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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.

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.

CPC Scientific is a world-leading peptide CMO. With headquarters in the San Francisco Bay Area, CPC has the world-largest >200,000 sq ft cGMP peptide facility. This cGMP peptide facility passed multiple inspections from FDA without any Form 483 observations, is certified by ISO9001:2000 and ISO13485, and has capacity of hundreds of kg/cGMP product. Under leadership from many peptide industry veterans, CPC has been an ideal "partner", beyond just being a "supplier", offering our global clients, including all the top 10 global pharmaceutical companies, with extensive technical expertise, the highest quality, fast delivery, local access, and cost-effective solutions.