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Company Background

Metabolomx is commercializing a non-invasive, rapid, and inexpensive breath test for cancer with the potential to revolutionize cancer diagnosis. 

Technology Overview

Metabolomx has developed technology enabling the identification of lung cancer from its metabolomic fingerprint in exhaled breath, currently in a second round of efficacy trials at Cleveland Clinic and other distinguished clinical centers. At the heart of the system is a high dimensional array of diversely reactive chemical indicators that change color upon interaction with volatile species or mixtures.

Using the first generation of the Metabolomx sensor a recent Cleveland Clinic study (Mazzone et al., 2012, Journal of Thoracic Oncology) reported 85 percent specificity and sensitivity for lung cancer detection, comparable to a CT scan, the present gold standard. In the first quarter of 2012, the Cleveland Clinic and National Jewish Health Center in Denver (led by Dr. Jim Jett, Editor-in-Chief of JTO) began testing a Metabolomx sensor over 100 times more sensitive than the version used in the Journal study. The Mayo Clinic, led by Dr. David Midthun, is scheduled to begin testing in the second quarter of this year. Metabolomx’s technology will be in the clinic at the country’s three top-ranked lung care centers (U.S. News and World Report), reflecting the promise of this new paradigm for lung cancer assessment.

Market Potential 

First indication: A companion diagnostic to CT scan 

Metabolomx’s first FDA indication will be as a diagnostic adjunct to an inconclusive CT scan. Results from the 53,000 patient National Lung Screening Trial (NSLT) indicate that a CT scan screening of high-risk patients extends life expectancy over 20 percent (NEJM, June 2011). The study has already prompted a powerful shift toward widespread use of CT, with Wellpoint recently announcing it will cover CT screening of the enormous at-risk population (more than 55 years of age, more than 30 pack years) identified in the study. However, CT generates a large number of false positives (the NLST found 27% of the high-risk group had a positive CT). Millions of people who have a positive CT are faced with the difficult decision of whether to monitor with follow-up CT or submit to an invasive and expensive biopsy, magnifying the need for a diagnostic adjunct to inconclusive CT. The estimated market size of this first indication is 10 million units per year in the U.S., with revenue of $1 billion.

Pre-screen of high-risk population to triage who should receive CT 

The financial costs of CT, risks involved with radiation exposure, and the enormous size of the at-risk population defined in the NLST calls for a non-invasive, inexpensive initial test to better triage who should be screened by CT. Metabolomx expects to gather data on more than 1,000 patients to confirm that the breath test is a candidate pre-CT screen of the high-risk population. The estimated market size to pre-screen the high-risk population is 25 million units per year in U.S., with revenue of $2 billion.

Monitor efficacy of chemotherapy

Metabolomx is gathering data to confirm that the breath signature declines when chemotherapy is effective in curtailing tumor growth. The estimated potential market to monitor treatment is 15 million units per year in the U.S., with revenue of $1.5 billion.

Correlation between metabolomic breath fingerprint and effective treatment

Metabolomx is compiling an unmatched database allowing the post-hoc assessment of whether there is a predictive correlation between the metabolomic breath fingerprint and the efficacy of a particular treatment. With each treatment often tailored to intervene in a particular metabolic pathway, Metabolomx predicts that the metabolomic fingerprint in exhaled breath will allow individualized selection of treatment.

Competitive Advantage 

The technology is a five minute, inexpensive (less than $100), non-invasive test for lung cancer and other cancers, based on the VOC profile present in the bloodstream and picked up in exhaled breath. Each cancer has its own “smell,” and canines have been documented more than a 95 percent accurate in detection across stages, signaling the extraordinary promise of this new paradigm. 

Financial Overview 

Metabolomx has received several government development contracts, including a NCI Phase I/Phase II Fastrack award of $1.135 million. The company seeks a $5 million Series A round to reach clinical quantification of the level of accuracy of the second generation lung cancer detection system (building on the 85 percent accuracy of the first generation system) and submission to the FDA. Financial information on Metabolomx is available to interested parties under NDA.

