The chosen companies were selected from a highly competitive field of applicants by an independent panel of experts from venture capital firms, bio-pharmaceutical and medical device companies, based on their strength of research, impact on cancer, product development, and market potential.
These companies include:
20/20 GeneSystems, Inc.
Advanced Cell Diagnostics, Inc.
Altor BioScience Corporation
Guided Therapeutics, Inc.
Intra-Medical Imaging LLC
Optimum Technologies, Inc.
Pathfinder Therapeutics, Inc.
Syntrix Biosystems, Inc.
Visualase, Inc. (BioTex, Inc.)
Zacharon Pharmaceuticals, Inc.
20/20 GeneSystems develops and commercializes innovative, proprietary diagnostics tests that aid in the fight against cancer. Our present focus is on tests for lung cancer that fall into two categories:
Early detection of lung cancer: 20/20 is developing a blood test for the early detection of lung cancer in parallel with Ortho Clinical Diagnostics (“OCD”) (a Johnson & Johnson company) who licensed rights to the technology from 20/20 (co-exclusive). This product will be used for screening smokers and former smokers to identify very early signs of cancer. (A related product for the early detection of head and neck cancer is also in development.)
Personalized medicine for lung cancer: Our patented platform technology for measuring biomarkers in tumors was awarded over $2.5 million in government funding in 2008. The technology is now being used by groups at the National Cancer Institute and leading cancer centers to develop tests to predict responses to several new targeted therapies for lung cancer.
Market opportunity / Competitive advantage: 20/20’s patented platform technology supports numerous companion diagnostics each with revenue potential exceeding $100 million. Development of two such products is now underway. The primary competitive advantage of this technology is its ability to simultaneously identify 10 or more biomarkers from a single tissue section without grinding it up and loosing morphology. Thus, core needle biopsies (small amounts of tissue) can be assessed for their anticipated response to several different targeted therapies, each of which targets a distinct signaling pathway.
20/20 also is the sole owner of a separate biodefense “spin-off” company called 20/20 BioResponse. That company sells a patented kit to emergency responders for screening suspicious powders BioCheck (www.BioCheckInfo.com ). This profitable business unit experienced 15% sales growth in 2008 with potential revenue growth of up to 50% growth in 2009 with additional capitalization.
Advanced Cell Diagnostics, Inc. (ACD) is the world leader in in situ RNA detection. Its proprietary RNAscope™ technology is the first multiplex fluorescent and chromogenic in situ hybridization platform capable of detecting and quantifying RNA biomarkers at single molecule sensitivity.
Based in the heart of Silicon Valley, ACD was founded and managed by experienced entrepreneurs in the life science industry, who previously founded Panomics, a life science research tool company. Panomics was acquired by Affymetrix in 2008 for $73 million.
ACD started and was initially funded by two government grants in 2006. Supported in part by a SBIR grant from NCI, ACD has since completed the development of its RNAscope™ technology and is developing a number of diagnostic products for cancer management. Its major product development effort is focused on the detection and molecular characterization of circulating tumor cells (CTCs) in blood. Another product would assist the selection of adjuvant therapy for early stage breast cancer patients by in situ detection of RNA biomarkers in FFPE tissue sections. ACD has started early phase clinical studies for these products.
ACD’s products are molecular diagnostic tests for personalized cancer treatment, which is one of the fastest growing market sectors representing the future of cancer management. The CTC detection product, in particular, has enormous market potential because not only can each cancer patient be tested multiple times during different stages of cancer progression but also the same approach is applicable to almost all forms of cancer. With more than 10 million cancer patients in US, the potential total market size for CTC detection products is estimated to be well over $10B.
ACD’s RNAscope™ is currently the only effective technology platform capable of robust RNA detection in situ, which unlocks the full potential of RNA biomarkers for clinical diagnostic applications. Compared to existing IHC technology for protein marker detection, RNAscope™ is much more sensitive, specific with higher multiplexing capability. Thus the diagnostic assays based on RNAscope™ can detect diseases earlier, are more robust, precise and information rich. From a business prospective, ACD enjoys an “unfair” competitive advantage as assay conditions for different RNA markers are substantially uniform. This enables ACD to develop products for different applications quickly with minimum development risk.
ACD applies the power of RNAscope™ to CTC detection, which enables its products to not only identify CTCs at higher sensitivity and reliability but also to simultaneously analyze their functional characteristics at molecular level. Such a molecular analysis capability is critically important to cancer management but less attainable with other CTC detection technologies.
In addition to its ongoing efforts to develop proprietary diagnostic tests for cancer management, ACD also establishes partnerships with pharmaceutical and biotechnology companies to validate biomarkers for targeted therapy and to co-develop companion diagnostics.
ACD received its Series A financing of $5.4 million from Morningside Ventures in 2008 and is expected to raise Series B in 2010. Series B funding will be used to conduct clinical trials and to obtain regulatory approval for the commercial launch of its first diagnostic test.
Altor BioScience Corporation (“Altor” or the “Company”) 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. The Company was formed in 2002 by Hing C. Wong, Ph.D., as a spin-off from Sunol Molecular Corporation (“Sunol”), which in turn was spun-out in 1996 from Baxter International. The Company is located in Miramar, Florida, has 21 employees and currently has three products in mid and late phases of clinical development.
