BioMarin to Host Second Quarter 2018 Financial Results Conference Call and Webcast on Thursday, August 2 at 4:30pm ET

On July 11, 2018 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Thursday, August 2, at 4:30 p.m. ET to discuss second quarter 2018 financial results and provide a general business update (Press release, BioMarin, JUL 11, 2018, View Source [SID1234527654]).

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Dial-in Number

U.S. / Canada Dial-in Number: (866) 502-9859

International Dial-in Number: (574) 990-1362

Conference ID: 3364409

Replay Dial-in Number: (855) 859-2056

Replay International Dial-in Number: (404) 537-3406

Conference ID: 3364409

Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, www.biomarin.com. A replay of the call will be archived on the site for one week following the call.

BioSight Launches a Phase 2b Clinical Trial of BST-236 as a First-Line Treatment of Acute Myeloid Leukemia

On July 11, 2018 BioSight Ltd., a pharmaceutical development company focused on the development of targeted oncology drugs, reported that it has received the FDA and the Israeli Ministry of Health clearance to launch a Phase 2b clinical trial of BST-236 for treatment of Acute Myeloid Leukemia (AML) (Press release, BioSight, JUL 11, 2018, View Source [SID1234527653]).

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The trial, which will be launched in the upcoming month, will be conducted in 25 medical centers in the US and Israel. BST-236 will be administered as a single agent treatment for newly-diagnosed AML patients, either de novo or secondary to myelodysplastic disorder (MDS) who are unfit for standard chemotherapy due to its severe toxicity. This population is estimated to account for a third to half of the AML patients.

In the Phase 1/2 study, presented at the Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting and Exposition on December 2017, 26 acute leukemia patients were treated with BST-236 as a single agent. The study enrolled mainly older patients with poor prognosis baseline characteristics, including prior treatment with hypomethylating agents for MDS. BST-236 was found to be safe and well tolerated at high doses, with no neurological or gastrointestinal toxicities or renal failure, all of which are life-threatening toxicities associated with the existing chemotherapy and which often attenuate or prevent its use in older patients. This encouraging safety profile allowed the treatment of older and medically unfit patients with high doses of BST-236 and led to 2-3-fold higher response rates compared to currently approved treatments for this patient population. The aim of the Phase 2b study is to repeat the results of the Phase 1/2 study in a larger number of patients in the US and Israel.

Dr. Ruth Ben Yakar, BioSight’s CEO said: "We are excited to launch this Phase 2b clinical trial of BST-236 for treatment of newly-diagnosed AML patients who are unfit for standard chemotherapy. The encouraging results of the Phase 1/2 study suggest that BST-236 may serve as an improved treatment option compared to the approved drugs available today for this population, including for patients at the age of 75 years or more. We think it is very important to approach these patients, mainly due to the general increase in the population’s age and quality of life, and we believe we can provide them with a safer and more effective treatment."

About BST-236

BST-236 is a novel pro-drug of the chemotherapeutic drug cytarabine. Cytarabine has been the backbone of first-line therapy for AML for the past 40 years, however, it is associated with severe bone marrow, gastrointestinal, and neurological toxicities which significantly limit its use, especially in older and medically unfit patients. BST-236 is designed to enable delivery of high cytarabine doses to leukemia cells with lower systemic exposure to the free drug and relative sparing of normal tissues. As such, BST-236 may serve as an ideal therapy for leukemia, particularly for delivering high doses of cytarabine to medically unfit or older adults.

Quest Diagnostics To Release Second Quarter 2018 Financial Results On July 24

On July 11, 2018 Quest Diagnostics Incorporated (NYSE: DGX), the world’s leading provider of diagnostic information services, reported that it will report second quarter 2018 results on Tuesday, July 24, 2018, before the market opens (Press release, Quest Diagnostics, JUL 11, 2018, View Source [SID1234527651]). It will hold its quarterly conference call to discuss the results beginning at 8:30 a.m. Eastern Time on that day.

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The conference call can be accessed in listen-only mode by dialing 773-756-0467, passcode 3214469. The company suggests participants dial in approximately 10 minutes before the call.

A replay of the call may be accessed online at www.QuestDiagnostics.com/investor or by phone at 866-483-9044 for domestic callers or 203-369-1586 for international callers. Telephone replays will be available from approximately 10:30 a.m. Eastern Time on July 24, 2018 until midnight Eastern Time on August 7, 2018.

Anyone listening to the call is encouraged to read the company’s periodic reports on file with the Securities and Exchange Commission, including the discussion of risk factors and historical results of operations and financial condition in those reports.

Janssen Provides Update on IMBRUVICA® (ibrutinib) Phase 3 PHOENIX Trial in Newly Diagnosed Non-Germinal Center B Cell (Non-GCB) Subtype of Diffuse Large B-Cell Lymphoma (DLBCL)

On July 11, 2018 The Janssen Pharmaceutical Companies of Johnson & Johnson reported that topline results from the Phase 3 PHOENIX trial evaluating the investigational use of IMBRUVICA (ibrutinib) in the treatment of newly diagnosed non-Germinal Center B cell (non-GCB) subtype of diffuse large B-cell lymphoma (DLBCL), an aggressive form of non-Hodgkin lymphoma (NHL) (Press release, Johnson & Johnson, JUL 11, 2018, View Source [SID1234527650]). The study compared IMBRUVICA plus rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) versus R-CHOP, the current standard of care in DLBCL. The clinical trial did not meet the primary endpoint of event-free survival in patients with non-GCB subtype of DLBCL, including activated B cell-like (ABC) subtype of DLBCL. Janssen is conducting further analyses of the PHOENIX study results to assess the potential efficacy of IMBRUVICA as observed in a patient sub-population. IMBRUVICA, a first-in-class Bruton’s tyrosine kinase (BTK) inhibitor, is jointly developed and commercialized by Janssen Biotech, Inc., and Pharmacyclics LLC, an AbbVie company.

