Data Presented at American Society of Hematology from Ibrutinib Phase 1b/2 and Phase 2 Trials Suggest High and Durable Response

Data Presented at American Society of Hematology from Ibrutinib Phase 1b/2 and
   Phase 2 Trials Suggest High and Durable Response in Chronic Lymphocytic
                                   Leukemia

PR Newswire

ATLANTA, Dec. 8, 2012

ATLANTA, Dec. 8, 2012 /PRNewswire/ -- Pharmacyclics, Inc. (NASDAQ: PCYC) today
announced follow-up results that its investigational oral therapy ibrutinib
(PCI-32765), as a single agent, resulted in durable responses for patients
over 65 years of age with treatment-naive chronic lymphocytic leukemia (CLL)
or small lymphocytic lymphoma (SLL) and for those with relapsed/refractory
(RR) or high-risk (HR) disease.

In a study of 116 patients, the overall response rate (ORR) was 68 percent in
elderly treatment-naive (TN) patients with an estimated 96 percent
progression-free survival (PFS) rate at 26 months. In patients with RR
CLL/SLL, including those with HR disease, the ORR was 71 percent with an
estimated PFS at 26 months of 75 percent.

A second study in 40 patients with HR CLL treated with the combination of
ibrutinib and rituximab therapy reported an ORR of 83 percent, with 38 of the
40 patients continuing on therapy without disease progression. Patients with
HR disease have inferior responses to standard chemo-immunotherapy and shorter
rates of PFS and OS.

These findings, from ongoing Phase 1b/2 and Phase 2 trials, were presented in
a press briefing today at the 54^th annual meeting of the American Society of
Hematology in Atlanta, GA. CLL and SLL are common B-cell non-Hodgkin
lymphomas.

In describing the findings of the Phase 1b/2 ibrutinib monotherapy trial, lead
investigator John C. Byrd, M.D., said, "We are very encouraged that these
results with ibrutinib continue to support the possibility that we can address
some of the critical unmet needs in CLL/SLL." Dr. Byrd is the D. Warren Brown
Chair of Leukemia Research and Director, Division of Hematology at Ohio State
University Comprehensive Cancer Center — Arthur G. James Cancer Hospital and
Richard J. Solove Research Institute (OSUCCC — James).

In his presentation, Dr. Byrd noted that current therapies have a number of
limitations in terms of how long they can be used in a patient and their
tolerability, especially in elderly patients. "Further, virtually all patients
relapse, and there are few salvage regimens that produce durable remissions,"
he pointed out.

The Phase 2 trial, evaluating ibrutinib in combination with rituximab, was led
by Jan A. Burger, M.D., Ph.D., associate professor, Department of Leukemia,
Division of Cancer Medicine, The University of Texas MD Anderson Cancer
Center, Houston, TX.

"We are very excited by the response rates we saw with ibrutinib-rituximab
combination therapy, and believe they emphasize the need for rapid, further
development of ibrutinib, especially for patients with high-risk CLL/SLL,"
said Dr. Burger.

Studies and Findings
Oral presentation 189, The Bruton's Tyrosine Kinase (BTK) inhibitor ibrutinib
(PCI-32765) promotes high response rate, durable remissions, and is tolerable
in treatment naive (TN) and relapsed or refractory (RR) chronic lymphocytic
leukemia (CLL) or small lymphocytic lymphoma (SLL) patients including patients
with high-risk (HR) disease: New and updated results of 116 patients in a
phase Ib/II study
John C. Byrd, M.D., The Ohio State University, Columbus, OH

This presentation was based on findings from a Phase 1b/2, multi-center trial
with 116 patients with TN (n=31), RR (n=61) or HR (n=24) CLL/SLL. Patients
were treated with oral ibrutinib monotherapy, either 420 mg or 840 mg daily.
Patients with RR disease had received at least two prior therapies; patients
with HR disease had relapsed within two years following chemo-immunotherapy.
The study objectives were to determine the ORR, PFS, overall safety (OS),
safety of the two dosing regimens and pharmacokinetics/pharmacodynamics.

  oThe overall response rates were:

       o68 percent in TN patients, all of whom were over 65 years old, after
         a median follow up of 20.3 months
       o71 percent in patients with RR disease, including patients with HR
         CLL/SLL, after a median follow up of 15.7 months

  oThe estimated PFS at 26 months is 96 percent for patients over 65 years of
    age with TN CLL and 75 percent for those with RR/HR disease

       oEstimated OS at 26 months is 96 percent in TN patients and 83 percent
         in patients with RR/HR disease

Responses were independent of high-risk clinical or genetic features, such as
a deletion of part of chromosome 17 (del17p).