Altor is utilizing its novel STAR™ (Soluble T-cell Antigen Receptor) platform technology to capture the precise, disease-targeting properties of T-cell receptors in creating next-generation therapeutic and diagnostic molecules. These innovative molecules promise to enhance the efficacy and reduce the toxicity of existing drugs by targeting a wide range of tumor and viral antigens.
Products: ALT-801, Altor’s lead product for cancer, is a T-cell receptor-targeted immunotherapeutic that recently concluded a Phase I/IIa clinical trial in patients with metastatic malignancies. A Phase II trial against metastatic melanoma will be initiated in late 2009. A second product, an antibody-based Tissue Factor antagonist, is in a multi-center, placebo controlled, randomized Phase II trial for Acute Respiratory Distress Syndrome and Acute Lung Injury, a life-threatening systemic inflammatory disease. These development efforts have been supported by more than $5.5 MM in SBIR awards from NIH, with an additional $3 MM Bridge grant from NCI awarded in September 2009. The Company’s third product, an antibody that prevents and treats staphylococcal infections in premature neonates, out-licensed to Biosynexus, is in a Phase III registration trial.
Background: In the human immune response, when the T-cell receptors (TCRs), on the surface of T-cells recognize and bind to a tumor or virally infected cell, the T-cells become “activated” and initiate immune responses that eliminate the diseased cells. TCRs do not recognize full-length tumor or viral protein, but instead recognize protein fragments (or peptide antigens produced by protease processing), presented on membrane receptors, known as the major histocompatibility complex (MHC) molecules. Through this antigen-presenting pathway, each cell in the body is able to display a cell-specific set of MHC-peptide antigens for T-cell surveillance. Every T-cell produces a TCR with unique and specific binding properties, providing the immune system with the potential to selectively target many different disease antigens, including antigens derived from intracellular protein targets. Altor has captured this potential with the development of its STAR™ (Soluble T-cell Antigen Receptor) molecules, to guide therapeutic drugs directly to the sites of disease where they will be most effective. This approach overcomes a major shortcoming of monoclonal antibodies, which mainly target cell-surface proteins but have enjoyed enormous success for treating cancer with 2008 annual sales exceeding $15 billion.
Technology: Altor has developed the means for producing biologically active, soluble TCR molecules in a single-chain format (scTCRs). The resulting scTCR or STAR™ molecules retain the TCRs’ ability to specifically recognize novel antigen targets on virus-infected or cancerous cells, including intracellular antigens. Altor’s unique proprietary platform provides the means to further increase the binding affinity and stability of the STAR™ molecules for commercialization. Moreover, fusion of scTCR to an effector molecule, drug, or drug carrier does not interfere with MHC-restricted, peptide-specific TCR binding activity. The simplicity of this one-step production process results in scTCR fusions with the effector molecule covalently bound, eliminating the need for chemical cross linking. Altor’s technology is protected by a broad, integrated patent portfolio that includes 30 issued U.S. & foreign patents and 57 pending patent applications.
Market: Altor’s lead STAR product, ALT-801, utilizes a p53-specific scTCR to target IL-2 to the site of human tumors. Proleukin®, a recombinant form of IL-2, is an approved treatment for advanced metastatic renal cell carcinoma and metastatic melanoma despite significant toxicities. Altor believes a less-toxic version of IL-2 could provide an opportunity to treat a much larger population of patients with renal cell carcinoma and melanoma. In Altor’s recently concluded Phase I/IIa trial, ALT-801 also provides clinical benefits to patients with prostate, head and neck and neuroendocrine cancer. An estimated 330,000 HLA-A2+ cancer patients would benefit from Altor’s STAR therapeutic candidates representing a market potential estimated at $10 B.
Guided Therapeutics, Inc. (Pink Sheets: GTHP) is a development stage company dedicated to early detection of disease that leads to cervical cancer in a rapid and painless test. The technology is designed to quickly eliminate false positive Pap and HPV results and discover cervical disease missed by existing tests. Our device, the LightTouch™ is a class III device and will require PreMarket Approval (PMA) by the FDA. Unlike Pap and HPV tests, the device does not require a painful tissue sample and results are known immediately. The annual market opportunity is estimated by the company at over $1 billion.
Operating as SpectRx, Inc., the company has previously developed and marketed the BiliChek®, a non-invasive, painless monitor for infant jaundice. The BiliChek product line was sold to Respironics in March, 2003. We are an FDA registered, ISO-13485:2003 certified designer and manufacturer of non-invasive diagnostic instruments. Our Quality System has been audited by the FDA and by several of our development partners for compliance with FDA part 820 and ISO-13485:2003. It was also audited by ETL-Semko division of the Intertek Group (http://www.intertek-etlsemko.com) on an annual basis, for compliance with the ISO 13485:2003 standard.
