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IMBRUVICA® (ibrutinib) Phase 2 RESONATE-17 Data Show Robust Clinical Activity and Promising Survival Outcomes in Patients with Relapsed or Refractory Chronic Lymphocytic Leukemia with del 17p
Phase 2 data presented at the 2015 American Society of Hematology (ASH) Meeting
This release corresponds to abstract #833

ORLANDO, Fla. and HORSHAM, Pa., Dec. 7, 2015 /PRNewswire/ -- Results from a sub-analysis of the Phase 2 RESONATE-17 (PCYC-1117) study showed IMBRUVICA® (ibrutinib) was associated with robust efficacy and a positive risk-benefit profile in patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have the genetic alteration del 17p. Notably, IMBRUVICA was associated with a high overall response rate (Independent Review Committee [IRC]-assessed ORR; the primary endpoint) and long progression-free survival (PFS) across a variety of baseline genetic characteristics or mutations. These data will be presented in an oral session today at the 2015 American Society of Hematology (ASH) meeting in Orlando, FL. IMBRUVICA is jointly developed and commercialized by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company.

These results will be presented in full by RESONATE-17 study investigator Stephan Stilgenbauer, M.D., Associate Professor, Department of Hematology, Oncology, Rheumatology and Infectious Diseases at the University of Ulm, Germany, during the "CLL: Therapy, excluding Transplantation: Relapsed/Refractory CLL Therapy Excluding Transplantation" session on Monday, December 7 at 5:30 p.m. ET. This sub-analysis was compiled from RESONATE-17 results first presented last year at the ASH 2014 annual meeting. IMBRUVICA was approved by the U.S. Food and Drug Administration (FDA) in July 2014 to treat patients with del 17p CLL.

"CLL patients with the genetic alteration del 17p are considered to be high risk and typically have poor prognoses," said Dr. Stilgenbauer. "IMBRUVICA was the first therapy approved specifically for this difficult-to-treat patient population and the positive data seen in this analysis reinforce its clinical utility in CLL patients with del 17p."

RESONATE-17 is one of the largest dedicated studies conducted in patients with relapsed or refractory CLL with del 17p. The study evaluated 144 previously treated patients with del 17p (137 with CLL, seven with SLL), who received single-agent IMBRUVICA once daily until progression or unacceptable toxicity. A total of 116 patients were determined to have baseline genetic characteristics with the potential to influence treatment outcomes. The primary endpoint of the open-label, single-arm, multi-center trial was ORR, as measured by the IRC. Duration of response (DOR), PFS and safety were key secondary endpoints. At the time of the data assessment, the median treatment duration was 11.1 months and 70 percent of people continued treatment with IMBRUVICA.

After a median follow-up of 11.5 months, the investigator-assessed ORR including partial response with lymphocytosis (PR-L) for all treated patients was 83 percent in the RESONATE-17 trial; specifically, 17 percent of patients experienced a PR-L. While median PFS and OS were not yet reached, the 12-month PFS and OS rates were 79 and 84 percent, respectively. Overall, results were consistent across subgroups with additional baseline characteristics or mutations (e.g., del 11q, del 13q, NOTCH1, TP53, etc.).

"IMBRUVICA was the first therapy to be approved by the FDA specifically for CLL patients with the del 17p genetic alteration. The RESONATE-17 trial is one of the largest prospective trials ever conducted solely in this patient population, whose outcome is usually quite poor when treated with available therapies," said Craig Tendler, M.D., Vice President, Late-Stage Development and Global Medical Affairs for Oncology, Janssen Research & Development. "As we look deeper into the role of IMBRUVICA for the treatment of patients with CLL, we are very encouraged by the durable remissions and well tolerated safety profile achieved with IMBRUVICA in patients with del 17p CLL, in spite of the poor prognosis associated with this genetic alteration."

The most common adverse events (AEs ≥20 percent) of any Grade included diarrhea (36 percent), fatigue (31 percent), cough (24 percent) and arthralgia (22 percent). Serious AEs (SAEs) occurred in 40 percent of patients; 38 percent of all SAEs were Grade 3 or greater.

