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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 

Altor BioScience Corporation is a privately held, venture-backed, development-stage company engaged in the discovery and development of high-value, targeted immunotherapeutic agents for the treatment of cancer, viral infection, and inflammatory diseases, based on three revolutionary technology platforms. Altor was formed in 2002 by Hing C. Wong, Ph.D., and is based in Miramar, Fla., with 22 employees.

Technology Overview

Altor, a clinical-stage biopharmacteutical company with multiple on-going Phase II trials, has developed technology to produce biologically active, soluble T-Cell-Receptor molecules in a single-chain format (scTCR). These scTCRs can be modified into STAR™ fusion agents, which retain the ability of the TCR to specifically recognize novel targets on cancerous or virus-infected cells, including intracellular antigens that are not accessible to therapeutic antibodies. STAR molecules are ideally suited to deliver anti-cancer and anti-viral drugs, such as immunomodulatory cytokines, cytotoxic drugs, radioisotopes, and imaging agents, directly to diseased cells. Altor has developed a high-affinity scTCR that recognizes a peptide antigen derived from p53, which is over-expressed in roughly 50 percent of all human cancers. ALT-801 is a fusion of this p53-specific scTCR and the approved anti-cancer drug, Interleukin-2 (IL-2). ALT-801 is designed to deliver the IL-2 directly to the tumor site providing greater efficacy, lower toxicity, and better quality of life for patients.

Market Potential

ALT-801 would provide benefit to patients with bladder cancer, multiple myeloma, and melanoma. In 2010 in the U.S., 68,130 new cases of melanoma were diagnosed and 8,700 deaths occurred due to melanoma. In 2008, there were approximately 822,770 people alive in the U.S. who had a history of melanoma. It also estimated that 70,530 new cases of bladder cancer were diagnosed and 14,680 deaths occurred due to bladder cancer in the U.S. in 2010, and that there were approximately 537,428 people alive in the U.S. who had a history of bladder cancer in January 2008. In addition, an estimated 64,615 people in the U.S. were alive in 2008 with a history of multiple myeloma. This represents a market opportunity of over $3 billion in the U.S. alone. Bladder cancer, a major unmet medical need, is currently Altor’s main development focus.

Competitive Advantage

STAR agents significantly broaden the spectrum of tumor- and virally-specific antigens that can be targeted for therapeutic intervention. Altor has demonstrated scTCRs can be used to create targeting molecules to recognize antigens that cannot be targeted by monoclonal antibodies for diagnostic or therapeutic purposes. Although there are no TCR-based products on the market, monoclonal antibodies for cancer had sales of $24 billion in 2010. 

Financial Overview

Altor has raised $35.5 million in paid-in capital from institutional investors, including Sanderling Ventures and Florida Growth Fund, as well as from high net worth, private individuals. The company has been awarded $14 million in SBIR awards from NIH, FDA, and Gates Foundation grants. Altor is seeking $20 million in financing to support and complete the pivotal trial using ALT-801 for locally advanced and metastatic bladder cancer to gain accelerated approval.

Intellectual Property

STAR technology and ALT-801 are the subjects of 37 issued patents and 51 pending applications, including USP #7,456,263, EP 1,546,188. 

Commercialization Strategy

Altor’s short-term objectives are to continue clinical development of its lead product candidates, ALT-801 and ALT-803, through proof-of-principle Phase II clinical trials and then license these to a major biopharma/pharma partner for further development/commercialization. On a case-by-case basis, Altor will consider conducting a registration trial for FDA product approval.

Pipeline Products 

• ALT-801 (p53-TCR/IL-2 fusion protein) 
• Phase II for treating metastatic melanoma (NCT01029873) 
• Phase II for locally-advanced/metastatic bladder cancer (NCT01326871)
• Other Phase IB/II trials for superficial bladder cancer, multiple myeloma - supported by $3 million SBIR Bridge grant
• Phase I/IIAdonor lymphocyte infusion to treat Acute Myeloid Leukemia (NCT01478074) 
• ALT-836 (anti-Tissue Factor Antibody partnered with Genentech) 
• Phase II for treating ALI/ARDS (NCT00879606) and Phase I/IIA for solid tumors (NCT01325558)
• ALT-803 (non-targeted IL-15 super agonist/IL-15R-Fc fusion complex) 
• Pre-IND for treating solid and hematological tumors

Management Team

Hing C. Wong, Ph.D., President and CEO, is a 28-year veteran providing leadership, overall direction, fundraising, IND filing, and oversight of multiple products in clinical trials/commercialized. He has raised $65 million in private capital. 

