Company Overview: Neurono is developing a device via which small molecular (<40,000 MW) therapeutics can diffuse through the skull into brain parenchyma. This technology will have a wide array of applications (e.g. inflammatory CNS processes). The relevant intellectual property will be in-licensed from the NIH. Competitive Analysis: This process is differentiated by its minimally-invasive nature. Currently, CNS drug delivery is accomplished via intrathecal administration or surgical implantation of a cranial shunt (invasive procedures with associated costs and complication risks). Value Proposition: Neurono’s novel process is preferable to current modalities, which are time-consuming, expensive, and associated with significant morbidity. We expect our consumers to benefit from reduced complications and readmissions of their patients as timely therapeutic administration lessens CNS disease burden. Milestones and Strategic Vision: R&D - Characterize diffusion characteristics of human skulls (has been tested in mice, rats, and pigs thus far), and intact meninges - Map bone architecture to determine optimal drug administration point (currently at lambda) - Develop a working prototype, establish safety and efficacy in humans, obtain FDA approval, and file for IP protection Business Strategy - Publish significant data on the efficacy of this process/device, and present at major conferences to build industry support - Outsource manufacturing - Begin with in-house small-scale rollout to hospitals. Then pursue a strategic partnership to achieve large-scale sales and marketing. Subsequently, selling the company to a major device manufacturer is a likely exit - R&D needed to bring this to trial could be accomplished in 3-5 years Funding - Will need ~$500,000 in initial funding for pig studies, and ~$700,000 total to bring to trials. Team: Neurono is led by Josh Warren and Keyvan Mirsaeedi-Farahani, two MD/MBA candidates at UPenn Med and Wharton/Harvard. Expert advisors include Drs. Nichtberger and Litt, who have 40+ years combined healthcare entrepreneurial experience. Dr. Nichtberger has had successful exits, including a $230M IPO, while Dr. Litt, in addition to entrepreneurial endeavors, is also an active neurology researcher and clinician as the Director of the Center of Neuroengineering and Therapeutics at UPenn.
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Executive Summary: Angio360 Diagnostics specializes in the development of antibody-based products to detect cancer recurrence and tumor vascular health in blood samples and in diagnostic imaging. Utilizing patented biomarkers of tumor-associated blood vessel growth, our AngioTracer Blood Panel, and Imaging Panel detects tumor recurrence earlier and with greater sensitivity providing more effective treatments, better outcomes, and is an effective tool to screen new therapeutics. Marketed to pharmaceutical companies for research pre-clinical and clinical trials, as well as oncologists, and hospital systems, these detection tools will help improve the drug development process and enhance targeted drug treatments. Prospective Uses: Blood Panel Monitor the efficacy of anticancer therapeutics. Screen patients for early tumor recurrence. Imaging Panel Pinpoint areas with active tumor growth and assess vascular health. Guide therapeutic intervention procedures. Market Opportunity: Getting a single drug to market can cost up to $350 million and has a high failure rate. Our diagnostic products offer pharmaceutical companies the tools to reduce costs and more successfully validate the effectiveness of new drugs in the lab, and in the patients. Our products can also be used by oncologists to design custom treatments for cancer patients. Since our products detect tumor-associated blood vessels, common to most cancers, our diagnostic tools can be used for a wide range of cancer types. Especially those in which there are currently no reliable blood biomarkers, such as glioma and other brain-related cancers. Timeline in months: 18 Commercialization of the Blood Panel for pre-clinical and clinical research studies. 36 Commercialization of the Imaging Panel for pre-clinical studies. 48 Commercialization of the Blood Panel for the oncology patient market. 84 Commercialization of the Imaging Panel for use in human research studies. Funding: Initial funding will be obtained through an SBIR grant application in 2016, matching non-profit seed fund and VC/Angel investment. Management & Advisors: Our team has extensive experience in cancer related angiogenesis, clinical trial design and execution as well as experience in biotech business development and management. Along with a team of advisors familiar with development of start-up companies, antibody development, cancer treatment, and local financial resources our success is ensured.
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We at NeuroNostix believe that a deeper understanding of a patient’s underlying physiology is essential to providing that patient with the best possible care. Schizophrenia is often the result of overactive or overexpressed dopamine D2 receptors (D2R); with schizophrenic patients in mind, we are developing highly selective D2R radioligands to categorize a traditionally heterogeneous population into groups with specific physiological characteristics that can guide physicians towards a better course of treatment. Our product is a D2R receptor antagonist that displays high selectivity for this receptor compared to other compounds available on the market. The patent for the compound is assigned to Dr. David Sibley of the National Institute of Neurological Disorders and Stroke (NINDS). Currently there is little competition in the marketplace for the molecular imaging of schizophrenic patients. The market for molecular imaging utilizing radioligands is expected to grow at a CAGR of 18% to $4.2 billion through 2017, with a greater growth rate of 25% for targeted ligands. We plan to take advantage of this expanding market for personalized medicine by offering solutions for the 150,000 newly diagnosed patients annually and the 3.2 million patients currently living with schizophrenia in the United States. Our general business strategy revolves around the completion of three milestones. Milestone 1 involves partnering to synthesize the raidoligand and completing pre-clinical and phase I clinical trials with the assistance of CROs. Milestone 2 involves establishing sales to university-based and clinical researchers interested in using our radioligand as well as beginning phase II and III clinical trials. Milestone 3 will be completed with the sale of NeuroNostix to a pharmaceutical company looking to pursue technologies in the imaging and diagnostic space. We expect the development and completion of these milestones to cost $1.5 million. We plan to seek out SBIR/STTR grants, and “BRAIN” Initiative grants as well as angel investment and crowd funding options in order to provide us with the capital needed to achieve our goals. We expect to generate revenue after the completion of milestone 1 from sales of our radioligand for research purposes and at milestone 3 with the sale of NeuroNostix.
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Our mission at Hemisphere Pharmaceuticals is to provide customers suffering from neurodegenerative diseases with medications that prevent the symptoms, delay the progression, or cure their illness. We seek to offer these medications as treatment options that are competitively priced with current therapy options but have far less undesirable and toxic side effects. It is our hope that the use of our drugs allows our customers to overcome their debilitating diseases and lead uninhibited lives. Hemisphere Pharmaceuticals plans to initially focus on the development of the company’s platform drug, TFP5, a cyclin dependent kinase 6 inhibitory peptide based drug. TFP5’s competitive advantages come from the lack of continuously effective current treatments available to halt the progression of Alzheimer’s disease (AD). Because the TFP5 product contains naturally occurring molecules, we can expect a lower risk for adverse reactions. Furthermore, this invention provides a useful platform for developing additional peptides for treatment of other neurodegenerative diseases; an important future step for Hemisphere Pharmaceuticals is to investigate other potential applications. This plan aims to outline a series of investments necessary to complete Phase II clinical trials in 6 years. The Pre-clinical studies, Phase I trial, and Phase II trial require $1.25 million, $2.5 million, and $16 million, respectively. However, we will assess exit opportunities after each phase. The immediate action items in the first year are to develop a delivery system for TFP5 and obtain lab space to complete necessary pre-clinical animal studies prior to filing an IND with the FDA. Hemisphere Pharmaceuticals plans to keep its facilities local as New Orleans is committed to increasing the presence of biotechnology within the city and making it a larger part of their economy. Hemisphere Pharmaceuticals boasts a core team of graduate students with diverse expertise in clinical trials, pharmacology, neurodegenerative disorder research, biomedical engineering, and health sciences start-up as well as an advisory board with extensive experience in start-up business. The local assets and strong management team equip Hemisphere Pharmaceuticals for success.