Intellectual Property 

The company holds exclusive, worldwide rights to an estate of fundamental enabling technology, including both the sensor itself and application IP. Additional private information is available to interested parties under NDA.

Commercialization Strategy 

The Metabolomx breath analysis instrument will be sold or leased to the same hospitals and testing centers that use CT. The bulk of the company revenue is from the disposable sensor arrays and the balance from equipment and support. The company’s first FDA indication will be as a diagnostic adjunct to indeterminate CT scan. 

Pipeline Products 

The first product is a breath test for lung cancer, but tests for other cancers will follow.

Management Team 

Paul Rhodes, Ph.D., CEO leads a diverse group of technology companies, which have received $13 million in DARPA contracts to develop next-generation sensory systems. 

Ray Martino, COO, who, during a 20-year career at Symbol Technologies was General Manager of its mobile business ($500 million in division sales) and then CTO of Symbol prior to its acquisition by Motorola for $3.9 billion in 2007. 

Sung Lim, Ph.D., Chief Scientist, is a co-inventor of the company’s proprietary nanoporous pigment array optical sensing technology, along with Metabolomx’ co-founder, University of Illinois Professor Ken Suslick.

A Clinical Advisory Board has been formed, and includes deep practical expertise in FDA approval processes.

Company Background

Celek Pharmaceuticals is addressing the need for new medicines to help patients suffering from cancers that are poorly served by current therapies. The company’s strategy is to enhance the value of in-licensed drug candidates by advancing them through proof-of-concept clinical trials. Formed as a Delaware LLC in 2009, Celek’s two founders, Graham Allaway, Ph.D., and Gary Robinson, Ph.D., are currently the sole employees. 

Technology Overview

Celek’s lead product, CEL-031, is a clinical-stage targeted anticancer drug that selectively induces apoptosis in tumor cells by inhibiting cyclic GMP phosphodiesterases, which are overexpressed in human tumors. Currently in preclinical development for non-muscle invasive bladder cancer (NMIBC), CEL-031’s mechanism of action involves the degradation of β-catenin, a cell signaling protein that plays a key role in bladder cancer tumorigenesis. In clinical studies against advanced cancers, orally-administered CEL-031 showed evidence of efficacy and a good safety profile. CEL-031 should have greater clinical efficacy against NMIBC, where it will be administered intravesically (i.e., instilled transurethrally), the standard drug delivery route for this indication. 

Market Potential

Bladder cancer is the fifth most common cancer in the U.S., with 70,000 new cases annually and 600,000 individuals living with the disease. Worldwide, there are approximately 400,000 new cases annually and the incidence is rising. 

About 70 percent of new bladder cancer diagnoses are made at the non-muscle invasive stage. Current NMIBC treatments involve transurethral resection (TUR), often followed by intravesical chemotherapy using non-specific cytotoxic drugs such as mitomycin C, or immunotherapy with Bacillus Calmette Guerin (BCG). These treatments often fail, with five-year recurrence and progression rates of 50-70 percent and 20-30 percent, respectively. Current drugs also cause adverse side-effects and are hazardous to health care workers.

Since NMIBC is a chronic disease requiring lifelong monitoring and treatment, the lifetime cost per patient of treating bladder cancer is the highest of all cancers.

Despite the pressing need, few new drugs are in development for NMIBC. Celek is developing CEL-031 for two NMIBC indications: (i) perioperative administration following TUR, and (ii) BCG-refractory NMIBC. CEL-031’s estimated peak annual sales in these indications range from $510 million to $660 million. 

Competitive Advantage

As the first targeted drug for NMIBC, CEL-031 represents a potential breakthrough in the treatment of patients with this disease. It should be possible to deliver CEL-031 safely at higher, more effective doses than current cytotoxic chemotherapies, resulting in dramatic reductions in rates of recurrence and progression. CEL-031’s favorable safety profile should also result in a substantial increase in the number of patients treated with CEL-031 compared to current drugs.