Guided Therapeutics has significant experience in commercializing non-invasive diagnostic technologies. The management team of CEO Mark Faupel, Ph.D., Senior VP of Engineering Richard Fowler and VP of R&D Shabbir Bambot, Ph.D have over 50 years of combined experience in the medical device field and have over 30 issued U.S. patents and numerous regulatory filings for novel technology in cancer detection and biophotonics. Beginning with our BiliChek infant jaundice monitor where we licensed the technology from the University of Texas M. D. Anderson Cancer Center and following product development, engineering and clinical testing prepared and filed a 510(k) submission for the initial claims. We subsequently filed for expanded claims for use of the product for monitoring during phototherapy after conducting additional clinical trials. In addition to US regulatory approval via the FDA, the BiliChek has been approved and is currently available for sale in over 60 countries, including the European Union, Japan, and much of South America. BiliChek sales began in 1998 and have been steadily growing. SpectRx has also prepared and filed a PMA application with its partner Roche Diagnostics for a diabetes-screening product that has not yet gone to market.
IMI has been in business for 11 years and has successfully introduced advanced products for surgical oncology. It develops and markets molecular imaging devices for the surgical detection of lymph nodes and the differentiation of cancer from normal tissue.
- Farhad Daghighian, Ph.D.: President and Chief Scientist – 11 Years with IMI: After attaining his Ph.D. in high-energy physics at UC Irvine, he worked as a postdoctoral scholar at the UCLA's Nuclear Medicine laboratory. After 2 years he joined Memorial Sloan-Kettering Cancer Center. For the following 9 years, he continued pioneering the development of a number of intra-operative nuclear medicine instruments. In 1998, he left New York to establish IntraMedical Imaging LLC in Los Angeles.
- Terry Groome, MBA: Vice President, Marketing and Sales - 6 Years with IMI: Has worked in established corporations and startups alike in capacities that include CEO, marketing, and sales. Terry has a BA from the University of Pennsylvania, as well as an MBA.
- Mike Loloyan, Ph.D.: Chief Operating Officer – 5 Years with IMI: Ph.D. in Computer & Imaging Sciences, he has been an assistant professor at UCLA radiology department.
- Barry Leon, MBA, CPA: Chief Financial Officer – 11 Years with IMI: Is partner with ABL Ventures, that has progressively invested in IMI until it became profitable.
- Node Seeker®. Radio-isotope guided sentinel node biopsy to determine the stage of cancers
- PET-Probes®. Radio-isotope guided identification of cancer tissue using F-18 FDG.
- Five years of sustainability, due to revenues from the Node Seeker® and PET Probe® line of products.
- Multiple research grants from the National Cancer Institute and US Army.
- Multiple issued patents protecting current and future products
- FDA clearance, ISO-13485 certification, and CE mark
- Exclusive distribution agreement with GE Healthcare in Europe, Middle East, Africa, and Asia
New Products in Development:
Our Phase-II NCI-funded technology is for the Marginator™ Beta Camera. The principle behind the Beta Camera is based on the detection of positrons or electrons that are emitted from certain isotopes. These emissions are collectively called “beta rays”. Beta rays have short ranges in tissue; a radiation detector that is selectively sensitive to beta rays would therefore be immune from the effect of the background and can identify small residual radio-active tissue on or near the surface.
Stage of Development of the Marginator™: The patent for the beta camera is pending. The first prototype has been tested successfully in the laboratory and the testing on surgical samples has started with good results.
Clinical Trials: In addition to this phase-II grant, NCI has granted supplemental money to support a multi-center clinical trial of the Marginator™, at Memorial Sloan Kettering (prostate and GI), Cornell (prostate), Michigan (prostate), John Wayne Cancer Institute (breast), Roswell Park Cancer Center (lung), Brigham Women’s (brain), and the U of Miami (breast).
IMI has other novel products in various stages of development, such as a position-tracked intra-operative gamma ray scanner, a biopsy needle incorporating a radiation detector for improved guidance, and a portable PET scanner for use during surgery.
Impact on Cancer Patients by Addressing the Unmet Need for Complete Excision of Tumor:
Breast Cancer: Breast cancer usually recurs in the breast because the original primary tumor was not completely resected and the remaining cells were not destroyed by adjuvant radiation or systemic therapy. The ability to intra-operatively detect the presence of cancer at the margins of resection within the lumpectomy bed would enable the surgeon to successfully complete the lumpectomy by resecting all involved tissue in one operation. This would avoid a second operation in thousands of women annually.
Current Methods: Lumpectomy specimen and several biopsy samples from the cavity are examined by pathologist. These methods suffer from under-sampling, also the results are available after the surgery is over , requiring re-operation in 20 - 40% of patients.
Our Solution: The patient is injected by FDG one hour prior to surgery. Fluorine-18 labeled deoxyglucose is commonly used as a contrast for PET scans. FDG is FDA approved and is universally accepted for having high uptake in aggressive breast cancer tissue. The Marginator™ will be used after the bulk of the tumor is removed to scan the remaining cavity for any leftover cancerous tissues. Positrons emitted from 18F in FDG guide the Marginator™ beta camera to detect residual tumor cells at the margin.
Prostate cancer: Annually, surgical treatment is offered to over 70,000 men. Radical prostatectomy involves removal of the prostate, seminal vesicles, surrounding fascia and often regional lymph nodes. This is often associated with postoperative impotence and sometimes residual cancer around the nerves (positive margins). The incidence of positive surgical margins in patients who have RRP for clinically localized prostate cancer has ranged from 14% to 46%. Cancer in the surgical margin has been shown to be a significant independent adverse factor associated with a greater risk of disease recurrence, local disease recurrence in the prostatic fossa, and systemic progression with death from prostate cancer.
