On January 13, 2020 Phoenix Biotechnology’s (PBI) (www.phoenixbiotechnology.com) reported that New research findings presented in November at the Italian Cancer Society’s 61st annual meeting in Naples, Italy confirm the ability of drug PBI-05204 to act synergistically with coadministration of temozolamide and radiotherapy to kill Glioblastoma Multiforme (GBM) in mice with orthotopically implanted human GBM cells Italian Cancer Society’s 61st annual meeting in Naples, Italy. These data are currently being prepared for publication.

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The new research results are the latest in a series of evidence in support of PBI-05204, PBI’s lead anticancer drug derived from Nerium oleander, as a potential treatment for GBM, according to Robert A. Newman, Ph.D., Phoenix Biotechnology’s President and Chief Science Officer. The American Brain Tumor Association reports that GBM kills more than 16,000 people each year in this country. Both Senator John McCain and Senator Edward Kennedy died from complications of GBM.

The Italian research, conducted through the University of L’Aquila, shows an ability of PBI-05204 to synergistically interact with standard of care chemotherapy (temozolamide) and radiotherapy in extending the life of mice with human GBM, according to Dr. Claudio Festuccia, the lead investigator of the project.

GBM is the most common glial brain tumor and results in extremely low patient survival. A better understanding of GBM biology has recently shown some of the important reasons why current new drugs fail to produce major tumor responses, Newman said.

These include, for example, inabilities of a drug (1) to gain access to malignant tissue across the blood barrier, (2) to selectively target only malignant cells, (3) to eliminate GBM stem cells, (4) to induce the synthesis of brain derived neurotrophic factor (BDNF) recently shown to be important for a meaningful GBM response and (5) to interact in a synergistic manner with currently used standard of care therapeutic strategies, he said. In addition to these criteria, an ideal drug would also be orally bioavailable.

"Recent data from research with PBI-05204, a modified supercritical extract of Nerium oleander, has shown that it can meet each of these challenges," Newman said. "Data from Phoenix Biotechnology as well as Duke University have shown the facile ability of oleandrin, an important API within PBI-05204, to cross the blood brain barrier following oral administration to mice."1-3

"Once in the brain, PBI-05204 induces the synthesis of brain derived neurotrophic factor (BDNF), something which very few other drugs have been able to do," he said.4 "PBI’s lead drug also represents targeted delivery against malignant cells through their selective expression of a particular subunit of Na, K-ATPase."5-6

"Very recent and exciting research which is also being prepared for publication has shown that PBI-05204 is very potent in elimination of human GBM stem cells, possibly eliminating the recurrence of tumor after therapy," Newman continued, noting that other scientific investigators from Istanbul, Turkey and Pozzilli, Italy have suggested that oleandrin represents a new approach to brain cancer therapy.7-8

PBI-05204 is the only patented drug containing oleandrin that has been through Phase I and Phase II clinical cancer trials in the United States.

On January 13, 2020 Imaging Endpoints reported that the U.S. Patent and Trademark Office has issued Patent No. 10,332,634 – "Systems And Methods For Reliably Diagnosing Breast Cancer Using Quantitative Textural Analysis" to Imaging Endpoints and its inventor, Chief Medical Officer Ron Korn, M.D., PhD (Press release, Imaging Endpoints, JAN 13, 2020, View Source [SID1234553120]).

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This breakthrough radiomic evaluation tool allows for rapid diagnosis of the nature of a patient’s breast abnormalities from standard mammography images. The technology enables a real-time evaluation that is otherwise available only through an invasive biopsy and avoids the time involved in obtaining and processing the biopsied tissue.

Imaging Endpoints is honored and excited to share with the market this patented technology that could provide patients and physicians the advantage of faster, less invasive information that is critical to treatment decisions and patient outcomes.

Imaging Endpoints is a pioneer and global leader in the non-invasive interrogation of tumors and diseased tissues by analyzing diagnostic images to identify imaging patterns linked to tumor biology. In cancer patients, image analysis provides a non-invasive, low-risk approach to assessing tumor biology prior to therapy and an objective pathway for monitoring treatment responses. The method of using signals detected on image data to characterize tumor biology is based on underlying tumor biology, growth kinetics and other drivers of oncologic transformation which may have novel expression patterns on standard of care radiological images. When used alone or in combination with other factors, a unique imaging phenotype may be developed to characterize lesions using both qualitative and quantitative approaches. Thus insights into the biologic processes of tumors may be measured, derived, inferred or predicted with the discovery of imaging phenotypes. This coupled with imaging’s ability to provide a comprehensive and real-time assessment of the entire tumor and its micro-environment make quantitative imaging an attractive tool for rapid assessment and prognosis.

