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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

On February 10, 2018 AVEO Oncology (NASDAQ: AVEO) and EUSA Pharma reported the presentation of preliminary results from the Phase 2 portion of the TiNivo study, a Phase 1b/2 multicenter trial of oral (PO) tivozanib (FOTIVDA) in combination with intravenous (IV) nivolumab (OPDIVO, Bristol-Myers Squibb), an immune checkpoint, or PD-1, inhibitor, for the treatment of metastatic renal cell carcinoma (mRCC) (Press release, AVEO, FEB 10, 2018, View Source;p=RssLanding&cat=news&id=2331694 [SID1234523891]). The results were presented today at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Genitourinary Cancers Symposium (ASCO GU), in a poster presentation titled "Tivozanib combined with nivolumab: Phase Ib/II study in metastatic renal cell carcinoma (mRCC)" (Abstract 618). A copy of the presentation is available at www.aveooncology.com or further information can be obtained via EUSA Pharma Medical Information.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Phase 1/2 study has enrolled a total of 27 patients. The Phase 2 portion of the study (n=21) was designed to assess the safety, tolerability, and anti-tumor activity of the full dose and schedule of PO tivozanib (1.5 mg/QD for 21 days followed by a 7-day rest period), as established in the Phase 1 portion of the study (n=6), in combination with IV nivolumab (240 mg every 2 weeks). The combination was generally well tolerated. Treatment-related Grade 3/4 adverse events occurred in 44% of patients, the most common of which was hypertension.

Preliminary efficacy was assessed in 14 patients treated with the full dose and schedule of PO tivozanib in combination with IV nivolumab and enrolled at least 4 months prior to the data cutoff date. Of these, seven had received at least one prior systemic therapy. An objective response rate was observed in 64% of patients (partial responses), and a disease control rate (partial response + stable disease) was observed in 100% of patients. At the time of data collection, 11 of 14 evaluable patients remained on study.

"These preliminary data continue to support the rationale for choosing a high-specificity VEGF inhibitor TKI, such as tivozanib, in building upon the benefit of immune checkpoint therapy in renal cancer," said Doctor Bernard Escudier, MD, ex-Chairman of the Genitourinary Oncology Committee, Gustave Roussy, and lead investigator of the study. "Combining VEGF TKIs and immune checkpoint inhibitors has been hampered by toxicity, potentially emerging with the use of other TKIs, while minimal off-target toxicities have been observed with tivozanib in this combination. These results open the possibility for triple-combination therapy using tivozanib, nivolumab and ipilimumab, an immune system activator targeting CTLA-4."

"We believe that VEGF TKI-immunotherapy combinations are the obvious next step in the evolution of treatment within mRCC, which underscores the need for a VEGF therapy with best-in-class safety," said Michael Needle, M.D., chief medical officer of AVEO. "The preliminary activity and favorable safety profile observed thus far in the TiNivo study are encouraging and support the further exploration of tivozanib combinations with immuno-oncology therapies. In addition to the TiNivo study, we continue to look forward to topline data in the second quarter of 2018 from our Phase 3 TIVO-3 study, which, together with the previously completed TIVO-1 trial of tivozanib in the first line treatment of mRCC, is designed to support a request for regulatory approval of tivozanib in North America as a first and third line treatment for mRCC."

Lee Morley, EUSA Pharma’s Chief Executive Officer said, "We are excited by the continued development potential for tivozanib and the data arising from initial studies in combination with checkpoint inhibitors. As an effective TKI with a favorable tolerability profile, we are already launching tivozanib across the EU in line with its recent approval as monotherapy in the first line setting, and on the basis of the TiNivo study, we look forward to the potential to develop new innovative treatment options for patients in the future."

About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI) discovered by Kyowa Hakko Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma (RCC) in the European Union plus Norway and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.1,2 Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models, enabling potentially enhanced activity when used in combination with immune modulating therapy. As part of a North American registration plan, Tivozanib is currently being studied in the Phase 3 TIVO-3 trial, a randomized, controlled, multi-center, open-label study to compare tivozanib to sorafenib in subjects with refractory advanced RCC. Tivozanib has been investigated in several tumors types, including renal cell, hepatocellular, colorectal and breast cancers.

lthough emerging immuno-oncology products have been able to elicit unprecedented responses in cancers that are easily accessible to immune cells (eg. leukemia), solid tumors may be resistant to such therapies. 7 Hills is developing cost effective, safe therapeutics to overcome such resistance. Our lead compounds have the potential to increase the antitumor activity of numerous emerging cell-based immunotherapies, and enable the use of recently approved drugs to treat solid tumors that are currently refractory to therapy.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

7HP compounds target validated cell adhesion pathways that are critical for T cell homing to specific tissues. They are the only known activators of both the VLA-4/VCAM-1 and LFA-1/ICAM-1 cell adhesion axes. Activation of these adhesion pathways could significantly increase the trafficking of endogenous effector lymphocytes (targets of checkpoint blockade therapeutics) or adoptively transferred effectors (like CAR-T cells) into solid tumors.

Pre-clinical studies have shown that 7HP lead compounds:

Increase the adhesion of activated T-cells to simulated tumor endothelium by 100 fold,
Increase the penetration of activated T-cells into simulated tumor stroma.

On February 9, 2018 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that the company will present a company overview at the upcoming 2018 RBC Capital Markets Global Healthcare Conference on Thursday, February 22nd at 2:35 pm ET (Press release, PTC Therapeutics, FEB 9, 2018, View Source [SID1234523882]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The presentation will be webcast live on the Events and Presentations page under the investor relations section of PTC Therapeutics’ website at www.ptcbio.com. The presentation will be archived for two weeks following the presentation. It is recommended that users connect to PTC’s website several minutes prior to the start of the webcast to ensure a timely connection.