Phase 3 Study Evaluating IMBRUVICA® (ibrutinib) in Combination with Rituximab in Waldenström’s Macroglobulinemia Met Primary Endpoint

On December 5, 2017 Janssen Research & Development, LLC (Janssen) reported that the Phase 3 iNNOVATE (PCYC-1127) study evaluating IMBRUVICA (ibrutinib) in combination with rituximab (RITUXAN) in relapsed/refractory and treatment-naïve patients with Waldenström’s macroglobulinemia (WM) successfully met its primary endpoint of progression-free survival (PFS) (Press release, Johnson & Johnson, DEC 5, 2017, View Source [SID1234522391]). An Independent Data Monitoring Committee (IDMC) recommended unblinding iNNOVATE based on efficacy results observed in the pre-specified interim analysis. IMBRUVICA is a first-in-class Bruton’s tyrosine kinase (BTK) inhibitor jointly developed and commercialized by Janssen Biotech, Inc., and Pharmacyclics LLC, an AbbVie company.

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"It is gratifying to see that patients with Waldenström’s macroglobulinemia – a rare, difficult-to-treat form of blood cancer – have achieved this magnitude of benefit with the IMBRUVICA combination with rituximab as compared to rituximab alone in either the relapsed/refractory or newly diagnosed setting," said Craig Tendler, M.D., Vice President, Late-Stage Development and Global Medical Affairs, Janssen Oncology. "Since the approval of IMBRUVICA in 2013 for Waldenström’s, we have added to the body of evidence and patient experience, by conducting this randomized Phase 3 trial and confirming IMBRUVICA’s clinical benefit first seen in the relapsed/refractory setting and now demonstrated with earlier use in the treatment journey for the WM patient."

Janssen and Pharmacyclics will share and discuss the unblinded data from the study with health authorities around the world. IMBRUVICA received FDA approval in WM in January 2015.

About iNNOVATE
iNNOVATE is a Pharmacyclics-sponsored, randomized, placebo-controlled, double-blind, Phase 3 study, which is evaluating IMBRUVICA (ibrutinib) in combination with rituximab, and placebo in combination with rituximab in 150 patients with relapsed/refractory and treatment-naïve Waldenström’s macroglobulinemia (WM). In the study, patients were randomized to receive intravenous rituximab 375 mg/m2 once weekly for four consecutive weeks, followed by a second four-weekly rituximab course following a three-month interval. All patients received either IMBRUVICA 420 mg or placebo once daily continuously until criteria for permanent discontinuation were met. The primary endpoint is progression-free survival, with secondary objectives including overall response rate, hematological improvement measured by hemoglobin, time-to-next treatment, overall survival, and number of participants with adverse events as a measure of safety and tolerability within each treatment arm. WM is a form of non-Hodgkin’s lymphoma and is a rare disease, with an incidence rate of about three cases per million per year in the U.S. Roughly 1,000 to 1,500 people are diagnosed with WM each year in the U.S.1

About IMBRUVICA
IMBRUVICA (ibrutinib) was one of the first therapies to receive U.S. approval after having received the FDA’s Breakthrough Therapy Designation.2 IMBRUVICA works by blocking a specific protein called Bruton’s tyrosine kinase (BTK). 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.3 IMBRUVICA targets and blocks BTK, inhibiting the survival and spread of cancer cells, and impacting signaling associated with other serious conditions. Worldwide, IMBRUVICA was used to treat more than 90,000 patients to date. For more information, visit www.IMBRUVICA.com.

Additional Information about IMBRUVICA
INDICATIONS
IMBRUVICA is indicated to treat adults with2

Chronic lymphocytic leukemia (CLL)/Small lymphocytic lymphoma (SLL)
Chronic lymphocytic leukemia (CLL)/Small lymphocytic lymphoma (SLL) with 17p deletion
Waldenström’s macroglobulinemia (WM)
Mantle cell lymphoma (MCL) patients 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 and description of clinical benefit in a confirmatory trial.
Marginal zone lymphoma (MZL) patients who require systemic therapy and have received at least one prior anti-CD20-based therapy
Accelerated approval was granted for this indication based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Chronic Graft-Versus-Host Disease (cGVHD) patients who failed one or more lines of systemic therapy
IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Hemorrhage: Fatal bleeding events have occurred in patients treated with IMBRUVICA. Grade 3 or higher bleeding events (intracranial hemorrhage [including 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 and patients should be monitored for signs of bleeding.

