Pharmacyclics Announces Presentation of Phase III and Long Term Follow Up Data on IMBRUVICA™ in Chronic Lymphocytic Leukemia

Pharmacyclics Announces Presentation of Phase III and Long Term Follow Up Data
         on IMBRUVICA™ in Chronic Lymphocytic Leukemia (CLL) at ASCO

PR Newswire

SUNNYVALE, Calif., April 21, 2014

SUNNYVALE, Calif., April 21, 2014 /PRNewswire/ --Pharmacyclics, Inc. (NASDAQ:
PCYC) today announced that data from the Phase III PCYC-1112 (RESONATE™) study
of single agent IMBRUVICA™ (ibrutinib) vs. ofatumumab, an established therapy
in relapsed refractory CLL, as well as data from an Independent Efficacy
Evaluation of IMBRUVICA after three years of follow-up, will be presented at
the American Society of Clinical Oncology (ASCO) 50th Annual Meeting in
Chicago, Illinois May 30 - June 3, 2014. IMBRUVICA is being jointly developed
and commercialized by Pharmacyclics and Janssen Biotech, Inc.

"At the interim analysis of the RESONATE Phase III study in January, the data
showed that IMBRUVICA improved with statistical significance the overall
survival of patients with relapsed or refractory CLL versus ofatumumab, an
anti-CD-20 antibody similar to rituximab. IMBRUVICA also provided a
significant progression-free survival benefit for these patients," said John
C. Byrd, M.D., Director, Division of Hematology, The Ohio State University
Comprehensive Cancer Center – Arthur G. James Cancer Hospital & Richard J.
Solove Research Institute and lead investigator on the RESONATE study. "I am
excited by the oral, once daily, single agent, 'chemo-free' profile IMBRUVICA
offers patients. This class of drugs is transformative to the way we treat
patients today. I look forward to providing detailed results of the benefits
seen in the IMBRUVICA patients at the ASCO meeting."

"We have two historic oral presentations at this year's ASCO, one showing for
the first time a Survival Benefit with IMBRUVICA in a large randomized trial,
and the other evaluating the three-year safety and efficacy of IMBRUVICA in
relapsed and refractory CLL patients with a median of four prior therapies,"
said Bob Duggan, CEO and Chairman of Pharmacyclics. "Our team is currently
exploring the clinical benefits of IMBRUVICA in 42 clinical trials, of which
10 are large Phase III studies in a broad variety of B-Cell malignancies. With
each passing month, I am increasingly pleased with the launch progress we are
making, in partnership with our colleagues at Janssen, as we bring IMBRUVICA
to market for CLL and MCL patients across the United States."

RESONATE represents the first randomized Phase III study of IMBRUVICA in
patients with CLL.

Oral Presentations

Title: Randomized comparison of ibrutinib versus ofatumumab in relapsed or
refractory (R/R) chronic lymphocytic leukemia/small lymphocytic lymphoma:
Results from the phase III RESONATE trial (Abstract #LBA7008)
Session: Leukemia, Myelodysplasia, and Transplantation Oral Abstract Session
Date: Tuesday, June 3, 2014
Presentation Time: 11:57 a.m.
Location: McCormick Place, Room E354A
Presenter: John C. Byrd, M.D., The Ohio State University

Title: Independent evaluation of ibrutinib efficacy 3 years post-initiation of
monotherapy in patients with chronic lymphocytic leukemia/small lymphocytic
leukemia including deletion 17p disease (Abstract #7014)
Session: Leukemia, Myelodysplasia, and Transplantation Oral Abstract Session
Date: Tuesday, June 3, 2014
Presentation Time: 11:33 a.m.
Location: McCormick Place, Room E354A
Presenter: Susan O'Brien, M.D., The University of Texas MD Anderson Cancer
Center

Selected Poster Presentations

Title: A phase 1b/2 study evaluating activity and tolerability of the BTK
inhibitor ibrutinib in combination with ofatumumab in patients with chronic
lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and related diseases
(Abstract #7009)
Session: Leukemia, Myelodysplasia, and Transplantation Poster Highlights
Session
Date: Saturday, May 31, 2014
Presentation Time: 1:15 p.m.
Location: McCormick Place, Room S405, Poster #1
Presenter: Samantha Jaglowski, M.D., M.P.H.

