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Company Background 

CellSight Technologies is a privately held company with six employees located at the UCSF QB3 incubator in San Francisco.  The company started operations in 2010 on the principle of providing innovative new PET imaging technologies to accelerate therapy development and to leverage these imaging tools to personalize patient treatments.

Technology Overview

CellSight is developing a PET probe, [18F]FAraG, for imaging anti-tumor immune response. [18F]FAraG is a fluorine-18 labeled analog of the lymphoblastic leukemia drug AraG, a guanosine analog. Since [18F]FAraG accumulates specifically in activated T cells, which is a major factor in anti-tumor immune response, detection of activated T-cells should enable early prediction of therapeutic efficacy, optimization of immunotherapeutic regimens, and personalization of immunotherapy.  Cellsight has other PET probes in various stages of development targeted at immunotherapy and cell therapy.  In addition CellSight has reporter gene technology to determine trafficking of therapeutic cells in living subjects, including humans.

Market Potential

Immunotherapy is a rapidly expanding market segment due to demonstrated success in various hard to treat cancers such as melanoma and glioma. ClinicalTrials.gov shows that there are currently 90 industry-sponsored, cancer specific cell-based immunotherapy trials actively recruiting, of which 60 trials are in the U.S. According to a June 2014 report by Citigroup, the immunotherapy market could exceed $35 billion by 2023. There is currently no imaging technology on the market that is focused on imaging immune activation in humans. An imaging tool that can help predict a patient’s response to an immunotherapy early could enable patients to find effective therapies and reduce unnecessary medical costs.

Competitive Advantage

There are no commercially available imaging technologies that help visualize the biodistribution of activated T-cells. As immunotherapies gain traction based on their clinical success, there will be increased demand for a companion test to determine if an immunotherapy agent is effective with a particular patient.  A scan that can be used to visualize immune cell activation post infusion or after treatment with immune modulating agents would provide invaluable information to guide future treatment options. 

Financial Overview

CellSight has received over $3 million in grants from NCI. The annual revenue from grants, product sales, and service contracts totaled $377,045 in 2011, $430,864 in 2012, and $669,707 in 2013 our estimate for 2014 is $ 1.1M.

CellSight is seeking a minimum of $7 million in external funding beyond the nondilutive grant funding. The company plans to obtain $10 million in grants and private funding in order to conduct a Phase I/II clinical trial for the [18F]FAraG imaging probe. CellSight will also continue to provide selective fee-based imaging services that have contributed about $200,000 per year.

Intellectual Property

CellSight has exclusively licensed the [18F]FAraG PET probe from Stanford and has two other patents focused on reporter gene technology.  The patent from Stanford includes claims on the composition of matter, methods of synthesis, and methods of use.  Also CellSight personnel are sponsors for an FDA IND for [18F]FHBG PET reporter imaging probe, which is used to image patients undergoing HSV1-tk gene therapy.  In addition, the company founder is inventor of several other immunotherapy/cell therapy focused PET probes and has expressed a strong desire to commercialize them through CellSight given the right opportunity and financial conditions.

Commercialization Strategy

CellSight is in the process of applying to the FDA for an IND approval for [18F]FAraG and expects approval by December 2014. The company’s initial focus is the oncology market where we hope to fully partner with immunotherapy companies and offer the probe as a companion-imaging tool. We are in the early stages of partnering with two large pharma companies and are actively seeking additional partnerships.  CellSight plans to expand the probe’s use to other markets such as rheumatoid arthritis and diabetes mellitus where immunotherapy is in early stages.

Pipeline Products

CellSight intends to expand the probe for use in the rheumatoid arthritis and diabetes mellitus markets as the immunotherapy models for these applications mature and our company’s capacity expands.  In addition we will exclusively license two additional PET probes that our founder has developed for immunotherapy and cell therapy.  The additional PET probes are specific to receptors on immune cells and could be used to image and monitor other processes affected by immunotherapies beyond T cell activation.