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 (including bacterial, viral, or fungal) have occurred with IMBRUVICA therapy. Grade 3 or greater infections occurred in 14% to 29% of patients. Cases of progressive multifocal leukoencephalopathy (PML) and Pneumocystis jirovecii pneumonia (PJP) have occurred in patients treated with IMBRUVICA. Consider prophylaxis according to standard of care in patients who are at increased risk for opportunistic infections.

Monitor and evaluate patients for fever and infections and treat appropriately.

Cytopenias: Treatment-emergent Grade 3 or 4 cytopenias including neutropenia (range, 13 to 29%), thrombocytopenia (range, 5 to 17%), and anemia (range, 0 to 13%) based on laboratory measurements occurred in patients with B-cell malignancies treated with single agent 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, hypertension, 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. Atrial fibrillation should be managed appropriately, and if it persists, consider the risks and benefits of IMBRUVICA treatment and follow dose modification guidelines.

Hypertension: Hypertension (range, 6 to 17%) has occurred in patients treated with IMBRUVICA with a median time to onset of 4.6 months (range, 0.03 to 22 months). Monitor patients for new onset hypertension or hypertension that is not adequately controlled after starting IMBRUVICA. Adjust existing anti-hypertensive medications and/or initiate anti-hypertensive treatment as appropriate.

Second Primary Malignancies: Other malignancies (range, 3 to 16%) including non-skin carcinomas (range, 1 to 4%) have occurred in patients treated with IMBRUVICA. The most frequent second primary malignancy was non-melanoma skin cancer (range, 2 to 13%).

Tumor Lysis Syndrome: Tumor lysis syndrome has been infrequently reported with IMBRUVICA therapy. Assess the baseline risk (e.g., high tumor burden) and take appropriate precautions. Monitor patients closely and treat as appropriate.

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 and for 1 month after cessation of therapy. 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. Advise men to avoid fathering a child during the same time period.

ADVERSE REACTIONS

B-cell malignancies: The most common adverse reactions (≥20%) in patients with B-cell malignancies (MCL, CLL/SLL, WM and MZL) were thrombocytopenia (62%), neutropenia (61%), diarrhea (43%), anemia (41%), musculoskeletal pain (30%), rash (30%), bruising (30%), nausea (29%), fatigue (29%), hemorrhage (22%), and pyrexia (21%).

The most common Grade 3 or 4 adverse reactions (≥5%) in patients with B-cell malignancies (MCL, CLL/SLL, WM and MZL) were neutropenia (39%), thrombocytopenia (16%), and pneumonia (10%).

Approximately 6% (CLL/SLL), 14% (MCL), 11% (WM) and 10% (MZL) of patients had a dose reduction due to adverse reactions. Approximately 4%-10% (CLL/SLL), 9% (MCL), and 9 % (WM [6%] and MZL [13%]) of patients discontinued due to adverse reactions.

cGVHD: The most common adverse reactions (≥20%) in patients with cGVHD were fatigue (57%), bruising (40%), diarrhea (36%), thrombocytopenia (33%), muscle spasms (29%), stomatitis (29%), nausea (26%), hemorrhage (26%), anemia (24%), and pneumonia (21%).

The most common Grade 3 or 4 adverse reactions (≥5%) reported in patients with cGVHD were fatigue (12%), diarrhea (10%), neutropenia (10%), pneumonia (10%), sepsis (10%), hypokalemia (7%), headache (5%), musculoskeletal pain (5%), and pyrexia (5%).

Twenty-four percent of patients receiving IMBRUVICA in the cGVHD trial discontinued treatment due to adverse reactions. Adverse reactions leading to dose reduction occurred in 26% of patients.

DRUG INTERACTIONS

CYP3A Inducers: Avoid coadministration with strong CYP3A inducers.

CYP3A Inhibitors: Dose adjustment may be recommended.

SPECIFIC POPULATIONS

Hepatic Impairment (based on Child-Pugh criteria): Avoid use of IMBRUVICA in patients with moderate or severe baseline hepatic impairment. In patients with mild impairment, reduce IMBRUVICA dose.

Please see Full Prescribing Information: View Source

Verastem to Present Results from Pivotal Phase 3 DUO Study in Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma at a Research and Development Event at ASH 2017

On December 5, 2017 Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to improve the survival and quality of life of cancer patients, will present clinical data from the Phase 3 DUO study evaluating the efficacy and safety of duvelisib in patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) at a Research and Development reception at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2017 Annual Meeting (Press release, Verastem, DEC 5, 2017, View Source;p=RssLanding&cat=news&id=2321050 [SID1234522395]). The event will take place on Sunday, December 10, 2017 in Atlanta.