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EncepHeal Therapeutics develops products based on innovative and unique neuroscience research. We are a CNS therapeutics company focused on early stage development with a goal of creating lucrative pharmaceutical collaborations. Our lead product (ECH-201) targets individuals who are either currently suffering from or are at a significant risk for developing drug addiction, attention-deficit/hyperactivity disorder (ADHD), and narcolepsy. A total of about 58 million children are diagnosed with ADHD, where the cost of illness can be as high as $52 billion. There are also about 2.5 million people who have been treated for drug addiction as well as 23 million people who currently struggle with drug addiction (Figure 1). ECH-201 is an inhibitor of the dopamine transporter (DAT) that selectively prevents dopamine removal from the synapse. Drugs in this class, such as modafinil, have been FDA approved to treat narcolepsy. Further, it has been suggested that the mechanism of these drugs would provide potential therapy in drug addiction and ADHD. The modified modfinil-like structure of ECH-201 provides a unique therapeutic action and improved binding affinity, resulting in improved efficacy and a lower dose for the patient over the currently approved product, modafinil. Initial preclinical studies that demonstrate the safety and effectiveness of ECH-201 in drug addiction, will provide the basis for an IND application for the initiation of Phase 1 clinical studies. At EncepHeal Therapeutics, we are investing in developing a therapeutic product which we intend to outlicense to or jointly develop with an established pharmaceutical company in neurology. Collaboration with large pharma allows for effective late-stage development, commercialization and marketing. Our management team will be seeking funds through venture investments, grants, and collaborative industry partnerships to balance cash flow against burn rate in the early stages. Our business model will be virtual, utilizing vendors to perform development activities, such as drug substance and drug product manufacturing. While we plan to seek strategic partnerships, we are open to exploring options such as acquisition and IPO. With success, our investors should expect a significant return on investment.
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Autoimmune diseases are chronic and often incurable disorders with few effective treatments. Among these treatments are powerful immunosuppressive drugs such as Tysabri - the last line of hope for Crohn’s Disease and Multiple Sclerosis patients. However, these drugs render patients at risk for fatal infections such as Progressive Multifocal Leukoencephalopathy (PML) which is caused by the John Cunningham (JC) virus. To safely use immunosuppressives, physicians need to identify patients at risk for PML using accurate tests. In the case of JC virus, this represents a significant challenge as 58% of the world population are infected. The current antibody-based diagnostic is efficient at detecting the JC virus and the test is used as a basis to deny patients treatment with immunosuppressives. However, the risk of PML is only significant in patients who carry a virulent variant of JC Virus and most patients carry a harmless variant. We propose to develop and market the JCKey Diagnostic Assay, a state-of-the-art test that can determine which strain of the JC virus a patient carries. Because non-virulent variants are known to seroconvert into virulent variants, it is prudent to test immunosuppressed patients every month, ensuring that they continue to receive the appropriate treatment. In addition to the superior sensitivity and specificity, the manufacturing cost of our diagnostic is significantly lower than the existing test. This is an advantage to patients and payors, however we feel that the primary beneficiaries of this assay will be Biogen, the makers of Tysabri. We estimate that if physicians and diagnostic laboratories were to be able to take advantage of our diagnostic test then the eligible patient population for Tysabri would increase significantly - potentially tripling in countries where the test is available. We plan to capitalize on the robust sales and distribution channels established by Biogen and Roche in order to commercialize the technology. Specifically, our exit strategy is to develop the intellectual property (IP) into a working diagnostic and make the technology available for acquisition. We view Biogen as the most likely bidder for this diagnostic, given that the company is subsidizing all the costs for the current antibody-based assay.
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There are more than 90,000 patients currently living with Neurofibromatosis Type I (NF1) in the USA. NF1 is a genetic disease caused by the malfunction of neurofibromin, a regulatory protein. Most of the patients develop benign tumors that can be removed by surgery. However, 1 in 20 will develop malignant tumors, such as malignant peripheral nerve sheath tumors (MPNST) and, optic and malignant gliomas. MPNST do not respond to standard chemotherapy or radiation therapy and current treatment options do not improve the 5-year survival beyond 50%. Current clinical trials for these tumors include drugs that also affect normal cells causing several side effects. Hecate OncoSolution is a Houston based startup with the goal to translate basic science findings into effective treatments by developing channels and collaborations to expedite this complex route for promising antineoplastic agents. Our first product, “Macar-A” (Schweinfurthin-A), is a suitable therapeutic solution for benign and malignant tumors developed in patients with NF1. Macar-A is a small molecule that affects only NF1 mutant cancer cells by disrupting their cytoskeleton without affecting normal cells. Currently Macar-A is in early experimental phase. We will be licensing the technology from National Institutes of Health (US Patent 8,686,016, Issued April 01, 2014). Our first milestone is to complete pre-clinical experiments by 2018 with an initial investment of $1 million and the collaboration of academic research laboratories. In 2019 we will initiate the Phase I clinical trial for the FDA approval process with an orphan disease indication for malignant glioma and MPNST; this require an investment of $1.5 million. Once Macar-A clears Phase 1 and 2 clinical trials we will introduce it as a complementary therapy to surgery for invasive neurofirbomas and other NF1 related cancers. Based on our conservative estimates, we project that Macar-A has a $112.5M annual revenue opportunity only for MPNST in NF1 patients in the US only with a market penetration rate od 25%. Furthermore, by addressing other NF1 related malignancies we will increase this revenue. Hecate OncoSolution expects to go through all the road to take Macar-A into clinical implementation. We are a very strong team with diverse scientific, medical, business and life science entrepreneurs from Texas Medical Center, the world largest medical center.
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At nüros, our mission is to revolutionize drug delivery to the brain. We aim to provide a viable treatment option for traumatic brain injury (TBI) and concussion. The platform can be leveraged to deliver drugs for strokes and other neurological disorders in the future. Our initial technology, to be branded nüpass™, will be developed as a first-ever therapy for TBI and concussion. It is a user-friendly micro-needle patch device that painlessly delivers therapeutic agents through the skull. This transcranial delivery technology can focally perfuse FDA-approved neuroprotective drugs directly to the brain injury site. It will prevent long-term brain damage while avoiding open-skull surgery. We expect the technology can be readily leveraged for stroke, encephalitis and multiple sclerosis. According to GlobalData, the TBI drug market in 2011 was valued at US$1.25 billion and expected to grow at 2.8% to reach US$1.55 billion by 2019. No competing concussion therapy products in the market have been identified. Potential competitors developing new drugs for concussion are still in the preclinical or Phase I stage. We can go to the market rapidly by providing an optimal vehicle of delivery for FDA-approved drugs with our new technology. Partner with neurosurgeons to administer and prescribe nüpass™ for post-surgical TBI and concussion patients. Subsequently, we will target defense, schools and sport organizations where there is a high occurrence of repeated concussions, as well as ambulances and emergency rooms with emergency treatment kits. We will perform pre-clinical testing of target tissue penetration and develop efficacy endpoints in large mammals using a Contract Research Organization (CRO). Our next stage is initiating a clinical trial with UCSF Brain and Spinal Injury Center. We will develop our IP position during both stages. Chief Executive Officer: Chuchu Zhang, senior neuroscience Ph.D. candidate, UC San Francisco Chief Scientific Officer: Hye Young Lee, Ph.D, Postdoc, Department of Physiology, UC San Francisco Chief Financial Officer: Huixin Yang, MBA candidate, UCLA Anderson, School of Management Chief Legal Officer: Lizzie Shaxuan Shan, J.D. Candidate, UC Berkeley We are seeking a $500,000 seed investment for preclinical CRO research and IP costs.