Financial Overview

Celek has raised more than $700,000 in funding, including investments by the principals and federal and state grants. The NCI awarded Celek a $176,000 Phase I SBIR contract supporting preclinical studies on CEL-031 for NMIBC. Celek is currently seeking to raise $3 million to support preclinical studies of CEL-031 in bladder cancer and acute myeloid leukemia (AML), and the initiation of a Phase I/II clinical trial in non-muscle invasive bladder cancer. 

Intellectual Property

Celek obtained exclusive rights to CEL-031 from OSI Pharmaceuticals. CEL-031 as a composition of matter and methods of treating cancer with CEL-031 are covered by four issued U.S. patents (plus foreign equivalents). Additional patents cover analogs, methods of identifying anticancer compounds and combination therapies. 

Commercialization Strategy

Celek plans to complete a proof-of-concept clinical trial of CEL-031 in NMIBC patients, then partner for later stage development/commercialization. Recent partnering deals in this therapeutic area have had attractive financial terms. Celek has already met with potential partners who indicated interest in the product. 

Pipeline Products

Celek is also developing CEL-031 to treat advanced cancers using novel formulation and delivery technologies to increase concentrations of the drug in the body, thereby maximizing efficacy. The company is focusing on: (i) advanced bladder cancer, and (ii) acute myeloid leukemia (AML). A recently published independent study reported that CEL-031 has potent activity against tumor cells from AML patients, including those resistant to current drugs, and recommended clinical testing of CEL-031 against AML. CEL-031 would be eligible for Orphan Drug status in this indication.

Management Team

Graham Allaway, Ph.D., President and C.E.O, has spent 22 years in the biotechnology industry. As founding CEO of Panacos Pharmaceuticals, he played a key role in building that company from a private venture-backed start-up to a public company, while raising more than $125 million in private and public equity financing. Dr. Allaway also led Panacos’ drug discovery and development programs. Prior to Panacos, Dr. Allaway was CEO of Manchester Biotech and he previously led therapeutic R&D at Progenics Pharmaceuticals. 

Gary Robinson, Ph.D., Chief Business Officer, has 20 years of experience in research, development, and commercialization of technologies and products in the physical and life sciences. Most recently, he was Senior Director of Business Development at Panacos Pharmaceuticals, where he led partnering, contracting, intellectual property and pre-launch marketing activities. Prior to Panacos, Dr. Robinson held business and corporate development positions at IGEN.

Company Background

AcuityBio is a preclinical stage company focused on the commercialization of the company’s unique biocompatible, biodegradable drug delivery platform technology to prevent post-surgical locoregional tumor recurrence in early-stage lung cancer patients. AcuityBio’s formation in 2010 stemmed from a collaboration between lung cancer thoracic surgeon Dr. Yolonda Colson (Brigham and Women’s Hospital) and Professor of Chemistry/Biomedical engineering Mark Grinstaff (Boston University). Together with Drs. Grinstaff and Colson, AcuityBio was co-founded in 2010 with CEO Jay Schwartz, Ph.D., and co-inventor of the technology and Director of Research, Jesse Wolinsky, Ph.D. AcuityBio currently has two full-time employees.

Technology Overview

AcuityBio has developed chemotherapy-eluting ABC Mesh™ based on their proprietary slow-release, biodegradable ABC Polymer™ for the prevention of locoregional lung cancer recurrence. It functions by delivering paclitaxel locally at a slow and predictable rate directly at the resection site following lung cancer tumor removal surgery. This maintains a localized high level of drug while minimizing side effects without affecting healing, which will result in better patient outcomes. AcuityBio is currently evaluating applying ABC MeshTM to select soft tissue orphan oncology indications that have a high recurrence rate. This will allow a shortened time through a first-in-human trial as an “orphan” indication and accelerated FDA approval. AcuityBio has developed ABC Mesh to the preclinical stage, focusing on Chemistry Manufacturing and Control, engineering, and securing key manufacturing and supplier CMO/CRO vendor relationships. 