Management Team

• Founder Sam Gambhir is Chair of Radiology at Stanford and is a world-renowned pioneer in the field of molecular imaging.
• CEO Aruna Gambhir has over 25 years of broad experience in startups, R&D, sales, marketing, and operations.
• CSO Shahriar Yaghoubi has over 18 years of experience in molecular imaging, including pre-clinical and clinical assessment of novel PET probes and FDA experience with IND.
• COO Sam Quezada has over 30 years of experience in marketing, business development, and operations.

Technology Name: 3D Cell-based Cancer Diagnostic

Company Background

KIYATEC is a privately held company that has developed a platform to enable accurate ex vivo prediction of cancer patients’ response to drug treatment.  Incorporated in 2010 by co-founders David Orr and Matt Gevaert, the company has created novel 3D cell-based models for drug response profiling that can generate information relevant to preclinical testing, clinical trials, and clinical diagnostics applications. By accurately predicting patient drug response without exposing patients to drugs, KIYATEC will enable informed drug selection that minimizes clinical trial failures and maximizes patient outcomes.

KIYATEC is located in Greenville, SC and is based in the Greenville Health System’s Institute for Translational Oncology Research (ITOR) which conducts phase I clinical trials and is a life science incubator for companies pursuing translational cancer therapeutics and/or diagnostics. KIYATEC currently has 9 employees.

Technology Overview 

KIYATEC’s  3DKUBE™is a technology platform that uses human clinical samples to generate 3D microtumors to accurately predict human response to cancer drugs ex vivo. The 3DKUBE™ is a 3D perfusion bioreactor that allows for co-culture of the different cell types found in tumors, and incorporates optimized 3D scaffolds and media conditions. In addition to many cell lines, KIYATEC has tested clinically annotated, patient derived primary ovarian and breast cancer cells in its drug response profiling and is expanding into primary Glioblastomas (GBMs).  KIYATEC’s 3D microtumors can be used in preclinical drug evaluation, as a co-clinical trial patient selection tool, and as an ex vivo diagnostic for real time clinical decision making.

Market Potential            

The global cancer/tumor profiling market was valued at $13.3 billion in 2012 while the global market for cell-based assays for drug discovery was valued at $6.2 billion in 2010. The US total available market opportunity for cell-based ovarian cancer, breast cancer, and GBM cancer diagnostics is approximately $1 billion. KIYATEC’s live cell cancer diagnostics will enable response based drug selection and will be offered via a laboratory service model.

Competitive Advantage

KIYATEC’s drug response profiling platform uses a 3D microenvironment to culture cells, enabling better prediction of clinical outcomes than 2D microenvironments. In KIYATEC’s Phase I contract, cells cultured in an actively perfused microenvironment had increased cell viability, richer paracrine interactions, and increased cell functionality compared to static cultures. The 3D cultures are maintained as closed systems without manipulations, saving time and cost, decreasing contamination risk, and increasing scalability. KIYATEC’s demonstrated ability to choose and utilize the best scaffold for a given cell type maximizes the number and type of tissues that can be effectively modeled and maximizes the potential for multi-cell interaction for better cell function. KIYATEC’s exceptional clinical connectivity results in more effective procedures with the clinical partner and more successful translation of laboratory results back into the clinic, aspects not possible for competitors relying on third party primary tissue sourcing.

Financial Overview

KIYATEC has secured over $5.1 million in committed capital since 2008 ($2.6 million in investment; $2.5 million non-dilutive funding including a recently awarded NCI Phase II contract) and is currently working to raise a series B round of $12.5 million, with an initial tranche of $7 million to support company milestones. 

Intellectual Property

KIYATEC’s IP portfolio consists of an exclusive license to issued utility patents (product and method claims).  The company has assignment of issued design patents and pending utility filings associated with the core technology for applications in drug screening, cancer diagnostics, and 3D cell-based assays.

