Mirati Therapeutics To Present At The 30th Annual Piper Jaffray Healthcare Conference

On November 20, 2018 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical stage targeted oncology company, reported that it will participate in a fireside chat at the 30th Annual Piper Jaffray Healthcare Conference in New York on Tuesday, November 27th at 12:30 p.m. EST/ 9:30 a.m. PST. Charles M. Baum, M.D., Ph.D., President and Chief Executive Officer will represent Mirati at the conference (Press release, Mirati, NOV 20, 2018, View Source [SID1234531534]).

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The presentation will be webcast and made available through the "Investors" section of www.mirati.com, and replays will be made available for 90 days following the events.

Ipsen and 3BP announce First Patient Dosed in Phase I/II study for first-in-class radionuclide (IPN01087)

On November 20, 2018 Ipsen (Euronext: IPN; ADR: IPSEY) and 3BP reported the first patient has been dosed in a Phase I/II study for the first-in-class radionuclide 177Lu-IPN01087 (formerly known as 3BP-227) (Press release, Ipsen, NOV 20, 2018, View Source [SID1234531478]). IPN01087 is a compound that targets cancer cells in patients with advanced solid tumors which express the Neurotensin Receptor Subtype 1 (NTSR1).

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The key objective of the Phase I dose-escalation trial (EUDRACT Number 2017-001263-20) is to evaluate the safety and activity, as well as to identify the optimum systemically-administered dose of radiation to treat patients with any of the following solid tumors expressing NTSR1: pancreatic ductal adenocarcinoma, colorectal cancer, gastric cancer, gastrointestinal stromal tumors, Ewing sarcoma and squamous cell carcinoma of the head and neck.

Alexandre Lebeaut, Executive Vice President, R&D and Chief Scientific Officer, Ipsen, said: "Ipsen is committed to bringing to cancer patients innovative systemic radiation therapy with targeted radiopharmaceuticals. We are pleased to report progress of the development of IPN01087 in this Phase I/II study. Our targeted theranostic approach – which we are advancing in partnership with 3B Pharmaceuticals- provides a novel and exciting potential therapeutic solution for unmet medical needs across a number of solid tumours."

"This is a great milestone for IPN01087 and for 3B Pharmaceuticals," said Dr. Ulrich Reineke, Managing Director of 3BP. "We are pleased that the compound is in clinical trials and we remain passionate about systemic radiation therapy and its potential to improve patients’ lives."

DelMar Presents Clinical Update on VAL-083 From Ongoing First- and Second-Line Trials in Patients with MGMT-unmethylated GBM at The Society for NeuroOncology Annual Meeting

On November 20, 2018 DelMar Pharmaceuticals, Inc. (NASDAQ: DMPI) ("DelMar" or the "Company"), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, reported scientific updates, including data from two ongoing clinical trials, at the 23rd Annual Meeting and Education Day of the Society for Neuro-Oncology (SNO) held on November 15-18, 2018 in New Orleans, LA (Press release, DelMar Pharmaceuticals, NOV 20, 2018, View Source [SID1234531494]).

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"In our much-awaited Phase 2 study of VAL-083 in patients with MGMT-unmethylated, Bevacizumab-Naïve Recurrent Glioblastoma Multiforme (rGBM), we are pleased with the accelerated enrollment of this study with the vast majority of subjects already enrolled. What has become amply clear is that in ‎this aggressive tumor type, which can double in size every 6-8 weeks, VAL-083 when used for two or more cycles can stabilize the tumor and slow down its incessant growth," commented Saiid Zarrabian, President and Chief Executive Officer.

"At this time, some subjects are still on drug and others are being followed for survival and we wait to see if this observed stabilization of the tumor favorably impacts median overall survival. The preclinical and clinical efficacy of VAL-083 in MGMT-unmethylated GBM population, along with the 2017 revised NCCN guidelines for MGMT-unmethylated patients which cautions against the use of temozolomide for MGMT-unmethylated GBM patients, creates a therapeutic opportunity not only for newly diagnosed patients, but also in the follow-on maintenance setting currently using temozolomide, all of which signals a path forward for VAL-083," added Mr. Zarrabian.