Current Method: positive margins is found postoperatively by the pathologist when cancer is detected at the surface of removed tissue.
Our Solution: Hand-held as well as laparoscopic beta cameras will come to contact with the resected cavity and scan for cancer cells.
Potential Benefits of Application of the Beta Camera Marginator™:
- Sparing breast cancer patients the pain and emotional trauma of another operation or mastectomy.
- Sparing patients the trauma of loss of urine control or sexual performance in the case of prostate cancer.
- Saving the national health care system money on repeated procedures and morbidity.
- Improving patient care by enhancing surgical outcomes in melanoma, breast, colorectal, lung, brain, ovarian, and prostate ca.
Koning Corporation is a medical imaging company developing advanced imaging systems that combine the advantages of Digital X-ray and Computed Tomography called Cone Beam CT (CBCT). Producing true isotropic, high spatial resolution images, CBCT scanners can be tailored to specific rather than general applications and have the potential to be faster, smaller and less expensive to own and operate compared to current technology. The Company’s first product, Koning Breast CT (KBCT) is a dedicated Cone Beam CT scanner producing thin multi slice and 3D images of the breast tissue for improved detection and diagnosis of breast cancer compared to mammography which is a limited 2D imaging technique.
Breast cancer is the most frequently diagnosed cancer in women, accounting for 32% of cancer occurrence. It is the second leading cause of cancer death among all women and the leading cause of death in females 40-59 years of age. At this time breast cancer can not be prevented, therefore early detection when it is most treatable and associated with an increased survival rate is critical.
Mammography is the current standard of care for early detection. When an abnormality is detected at screening mammography, the patient undergoes diagnostic workup. The workup includes additional mammographic views, spot views, and magnification views, which in total are referred to as a diagnostic mammography exam. Between 2 to 11 additional views are common. These additional diagnostic views can be non-conclusive as they are restricted by mammography’s known limitations. Also, radiation dose to the patient can be substantial as more views are taken. In addition to superior clinical performance over diagnostic mammography, KBCT offers the following competitive advantages:
- Isotropic Resolution
- Full 3d Visualization of the Entire Breast.
- Ultra Thin Slice Data in Any Plane
- 90 Second Image Reconstruction
- 3D -Add-on Biopsy Device
- HIS, RIS, and PACS Connectivity
- Internet Access to Images and Patient Data
The Company was established in 2002 as a Delaware “C” Corporation and is registered with the Food and Drug Administration (FDA) as a medical device establishment. It was founded on extensive scientific research on CBCT funded by $8.3 million in grants from NIH and conducted at the University of Rochester Medical Center (URMC). Koning’s founder and President, Ruola Ning PhD, a Professor of Imaging Sciences, Biomedical and Computer Engineering at URMC, is a renowned scientist and a leading authority on Cone Beam CT. He and his team have published over 35 papers on CBCT and his research has resulted in nine US patents licensed exclusively by URMC to Koning Corporation on a worldwide basis.
San Diego-based Lpath is the category leader in lipidomics-based therapeutics, an emerging field of medicine that targets bioactive signaling lipids for treating a wide range of human disease. Lpath’s ImmuneY2™ drug-discovery engine has the unique ability to generate therapeutic antibodies that bind to and inhibit bioactive lipids that contribute to disease, thereby opening up an entire universe of opportunity in drug discovery. Lpath has developed a humanized mAb directed against an important tumor growth-like factor, Sphingosine-1-Phosphate (S1P). We believe that this antibody can be effective in reducing tumor cell proliferation and protection from apoptosis, tumor-associated angiogenesis, metastatic potential and resistance to chemotherapeutic agents.
The first mAb used in the clinic for the treatment of cancer was Rituxin (Rituximab) which was launched in 1997. Since then it has reached sales in excess of $2.5 bill per year and has demonstrated the utility of biospecific mAb as therapeutic agents. Not surprisingly, 17 other mAb have since been approved of marketing, including 7 that are prescribed for cancer. The success for these products, as well as the reduced time to develop mAb has made mAb therapeutic the second largest category of drug candidates behind small molecules. Further, the exquisite specificity of antibodies as compared to small molecule therapeutics has proven to be a major advantage both in term of efficacy and toxicity.
S1P is a novel target for the treatment of cancer. Cancer cells exploit the sphingolipid rheostat by promoting conditions that favor the production of S1P release into the extracellular compartment. The ability of cancer cells to release S1P into the tumor microenvironment promotes the infiltration of platelets, fibroblasts, mast cells and neutrophils resulting in an inflammatory response. The infiltrating cells promote further release of S1P into the tumor microenvironment with the resulting manifestation of tumorigenic and pro-angiogenic effects of S1P. Targeting S1P with Lpath’s mAb approach is a potential new way to attack cancer. The company is currently advancing three drug candidates, two of which—ASONEP™ for cancer and iSONEP™ for AMD—are in late-Phase 1 clinical trials. For ASONEP, Lpath has joined with international drug giant Merck-Serono under a worldwide exclusive license and development partnership.
