Pharmacyclics Announces Third Breakthrough Therapy Designation for Ibrutinib from the U.S. Food and Drug Administration

 Pharmacyclics Announces Third Breakthrough Therapy Designation for Ibrutinib
                  from the U.S. Food and Drug Administration

PR Newswire

SUNNYVALE, Calif., April 8, 2013

SUNNYVALE, Calif., April 8, 2013 /PRNewswire/ --Pharmacyclics, Inc. (Nasdaq:
PCYC) announced today that the U.S. Food and Drug Administration (FDA) has
granted an additional Breakthrough Therapy Designation for the investigational
oral agent ibrutinib as monotherapy for the treatment of chronic lymphocytic
leukemia (CLL) or small lymphocytic lymphoma (SLL) patients with deletion of
the short arm of chromosome 17 (deletion 17p). Patients harboring a deletion
within chromosome 17 generally have poor response to chemoimmunotherapy and
have limited treatment options. The presence of deletion 17p is one of the
worst prognostic factors in patients with CLL.

In February 2013, FDA granted Breakthrough Therapy Designations for ibrutinib
as a monotherapy for the treatment of patients with relapsed or refractory
mantle cell lymphoma (MCL) and as a monotherapy for the treatment of patients
with Waldenstrom's macroglobulinemia (WM), both of which are also B-cell
malignancies. Ibrutinib is jointly being developed by Pharmacyclics and
Janssen for treatment of B-cell malignancies.

The Breakthrough Therapy Designation is intended to expedite the development
and review of a potential new drug for serious or life-threatening diseases
where "preliminary clinical evidence indicates that the drug may demonstrate
substantial improvement over existing therapies on one or more clinically
significant endpoints, such as substantial treatment effects observed early in
clinical development." The designation of a drug as a Breakthrough Therapy was
enacted as part of the 2012 Food and Drug Administration Safety and Innovation
Act. Pharmacyclics, together with Janssen, is working with the FDA to
determine the implications of this Breakthrough Therapy Designation to the
ongoing and planned development and the FDA filing requirements for the use of
ibrutinib in CLL patients with deletion 17p.

The FDA Breakthrough Therapy Designation for ibrutinib in CLL patients with
deletion 17p was based on data from pre-clinical and clinical studies where
ibrutinib as a monotherapy was used to treat patients with this disease.
Ibrutinib has the potential to improve the outcome in this serious and
life-threatening disease which has a poor prognosis. In addition,
Pharmacyclics and Janssen have recently initiated a Phase II study of
ibrutinib in patients with CLL deletion 17p, RESONATE™ -17, which is a
single-arm, open-label, multi-center trial using ibrutinib as a monotherapy in
patients who have deletion 17p and who did not respond to or relapsed after at
least one prior CLL treatment (a high unmet need population). The primary
endpoint of the study will be overall response rate. This global study opened
this year and Pharmacyclics plans to enroll 111 patients worldwide.

About Chronic Lymphocytic Leukemia

Chronic Lymphocytic Leukemia (CLL) 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 16,000 patients in the US are
diagnosed each year with CLL. The prevalence of CLL is approximately 113,000
in the US. The disease is a chronic disease of the elderly with an average
survival of about 5 years. Patients commonly receive multiple lines of
treatment over the course of their disease.

In CLL the genetic mutation 17p deletion occurs when the short arm of
chromosome 17 is missing. Del 17p 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 7% of
treatment naive CLL patients and is estimated to be approximately 20% to 40%
of relapsed or refractory patients harboring the mutation.

About Ibrutinib

Janssen Biotech, Inc. and Pharmacyclics entered a collaboration and license
agreement in December 2011 to co-develop and co-commercialize ibrutinib.
Ibrutinib was designed to specifically target and selectively inhibit 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 five Phase III trials have been initiated with
ibrutinib and a total of 26 trials are currently registered on
www.clinicaltrials.gov.

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.

The Company 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 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", "should",
"would", "project", "plan", "predict", "intend" 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 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
annual report on Form 10-K 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.

SOURCE Pharmacyclics, Inc.

Website: http://www.pharmacyclics.com
Contact: Ramses Erdtmann, SVP Investor Relations & Administration,
+1-408-215-3325
 
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