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"While we are disappointed that the overall study did not result in the outcome that we had hoped for in patients living with DLBCL, we are conducting additional analyses to further understand potential benefits we have observed in a sub-population of patients," said Craig Tendler, M.D., Vice President, Clinical Development and Global Medical Affairs, Janssen Research & Development, LLC. "These analyses will be informative in preparation for upcoming consultations with health authorities. We look forward to these regulatory discussions and to submitting the PHOENIX data for presentation at a major medical conference later this year."

DLBCL is a fast-growing type of NHL that affects B-cell lymphocytes, a type of white blood cell.1 DLBCL is the most common form of NHL in adults, accounting for up to 40 percent of all cases worldwide.2 DLBCL can arise in lymph nodes or outside of the lymphatic system, in the gastrointestinal tract, testes, thyroid, skin, breast, bone, or brain.1 There are two main subtypes of DLBCL: GCB subtype and non-GCB subtype, which includes unspecific subtype and ABC subtype.3 The disease occurs more commonly in the elderly and is slightly more prevalent in men.1

About PHOENIX

PHOENIX (NCT01855750) is a randomized, double-blind, placebo-controlled, multicenter, Phase 3 study comparing the efficacy and safety of ibrutinib in combination with R-CHOP versus placebo in combination with R-CHOP in patients with newly diagnosed non-GCB subtype of DLBCL, including ABC subtype of DLBCL. In the study, patients were randomized in a 1:1 ratio to receive either placebo plus R-CHOP or 560 mg ibrutinib plus R-CHOP stratified by Revised International Prognostic Index, geographic region, and number of pre-specified treatment cycles (6 vs. 8 cycles). The primary endpoint was event-free survival, defined as the time interval from randomization to the date of disease progression, relapse from complete response as assessed by the investigator, initiation of systemic anti-lymphoma therapy for either positron emission tomography-positive or biopsy-proven residual disease upon completion of at least 6 cycles of R-CHOP therapy, or death, whichever occurred first.

About IMBRUVICA

IMBRUVICA (ibrutinib) was one of the first therapies to receive U.S. approval after having received the FDA’s Breakthrough Therapy Designation.4 IMBRUVICA works by blocking a protein called Bruton’s tyrosine kinase (BTK). The BTK protein transmits important signals that cause B cells to mature and produce antibodies and is needed by specific cancer cells to multiply and spread.5 IMBRUVICA targets and blocks BTK, inhibiting the survival and spread of cancer cells, and impacting signaling associated with other serious conditions. Worldwide, IMBRUVICA has been used to treat more than 100,000 patients to date. For more information, visit www.IMBRUVICA.com.

Additional Information about IMBRUVICA

INDICATIONS

IMBRUVICA is indicated to treat adults with4

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.

Cardiac Arrhythmias: Fatal and serious cardiac arrhythmias have occurred with IMBRUVICA therapy. Grade 3 or greater ventricular tachyarrhythmias occurred in 0 to 1% of patients, and Grade 3 or greater atrial fibrillation and atrial flutter occurred in 0 to 6% of patients. These events have occurred particularly in patients with cardiac risk factors, hypertension, acute infections, and a previous history of cardiac arrhythmias.

Periodically monitor patients clinically for cardiac arrhythmias. Obtain an ECG for patients who develop arrhythmic symptoms (e.g., palpitations, lightheadedness, syncope, chest pain) or new onset dyspnea. Manage cardiac arrhythmias 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%), bruising (30%), rash (30%), fatigue (29%), nausea (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%)*, stomatitis (29%), muscle spasms (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.

*Treatment-emergent decreases (all grades) were based on laboratory measurements and adverse reactions.

DRUG INTERACTIONS

CYP3A Inhibitors: Dose adjustment may be recommended.

CYP3A Inducers: Avoid coadministration with strong CYP3A inducers.

SPECIFIC POPULATIONS

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

FDA Accepts Biologics License Application For Subcutaneous Formulation Of Herceptin

On July 11, 2018 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported that the U.S. Food and Drug Administration (FDA) has accepted a Biologics License Application from Genentech, a member of the Roche Group, for a subcutaneous (SC) formulation of trastuzumab (Herceptin) in its FDA-approved breast cancer indications (Press release, Halozyme, JUL 11, 2018, View Source [SID1234527649]). This is a co-formulation with Halozyme’s proprietary recombinant human hyaluronidase enzyme (ENHANZE technology), which is approved and marketed under the Herceptin SC brand in many countries outside the U.S.

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"We are excited to see Genentech and Roche taking steps to bring a subcutaneous formulation of trastuzumab to patients in the United States," said Dr. Helen Torley, president and chief executive officer. "If approved, this formulation would provide a new treatment administration option for patients and health care practitioners."

Roche reported total 2017 sales of Herceptin in the United States of 2.7 billion CHF. Sales of subcutaneous trastuzumab will be dependent on market adoption.

Herceptin (trastuzumab) is a registered trademark of Genentech, a member of the Roche Group.