The Breast Imaging Reporting and Data System (BI-RADS) was established by the American College of Radiology to assign mammogram screenings into discrete categories of perceived severity. A follow-up biopsy is typically recommended for BI-RADS category 4 and higher. However, biopsies ultimately confirm that many BI-RADS 4 designations are benign. Consequently, many women undergo painful and expensive biopsies only to find that the tumor is benign due to the rate of false positives.

Ronald Korn, M.D., PhD commented: "A reliable imaging signature for differentiating between malignant and non-malignant BI-RADS 4 mammographic lesions has remained elusive until now. The Imaging Endpoints invention provides a biomarker signature for determining whether a lesion identified in a breast image is malignant. The signature is derived from processing mammography data using a Quantitative Textural Analysis platform (TexRAD); generating respective histograms and related quantitative metrics, and performing logistical regression to yield a model predictive signature. Imaging Endpoints believes that its technology offers a real-time advantage with rapid results, however additional studies are needed to further validate the correlation of the signature with pathologic variables."

Imaging Endpoints currently offers imaging CRO services for the use of its advanced imaging technologies in clinical trials, and is actively seeking partners to commercialize its technologies for routine patient care.

On January 13, 2020 Helix BioPharma Corp. (TSX: "HBP") ("Helix" or the "Company"), an immunooncology company developing innovative drug candidates for the prevention and treatment of cancer, reported it has closed a private placement financing of 2,940,000 units of the Company ("Units") and the disposition of shares of the Company’s Polish subsidiary, Helix Immuno-Oncology S.A. ("HIO"), representing an 8.5% stake in HIO (together with the Units, the "Purchased Securities") for aggregate gross proceeds of approximately CAD $3,000,000, or $1.02 per Purchased Security (Press release, Helix BioPharma, JAN 13, 2020, View Source [SID1234553139]).

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Each Unit is comprised of one common share and one common share purchase warrant. Each common share purchase warrant will entitle the holder to purchase one common share at an exercise price of $1.43 and have an expiry of five years from the date of issuance. The terms of the private placement also include the disposition by the Company of shares of HIO, representing 8.5% of the outstanding shares of HIO. Following the disposition, the Company owns 66.5% of the outstanding shares of HIO.

The Company intends to use the net proceeds of the private placement for working capital and research and development activities.

ACM Alpha Consulting Management AG provided financial advisory services to Helix in connection with the private placement

On January 13, 2020 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported preliminary net revenues for 2019, as well as outlined key BPDCN market successes and upcoming commercial and clinical milestones (Press release, Stemline Therapeutics, JAN 13, 2020, View Source [SID1234553177]).

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Unaudited preliminary 2019 results include:

$43.2 million in estimated ELZONRIS net revenues for the year-ended December 31, 2019
$11.8 million in estimated ELZONRIS net revenues for the fourth quarter of 2019
The above financial information is based on preliminary unaudited information, is subject to adjustment, and does not present all information necessary for an understanding of the Company’s full-year and fourth quarter financial results for 2019. Stemline expects to report complete audited 2019 financial results on or before March 16, 2020.

Robert Francomano, Chief Commercial Officer of Stemline, stated, "We are very pleased with the solid uptake seen in the first year of the ELZONRIS launch, as we continue to successfully create, penetrate and grow a new market in BPDCN. Given the orphan nature and unique features of this disease, we believe patient starts were subject to significant quarterly variance – a phenomena that will likely continue throughout 2020. We are actively implementing a host of tactics to expand and further penetrate this emerging market."

Ivan Bergstein, CEO of Stemline, commented, "2019 was a transformational year for Stemline as we launched ELZONRIS, the first and only CD123 targeted agent and first agent ever approved for patients with BPDCN. We continue to pursue growth opportunities not only in BPDCN but also in a number of malignancies where targeting CD123 could provide therapeutic benefit. We look forward to data readouts in CMML, MF, and AML, including in patient subsets with high CD123, later this year and on into next year. Given our continued commercial and clinical progress, we look forward to a productive 2020 and beyond."