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The event, which follows the oral presentation of the DUO data results at ASH (Free ASH Whitepaper), will feature a slide presentation and moderated panel discussion with recognized experts in the treatment of hematologic malignancies, including CLL/SLL, and a live Q&A session. Speakers will include Ian Flinn, MD, PhD, Sarah Cannon Research Institute, who will present results from the Phase 3 DUO study, and Steven Horwitz, MD, Memorial Sloan Kettering Cancer Center, who will provide an update on duvelisib for the treatment of Peripheral T-Cell Lymphoma (PTCL). In addition, Lori Kunkel, MD, Verastem Clinical and Scientific Advisory Board, and Steven Bloom, Verastem Chief Strategy Officer, will participate in the discussion panel and Q&A session, which will be moderated by Robert Forrester, Verastem President and Chief Executive Officer.

The event will take place during the ASH (Free ASH Whitepaper) 2017 Annual Meeting and is open to analysts and institutional investors. Interested parties can access a live webcast of the event beginning Sunday, December 10, 2017 at 8:15 p.m. ET on the "Presentations" page of the company’s website View Source;p=irol-calendar. A replay of the webcast will be archived on the company’s website for 90 days following the event. For more information or to RSVP, please contact Maeve Conneighton at [email protected].
Duvelisib is Verastem’s first in class oral dual inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma which is currently being developed for the treatment of CLL/SLL, peripheral T cell lymphoma (PTCL), and other hematologic malignancies.

Moleculin’s WP1066 Drug gets FDA Brain Tumor IND Clearance

On December 5, 2017 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the MD Anderson Cancer Center ("MD Anderson"), reported the physician-sponsored Investigational New Drug ("IND") application for a Phase I trial of Moleculin’s drug WP1066 in patients with recurrent malignant glioma and brain metastasis from melanoma has been allowed by the US Food and Drug Administration ("FDA") (Press release, Moleculin, DEC 5, 2017, View Source [SID1234522382]).

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"We are so pleased to now have a second drug enter the clinical stage," commented Walter Klemp, Chairman and CEO of Moleculin. "We believe WP1066 represents a new class of anticancer drugs able to fight tumors on two fronts by directly inhibiting cell signaling supporting tumor activity, and independently stimulating a natural immune response. This constitutes a new approach to treating brain tumors and tumor metastasis to the brain.

Mr. Klemp concluded, "Since the discovery of WP1066 at MD Anderson by Prof. Waldemar Priebe, it has now been studied by many independent groups and is widely recognized as a potent inhibitor of the activated form of a protein called STAT3, which has been implicated in many difficult to treat tumors, including brain tumors. Animal studies have shown that inhibition of STAT3 directly blocks tumor proliferation and its survival, while most importantly boosting the immune system’s ability to fight cancer. We finally have our first opportunity for a clinical proof of concept and confirmation of promising preclinical activity."
This IND was sponsored by Dr. Amy Heimberger, who will serve as the principal investigator for the Phase I trial at MD Anderson Cancer Center to evaluate safety and efficacy. Details about the trial can be viewed on www.clinicaltrials.gov.

Moleculin Biotech, Inc. Investor Presentation

On December 5, 2017 Moleculin Biotech, Inc. presented Investor Presentation (Presentation, Moleculin, DEC 5, 2017, View Source [SID1234522381]).

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Medidata Expands Partnership with CytomX Therapeutics to Centralize Management of Regulated and Nonregulated Content

On December 5, 2017 Medidata (NASDAQ:MDSO), the leading global provider of cloud-based technology and data analytics for clinical research, reported that CytomX Therapeutics, Inc., a biopharmaceutical company developing investigational Probody therapeutics for the treatment of cancer, will expand its partnership to include Medidata Regulated Content Management (RCM) solutions, eTMF Archive and SOP Management (Press release, CytomX Therapeutics, DEC 5, 2017, View Source [SID1234522418]).

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"We’ve created a seamless offering with the Medidata platform that can facilitate everything during clinical research, from data collection and management to compliance"
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With the addition of Medidata RCM, CytomX will now manage regulated and nonregulated content in a single, unified platform. Accessible through the Medidata Clinical Cloud, CytomX will integrate standard operating procedure (SOP) management and electronic trial master file (eTMF) archive to manage all content, data and workflows.

CytomX is also currently using Medidata Rave, the world’s leading solution for capturing, managing and reporting patient data and Medidata Balance, randomization and trial supply management (RTSM) application, for its oncology research studies.

"We’ve created a seamless offering with the Medidata platform that can facilitate everything during clinical research, from data collection and management to compliance," said Mike Capone, chief operating officer at Medidata. "Medidata’s industry difference is that we enable our customers to take control of their data, helping them meet regulatory requirements and ensure efficient clinical trials."