Most adverse events (AEs) were Grade 1 or 2 in severity, with the most common
attributed to ibrutinib being diarrhea, fatigue, nausea and rash. Adverse
hematologic events were relatively infrequent. In patients with cytopenia (low
blood cell counts) at the beginning of the study, sustained improvements of
platelet counts (78 percent) and hemoglobin (82 percent) were seen after
treatment. There was no evidence of cumulative toxicity or long-term safety
concerns.

Oral presentation 187, The BTK inhibitor ibrutinib (PCI-32765) in combination
with rituximab is well tolerated and displays profound activity in high-risk
chronic lymphocytic leukemia (CLL) patients
Jan A. Burger, M.D., Ph.D., MD Anderson Cancer Center, Houston, TX

This presentation was based on findings from a Phase 2, single-center trial
with 40 patients with HR CLL treated with 420 mg/day ibrutinib in combination
with rituximab, a current standard CLL therapy. Patients with HR disease were
previously treated and had one of the following characteristics: deletion of a
part of chromosome 17 (del17p), which is associated with poorer treatment
outcomes; a gene mutation called TP53; del11q, another partial chromosome
deletion associated with poorer outcomes; or a short remission duration (less
than three years) after first-line chemo-immunotherapy.

The results after a follow-up of three to six months are:

  oThe ORR was 83 percent
  o38 of 40 patients continue on therapy without disease progression
  o95 percent of all patients and 90 percent of patients with del17p had not
    progressed
  oThere was a large and rapid reduction in lymph node and spleen sizes, with
    84 percent of patients (26/31) experiencing more than a 50 percent
    decrease in lymph node size
  oTreatment was well tolerated overall, with Grade 3 or Grade 4 toxicities
    infrequent and transient in nature, including febrile neutropenia (fever
    with low white blood cell count), anemia, mucositis and pneumonia, in this
    clinical study

"We believe the updated findings from these two trials further affirm the
possibilities of ibrutinib," said Bob Duggan, CEO and Chairman of the Board of
Directors of Pharmacyclics. "We look forward to continuing our clinical
development program for this therapy, and hope it can help us in achieving
Pharmacyclics' mission of improving the quality and duration of life for
patients with oncologic diseases."

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 –
regulating B-cell apoptosis, cell adhesion, and lymphocyte migration and
homing. Data are consistent with a mechanistic model whereby ibrutinib blocks
BCR signaling, which drives cells into apoptosis and/or disrupts cell
migration and adherence to tumor-protective microenvironments.

The effectiveness of ibrutinib alone or in combination with other treatments
is being studied in several B-cell malignancies, including CLL/SLL,
relapsed/refractory mantle cell lymphoma, diffuse large B-cell lymphoma,
follicular lymphoma and multiple myeloma. A comprehensive late stage Phase 2
and 3 program is under way.

Conference Call and Webcast Details
Pharmacyclics will be holding a conference call on Wednesday, December 12,
2012 at 8:30 AM ET. 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 http://ir.pharmacyclics.com/events.cfm. The archived
version of the webcast will be available for 30 days on the Investor Relations
section of the company's website at www.pharmacyclics.com.

About the Pharmacyclics and Janssen Collaboration
Pharmacyclics and Janssen Biotech, Inc. entered into a worldwide collaboration
on December 8, 2011, to develop and commercialize ibrutinib. Following
regulatory approval, Pharmacyclics and Janssen will co-commercialize
ibrutinib. Each company will lead development for specific indications as
stipulated in a global development plan.

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 medial 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. We are committed to
high standards of ethics, scientific rigor, and operational efficiency as we
move 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.

Note: Data in this release correspond to ASH Abstracts 189 and 187.

SOURCE Pharmacyclics, Inc.

Website: http://www.pharmacyclics.com
Contact: Ramses Erdtmann, Vice President, Investor Relations and Finance,
+1-408-215-3325
 
Press spacebar to pause and continue. Press esc to stop.