On February 6, 2019 KIYATEC, Inc., reported that Oregon Health & Science University (OHSU) Knight Cancer Institute has initiated patient enrollment into KIYATEC’s clinical study, 3D-PREDICT, to validate the company’s test as a patient-specific predictor of response to cancer therapies for solid tumors (Press release, KIYATEC, FEB 6, 2019, View Source [SID1234533099]).

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In this clinical study, the test analyzes a patient’s live cancer cells, grown in KIYATEC’s laboratory within a biologically-relevant 3D microenvironment, to determine whether or not those cells respond to guideline-recommended cancer drugs. Evidence from the company’s earlier pilot study established a correlation between patient-specific predicted tumor response and actual patient clinical response to cancer therapy. The 3D-PREDICT study is a fully prospective, multi-institutional effort to validate the predictive accuracy of the test and correlate response predictions to clinical outcomes among patients with newly diagnosed and relapsed ovarian cancer, glioblastoma and certain rare tumors.

At present, the OHSU Knight Cancer Institute is enrolling newly diagnosed and relapsed ovarian cancer patients into the 3D-PREDICT Study.

"As a pioneer in personalized cancer care, the OHSU Knight Cancer Institute is deeply committed to optimizing appropriate therapy for our patients as early as possible following diagnosis, when the disease is most treatable," said Dr. Koen De Geest, lead investigator of the clinical trial at OHSU. "Five-year survival among high-grade ovarian cancer patients is 30%, and we believe this test has the potential to help improve outcomes in the clinic."

"With cancer treatment, and especially ovarian cancer, time is of the essence and being able to measure patient-specific evidence of response and non-response before treatment begins can truly change the future of cancer care," said Matthew Gevaert, CEO of KIYATEC. "We welcome OHSU to our clinical study and their participation will be integral as we work to deliver accurate predictions of patient response to cancer therapies, reducing the need for patients to undergo treatments that may not work."

The 3D-PREDICT study is anticipated to continue through 2022. Details on the trial can be found on View Source

On February 6, 2019 GT Biopharma, Inc. (OTCQB: GTBP and Euronext Paris GTBP.PA) ("GT Biopharma" or the "Company"), an immuno-oncology biotechnology company focused on innovative treatments based on the Company’s proprietary NK-engager and Bispecific Antibody Drug Conjugate platforms, reported that it has entered into a Securities Purchase Agreement with existing investors pursuant to which the Company has issued to the Purchasers Convertible Secured Notes in an aggregate principal amount of $1,352,224, consisting of gross proceeds of $1,052,224 and settlement of existing debt of $300,000, which Notes shall be convertible into the Company’s common stock, par value $0.001 per share at a price of $0.60 per share (Press release, GT Biopharma , FEB 6, 2019, View Source [SID1234539516]).

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GT Biopharma also provided an update on its corporate progress, clinical status and anticipated milestones for its pipeline of immuno-oncology products based off the Company’s proprietary bi-specific Antibody Drug Conjugate (ADC), Tri-specific Killer Engager (TriKE) and Tetra-specific Killer Engager (TetraKE) technology platforms.

"2018 represented a critical transitional phase for our Company, and I believe we have built significant momentum positioning us to achieve multiple corporate, clinical and regulatory milestone over the course of 2019. Importantly, we are very pleased to have closed the initial bridge financing, which represents an important step in our strategy to ensure we are properly funded to propel the Company to our next phase of growth. We are grateful to our loyal stockholders who have provided continued support of the Company through this investment," commented Raymond Urbanski, M.D., Ph.D., Chief Executive Officer of GT Biopharma. "We continue focusing all of our resources on the flawless execution of our strategy and believe 2019 will be a transformational year for the Company, enabling us to drive significant shareholder value."

Clinical Program Updates

GTB-1550 (OXS-1550): Most Advanced Bi-specific ADC Candidate

The Company’s most advanced bi-specific ADC in development, GTB-1550, targets CD19+ and/or CD22+ hematological malignancies and is currently in the Phase 2 component of a Phase 1/2 Non-Hodgkin’s Lymphoma (NHL)/Acute Lymphocytic Leukemia (ALL) trial which is an open-label, investigator-led study.