Lpath’s evolution from a start-up incubated in an academic lab to a publicly-traded entity with an already established management team, complete with a scientific advisory board and two clinical advisory boards, provides evidence that we can handle the ever-changing management needs of a growing biotech company.
MagArray Inc. is a start-up company spun out of Stanford University to commercialize the magnetic biochip technology developed under Federal funding. All the founders participated in the original biomolecular assay development program focused on labeling target molecules with magnetic nanoparticles and detecting them with an ultrasensitive magnetic sensor array. MagArray Inc. presently occupies 5,000 square feet of lab space in a molecular sciences incubator facility, and has access to the Stanford Nanofabrication Facility on a fee-for-services basis. MagArray Inc. is financed by private investments and government grants, and has exclusive licenses of core intellectual properties developed at Stanford University as well as within MagArray.
There is an evolving revolution in diagnostic medicine being driven by the availability of molecular diagnostic assays. This revolution will accelerate as biomarker panels are established for more and more diseases. Perhaps the most active area in cancer research is the search for biomarker panels specific to the different species of cancer, either for therapy prediction/monitoring or for earlier diagnosis. When such panels are identified, the need for a high-sensitivity multiplexed assay will also explode. Such diagnostic assays will be necessary in the research stage as platforms for the establishment of the clinical efficacy for the biomarker panels, but the market will reach its peak size once the biomarker panels reach the clinical application stage. No presently available molecular diagnostic assay such as ELISA or Luminex meets the need of the marker panel application. The unique and remarkable capabilities of the MagArray assay system make it a prime candidate to facilitate and underpin the revolution in diagnostic technology presently emerging in diagnostic medicine. MagArray Inc.’s vision is that we become a key assay platform supplier to the molecular diagnostics field. Our initial focus is on a cancer diagnostic assay for the research market and a cardiac assay for the clinical market.
MagArray Inc. has demonstrated the detection of proteins such as carcinoembryonic antigen (CEA) down to 0.01 pg/mL (Nature Medicine, Oct. 11, 2009) and troponin (Tn) down to 1 pg/mL concentrations. We have also demonstrated high sensitivity and selectivity in multiplex assays where 4-20 biomarkers are detected simultaneously (PNAS, Dec. 30, 2008). MagArray Inc. is actively developing both the bioassay chemistry and the actual hardware on which the measurements are carried out, including the giant magnetoresistive (GMR) sensor chip (Figure), the chip cartridge and the chip cartridge reader station. The GMR sensor chip features 64 sensors in an 8x8 array, which can be individually functionalized with different antibodies.
Clinical Needs - Improved minimally invasive products are needed to differentiate cancerous and non-cancerous tissues in vivo. The present standard of care is invasive, costly, and inefficient. In our targeted applications (bladder and gastro-intestinal tract), there is no clinically accepted alternative to the technique that has been used for over 50 years: excisional biopsy and histo-pathological assessment of polyps or other suspicious lesions. These methods cause bleeding, pain, risk of organ perforation, and other complications. Due to the invasive nature of these existing techniques, only a few samples of tissue may be collected during a procedure, and the selection of sampling sites is based solely on visual appearance. Results are sometimes not available for days, causing unnecessary patient anxiety and delays in scheduling treatment. Lab samples can be mislabeled or lost, which can result in unnecessary treatments that include a full regimen of chemotherapy. A technique is needed that mitigates these risks, costs, morbidity, and limited sampling.
Technology and Benefits – With a flash of light, Optimum Technologies’ (OTI’s) minimally invasive technology differentiates normal and malignant tissues. The system uses the technology of polarized elastic scattering spectroscopy (PESS). It therefore requires no indicator dyes, molecular markers, or other exogenous (foreign) agents, so no new FDA drug clearance is needed. This means faster time to market than many other methods currently under development. Because it is a point measurement system rather than an imaging modality such as NMR, CT, OCT and ultrasound, PESS is simple, low cost, and easy to use with minimal training. The system will produce a probability of cancer for each reading. There are no complex images to interpret, and resultant inter-observer variations are eliminated. OTI holds 2 issued patents for PESS, has filed 2 provisionals, and has other patent applications in preparation. Regulatory and reimbursement strategies are defined.
Business Opportunity – Worldwide each year there are 356,000 new cases of bladder cancer, 410,000 new cases of esophageal cancer, and 940,000 new cases of colorectal cancer. PESS is capable of detecting carcinomas in all of these organ systems, as well as skin, mouth, brain, sinuses, lungs, and cervix. Ongoing clinical studies have demonstrated the potential of this “optical biopsy” technique to differentiate normal and diseased tissues with very high accuracies. The overall US markets for gastrointestinal devices exceeded $1.2 billion in 2007. Driven in part by demand for minimally invasive devices to diagnose colorectal cancer, this market is expected to increase to over $1.6 billion in 2011. Moreover, the use of instant diagnostic technologies such as PESS will produce a cost savings of over $95 million just for the 1.6 million annual colonoscopies which will no longer require pathology workups in the lab. Most gastrointestinal endoscopy is performed in endoscopy suites in hospitals. There are ~6,000 hospitals in the U.S. and double that number abroad.