At the SNO 2018 conference, DelMar provided an update on the company’s ongoing Phase 2 clinical study in a poster entitled "Phase 2 Study of Dianhydrogalactitol (VAL-083) in Patients with MGMT-unmethylated, Bevacizumab-Naïve Recurrent Glioblastoma." This study is being conducted in collaboration with The University of Texas MD Anderson Cancer Center (MDACC). This biomarker-driven trial (testing for MGMT methylation status) is designed to enroll up to 48 patients to determine if VAL-083 treatment improves overall survival compared to historical reference control of 7.15 months with lomustine.

As of October 31, 2018, 44 (of 48) patients have been enrolled
41 of those enrolled have received at least 1 cycle of VAL-083
7 patients are currently receiving treatment; 22 being followed for survival; 19 deceased thus far
Study subjects received a median of 2 cycles of therapy
12 patients received only 1 cycle of VAL-083
2 patients received 1 cycle and are ongoing
27 patients completed 2 or more cycles of therapy
Of the 27 subjects that completed at least 2 cycles of treatment, 9/27 (33.33%) subjects exhibited stable disease (SD) at the end of cycle 2
8/23 initially receiving 40 mg/m2 exhibited SD at the end of cycle 2
1/4 initially receiving 30 mg/m2 exhibited SD at the end of cycle 2
The most prevalent side effect with the 40 mg/m2/day dose of VAL-083 was myelosuppression (thrombocytopenia and neutropenia)
Myelosuppression was also correlated with prolonged (> 5 cycles) prior front-line temozolomide use
in such patients a VAL-083 dose reduction to 30 mg/m2 was found optimal
The Company also provided an update on its Phase 1/2 clinical study in a poster entitled "Phase I/II Study of Dianhydrogalactitol (VAL-083) with Radiation Therapy with Newly Diagnosed MGMT-unmethylated Glioblastoma." This trial is being conducted at the Sun Yat-sen University Cancer Center (SYSUCC) in Guangzhou, China in collaboration with Guangxi Wuzhou Pharmaceutical Company. The trial is designed to enroll up to 30 patients to determine if first-line therapy with VAL-083 treatment, in lieu of first-line temozolomide, improves progression free survival (PFS).

As per the 2017 National Comprehensive Cancer Network (NCCN) guidelines, this trial sets out with the vision of eradicating the unnecessary use of temozolomide in the approximately 60% of GBM patients, as noted in prior studies, with unmethylated MGMT gene promoter
The Company reported that 10 patients have been enrolled as of October 31, 2018
These 10 patients were part of the "3+3" dose escalation cohorts, and were treated with VAL-083 at each different dose on days 1 to 3 of a 21-day cycle along with radiation at 2Gy/day x 5 days for 6 weeks. The same dosing regimen of VAL-083 would be applied during the maintenance stage following six-week chemo-radiation
In the dose-escalation stage, grade 3+ myelosuppression was observed in 2 of 3 patients treated with VAL-083 at 40 mg/m2/day
The lower VAL-083 dose of 30 mg/m2/day was hence moved forward into the expansion phase of the trial
A 20-patient expansion cohort has now commenced enrolling
The primary endpoint of this trial is progression free survival and secondary endpoints include tumor response, overall survival and pharmacokinetics
In addition, DelMar presented three preclinical updates during the conference:

VAL-083 Inhibits Proliferation of a Panel of Eight Glioblastoma Stems Cell Lines: Downregulation of BDR4 as a Novel Anti-Neoplastic Mechanism

In this poster, the authors discuss their preclinical finding that when glioblastoma stem cell lines are treated with VAL-083 there is a downregulation of the transcription activator bromodomain-containing protein 4 (BRD4).

Chromatin remodeling through histone acetylation is a key step in the regulation of the gene expression in both normal and tumor cells. Members of the bromodomain family of proteins, such as BRD4, interact with acetylated histones to assemble chromatin complexes and transcription activators at specific gene promoter sites, including tumor oncogenes. Selective downregulation of bromodomain proteins such as BRD4 by agents such as VAL-083 can therefore inhibit the interaction of BDR4 with acetylated histones at promoter sites, resulting in a reduction of downstream signaling events. Thus, it is possible that VAL-083 may elicit its DNA-damaging action, at least in part, by interrupting chromatin remodeling in cancer cells.