GTB-1550 targets cancer cells expressing the CD19 receptor or CD22 receptor or both receptors. When GTB-1550 binds to cancer cells, the cancer cells internalize GTB-1550, and are killed due to the action of drug’s cytotoxic diphtheria toxin payload. GTB-1550 has demonstrated success in a Phase 1 human clinical trial in patients with relapsed/refractory B-cell lymphoma or leukemia.

Dr. Veronika Bachanova, Associate Professor of Medicine, Division of Hematology, Oncology and Transplantation at the University of Minnesota and the Principle Investigator for this study, is currently analyzing the final data and is anticipating submitting an abstract for the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) conference.

GTB-3550 (OXS-3550): TriKE product candidate

GTB-3550 is the Company’s first Tri-specific Killer Engager (TriKE) product candidate being initially developed for the treatment of acute myelogenous leukemia (AML). GTB-3550 is a single-chain, tri-specific scFv recombinant fusion protein conjugate composed of the variable regions of the heavy and light chains of anti-CD16 and anti-CD33 antibodies and a modified form of IL-15. When the NK stimulating cytokine human IL-15 is used as a crosslinker between the two scFvs, it provides a self-sustaining signal that activates NK cells and enhances their ability to kill.

GT Biopharma recently announced that its Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) is now open and it is authorized to initiate a first-in-human Phase 1 study with GTB-3550, its first-in-class TriKE, for the treatment of AML, myelodysplatic syndrome (MDS) and mastocytosis. The study will be led by Principal Investigator, Erica Warlick, Associate Professor, Division of Hematology, Oncology and Transplantation at Masonic Cancer Center, University of Minnesota.

This single center, first-in-human Phase 1 clinical trial of GTB-3550 will enroll up to 60 subjects with CD33-expressing refractory/relapsed AML, high-risk MDS, or advanced systemic mastocytosis. Subjects will receive a single course of GTB-3550 given as 3 weekly treatment blocks. Each block consists of four consecutive 24-hour continuous infusions of GTB-3550 followed by a 72-hour break after Block #1 and #2. Disease response will be assessed by bone marrow biopsy performed between Day 21 and Day 42 after the start of the 1st infusion. Follow-up for response and survival continues through 6 months from treatment start. The primary objective from the Phase 1 dose finding portion of the study will be to identify the maximum tolerated dose (MTD) of GTB-3550 defined as the dose level that most closely corresponds to a dose limiting toxicity rate (DLT) of 20%. The primary objective from the Phase 2 extended portion of the study will be the potential efficacy of GTB-3550, measured using rates of complete and partial remission. Subjects experiencing clinical benefit and no unacceptable side effects may be considered for a 2nd course of GTB-3550 on a compassionate basis.

The Company believes that GTB-3550 could serve as a relatively safe, cost-effective, and easy-to-use therapy for refractory/relapsed AML, high-risk MDS and advanced systemic mastocytosis and could also be combined with chemotherapy and/or other agents as frontline therapy thus targeting a much larger patient population.

GT Biopharma’s initial and ongoing work is being conducted in collaboration with the Masonic Cancer Center at the University of Minnesota under research agreements led by Dr. Jeffrey Miller, the Deputy Director and Dr. Daniel Vallera, Director, Section of Molecular Cancer Therapeutics.

GT Biopharma has an exclusive worldwide license agreement with the University of Minnesota to further develop and commercialize cancer therapies using proprietary TriKE technology developed by researchers at the university to target NK cells to cancer.

Upcoming Milestones Expected to Drive Value

Announce topline results from Phase 1/2 trial of GTB-1550 in Q1 2019;
Initiate Phase 1 first-in-human clinical trial of GTB-3550 for the treatment of Relapse/Refractory AML, High Risk MDS, and Advanced Systemic Mastocytosis in the first half of 2019;
Conduct meeting for GTB-1550 with U.S. FDA in the first half of 2019;
Advance ongoing GTB-C3550 IND-enabling studies & TetraKE pre-clinical program to target the larger solid tumor population and are working towards beginning clinical trials in 2019;
Bolster executive management team and board with key expertise to continue to transform the Company;
Participate in key scientific conferences;
Make progress in advancing potential corporate and business development opportunities; and
Uplist to a National Exchange.
This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On February 6, 2019 Arch Oncology, Inc., a clinical-stage immuno-oncology company focused on the discovery and development of next-generation anti-CD47 antibody therapies for cancer, reported the initiation of a new Phase 1 clinical trial of AO-176 in select solid tumors with the first patient dosed (Press release, Arch Oncology, FEB 6, 2019, View Source [SID1234533100]). AO-176 is an anti-CD47 antibody with a best-in-class profile that works by blocking the "don’t eat me" signal and also by directly killing tumor cells, with preferential binding to tumor versus normal cells.

