years of market exclusivity. The granting of an orphan drug
designation does not alter the standard regulatory requirement to
establish the safety and effectiveness of a drug through adequate and
well-controlled studies to support approval.

About Biliary Tract Cancer (BTC)

BTC is a collective term for a group of rare and aggressive
gastrointestinal cancers, including intrahepatic cholangiocarcinoma
(ICC), extrahepatic cholangiocarcinoma (ECC), and gallbladder
carcinoma (GBC).[1] Surgery is the only curative treatment, but most
patients present with advanced disease and therefore have a limited
survival.[1] Approximately 186,000 cases of BTC are estimated to
occur annually world-wide.[2] However, incidence of BTC varies in
different parts of the world: the incidence of cholangiocarcinomas is
rising in the Western world, with reports of up to 2 in 100,000.[4]
By contrast, in Asian countries, the incidence is much higher.[4] GBC
also has an incidence of 2 in 100,000,?but is much more prevalent in
parts of South America.[4] Collectively, these cancers present late
in the majority of patients and long-term outcomes for resectable
patients are poor with median survival in the advanced setting less
than 1?year.[1],[3]

References

1. Blair A B et al. Immunotherapy as a treatment for biliary tract
cancers: A review of approaches with an eye to the future. Curr
Probl Cancer. 2018;42:49-58.
2. Naghavi M et al. The global burden of cancer 2013. JAMA Oncol.
2015;1: 505-527.
3. Hezel AF et al. Genetics of biliary tract cancers and emerging
targeted therapies. J Clin Oncol. 2010;28:3531-3540.
4. Goldstein D et al. New molecular and immunotherapeutic approaches
in biliary cancer. ESMO Open. 2017; 2(Suppl 1):e000152.
5. Lamarca A, et al. Second-line chemotherapy in advanced biliary
cancer: a systematic review. Ann Oncol. 2014;25:2328-2338.

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