Commercialization Strategy

KIYATEC is currently generating revenue from late stage preclinical 3D cell-based assay services for pharma, biotech and CRO clients with potential to expand quickly into co-clinical trials.  Future ex vivo cancer diagnostics that enable response based drug selection will be offered as a CLIA lab based service offering either as an LDT or as an FDA regulated diagnostic as required.

Pipeline Products

KIYATEC’s first generation platform (3DKUBE™) has been tested with patient derived ovarian and breast cancer cells / tissues; GBM studies will begin in Q4, 2014.  A second generation, higher throughput platform is under development.  KIYATEC’s platforms can be further expanded into additional solid tumor types (e.g. lung, colon, pancreas, and liver).

Management Team

• CEO Matt Gevaert, Ph.D., has IP commercialization/technology startup experience as commercialization officer at Clemson University and has experience with Merck and 3M.
• COO David Orr, Ph.D., MBA, inventor of KIYATEC technology, has biomedical, industrial operations and financial management experience at Cook Medical, Ingersoll-Rand, and Dwyer Instruments.
• Chief Medical Officer Hal Crosswell, M.D., is a practicing oncologist and the lead investigator for more than 40 industry and NCI-sponsored clinical research studies.
• Sr. Director of Business Development Bryce Chaney, MBA, has business development and technical experience at Cirrus Pharmaceuticals, Azopharma, Scynexis and BD Technologies.
• Chairman of the board Robert Silverman is a diagnostics industry veteran whose last company was acquired by Roche for $270 million. 

Vaxiion Therapeutics is dedicated to the further development of VAX-IP, a promising investigational bacterial-minicell based biologic product initially intended for the topical intravesical treatment of Non-muscle Invasive Bladder Cancer (NMIBC).  VAX-IP exerts rapid selective tumoricidal effects against human urothelial carcinomas in addition to synergistic secondary immunotherapeutic effects, a combination unlike that of any other product under development or on the market in the NMIBC space. With compelling pre-clinical efficacy data in several clinically relevant variations of the gold standard mouse model of NMIBC, Vaxiion is now committed to the commercial and clinical development of VAX-IP for initial use as an intravesical agent for the treatment of NMIBC.   Currently, Vaxiion is initiating IND-enabling studies with the intention of filing an IND application with the FDA in Q1 of 2016.  Clinical trials would focus on serving as a salvage therapy for BCG-refractory and BCG-intolerant NMIBC patients, and if successful,  quickly be expanded to other areas of unmet need within the NMIBC treatment algorithm.

Vaxiion estimates the market size for an effective and reimbursable bladder cancer product addressing this admixed disease niche to be ~$937 - ~$1.87 billion depending on the number of approved indications. The company feels that it will take ~$7-10M and 3.5-4 years to complete two Phase 2a efficacy studies and demonstrate human proof-of-concept. Vaxiion has raised ~$5 million for this effort to date, and is looking for $3-$4 million in addition to proceeds from a pending SBIR Phase II award to advance the program through this inflection point.

Technology Name: Small molecule CDK8/19 inhibitor

Company Background

The mission of Senex Biotechnology is to develop novel therapeutics for the treatment of cancer and other major diseases by targeting key disease-promoting pathways induced by cellular damage and aging, and by identifying and attacking novel cancer-specific molecular targets. Founded by Dr. Igor Roninson, on the basis of discoveries in his laboratory in 2002, the company is located in Columbia, SC, and currently supports three scientists.  Senex has won a series of grants, including two Phase II SBIR grants, and concluded a strategic licensing agreement with a foreign pharmaceutical company in 2014. Senex has identified several novel targets and generated first-in-class small molecules against three of these targets. The drug for the most advanced program is about a year from clinical trials.

