Phase II Ibrutinib Monotherapy Study Shows CLL Patients Achieved Rapid and
Sustainable Disease Control Irrespective of Age or High Risk Prognostic Factor
-Nine Ibrutinib Presentations at the American Association of Cancer Research -
SUNNYVALE, Calif., April 8, 2013
SUNNYVALE, Calif., April 8, 2013 /PRNewswire/ -- Pharmacyclics, Inc. (Nasdaq:
PCYC) today announced results from a Phase II trial of the investigational
oral agent ibrutinib which demonstrated rapid and sustained disease control as
a monotherapy in untreated, relapsed and refractory chronic lymphocytic
leukemia (CLL) patients, irrespective of characteristics that predict poor
outcomes to chemoimmunotherapy.
This study was discussed at today's American Association for Cancer Research
(AACR) annual meeting in Washington, DC together in addition to 8 other
presentations covering advances in clinical and pre-clinical research with
ibrutinib. The Phase II study, which was sponsored by the National Heart, Lung
and Blood Institute, included an analysis of two CLL patient cohorts: the
elderly, above 65 years of age, (n=24, of which 8 were treatment naive) and
the high risk genetic group with a deletion of chromosome 17p (del 17p) (n=29,
of which 15 were treatment naive). Many elderly patients with CLL are unable
to tolerate aggressive therapies. Patients with deletion of chromosome 17p
typically are poor responders to chemoimmunotherapy and have limited treatment
options with no standard of care defined. Of all CLL patients enrolled in this
trial, 72% had been characterized as Rai stage 3-4, indicating an advance
stage, high risk patient population.
The results of the study were presented by lead investigator Adrian Wiestner,
M.D., PhD, Hematology Branch, NHLBI, National Institutes of Health. "Ibrutinib
was highly efficacious as a single agent in patients with untreated, relapsed
and unresponsive CLL, irrespective of their del 17p status," Dr. Wiestner
said. "Responses in this study appear to be durable, and results indicate the
drug is effective against the disease in lymph nodes, spleen and bone marrow.
This is important because existing therapies often fail to effectively
eliminate cancer cells in these tissue sites. Targeted therapy for CLL is
becoming a reality, and this new approach may greatly improve the lives of
patients with this disease."
The study also evaluated in vivo effects of ibrutinib using blood and tissue
samples collected before and during treatment. Ibrutinib demonstrated rapid
and sustained disease control in blood, lymph nodes, spleen and bone marrow.
After 6 months, 95 percent of patients experienced a reduction in lymph node
size and all showed reduction in spleen enlargement, with a median reduction
of 55 percent. In 26 patients, for whom a bone marrow biopsy was done, tumor
infiltration decreased by 82 percent.
The Progression Free Survival probability for these patients at 12 months was
estimated to be 94 percent. Most adverse events were mild and manageable and
included diarrhea, fatigue and rash, severe events occurred in less than 13
percent of patients.
"We are very pleased with the continued research that is being demonstrated at
AACR across 9 different presentations. These advances show the potential
breadth of the ibrutinib program and provide further opportunities for us to
pursue. We are grateful for the continued support of our collaborators,
investigators, patients and shareholders," said Bob Duggan, CEO and chairman
of the board.
Ibrutinib has been designated as a FDA Breakthrough Therapy in CLL patients
with deletion 17p, based on data from completed Phase I/II clinical studies,
where ibrutinib as a monotherapy was used to treat patients with this disease.
Ibrutinib has the potential to improve the outcome in this serious and
life-threatening disease, and may provide a substantial improvement over
existing therapies for this indication.
About Chronic Lymphocytic Leukemia
CLL is the most common type of leukemia, a cancer of the white blood cells.
CLL is a B cell cancer that originates in the bone marrow. Approximately
16,000 patients in the US are diagnosed each year with CLL. The prevalence of
CLL is approximately 113,000 in the US. The disease is a chronic disease of
the elderly with an average survival of about 5 years. Patients commonly
receive multiple lines of treatment over the course of their disease. CLL and
Small lymphocytic lymphoma (SLL) are considered the same underlying disease.
SLL is a disease that is limited to the lymph nodes.
Janssen Biotech, Inc. and Pharmacyclics entered a collaboration and license
agreement in December 2011 to co-develop and co-commercialize 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, 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
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 five Phase III trials have been initiated with
ibrutinib and a total of 26 trials are currently registered on
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 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.
SOURCE Pharmacyclics, Inc.
Contact: Ramses Erdtmann, SVP Investor Relations & Administration,
Press spacebar to pause and continue. Press esc to stop.