Market Potential

Lung cancer is responsible for more deaths annually in the U.S. than breast, colorectal, and prostate cancers combined. Surgical removal of the tumor (resection) is the standard of care for early-stage lung cancer patients, yet lethal, locoregional tumor recurrence continues to afflict about half of the 50,000 patients per year who receive surgical treatment with curative intent. 

Each year in the U.S., 221,000 new lung cancer cases are reported. Roughly one-quarter of these patients (about 50,000/year in the U.S.) will be considered candidates for surgical resection (the standard of care). The cost to the health care system resulting from the 50 percent endemic cancer treatment failure in the U.S. alone is a staggering $1 to $2 billion per year. Other early stage cancers also suffer from high locoregional recurrence rates, including stage II colorectal cancer (50,000 patients) and breast cancer lumpectomy patients (71,000). Several soft tissue “orphan” oncology indications more than 50,000 patients per year.

Competitive Advantage

Existing drug-eluting polymer products on the market do not possess the properties that are required for the development of an ideal soft tissue compliant, biocompatible, biodegradable drug-eluting implant with tunable drug release. ABC Mesh has all these features. ABC Mesh is an easy-to-manufacture, widely applicable drug delivery platform, which can predictably and locally deliver insoluble and water-sensitive drugs to soft tissue for more than 50 days. The implant has already been proven to be biocompatible (ISO10993), biodegradable, and physically compliant and capable of slow, controlled drug delivery.

Financial Overview

AcuityBio has received almost $3 million in non-dilutive capital through NIH, SBIR Phase I and II, government and Coulter Foundation grants, which will be sufficient to advance their ABC MeshTM program to a first IND. The company seeks $3.5 million to help propel its first product through preclinical and to Phase I safety trials for early-stage non-small-cell lung cancer and to support the clinical development of its orphan drug first-in-human trials

Intellectual Property

AcuityBio has negotiated with Boston University and Brigham and Woman’s Hospital-Harvard/Partners to obtain exclusive world-wide rights to issued patent (U.S. 7,671,095) and three pending patents covering compositions and methods of use. The company has determined it will have a freedom to operate clear of prior art.

Commercialization Strategy

The value proposition of ABC Mesh is that it combines clinical differentiation, ease of use, and positive patient impact at lower overall cost per patient. Thoracic surgeons, hospital purchasing agents, group purchasing organizations will be the company’s main customers. Customers will be compelled to choose ABC Mesh because of its compelling clinical data, key opinion leader, and contract sales channels. 

Pipeline Products

ABC Mesh is a platform technology that has been specialized to deliver water-insoluble or water-sensitive drugs that are traditionally difficult to formulate for administration. The company can tune the drug release rate and the implant’s degradation rate depending on the application. AcuityBio is actively pursuing other clinical indications including orphan indication that would benefit from their localized delivery technology. 

Management Team

John ‘Jay’ Schwartz, Ph.D., Co-Founder, CEO, and Chairman, has more than 20 years in life sciences technology development. He was research faulty at MIT working on drug delivery, is experienced in private equity fundraising, and has succeeded in securing multiple U.S. government grants. He co-founded the venture-backed engeneOS, bought by ADNEXUS, which was acquired by Bristol-Myers Squibb in 2007. Jay received his Ph.D. in Biochemistry and Molecular Biology from New York Medical College and pursued postdoctoral work at Harvard and MIT in drug delivery, protein engineering, and cardiovascular medicine.

Jesse Wolinsky, Ph.D., Co-Founder and Director of Research, is a co-inventor of the ABC Mesh. He holds a doctorate in Biomedical Engineering from Boston University and a Bachelor of Science in Materials Science and Engineering from the University of Florida where he specialized in Polymer Chemistry. 

