New Drug Application for Ibrutinib Submitted to the U.S. FDA
SUNNYVALE, Calif., July 10, 2013
SUNNYVALE, Calif., July 10, 2013 /PRNewswire/ -- Pharmacyclics, Inc. (Nasdaq:
PCYC) today announced that it has submitted a New Drug Application (NDA) to
the U.S. Food and Drug Administration (FDA) for the investigational oral
Bruton's tyrosine kinase (BTK) inhibitor, ibrutinib, for two
relapsed/refractory B-cell malignancy indications: mantle cell lymphoma (MCL)
and chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL). The
submission was based on data from Phase II studies in patients with
relapsed/refractory MCL and in patients with relapsed/refractory CLL/SLL. With
this submission, Pharmacyclics is also requesting Priority Review.
Pharmacyclics is jointly developing ibrutinib with Janssen.
The NDA submission follows the receipt of a Breakthrough Therapy Designation
from the FDA in February 2013 for ibrutinib as a monotherapy for the treatment
of patients with relapsed/refractory MCL, and receipt of a second Breakthrough
Therapy Designation for the treatment of patients with CLL/SLL with deletion
of the short arm of chromosome 17 (del 17p). Further information on the
implications of this filing for potential commercialization will be provided
subsequent to the FDA rendering a decision on the filing.
"We are very excited having achieved this major milestone. This first NDA for
ibrutinib was made possible in record time because of the continuous support
and consultations we received from the FDA," said Dr. Urte Gayko, Senior Vice
President of Global Regulatory Affairs, Pharmacyclics. "We look forward to
continuing to work with the FDA as they review the application for ibrutinib
through the new Breakthrough Therapy Designation process."
"These past months have been enormously active and productive for
Pharmacyclics and our ibrutinib partner, Janssen. We received our first of
three Breakthrough Therapy Designations just this past February and since then
published various clinical results in patients with CLL del17p and also in
patients with Waldenstroms; in April we completed enrollment of our first
Phase III study; last month, results of two of our trials were published in
the prestigious New England Journal of Medicine and today Pharmacyclics
announced the filing of its first NDA for ibrutinib with the Food and Drug
Administration," said Bob Duggan CEO and Chairman of Pharmacyclics. "Thus far,
more than 1600 patients have been treated in our studies with ibrutinib and we
are making excellent progress in the development and preparation for
commercialization of this investigational drug. As of today, we have initiated
7 Phase III studies together with our partner Janssen and have currently
registered with the US National Institute of Health 31 clinical trials using
ibrutinib. It is Pharmacyclics's goal to advance science and drug development
in the hopes of making a significant difference for the betterment of patients
with serious unmet healthcare needs, and with the announcement today we have
accomplished an important milestone in our timeless journey."
About CLL / SLL
CLL, a B-cell malignancy, is a slow-growing blood cancer that starts in the
white blood cells (lymphocytes), most commonly from B-cells. CLL is the second
most common adult leukemia. Approximately 15,680 patients in the US are
diagnosed each year with CLL. The prevalence of CLL is approximately 113,000
in the U.S. It is a chronic disease of the elderly with a five-year survival
of approximately 82 percent.^1 Patients commonly receive multiple lines of
treatment over the course of their disease. When cancer cells are located
mostly in the lymph nodes, the disease is called SLL. CLL and SLL are
considered to be different manifestations of the same underlying disease; they
share similarities in signs and symptoms, genetic features, disease
progression and treatment.
In CLL, the genetic mutation del 17p occurs when the short arm of chromosome
17 is missing. Del 17p CLL is associated with abnormalities of a key tumor
suppressor gene, TP53, which results in poor response to chemoimmunotherapy
and worse treatment outcomes. It occurs in about seven percent of treatment
naïve CLL patients, with approximately 20-40 percent of relapsed/refractory
patients harboring the mutation.
