Pharmacyclics First to Announce Breakthrough Therapy Designation in Oncology from the U.S. Food and Drug Administration

 Pharmacyclics First to Announce Breakthrough Therapy Designation in Oncology
                  from the U.S. Food and Drug Administration

PR Newswire

SUNNYVALE, Calif., Feb. 12, 2013

SUNNYVALE, Calif., Feb. 12, 2013 /PRNewswire/ -- Pharmacyclics, Inc. (the
"Company") (Nasdaq: PCYC) announced today that the U.S. Food and Drug
Administration (FDA) granted Breakthrough Therapy Designation to the
investigational oral agent ibrutinib monotherapy for the treatment of patients
with relapsed or refractory mantle cell lymphoma (MCL) and to ibrutinib
monotherapy for the treatment of patients with Waldenstrom's macroglobulinemia
(WM), both of which are 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."[i] The designation of a drug as a Breakthrough Therapy
was enacted as part of the 2012 Food and Drug Administration Safety and
Innovation Act.

Pharmacylics, 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 filing requirements for the use of ibrutinib in
patients with MCL and WM. The company expects to finalize the MCL filing prior
to year end and will provide guidance on the WM filing after further
discussions with the FDA. Pharmacyclics will provide regulatory updates as
further information on implementing the requirements with respect to
Breakthrough Therapies are developed by the Secretary of Health and Human

"This is a historic moment in oncology. We are truly honored to have received
this Breakthrough Designation and are pleased for patients and clinicians with
the FDA's decision to expedite the development of ibrutinib," said Bob Duggan,
CEO and Chairman of the Board. "This compound entered the clinic in 2009 and
has demonstrated tremendous clinical progress over the past four years. I
would like to thank our collaboration partner, Janssen, for their support.
Together we are committed to bringing this new therapeutic to patients and
health care providers."

Dr. Ellen Sigal, Chair & Founder of Friends of Cancer Research, a think tank
and advocacy organization based in Washington, D.C., said, "As an oncology
product, ibrutinib receiving the Breakthrough Therapy Designation is an
example of progress and hope for patients fighting a range of cancers. This
designation shows that the FDA is dedicated to using an 'all hands on deck
approach' to work on products that show promise in treating serious and
life-threatening diseases. The breakthrough pathway that our organization
worked to create is intended to speed up the development and review of
treatments that may demonstrate substantial improvement over existing
therapies, and ibrutinib is a great example of using this new designation to
potentially accelerate patient access to promising treatments."

TheFDA Breakthrough Therapy Designation for ibrutinib in MCL patients was
based on data from clinical and pre-clinical studies. Ibrutinib has the
potential to improve the outcome in this serious and life-threatening disease
which has a poor prognosis. The company and Janssen have designed a
comprehensive development program of ibrutinib in MCL which includes the
following studies:
- A Phase III randomized, multi-center registration trial of ibrutinib as a
monotherapy versus temsirolimus in relapsed or refractory MCL patients who
received at least one prior rituximab-containing chemotherapy regimen, RAY
- A Phase III randomized, double-blind, placebo-controlled study of ibrutinib
plus bendamustine and rituximab versus placebo plus bendamustine and rituximab
in subjects with newly diagnosed MCL, SHINE (MCL3002).
- A Phase II single-arm study of ibrutinib as a monotherapy in patients with
MCL who received at least one prior rituximab-containing chemotherapy regimen
and who progressed after bortezomib therapy, SPARK (MCL2001).
- A Phase II single-arm study of ibrutinib as monotherapy in patients with
relapsed or refractory MCL (PCYC-1104).

Conference Call
Further updates will be provided during the upcoming quarterly conference call
on February 14, 2013 at 4:30 p.m. EDT. To participate in the conference call,
please dial 1-877-407-0778 for domestic callers and 1-201-689-8565 for
international callers. To access the live audio broadcast or the subsequent
archived recording, log on to The
archived version of the webcast will be available for 30 days on the Investor
Relations section of the company's Web site at

About Mantle Cell Lymphoma
MCL is an aggressive type of B-cell non-Hodgkin lymphoma (NHL) that usually
occurs in older adults. The disease typically begins in the lymph nodes but
can spread to other tissues, such as bone marrow and liver. Ibrutinib targets
the B-cell receptor pathway an important pathway in malignant B-cell growth
and proliferation. In the United States there are approximately 5,000 new
cases of MCL each year.

About Waldenstrom's macroglobulinemia
Waldenstrom's macroglobulinemia is rare type of lymphoma. The disease begins
with a malignant change to the B-cell during its maturation so that it
continues to reproduce more malignant B-cells resulting in an overproduction
of monoclonal immunoglobulin M antibody (IgM). WM is a hematologic malignancy
for which no established standard of care - or approved therapeutic exists. In
the United States there are approximately 1,500 new cases each year.

About Ibrutinib
Ibrutinib isan investigational drug 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 – regulating apoptosis, adhesion, and cell migration and
homing. Through these multiple actions, BTK helps to direct malignant B-cells
to lymphoid tissues, thus allowing access to a micro environment necessary for

The effectiveness and safetyof 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 27 trials are currently registered on

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

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 

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",
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intended to identify such forward-looking statements. These forward-looking
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substantial additional financing and the availability and terms of any such
financing, the safety and/or efficacy results of clinical trials of our
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achieve market acceptance of any of our product candidates, for which we rely
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expectations reflected in the forward-looking statements are reasonable, we
cannot guarantee future results, performance or achievements and no assurance
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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
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.

Contact: Ramses Erdtmann, Vice President of Investor Relations,
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