Dean Taylor, Ph.D., Chief Business Development Officer, has 30 years of experience and is responsible for business development, contracts, strategy, and concluding deals.

Peter Rhode, Ph.D., Vice President, R&D, leads product development, supervises R&D and manages the manufacturing and IP portfolio, and oversees IND filings.

Jeff Weber, M.D., Ph.D., Consulting Medical Director, supports clinical development strategy and planning, and is a renowned clinical research oncologist and senior member of Moffitt Cancer Center.

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

Centrose is a biotechnology company formed in 2006 and is focused on developing a novel antibody-drug conjugation (ADC) technology that targets a wide variety of diseased cells. Centrose discovered the first-ever synergistic drug targeting system called the Extracellular Drug Conjugate System (EDC). 

Centrose has 10 employees and projects to grow to 25 staff. 

Technology Overview

Centrose is a preclinical stage company developing a novel ADC technology that targets a wide variety of diseased cells. Centrose discovered the first-ever synergistic drug targeting system called the EDC System. EDCs are like (ADCs), but are safer and more effective because they are not pro-drugs and only affect diseased cells. To modulate cell growth and activity, EDCs use antibodies (specific to diseased cells) attached to Centrose’s proprietary modulating drugs to work in concert together – the two must be attached to work. Currently, Centrose has four EDC lead drug candidates. As a platform, the EDC system allows for the construction and development of targeted drugs that can be developed for multiple indications including cancer, inflammation, and diabetes.

Market Potential 

Currently, Centrose has four lead programs that it anticipates moving into clinical trials in the next 24 months. The company’s lead program, EDC1, is focused on the lung and metastatic cancer markets; specifically non-small cell lung cancer (NSCLC) and pancreatic cancer. 

Competitive Advantage

There are limitations with regards to traditional antibody drug conjugates technologies:

• First, ADC cell internalization is inefficient and requires the use of very toxic drugs; 
• Second, to become activated, the drugs must be released from the antibody;
• Third, once released, the drugs can interact with normal surrounding tissue leading to toxicity concerns.

In combination, these requirements present formidable design challenges and seriously limit the power of traditional antibody drug conjugates.

To address these problems, Centrose discovered and developed a revolutionary new type of ADC, called EDC. The Centrose EDC system is composed of three parts: a binding component that specifically targets diseased cells, a proprietary drug, and a linker that connects them. This is similar to the ADC system except that the EDC never requires drug dissociation or cell internalization, negating the three major problems of the ADC system. 

Financial Overview

Centrose has raised $3.5 million from individuals and $1.5 million from government grants. The company is currently looking to raise $20 million under a Series A round to move Centrose’s lead compound into and through Phase I clinical trials.

Intellectual Property 

Centrose technology is the sole property of Centrose. Centrose has applied for multiple U.S. and worldwide patents covering EDC technology. Centrose also has the freedom-to-operate in the space. 

Commercialization Strategy 

Centrose’s business strategy is focused on producing the next generation of targeted therapies and to out-license these assets to select pharmaceutical companies. Strategic partnering is therefore critical to advance Centrose's novel therapeutics programs into clinical development and then to the market.

Pipeline Products 

In addition to EDC1, Centrose has four other EDC programs:

EDC2

The antibody target is CD147 and is highly expressed on cancer cells where it facilitates invasion and metastasis. CD147 is also a biomarker for wide range of cancers. As proof of efficacy, Centrose has tested EDC2 and with gemcitabine on pancreatic cell line and demonstrated that EDC2 shows picomolar activity on PANC1 cell line verses gemcitabine, which demonstrated only micromolar activity. Gemcitabine is approved for the treatment of pancreatic cancer.

EDC3

The antibody target is CD44v6 and is associated with tumor progression, metastasis, and specifically with NSCLC lymph node metastasis. Centrose studies show Na,K-ATPase-and CD44v6 complexes on certain cancer cells, yet EDC3 is not toxic to human skin cells in culture (warhead target is low on normal skin). 

EDC7

The antibody target for EDC7 is CD56 (aka NCAM-Neural Cell Adhesion Molecule). The mAB target, CD56, is also the target of ImmunoGen’s lead internal program: IMGN-901. CD56 is highly expressed on the following human tumors SCLC, multiple myeloma, ovarian, and other related indications such as leukemia and Wilms’ Tumor. Studies show Na,K-ATPase-and NCAM, form a complex on SCLC cells. EDC7 demonstrated low picomolar level activity when cancer cells express CD56; thus EDC7 may be an excellent candidate for SCLC.