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According to the National Institute of Health, 23.2 million Americans are trapped in detrimental addictions. Addictions affect not only the addict, but also family, friends, and even strangers. With this behavior costing the U.S. over $600 billion each year, and harming loved ones every day, consider this a nationwide call to action. Crimson Pharma is a drug research and development company dedicated to helping those whose lives are affected by substance abuse, sleep deprivation, and attention disorders. Our first product line Dopatin will target substance abuse, specifically cocaine. Dopamine is a neurotransmitter that stimulates pleasure and is increased tenfold with the use of stimulants. Amy Newman and coworkers from the National Institute on Drug Abuse have filed a provisional patent for Dopatin that acts as a non-addictive dopamine reuptake inhibitor eliminating the withdrawal process. Dopatin is derived from Modafinil, a non-addictive stimulant currently approved to treat sleep and wake disorders. By altering functional groups, the analogues have greater solubility and higher binding affinity to dopamine transporters, which yields smaller doses and greater bioavailability. Current research includes promising pre-clinical assays assessing metabolism, pharmacokinetics, and behavioral studies in rodents with cocaine dependence. Future behavioral studies will concentrate on toxicity and delivery methods. $8.5 million is needed for the five years to get through Phase II clinical trials. Contingent on promising results, the product will be out-licensed to a larger pharmaceutical company to continue manufacturing. Crimson Pharma will then take the revenue and royalties from the licensing to develop analogues for the other indications mentioned previously. Medication-assisted treatment clinics (MAT), hospitals, and the justice system will serve as the market for Dopatin. MAT clinics do not have any FDA approved drugs for the treatment of cocaine addiction, but 25% of opioid treatments occur in MAT’s. Just over 150,000 persons were treated for cocaine dependence in the US in 2010; assuming a similar rate to MAT of opioid dependence plus market growth due to its one-of-a-kind nature, we plan on Dopatin reaching about 50% of those treated for cocaine. Key officers in the company include CEO Megan Torman and CSO Pandora White, accompanied by boards of advisors including entrepreneurial, marketing, engineering, and business expertise.
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Artifix has developed a technology that can vastly improve motion correction in medical imaging. The most common reason for motion ‘blurring’ artifacts in MRI images is also fairly inevitable. Staying motionless in a noisy and confined space for an extended period of time is not an easy task for a healthy individual let alone a patient or a child being scanned. Our system significantly improves the quality of images and can be seamlessly integrated with existing and new MRI systems. Current technologies aimed at tackling the motion artifact problem have certain disadvantages such as increasing scan time, patient discomfort or limited correction efficiency. Our proprietary system circumvents these issues through the use of external cameras and innovative algorithms that track subject movement to enable prospective compensation for the measured motion. This approach has many advantages, the most important of which is the improved quality of health care made possible by our product and the reduced dependence on anesthesia. Every day, motion causes MRI clinics to experience opportunity losses in machine utilization that hurts both the bottom line and quality of care. Artifix aims to not only tap this market opportunity, but also to advance innovation in motion correction technology by augmenting one of the most important diagnostic tools used in healthcare. We have devised a straight-forward strategy to realize the market potential of this product. VC and other financing in the amount of around $700K would go towards accomplishing our milestones of prototyping, developing, and building commercial relationships with large MRI manufacturers. We aim to generate revenue through the licensing of our technology to major manufacturers or ultimately through an acquisition. With a versatile and interdisciplinary team, comprised of a physicist, an experienced MRI engineer and a business student along with mentorship of established researchers and entrepreneurs in biomedical engineering, we are well equipped to face the nuances of developing and commercializing such a product and take on the challenges that come with it on our path to being a leader in prospective motion correction technology.
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Avastin is a monoclonal antibody that nonspecifically inhibits angiogenesis. It currently sells $6.5B annually and is effective for the first- and second-line combination treatment of multiple forms of advanced cancers, including brain tumors. Off-target and systemic adverse effects such as bleeding and impaired wound healing in normal tissues limit its use. The benefits of Avastin can be made better by targeting tumor-specific angiogenesis, which would enhance both efficacy and safety. We propose a novel anti-angiogenic therapy for brain tumors using the L2 monoclonal antibody against TEM8, a highly conserved receptor that is overexpressed on tumor blood vessels of multiple cancers. We envision two distinct clinical applications for L2: 1) Control of advanced disease in the brain by combining it with the established chemotherapeutic regimens, such as in the cases of multiple brain metastasis or malignant gliomas. 2) Prophylactic use in patients with cancer at high risk of developing brain metastases that can prevent upto 1,50,000 new cases of brain tumor every year. NIH-funded research has shown that anti-TEM8 antibody have inhibited tumor-induced angiogenesis, displayed broad anti-tumor activity and augmented the activity of clinically approved anti-cancer agents without added toxicity. Our initial research strategy includes product optimization including in vitro and animal experiments to further determine the efficacy of L2. This will be followed by clinical trials to examine the safety profile and potency of L2 in human patients. The initial phase of the project will require up to 3 years and $5 million of seed money to secure a license and support academia-based research. Our initial funding would come from private investment. Following the success of initial phase, additional funding would be secured from private investors or partners to formally commence the company operations. The attractiveness of the product concept, i.e. “next-generation, targeted Avastin therapy,” coupled with the large number of potential patients who can benefit from such therapy, will ensure attractive investment returns if the drug is successfully commercialized. This will not only capture the market of Avastin but also of other anti-angiogenic drugs. This construct and expectation is based on a number of precedent start-up companies in oncology.