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"Dosing the first patient in this new trial is a significant milestone for our Company," said Julie M. Cherrington, Ph.D., President and Chief Executive Officer of Arch Oncology. "AO-176 is an anti-CD47 antibody with a best-in-class profile and we are excited to be part of the effort to advance this new class of therapeutics. Our team has worked diligently to bring AO-176 into the clinic, and our aim is to develop this antibody as a new treatment approach for patients with cancer."

Howard A. "Skip" Burris, III, M.D., President, Clinical Operations, Chief Medical Officer, and Principal Investigator, Sarah Cannon Research Institute, commented, "There is a growing body of research on AO-176, which has demonstrated in preclinical studies a highly differentiated mechanism among the anti-CD47 agents. With greater insights into the number of tumors that overexpress CD47, we are excited to participate in this study to assess this next-generation anti-CD47 antibody’s safety and preliminary efficacy profile."

Sarah Cannon Research Institute dosed the first patient in the Phase 1 clinical trial of AO-176. The multicenter, open-label, dose-escalation and dose-expansion study is evaluating the safety, pharmacokinetics, pharmacodynamics, and preliminary anti-tumor activity of AO-176 in patients with select solid tumors.

About AO-176

AO-176 is a humanized anti-CD47 IgG2 antibody with a best-in-class profile. Arch Oncology’s next-generation anti-CD47 antibody AO-176 is highly differentiated, with the potential to improve upon the safety and efficacy profile relative to other agents in this class of checkpoint inhibitors. AO-176 works by blocking the "don’t eat me" signal, the standard mechanism of anti-CD47 antibodies. Beyond blocking this signal, AO-176 also works by directly killing tumor cells. Importantly, AO-176 binds preferentially to tumor cells, instead of to normal cells, and binds even more potently to tumors in their acidic microenvironment (low pH). AO-176 is in a Phase 1 clinical trial for the treatment of patients with select solid tumors.

Data presented on AO-176 at recent medical and scientific meetings can be found at View Source

On February 6, 2019 Vaxart, Inc., a clinical-stage biotechnology company developing oral recombinant vaccines that are administered by tablet rather than by injection, reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Vaxart, FEB 6, 2019, View Source [SID1234533101]).

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"As we execute on our objective of building a leading oral vaccine company, we continue to expand our understanding of the unique properties of our oral vaccine platform and the important advantages we believe it can offer over conventional injectable vaccines, particularly for mucosal pathogens such as norovirus, flu and RSV," said Wouter Latour, M.D., chief executive officer of Vaxart. "We are focused on our lead product candidate, the first oral vaccine against norovirus, a disease with a $34 billion economic impact in high income countries including the United States, Europe and Japan. After laying the groundwork in 2018, we expect to initiate our norovirus Phase 1 bivalent study and Phase 2 monovalent challenge study during the first half of 2019. In parallel, we are advancing our first therapeutic vaccine targeting HPV-associated dysplasia and cancer toward the clinic."

2018 Highlights:

Corporate:

In February, Vaxart commenced trading on the Nasdaq Capital Market under the symbol "VXRT" following the closing of its merger with Aviragen Therapeutics.
In October, at ID Week in San Francisco, the Company presented data from its H1 influenza Phase 2 challenge study demonstrating that its oral H1 flu vaccine, while providing 39% reduction in flu illness compared to 27% for Fluzone, protected primarily through mucosal immunity, in contrast to Fluzone which primarily protected through serum antibodies. This finding provides evidence that Vaxart’s oral vaccines may deliver better protection against mucosal pathogens than injectable vaccines.
In July, Vaxart announced the publication of the comprehensive results of the previously disclosed Phase 1 clinical trial with its norovirus oral tablet vaccine in the Journal of Clinical Investigation Insight. As reported in the article, the vaccine generated robust systemic and mucosal immune responses, including mucosal IgA, memory B cells, and serum blocking antibody titers (BT50), all potential correlates of protection.
In October at the 32nd International Papillomavirus Conference, the Company presented preclinical data on its human papillomavirus (HPV) vaccine trial. The Vaxart HPV vaccine created CD8 tumor-infiltrating T cells and eliminated or significantly reduced the majority of tumors with or without a checkpoint inhibitor.
In June, the Company announced the publication of preclinical results from its oral F-protein based Respiratory Syncytial Virus (RSV-F) vaccine in Vaccine. As described in the article, the oral RSV-F vaccine candidate provided complete sterilizing protection against RSV infection in the cotton rat challenge model at the target dose.
Financial Results for the Three Months and Year Ended December 31, 2018

