On September 30, 2018 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, today is presenting data describing the tumor microenvironment and immunogenicity of Toca 511 (vocimagene amiretrorepvec) & Toca FC (flucytosine, extended-release) in patients with solid tumor malignancies at the International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) hosted by The Cancer Research Institute (CRI), the Association for Cancer Immunotherapy (CIMT) (Free CIMT Whitepaper), the European Academy of Tumor Immunology (EATI), and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in New York City (Press release, Tocagen, SEP 30, 2018, View Source;p=RssLanding&cat=news&id=2369481 [SID1234529699]). The lead author is Jaime Merchan, M.D., director, Phase 1 clinical trials program at Sylvester Comprehensive Cancer Center; associate professor of medicine at the University of Miami Miller School of Medicine.

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The poster describes data available as of August 31st for 20 patients with advanced solid tumors, including colorectal, sarcoma, pancreas and non-small cell lung cancer, treated with intravenous Toca 511 followed by oral Toca FC in the Phase 1b Toca 6 clinical trial. Among these patients, 15 also received Toca 511 via intratumoral administration.

In this preliminary analysis of immune activation in patients with advanced solid tumors, analysis of peripheral blood shows immune cell modulation that is consistent with what has been observed in preclinical studies and in patients with recurrent high grade glioma treated with Toca 511 & Toca FC in previous clinical trials. In addition, available tumor samples from three patients show Toca 511 infected both "hot" (T cells present) and "cold" (T cells low or absent) areas of metastatic tumor, suggesting Toca 511 can penetrate multiple tumor microenvironments. Toca 511 & Toca FC treatment was well tolerated.

"The encouraging preliminary immune activity data from the Toca 6 trial continue to support the proposed mechanism of action for Toca 511 & Toca FC and its potentbiial in the treatment of multiple cancers," said Asha Das, M.D., chief medical officer of Tocagen. "We look forward to advancing expansion opportunities for our lead product in patients with solid tumors."

CRI-CIMT-EATI-AACR Abstract/Poster Number: A018
Abstract Title: Effects of Toca 511 & Toca FC on tumor microenvironment and peripheral blood populations in patients with advanced malignancies
Date: Sunday, September 30, 2018
Time: 11:45 a.m. – 2:15 p.m. ET
The poster is available on Tocagen’s website.

About Toca 6
Toca 6 is a multi-center, open-label Phase 1b study evaluating Toca 511 & Toca FC in patients with advanced solid tumors. The study will evaluate the safety and presence of Toca 511 genes in tumors of patients with widely disseminated disease, immunologic activity in blood and tumor, and clinical activity such as tumor response and clinical benefit. More information can be found at www.tocagen.com/toca6 or by searching clinicaltrials.gov using the clinical trial identifier NCT02576665.

About Toca 511 & Toca FC
Tocagen’s lead product candidate is a two-part cancer-selective immunotherapy comprising an investigational biologic, Toca 511, and an investigational small molecule, Toca FC. Toca 511 is a retroviral replicating vector (RRV) that selectively infects cancer cells and delivers a gene for the enzyme, cytosine deaminase (CD). Through this targeted delivery, only infected cancer cells carry the CD gene and produce CD. Toca FC is an orally administered prodrug, 5-fluorocytosine (5-FC), which is converted into an anti-cancer drug, 5-fluorouracil (5-FU), when it encounters CD. 5-FU kills cancer cells and immune-suppressive myeloid cells resulting in anti-cancer immune activation and subsequent tumor killing.

On October 29, 2018 Euronext Paris: FR0010331421 – IPH), reported new data from the Phase I clinical trial of IPH4102 in patients with relapsed/refractory cutaneous T-cell lymphomas (CTCL) (Press release, Innate Pharma, SEP 29, 2018, View Source [SID1234530303]). The data, including longer follow up for patients treated in the dose-escalation and observations from an additional patient cohort, will be presented today at the EORTC Cutaneous Lymphoma Group meeting in St. Gallen, Switzerland, by Pr Martine Bagot, Principal Investigator and Head of the Dermatology Department at the Saint-Louis Hospital, Paris. IPH4102 is Innate Pharma’s wholly-owned first-in-class anti-KIR3DL2 antibody, designed for treatment of T-cell lymphoma.