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Opportunity: >In 2010, over 2.5M Americans visited the ER or were hospitalized due to traumatic brain injury (TBI) >More than 100,000 players of contact sports suffer from TBI each year >Lack of immediate medication post-injury leaves the brain susceptible to the damaging effects of secondary inflammation, which may account for high incidence of dementia or other cognitive issues in retired NFL players (~28%) >There is a need for speedy treatment of TBI, especially in contact sports, military, and accidents CranioPore Inc.: >Our solution is a portable method for rapid transcranial delivery of anti-inflammatory and other high molecular weight (MW) drugs directly to the brain to treat TBI, stroke, and tumors >Our Products: -Programmable pump: Releases designated doses of drugs onto intact skull -Adhesive Patch: Seals wound and delivers drugs for brain injury -Dissolving subcutaneous implant: Slow and targeted delivery of anticancer drugs >Lead Investigator: Dr. Dorian McGavern (NINDS) >Patent Status: US App.# 61/599,107, PCT App.# PCT/US13/24741, PCT application filed Feb 5, 2013 Target customers: >Paramedics >Contact sports team physicians >TBI, brain cancer and stroke patients >Department of Defense and Veterans Hospitals >Pharmaceutical companies with appropriate drug candidates Market Competition: >Current treatment methods require passage through the Blood Brain Barrier(BBB): -TBI - oral/injectable drugs, ultrasound permeabilization, or craniotomy -Brain Cancer - oral/injectable drugs or surgery -Stroke - injectable drugs Business Strategy: >License technology to pharmaceutical companies for manufacture and sale Monetization strategy: >Licensing negotiations with drug companies >Royalty per unit sale Benefit for Investors: >High return from large patient base >Low risk from licensing to pharmaceutical companies Potential Funding: >Non-dilutive funding from NIH, DoD, and Cancer research foundations >Industry >NFL, NHL, MLS Exit strategy: >Acquisition by a pharmaceutical company Financial Summary: >Expense Outlook: Research cost by Contract Research Organizations, and Class II Device Human Trials for FDA approval (~20M) >Consumer base in USA : ~3.3 M people/year >Projected Cumulative Revenue after 5 years: $367M >Projected Annual revenue after 5 years : $97M >Growth opportunity: Revenue from licensing technology to pharmaceutical companies with current pipeline of high MW neurological drug
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Patient movement is the single largest contributor to image acquisition time, rescan rate, and image quality degradation in brain MRI. This costs the US healthcare system $6.3B annually, of which $2.8B is carried directly by our customers--hospitals and imaging clinics. What do we offer? Buckeye SmarterImage (BSI) offers a patent-pending motion correction system (MCS) comprising of a camera and software algorithm. The BSI-MCS compensates for patient motion in real time to make what would have been an unusable scan, usable. Moreover, it has been validated on human subjects and eliminates key problems associated with the solutions offered by major MRI vendors (GE’s PROPELLER, Siemen’s BLADE, etc.). Unlike any system in the market, BSI-MCS: • Can be installed on existing machines (10,000+ machines in US alone). • Does not require calibration nor lengthen scan time. • Does not distort the image. Market and Financial Projections: BSI offers a unique opportunity in an underserved market, with a rapid growth and high recurring revenue product. The BSI-MCS saves healthcare facilities $220k per machine per year, while decreasing acquisition time and increasing diagnostic quality. Initial investment of $0.75M will fund 9 months of product development, in collaboration with the inventor. Additional development during preclinical studies ($0.5M), carried out in partnership with the renowned Ohio State University’s Medical Center, will allow BSI to strengthen the IP position as well as fast track FDA approval through substantial equivalence. FDA approval represents a significant value inflection point for the company. Finally, market entry is strengthened through early sales to development collaborators and industry partners, including the Wexner Medical Center, Nationwide Children’s Hospital and Cleveland Clinic. This further allows BSI to leverage their brand in the community, and rapidly scale market presence. The venture is highly profitable due to recurring subscription fees and low cost manufacturing. With a monthly burn rate of $90-120k, break-even can be achieved within the first four months of active sales. A cash balance of $20M is projected when capturing 3% of the market within 3 years of sales. BSI will be an attractive target for acquisition well before this point.
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Overview We are at the forefront of developing targeted therapies, enabling people to function more naturally in society. We do this by producing a highly selective drug, named ML321, which alleviates brain mismanagement of dopamine without producing side effects that disrupt their lives. Dopamine plays a major role in the regulation of diseases such as schizophrenia, but we believe that ML321 may help patients with various other ailments, such as PTSD and autism. We would like ML321 to be one of many highly selective pharmaceuticals used in tandem with one another in doses tailored to patients. Product Our drug is the first to selectively bind to the D2 dopamine receptor, so that dopamine cannot bind to it instead. Because ML321 is so much more selective to D2 than its closest competitor, it does not trigger other side effects in the body. Dr. David Sibley of the National Institutes of Health currently owns this patent. Competitive Landscape Various other treatments help to cure the same ailments or more than one ailment at a time. However, no drug currently available or known to be in the pipeline only target the D2 receptor, long suspected to be the culprit for hallucinations and other symptoms of diseases such as schizophrenia. ML321 is the only drug that can decrease disease symptoms without negative side effects seen in other drugs. Business Strategy The end goal is to develop ML321 as a targeted therapeutic for diseases involving the D2 dopamine receptor, ranging from schizophrenia to PTSD. Revenues will be created by licensing/selling ML321 to a pharmaceutical company, who will, in turn, make money when patients use the drug. Pharmaceutical companies benefit by getting in on the ground floor of the coming wave of tailored medicine. Major Milestones 1. Secure funding from venture capitalists/angel investor 2. Scale up of drug production a. Market as research tool for elucidating dopamine-related disease pathways b. Perform pharmacokinetic studies and perform additional structure modifications if needed 3. Conduct clinical trials 4. Exit Plan a.Sell product to major pharmaceutical company for patient treatment or as a research tool
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Gliomacare Inc., helps clinicians to diagnose brain tumor from patients presenting with minimal symptoms. Using a comprehensive blood based biomarker bio-chip test, the company aims to provide an inexpensive and an easy to integrate method to screen brain tumors at an early stage. By developing an easy-to-integrate, quick and an inexpensive blood based diagnostic tool, Gliomacare inc., aims to capture 50% of the global market potential approximately, $ 5 billion USD. Proceedings have begun to reach out to the North American markets and also to capture the Asia- Pacific (APAC) regions starting from India. Because of the more recent market developments, India is expected to experience the highest growth rate in the forecast period, at a compound annual growth rate (CAGR) of 14.4%. Further plans are also in place to capture other APAC regions which account for an estimated of USD $105.8m by 2020. The company aims to deliver the product by matching the various types of biomarker tests available in the market and by strengthening the research and development (R&D) pipeline globally. Gliomacare Inc., has made collaborations with diagnostic and drug marketing companies to capture market in Canada and India. The competitive landscape analysis indicates that the company might compete with major cancer biomarker tests such as prostate specific antigen (PSA) tests, human epidermal growth factor receptor 2 (HER2) tests and circulating tumor cells (CTC) tests. Gliomacare Inc. is seeking $1.3m USD to support initial investments to manufacture biochips, establish its offices and payroll, and run its sales and marketing campaigns. Pro forma projections estimate a capture of at least 10% of the global glioblastoma diagnostic market. Cash and accounts receivable by the end of year 5 are estimated to $1.5m USD. The company aims to attract venture capitalist firms by online networking, crowdfunding resources and through collaborators. The company has already established contacts with venture capitalists and collaborations with marketing companies in India. Gliomacare Inc., is led by Ravishankar Palanisamy who will be the President and CEO of the company. The core team involved in the development of the diagnostic tool will own the technology. Believing that the company will perform better, Gliomacare Inc aims to adopt “owner buyout” as an exit strategy and plans to strike an agreement with the investors and stockholders.