Corporate Highlights and Key Commercial and Clinical Milestones

BPDCN

$43.2 million in estimated, unaudited, net revenues for ELZONRIS in 2019
Marketing Authorization Application (MAA) under review by European Medicines Agency (EMA) for potential approval in the E.U.
Phase 1/2 trial of ELZONRIS in patients with BPDCN in the maintenance setting, post-stem cell transplant (SCT), has regulatory authorization to proceed; targeting treatment of the first patient in ~1Q20
Chronic Myelomonocytic Leukemia (CMML)

The CMML expansion cohort, Stage 3a, is open for enrollment of two patient populations: relapsed/refractory patients, and first-line, poor prognosis patients not expected to benefit from first line cytoreductive treatment
Results from Stage 3a is expected to inform the design of the subsequent Stage 3b confirmatory cohort for potential registration
We expect to report initial data from this trial in ~4Q20
Myelofibrosis (MF)

The MF cohort of the ongoing trial has been expanded to include 20-25 additional patients
We are evaluating relapsed/refractory patients and specific subsets of patients, including patients with monocytosis, thrombocytopenia, and CD123 positivity.
We expect to report updated data from this trial in ~4Q20
Acute Myeloid Leukemia (AML)

A Phase 1/2 trial of ELZONRIS in combination with other agents in patients with relapsed/refractory AML, treatment-naive AML unfit for chemotherapy, and high-risk myelodysplastic syndrome (MDS) is currently enrolling patients. We expect to provide data updates later this year.
About ELZONRIS
ELZONRIS (tagraxofusp), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

About BPDCN
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

About CD123
CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

On Januaruy 13, 2020 Ontario Institute for Cancer Research (OICR) and the University Health Network (UHN) have discovered detailed new information about the subtypes of pancreatic cancer (Press release, OICR, JAN 13, 2020, View Source [SID1234553214]). A better understanding of the disease groups may lead to new treatment options and improved clinical outcomes for this lethal disease.

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The study, published today in Nature Genetics, represents the most comprehensive analysis of the molecular subtypes of pancreatic cancer to date. Through detailed genomic and transcriptomic analyses, the research group identified five distinct subtypes of the disease (Basal-like-A, Basal-like-B, Classical-A, Classical-B, and Hybrid) with unique molecular properties that could be targeted with novel chemotherapies, biologics and immunotherapies.

"Therapy development for pancreatic cancer has been hindered by an incomplete knowledge of the molecular subtypes of this deadly disease," says lead author Dr. Faiyaz Notta, Co-Leader of OICR’s Pancreatic Cancer Translational Research Initiative (PanCuRx) and Scientist at UHN’s Princess Margaret Cancer Centre. "By rigorously analyzing advanced pancreatic cancers – which is the stage of disease that most patients have when they’re diagnosed – we were able to create a framework. This will help us develop better predictive models of disease progression that can assist in personalizing treatment decisions and lead to new targeted therapies."

The study is based on data from more than 300 patients with both early stage and advanced pancreatic cancer who participated in COMPASS, a first-of-its-kind clinical trial that is breaking new ground in discovery science and personalized pancreatic cancer treatment. COMPASS is enabled by advanced pathology laboratory techniques at UHN and OICR, and next generation sequencing at OICR.

"Most pancreatic cancer research is focused solely on early stage – or resectable – tumours, but in reality, pancreatic cancer is often found in patients after it has advanced and spread to other organs," says Notta. "COMPASS allowed us to look into these advanced cancers while treating these patients, develop a better understanding of the biology behind metastatic pancreatic cancer, and shed light on the mechanisms driving disease progression."

Interestingly, the Basal-like-A subtype, which had been difficult to observe before this study, was linked with a specific genetic abnormality. Most of the Basal-like-A tumours harboured several copies of a mutated KRAS gene, also known as a genetic amplification of mutant KRAS. The research group hypothesizes that some of the subtypes arise from specific genetic changes that occur as pancreatic cancer develops.

"This research opens new doors for therapeutic development," says Dr. Steven Gallinger, Co-Leader of OICR’s PanCuRx, Surgical Oncologist at UHN and Senior Investigator, Lunenfeld Tanenbaum Research Institute at Mount Sinai Hospital. "We look forward to capitalizing on the promise of these discoveries, building on our understanding of pancreatic cancer subtypes, and bringing new treatments to patients with the disease."

This research was supported by OICR through funding provided by the Government of Ontario, and by the Wallace McCain Centre for Pancreatic Cancer by the Princess Margaret Cancer Foundation, the Terry Fox Research Institute, the Canadian Cancer Society Research Institute, the Pancreatic Cancer Canada Foundation, the Canadian Friends of the Hebrew University and the Cancer Research Society (no. 23383).