Dianhydrogalactitol (VAL-083) has the Potential to Overcome Major Challenges in the Treatment of Diffuse Intrinsic Pontine Glioma (DIPG)

In this poster, the authors discuss the potential for VAL-083 either as a single-agent, or as part of combination therapy regimens, for the treatment of diffuse intrinsic pontine glioma (DIPG). DIPG is a difficult-to treat, inoperable, rare pediatric brain tumor with very poor prognosis and a dismal survival outlook. In this poster the authors report that VAL-083 is active as a single-agent and synergistic with AZD1775, a Wee1 inhibitor, against DIPG cell lines with varying genetic profile.

Dianhydrogalactitol (VAL-083) Reduces Glioblastoma Tumor Growth In Vivo Upon Bevacizumab-induced Hypoxia

Treatment of GBM with second-line bevacizumab after progression on first-line temozolomide is the standard-of-care for this disease. However, bevacizumab has not only failed to show an improved benefit in these patients, but has also been found to induce intratumor hypoxia, which is then implicated in increased chemoresistance. Preclinically, it has been previously demonstrated that bevacizumab hypoxia upregulates GLUT-1/GLUT-3 glucose transporters. In such a milieu, VAL-083, due to its simple structure, has a unique advantage of enhanced intra-tumoral transport and uptake. The authors seek confirmation of this in-vitro observation in a GBM xenograft model. The data shows that in such mouse models the GBM tumor shrinkage is best when bevacizumab and VAL-083 are administered together compared to when either agent is used as monotherapy.

DelMar’s poster presentations can be viewed on the company’s website at View Source

About VAL-083

VAL-083 (dianhydrogalactitol) is a "first-in-class," bifunctional DNA-targeting agent that introduces interstrand DNA cross-links at the N7-position of guanine leading to DNA double-strand breaks and cancer cell death. VAL-083 has demonstrated clinical activity against a range of cancers including GBM and ovarian cancer in historical clinical trials sponsored by the U.S. National Cancer Institute (NCI). DelMar has demonstrated that VAL-083’s anti-tumor activity is unaffected by common mechanisms of chemoresistance, including MGMT, in cancer cell models and animal studies. Further details regarding these studies can be found at:

View Source.

VAL-083 has been granted orphan drug designations by the U.S. FDA Office of Orphan Products for the treatment of glioma, medulloblastoma and ovarian cancer, and in Europe for the treatment of malignant gliomas. VAL-083 has been granted fast-track status for the treatment of recurrent GBM by the US FDA

Takeda Receives Clearance from the European Commission for the Proposed Acquisition of Shire plc

On November 20, 2018 Takeda Pharmaceutical Company Limited ("Takeda") reported that it has received clearance from the European Commission (the "EC") for the proposed acquisition of Shire plc ("Shire") announced on May 8, 2018 (the "Acquisition") (Press release, Takeda, NOV 20, 2018, View Source [SID1234531512]).

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The EC’s approval is conditional on Takeda and Shire fulfilling commitments given to the EC in connection with the clearance. Specifically, in relation to the future potential overlap in the area of inflammatory bowel diseases between Takeda’s marketed product Entyvio (vedolizumab) and Shire’s pipeline compound SHP647, Takeda and Shire have committed to divest the pipeline compound SHP647 and certain associated rights. The divestment of SHP647 and certain associated rights is not a condition to the completion of the Acquisition.

SHP647 is an exciting pipeline compound and Takeda expects the asset to attract interest from a number of potential buyers. Takeda remains committed to Entyvio, which has been granted marketing authorization in more than 60 countries and is the cornerstone of Takeda’s diverse specialty gastrointestinal portfolio.