Technology Overview 

Senex’s most advanced program targets CDK8/19, a transcription-regulating oncogenic kinase. CDK8/19 inhibition has multiple anti-cancer effects at the molecular level, including the inhibition of oncogenic transcription factors, as well as stimulating immune surveillance by NK cells. The lead molecule, Senexin B, is fully optimized and exceptionally selective for CDK8/19. It is orally available, non-toxic, and extremely potent in numerous in vivo studies: Senexin B directly suppresses prostate and breast tumor growth, exhibits strong synergistic effects with several widely used drugs, and has anti-metastatic activity in several cancer types.

Market Potential

Senex will initially develop Senexin B for treatment of metastatic castration-resistant prostate cancer (mCRPC), the second leading cause of cancer related death in the United States. Over 29,000 people die from prostate cancer every year; 1 out of every 36 men will die from prostate cancer.  Median survival for mCRPC is less than 2 years.  Although several new drugs have recently been approved, resistance to these drugs develops within several months so there is a large unmet need.  Senexin B acts against those cancers that do not respond to any class of androgen receptor inhibitors, including cancers that are resistant to the newly approved drugs Xtandi and Zytiga.

Competitive Advantage

CDK8/19, Senex’s primary target, belongs to the CDK family, but unlike better-known CDKs, CDK8/19 does not mediate cell cycle progression, and is not required by normal cells under homeostatic conditions. As a consequence Senexin B is extremely well tolerated. Senex is the only company to describe selective CDK8/19 inhibitors in a peer-reviewed article. Based on the known poster presentations and published patent applications, CDK8/19 inhibitors have been recently developed by Selvita (Poland), Bayer Pharma, and CNIO (Spain). Based on the available information, none of the competitors’ compounds appear to be as selective as Senex’s CDK8/19 inhibitors, and no comparable in vivo studies have been reported.

Financial Overview

Senex has received over $2.8 million in NIH funding and over $3.5 million from other sources, including angel investors, licensees, charitable foundations and the DOD. Senex is seeking $10 million to fund additional pre-clinical and clinical studies through proof-of-concept in castration-resistant prostate cancer.  The plans for this study have been developed with a NCI-designated cancer center.

Intellectual Property

The key issued patents for Senex’s CDK8/19-related IP are US patents 8,598,344 protecting the composition-of-matter of its CDK8/19 inhibitors and 8,592,147 protecting the general screening method for identifying inhibitors of transcriptional pathways including those regulated by CDK8/19. Senex also has several pending utility patent applications protecting other novel applications of CDK8/19 inhibitors that Senex and its collaborators have discovered.

Commercialization Strategy

Senexin B has been licensed to a foreign pharmaceutical company for minor markets. Marketing rights for all major markets are retained by Senex. The licensee will provide Senex with GMP manufactured Senexin B, the results of FDA acceptable preclinical safety studies, and clinical trials that will be conducted to international GCP standards. Senex will receive milestone payments and royalties from sales of drugs in the licensee’s minor markets. These results will enable Senex to partner with a major pharmaceutical company to perform additional clinical trials and to market the drug.  Anticipated milestone payments will fund other programs in Senex’s pipeline.

Pipeline Products

Senex also has programs targeting CDK3 and COPZ1. Senex has identified CDK3 as a cancer-specific target and is optimizing the first CDK3-selective small molecule inhibitors. COPZ1 is a component of the vesicle-coating complex and Senex has discovered the first COPZ1-targeting small molecules. COPZ1 inhibition should kill most types of tumor cells, including dormant cells and cancer stem cells, which are resistant to conventional therapy.

Management Team

• President and CSO Igor Roninson is the founder of Senex and the inventor on 41 issued US patents.
• CEO Lawrence Friedhoff has a long history of successful and rapid FDA approval of new drugs, including two blockbusters, one of which is Aricept, the main drug used to treat Alzheimer’s disease.
• Karthik Gopalakrishnan brings several years of experience in business development and negotiation skills to Senex.  He has successfully concluded business transactions with several pharmaceutical companies.

