Ibrutinib Monotherapy Clinical Trial Data in Patients with Waldenstrom's Macroglobulinemia Presented at the International

   Ibrutinib Monotherapy Clinical Trial Data in Patients with Waldenstrom's
   Macroglobulinemia Presented at the International Conference on Malignant
                       Lymphoma in Lugano, Switzerland

PR Newswire

SUNNYVALE, Calif., June 20, 2013

SUNNYVALE, Calif., June 20, 2013 /PRNewswire/ --Pharmacyclics, Inc. (the
"Company") (Nasdaq: PCYC) today announced results of a study evaluating
ibrutinib, an investigational oral Bruton's tyrosine kinase (BTK) inhibitor in
patients with Waldenstrom's Macroglobulinemia (WM). Pharmacyclics is jointly
developing ibrutinib with Janssen Research & Development, LLC. The data were
presented today at the International Conference on Malignant Lymphoma (ICML),
taking place in Lugano, Switzerland.

A Phase II study examining the efficacy and tolerability of ibrutinib in
patients with relapsed/refractory WM was led by Dr. Steven Treon at the Bing
Center for Waldenstrom's Research in Boston, MA. In WM the malignant B-cells
produce large amounts of a normal antibody called immunoglobulin M (IgM).
Antibodies such as IgM help the body to fight infection; however, in WM excess
IgM causes the blood to thicken and causes many of the symptoms of the

Thirty five relapsed/refractory WM patients who received a median of 2 prior
therapies were evaluated. Key findings presented at ICML include:

  oThe best overall response rate was 83% (11.4% very good partial response;
    54.3% partial response; 17.1% minor response)
  oAfter six treatment cycles (a cycle = 4 weeks of treatment) bone marrow
    disease burden decreased from 70% down to 40% (p=0.0004)
  oLevels of IgM reduced from 3,190 mg/dL at baseline to 1,232 mg/dL (6
    cycles best response; p=5.1x10^-9)
  oRed blood cell production improved with levels of hematocrit increasing
    from 30.8% to 39.7% (6 cycles best response; p=1.1x10^-11)
  oThe safety profile observed in patients with WM was similar to the
    established safety profile in other B-cell malignancies. Grade 3 and
    higher adverse events associated with the treatment of ibrutinib were
    infrequent with thrombocytopenia and neutropenia seen in 8.6%, and single
    cases of stomatitis and atrial fibrillation. None of these events led to
    ibrutinib discontinuation.
  oAs of June 3, 2013, 91.4% of patients remain on the study with a minimum
    of 6 cycles of follow up. The study was extended by an additional 28
    patients to further evaluate the safety and efficacy of ibrutinib in WM
    and a total of 63 patients are now enrolled.

"These data suggest that ibrutinib was a highly active and well-tolerated
treatment for Waldenstrom's patients in this study, and provide further
evidence of the role of BTK in tumor growth," noted Steven Treon, M.D., Ph.D,
Director of the Bing Center for Waldenstrom's Research and an attending
physician at Dana-Farber Cancer Institute and Brigham and Women's Hospital, in
Boston, MA. "Despite the relatively small patient population, there are
currently no approved treatments specifically for Waldenstrom's, so the need
for novel therapies such as ibrutinib should not be under-estimated."

About Ibrutinib
Ibrutinib is an investigational agent 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 six Phase III trials have been initiated with
ibrutinib and a total of 31 trials are currently registered on
www.clinicaltrials.gov. Janssen Biotech, Inc. and Pharmacyclics entered a
collaboration and license agreement in December 2011 to co-develop and
co-commercialize ibrutinib.

About WM
Slow-growing and rare, with an incidence in the U.S. and Europe of
approximately 3-5 cases a year per million people,^[3] WM belongs to a family
of cancers classified as non-Hodgkin lymphoma (NHL). WM originates from
B-cells, a type of white blood cell (lymphocyte) that develops in the bone
marrow. Approximately 70 percent of WM cases are diagnosed in those over the
age of 65,^[4] and the median overall survival is 5-11 years.^[5],[6]

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 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] Pub Med Health. Waldenstrom's macroglobulinemia; macroglobulinemia -
primary; Lymphoplasmacytic lymphoma. Available from:
http://www.ncbi.nlm.nih.gov/pubmedhealth/PMH0001614/. Accessed May 14, 2013.

[2] American Cancer Society. Waldenstrom macroglobulinema. Available from:
Accessed May 14, 2013.

[3] Garcia-Sanz R, Ocio E. Novel treatment regimens for Waldenstrom's
macroglobulinemia. Expert Rev Hematol. 2010; 3:339-50.

[4] American Cancer Society. Waldenstrom macroglobulinemia: Key Statistics.
Available from:
Accessed May 14, 2013.

[5] Vijay A, Gertz MA. Waldenstrom macroglobulinemia. Blood.

[6] Fonseca R, Hayman S. Waldenstrom macroglobulinaemia. Br J

SOURCE Pharmacyclics, Inc.

Website: http://www.pharmacyclics.com
Contact: Ramses Erdtmann, SVP of Investor Relations, Phone: 408-215-3325, or
U.S. Medical Information, Phamacyclics, 855-ibrutinib [(855) 427-8846]
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