"We are very pleased to have secured clearance from the European Commission, the final regulatory approval required to proceed with our acquisition of Shire," said Christophe Weber, President and Chief Executive Officer of Takeda. "We are another step closer to creating a global, values-based, R&D-driven biopharmaceutical leader, and after several months of constructive dialogue, we are optimistic that our shareholders recognize the significant long-term value creation potential of this powerful combination."

The Acquisition has now received clearances from the European Commission, the United States Federal Trade Commission, the Japan Fair Trade Commission, the State Administration for Market Regulation in China and the Brazilian Administrative Council for Economic Defense, among other regulatory authorities.

As announced on November 12, 2018, Takeda has published a circular containing a notice of its decision to hold an Extraordinary General Meeting of Shareholders (the "EGM") to vote on the necessary matters relating to the proposed Acquisition. The EGM is to be convened at 10:00 a.m. (Tokyo time) on December 5, 2018 at INTEX Osaka, Hall 6B Zone.

Takeda also confirms its previously announced expectation that, subject to receiving the necessary shareholder approvals and sanction of the scheme of arrangement by the Jersey court, completion of the Acquisition will take place on January 8, 2019. Further announcements will be made as appropriate.

About Entyvio (vedolizumab)
Vedolizumab is a gut-selective biologic and is approved as an intravenous (IV) formulation.1 It is a humanized monoclonal antibody designed to specifically antagonize the alpha4beta7 integrin, inhibiting the binding of alpha4beta7 integrin to intestinal mucosal addressin cell adhesion molecule 1 (MAdCAM-1), but not vascular cell adhesion molecule 1 (VCAM-1).2 MAdCAM-1 is preferentially expressed on blood vessels and lymph nodes of the gastrointestinal tract.3 The alpha4beta7 integrin is expressed on a subset of circulating white blood cells. These cells have been shown to play a role in mediating the inflammatory process in ulcerative colitis (UC) and Crohn’s disease (CD).4 5 By inhibiting alpha4beta7 integrin, vedolizumab may limit the ability of certain white blood cells to infiltrate gut tissues.

Vedolizumab IV is approved for the treatment of adult patients with moderately to severely active UC and CD, who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a tumor necrosis factor-alpha (TNFα) antagonist. Vedolizumab IV has been granted marketing authorization in over 60 countries, including the United States and European Union, with over 200,000 patient years of exposure to date. 6

Therapeutic Indications

Ulcerative colitis
Vedolizumab is indicated for the treatment of adult patients with moderately to severely active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a tumor necrosis factor-alpha (TNFα) antagonist.

Crohn’s disease
Vedolizumab is indicated for the treatment of adult patients with moderately to severely active Crohn’s disease who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a tumor necrosis factor-alpha (TNFα) antagonist.

Important Safety Information

Contraindications
Hypersensitivity to the active substance or to any of the excipients.

Special warnings and special precautions for use
Vedolizumab should be administered by a healthcare professional equipped to manage hypersensitivity reactions, including anaphylaxis, if they occur. Appropriate monitoring and medical support measures should be available for immediate use when administering vedolizumab. Observe all patients during infusion and until the infusion is complete.

Infusion-related reactions
In clinical studies, infusion-related reactions (IRR) and hypersensitivity reactions have been reported, with the majority being mild to moderate in severity. If a severe IRR, anaphylactic reaction, or other severe reaction occurs, administration of vedolizumab must be discontinued immediately and appropriate treatment initiated (e.g., epinephrine and antihistamines). If a mild to moderate IRR occurs, the infusion rate can be slowed or interrupted and appropriate treatment initiated (e.g., epinephrine and antihistamines). Once the mild or moderate IRR subsides, continue the infusion. Physicians should consider pre-treatment (e.g., with antihistamine, hydrocortisone and/or paracetamol) prior to the next infusion for patients with a history of mild to moderate IRR to vedolizumab, in order to minimize their risks.

