On February 12, 2018 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address liver and inflammatory diseases, reported that pre-clinical studies show Namodenoson’s novel mechanism of action which entails de-regulation of 3 key signaling pathways which mediate the etiology and pathology of NAFLD/NASH and are responsible for the anti-inflammatory and anti-fibrogenic effect in the liver (Press release, Can-Fite BioPharma, FEB 12, 2018, View Source [SID1234524644]).

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Pre-clinical studies were conducted in hepato-stellate cells in vitro and in an experimental NASH CCL4 model, showing that in both systems, the molecular mechanism of action of Namodenoson was conferred by decreased expression levels of the signaling protein phosphoinositol-3-phosphate (PI3K) which controls 3 downstream signal transduction pathways, the Wnt, NF-kB and α-SMA, which control liver inflammation and liver fibrosis. The detailed data is scheduled to be presented at the 2018 International Liver Congress (ILC), which is the annual meeting of the European Association for the Study of the Liver (EASL) (View Source).

The Company is currently conducting a Phase II trial with its drug candidate Namodenoson for the treatment of 60 patients with non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). There is currently no U.S. FDA approved drug for the treatment of NASH, which is an addressable pharmaceutical market estimated to reach $35-40 billion by 2025. Can-Fite’s 12-week study has been designed by leading Key Opinion Leaders in the area of NASH and liver diseases in the US including the company CAB members, Dr. Scott Friedman, Chief of the Division of Liver Diseases at the Icahn School of Medicine at Mount Sinai in New York; Dr. Arun Sanayal, Professor of Medicine, Physiology and Molecular Pathology at Virginia Commonwealth University School of Medicine; Dr. Rifaat Safadi, Head of the Liver Unit, Gastroenterology and Liver Diseases, Division of Medicine at Hadassah Medical Center and Dr. Stephen A. Harrison, the Medical Director of Pinnacle Clinical Research.

Namodenoson has been tested in over 100 subjects with other liver diseases, with clinical data suggesting a very favorable safety profile.

"We believe that the anti-inflammatory and anti-fibrogenic effects of Namodenoson, together with the favorable safety profile to date make it a promising drug candidate for the treatment of NAFLD/NASH," stated Prof. Rifaat Safadi.

About NAFLD/NASH

NAFLD is characterized by excess fat accumulation in the form of triglycerides (steatosis) in the liver. According to a study published in Hepatology, an estimated 17%-33% of the population in the U.S. has NAFLD, with a higher prevalence in people with type II diabetes. Incidence is increasing based on rising obesity rates. NAFLD includes a range of liver diseases, with NASH being the more advanced form, manifesting as hepatic injury and inflammation. According to the NIH, the incidence of NASH in the U.S. is believed to affect 2-5% of the population. The spectrum of NAFLDs resembles alcoholic liver disease; however, they occur in people who drink little or no alcohol. If untreated, NASH can lead to cirrhosis and liver cancer. By 2025, the addressable pharmaceutical market for NASH is estimated to reach $35-40 billion.

About Namodenoson (CF102)

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson is being evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect is believed to account for the excellent safety profile of the drug. Can-Fite has received Orphan Drug Designation for Namodenoson in Europe and the U.S., as well as Fast Track Status in the U.S. as a second line treatment for hepatocellular carcinoma

On February 12, 2018 Supernus Pharmaceuticals, Inc. (NASDAQ:SUPN), a specialty pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported that the Company expects to report the financial results for the fourth quarter and full year of 2017 after 5:00 PM ET on Tuesday, February 27, 2018 (Press release, Supernus, FEB 12, 2018, View Source [SID1234523918]).

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Jack Khattar, President and Chief Executive Officer, and Greg Patrick, Chief Financial Officer, will host a conference call to present the fourth quarter and full year 2017 business results on Wednesday, February 28, 2018 at 9:00 AM ET. Following the management presentation, the call will be open for questions.

A live webcast will be available at www.supernus.com.

Please refer to the information below for conference call dial-in information. Callers should dial in approximately 10 minutes prior to the start of the call.