Management Team 

Dr. James Prudent is the CEO and founder of Centrose and brings more than 20 years of biotechnology. Before Centrose, Dr. Prudent served as Chief Scientific Officer and on the Board of Directors at EraGen Biosciences (sold to Luminex). Dr. Prudent received his doctorate in chemistry from the University of California at Berkeley.

Steve Worsley is the Chief Business Officer and brings 25 years in the biotechnology industry to Centrose. Mr. Worsley has executed numerous transactions in the mAB market; most notably with the companies Abgenix and Raven Biotechnologies. Mr. Worsley out-licensed Vectibix®, the first fully human mAB specific to the EGFr (HER1). He received his MBA from the University of Washington.

The technical staff at Centrose includes two managers, Dave Marshall, Director of EDC Technologies,and Dr. Mohammed Shekhani, Director of Chemistry, who manage the biotechnology and chemistry groups respectively. 

The technical group is provided consultation by Dr. Homer Pearce who developed gemcitabine (Gemzar) and has numerous years of experience in oncology while at Eli Lilly and numerous other technical advisors.

MyeloRx is developing proprietary prodrugs and derivatives of the natural product triptolide. This agent has demonstrated clinical efficacy in acute leukemias. The company is currently funded by an NCI Fast Track SBIR contract enabling the development of its lead product, MRx102. MRx102 has demonstrated efficacy in a variety of preclinical models of AML; this work, performed in collaboration with Dr. Michael Andreeff of the M.D. Anderson Cancer Center, was recently published in Leukemia.  It has also shown a high degree of safety in rodent toxicology models. 

 Based on its novel mechanism of action, the inhibition of XPB, it is expected to enhance the effects of radiation in solid tumors. Preclinical data support this hypothesis.

 The compound is currently undergoing additional toxicology studies in rodents as well as toxicology studies in dogs. Is has patent coverage in the U.S., E.U., Japan and China.

The principals have extensive pharmaceutical experience at both large (Pfizer, Wyeth, DuPont) and small (Cetus, Cygnus and Pharmagenesis) pharmaceutical/biotechnology organizations. 

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.

Company Background

Omniox is a biotechnology company commercializing a breakthrough oxygen delivery technology called H-NOX for a broad range of peripheral hypoxia diseases including cancer, acute cardiovascular ischemia, wounds, and trauma. The H-NOX technology directly overcomes key reasons for the failure of prior efforts in this area. The technology was originally developed in the laboratory of Michael Marletta, currently President and CEO of The Scripps Research Institute. Omniox currently employs seven full-time scientists and has laboratory operations in Mission Bay, San Francisco, and Sunnyvale, Calif. 

Technology Overview

Omniox is a preclinical/IND-stage company initially focused on developing an H-NOX product that sensitizes hypoxic tumors to radiation and chemotherapy. Preclinical data with the lead H-NOX candidate demonstrate substantial re-oxygenation of hypoxic tumors. When combined with radiation, there is a significant delay in tumor growth and enhanced survival in relevant mouse models of human cancer including glioblastoma, with a promising safety profile.

The University of California, San Francisco Neuro-Oncology Clinical Site Committee has approved H-NOX for parallel Phase IB clinical trials in recurrent and newly diagnosed glioblastoma. A real-time pharmacodynamic biomarker for hypoxia has been validated in the clinic and will be used to identify appropriate patients and measure the biological effects of H-NOX in reducing tumor hypoxia. 

Market Potential

Radiation therapy is the most common non-surgical treatment for cancer patients (more than chemotherapy and targeted therapies combined). Needham & Company estimates that an oxygen-delivery therapy to improve chemo-radiation would command $4,000 to $20,000 per round of chemo-radiation treatment and may represent a market of $3 to $5 billion per year. The competitive, regulatory, clinical, and reimbursement landscapes for this indication are compelling.

Competitive Advantage 

Omniox’s H-NOX oxygen carriers are designed to penetrate deep into the tumor tissue, beyond the reach of red blood cells. This approach is a major improvement over prior clinical efforts relying on manipulating red blood cells: this only succeeded in hyper-oxygenating normoxic tissues with minimal effects on hypoxic tumors. H-NOX is an entirely new approach to re-oxygenating hypoxic tumors to enhance chemo/radiosensitiation.

Financial Overview 

Omniox has secured more than $4 million in NIH SBIR funding since 2009. We are actively seeking equity financing to match the NCI Phase IIB $3 million Bridge Award to advance a lead candidate through Phase IB clinical trials. This clinical milestone will create a significant value inflection for investors joining at this stage of development.

