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

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- FDA grants M7824, an investigational bifunctional immunotherapy,
orphan drug designation in biliary tract cancer
- First regulatory designation for M7824 following recent
presentation of first clinical data in BTC
- 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 US Food and Drug Administration (FDA) has granted orphan
drug designation (ODD) to M7824, the first regulatory designation for
the bifunctional immunotherapy, for the treatment of biliary tract
cancer (BTC). The FDA orphan drug designation follows the recent
presentation of the first clinical data for M7824 in BTC at the
European Society of Medical Oncology (ESMO) congress in October.
M7824 is an investigational bifunctional immunotherapy designed to
combine co-localized blocking of the transforming growth factor-? and
anti-PD-L1 immune escape mechanisms.

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 16,000 cases of BTC are estimated
to occur every year in the US and collectively these cancers present
late in the majority of patients.[1],[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]

"Biliary tract cancer is a rare, notoriously hard-to-treat tumor
where existing treatment approaches, such as surgery or chemotherapy,
are either not viable or simply don't deliver acceptable patient
outcomes," said Luciano Rossetti, Head of Global Research &
Development at the Biopharma business of Merck. "As the first
regulatory designation for M7824, Merck is excited about the
potential of this new class of immunotherapy in a number of
challenging cancers and settings."

The first clinical data for M7824 in BTC, presented at the ESMO
congress in October, demonstrated clinical activity in Asian patients
who had progressed after platinum-based first-line treatment. The ORR
among the total of 30 patients was 20%, as assessed by 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
TRAEs were experienced by 10 patients (33.3%) and the most common
Grade 3 TRAEs were rash (10%) and lipase increase (10%).

FDA Orphan Drug Designation (ODD) is granted to medicines intended
to treat rare diseases or disorders that affect fewer than 200,000
people in the US, or those that affect more than 200,000 people but
are unlikely to recover the costs of developing and marketing the
drug. Medicines that meet the FDA's ODD criteria qualify for a number
of incentives to help support advancement.

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, 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 FDA Orphan Designation

FDA orphan drug designation is granted to drugs intended to treat
rare diseases or disorders that affect fewer than 200,000 people in
the US, or those that affect more than 200,000 people, but are
unlikely to recover the costs of developing and marketing the drug.
Orphan drug designation by the FDA qualifies the sponsor for
incentives provided for in the Orphan Drug Act, which can include
protocol assistance for clinical trials, prescription drug user fee
waivers, tax incentives and seven 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. The orphan drug designation for M7824 applies only to BTC.

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 140,000 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]

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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. Current
Problems in Cancer (2018) https://www.sciencedirect.com/science/ar
ticle/pii/S0147027217301083?via%3DihubAccessed November 2018
2. Global Burden of Disease Study 2013. The Lancet
2015;385(9963):117-171.
3. Hezel AF et al. Genetics of biliary tract cancers and emerging
targeted therapies. J Clin Oncol 2010;28:3531-40.
http://dx.doi.org/10.1200/JCO.2009.27.4787 Accessed November 2018
4. Goldstein D et al. New molecular and immunotherapeutic approaches
in biliary cancer. ESMO Open (2017). Published online 2017 Mar 27.
doi: https://dx.doi.org/10.1136%2Fesmoopen-2016-000152 Accessed
November 2018
5. Lamarca A, et al. Ann Oncol. 2014;25(12):2328-2338.

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