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ChromriScan: Executive Summary ChromriScan develops and manufactures next-generation MRI contrast agents enabling imaging of multiple biomarkers simultaneously. Our first focus is Alzheimer’s disease, a global aging illness. The current contrast agents can only detect one biomarker and thus causing a high false positive rate. With our product, the diagnosis of Alzheimer’s disease will be more accurate. Problem: Alzheimer’s disease affects over 44 million people worldwide, causing $600 billion in economic loss each year. The complexity of the disease presents a crucial obstacle in clinical diagnosis. The imaging agents for Alzheimer’s on the market or in development are PET contrast agents, which can only detect one biomarker, beta-amyloid. Furthermore, PET is expensive and not widely available. These disadvantages limit their use and diagnostic accuracy. Product/Solution: Our MRI contrast agent is based on an invention by NIH. It is the first and only agent that can image multiple biomarkers simultaneously, thereby increasing our diagnostic accuracy in Alzheimer’s disease. In addition, each MRI scan costs approximately ½ the price of a PET scan. Market: The Alzheimer’s biomarker market is $2.9 billion with 20% CAGR. The incidence of Alzheimer’s disease is increasing with our aging population, making this a fast growing market. Business Strategy / Model: Like most other imaging agents, our revenue will mainly come from diagnostic usage through healthcare reimbursement. For example, Amyvid, an Alzheimer’s contrast agent, is sold for $1600 per scan. We will also have research-use-only sales directly from our company to research labs. R&D Path and Major Milestones: Month 1-12: complete product precursor development Month 12-18: complete product prototype development, animal testing and start marketing for research uses Month 19-42: Initial human testing, product optimization Month 43-90: Clinical trial Use of Initial Funds: Term: Budget Explanation Consultant payment: $360,000 Providing R & D advice, 50h/month, $300-350/h Technician payment: $240,000 Assisting R & D, 2 people, 160h/month each, $30-40/h Lab space: $300,000 Space and equipments, $14000-$16000/month Experimental materials: $100,000 Alzheimer's disease mouse models, lab consumables Delivery: 18 months Total Spending: $1,000,000
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Cortexius Corp. is developing safer, effective CNS therapeutics needed to improve treatments for neurological disorders and provide better, more active lives for our patients Business Summary Cortexius Corp. is a research and development pharmaceutical company addressing the need for improved, non-stimulate treatments to manage chronic neurological disorders. Product Cortexius’s pipeline consists of a novel analogue of modafinil (Somnusin), which demonstrates lower cytotoxicity than generic modafinil, and a higher affinity for inhibiting the reputake action of neuronal plasma membrane transporters, namely dopamine, serotonin and norepinephrine. These features translate into elevated monoamine concentrations through lower dosing and side effect risks, such as toxic epidermal necrolysis (TEN), Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS) syndrome, and Stevens-Johnson syndrome (SJS). Go-to-Market Strategy Cortexius will target indications where there is a need for safer, effective, non-stimulant therapeutics to manage chronic neurological disorders. In adult Attention-Deficit Hyperactivity Disorder (ADHD) there is a growing demand for non-stimulant, reduced dosing treatments due to potential substance abuse and long-term treatment duration. These concerns are also expressed in other indications such as ADD, excessive day time sleepiness (EDS), substance abuse and potentially weight-loss. Please see Business Plan for Market Entry strategy. Advantages - Improved analogue of marketed drug modafinil (Provigil) - Efficacy – Higher affinity to inhibit monoamine reuptake transporters - Safety/AE – Lower dosing and cytotoxicity - PK – Greater water solubility Market Analysis Worldwide, the ADHD therapeutic market is projected to grow 5.3% annually from $6.9B in 2013 to $9.9B by 2020; key drivers include broadening diagnostic criteria and increased adult treatment. EDS is expected to grow by 0.6% to $821M by 2019, and substance abuse by 5.3% to $12.7B by 2018. (GBI Research and GlobalData) Future Milestones - Patent – secure exclusive licensing rights - Funding – Grants, angels and/or strategic partners for in-vivo studies)
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At Vigilance Diagnostics, we believe the best treatment decisions are those informed by an accurate assessment of therapeutic benefits and risks. While several immunomodulating therapeutics are effective in managing chronic auto-immune disorders, such as Multiple Sclerosis (MS), in some instances, patients have contracted the often fatal condition Progressive Multifocal Leukoencephalopathy (PML). Fear of developing PML often leads to discontinued treatment and consequently, diminished quality of life. Vigilance Diagnostics’ technology refines PML risk profiles, improving patient stratification. Exposure to JCV, a virus carried by a majority of the population, is the predominant risk factor in developing PML, for those using immunomodulatory treatments. Presently, ELISA based diagnostics detect the presence of JCV antibodies. Unlike our proprietary qPCR probes, however, ELISAs cannot distinguish between the latent and pathogenic states of the virus. Furthermore, up to 30% of MS patients receive false negative results, causing heightened risk. Our technology fills a void in the market by augmenting current methods for risk stratification. Our diagnostic relies on CLIA-approved technology platforms that are amenable to mass production with low production costs. We are presently approaching private investors, the PML consortium (Biogen, Pfizer, and Roche) and non-profit patient advocacy groups, such as MS Society of Canada, for funding. Our timeline and estimated initial funding requirements are as follows: • Phase I (1 year)/$250K: Retroactive analysis, utilizing thousands of MS patient serum and plasma samples banked during the course of treatment with Tysabri, a therapeutic associated with risk of PML. • Phase 2 (2 years)/$2M: Collaboration with Biogen Idec to demonstrate the clinical efficacy of the diagnostic for patients taking Tysabri. • Phase 3 (Post Year 3): Licensing to pharmaceutical companies developing therapies that carry a risk of PML. Our diagnostic could increase the number of patients on Tysabri by 5%, creating $50M in additional revenues for Biogen Idec. We would seek to negotiate licenses with a target of $10M upfront, plus 2% royalty/test. We would also pursue similar emerging therapies that carry a risk for PML, broadening our income base prior to being acquired or going public after Year 4. Importantly, investors’ ROI will be 20-fold their initial investment. At Vigilance Diagnostics, we help make the decisions that matter.
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Protegem is a development-stage biopharmaceutical company pursuing a pipeline of peptide-based therapeutics with an initial focus on Alzheimer’s disease (AD). AD is an incurable disorder, affecting more than 5 million Americans with healthcare costs of over $200 billion annually. Product/Technology Encouraged by earlier favorable results, Protegem plans to commercialize the peptide, TFP5, for the treatment of AD. Competitive Analysis •Protegem is engaged in an intensely competitive field characterized by extensive research efforts and rapid technology development. •At best, available therapies treat only cognitive symptoms and cannot slow, stop, or reverse AD-associated tissue damage. •Protegem believes its proprietary technology, strategic intracellular approach, and exceptional scientific/business management team will ensure a sustainable, competitive advantage to address the underlying causes of AD. Business Strategy/Model •Protegem is seeking $5 million in equity financing to complete IND enabling and Phase-I studies. •An additional $15 million in funding in 2017 will advance products through Phase-II trials. •Concurrently, Protegem will seek various grants, including SBIR and STTR. •Securing strategic alliances/joint venture with a major pharmaceutical company by 2017 will: -Providing financial/strategic resources, including legal, regulatory, manufacturing, and marketing expertise. -Milestone/royalty revenue stream. •Recent comparable deals include the AC Immune deal in 2012 (valued at $418 million) and the iPierian acquisition in 2014 (valued at $725 million) suggesting that Protegem’s valuation within the next 3 – 5 years, will likely be upward of $500 million or higher. •Potential first product launch by 2022. •Possible exit scenarios include, IPO, M&A, JV and/or strategic alliance. R&D Path and Major Milestones •Complete IND enabling studies in next 18 months: -ADME/tox -Peptide/protein binding -Peptide stability/degradation in body -Dosing/administration route •Submit IND application Q3-2016 •Initiate Phase-I trials Q4-2016 •Start Phase-II trials Q4-2017 •Seek fast track approval for lead compound •Begin Phase-III trials 2020-2021 Management Protegem is led by strong biopharmaceutical veterans with expertise in startup and biopharmaceutical product/business development. The business team is complemented by world-class scientists from major academic institutions with expertise in molecular biology, neuroscience, and drug development.
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Every 15 seconds, an American suffers a traumatic brain injury (TBI), hospitalizing 230,000 Americans annually at a cost of more than $50 billion. Patients are typically classified as mild, moderate or severe depending on the extent of injury. Because of a lack of specific therapies for TBI, patients hospitalized with moderate injuries receive care consisting mainly of passive monitoring rather than active treatment. Patients with injuries classified as mild are currently not admitted for prolonged hospitalization, however, current estimates suggest that upwards of 50% of these patients have cognitive and functional impairments lasting for months to years following injury. Beyond Barriers Therapeutics’ aim is to change this. Our company is developing a new treatment for TBI that would actively and directly help reduce injury to neural tissue following injury. Our product would decrease cost of care by speeding recovery after injury and diminishing risk of long-term disability. In the hours and days following initial injury, toxic molecules and dangerous inflammation can occur in the brain, contributing to long-term disabilities. By using a novel delivery method during this treatment window, our product would directly slow or prevent this harmful process from further damaging neural tissue. Our solution of rapid and targeted administration of antioxidant therapeutics to the brain would result in drastically reduced long-term patient injury and substantial cost savings to the healthcare system, potentially more than $1 billion due to hospital stays alone. We have a unique and compelling roadmap for successes. Our business strategy emphasizes rapid preclinical and clinical development, leveraging regulatory pathways and our team’s relationships with Northwestern University to generate value quickly and efficiently. Our team has the technical and medical background to oversee product development and research at all stages. We, along with our advisory board, also have the strategic, financial, and legal expertise to successfully bring our product to market. In addition to significantly improving standard of care for patients suffering from TBI, our proprietary technology will lay the foundation for use of a novel, more effective delivery mechanism of small molecules to the brain. After successful testing, this technology can be used to treat a number of other neurological conditions--generating value throughout the healthcare system.
