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]).

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 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]).

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 12, 2018 EUSA Pharma (EUSA), reported that the National Institute for Health and Care Excellence (NICE) has published a Final Appraisal Determination (FAD) recommending tivozanib as a first-line treatment option for advanced renal cell carcinoma (aRCC) in line with its licensed indication (Press release, EUSA Pharma, FEB 12, 2018, View Source [SID1234523911]). Tivozanib was licensed by the European Commission (EC) in August 20171 and is an oral, once-daily, potent selective vascular endothelial growth factor receptor tyrosine kinase inhibitor (VEGFR-TKI). VEGFR-TKIs work by reducing the supply of blood to the tumour – denying it the food and oxygen it would need to grow in size.2

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!

In arriving at its positive recommendation, NICE considered data from the global, open-label, randomised, multi-centre Phase III trial (TiVO-1)3 which evaluated the efficacy and tolerability of tivozanib compared to a currently available comparator VEGFR-TKI (sorafenib) in the treatment of 517 patients with aRCC. Patients treated with tivozanib experienced superior progression-free survival (PFS) (11.9 vs. 9.1 months in the overall population and 12.7 vs. 9.1 months in treatment naïve patients) versus sorafenib.3 There was also an improved side effect profile with tivozanib, with only 14% (versus 43% with sorafenib) requiring a dose reduction due to adverse events (AEs).3 In addition, fewer people on tivozanib experienced burdensome side effects, such as diarrhoea (23% vs 33%), and hand-foot syndrome (14% vs 54%).3

Dr Lisa Pickering, Consultant Medical Oncologist at St George’s Hospital, London, commented: This is excellent news for patients with aRCC. A key treatment goal is to enable patients to live without their cancer progressing for longer periods of time – we cannot cure their cancer but we can help to preserve normal living for as long as possible. That is why I’m delighted that NICE has recommended tivozanib as a treatment option."

Around 12 people in the UK die every day of kidney cancer, with more than 40% of cases in England diagnosed at a late (advanced) stage. Kidney cancer is expected to be one of the fastest increasing cancers over the next 10 years, as a result of the UK’s ageing population, and with smoking and a rise in obesity also playing a part.4

Nick Turkentine, Chief Executive of Kidney Cancer UK, said: "With over 12,500 new cases of kidney cancer being diagnosed every year this is a great result for people with aRCC and the overall fight with the disease. It is vitally important patients have access to effective, well-tolerated treatments, so they can spend more precious time with their families and loved ones. We applaud EUSA Pharma, NICE and NHS England for making this happen."

Patients with advanced renal cell carcinoma (aRCC) who may benefit from treatment with tivozanib will be able to access it from today as the final guidance is published, and should be able to have immediate access through the CDF.

Lee Morley, EUSA Pharma’s Chief Executive Officer commented "We are very pleased that NICE are recommending the use of Fotivda for the first line treatment of advanced Renal Cell Carcinoma and that Fotivda will be funded immediately in England and Wales, meaning physicians and patients can experience the benefits of Fotivda. Fotivda has the potential to become an important new first-line therapy and the recommendation by NICE is a great achievement for the EUSA team delivering on their mission to improve the lives of those patients suffering from cancer."

On February 12, 2018 Lipocine reported to have presented its Corporate presentation (Press release, Lipocine, DEC 12, 2018, View Source [SID1234523947]).

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!

On February 12, 2018 MabVax Therapeutics Holdings, Inc. (Nasdaq: MBVX), a clinical-stage biotechnology company focused on the development of antibody-based products to address unmet medical needs in the treatment of cancer, reported positive interim results from the Company’s ongoing Phase 1 trial evaluating MVT-5873 in combination with standard of care chemotherapy in patients newly diagnosed with pancreatic and other CA19-9 positive malignancies (Press release, MabVax, FEB 12, 2018, View Source [SID1234523919]). At the dose tested, all six patients in the cohort had meaningful reductions in tumor volume by RECIST.

In the Phase 1 study, MabVax’s MVT-5873, a fully human antibody, was given in combination with nab-paclitaxel and gemcitabine to patients newly diagnosed with CA19-9 positive pancreatic cancer. MVT-5873 at a dose of 0.125 mg/kg when added to first-line chemotherapy was generally well tolerated by all subjects. The Company reported that all six patients had measurable tumor reductions, with four patients meeting the criteria for partial response (PR) and two patients meeting the criteria for stable disease (SD). These results help confirm results reported from a group of patients treated earlier. Patient CA19-9 levels, which are a prognostic indicator of the disease state, were markedly reduced in all subjects with this combination therapy. The Company plans to enroll additional patients at this dose to further explore safety and potential response.

"We are highly encouraged by the continued positive response across all of the initial patients at this dose of MVT-5873. We are enrolling additional patients at this dose level to confirm our early clinical results with a goal to determine if these clinically meaningful initial results can continue to be replicated in a larger patient population. With additional confirmatory data, we could establish the potential of combining MVT-5873 with first line therapy in very difficult to treat cancer patients," commented David Hansen, MabVax’s President and Chief Executive Officer.

This Phase 1 clinical trial is an open-label, multi-center nonrandomized study evaluating the safety and recommended Phase 2 dose of MVT-5873 in combination with a standard of care chemotherapy in subjects with pancreatic and other CA19-9 positive malignancies. Secondary objectives include evaluating tumor response rate by RECIST 1.1, duration of response, and to determine pharmacokinetics. This study utilizes a conventional 3+3 design to identify the recommended Phase 2 dose. Dr. Eileen O’Reilly, Associate Director of the David M. Rubenstein Center for Pancreatic Cancer Research, attending physician, member at Memorial Sloan Kettering Cancer Center and Professor of Medicine at Weill Cornell Medical College is the lead investigator in the MVT-5873 Phase 1 clinical trial.

For additional information about the Phase 1 MVT-5873 clinical trial, please visit clinicaltrials.gov, and reference Identifier NCT 02672917.

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!