On September 27, 2018 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a clinical stage biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that it has successfully completed a study to determine the best assay method for the accurate determination of the number of viable encapsulated cells at any given time inside PharmaCyte’s Cell-in-a Box capsules, which will be used for the treatment of locally advanced, non-metastatic, inoperable pancreatic cancer (LAPC) (Press release, PharmaCyte Biotech, SEP 27, 2018, View Source [SID1234529632]).

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The decision on which viability assay to use is an important component required for the filing of PharmaCyte’s Investigational New Drug Application (IND) with the U.S. Food and Drug Administration (FDA), since the viable cell assay is needed to show how many living cells are in the capsules at any given moment. This is a parameter that influences the biological activity of PharmaCyte’s Cell-in-a-Box encapsulated cell product.

This particular study compared three different methods for determining the number of viable cells in the capsules as well as the growth rate of the cells within the capsules. Although the rate of growth has already been fixed as part of the production process (and is not affected by this study), the new data revealed that only one of the tested methods can accurately estimate the number of cells within a capsule, particularly when the capsules are populated at high cell densities as they will be for use in PharmaCyte’s clinical trial in patients with LAPC. Therefore, this study provides the justification for the use of the most accurate and sensitive assay chosen by this study, which will also be used for quality control of the product release for the upcoming planned clinical trial for LAPC.

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, explained the significance of the study saying, "The cells that are encapsulated using the Cell-in-a-Box technology are the engine for the final product in that they activate the chemotherapeutic agent ifosfamide at the site of the tumor in the pancreatic cancer patient. This study is important since it shows we now have at our disposal the best method to determine the actual number of living cells in the capsules.

"The data from this study is another key piece of information required for PharmaCyte to comply with FDA guidelines and recommendations for our planned, upcoming clinical trial in patients with LAPC. This viability information must be reliable; therefore, it is also valuable for ensuring good quality control of our final product. In other words, using the chosen viability measuring method will help us ensure that the Cell-in-a-Box encapsulated product is reproducible from batch to batch. This is an essential requirement for any medicinal product."

On September 27, 2018 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported that members of the leadership team will present at the following upcoming investor conferences in New York City (Press release, Tocagen, SEP 27, 2018, View Source;p=RssLanding&cat=news&id=2369348 [SID1234529674]):

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Tuesday, October 2, 10:30 a.m. ET
Ladenburg Thalmann 2018 Healthcare Conference
Presenter: Chief Executive Officer Marty Duvall

Wednesday, October 3, 8:00 a.m. ET
Cantor Global Healthcare Conference
Presenter: Chief Executive Officer Marty Duvall

Wednesday, October 3, 9:00 a.m. ET
Leerink Roundtable Series: Rare Disease & Oncology
Presenters: Chief Financial Officer Mark Foletta and Chief Medical Officer Asha Das, M.D.

Tuesday, October 9, 3:45 p.m. ET
Chardan Capital Markets 2nd Annual Genetic Medicines Conference
Presenter: Chief Executive Officer Marty Duvall

The live audio webcasts from the conferences and subsequent replay may be accessed by visiting the "Events & Presentations" page in the investors section of Tocagen’s website. The webcasts will be available shortly after conclusion of the presentation and archived on the company’s website for 90 days following the presentation.

On September 27, 2018 Veracyte, Inc. (Nasdaq: VCYT) reported that Bonnie H. Anderson, chairman and chief executive officer, will present at the 2018 Cantor Fitzgerald Global Healthcare Conference on Tuesday, October 2, 2018, at 1:05 p.m. Eastern Time (ET) (Press release, Veracyte, SEP 27, 2018, View Source [SID1234529633]).

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A live audio webcast of the presentation will be available by visiting Veracyte’s website at View Source A replay of the webcast will be available for 90 days following the conclusion of the live presentation broadcast.