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Traumatic brain injury (TBI) is a $76 billion dollar problem. Each year there are over 50,000 deaths and 1.8M patients diagnosed with TBI in the US alone. ShiraDevices offers a solution to this problem through our revolutionary Brain Balm delivery system (patent pending). Brain Balm is an easy, preloaded disposable injection of a hydrogel formulated anti-inflammatory, applied subcutaneously onto the cranial surface. Transcranial diffusion of the anti-inflammatory agent occurs from the hydrogel depot into the cerebrospinal fluid reducing cell death and effectively treating TBI. Our unique product bypasses the blood brain barrier – a structural blockade for many pharmacological products. Unlike our competitors, all early stage biotechnology companies, we have a direct therapy to meet current market needs. Given the current wait and see approach used by medical personnel, our innovative product will be easily adopted in emergency rooms and EMS vehicles. With the disposable nature of Brain Balm, we forecast these customers purchasing large quantities to combat the increasing prevalence of TBI. Furthermore, the reduced patient recovery time is an incentive for insurance reimbursement to our customers. Current preclinical data are based on rodent and swine models, which will be expanded to include cadaver studies. To launch Brain Balm, we will conduct clinical trials for regulatory approval, funded by SBIR awards and venture capital funding. As a kit, Brain Balm will include disposable FDA approved syringes preloaded with approved anti-inflammatory agents, effectively reducing R&D, manufacturing costs, and regulatory hurdles. We are prepared to grow as a company or leverage our value to a large medical device partner. ShiraDevices is founded by entrepreneurial researchers and our management team has R&D experience in neurological discovery, bioengineering and biomedical device development. Together, we have over 20 years of startup experience including securing venture capital funds of over $6.5M and profitable exits of four biotech companies. A compromised brain may lead to long-term mental defects including cognitive and emotional disorders, a growing problem for the military and professional sports. Our platform technology will allow for increased valuation when we expand to treat other neurological conditions, such as Parkinson’s disease, and incorporate other anti-inflammatory agents.
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Attention-deficit/hyperactivity disorder (ADHD) is a neurobehavioral disorder that affects 6.4M children between 4-17 years of age. Approximately 3.55M of these children are currently receiving medication to treat the symptoms of ADHD contributing to the $6.9B global market in 2013. Historically, the most common medications prescribed for the treatment of ADHD are psychostimulants, such as amphetamine salts (Adderall) and methylphenidate (Ritalin). These medications are effective in treating ADHD, but have come under scrutiny recently because of their high abuse potential and side effects such as anxiety, headache, and loss of appetite. Modafinil, which has alertness-promoting properties most recently used for treatment of narcolepsy, has been proposed as an alternative ADHD treatment. Modafinil has a reduced potential for abuse compared to current psychostimulants, and our novel formulation lowers side effects through anticipated lower effective dosage than traditional modafinil. Our modafinil formulation has the potential to become the industry standard for the safe and effective treatment of one of the most prevalent mental disorders, ADHD. Our team will manage the development of the novel drug from discovery to commercial success. Through collaboration with Dr. Amy Newman and the New Orleans BioInnovation Center, we will oversee preclinical testing for IND filing. Our strong team of industry advisors (PPD, inVentiv Health Clinical, CNS Vital Signs) will facilitate trial design and effective outsourcing to minimize cost and time required to get to market. Initial analysis indicates $37M overall investment over 4 years to execute our business plan. Initial investment of $2M will deliver preclinical work, followed by $10M for phase 1 and $25M for phase 2. Iterative milestones, including data on toxicity, pharmacokinetics, and clinical phases, will allow us to leverage financial resources and de-risk our progress. Our exit strategy will be to market this medication to companies active in ADHD and modafinil-based treatments (Teva, Shire, Eli Lilly) after Phase II clinical trials. Our exit value is anticipated to be $250M yielding a 6.75X multiple. Our team has the knowledge to drive successful product development of this novel formulation, potentially revolutionizing treatment for millions of children and adults with ADHD.
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About Orphaceuticals Orphaceuticals Ltd. is an anti-cancer company specializing in Neurofibromatosis Type 1 (NF-1) therapeutics. The company is developing an orphan drug to treat Malignant Peripheral Nerve Sheath Tumors (MPNSTs) caused by NF-1 utilizing the compound Schweinfurthin A (SA). The management of the project boasts a mix of talents with expertise in both science and business, supported by a scientific advisory board from Queen Mary Hospital and partners from Ventac Partner, a life science consulting firm. The Disease (MPNST) and SA Approximately 20,000 patients suffer from MPNSTs due to NF-1 disorder in the US and EU. Current prognosis with surgical removal and adjuvant radiotherapy is destitute with a 5-year survival rate of 35%. The development of SA would meet this medical need. SA’s action is novel and specific within the industry in that it could inhibit the growth factor-induced Rho signal to disrupt the F-actin cytoskeleton of NF-1 deficient tumor cells. The preclinical product is at lead optimization, which is estimated to be commercialized in approximately 8.5 years. A detailed timeline is displayed in Exhibit 1. Business Model and Commercialization Currently, the project is funded by the Children’s Tumor Foundation. It is also eligible to be financed by other government and private funds expediting orphan drug and NF-1 research development. Once it proceeds into the clinical trial stage, venture capital would take a substantial amount of financing. The company aims to exit through acquisition by a pharmaceutical company at Clinical Trial Phase III. As there is no direct competition, the product will be able to acquire market dominance and potent pricing structure. The orphan drug status grants market exclusivity for 7 and 10 years in the US and EU respectively, with 50% tax credit on R&D cost and FDA fees waiver which reduce development cost and allow for a longer cost recovery period. Key figures of financial projection are illustrated in Exhibit 2. Continuing Development Possible developments include Blood Brain Barrier penetration for Central Nervous System tumors and prophylaxis against Plexiform Neurofibroma. These potential areas are all research gaps related to NF-1 and could substantially enlarge the target market.
