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Company Background 

Accelerated Medical Diagnostics, Inc. (AMD) was founded in 2008 and is enabling personalized chemotherapy for cancer patients through the development of PlatinDx, a test for predicting response to platinum-based chemotherapeutics. The test works by directly measuring in vivo the patient’s individual tumor susceptibility to platinum agents. We are presently conducting a multisite clinical feasibility study of PlatinDX for carboplatin/cisplatin in lung and bladder cancers, and a single site feasibility study oxaliplatin for advanced, metastatic breast cancer.

Technology Overview

The PlatinDx assay is a drug-device combination product that depends upon accelerator mass spectrometry (AMS) to analyze biopsy samples after patients are given microdoses of radiolabeled carboplatin or oxaliplatin. AMS is capable of detecting as little as one 14C atom per human cell, which enables administering humans with extremely low “microdoses” of labeled drugs for diagnostics purposes. Microdosing creates very low levels of a transient, radiolabeled biomarker that allows the in vivo measurement of the pharmacodynamic effect of this drug via the formation of drug-target complexes (adducts). The measurement of this label by AMS forms the basis for the ability of the PlatinDx test to predict response to subsequent full dose platinum-based therapy. The assay will identify those potential responders who have relatively high drug-DNA adducts and are likely to respond to platinum-based chemotherapy

Market Potential

The addressable market for PlatinDx is $500 million for the United States lung cancer market, $250 million each for the U.S. bladder and breast cancer markets, and $3-$5 billion globally for multiple cancers and drugs.

Competitive Advantage

PlatinDx directly measures in vivo the patient’s individual tumor susceptibility to specific chemotherapeutic agents, which will improve survival for one group and reduce acquired drug resistance caused by needless exposure of non-responders to platinum-based therapy. PlatinDx does not require patient or tumor genotype information or culturing of tumor cells. Other tests rely on quantitation of a single or a few gene mutations or expression levels, which is inadequate for prediction of platinum-based drug efficacy. FDA approval will lower barriers to reimbursement.

Financial Overview

AMD has been primarily funded through NIH/NCI contracts to develop PlatinDx for bladder and lung cancer. The company has raised over $2 million since 2011. Currently accruing patients for feasibility trials, AMD has funding for operations through early 2015. The company will be raising $32 million in additional capital over the next five years to complete the pivotal trials, establish the clinical and sales teams, develop the reimbursement and manufacturing processes, obtain CLIA certification, and gain FDA approval. Product launch is set for 2019.

Intellectual Property

AMD is presently refining and reducing to practice specific diagnostic methods for platinum-based chemotherapeutics through clinical evaluations. The company is pursuing protection of the PlatinDx platform via a series of patent filings for the global market, the first of which is a U.S. patent expected to be filed in November 2014. Microdose-based diagnostic applications to be covered include specifics of the microdose-based assay used for human studies, including useful drug concentrations, formulations, and specific activities, the resulting useful drug-DNA adduct frequencies in patients induced by microdosing, and sample processing methods optimized for AMS analysis. When issued, patent protection should extend until at least 2034. In addition to patent protection, FDA approval of the drug component as a “similar drug” will result in a period of market exclusivity for the drug component.

Commercialization Strategy

AMD is seeking regulatory approval for its diagnostic assays. AMD has key opinion leaders participating in the clinical trials and expects them to be early adopters upon launch. AMD plans to do a health economic study to justify reimbursement. AMD plans to develop regional laboratories that will serve U.S. and international markets. As an FDA approved test, AMD can license to large diagnostics companies.

Pipeline Products

The PlatinDX assays under development predict patient response for the three approved platinum chemotherapy agents (cisplatin, carboplatin and oxaliplatin) in the lung, bladder and breast cancer areas. The platform technology will be applied to other cancer drugs and tumor types.

Management Team

• CEO and founder Paul Henderson, PhD is a UC Davis faculty member with 15 years of experience in designing and implementing experiments to assess drug resistance, mostly with respect to accelerator mass spectrometry analysis.
• Vice president of development George Cimino, PhD is the co-founder and former vice president of development for Cerus Corporation and has over 30 years of research experience with small molecules that interact with DNA and 25 years of experience in bringing regulated drug/device combination products to market, including CE Mark and FDA approvals.
• Founder Chong-xian Pan, MD, PhD is a UC Davis faculty member and practicing medical oncologist who led the clinical implementation of two trials for PlatinDx.

Actinobac Biomed is conducting IND-enabling studies for their primary drug candidate, Leukothera®, a highly effective therapeutic for the treatment of hematological malignancies including B-cell lymphoma. Leukothera™ is a naturally derived biologic that specifically targets the subset of white blood cells (WBCs) expressing activated leukocyte function antigen-1 (LFA-1). Since LFA-1 is only present on WBCs and its activated form is uniquely expressed on cancerous and hyper-inflammatory WBCs, other cells and tissues are not affected, minimizing negative side effects.

Actinobac has exclusively licensed the patent portfolio established by Rutgers University covering the use of Leukothera® for the treatment of multiple medical conditions including cancer, autoimmune/inflammatory diseases, and HIV infection. The company has raised $960,855 since 2009 and is presently seeking funds to support the GMP production and formulation ($700,000) of their drug candidate. This high quality material will be used to complete preclinical research and development activities ($900k), stability and storage testing ($500k), the assembly and filing of regulatory documentation and an IND application ($1 million), and perform safety and preliminary efficacy clinical studies ($1.5 million). Actinobac is presently in discussions with a number of major pharmaceutical companies and investment groups to provide scientific support and financing for these activities.