About Mantle Cell Lymphoma
MCL is a B-cell malignancy, an aggressive type of B-cell non-Hodgkin lymphoma
(NHL) that usually occurs in older adults.^2 The disease typically begins in
the lymph nodes, but can spread to other tissues, such as bone marrow, liver,
and spleen^3. Patients typically survive an average of five years.^4In the
U.S., there are approximately 5,000 new cases of MCL each year.^3
Ibrutinib is an investigational agent designed to provide potent and sustained
inhibition of an enzyme called Bruton's tyrosine kinase (BTK). BTK is a key
mediator of at least three critical B-cell pro-survival mechanisms occurring
in parallel – regulation of apoptosis, adhesion, and cell migration and
homing. Through these multiple signals, BTK regulation helps to direct
malignant B-cells to lymphoid tissues, thus allowing access to a
micro-environment necessary for survival.
The effectiveness of ibrutinib alone or in combination with other treatments
is being studied in several B-cell malignancies, including chronic lymphocytic
leukemia/small lymphocytic lymphoma, mantle cell lymphoma, diffuse large
B-cell lymphoma, follicular lymphoma, Waldenström's macroglobulinemia and
multiple myeloma. To date, 7 Phase III trials have been initiated with
ibrutinib and a total of 31 trials are currently registered on
www.clinicaltrials.gov. Janssen and Pharmacyclics entered a collaboration and
license agreement in December 2011 to co-develop and co-commercialize
Pharmacyclics^® is a clinical-stage biopharmaceutical company focused on
developing and commercializing innovative small-molecule drugs for the
treatment of cancer and immune mediated diseases. Our mission and goal is to
build a viable biopharmaceutical company that designs, develops and
commercializes novel therapies intended to improve quality of life, increase
duration of life and resolve serious unmet medical healthcare needs; and to
identify promising product candidates based on scientific development and
administrational expertise, develop our products in a rapid, cost-efficient
manner and pursue commercialization and/or development partners when and where
Presently, Pharmacyclics has three product candidates in clinical development
and several preclinical molecules in lead optimization. The company is
committed to high standards of ethics, scientific rigor, and operational
efficiency as it moves each of these programs to viable commercialization.
Pharmacyclics is headquartered in Sunnyvale, California and is listed on
NASDAQ under the symbol PCYC. To learn more about how Pharmacyclics advances
science to improve human healthcare visit us at http://www.pharmacyclics.com.
NOTE: This announcement may contain forward-looking statements made in
reliance upon the safe harbor provisions of Section 27A of the Securities Act
of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934,
as amended, including statements, among others, relating to our future capital
requirements, including our expected liquidity position and timing of the
receipt of certain milestone payments, and the sufficiency of our current
assets to meet these requirements, our future results of operations, our
expectations for and timing of ongoing or future clinical trials and
regulatory approvals for any of our product candidates, and our plans,
objectives, expectations and intentions. Because these statements apply to
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"intend", "target" and similar expressions are intended to identify such
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information currently available to us and are subject to a number of risks,
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that may cause such a difference include, without limitation, our need for
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expectations reflected in the forward-looking statements are reasonable, we
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forward-looking statements. For more information about the risks and
uncertainties that may affect our results, please see the Risk Factors section
of our filings with the Securities and Exchange Commission, including our
transition report on Form 10-K for the six month period ended December 31,
2012 and quarterly reports on Form 10-Q. We do not intend to update any of the
forward-looking statements after the date of this announcement to conform
these statements to actual results, to changes in management's expectations or
otherwise, except as may be required by law.
^1 Cancer.net. "Leukemia – Chronic Lymphocytic - CLL".
Accessed June 2013.
^2 Cancer.net. "Lymphoma – Non-Hodgkin".
^3 Know Cancer. "Mantle Cell Lymphoma". Available at:
http://www.knowcancer.com/oncology/mantle-cell-lymphoma/. Accessed April 2013.
^4 Geisler, C. (2010) "Front-line treatment of mantle cell lymphoma."
Haematologica, 95:8:1241-1243. Available at:
SOURCE Pharmacyclics, Inc.
Contact: Ramses Erdtmann, SVP of Investor Relations and Corporate
Communications, +1- 408-215-3325, or U.S. Medical Information, Pharmacyclics,
+1-855-ibrutinib +1-855-427-8846, email@example.com
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