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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.

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.

Prof. Suzie Chen and her colleagues at Rutgers University have recently discovered that the metabotropic glutamate receptor 1 (GRM1, mGluR1) plays a significant role in the development and growth of melanoma tumors.  They found that riluzole, the only drug approved by the U.S. FDA for the treatment of amyotrophic lateral sclerosis (ALS), which also blocks glutamate release from GRM1 cells, inhibits melanoma growth and proliferation both in vitro, in mouse xenograft models, and in limited human clinical trials in melanoma patients.  However, the clinical use of riluzole for ALS, and potentially for melanoma, is severely limited by variable CYP1A2-mediated first pass metabolism.  Fox Chase Chemical Diversity Center, Inc. has developed novel and innovative prodrug derivatives of riluzole which avoid CYP1A2 in vitro metabolism and are expected to provide more regular pharmacokinetics and exposure in patients, with a longer half-life, allowing for once daily oral dosing versus the current twice daily dosing that is required, and potentially greater patient compliance.  

The company has filed several PCT provisional U.S. patent applications on riluzole prodrugs, with additional composition of matter patent filings anticipated. Other than riluzole, there are no compounds that target the glutamate pathway for treatment of metastatic melanoma.  The company has raised more than $2.2 million specifically for this program since 2010, and is looking to raise an additional $3 million by the third quarter of 2015, in order to perform the required IND-enabling studies for an IND application and enter Phase I clinical trials at the Cancer Center of New Jersey