Title: Association of disease progression on ibrutinib therapy with the
acquisition of resistance mutations: A single-center experience of 267
patients (Abstract #7010)
Session: Leukemia, Myelodysplasia, and Transplantation Poster Highlights
Session
Date: Saturday, May 31, 2014
Presentation Time: 1:15 p.m.
Location: McCormick Place, Room S405, Poster #2
Presenter: Jennifer Woyach, M.D.

Title: Preliminary safety and efficacy of the Bruton's tyrosine kinase (BTK)
inhibitor ibrutinib (IBR) in patients (pts) with hairy cell leukemia (HCL)
(Abstract #7063)
Session: Leukemia, Myelodysplasia, and Transplantation General Poster Session
Date: Monday, June 2, 2014
Presentation Time: 1:15 p.m.
Location: McCormick Place, S Hall A, Poster #348
Presenter: Jeffrey Jones, M.D., M.P.H.

INDICATIONS

IMBRUVICA is indicated for the treatment of:

  oPatients with mantle cell lymphoma (MCL) who have received at least one
    prior therapy.
  oPatients with chronic lymphocytic leukemia (CLL) who have received at
    least one prior therapy.

The FDA's accelerated approval of these indications was based on the overall
response rate of patients in the Phase II clinical trials of PCYC-1102 and
PCYC-1104. Improvements in survival or disease-related symptoms were not
established in these studies.

The following safety information is described in the package insert for the
use of IMBRUVICA in patients with mantle cell lymphoma who have received at
least one prior therapy or chronic lymphocytic leukemia who have received at
least one prior therapy:

IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS

Hemorrhage – Five percent of patients with MCL and 6% of patients with CLL had
Grade 3 or higher bleeding events (subdural hematoma, ecchymoses,
gastrointestinal bleeding, and hematuria). Overall, bleeding events including
bruising of any grade occurred in 48% of patients with MCL treated with 560 mg
daily and 63% of patients with CLL treated at 420 mg daily.

The mechanism for the bleeding events is not well understood. IMBRUVICA may
increase the risk of hemorrhage in patients receiving antiplatelet or
anticoagulant therapies. Consider the benefit-risk of withholding IMBRUVICA
for at least 3 to 7 days pre and post-surgery depending upon the type of
surgery and the risk of bleeding.

Infections - Fatal and non-fatal infections have occurred with IMBRUVICA
therapy. At least 25% of patients with MCL and 35% of patients with CLL had
infections Grade 3 or greater NCI Common Terminology Criteria for Adverse
Events (CTCAE). Monitor patients for fever and infections and evaluate
promptly.

Myelosuppression - Treatment-emergent Grade 3 or 4 cytopenias were reported in
41% of patients with MCL and 35% of patients with CLL. These included
neutropenia (29%), thrombocytopenia (17%) and anemia (9%) in patients with MCL
and neutropenia (27%) and thrombocytopenia (10%) in patients with CLL. Monitor
complete blood counts monthly.

Renal Toxicity - Fatal and serious cases of renal failure have occurred with
IMBRUVICA therapy. Treatment-emergent increases in creatinine levels up to 1.5
times the upper limit of normal occurred in 67% of patients with MCL and 23%
of patients with CLL. Increases in creatinine 1.5 to 3 times the upper limit
of normal occurred in 9% of patients with MCL and 4% of patients with CLL.
Periodically monitor creatinine levels. Maintain hydration.

Second Primary Malignancies - Other malignancies have occurred in 5% of
patients with MCL and 10% of patients with CLL who have been treated with
IMBRUVICA. Four percent of patients with MCL, had skin cancers, and 1% had
other carcinomas. Eight percent of patients with CLL had skin cancers and 2%
had other carcinomas.

Embryo-Fetal Toxicity - Based on findings in animals, IMBRUVICA can cause
fetal harm when administered to a pregnant woman. Advise women to avoid
becoming pregnant while taking IMBRUVICA. If this drug is used during
pregnancy or if the patient becomes pregnant while taking this drug, the
patient should be apprised of the potential hazard to a fetus.

ADVERSE REACTIONS –

MCL: The most commonly occurring adverse reactions (≥20%) in the clinical
trial were thrombocytopenia*, diarrhea (51%), neutropenia*, anemia*, fatigue
(41%), musculoskeletal pain (37%), peripheral edema (35%), upper respiratory
tract infection (34%), nausea (31%), bruising (30%), dyspnea (27%),
constipation (25%), rash (25%), abdominal pain (24%), vomiting (23%), and
decreased appetite (21%).

