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.

On January 10, 2018 LIDDS reported that it has successfully completed another preclinical study in mice assessing the feasibility of using the NanoZolid drug delivery technology for intratumoral immunotherapy (Press release, Lidds, JAN 10, 2018, https://www.globenewswire.com/news-release/2018/01/10/1286736/0/en/LIDDS-NanoZolid-with-immune-stimulating-agent-confirms-efficacy-in-an-additional-cancer-model.html [SID1234555920]). Clear anti-tumor effects have been observed in another cancer model in mice where significant decreases in tumor growth was demonstrated.

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LIDDS has several projects ongoing that focus on assessing the feasibility of using the NanoZolid-technology for local or intratumoral immunotherapy. These studies aim at a local intratumoral delivery of an immune-stimulatory agent.

The confirmed anti-tumor effect of an immune-stimulating agent formulated using the NanoZolid-technology in two independent cancer models in mice, will trigger the initiation of follow-on studies. The possibility that local immune-stimulation using the NanoZolid-technology will increase the effects of systemic immunotherapy will be investigated. Results are expected during the next four months.

A locally delivered immunotherapy has the potential to act either as a monotherapy or in combination with systemic immunotherapies e.g. checkpoint inhibitors. Successful combination treatments could significantly increase the response rates and efficacy rates of current immunotherapies. As immuno-oncology is the fastest growing area in oncology, reaching a market size over 100 billion USD by 2022, these results should be of great interest to pharmaceutical companies seeking new combination modalities to increase the response rate and efficacy of their immunotherapy drugs.

A recent review article in the highly ranked journal Annals of Oncology highlights the unique opportunity of intratumoral treatments to increase the efficacy of immunotherapy while reducing the potential side-effects, View Source

Immunotherapy for the treatment of cancer aims to activate and utilize the body’s own immune system to recognize and attack tumors and cancer cells and is today the most promising area of cancer research.

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.

On January 10, 2018 Abbvie presented JP Morgan Conference presentation (Presentation, AbbVie, JAN 10, 2018, View Source;t=1 [SID1234523053]).

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On January 9, 2018 Neuralstem presented at the Neuroscience Innovation Forum (Presentation, Neuralstem, JAN 9, 2018, View Source [SID1234523044]).

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