KSQ Therapeutics Secures $80 Million Financing and Advances Broad Pipeline of Cancer Therapies Including Its First Adoptive T-Cell Therapy Program for PD-1 Resistant Solid Tumors

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.

Exicure, Inc. to Present at Upcoming Investor Conference and Participate in Oncology Roundtable

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.

Genprex™ Taps Aldevron to Supply TUSC2 Plasmid DNA for Oncoprex Clinical Development Program

On September 27, 2018 Genprex, Inc. (NASDAQ:GNPX), a clinical stage gene therapy company developing a new approach to treating cancer based upon a novel proprietary technology platform, reported that it has signed an agreement with Aldevron, a leading contract manufacturing organization, to supply TUSC2 (Tumor Suppressor Candidate2) plasmid DNA for use in Genprex’s clinical development program evaluating its immunogene therapy Oncoprex for the treatment of non-small cell lung cancer (Press release, Genprex, SEP 27, 2018, View Source [SID1234529631]).

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"Plasmid DNA plays an important role in the delivery of gene therapies into target cells," said Rodney Varner, Chairman and Chief Executive Officer of Genprex. "Aldevron is a leading plasmid manufacturer that offers world-class laboratory and manufacturing programs. Through this agreement, we have established a GMP-validated supply of plasmid DNA vectors for our expanding clinical program evaluating the utility of Oncoprex in combination with some of the most promising new therapies for cancer."

"Aldevron is honored to partner with Genprex in the development of Oncoprex," said Michael Chambers, CEO of Aldevron. "This collaboration furthers our goal to provide high-quality plasmid DNA and other biologics to leading companies such as Genprex that are creating treatments that could have a significant impact on human health."

PharmaCyte Biotech Successfully Completes Comparative Analysis of Methods for Measuring Viability of Encapsulated Cells

On September 27, 2018 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a clinical stage biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that it has successfully completed a study to determine the best assay method for the accurate determination of the number of viable encapsulated cells at any given time inside PharmaCyte’s Cell-in-a Box capsules, which will be used for the treatment of locally advanced, non-metastatic, inoperable pancreatic cancer (LAPC) (Press release, PharmaCyte Biotech, SEP 27, 2018, View Source [SID1234529632]).

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The decision on which viability assay to use is an important component required for the filing of PharmaCyte’s Investigational New Drug Application (IND) with the U.S. Food and Drug Administration (FDA), since the viable cell assay is needed to show how many living cells are in the capsules at any given moment. This is a parameter that influences the biological activity of PharmaCyte’s Cell-in-a-Box encapsulated cell product.

This particular study compared three different methods for determining the number of viable cells in the capsules as well as the growth rate of the cells within the capsules. Although the rate of growth has already been fixed as part of the production process (and is not affected by this study), the new data revealed that only one of the tested methods can accurately estimate the number of cells within a capsule, particularly when the capsules are populated at high cell densities as they will be for use in PharmaCyte’s clinical trial in patients with LAPC. Therefore, this study provides the justification for the use of the most accurate and sensitive assay chosen by this study, which will also be used for quality control of the product release for the upcoming planned clinical trial for LAPC.

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, explained the significance of the study saying, "The cells that are encapsulated using the Cell-in-a-Box technology are the engine for the final product in that they activate the chemotherapeutic agent ifosfamide at the site of the tumor in the pancreatic cancer patient. This study is important since it shows we now have at our disposal the best method to determine the actual number of living cells in the capsules.

"The data from this study is another key piece of information required for PharmaCyte to comply with FDA guidelines and recommendations for our planned, upcoming clinical trial in patients with LAPC. This viability information must be reliable; therefore, it is also valuable for ensuring good quality control of our final product. In other words, using the chosen viability measuring method will help us ensure that the Cell-in-a-Box encapsulated product is reproducible from batch to batch. This is an essential requirement for any medicinal product."

Tocagen to Participate in Four Investor Conferences in October

On September 27, 2018 Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, reported that members of the leadership team will present at the following upcoming investor conferences in New York City (Press release, Tocagen, SEP 27, 2018, View Source;p=RssLanding&cat=news&id=2369348 [SID1234529674]):

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Tuesday, October 2, 10:30 a.m. ET
Ladenburg Thalmann 2018 Healthcare Conference
Presenter: Chief Executive Officer Marty Duvall

Wednesday, October 3, 8:00 a.m. ET
Cantor Global Healthcare Conference
Presenter: Chief Executive Officer Marty Duvall

Wednesday, October 3, 9:00 a.m. ET
Leerink Roundtable Series: Rare Disease & Oncology
Presenters: Chief Financial Officer Mark Foletta and Chief Medical Officer Asha Das, M.D.

Tuesday, October 9, 3:45 p.m. ET
Chardan Capital Markets 2nd Annual Genetic Medicines Conference
Presenter: Chief Executive Officer Marty Duvall

The live audio webcasts from the conferences and subsequent replay may be accessed by visiting the "Events & Presentations" page in the investors section of Tocagen’s website. The webcasts will be available shortly after conclusion of the presentation and archived on the company’s website for 90 days following the presentation.