Financial Overview - OTI’s business plan calls for cumulative sales in the first 5 years of $1B. Much of this revenue will be comprised of disposable probes with gross margins in the range of 70% and pre-tax earnings in the range of 15-20% of revenues. OTI seeks funding to augment its NIH support. Funds will be used to continue product development, conduct pivotal human studies, execute it’s regulatory and reimbursement strategies, and establish distribution partners. Initial products can be launched in less than 30 months.
Company and Team – OTI is a medical technology- and product-development company founded in 1994 that has assisted hundreds of companies commercialize new devices. OTI has been developing the PESS technology on its own using NIH and internal funding. Founder/CEO Randal Chinnock has 30 years of experience in the development and commercialization of electro-optical, opto-mechanical, and surgical devices and instrumentation. VP of R&D Fred Bargoot holds a Ph.D. in molecular physics and has 35 years of experience successfully developing and managing the development of products for medical device, industrial, biotechnology, clinical diagnostics, and food and dairy markets. Program Manager George Grubner holds an MS degree in Electrical Engineering from Harvard University and an MBA from Babson College, and has over 30 years of experience developing and commercializing electronic instrumentation systems encompassing hardware, electronics, firmware, and control architecture. Long-time collaborator Irving Bigio, Ph.D. is a professor of Biomedical Engineering and Computer and Electrical Engineering at Boston University and Director of the Biomedical Optics Laboratory at BU. He is a pioneer in the use of elastic scattering spectroscopy for optical cancer detection, and has been a key contributor to OTI’s technology development efforts. Satish Singh, M.D. is conducting a clinical study in the colon using OTI hardware at Boston Medical Center. Louis Liou M.D. is conducting a clinical study for OTI in the bladder at a Harvard affiliate hospital. Lev Perlman, Ph.D. is a collaborator at Beth Israel Deaconess Medical Center and is conducting a clinical study in the esophagus using OTI hardware.
Pathfinder Therapeutics, Inc. ("PTI" or "the Company") is pioneering the development of 3 dimensional imaging and real time abdominal navigation to improve disease management, treatment planning and intra abdominal guidance. The Company has developed core image guided software and guidance system that improves the ability of the clinicians to identify the best course of treatment for a patient, enhance the ability to deliver that treatment, and measure the effectiveness of that treatment over time. The Company’s initial focus is on liver cancer with products to include the kidney and pancreas to be released near term. Other key organs will be added to the product offering as well.
Specifically, the Company has developed an image based guidance platform consisting of imaging software for pre-treatment – PlaniSight – and SurgiSight, an intra abdominal navigation system. PlaniSight and SurgiSight are FDA cleared for open liver resection and ablation procedures.
The Planisight planning module operates initially as a disease management platform, rapidly and automatically producing high quality images and volumetric data to enable clinicians to identify tumor locations and progression over time. Key data produced through this analysis enables physicians to assess the effectiveness of previous treatments and determine future courses of treatment. If the treatment solution is a surgical resection or ablation of a tumor, PlaniSight automatically creates 3D image segmentations of the liver, tumor, and vascular structure. The physician can then create a resection plane to optimize the surgical outcome. The software automatically provides the physician the volume measurements of the total liver, functional liver, the resected and remnant portions of the liver.
The SurgiSight Navigation system enables real time guidance to accurately follow the pre-operative plan once in the OR environment. The objectives of a liver resection procedure is to remove the necessary volume of the organ to ensure the removal of the tumor and tumor cells while leaving as much healthy liver tissue as possible to enhance patient recovery and accelerate organ regeneration. The navigation system provides the surgeon with a tool to reach these objectives more effectively.
PTI is developing intra abdominal guidance systems for use in minimally invasive resection and ablation procedures for the liver, kidney, and pancreas. Our minimally invasive navigation system will provide the same benefits found in open resections and ablations. These applications will allow more patients to be treated more effectively than current treatments offered and will increase the utilization of the Company’s products across physician specialties.
As PTI establishes intra abdominal guidance as a standard of care for minimally invasive and open resection and ablation procedures for the over 700,000 patients diagnosed annually with liver, kidney, and pancreatic cancer, the Company will be a prime target for acquisition. Medical device companies will see the improvement in outcomes of the ablation procedures where their products are utilized due to accurate placement of their devices. Current image guided companies focusing on rigid structures including skull, spine, and orthopedic joint will see the opportunity in this new burgeoning field of soft tissue guidance and realize the opportunity to add a vibrant market to supplant their maturing markets. It is the company’s belief Covidien, Acculis, BSD Medical from the device industry and Medtronic, GE Medical, and Stryker from the image guided industry, to name a few, would have an interest in acquisition. Each has a history of acquiring companies and technology to enter new markets.
Progenra Inc is a leader in companies focusing on ubiquitin cell pathways, which have an essential role in a number of pathophysiologies. The company’s mission is to discover new medicines addressing unmet medical needs. Progenra’s drug discovery platform and unique profiling capacity have identified first in class selective inhibitors of critical ubiquitin pathway enzymes associated with cancer, osteoporosis, and other debilitating diseases. Among these is a highly promising anti-cancer compound, currently in development for the oncology market.