Company Background

A&G Pharmaceutical (A&G), founded in 2000, is a privately held company based in Columbia, Md. A&G uses proprietary technology for rapid development of monoclonal antibodies (mAB) to unique cancer-specific theranostic targets, to develop novel therapy/diagnostic combination products that address a broad range of cancers. A&G holds a proprietary position on the detection and treatment of diseases related to the growth factor GP88 (progranulin). The company is currently developing a therapeutic mAB to treat lung and breast cancer. A&G has also developed clinically validated proprietary companion diagnostic products to identify and monitor patients treated with mAB. The company employs 22 people.

Technology Overview

Several peer reviewed studies have demonstrated that glycoprotein GP88 has a critical role in the proliferation and survival of cancer cells. A&G has developed a recombinant therapeutic anti-GP88 to treat cancers overexpressing GP88. Direct validation of GP88, as a novel therapeutic target, was provided by inhibition of GP88 expression and function in breast carcinoma cells resulting in both reduced proliferation in vitro and reduced malignancy in vivo. Xenograft data demonstrates that anti-GP88 is useful for the treatment of breast and lung cancers as a single agent. When used in combination with Tamoxifen in Tamoxifen-resistant tumors, anti-GP88 restores Tamoxifen sensitivity, leading to significant tumor reduction. Restored sensitivity to Tamoxifen and other anti-estrogen therapies is a major breakthrough, more than 50 percent of all patients on anti-estrogen develop resistance de novo or during treatment. Similar results were obtained with chemo resistant lung cancers. A&Gs pre-clinical candidate is ready to enter IND-enabling acute and repeat dose toxicology studies in primates.

Market Potential

GP88 therapy can address two leading cancers in the U.S., including: breast (220,000 new cases; 40,000 deaths annually) and lung (200,000 cases;160,000 deaths) cancers. More than 50 percent of lung cancer patients die within 5 years. There is an unmet need for targeted lung cancer therapies, especially among cancers that are chemo resistant. In the case of breast cancer even for Tamoxifen, a leading drug used to treat breast cancer, 40-50 percent of patients do not respond to initial treatment, while the majority of those patients that do respond can have the cancer become resistant during treatment. A&G has developed diagnostic kits to identify patients who are de novo resistant or who are becoming resistant and thus identify patients that are suitable for anti-GP88 therapy.

Competitive Advantage

• GP88 is uniquely placed as a novel biological target for development of products for oncology:
• GP88 is a critical biological player in development, proliferation and survival, and drug resistance for several cancers
• GP88 expression in tumor tissue has been statistically shown to be a prognostic indicator of poor patient outcome (disease-free and overall survival)
• GP88 is secreted by cancer cells and detectable in blood, making it an important target for therapeutic and diagnostic product development
• Inhibiting GP88 with mAB reduces tumor growth and reverses resistance to hormone therapy in breast cancer
• 2 GP88 Diagnostic tests have been clinically evaluated: (1) Tumor levels are prognostic, (2) Blood levels are linked to tumor growth and can be used to monitor treatment

Herceptin remains the last major combination therapeutic and companion diagnostic co-development in oncology. GP88 is positioned to be the next major theranostic product.

Financial Overview

A&G Pharmaceutical’s financial overview includes:

• Seed capitalization of $1.5 million in 2002
• Closed a Series A round of $2 million in 2005
• Raised a total of $6.4 million in 2006 in form of strategic investment. As part of license agreement with Celltrion they agreed to provide cash and candidate development including initial manufacturing process development and materials for toxicology.
• Completed Series B prime financing of $4 million 2008.
• Profits from sales of custom monoclonal antibodies (www.precisionantibody.com); anticipated revenues for 2012 are more than $2.8 million.
• Seeking a $5.0 million investment to fund toxicology and first-in-human clinical studies.

Intellectual Property

Fifty patent applications and 49 patents (15 U.S. patents) granted worldwide covering therapy and diagnostic use of GP88.

Commercialization Strategy

A&G will enter safety/efficacy clinical studies of anti-GP88 in lung/breast cancer. During early clinical trials, A&G will pursue agreement(s) with key player (s) in the pharmaceutical/biotech industry active in the field of oncology. Such agreement(s) will dictate the commercialization strategy for anti-GP88.