Infections
Vedolizumab is a gut-selective integrin antagonist with no identified systemic immunosuppressive activity. Physicians should be aware of the potential increased risk of opportunistic infections or infections for which the gut is a defensive barrier. Vedolizumab treatment is not to be initiated in patients with active, severe infections such as tuberculosis, sepsis, cytomegalovirus, listeriosis, and opportunistic infections until the infections are controlled, and physicians should consider withholding treatment in patients who develop a severe infection while on chronic treatment with vedolizumab. Caution should be exercised when considering the use of vedolizumab in patients with a controlled chronic severe infection or a history of recurring severe infections. Patients should be monitored closely for infections before, during and after treatment. Before starting treatment with vedolizumab, screening for tuberculosis may be considered according to local practice. Some integrin antagonists and some systemic immunosuppressive agents have been associated with progressive multifocal leukoencephalopathy (PML), which is a rare and often fatal opportunistic infection caused by the John Cunningham (JC) virus. By binding to the α4β7 integrin expressed on gut-homing lymphocytes, vedolizumab exerts an immunosuppressive effect on the gut. Although no systemic immunosuppressive effect was noted in healthy subjects, the effects on systemic immune system function in patients with inflammatory bowel disease are not known. Healthcare professionals should monitor patients on vedolizumab for any new onset or worsening of neurological signs and symptoms, and consider neurological referral if they occur. If PML is suspected, treatment with vedolizumab must be withheld; if confirmed, treatment must be permanently discontinued. Typical signs and symptoms associated with PML are diverse, progress over days to weeks, and include progressive weakness on one side of the body, clumsiness of limbs, disturbance of vision, and changes in thinking, memory, and orientation leading to confusion and personality changes. The progression of deficits usually leads to death or severe disability over weeks or months.

Malignancies
The risk of malignancy is increased in patients with ulcerative colitis and Crohn’s disease. Immunomodulatory medicinal products may increase the risk of malignancy.

Prior and concurrent use of biological products
No vedolizumab clinical trial data are available for patients previously treated with natalizumab. Caution should be exercised when considering the use of vedolizumab in these patients. No clinical trial data for concomitant use of vedolizumab with biologic immunosuppressants are available. Therefore, the use of vedolizumab in such patients is not recommended.

Vaccinations
Prior to initiating treatment with vedolizumab all patients should be brought up to date with all recommended immunizations. Patients receiving vedolizumab may receive non-live vaccines (e.g., subunit or inactivated vaccines) and may receive live vaccines only if the benefits outweigh the risks.

Adverse reactions include: nasopharyngitis, headache, arthralgia, upper respiratory tract infection, bronchitis, influenza, sinusitis, cough, oropharyngeal pain, nausea, rash, pruritus, back pain, pain in extremities, pyrexia, and fatigue.

Please consult with your local regulatory agency for approved labeling in your country.

For U.S. audiences, please see the full Prescribing Information including Medication Guide for ENTYVIO.7

For EU audiences, please see the Summary of Product Characteristics (SmPC) for ENTYVIO.

Takeda’s Commitment to Gastroenterology
Gastrointestinal (GI) diseases can be complex, debilitating and life-changing. Recognizing this unmet need, Takeda and our collaboration partners have focused on improving the lives of patients through the delivery of innovative medicines and dedicated patient disease support programs for over 25 years. Takeda aspires to advance how patients manage their disease. Additionally, Takeda is leading in areas of gastroenterology associated with high unmet need, such as inflammatory bowel disease, acid-related diseases and motility disorders. Our GI Research & Development team is also exploring solutions in celiac disease and liver diseases, as well as scientific advancements through microbiome therapies.

ARVINAS TO PRESENT AT THE 30TH ANNUAL PIPER JAFFRAY HEALTHCARE CONFERENCE

On November 20, 2018 Arvinas Inc. (Nasdaq: ARVN), a biotechnology company creating a new class of drugs based on protein degradation, reported that John G. Houston, Ph.D., President and Chief Executive Officer, will participate in a fireside chat at the 30th Annual Piper Jaffray Healthcare Conference on Tuesday, November 27 at 8:00 a.m. ET in New York City (Press release, Arvinas, NOV 20, 2018, View Source [SID1234531495]).

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A live audio webcast of the presentation will be available at www.arvinas.com on the Events page. A replay of the webcast will be archived on the Arvinas website for 30 days following the presentation.