Vaxart reported a net loss of $4.9 million for the fourth quarter of 2018 compared to a net loss of $1.1 million for the fourth quarter of 2017. For the year ended December 31, 2018, the net loss was $18.0 million compared to a net loss of $9.6 million for 2017.
Vaxart ended the year with cash and cash equivalents of $11.5 million compared to $17.9 million at September 30, 2018. The decrease was primarily due to cash used in operations.
Revenue for the quarter was $1.8 million compared to $0.8 million in the fourth quarter of 2017. The increase was due to royalty revenue resulting from our merger with Aviragen, offset by lower revenues from the contract with BARDA, which ended on September 30, 2018.
Research and development expenses were $4.5 million for the quarter compared to $1.9 million for the fourth quarter of 2017. The increase was mainly due to higher clinical and manufacturing costs incurred in the Company’s norovirus program and amortization of intangible assets acquired in the merger with Aviragen, offset by lower expenditures incurred under the BARDA contract.
General and administrative expenses were $1.2 million for the quarter compared to $1.5 million for the fourth quarter of 2017. The decrease was a result of significant one-off costs incurred in the 2017 period in connection with the merger with Aviragen

On February 6, 2019 Pathios Therapeutics ("Pathios"), an innovative biotech company focused on the development of first in class therapies for autoimmune diseases and immuno-oncology and Sygnature Discovery ("Sygnature"), reported a strategic partnership to accelerate Pathios’ drug discovery and development programmes (Press release, Pathios Therapeutics, FEB 6, 2019, View Source [SID1234650160]).

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As part of the agreement, Pathios and Sygnature will collaborate on an integrated drug discovery programme against a novel G Protein-Coupled Receptor (GPCR) target. Specifically, Sygnature are providing their industry leading expertise in GPCR bioscience and medicinal chemistry to expand Pathios’ current hit-to-lead programme. Sygnature are also deploying their computational chemistry, library design and synthesis and medium throughput screening capability to broaden Pathios’ hit finding. As part of this agreement, Sygnature will receive equity in addition to fees.

Pathios Therapeutics brings together cutting-edge European science and a leading drug discovery and development team to modulate the activity of GPR65, a pH sensing GPCR. This drug target is characteristic of certain T helper 17 (Th17) cell populations which have been shown to contribute significantly to the pathology of autoimmune conditions, such as ankylosing spondylitis and psoriatic arthritis. In addition, recently published studies have demonstrated GPR65 drives tumour associated macrophages (TAM) to adopt a phenotype that supports cancer immune evasion.

Tom McCarthy, Executive Chairman and Co-founder of Pathios Therapeutics Limited, commented: "We founded Pathios to build on emerging science that demonstrated GPR65 sits at the nexus of autoimmune disease and immuno-oncology as this receptor links pathology caused by a low pH environment. The ultimate aim is to block the pathological process that GPR65 initiates without interfering with the physiological role of this receptor. In addition to developing potent and selective drugs to modulate GPR65, we are continuing to broaden the understanding of the fundamental biological processes that link to GPR65’s effects in Th17 cells, TAMs and other cell types. Our team has worked closely with Sygnature in the past and know they have the deep experience, expertise and state-of-the-art drug discovery and development infrastructure to drive our programme forward. I’m delighted on this rare occasion Sygnature also chose to invest in Pathios and expand our GPR65 drug discovery efforts while we explore Series A funding opportunities".

Simon Hirst, CEO of Sygnature, added: "We are extremely pleased to take this exceptional opportunity to partner with, and invest in Pathios on their drug discovery projects. Based on our diligence activities, we are excited about the potential for GPR65 modulation to be central to new treatments for autoimmune disease and a critical mechanism of action in next generation immunooncology drugs targeting the tumour micro-environment. We are excited to have the opportunity to work with Tom and the rest of Pathios’ tremendously talented team again and contribute to the potential positive impact their therapeutics will have on patients’ lives".