Technology Name:  Cytomegalovirus Therapeutic Vaccine – Glioblastoma and other cancers 

Company Background

Annias Immunotherapeutics, Inc., is a biopharmaceutical company focused on the development of novel immunotherapeutic approaches to treat cancer.  The company was founded in 2009 by Senior Investigators at the Duke Medical Center and is led by CEO Reiner Laus, MD. It is based on a patented and proprietary immunotherapeutic platform, discovered by John Sampson and Duane Mitchell at Duke University that targets human Cytomegalovirus (CMV). CMV is over-expressed in a variety of human cancers including significant and homogeneous expression in almost all glioblastoma (GBM) but not in normal brain tissue.  Annias Immunotherapeutics is focused on this promising opportunity to utilize CMV proteins as tumor-specific targets.  Additionally, the company has developed superior methods to immunize humans against cancer, and has already tested this approach in human clinical trials. This novel immunotherapeutic platform has shown extraordinary promise in eradicating GBM with virtually no toxicity experienced in the treated patients.

Technology Overview

Annias has exclusive rights to the next generation of proprietary immunotherapeutic platforms for peptide-based and dendritic cell approaches developed at Duke by Senior Investigators in the Preston Robert Tisch Brain Tumor Center who have completed two consecutive clinical trials using CMV pp65 loaded dendritic cells (DCs) in patients with GBM. The first of these trials was randomized and blinded and demonstrated the induction of CMV-specific immunologic responses along with remarkable progression-free (PFS) exceeding 36.6 months vs. 10.8 months for the control group and a median overall survival (OS) exceeding 36.6 months vs. 18.5 months for the control group.  The CMV vaccine is currently being reformulated into a cutting-edge rationally designed multi-epitope peptide conjugate, PEP-CMV. This proprietary technology platform combining chemotherapy and vaccination will be used to clinically evaluate the efficacy and immunologic effects of PEP-CMV vaccines in patients with newly diagnosed GBM. The poor prognosis for patients with GBM will enable Annias to obtain efficacy data from a randomized, controlled trial within two years of trial initiation. Product candidates based on this platform can be rapidly advanced in breast cancer, prostate cancer, and colorectal cancer.

Market Potential

Despite aggressive treatment, GBM remains uniformly lethal with median survival rates for patients with GBM being less than 15 months from the time of diagnosis. Thus, there is a large and urgent need for improved therapies for GBM. World-wide sales of the chemotherapeutic temozolomide (Temodar®) for GBM have reached one billion dollars in spite of its very limited efficacy. The markets for the products in the Annias pipeline for treatment of breast, colorectal, and prostate cancer present multi-billion dollar opportunities.

Competitive Advantage

The recent discovery and confirmation by five independent laboratories, that CMV propagates within a high proportion of GBMs, without infecting surrounding normal brain tissue provides an unparalleled opportunity to utilize the highly immunogenic antigens from CMV as tumor-specific targets. A distinct advantage of immunotherapeutic targeting of viral antigens is that the immune system is better equipped to target viral antigens compared to autoantigens. The frequency of CMV-specific T-cells is several orders of magnitude higher than what can be achieved with approaches targeting autoantigens.

Financial Overview

Annias secured $246,000 in NCI Phase I SBIR funding in 2012 and has recently submitted a Phase II application for $1.9 million. Through the Duke University Medical Center, several million dollars were secured in grant funding for CMV-targeted vaccine research.  The company expects that taking PEP-CMV through a randomized Phase II program, developing its pipeline products, and operating the company over the next four years will require significant incremental investment.

Intellectual Property

The company has exclusive rights to the Duke University portfolio of Immunotherapy patents. This portfolio includes both issued and pending patents, as well as published and as of yet unpublished patent applications. The issued patent includes claims that cover Annias approach both broadly (immunotherapy of cancer by targeting CMV) and narrowly (composition of PEP-CMV therapeutic).

Commercialization Strategy

Annias plans to start the next clinical trial for PEP-CMV this year. The initial capitalization of the company will serve to conduct and collect data from a large-scale, randomized controlled clinical trial for PEP-CMV. This trial will position the company well for product registration, partnering, and further development of its pipeline in additional diseases.

Pipeline Products

The company is developing PEP-CMV with GBM as the lead indication. Follow-on indications that can be targeted with the same product include the four leading cancers: prostate, breast, lung, and colorectal cancer.

Management Team

• CEO and President Reiner Laus, MD. Dr. Laus has worked on developing cancer immunotherapeutics for over 20 years. He was most recently CEO of BN Immunotherapeutics and prior to that he was VP of R&D at Dendreon, where he was a co-inventor of Provenge.
• Chief Scientific Collaborator and Founder, John Sampson, MD, PhD, is leader of the Neuro-Oncology Program at Duke University and head of the Brian Tumor Immunotherapy Program there. He has published extensively on CMV as a tumor-specific antigen and immunotherapy approaches. His laboratory at Duke developed the EGFRvIII-targeted vaccine which is now completing worldwide Phase III trials in patients with GBM.
• Vice president and corporate secretary James Sheldon was founder of Embrex, Inc. (acquired by Pfizer) and EnSys, Inc. (merged into SDOI).

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.