Pipeline Products

GP88 has been implicated in several cancers and as such A&G is interested in developing its proprietary theranostic pipeline for use in cancers of the GI, prostate, and brain. Using A&G’s proprietary antibody development technology the company is researching other cancer biomarkers for development along the theranostic pathway.

Management Team

A&G’s CEO, Ginette Serrero, Ph.D., has 25 years of experience in cancer research and 10 years in biotech management and has been instrumental in directing A&G’s vision and assembling the management team.

VP of Drug Discovery, Randy Barton, Ph.D., was previously the director of drug discovery, Boehringer Ingelheim, and has 20 years of experience validating small-molecule and biological drug candidates.

VP of R&D Jun Hayashi, Ph.D., is an immunologist and inventor of A&G’s proprietary mAB technology.

COO Michael Keefe, MBA, is seasoned in raising capital and managing the growth of start-ups.

VP of Product Management, David Hicks, has more than 20 years of experience with diagnostic products and clinical development.

Company Background

NovoMedix LLC specializes in the development of small molecule inhibitors of multiple biological pathways that are critical drivers of disease and are relatively inactive in normal tissues and housekeeping processes, with an initial focus on cancer. NovoMedix targets underserved markets with unmet clinical needs, including triple negative breast cancer (TNBC), high risk B-cell acute lymphoblastic leukemia (B-ALL), and melanoma.

Technology Overview 

NovoMedix has developed two new classes of small molecule translation initiation inhibitors with unique mechanisms of action as targeted therapies for high risk TNBC (estrogen and progesterone receptor, and HER2/neu-negative breast cancer). Lead compounds are currently in the preclinical stage and have been tested in an animal model of TNBC in which they significantly reduced tumor growth (better than paclitaxel) with no apparent toxicity. These novel compounds are promising clinical candidates and represent first-in-class small molecule therapeutics aimed at reducing recurrence and increasing survival rates for TNBC. Since these drug candidates are small molecules, they will be less expensive and easier to administer than biologics and should fit easily within the current treatment regimen.

Market Potential 

Breast, prostate, and colorectal cancer account for more than half of cancer patients in the United States. One in eight women in the U.S. will develop breast cancer during her lifetime. Although the overall survival rate for early stage breast cancer is high, triple negative breast cancers are particularly aggressive and are more likely to recur than other subtypes, resulting in a significantly increased risk of death. Currently, no targeted therapies exist for TNBC. Since more than 60 percent of triple negative breast tumors overexpress eIF4E (a critical factor in translation initiation), and high levels of eIF4E are correlated with recurrence and death, inhibitors of protein translation initiation should prove to be a viable targeted therapy for TNBC with high eIF4E.

Competitive Advantage

NovoMedix’s most advanced drug candidates for the treatment of TNBC represent two new classes of translation initiation inhibitors with unique mechanisms of action. Besides the anti-viral drug, ribavirin, there are no viable drug-like inhibitors of translation initiation have been reported to date. More importantly, there are virtually no novel therapies in clinical trials for TNBC. Most ongoing trials for TNBC are on various combinations of existing chemotherapy drugs. Recent data suggests that at least one of these “first-in-class” compounds has the potential to enter into a Phase I clinical trial for TNBC.

Financial Overview 

NovoMedix LLC was established as a partnership in 2001 and converted to an LLC in 2010 in anticipation of angel or VC funding and/or corporate partnerships. NovoMedix is currently privately owned and has no venture capital investment. NovoMedix has raised $1.75 million in equity, government grant, and tax credit revenue. SBIR funding has allowed the company to increase its value without dilution. NovoMedix is seeking a strategic investment of $5 million to complete preclinical studies and file an IND for TNBC within 24 months. NovoMedix would then partner with a larger pharmaceutical company for clinical development and commercialization of a novel therapy for TNBC.