Company Background

Avidity NanoMedicines is pioneering a new class of therapeutics, antibody siRNA complexes (ARCsÔ), which draw on the best features of antibody-drug conjugates and nucleic acid-based medicines.  Through partnerships with academic and industry experts, Avidity is applying its technology to the discovery and development of novel, targeted drugs. Founded in 2013, the company completed a $9 million Series A financing in January 2014 and entered into a collaboration with a major pharmaceutical company.

Technology Overview 

ARCs™ are self-assembling, polymeric nanoparticles that encapsulate one or more siRNA and are decorated with monoclonal antibodies for cell-specific binding and internalization. Avidity’s core technology was invented by Mark Davis, professor of chemical engineering at Caltech. ARCs™ represent the culmination of Professor Davis's nearly two decades of R&D in nanomedicines and are optimized for distribution, half-life, cell internalization, and tolerability. In the SBIR Phase I project, Avidity has assessed the feasibility of the ARCs™ via 1) identification of candidate siRNAs via bioinformatics and in vitro screening, 2) characterization of physicochemical properties of siRNA-containing ARCs™, 3) demonstration of siRNA-mediated target knockdown and induction of apoptosis in cultured cells, and 4) evaluation of target knockdown and tumor regression in a xenograft tumor model.

Market Potential

By utilizing nano-scale self-assembly to combine the potency and specificity of biologics with siRNA payloads, ARCs™ create a disruptive approach to the treatment of cancer and other serious diseases. Preliminary results suggest that treatment with an ARC™ is superior to treatment with native antibody alone and results in profound tumor regression in mice. Avidity currently has a strategic collaboration with a top 10 pharmaceutical company and seeks to create exceptional collaborations around specific ARC™ products in cancer and other serious diseases based on its breakthrough technology. Importantly, Avidity plans to advance independent programs through clinical proof-of-concept utilizing proceeds from collaborations in addition to venture financing.  

Competitive Advantage

ARCs™ overcome the cell delivery barrier that has historically limited siRNAs to primarily targeting liver diseases. The physicochemical properties (e.g., size, charge, stability) of ARCsTM have been optimized for in vivo delivery of siRNAs to solid tumors. Thus, ARCsTM allow gene targets to be silenced with exquisite specificity and efficiency, and have the potential to exceed the efficacy of existing drugs.

Financial Overview

Avidity receives full-time equivalent (FTE) revenue and reimbursement of direct research expenses through an Evaluation Agreement with a large pharma company. Avidity was awarded a Phase I SBIR grant in December 2013 and completed a $9 million Series A financing in January 2014. The company will close a $5 million convertible note in fall 2014.

Avidity forecasts having adequate resources to allow for advancement of the candidate therapeutic through IND-enabling animal studies and for completion of a Phase I clinical evaluation in cancer patients.

The company intends to raise additional funding to support continued clinical development through Phase II and Phase III clinical studies, culminating with NDA submission.

Intellectual Property

Avidity licensed the intellectual property for the cMAP-based nanoparticle system from Caltech, including a patent issued in 2009. The company is filing provisional and non-provisional patent applications for specific candidate siRNAs and will seek and required licenses or agreements at an appropriate stage of product development.

Commercialization Strategy

Avidity plans to initiate a second pharma research collaboration, nominate 1-2 development candidates, close a convertible note and Series B financing, and have the first-in-human dose within the next two years.

Pipeline Products

Avidity is advancing multiple ARCs™ for the treatment of cancer, with a focus on solid tumors.

Management Team

• Troy Wilson, president and CEO, is also the president and CEO of Wellspring Biosciences and its affiliated company, Araxes Pharma.
• Kent Hawryluk, chief business officer, has been an entrepreneur for 25 years and is also a partner of Twilight Venture Partners.
• Arthur Levin, executive vice president, R&D, has an immense background in nucleic acid-based therapeutics and previously held the same position at miRagen Therapeutics.

Company Background

Modulation Therapeutics is an early discovery and research startup company, which has been spun out of the Moffitt Cancer Center as part of their initiative to commercialize the research being conducted at the research facility. Modulation is dedicated to the development of novel peptidomimetic drugs for multiple myeloma (MM) and other cancers that home to bone. The company is currently focused on development of its lead candidate, MTI-101, a novel drug with a first-in-class mechanism of action.

