Merck Announces FDA Acceptance for Review of MK-3475 Biologics License Application for Advanced Melanoma


May 6, 2014 7:40 am ET

Priority Review Designation for MK-3475 BLA for Proposed Indication of Unresectable or Metastatic Melanoma in Patients Who Have Been Previously Treated with Ipilimumab

Advancing New Phase 3 Studies with MK-3475 in Adjuvant Melanoma, Previously-Untreated Non-Small Cell Lung Cancer, Advanced Head & Neck and Bladder Cancers

Data from Studies in Three Different Cancer Types to be presented at 2014 ASCO Annual Meeting

Initiating Phase 1 Study with Investigational Anti-GITR Antibody (MK-4166)

BOSTON–(BUSINESS WIRE)–Merck (NYSE:MRK), known as MSD outside the United States and Canada,
today announced that the U.S. Food and Drug Administration (FDA) has
accepted for review the Biologics License Application (BLA) for MK-3475,
Merck’s investigational anti-PD-1 antibody, for the treatment of
unresectable or metastatic melanoma in patients who have been previously
treated with ipilimumab. The FDA granted Priority Review designation
with a PDUFA date of October 28, 2014, and the MK-3475 BLA will be
reviewed under the FDA’s Accelerated Approval program. The FDA
previously granted MK-3475 Breakthrough Therapy designation for advanced
melanoma, the most dangerous type of skin cancer. If approved by the
FDA, MK-3475 has the potential to be the first anti-PD-1 antibody in a
new class of immune checkpoint modulators.

Merck also announced it plans to file a Marketing Authorization
Application for MK-3475 in Europe for advanced melanoma by the end of

“Patients with advanced melanoma have few therapeutic options and often
fail to respond to all available treatments,” said Dr. Roger M.
Perlmutter, president, Merck Research Laboratories. “We are hopeful that
the FDA, through their priority review of our application, will agree to
make MK-3475 available to patients with advanced melanoma who have no
other therapeutic options.”

MK-3475 Development Ongoing in 30 Tumors, as Monotherapy and in

Today, Merck highlighted progress on the advancement of the MK-3475
development program – currently ongoing in 30 tumor types as monotherapy
and in combination. It is anticipated that by the end of 2014, the
MK-3475 development program will grow to more than 24 clinical trials
across 30 different tumor types, enrolling an estimated 6,000 patients
at nearly 300 clinical trial sites worldwide, including four new Phase 3
studies. Ongoing and planned late-stage monotherapy and combination
studies include:

  • Seven Phase 3 registrational trials spanning advanced melanoma
    (adjuvant, ipilimumab-naïve, and ipilimumab-refractory), advanced
    non-small cell lung cancer (NSCLC) (previously-treated and
    previously-untreated), advanced head & neck cancer and advanced
    bladder cancer; and,
  • Ten combination studies, including advanced melanoma, advanced
    NSCLC, advanced renal cell carcinoma, HER2+ breast cancer and other
    solid tumors.

Merck also said today that based on encouraging preclinical data, it
plans to initiate a Phase 1 dose-ranging study with its investigational
anti-GITR agonistic antibody, MK-4166, in patients with advanced
malignances. GITR (glucocorticoid-induced TNFR receptor) is an
activating immune checkpoint receptor, which is believed to stimulate
immune activity against cancer cells. This will be the second
investigational immune checkpoint antibody within Merck’s
immuno-oncology discovery program to enter clinical development.

Dr. Perlmutter added, “Our priority is advancing breakthrough
immunomodulatory molecules that reveal the ability of the immune system
to eliminate cancer cells. While MK-3475 provides a firm foundation for
Merck’s research and development strategy in oncology, we are also
advancing a broad pipeline of immune checkpoint agonists and

MK-3475 Presentations at 2014 ASCO Annual Meeting

At the 2014 American Society of Clinical Oncology (ASCO) Annual Meeting,
Merck will have more than 15 company-sponsored abstracts on MK-3475,
including six oral presentations. Clinical data will be presented from
studies in advanced melanoma, advanced NSCLC, as well as advanced head &
neck cancer, which is the first time data for MK-3475 will be presented
in this cancer type.

About Merck

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well. Merck is known as MSD outside the United States and Canada.
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Ian McConnell, 973-901-5722
Claire Mulhearn, 908-423-7425
Carol Ferguson, 908-423-4465
Justin Holko, 908-423-5088

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