New Drug Application Filing for Ibrutinib Accepted in Two B-cell Malignancies by the U.S. FDA

New Drug Application Filing for Ibrutinib Accepted in Two B-cell Malignancies
                               by the U.S. FDA

Priority Review Granted

PR Newswire

SUNNYVALE, Calif., Aug. 29, 2013

SUNNYVALE, Calif., Aug. 29, 2013 /PRNewswire/ -- Pharmacyclics, Inc. (Nasdaq:
PCYC) today announced that the U.S. Food and Drug Administration (FDA) has
accepted for filing its New Drug Application (NDA) for the investigational
oral Bruton's tyrosine kinase (BTK) inhibitor ibrutinib, for two B-cell
malignancy indications: previously treated mantle cell lymphoma (MCL) and
previously treated chronic lymphocytic leukemia (CLL)/small lymphocytic
lymphoma (SLL). On June 28, 2013 Pharmacyclics submitted a New Drug
Application (NDA) under section 505(b) of the Food, Drug & Cosmetic Act for
ibrutinib. On Aug 27, 2013 the FDA notified Pharmacyclics that they have
completed their filing review and determined that the application is
sufficiently complete to permit a substantive review. The FDA's acceptance of
the NDA triggers a $75 million milestone payment to Pharmacyclics under its
Collaboration Agreement with Janssen Biotech Inc.

"We are very excited to have received the official FDA acceptance of our first
NDA filing for ibrutinib," said Dr. Urte Gayko, Senior Vice President of
Global Regulatory Affairs, Pharmacyclics. "We look forward to continuing to
work with the FDA as they complete their review of the ibrutinib application
which includes the new Breakthrough Therapy Designation process."

About CLL / SLL

CLL, a B-cell malignancy, is a slow-growing blood cancer of the white blood
cells (lymphocytes), most commonly from B-cells. CLL is the second most common
adult leukemia. Approximately 16,000 patients in the US are diagnosed each
year with CLL. The prevalence of CLL is approximately 113,000 in the U.S. CLL
is a chronic disease that predominantly occurs in 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.

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 2,500 new cases of MCL each year and a
prevalence of approximately 10,000 ^ (Decision Resources 2012).

About Ibrutinib

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, Waldenstrom'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 2011to co-develop and co-commercialize
ibrutinib.

About Pharmacyclics

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
appropriate.

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
future events, they are subject to risks and uncertainties. When used in this
announcement, the words "anticipate", "believe", "estimate", "expect",
"expectation", "goal", "should", "would", "project", "plan", "predict",
"intend", "target" and similar expressions are intended to identify such
forward-looking statements. These forward-looking statements are based on
information currently available to us and are subject to a number of risks,
uncertainties and other factors that could cause our actual results,
performance, expected liquidity or achievements to differ materially from
those projected in, or implied by, these forward-looking statements. Factors
that may cause such a difference include, without limitation, our need for
substantial additional financing and the availability and terms of any such
financing, the safety and/or efficacy results of clinical trials of our
product candidates, our failure to obtain regulatory approvals or comply with
ongoing governmental regulation, our ability to commercialize, manufacture and
achieve market acceptance of any of our product candidates, for which we rely
heavily on collaboration with third parties, and our ability to protect and
enforce our intellectual property rights and to operate without infringing
upon the proprietary rights of third parties. Although we believe that the
expectations reflected in the forward-looking statements are reasonable, we
cannot guarantee future results, performance or achievements and no assurance
can be given that the actual results will be consistent with these
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".
http://www.cancer.net/cancer-types/leukemia-chronic-lymphocytic-cll/statistics.
Accessed June 2013.

^2 Cancer.net. "Lymphoma — Non-Hodgkin".
http://www.cancer.net/cancer-types/lymphoma-non-hodgkin/subtypes. Accessed
April 2013.

^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:
http://www.haematologica.org/content/95/8/1241.full

SOURCE Pharmacyclics, Inc.

Website: http://www.pharmacyclics.com
Contact: Ramses Erdtmann, SVP of Investor Relations and Administration,
408-215-3325; Manisha Pai, Sr. Director Public Relations & Corporate
Communications, 408-215-3720; U.S. Medical Information, Pharmacyclics,
855-ibrutinib [(855)-427-8846], medinfo@pcyc.com
 
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