On September 27, 2018 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), reported that the European Patent Office ("EPO") has issued European Patent No. EP2940014B1 for CG-806, a first-in-class, highly potent oral small molecule being developed for acute myeloid leukemia (AML), B cell and other hematologic malignancies (Press release, Aptose Biosciences, SEP 27, 2018, View Source [SID1234529656]). The granted patent claims various compounds, including the CG-806 compound, pharmaceutical compositions comprising the CG-806 compound, and uses for the treatment of various diseases, such as cancer. This European patent will be nationalized in, and cover, approximately forty European countries including the United Kingdom, France, Germany, Italy, Netherlands and Spain. The patent is expected to provide protection until the end of 2033.

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"This is a meaningful decision by the EPO and adds to the previously issued patents in the US and Japan for CG-806," stated Dr. William G. Rice, Chairman, President and Chief Executive Officer of Aptose. "We will continue to strengthen the patent portfolio through additional findings and applications."

About CG-806

CG-806 is an oral, first-in-class pan-FLT3/pan-BTK multi-kinase inhibitor. This small molecule demonstrates potent inhibition of wild type and mutant forms of FLT3 (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region), eliminates acute myeloid leukemia (AML) tumors in the absence of toxicity in murine xenograft models, and represents a potential best-in-class therapeutic for patients with AML. Likewise, CG-806 demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser mutant forms of the BTK enzyme, as well as other oncogenic kinase pathways operative in B cell malignancies, suggesting CG-806 may be developed for various B cell malignancy patients (including CLL, MCL, DLBCL and others) that are resistant/refractory/intolerant to covalent BTK inhibitors.

About CrystalGenomics

CrystalGenomics, Inc. is a commercial stage biopharmaceutical company focused in the structure-based drug discovery and development of novel therapeutics in unmet medical need areas of inflammation, oncology, and infectious disease. In addition to several drug programs in the R&D pipeline, the Company has an osteoarthritis drug on the market and, has recently added manufacturing and commercialization capabilities through multiple acquisitions. For more information, please visit: www.cgxinc.com or www.crystalgenomics.com. CrystalGenomics, Inc. is listed on KOSDAQ (083790).

On September 27, 2018 VBI Vaccines Inc. (NASDAQ: VBIV) ("VBI"), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported that the independent Data and Safety Monitoring Board (DSMB) completed its second safety assessment of the ongoing Phase 1/2a clinical study of VBI-1901 in recurrent GBM (Press release, VBI Vaccines, SEP 27, 2018, View Source [SID1234529675]). The DSMB reviewed the complete safety data from the fully enrolled, intermediate-dose patient cohort, and unanimously recommended the continuation of the study without modification.

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Following this recommendation, VBI has initiated enrollment in the high-dose arm of the study. One final, pre-specified DSMB review is expected to occur after completion of enrollment in the high-dose cohort, concluding the dose-escalation phase of the study.

"We are encouraged by the sustained clean safety profile of VBI-1901 as concluded by this second DSMB assessment," said Jeff Baxter, VBI’s President and CEO. "These positive safety reviews are critical milestones for the program and for patients diagnosed with this extremely aggressive tumor who currently have no effective treatment options. With active clinical study sites at the Columbia University Medical Center, Dana-Farber Cancer Institute, and Massachusetts General Hospital, we hope to complete enrollment in this high-dose cohort quickly and look forward to announcing initial immunologic data from the low- and intermediate-dose cohorts expected later this year."

About the Phase 1/2a Study Design

VBI’s two-part Phase 1/2a study is a multi-center, open-label, dose-escalation study of VBI-1901 in up to 28 patients with recurrent GBM:

Part A: Dose-escalation phase to define the safety, tolerability, and optimal dose level of VBI-1901 in recurrent GBM patients. This phase is expected to enroll up to 18 patients in three dose cohorts.
Part B: A subsequent extension of the optimal dose level, as defined in the dose escalation phase. This phase is expected to enroll an expanded cohort of approximately 10 additional patients.

VBI-1901 is administered intradermally and is adjuvanted with granulocyte-macrophage colony-stimulating factor (GM-CSF), a potent adjuvant that mobilizes dendritic cell function. Patients in both phases of the study will receive vaccine every four weeks until tumor progression.

Additional information, including a detailed description of the study design, eligibility criteria, and investigator sites, is available at ClinicalTrials.gov using identifier NCT03382977.