On January 10, 2018 Intrexon Corporation presented Presentation, dated January 10, 2018 (Presentation, Intrexon, JAN 10, 2018, View Source [SID1234523050]).

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On January 10, 2018 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the MD Anderson Cancer Center ("MD Anderson"), reported it has expanded the Company’s development pipeline for the treatment of acute myeloid leukemia ("AML") with an immuno-stimulating STAT3 inhibitor (Press release, Moleculin, JAN 10, 2018, View Source [SID1234523052]).

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"Leading experts in the treatment of AML, Dr. Jorge Cortes and Dr. Sanjay Awasthi have now asked us to expand our clinical research to include WP1066, our immuno-stimulating agent and STAT3 inhibitor, to increase therapeutic options for AML patients," commented Walter Klemp, Chairman and CEO of Moleculin. "This would potentially be complementary and synergistic with Annamycin and existing first line treatments and could position us as a leader in the advancement of leukemia treatments."

Dr. Sanjay Awasthi, Professor of the Department of Internal Medicine, Division of Hematology & Oncology, Texas Tech University Health Sciences Center and Medical Director, Southwest Cancer Center Lubbock, Texas, added, "The apparent ability in pre-clinical trials of WP1066 to stimulate the patient’s natural immune response and simultaneously inducing tumor cell death by inhibiting the activated form of STAT3 is highly promising and unique. Importantly, based on current scientific findings, such properties should be extremely valuable in developing improved treatments for AML patients and expanding their therapeutic options.

"We are clearly excited to see what Moleculin’s Annamycin can do for relapsed or refractory AML patients in the Company’s recently launched clinical trial," continued Dr. Awasthi, "thus given the potential for an even broader arsenal of AML drugs, we are encouraging Moleculin to expand their AML clinical research to include this novel immuno-stimulating STAT3 inhibitor drug candidate."

Another noted AML expert, Dr. Jorge Cortes, is also encouraging Moleculin’s clinical expansion, commenting: "AML appears to be associated with a significant increase in the activation of STAT3 and many of us in the AML clinical community have been eager to test the ability of a STAT3 inhibitor to treat AML patients. Part of the difficulty in pursuing this path has been finding a safe and effective STAT3 inhibitor and, if successful, WP1066 may have finally opened this pathway."

Mr. Klemp concluded: "Of course, our first priority will be to demonstrate single agent activity in both Annamycin and WP1066, but we see exploring the potential for synergistic effect as a longer-term opportunity as well."

Researchers have shown that ritanserin is a novel DGK-alpha inhibitor, with activity against glioblastoma (GBM) in vitro and in vivo. They have shown that it is synergistic with temozolomide chemotherapy and radiation against GBM cells. Furthermore, ritanserin has preferential cytotoxicity against mesenchymal GBM cells, a particularly treatment-resistant subset lacking any selective and effective therapies at this time.

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On January 10, 2018 Delcath Systems, Inc. (OTCQB:DCTH), an interventional oncology company focused on the treatment of primary and metastatic liver cancers, reported that it has concluded a modification agreement with the U.S. Food and Drug Administration (FDA) for its Phase 3 clinical trial of Melphalan Hydrochloride for Injection for use with the Delcath Hepatic Delivery System (Melphalan/HDS) to treat patients with hepatic dominant ocular melanoma (The FOCUS Trial). The modification agreement revises the FOCUS trial’s eligibility criteria to permit a greater extent of extra-hepatic disease by removing the size restriction, number and location of extra-hepatic lesions, in conjunction with a treatment plan for the extra-hepatic metastases.

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Commenting on the announcement, Jennifer K. Simpson, Ph.D., President and CEO of Delcath Systems, "We requested this protocol modification to improve patient access to this important clinical trial for appropriately selected and managed patients. In an ultra-orphan indication like ocular melanoma, striking the appropriate balance between eligibility criteria and patient access can be a challenge. We are pleased that the FDA agreed to this modification, and hope that once approved by the institutional review boards of our participating clinical trial sites, that this modification will help accelerate enrollment in this registrational trial."

PHP Therapy with Melphalan/HDS was developed by Delcath Systems as a targeted, whole organ therapy for the liver. It is commercially available as a device in Europe, where it is marketed as CHEMOSAT. The system has not been approved by the U.S. Food and Drug Administration, and is undergoing Phase 3 clinical testing in the U.S. as an investigational product.

Abstract #202913

Clinical significance of high expression of a specific solute carrier transporter in HCC (Abstracts, GenoScience, JAN 10, 2018, View Source [SID1234523060]).

Patricia Gifu, Sonia Brun, Guanxiong Wang, Firas Bassissi, Claude Caron de Fromentel, Philippe Merle, Philippe Halfon; Cancer Research Center of Lyon, Lyon, France; Genoscience Pharma, Marseille, France; Service d’Hepatogastroentérologie, Hôpital de la Croix Rousse, Lyon, France

Background: Hepatocellular carcinoma (HCC) is the third cause of cancer-related death. GNS561 (Genoscience Pharma, France) is a small molecule inducing apoptosis by lysosome inhibition and caspase activation. GNS561 has high hepatotropism and anti-tumorigenic capacity on tumor bulk and cancer stem cells. Its main target is a specific solute carrier transporter (SLCt). We describe here the clinical relevance of the overexpression of SLCt in HCC patients.

Methods: The expression of SLCt was investigated by iQRT-PCR in surgically resected HCC tumors (T) and the matched non-tumor (NT) liver tissues (n = 180) as well as healthy livers (HL) devoid of chronic or acute disease (n = 10). Pearson’s chi-squared test was employed for significant correlation tests while univariate and multivariate survival analysis were performed by Cox proportional hazard ratio (HR) method.

Results: SLCt was overexpressed in 40.5% of T and 30.3% of NT as compared to HL. High SLCt in T was associated with microvascular emboli (p = 0.034) and expression of the cancer stem cell markers Sox2 (p = 0.025) and CD133 (p = 0.034). High SLCt in NT correlated with cirrhosis (p = 0.009) and presence of satellite nodules (p < 0.001). Univariate analysis showed association between high SLCt in T and shortened overall survival (OS) (HR = 1.08, p = 0.020), lower progression-free survival (PFS) (HR = 1.76, p = 0.006) as well as early recurrence-free survival (ERFS) (within 2 years post-surgery) (HR = 1.88, p = 0.008). In multivariate analysis, high SLCt tended to be an independent factor for OS, and this was strongly significant for PFS and ERFS. High SLCt in NT, is quite a poor outcome factor on late recurrence-free survival (HR = 2.33, p = 0.056) together with cirrhosis in univariate analysis, but remained dependent of cirrhosis in multivariate analysis. Late recurrence is linked to the intrinsic tumorigenic status of the liver.

Conclusions: Overexpression of the SLCt in HCC tumors is associated to stemness features and appears as a poor outcome factor impacting on early recurrence. Further, SLCt in NT is associated with development of de-novo HCC. Validation cohorts and prospective assessments are needed to define high SLCt as a potential biomarker of HCC stemness and patient outcome.