*Treatment-emergent decreases (all grades) of platelets (57%), neutrophils
(47%) and hemoglobin (41%) were based on laboratory measurements and adverse
reactions.

The most common Grade 3 or 4 non-hematological adverse reactions (≥5%) were
pneumonia (7%), abdominal pain (5%), atrial fibrillation (5.4%), diarrhea
(5%), fatigue (5%), and skin infections (5%). Treatment-emergent Grade 3 or 4
cytopenias were reported in 41% of patients. Ten patients (9%) discontinued
treatment due to adverse reactions in the trial (N=111).

The most frequent adverse reaction leading to treatment discontinuation was
subdural hematoma (1.8%). Adverse reactions leading to dose reduction occurred
in 14% of patients.

CLL: The most commonly occurring adverse reactions (≥ 20%) in the clinical
trial were thrombocytopenia*, diarrhea (63%), bruising (54%), neutropenia*,
anemia*, upper respiratory tract infection (48%), fatigue (31%),
musculoskeletal pain (27%), rash (27%), pyrexia (25%), constipation (23%),
peripheral edema (23%), arthralgia (23%), nausea (21%), stomatitis (21%),
sinusitis (21%), and dizziness (21%).

*Treatment-emergent decreases (all grades) of platelets (71%), neutrophils
(54%) and hemoglobin (44%) were based on laboratory measurements per IWCLL
criteria and adverse reactions.

The most common Grade 3 or 4 non-hematological adverse reactions (≥ 5%) were
pneumonia (8%), hypertension (8%), atrial fibrillation (6.3%), sinusitis (6%),
skin infection (6%), dehydration (6.4%), and musculoskeletal pain (6%).
Treatment-emergent Grade 3 or 4 cytopenias were reported in 35% of patients.

Five patients (10%) discontinued treatment due to adverse reactions in the
trial (N=48). These included 3 patients (6%) with infections and 2 patients
(4%) with subdural hematomas. Adverse reactions leading to dose reduction
occurred in 13% of patients.

DRUG INTERACTIONS

CYP3A Inhibitors - Avoid concomitant administration with strong or moderate
inhibitors of CYP3A. If a moderate CYP3A inhibitor must be used, reduce the
IMBRUVICA dose.

CYP3A Inducers - Avoid co-administration with strong CYP3A inducers.

SPECIAL POPULATIONS - Hepatic Impairment - Avoid use in patients with baseline
hepatic impairment.

For the full prescribing information, visit
http://www.imbruvica.com/downloads/Prescribing_Information.pdf

About IMBRUVICA

IMBRUVICA is indicated for the treatment of patients with mantle cell lymphoma
or chronic lymphocytic leukemia who have received at least one prior
therapy.^1 For more information about IMBRUVICA, including the full
prescribing information, please visit www.IMBRUVICA.com. IMBRUVICA is a first
in class, oral therapy and is a new agent that inhibits a protein called
Bruton's tyrosine kinase (BTK).^1 BTK is a key signaling molecule of the
B-cell receptor signaling complex that plays an important role in the survival
and spread of malignant B cells.^8,9,10 IMBRUVICA blocks signals that tell
malignant B cells to multiply and spread uncontrollably.^1,11 It is one of the
first medicines to file for FDA approval via the new Breakthrough Therapy
Designation pathway, enabling Pharmacyclics to rapidly bring this medicine to
patients in need.

To date, 11 Phase III trials have been initiated with ibrutinib and a total of
42 trials are currently registered on www.clinicaltrials.gov. Janssen and
Pharmacyclics entered a collaboration and license agreement in December 2011
to co-develop and co-commercialize IMBRUVICA.

About Pharmacyclics

Pharmacyclics^® is a 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 and control
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.

Pharmacyclics markets IMBRUVICA and 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 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.

Dr. Byrd serves as national principal investigator of this
Pharmacyclics-sponsored clinical study. He has served as an unpaid advisor to
both Pharmacyclics and Janssen in developing the compound ibrutinib. Byrd does
not have a financial interest in either company.

SOURCE Pharmacyclics, Inc.

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