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"These data support the positive trends observed in the dose-escalation part of the trial and demonstrated a high response rate and long progression-free survival for these heavily pretreated CTCL patients, a majority being Sézary syndrome," commented Pierre Dodion, Chief Medical Officer of Innate Pharma. "Additionally, these data will serve as a basis for the initiation of a broader Phase II clinical program in Sézary syndrome and other subtypes of T-cell lymphomas. We look forward to providing more insight into the data and subsequent clinical development plans in the near future."

As of June 28, 2018, a total of 44 patients with relapsed/refractory CTCL were evaluable for safety and clinical activity. The study consisted of two parts: a dose-escalation (n=25) and a cohort expansion (n=19). Patients received a median of 3 prior systemic therapies. IPH4102 demonstrated a favorable safety profile and was well-tolerated. The study showed clinical activity that was demonstrated by a high response rate and long progression free survival.

In the total study population, the objective response rate (ORR) was 36% and median duration of response (DOR) and progression free survival (PFS) were 13.8 and 8.2 months, respectively. Sézary syndrome (SS) subset patients treated in the dose-escalation part (n=20) now show median PFS of close to 1 year. At the cut-off date of June 28, 2018, median follow-up was 12.7 months and nine patients were still ongoing treatment.

Better outcomes were observed in patients without evidence of histologic large cell transformation (LCT) (n=29); these patients achieved an ORR of 51.7% and PFS of 12.8 months. LCT is present in approximatively 10% of all Mycosis fungoides/Sézary syndrome patients* and is associated with poorer prognosis and shorter survival using currently available therapies.

"This patient population remains a high unmet medical need as they continue to progress through several lines of treatments," commented Pr Martine Bagot, Principal Investigator. "The patients with complete response, partial response and even those with stable disease showed an improvement in quality of life parameters overtime including pruritus. IPH4102’s encouraging clinical activity provides substantial support to explore its potential therapeutic benefits not only in SS patients but also in other T cell lymphoma patient populations. Together with a favorable safety profile, IPH4102 could emerge as a key therapeutic option in aggressive T-cell lymphomas."

On September 28, 2018 QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported that the U.S. Food and Drug Administration (FDA) has approved a PMA Supplement expanding the labelling claim of the therascreen EGFR RGQ PCR Kit to allow its use as a companion diagnostic with Pfizer’s VIZIMPRO (dacomitinib) for first-line treatment of patients with non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions or an exon 21 L858R mutation (Press release, Qiagen, SEP 28, 2018, View Source [SID1234529648]). The therascreen EGFR RGQ PCR kit is now approved as a companion diagnostic to guide the use of three FDA-approved therapies, including also GILOTRIF (Afatinib) from Boehringer Ingelheim and Iressa (Gefitinib) from AstraZeneca. It is registered in more than 40 countries globally. This was a project governed under an agreement between QIAGEN and Pfizer.

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"As precision medicine becomes the standard of care in oncology, we are pleased to provide benefits to more lung cancer patients with our clinically proven therascreen EGFR RGQ PCR Kit. Our collaboration with Pfizer has made great strides already and will continue to improve personalized healthcare for patients around the world," said Jonathan Arnold, Vice President, Head of Oncology and Precision Diagnostics for QIAGEN. "In addition to detecting a comprehensive panel of EGFR mutations, the therascreen EGFR kit offers laboratories an efficient workflow on the Rotor-Gene Q MDx, the real-time PCR module in our widely-used QIAsymphony family of instruments."

On September 28, 2018 KSQ Therapeutics reported that the company has secured an $80 million Series C financing to advance oncology drug candidates generated from the company’s proprietary CRISPRomics drug discovery engine into clinical studies (Press release, KSQ Therapeutics, SEP 28, 2018, View Source [SID1234529649]). With the financing, KSQ will advance its first drug program into the clinic within the next 18 months and up to three additional oncology drug programs into IND-enabling studies. The company’s first drug program is a modified adoptive T-cell immunotherapy which has shown efficacy in multiple animal models of PD-1 resistance.