Conference dial-in: (877) 288-1043
International dial-in: (970) 315-0267
Conference ID: 9385269
Conference Call Name: Supernus Pharmaceuticals Fourth Quarter and Full Year 2017 Earnings Conference Call

Following the live call, a replay will be available on the Company’s website under the ‘Investors’ section. The webcast will be available on the Company’s website for 60 days following the live call.

On February 12, 2018 ERYTECH Pharma (Euronext: ERYP- Nasdaq: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported the selection of Triple Negative Breast Cancer as the next target indication for broadening the scope of eryaspase (GRASPA) development in solid tumors (Press release, ERYtech Pharma, FEB 12, 2018, View Source;p=RssLanding&cat=news&id=2332012 [SID1234523923]).

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L-asparaginase is a cornerstone treatment in acute lymphoblastic leukemia (ALL), especially for pediatric patients, but the excessive toxicity of conventional L-asparaginase formulations has limited its use in other indications.

The positive Phase 2b result of eryaspase (L-asparaginase encapsulated in red blood cells) in metastatic pancreatic cancer, reported in 2017, represents, to our knowledge, the first-ever evidence of clinical benefit of an asparaginase-based product in a solid tumor indication. In this 141-patient randomized Phase 2b study, eryaspase in combination with chemotherapy demonstrated a 40% reduction in risk of death rate (HR=0.60; p=0.009) compared to chemotherapy alone.

Following these very encouraging results, ERYTECH conducted a comprehensive evaluation to determine other potential solid-tumor indications for developing eryaspase. Metastatic Triple-Negative Breast Cancer (TNBC) has now been selected as the next indication to expand the potential use of eryaspase in solid tumors. TNBC is an aggressive and metabolically active form of breast cancer with high rates of symptomatic metastases. A recent publication in Nature1 supports the hypothesis that restricting availability of asparagine can reduce metastatic progression of cancer cells in breast cancer. One of the most striking observations from our Phase 2b trial in pancreatic cancer was the 40% reduction of new lesions in the eryaspase arm compared to the control arm.

"TNBC is a heterogenous subgroup of breast cancer associated with poor patient outcome and high risk of recurrence compared to other breast cancer subtypes. Aside from BRCA1/2 mutation status, treatment options for TNBC remain limited," commented Iman El-Hariry, MD, PhD, Chief Medical Officer of ERYTECH. "There is growing evidence of altered metabolism in TNBC. The evaluation of eryaspase in metastatic TNBC provides a promising new therapeutic approach, which capitalizes on reprogramming of the metabolic pathways in this disease."

Gil Beyen, Chairman and CEO of ERYTECH, added, "The selection of TNBC as the second solid tumor indication for evaluating eryaspase anti-tumor activity brings hope for improving the health of these women. The safety profile of eryaspase provides additional rationale for combination with currently existing therapies to increase treatment options in TNBC."

The development in TNBC complements ERYTECH’s pipeline of programs, which focus on the development of therapies that target amino acid metabolism of tumor cells. Set-up activities of a Phase 2 proof-of-concept clinical study have started and ERYTECH expects to enroll the first patient in Q3 2018.

About Triple-Negative Breast Cancer (TNBC)

Breast cancer is the most commonly diagnosed cancer in women globally with nearly 1.8 million new cases diagnosed annually2. It is estimated that over 600,000 women each year are diagnosed with breast cancer in the United States and Europe in aggregate3. Approximately 10-20% of breast cancers are TNBC, a form of breast cancer that lacks expression of estrogen receptor (ER), progesterone receptor (PR) and does not overexpress HER24. TNBC is associated with a poorer prognosis when compared to other breast cancer subtypes. As commonly utilized hormone therapy and HER2 targeting agents are not treatment options for women with TNBC, there is significant unmet need for novel therapeutic approaches in this subtype of breast cancer.