Company and the team: Progenra is a seven year old biotechnology company driven partly by private investments. The company has built state of the art laboratory facilities in the Great Valley Corporate Center, a biotech hub of the western Philadelphia suburbs. The Progenra team is composed of seasoned management, formerly from GSK. Dr. Tauseef Butt, President and CEO of Progenra, has raised several million dollars and brought numerous technologies and products to the market. Dr. Michael Mattern, COO of Progenra, has 20 years of experience at GSK in anticancer drug development. In addition, Progenra is honored to have a highly distinguished Scientific Advisory Board.
Competitive Edge: Progenra is at the forefront in the discovery of innovative medicines modifying the ubiquitin pathway: Its research program is enabled by novel targets, superior platform technologies, and in-house medicinal chemistry facilities. Progenra’s competitive advantage is its single minded focus on the ubiquitin pathway, for example, the company has expressed, purified and characterized all the enzymes of the human de-ubiquitylase family (~100 members). Progenra is thus set up to reject non-selective compounds early in the development cycle, thereby reducing the risk of failure of compounds in clinical stages. The most advanced program at Progenra is a first in class unique de-ubiquitylase targeting compound.
Market Opportunity: The ubiquitin pathway affords numerous novel therapeutic targets. Progenra pursues a discovery program that has created several opportunities to establish partnerships with other companies. Progenra is interested in discussing: Discovery Collaborations with companies interested in exploring their internal compound libraries. Co-development Partnerships with companies seeking the power of Progenra’s repertoire of platforms to enable the selection of chemical entities most likely to succeed in the clinic. The company’s most advanced program has identified a de-ubiquitylase inhibitor currently in pre-clinical development. This molecule is first in its class of drugs with a unique anticancer mechanism. Progenra is actively seeking investment and partnership opportunities centered on this molecule.
Syntrix Biosystems, Inc. (Auburn, WA) is a privately held pharmaceutical company that opportunistically identifies low- to moderate-risk small-molecule development opportunities that address high-value unmet needs in therapeutic markets that include oncology, rheumatology, dermatology and pulmonary disease. The company’s strategy is to rapidly move its pipeline of therapeutic candidates through clinical and regulatory development to maximize deal valuation at the time of partnering with established multi-national pharmaceutical companies. Since its inception, Syntrix Biosystems has been awarded over $15 million in non-dilutive financing from the National Institutes of Health in support of its development efforts.
Pipeline: The company’s preclinical and clinical-stage pipeline of oral small-molecule candidates include:
- rac-aminopterin (a new antifolate aimed at capturing key high-value markets in rheumatology and dermatology, highlighted below),
- the neutrophil chemotaxis inhibitor SX576 aimed at the COPD and other inflammatory disease markets (SX576 allosterically inhibits the G-protein coupled receptor CXCR2), and
- novel formulations and analogues of a non-opioid analgesic aimed at capturing the existing tramadol market (there were 18,526,000 tramadol prescriptions in 2007 and $281 million in retail sales).
Highlighted Pipeline Investment Opportunity: Given the critical dual role that antifolate therapy with the mainstay methotrexate (MTX) plays in both oncology and inflammatory disorders, there has been intense interest in developing improved antifolates (there are ~4,000,000 MTX prescriptions per year, mainly for arthritis and psoriasis). In oncology, the recent approvals of pralatrexate (Folotyn™, Allos Therapeutics) for peripheral T cell lymphoma (PTCL) and pemetrexed (Alimta®, Eli Lilly & Co.) for mesothelioma and non-small cell lung cancer (NSCLC) provide vivid evidence that interest in improved antifolates in oncology is brisk.
In the treatment of inflammatory disorders, 87% of MTX users have adverse side effects and 30% have to withdraw within a year due to either its toxicity or lack of efficacy (i.e. MTX ‘failures’). Patients with rheumatoid arthritis (RA) and psoriasis who fail standard therapy with MTX are additionally treated with biologic (anti-TNF) drugs such as Enbrel®, Remicade® and Humira®. The cost of biologic therapy is approximately $40,000 per year for each patient, and total biologic sales for RA and psoriasis in 2006 were over $10 billion.
There is therefore an urgent and high-value unmet opportunity in the current pharmacopeia for a new antifolate that provides superior clinical efficacy and less toxicity than MTX in oncology and inflammatory indications, and that is priced between MTX and biologics.
Racemic-aminopterin (rac-AMT) is a proprietary antifolate being developed by Syntrix Biosystems, Inc. as a “better methotrexate” that is aimed at meeting these unmet needs in oncology, RA and psoriasis. Laboratory and clinical data suggest that rac-AMT is more effective than MTX in oncology and inflammation and causes fewer side effects. For those with RA and psoriasis who have failed therapy with MTX, rac-AMT thus offers a therapeutic value proposition similar to that of biologics but at a lower cost, and with the added benefit of convenient once-weekly oral dosing. A superior and better tolerated oral therapy with otherwise identical prescribing to MTX would be adopted quickly by clinicians. The domestic rac-AMT market is estimated at $1.1 to $2.6 billion.