Intellectual Property 

NovoMedix has filed a composition of matter patent application (PCT/US2011/039377) for the NM043 series of compounds for the treatment, prevention, and/or amelioration of various disorders, including cancer. In addition, NovoMedix is in the process of filing provisional patents on several other lead series.

Commercialization Strategy 

NovoMedix’s commercialization strategy is to design and execute an IND-enabling nonclinical safety program to support a Phase I clinical trial in patients with advanced metastatic disease and enter into partnerships with pharmaceutical companies for the clinical development and ultimate commercialization of novel small molecule drugs. NovoMedix plans to license its compounds in exchange for licensing fees, milestone payments, and royalties.

Pipeline Products 

The NovoMedix pipeline contains several novel compounds in various stages of development. Most relevant to this project are follow-up studies that are planned to determine the efficacy of previously identified lead compounds for the treatment of metastatic breast cancer. In addition, several different novel lead compounds are currently under development for the treatment of high risk pediatric B-ALL. These compounds have demonstrated in vitro safety and efficacy and preliminary safety in animals. In vivo studies in mouse models of high risk B-ALL are the subject of a recently submitted Phase I SBIR proposal.

Management Team 

Cathy Swindlehurst, Ph.D., Founder and CEO, has 22 years of experience in biotechnology. Former V.P. at PanCel, MagneSensors, and NovaDx.

Leah Fung, Ph.D., Founder and Exec. Director, Drug Discovery, has 20 years of experience in medicinal chemistry. Management positions at Structural Genomics, Structural Bioinformatics, and Celgene.

Sabine Ottilie, Ph.D., Director, Molecular Oncology, has 20 years of molecular oncology research experience in academia and biotechnology.

Molecular Express, Inc., a wholly owned subsidiary of Molecular GPS Technologies has an exclusive license to a virus electrode technology, "Viratrode™", from the University of California, Irvine to pursue commercialization opportunities in pharmaceutical and biotechnology applications. 

The Viratrode™ is an exciting new platform technology capable of monitoring biological environments at the molecular level.  By coupling the desirable features of biological systems with nanomaterials, hybrid technologies such as Viratrodes™ will lead to a whole new generation of extremely sensitive detection devices that would be highly competitive in todays global market.

The Viratrodes™ technology possesses several advantages including high specificity and sensitivity for a target analyte in an easy-to-use format that provides results within a short period of time.  These attractive features will revolutionize many areas of science and technology since the technical capabilities of the Viratrodes™ could be used not only as a diagnostic, but as a real-time analytical tool to accelerate the pace of research and development throughout the world.

Orphagen focuses on orphan nuclear receptors for which pharmacological data is limited or non-existent and industry competition is minimal. Orphagen’s strengths lie in the validation of novel drug-like small molecule ligands in receptor assays and subsequent clarification of therapeutic utility in target cells and animal models. This "first-to-ligand" strategy attracts industry partners earlier in the drug development cycle than is possible in a more crowded area. By taking this approach, Orphagen signed a discovery and development partnership in autoimmune disease in 2008 with a mid-sized Japanese pharmaceutical company.

Orphagen Pharmaceuticals has been funded through partnership revenues and more than $5.5 million in external grant funding.

Orphagen's project pipeline:

1. Castration-resistant prostate cancer, Cushing's syndrome and adrenocortical cancer based on antagonists to steroidogenic factor-1 (SF-1).
2. Retinitis pigmentosa (novel target in retina) and the dry form of age-related macular degeneration
3. Mood disorders (novel target in CNS) based on modulation of circadian rhythm

TECHNOLOGY

Nuclear receptors are ligand-mediated transcription factors. As a drug class they have been very successful, but 23 of 48 remain unexplored (orphans).

Orphagen has performed the first small molecule validation for several of these orphan nuclear receptors and continues discovery on new targets.

Orphagen chooses targets based on key criteria:

1. Restricted expression (not ubiquitous)
2. Link between tissue distribution and knockout phenotype
3. Plausible therapeutic hypothesis
4. Evidence for ligand-binding pocket