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Innovative Vascular Therapeutics (IVT) is a company based in New Orleans working towards developing the first line of novel stroke therapeutics. Stroke is the 4th leading cause of death in the United States, claiming over 130,000 lives annually. For the majority of stroke victims, this event imposes a $200,000 cost over the course of their lives, and places an economic burden of $36.5 billion through medical costs and lost productivity every year. Currently, the only FDA approved treatment of ischemic strokes is tissue plasminogen activator (tPa) which works by dissolving the clot and improving blood flow to the brain. However, tPa targets only the initial vascular blockage and does not mitigate stroke-associated brain damage. Much of this damage is caused by the inflammatory response mediated by RAGE, or the Receptor for Advanced Glycation End products. IVT has developed a novel soluble form of this protein, N-glycoform sRAGE, to act as a decoy and competitively bind to the inflammatory ligands that would otherwise activate RAGE. This mechanism has been shown in preclinical studies to significantly reduce the area of damage in the brain following stroke. Our inventors have been awarded a full US patent for this formulation, and we foresee this therapy as a reimbursable treatment through Medicare. Our path to commercialization begins with pre-clinical validation of our therapy funded through small business and research grants, and supported through a 40% tax credit in research expenditures exclusive to Louisiana. As this therapy is eligible for the FDA’s Fast Track program, we estimate an investment of $5 million will enable accelerated progress through clinical trials to our end goal of partnership with a major pharmaceutical company once we enter Phase II. Our team is motivated and backed by advisors and mentors with decades of expertise in entrepreneurial management, raising capital, and conducting research. Our collaborations span from the New Orleans BioInnovation Center to Tulane’s Stroke Program, the only Gold Plus Stroke Center in the region. Together, IVT has the opportunity and the advantages to make unmatched progress in improving stroke patient outcomes.
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COMPANY OVERVIEW NOLA Therapeutics develops cancer drug treatments using nanotechnology and antibody therapy. CORE TEAM Technical Michael Strong, MD/PhD candidate James Morgan Dixon, MS Biomedical Engineering, MBA candidate Gary Mouradian, PhD, Postdoctoral fellow Juan Carlos Vera, MD, MS candidate in Clinical Research Business Development Yingchun Ba, MBA candidate Arpit Bhopalkar, MBA candidate Ryan Hildebrand, MBA candidate Christine Soliva, MBA PROBLEM Glioblastoma multiforme (GBM), although rare compared to other cancers, is the most common malignant brain tumor. Temozolomide is the standard of care for treatment of GBM in conjunction with surgery and radiation. Unfortunately, 60% of GBM patients treated with Temozolomide experience tumor regrowth within one year of treatment. Since Temozolomide is a non-specific chemotherapeutic agent, there are side effects including immunosuppression and seizures. SOLUTION Scientists at the NIH discovered a key protein called Anti-Tumor Necrosis Factor Induced Apoptosis (ATIA) that protects and promotes the spread of GBM cells. Our product, N1, selectively targets and inhibits ATIA. The drug is a biologically enhanced antibody-targeted radiosensitizing chemotherapeutic that links an RNA-based inhibitor, surrounded by a biodegradable nanoparticle polymer, to a monoclonal antibody specific for ATIA. MARKET The GBM treatment market is forecasted to grow from $301 M in 2013 to $623 M in 2020 with a CAGR of 10.9%. There are 15,000 GBM new cases diagnosed annually in the US. GO-TO-MARKET STRATEGY We will validate the technology through pre-clinical and Phase 1 clinical trials, and seek orphan drug designation. To attract strategic partners, we will publish in peer-reviewed journals and attend oncology conferences. COMPETITIVE ADVANTAGE Compared to other anti-cancer agents that kill both healthy and GBM cells, N1 specifically targets GBM, resulting in fewer side effects, shorter treatment time, and fewer recurrences. INTELLECTUAL PROPERTY In addition to the US, the company will pursue patents in UK, France, Germany, Italy, and Spain, where the incidence of GBM is high. FINANCIALS We need $3M to fund the 5-year pre-clinical and Phase 1 clinical trials that will produce validation data, making the company an attractive acquisition target for pharmaceutical companies.
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Please click into this link for easy-read access: https://claremontmckenna.box.com/s/o016jk2npxv7vxp71gj1 BDCure is a startup pharmaceutical company specializing in developing the drug NtBuHA that can potentially cure the fatal Infantile Neuronal Ceriod Lipofuscinosis (INCL) disease. The target customers are all INCL affected orphans and their affiliates. The product can functionally mimic a key enzyme that is missing in INCL patients, which can potentially preserve the motor abilities and extend the life span of INCL patients. The ownership of the patent and the major inventor of this therapeutic is Dr. Mukherjee. This drug has a promising development because it is potentially the only candidate that can adequately deal with INCL at present. This drug enjoys a promising future after it passes the clinical trial stages, approves by FDA and puts into production. BDCure is exploiting this opportunity through the sales of NtBuHA to generate profit. Revenue is projected at around $150,000 per patient per year and it requires patients’ life-long spending. The R&D path includes both the pre-clinical development stage as well as the three-phase clinical trials. The succeeded large scale mice test emblems the completion of the pre-clinical development stage. Managing members of the pharma startup are ensured a high degree of skills and management experiences pertaining to the drug development and management. The total initial investment required should be around $5000 million, among which the marketing and other general expenses are forecast to consist of 36% of the allocated funding. The manufacturing cost and the purchase of long-term asset will be approximately 19% and 12% respectively of the initial funding during the discounted payback period. The R&D stages funding will mainly come from the government and NGOs whereas venture capital funding will mostly support the marketing and production cost after FDA approval. The discounted payback period for venture capital is approximately 9 years, counting from when the drug is put into production. The burn rate during R&D stage is around $26M. However, with a promising future of consistent revenue from patients, the invention give a positive and strong forecasted financial position. School of Accountancy, Level 10, Cheng Yu Tung Building, 12 Chak Cheung Street, Shatin, Hong Kong +852 9662 9000 leungsheklun@gmail.com
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Cogentis Therapeutics is developing a breakthrough therapeutic for Alzheimer’s disease, a severe neurodegenerative condition that involves progressive loss of memory and other brain functions, eventually resulting in death. The disease affects an estimated 30-40 million people globally, a figure that will double by 2030, and is the 6th leading cause of death in the US. Alzheimer’s exerts a major psychological and financial toll on patients and their families and caregivers. Existing treatments for Alzheimer’s have limited efficacy and focus primarily on relieving symptoms rather than slowing the underlying disease. There is a major demand for new treatments, but, despite significant efforts, few drug candidates have shown promising results. Cogentis’ unique solution is to target a key brain enzyme called CDK5 that is hyperactivated in Alzheimer’s brains and plays a key role in disease progression. Our drug, TFP5, inhibits abnormal CDK5 activity and is one of the only candidates that targets all the major hallmarks of the disease: beta amyloid plaques, neurofibrillary tangles, and cell death. In a well-accepted disease model, the drug reversed memory loss as well as other brain deficits and significantly extended lifespan, with no detectable toxic side effects. These dramatic results have generated a great deal of interest in this novel Alzheimer’s treatment approach. The global market for Alzheimer’s therapeutics is predicted to reach $15 billion by 2020 due, in part, to population aging. Analysts project that an effective Alzheimer’s drug would have peaks sales well in excess of $1 billion. Cogentis plans to test TFP5 for efficacy in other neurodegenerative diseases, including Parkinson’s disease and ALS, which would enhance the drug’s market. Cogentis intends to close a Series A financing of $2 million in the near future, which will be used to complete preclinical studies and file an IND to initiate clinical testing of TPF5. Our goal is to expedite development and marketing of the drug so that it can become available to Alzheimer’s patients as soon as possible. We anticipate partnering with a large pharmaceutical company to help fund and accelerate late-stage clinical trials and commercialization of this revolutionary treatment.