Business / General: The business of Visualase is to develop, manufacture, and sell minimally invasive laser ablation systems and their related disposables, for the purpose of destroying tumors and other soft tissue. The Visualase technology is proprietary, strongly IP protected, and game changing. The Visualase system is fully developed, already FDA cleared with a broad indications, and is currently being used commercially in the US. Visualase, because of the unprecedented level of control and precision offered by the technology, faces several large patient populations who have no options, in markets that have never been accessed by previous ablative technologies. Excellent reimbursement exists and average sales price will reflect this fact. Manufacturing, completed at our Houston facility is simple, scalable, and low cost leading to high gross margins. Disposables will represent > 80% of the total revenue stream.
Technology & Market: The Visualase system allows visualization, control, and monitoring of laser ablation procedures in real time, as well as modeling of the of the kill zone while it is being created. The sharp demarcation of dead vs. unharmed tissue (<1mm) associated with laser, combined with the ability to monitor and control the to sub-millimeter levels of accuracy, offers a level of precision never before attained with ablative therapies. Visualase is actively pursuing applications in brain tumors, focal (tumor specific) prostate cancer, and epilepsy. The Visualase system is a platform technology facing several large markets (as shown in the table) in which there are limited or poor patient options, poor or no competition, aggressive physician call points and high reimbursement.
- Proven platform technology that solves unmet clinical need in multiple markets
- Human clinical study on brain tumors completed, published in peer reviewed journal Neurosurgery
- Patients currently being treated and enrolled in clinical protocols for prostate cancer.
- Low technical and regulatory risk: all products FDA 510(k) cleared for marketing in the US
- $3.5M Series A financing recently completed through Toronto based Eventi Capital Partners
- Patented technology: five patents granted, eleven pending, >200,000 lines of proprietary software
Bill Hoffman, CEO — Bill Hoffman has extensive commercial experience with early stage medical device companies, especially those focused on novel and potentially disruptive technologies. Bill’s previous positions include Chief Operating Officer at Rubicor Medical which was focused on image guided percutaneous lumpectomy and a novel hand held breast biopsy device. Prior to that, Bill was VP of Sales at FoxHollow Inc., building from scratch a team of 250 sales professionals, and driving arguably the fastest revenue ramp in the medical device history: from $0 in Q2 2003 to $138M in calendar year 2005. Bill also served as Director of Sales for RITA Medical Systems, the first volumetric RF ablation company in the US and helped execute a plan which resulted in a successful IPO in 2000.
Ashok Gowda Ph.D., Founder and COO — Dr. Gowda has over 10 years experience in directing medical device technology development and has contributed to the Visualase effort on both the scientific and management fronts. Dr. Gowda led the company through the development phases and through regulatory approvals including the initial human clinical studies. Dr. Gowda received his B.E. degree in Biomedical Engineering from Vanderbilt University in 1991 and the M.S. and Ph.D. degrees in Bioengineering from Texas A&M University in 1998.
Roger McNichols Ph.D., Founder and CTO — Dr. McNichols is responsible for overseeing all aspects of research and development of the Visualase technology. Dr. McNichols has extensive experience in biomedical optics, software, and magnetic resonance technology. Dr. McNichols received a BS in Electrical Engineering from the Ohio State University in 1992 and a Ph.D. in Biomedical Engineering from Texas A&M University in 1998.
Zacharon is leveraging innovative drug discovery technology to enable, for the first time, development of small molecule drugs targeting glycan biosynthesis. Glycans are the carbohydrate chains of glycoproteins, proteoglycans, and glycolipids and encompass a broad selection of specific and potent drug targets. Our most advanced programs are in lead optimization for oncology and lysosomal storage disease. The lysosomal storage disease market currently exceeds $3B and is characterized by strong growth rates, attractive operating margins, and limited competition.
Based on strong preclinical evidence, targeting discrete points in glycan biosynthesis represents a highly specific and potent therapeutic strategy for cancer, lysosomal storage disease, inflammatory disease, and other applications. However, several key challenges have until now precluded the development of drugs targeting glycan biosynthesis:
- The biosynthetic pathway, structure, and resulting function of glycans were not well understood
- Methods for analyzing the highly complex glycan structures, central to glycan-driven pathology, were not available
- Effective high-throughput small molecule screening technology did not exist
To unlock this potential, over the past 5 years Zacharon has created breakthrough assay technologies integrating cellbased high-throughput screening with highly sensitive glycan structural analysis tools. Combined with recent advances in the understanding of glycan biosynthesis and function, Zacharon’s technology provides a powerful and proprietary platform for novel small molecule drug discovery of an entire new class of therapeutics. By coupling this drug discovery engine with traditional downstream small molecule drug development, Zacharon is creating a broad pipeline of novel therapeutics for many diseases where glycans are strongly implicated in disease pathogenesis.
Since its inception in 2004, Zacharon has been awarded nearly $3M in SBIR grants and recently received Series A funding from Avalon Ventures. The company has executed an intellectual property strategy involving patent filings and trade secrets surrounding its assay technologies, glycan targeting approach, and small molecule drugs.
Zacharon has completed high throughput screening and has initiated medicinal chemistry and other preclinical activities. Proof of concept has been demonstrated in oncology and lysosomal storage disease models for the Heparan Sulfate program.