On February 12, 2018 Ziopharm Oncology, Inc. (Nasdaq:ZIOP), a biotechnology company focused on development of next generation immunotherapies utilizing gene- and cell-based therapies to treat patients with cancer, reported data demonstrating point-of-care (P-O-C) manufacturing of human T cells expressing chimeric antigen receptor (CAR) that persist and have an anti-tumor effect in preclinical models were presented at the Keystone Symposia Emerging Cellular Therapies: T Cells and Beyond in Keystone, Colorado (Press release, Ziopharm, FEB 12, 2018, View Source [SID1234523928]).

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The data presented showed T cells expressing CD19-specific CAR with membrane-bound IL-15 (mbIL15) were generated with the non-viral Sleeping Beauty system in less than two days and did not require ex vivo activation or propagation. T cells designed to express mbIL15 showed greater persistence and more potent antitumor activity than comparator T cells without mbIL15 in these studies.

Lenka V. Hurton, Ph.D., a researcher in the Division of Pediatrics at the University of Texas MD Anderson Cancer Center, presented the findings in a talk entitled, "Rapid production of T cells co-expressing CAR and membrane-bound IL-15 potentiates antitumor activity and promotes in vivo memory." She also presented a poster under the same title during the Keystone Symposia.

Ziopharm is advancing its non-viral Sleeping Beauty platform towards using its point-of-care, or P-O-C, technology, a very rapid manufacturing process of genetically modified CAR+ T cells co-expressing mbIL15, with the first in-human trial utilizing this approach expected to commence in 2018. Ziopharm believes that manufacturing under P-O-C has the potential to reduce the costs associated with T-cell therapies and the potential to broaden application based on avoiding the need for centralized manufacturing as is the case when using a virus to genetically modify T cells.

Dr. Hurton’s poster and presentation slides are based on research conducted in collaboration with The University of Texas MD Anderson Cancer Center and Precigen Inc., a wholly-owned subsidiary of Intrexon Corporation (NYSE:XON). The poster is available in the Presentations and Publications section of the Company’s website, www.ziopharm.com.

On February 12, 2018 VistaGen Therapeutics, Inc. (NASDAQ: VTGN), a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders, reported financial results for its third fiscal quarter ended December 31, 2017 (Press release, VistaGen Therapeutics, FEB 12, 2018, View Source [SID1234523927]).

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"Building on our significant progress last quarter, our team is prepared and eager to launch, during the current quarter, our AV-101 Phase 2 clinical development program, initially focused on adjunctive treatment of Major Depressive Disorder patients with an inadequate response to standard, FDA-approved antidepressants. This year has the potential to be transformative for VistaGen and the millions of depression patients seeking new generation treatment options that are fundamentally different from all currently available therapies," commented Shawn Singh, Chief Executive Officer of VistaGen.

Financial Results for the Fiscal Quarter Ended December 31, 2017:
Net loss attributable to common stockholders for the fiscal quarter ended December 31, 2017 was approximately $3.5 million, compared to $2.9 million for the fiscal quarter ended December 31, 2016.

Research and development expense totaled approximately $1.6 million for the fiscal quarter ended December 31, 2017, compared with approximately $1.6 million for the fiscal quarter ended December 31, 2016. Research and development expense was primarily attributable to the Company’s development of AV-101, its oral, new generation CNS drug candidate initially focused on displacing adjunctive atypical antipsychotics in the current Major Depressive Disorder (MDD) treatment paradigm, including final preparations to launch its AV-101 MDD Phase 2 adjunctive treatment study in patients with an inadequate response to standard FDA-approved antidepressants.

General and administrative expense was approximately $1.3 million in the fiscal quarter ended December 31, 2017, compared to approximately $2.3 million in the fiscal quarter ended December 31, 2016. The decrease was primarily attributable to decreased professional services expenses, a decrease in noncash expense attributable to grants of common stock for services, and a decrease in noncash warrant modification expense, partially offset by increased salary and benefits and noncash stock compensation expenses.

At December 31, 2017, the Company had cash and cash equivalents of approximately $13.0 million, compared to approximately $2.9 million at March 31, 2017.