Pharmacyclics Completes Enrollment of First Phase III Ibrutinib CLL Study - RESONATE™ and Announces Completion of Enrollment

 Pharmacyclics Completes Enrollment of First Phase III Ibrutinib CLL Study -
  RESONATE™ and Announces Completion of Enrollment of Phase II Ibrutinib MCL
                                Study - SPARK

PR Newswire

SUNNYVALE, Calif., April 18, 2013



SUNNYVALE, Calif., April 18, 2013 /PRNewswire/ -- Pharmacyclics, Inc. (Nasdaq:
PCYC) announced today that the enrollment target of 350 patients for its Phase
III study using ibrutinib monotherapy versus ofatumumab in patients with
relapsed or refractory chronic lymphocytic leukemia / small lymphocytic
lymphoma (CLL/SLL), (RESONATE™) was achieved on April 3, 2013. As of today,
additional 41 patients were screened and are allowed to participate in this
study which has now been officially closed to enrollment.

The primary endpoint of this study is to demonstrate a clinically significant
improvement in progression-free survival with ibrutinib when compared to
ofatumumab. A pre-defined number of progression events will trigger an interim
analysis. We would expect to have a read out from the interim analysis during
the 1st quarter 2014. If the treatment effect of ibrutinib in comparison to
ofatumumab is deemed statistically favorable by an independent review
committee a discussion with the FDA and other health authorities for a
potential early filing can take place.

"In addition to our outstanding team, we are grateful to the patients, their
treating physicians, and the clinical sites for their participation in our
RESONATE study and their strong support of the ibrutinib program," said Maria
Fardis, Chief of Oncology Operations and Alliances at Pharmacyclics.

Pharmacyclics also announced the completion of enrollment of the planned 110
patients in the Phase II single-arm study using ibrutinib in patients with
mantle cell lymphoma (MCL) who progress after bortezomib therapy and have
received at least one prior rituximab-containing chemotherapy regimen, SPARK
(MCL2001). This global study is conducted by Janssen Research & Development,
LLC, and its primary endpoint is overall response rate, which is scheduled to
be evaluated 6 months from the completion of enrollment. Further updates to
this study will be provided by Janssen.

As previously announced Pharmacyclics expects to file a New Drug Application
(NDA) with the FDA for the use of ibrutinib in patients with relapsed or
refractory MCL prior to year-end. In the time before a potential U.S.
marketing approval Pharmacyclics will strive to provide early access to
ibrutinib under an Early Access Program (EAP). EAPs are clinical studies and
allowed under certain circumstances by the FDA. They are designed to provide a
mechanism for access to an investigational drug to treat patients with a
serious or immediately life-threatening disease or condition until the time of
an anticipated U.S. marketing approval. A multicenter, open-label EAP,
conducted by Janssen, will be initiated in the United States with ibrutinib
for relapsed or refractory MCL patients. Information about this program is
posted and updated on www.clinicaltrials.gov.

"The completion of the enrollment of our first Phase III study with ibrutinib,
ahead of schedule, is an important milestone for our clinical development plan
and a real achievement for our company," said Bob Duggan CEO and Chairman of
Pharmacyclics. "As of today we have initiated 5 Phase III studies together
with our partner Janssen and we currently have registered with the US National
Institute of Health 28 clinical trials using ibrutinib. Thus far over 1200
patients have been dosed in our studies and we are making excellent progress
in the development of this investigational drug, as underscored by the most
recent three Breakthrough Therapy Designations we received from the FDA. It is
Pharmacyclics' goal to advance science and drug development in the hopes of
making a significant difference for the betterment of patients with serious
unmet needs, and we are steadily progressing towards that worthy goal."

About 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, cell adhesion, and cell migration and homing.

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 5 Phase III trials have been initiated with
ibrutinib and a total of 28 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.

Ofatumumab is manufactured by Glaxo Group Limited and distributed by
GlaxoSmithKline.

Bortezomib is marketed and distributed by Millennium Pharmaceuticals, Inc.

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", "goal",
"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, Phone:
408-215-3325
 
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