About IMBRUVICA® (ibrutinib)
IMBRUVICA was one of the first therapies to receive U.S. approval after having received the FDA's Breakthrough Therapy Designation. IMBRUVICA works by blocking a specific protein called Bruton's tyrosine kinase (BTK).1 The BTK protein transmits important signals that tell B cells to mature and produce antibodies and is needed by specific cancer cells to multiply and spread.1,2 IMBRUVICA targets and blocks BTK, inhibiting cancer cell survival and spread.1 For more information, visit www.IMBRUVICA.com.

Additional Information about IMBRUVICA® 
INDICATIONS  
IMBRUVICA® is indicated to treat people with:

  • Chronic lymphocytic leukemia (CLL) who have received at least one prior therapy
  • Chronic lymphocytic leukemia (CLL) with 17p deletion
  • Waldenström's macroglobulinemia (WM)
  • Mantle cell lymphoma (MCL) who have received at least one prior therapy
    • Accelerated approval was granted for this indication based on overall response rate. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trials.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Hemorrhage - Fatal bleeding events have occurred in patients treated with IMBRUVICA®. Grade 3 or higher bleeding events (subdural hematoma, gastrointestinal bleeding, hematuria, and post-procedural hemorrhage) have occurred in up to 6% of patients. Bleeding events of any grade, including bruising and petechiae, occurred in approximately half of patients treated with IMBRUVICA®.

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. Grade 3 or greater infections occurred in 14% to 26% of patients. Cases of progressive multifocal leukoencephalopathy (PML) have occurred in patients treated with IMBRUVICA®. Monitor patients for fever and infections and evaluate promptly.

Cytopenias - Treatment-emergent Grade 3 or 4 cytopenias including neutropenia (range, 19 to 29%), thrombocytopenia (range, 5 to 17%), and anemia (range, 0 to 9%) occurred in patients treated with IMBRUVICA®. Monitor complete blood counts monthly.

Atrial Fibrillation - Atrial fibrillation and atrial flutter (range, 6 to 9%) have occurred in patients treated with IMBRUVICA®, particularly in patients with cardiac risk factors, acute infections, and a previous history of atrial fibrillation. Periodically monitor patients clinically for atrial fibrillation. Patients who develop arrhythmic symptoms (e.g., palpitations, lightheadedness) or new-onset dyspnea should have an ECG performed. If atrial fibrillation persists, consider the risks and benefits of IMBRUVICA® treatment and dose modification.

Second Primary Malignancies - Other malignancies (range, 5 to 14%) including non-skin carcinomas (range, 1 to 3%) have occurred in patients treated with IMBRUVICA®. The most frequent second primary malignancy was non-melanoma skin cancer (range, 4 to 11%).

Tumor Lysis Syndrome - Tumor lysis syndrome has been reported with IMBRUVICA® therapy. Monitor patients closely and take appropriate precautions in patients at risk for tumor lysis syndrome (e.g., high tumor burden).

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
The most common adverse reactions (≥25%) in patients with B-cell malignancies (MCL, CLL, WM) were thrombocytopenia* (57%, 52%, 43%), neutropenia* (47%, 51%, 44%), diarrhea (51%, 48%, 37%), anemia* (41%, 36%, 13%), fatigue (41%, 28%, 21%), musculoskeletal pain (37%, 28%**, NA***), bruising (30%, 12%**, 16%**), nausea (31%, 26%, 21%), upper respiratory tract infection (34%, 16%, 19%), and rash (25%, 24%**, 22%**).

*Based on adverse reactions and/or laboratory measurements (noted as platelets, neutrophils, or hemoglobin decreased).

**Includes multiple ADR terms.

***Not applicable; no associated ADRs.

The most common Grade 3 or 4 non-hematological adverse reactions (≥5%) in MCL patients were pneumonia (7%), abdominal pain (5%), atrial fibrillation (5%), diarrhea (5%), fatigue (5%), and skin infections (5%).
Approximately 6% (CLL), 14% (MCL), and 11% (WM) of patients had a dose reduction due to adverse events.
Approximately 5% (CLL), 9% (MCL), and 6% (WM) of patients discontinued due to adverse events. Most frequent adverse events leading to discontinuation were infections, subdural hematomas, and diarrhea in CLL patients and subdural hematoma (1.8%) in MCL patients.