Omniox has received firm commitments for $1 million from high net worth investors, and is seeking a minimum of $2 million in additional investments to match the NCI Bridge award. 

Intellectual Property 

In 2006, UC Berkeley filed broad patent claims to protect the core technologies, and Omniox continues to file for further protection of specific applications. Omniox holds an exclusive option to negotiate (with capped financials) for an exclusive worldwide license for all therapeutic and industrial uses of these technologies. The company has retained the law firm of Morrison & Foerster to oversee IP matters and the firm of Latham & Watkins for corporate affairs. More details on the current status of national filing phases of the core patents are available upon further request.

Commercialization Strategy 

Omniox expects to partner with or be acquired by a pharmaceutical company to successfully commercialize H-NOX for peripheral oxygen delivery. All major pharmaceutical companies are currently conducting clinical trials with chemotherapeutics or targeted therapies in combination with radiation, with the goal of enhancing the efficacy of radiation. 

The lead H-NOX product will be best utilized by medical oncologists who oversee patient treatment plans as part of a team of oncology professionals, including a radiation oncologist. More than 90 percent of radiation oncologists practice within two blocks of medical oncology clinics, therefore, radiosensitizers can be infused at the medical oncology office prior to transport of the patient for radiation treatment.

Pipeline Products 

H-NOX oxygen carriers have the potential to reduce tissue loss during myocardial infarctions and stroke, as well as in acute and chronic wound settings, a range of transplant surgeries, and ultimately may function as part of a resuscitation fluid in emergent situations. There is tremendous life cycle potential for H-NOX proteins beyond their utility in oncology.

Management Team 

Omniox is led by CEO and co-founder Stephen Cary, formerly in Research and Development/Market Strategy at Genentech. 

The Chair of the Scientific Advisory Board is co-founder, Michael Marletta, currently President/CEO of The Scripps Research Institute, member of the SAB of HHMI, and member of NAS and IOM. He has extensive experience in advising pharmaceutical companies in drug development. 

The business co-founder is Ajit Shah, who has a combined 24 years of experience as an entrepreneur, operating executive, and venture capitalist. He is active in Silicon Valley as an outstanding scientific and strategic advisor to start-ups. 

The IND Core Team is made up of experienced drug development veterans from Genentech, Quintiles, and Baxter Healthcare. 

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 

Company 

Qualiber, Inc. is a nanomedicine company with the mission to deliver innovative treatments for cancer and other life threatening diseases.  Utilizing nanotechnology it is creating medicines that have superior efficacy and safety profile. In addition, Qualiber is partnering with pharmaceutical/biotechnology companies to address delivery challenges of partner’s treatments.

The company has secured funding from the National Cancer Institute, UNC-CH, the North Carolina Biotechnology Center and pharmaceutical company partners.  The company is seeking $500K first tranche of $5M Series A funding to progress lead cancer product, NanoGTP™ (Nanomedicine delivering Gemcitabine Tri-Phosphate) towards human clinical trials.

1st Product: NanoGTP™, demonstrated superior efficacy & safety in animal models of human cancer 

Cancer (especially pancreatic and lung cancer) is a continuing leading cause of death and suffering in the US and the World. Though there are chemotherapeutic drugs on the market, most have limited effectiveness and cause serious side effects/toxicities. Qualiber is developing its first product NanoGTP for pancreatic and lung cancers. NanoGTP substantively improves the delivery of existing anticancer drug, gemcitabine, and enhances the potential for greater clinical benefit and expanded use.  In preclinical animal models of human cancer NanoGTP has demonstrated superior efficacy and safety than gemcitabine.  Company expects to complete scale-up manufacturing and preclinical toxicology studies by end-2013 followed by Investigational New Drug (IND) application filing to the FDA in 2014. The market for pancreatic cancer treatments is estimated to be $1.2Billion in 2015 and for lung cancer to be $9.5Billion in 2018.

Founders and Management Team

·        Dr. Anil K. Goyal, President & CEO. Over 20 years of leadership experience at venture-backed and public biotechnology companies. Managed and led exits: Optherion’s diagnostics division sale and Serenex >$300M sale to Pfizer.

·        Dr. Leaf Huang, CSO and Co-founder.  Distinguished Professor and Chair, Division of Molecular Pharmaceutics, UNC-Chapel Hill. More than 30 years research experience and world-renowned in development of liposome based drug and gene-delivery systems. Co-founder of four other companies.