Merck Announces Breakthrough Therapy Designation for Lambrolizumab an Investigational Antibody Therapy for Advanced Melanoma


April 24, 2013 6:30 am ET

Merck (NYSE: MRK), known as MSD outside the United States and Canada,
announced today that the U.S. Food and Drug Administration (FDA) has
designated lambrolizumab (MK-3475) as a Breakthrough Therapy for the
treatment of patients with advanced melanoma. Lambrolizumab is Merck’s
investigational antibody therapy targeting Programmed Death receptor
(PD-1) that is currently being evaluated for the treatment of patients
with advanced melanoma, and other tumor types.

“We are pleased that the FDA has designated lambrolizumab a Breakthrough
Therapy for patients with advanced melanoma,” said Gary Gilliland M.D.,
Ph.D., senior vice president and oncology franchise head, Merck Research
Laboratories. “The FDA’s decision to place lambrolizumab in a category
that may enable expedited development and review is an important
milestone for Merck as we advance ongoing programs in multiple cancer

The designation of an investigational drug as a Breakthrough Therapy is
intended to expedite the development and review of a candidate that is
planned for use, alone or in combination, to treat a serious or
life-threatening disease or condition when preliminary clinical evidence
indicates that the drug may demonstrate substantial improvement over
existing therapies on one or more clinically significant endpoints. The
Food and Drug Administration Safety and Innovation Act (FDASIA) includes
a provision that allows sponsors to request that an investigational drug
be designated as a Breakthrough Therapy. The implications of
Breakthrough Therapy Designation cannot be determined at this time.

In November 2012, early interim results from a single-arm, open-label
Phase Ib study of lambrolizumab (MK-3475) administered to 85 patients
with advanced (inoperable and metastatic) melanoma were presented at the
Society for Melanoma Research of the 9th International Congress of the
Society for Melanoma Research (SMR) in Hollywood, Calif. (see news

Advanced melanoma

Advanced melanoma accounts for more than 80 percent of skin
cancer-related deaths and one to two percent of all cancer deaths in the
United States1. According to the American
Cancer Society
, in 2012 in the U.S. an estimated 9,180 people died
from advanced melanoma.

About PD-1

Researchers have shown that several tumor types are able to hide in
plain sight by establishing a “molecular camouflage” that deceives the
body’s immune system into thinking they are normal and therefore allow
them to grow unchecked. The interaction between the immune checkpoint
receptor PD-1 (programmed death) and its ligands represents a
potentially important tumor-specific immunomodulatory mechanism. By
utilizing the PD-1 pathway, a tumor cell can prevent the activation of
T-cells and therefore may block a key step that triggers the immune

About lambrolizumab

Lambrolizumab is an investigational antibody therapy designed to disrupt
the action of the immune checkpoint protein PD-1 and therefore inhibit
the ability of some cancers to evade the body’s immune system.
Lambrolizumab is being studied in multiple cancer types including
melanoma and non-small cell lung cancer. For further details, please

About Merck

Today’s Merck is a global healthcare leader working to help the world be
well. Merck is known as MSD outside of the United States and Canada.
Through our prescription medicines, vaccines, biologic therapies, and
consumer care and animal health products, we work with customers and
operate in more than 140 countries to deliver innovative health
solutions. We also demonstrate our commitment to increasing access to
healthcare through far-reaching policies, programs and partnerships. For
more information, visit
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Merck forward-Looking Statement

This news release includes “forward-looking statements” within the
meaning of the safe harbor provisions of the United States Private
Securities Litigation Reform Act of 1995. These statements are based
upon the current beliefs and expectations of Merck’s management and are
subject to significant risks and uncertainties. There can be no
guarantees with respect to pipeline products that the products will
receive the necessary regulatory approvals or that they will prove to be
commercially successful. If underlying assumptions prove inaccurate or
risks or uncertainties materialize, actual results may differ materially
from those set forth in the forward-looking statements.

Risks and uncertainties include but are not limited to, general industry
conditions and competition; general economic factors, including interest
rate and currency exchange rate fluctuations; the impact of
pharmaceutical industry regulation and health care legislation in the
United States and internationally; global trends toward health care cost
containment; technological advances, new products and patents attained
by competitors; challenges inherent in new product development,
including obtaining regulatory approval; Merck’s ability to accurately
predict future market conditions; manufacturing difficulties or delays;
financial instability of international economies and sovereign risk;
dependence on the effectiveness of Merck’s patents and other protections
for innovative products; and the exposure to litigation, including
patent litigation, and/or regulatory actions.

Merck undertakes no obligation to publicly update any forward-looking
statement, whether as a result of new information, future events or
otherwise. Additional factors that could cause results to differ
materially from those described in the forward-looking statements can be
found in Merck’s 2012 Annual Report on Form 10-K and the company’s other
filings with the Securities and Exchange Commission (SEC) available at
the SEC’s Internet site (

Accessed on April 17, 2013

Caroline Lapppetito, 267-305-7639
Carol Ferguson, 908-423-4465
Justin Holko, 908-423-5088

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