DRUG INTERACTIONS

CYP3A Inhibitors - Avoid co-administration with strong and moderate CYP3A inhibitors. If a moderate CYP3A inhibitor must be used, reduce the IMBRUVICA® dose.

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

SPECIFIC POPULATIONS

Hepatic Impairment - Avoid use in patients with moderate or severe baseline hepatic impairment. In patients with mild impairment, reduce IMBRUVICA® dose.

Please see full Prescribing Information: http://www.imbruvica.com/downloads/Prescribing_Information.pdf

About Chronic Lymphocytic Leukemia
Chronic Lymphocytic Leukemia is a slow-growing blood cancer that most commonly arises from B cells, a type of white blood cell (lymphocyte) that originates in the bone marrow.3,4 CLL is predominantly a disease of the elderly, with a median age of 71 at diagnosis.3 In CLL, the genetic mutation del 17p occurs when part of chromosome 17 has been lost. CLL patients with del 17p have poor treatment outcomes.5 Del 17p is reported in seven percent of treatment-naive CLL cases,6 with approximately 20 to 40 percent of patients with relapsed or refractory CLL harboring the mutation.7

About Janssen Biotech, Inc.
Janssen Biotech, Inc. redefines the standard of care in immunology, oncology, urology and nephrology. Built upon a rich legacy of innovative firsts, Janssen Biotech has delivered on the promise of new treatments and ways to improve the health of individuals with serious disease. Beyond its innovative medicines, Janssen Biotech is at the forefront of developing education and public policy initiatives to ensure patients and their families, caregivers, advocates and health care professionals have access to the latest treatment information, support services and quality care.

Janssen Biotech, Inc. and Janssen Research & Development, LLC are part of the Janssen Pharmaceutical Companies of Johnson & Johnson. For more information on Janssen Biotech, Inc. or its products, visit http://www.janssen.com. Follow us on Twitter at www.twitter.com/JanssenUS.

Janssen Oncology
Our goal is to fundamentally alter the way cancer is understood, diagnosed and managed, reinforcing our commitment to the patients who inspire us. In looking to find innovative ways to address the cancer challenge, our primary efforts focus on several treatment and prevention solutions. These include a focus on hematologic malignancies, prostate cancer and lung cancer; cancer interception with the goal of developing products that interrupt the carcinogenic process; biomarkers that may help guide targeted, individualized use of our therapies; as well as safe and effective identification and treatment of early changes in the tumor microenvironment. Please visit http://www.janssen.com for more information.

Cautions Concerning Forward-Looking Statements

This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, Janssen Biotech, Inc. and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in new product development, including the uncertainty of clinical success and of obtaining regulatory approvals; competition, including technological advances, new products and patents attained by competitors; challenges to patents; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and description of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 28, 2014, including in Exhibit 99 thereto, and the company's subsequent filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. None of the Janssen Pharmaceutical Companies or Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.


1 IMBRUVICA Prescribing Information, January 2015
2 Genetics Home Reference. Isolated growth hormone deficiency. Available at: http://ghr.nlm.nih.gov/condition/isolated-growth-hormone-deficiency. Accessed December 2015.
3 American Cancer Society. Detailed guide: what is chronic lymphocytic leukemia. Available from: http://www.cancer.org/acs/groups/cid/documents/webcontent/003111-pdf.pdf. Accessed December 2015.
4 Shaffer AL, Rosenwald A, Staudt LM. Lymphoid malignancies: the dark side of B-cell differentiation. Nat Rev Immunol. 2002;2(12):920-932.
5 NCCN Clinical Practice Guidelines in Oncology. Non-Hodgkins Lymphomas. Version 1.2014.http://www.nccn.org/professionals/physician_gls/pdf/nhl.pdf. Accessed November 2015.
6 Schnaiter A, Stilgenbauer S. 17p deletion in chronic lymphocytic leukemia: risk stratification and therapeutic approach. Hematol Oncol Clin North Am. 2013;27:289-301.
7 Stilgenbauer S, Zenz T. Understanding and managing ultra high-risk chronic lymphocytic leukemia. Hematology Am Soc Hematol Educ Program. 2010;2010: 481-8.

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SOURCE Janssen Biotech, Inc.