Technology Overview 

MTI-101 is a cyclic peptidomimetic  that binds  CD44 and induces an agonistic signal leading to toxic increases in the levels of intracellular Ca2+ that trigger cell necrosis in MM cells. Recent evidence indicates that metastatic tumors rewire their Ca2+ circuitry. We propose that rewiring of Ca2+ signaling renders tumor cells vulnerable to Ca2+ overload. The in vivo efficacy of Modulation’s lead compound, MTI-101, has been demonstrated as a single agent and in combination with the proteasome inhibitor bortezomib using two independent MM in vivo models that considers the tumor microenvironment.  Importantly, MTI-101 shows increased activity in primary MM specimens obtained from patients who have relapsed on therapy; a finding that has been used to develop predictive biomarkers to be further explored in early phase clinical trials.

Intellectual Property

Modulation Therapeutics has licensed MTI-101 and several analogs from the Moffitt Cancer Center. The composition of matter and use patent was filed in 2012. Additional filed patents include coverage of analogs, combination strategies as well as biomarkers of response.

Market Potential

Multiple Myeloma is a disease that initially responds to therapy. However, inevitably all patients will relapse with disease that is refractory to standard of care agents. Thus novel treatment strategies are required to improve patient outcome. The market for MM is very large, and with improved diagnosis/detection combined with an aging population, is projected to exceed $10 billion worldwide by 2018.

Competitive Advantage

There are no drugs on the market or in development that would provide direct competition to Modulation’s lead candidate, MTI-101.

• MTI-101 induces necrotic cell death in myeloma cells and thus does not require the apoptotic machinery to induce cell death.
• Inhibits osteoclast formation and function and augments differentiation of osteoblasts using in vitro cultures and demonstrates decreased myeloma induced lytic bone lesions in vivo. MTI-101 uniquely targets the both the tumor and the bone marrow niche.
• Shows increased ex-vivo activity in specimens obtained from patients relapsing on therapy.

The crucial unmet need in myeloma is for drugs effective in patients that have relapsed on existing therapies. Modulation Therapeutics is well positioned to develop MTI-101 for the treatment of drug refractory myeloma.

Financial Overview

Modulation is an early-stage, pre-clinical research organization and has not generated any company revenues to date, nor have they developed any revenue projects beyond understanding the potential size of the market opportunity.

The company has raised over $1.7 million to date through a combination of research grants, foundation investments, and local commercialization debt.

Commercialization Strategy

Modulation is looking to raise $3 million to build and fund the infrastructure and partnerships required to plan, manage, execute, and document the IND-enabling studies to include the IND application and approval process. Modulation expects to complete the IND approval process within 18 months of receiving the required funding. Currently, Modulation has been able to advance the development of MT-101 through initial PK studies.

Pipeline Products

The initial focus for Modulation is on MTI-101 for the treatment of MM. Additional indications currently being validated are EGFR driven lung cancer, metastatic prostate and breast cancer. Targeting antibody conjugation strategies are in development and the patent has been filed for this strategy. Moreover a peptoid-peptide library is currently in development for covering additional chemical space surrounding MTI-101.

Management Team

• Co-founder and CSO Lori Hazlehurst, PhD is an expert in defining strategies to target the tumor microenvironment and is the co-inventor of multiple patents, including MTI-101.
• Director of Synthetic Chemistry Mark McLaughlin, PhD is an experienced synthetic organic and peptide chemist with more than 100 publications, is a co-inventor of MTI-101, and a senior member of the Moffitt Cancer Center.
• Board Member William Dalton, MD, PhD is the current CEO of M2GEN and former CEO of the Moffitt Cancer Center.
• Board Member Anne Cress, PhD is a professor of Cellular & Molecular Medicine at the University of Arizona, and Deputy Dean of Research.