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In the past 12 months, utilizing its CRISPRomics platform, KSQ has initiated and advanced 12 drug discovery programs across three oncology drug categories: adoptive T-cell therapies, immuno-oncology, and targeted therapies. In addition, KSQ is continuing to expand CRISPRomics for use in other therapeutic areas, including immunology and rare diseases. The Series C financing includes new investors Baillie Gifford, Cowen Healthcare Investments, Invus, and Lilly Asia Ventures, as well as full participation from its founding investors Flagship Pioneering and Polaris Partners, as well as existing investors ARCH Venture Partners and Alexandria Equities.

"KSQ has made remarkable progress in the past 12 months taking an unbiased, whole-genome approach to target identification with the goal of changing the probabilities of drug discovery and development. This approach has rapidly generated a broad pipeline of cancer programs and positions us to create new medicines with higher success rates and better outcomes for patients," said David Meeker, MD, Chief Executive Officer of KSQ. "The power of our platform is evident in our first drug program, a modified adoptive T-cell immunotherapy with strong activity in PD-1 resistant solid tumors."

"KSQ has made impressive progress in demonstrating the power of its pioneering CRISPRomics discovery engine," said Jim Gilbert, Senior Partner at Flagship Pioneering and Chairman of the Board at KSQ. "We see a compelling opportunity for KSQ’s high-confidence drug development approach to identify untapped opportunities and improve productivity in developing innovative medicines."

Using the genome-scale analysis of its CRISPRomics technology, KSQ has uncovered the most relevant therapeutic targets while ruling out thousands of less relevant targets at the outset. To date, KSQ has applied this high-confidence drug development approach in two areas: a tumor-genome platform for targeted cancer therapies and a T-cell genome platform for immuno-oncology monotherapies.

With the tumor-genome platform, KSQ has interrogated the function of all 20,000 human genes across more than 600 cancer models – a massive data base which allowed the company to pinpoint the optimal therapeutic targets and patient selection biomarkers for multiple cancer types.
With the T-cell genome platform, KSQ has comprehensively mined the function of all genes in the T cell in vivo, allowing identification of adoptive T-cell enabling targets and the next generation of monotherapy targets in immuno-oncology.
Dr. Meeker continued, "Our CRISPRomics engine has exceeded expectations in its performance and productivity, as it has systematically pinpointed cancer targets across two platforms – one for targeted therapies and another for immuno-oncology – and shown extraordinary scale, precision and speed in generating our pipeline of more than a dozen drug programs. With this financing and strong syndicate of investors, we are well positioned to take the next steps in realizing the potential of CRISPRomics to advance medicines that will have meaningful impact for patients."

About CRISPRomics

KSQ Therapeutics has built a genome-scale, functional-genomics drug discovery engine, called CRISPRomics, to pinpoint therapeutic nodes that directly correlate genomic function to disease with unprecedented certainty and speed. Using CRISPRomics, KSQ is elucidating the function that each human gene plays in a multitude of diseases providing a unique and more comprehensive understanding of disease biology. The quality of these insights enables KSQ to identify a multitude of high-confidence, patient-tailored, targets for drug development and rapidly rule-out thousands of less relevant targets from the outset; thereby, focusing R&D investment on the development of medicines with the greatest potential to meaningfully impact the treatment of human disease.

On September 28, 2018 Exicure, Inc. (OTCQB:XCUR), the pioneer in the development and application of three-dimensional Spherical Nucleic Acid (SNA) constructs as gene regulatory and immunotherapeutic agents, reported that Dr. David Giljohann, Chief Executive Officer of Exicure, will present at the Ladenburg Thalmann 2018 Healthcare Conference and Leerink Partners Roundtable Series: Rare Disease & Oncology. Details of these presentations are as follows (Press release, Exicure, SEP 28, 2018, View Source [SID1234529650]):

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Ladenburg Thalmann 2018 Healthcare Conference

Date/Time: Tuesday, October 2, 2018 at 2:00 p.m. EDT
Location: Sofitel Hotel, New York City
Leerink Partners Roundtable Series: Rare Disease & Oncology

Date/Time: Wednesday, October 3, 2018 at 9:30 a.m. EDT
Location: Lotte New York Palace, New York City
A live webcast of these presentations will be available on the Events & Presentations section of Exicure’s website, where it will also be archived and available for replay following the presentation for 30 days.