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Great Lakes Neuroscience (GLN) is a privately held company developing therapies for neurodegenerative diseases. GLN has a strong, well-qualified team with experience in research, product development, clinical trials, and current good manufacturing practices. Our Scientific Advisory Board is composed of experts from companies including AbbVie, Astellas, Retrophin, Quintiles, IDEA Pharma, Navigant Life Sciences, Fidelity Biosciences, Silicon Valley Bank, and Heartland Angels. Using an exclusively licensed patent, GLN is currently developing its first product, TPfive, a novel drug for treating multiple sclerosis (MS). MS is a neurodegenerative autoimmune disorder affecting over 2.5 million people worldwide. There are no FDA-approved drugs to treat progressive MS; those drugs indicated for relapsing MS have only modest clinical efficacy and many have serious side effects. TPfive has the potential to treat all forms of MS, including progressive. TPfive works to re-establish normal levels of cyclin-dependent kinase 5 (Cdk5). In the mature brain, dysregulated and elevated Cdk5 activity is associated with neurodegeneration, loss of microglia and oligodendrocytes, neurofibrillary tangle formation, and tau-opathy. Research done by (among others) Dr. Siddharthan Chandran’s group of the University of Cambridge on the pathology of MS would suggest Cdk5 to be a novel target in the treatment of MS. TPfive is a highly targeted peptide conjugated to a transactivator of transcription protein transduction domain, allowing it to cross the blood-brain barrier where it inhibits a pathological Cdk5 activator, p25. In multiple mouse models, TPfive has been shown to lower Cdk5 levels and to ameliorate the adverse effects of prior elevated Cdk5 without off-target effects or toxicities up to 200 mg/kg. Using collaborations with Chicago-area research institutions, GLN aims to demonstrate the efficacy of TPfive in animal models of MS. GLN intends to partner with a larger pharmaceutical company as early as Phase 1 clinical trials. GLN is now seeking seed investment of $1M to support its preclinical work which will be supplemented by SBIR grants and/or angel investing. Based on the validations from preclinical trials, GLN aims to collaborate with companies that crowd-source expertise and the Patient Centered Outcomes Research Institute in order to develop patient-focused clinical trials.
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TAQ Technologies (TT) is a biotech company working to commercialize a targeted, affordable, quick diagnostic test to detect the John Cunningham virus (JCV). While latent in nearly 70% of the US population, in severely immunocompromised patients, JCV can become active and cause the lethal neurological disorder progressive multifocal leukoencephalopathy (PML) . Conditions such as AIDS and cancer and medications called monoclonal antibody (mAb) therapies, used for immunosuppression in organ transplant patients and in treating multiple sclerosis, psoriasis, and rheumatoid arthritis, often promote JCV’s active form. A simple, accurate test is urgently needed to determine and regularly monitor JCV status in at-risk patients. The available test, STRATIFY JCV Antibody ELISA, infers whether a patient is JCV positive from the presence of antibodies in plasma; a definitive diagnosis for PML cannot be made without an MRI scan. In contrast, the CLIA-approved TT JCV Diagnostic Test, which uses a technique called qrtPCR to distinguish a variation in the JCV genome upon its activation, can detect, quantify, and distinguish between the latent and active forms. TT has filed for articles of incorporation and plans to establish a full-service laboratory in Winston-Salem, NC, to perform it on patient samples.TT has already secured $5,500 in non-dilutive funding and seeks to partner with Biogen, which manufactures a common mAb therapy, TYSABRI. The number and diversity of patients who will benefit from this test indicates an incredible opportunity to succeed financially, to advance modern healthcare and save individual lives.
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Temporada Pharmaceuticals is committed to finding breakthrough alternatives for the early diagnosis and treatment of Alzheimer’s disease through the development of TFP5 (Invention 2). With a unique blend of dedication, skill, and experience Temporada stands poised to give hope to the millions of patients, families, and caregivers affected by Alzheimer’s. TFP5 was shown to block aberrant Cyclin-Dependent Kinase-5 (CDK-5) activity in animal models while maintaining normal CDK-5 levels. While other CDK inhibitors exist, the TFP5 peptide specifically attacks aberrant CDK-5 activity which has been implicated in numerous disorders, from neurodegenerative disease to cancer. As such, the development of this drug could be a platform for the treatment of a host of diseases. Current Alzheimer’s treatments address symptoms, but do not slow its progression. Other companies are doing research, but current clinical trials target different mechanisms. Additionally, the field lacks an early diagnostic tool. TFP5 stands alone in slowing the progression of Alzheimer’s. As such, there is great market potential for Alzheimer’s therapeutics and early diagnostics (Figure-1). Temporada’s strategy (Figure-2) is to pursue a two-pronged approach toward developing an Alzheimer’s therapeutic concurrently with an early-stage diagnostic using radio-labelled TFP5 for PET imaging. We plan to conduct an efficacy indicating Phase I clinical trial, using healthy volunteers and mild-to-moderate Alzheimer’s patients. Temporada’s Team (Figure-3) has extensive experience in overcoming the challenges that face start-up pharma companies. Temporada’s collaborators have a proven track-record in drug development. Temporada’s advisory board has extensive international-level Alzheimer’s expertise. All this, combined with TFP5’s known ability to cross the blood brain barrier, shows great promise for TFP5. Temporada’s development plan provides immense value for patients, caregivers and investors. While the start-up costs are high (Figures 4 and 5), comparative studies of similarly positioned companies revealed a 10x ROI. The company believes that successfully meeting its milestones will create an attractive exit opportunity for investors via partnership or acquisition in Q4Y3. At that point, TFP5 research could expand to other diseases (Figure-2). Ultimately, Temporada and TFP5 promise to give those affected by Alzheimer’s more hope, more life, and more time.
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Market ● About 20% of all MRI examinations are subject to patient movement, costing over $1B/year in rescans across 11,000 MRI machines in North America. Company Objectives ● MotionCorrect makes MRI scans simpler and shorter for patients by using a visual tracking system to compensate for movement in real-time. ● Our platform increases MRI efficiency and effectiveness by allowing the incorporation of cutting-edge scanning sequences. Value Proposition ● By increasing image quality, augmenting diagnostic accuracy and reducing re-scans, we improve patient care and throughput. ● We offer a comprehensive, NIH-patented real-time solution, combining off-the-shelf cameras with tracking and scanning optimization software that interfaces with OEM’s MRI platforms. Competitive Landscape ● The only direct competitor is at the pre-FDA approval startup stage and provides a single-camera solution with reduced accuracy but simpler setup. Business Model ● We are mostly a low cost, highly scalable software company. ● Our service agreement provides low entry cost and peace of mind. ● A capital expense model will also be available. ● Through future add-ons, we will expand our value proposition beyond motion correction. Roadmap ● 2015, 2016: polish the product, establish alpha sites and begin clinical trials ● 2017: submit to FDA and expand beta sites ● 2018 and 2019: obtain FDA approval and launch ● 2025: reach 20% market share Operations ● Montreal is uniquely suited with its world renowned research centers in neuroimaging. Our R&D team has ties with the Montreal Neurological Institute, Douglas Institute, and l’Unité de Neuroimagerie Fonctionnelle. ● We will collaborate with clinics specialized in pediatric, geriatric care, and sports medicine, such as Montreal Children’s Hospital, Ste-Justine, Concordia PERFORM Centre. ● We will work with MRI OEMs to ensure compatibility and ease of integration. Management ● Current team composition: ○ MRI R&D ○ Medicine and clinical care ○ Real-time robotic vision ○ Licensing and IP ○ Forensic accounting ○ Contract negotiation ● Planned expertise: ○ Three full-time developers ○ One imaging specialist ○ One clinical research coordinator ○ FDA approval consulting services Financial Summary ● We are raising $2M to develop and clinically validate our prototype for FDA submission by 2017. ● Half of the funding will go towards product R&D; the other half will go towards clinical trials and fulfilling FDA application requirements
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Executive Summary: NeuroExP is a startup with its founding members at the University of Pennsylvania in Philadelphia. The company is based on the intellectual property owned by the US Dept. of Health and Human Services, surrounding its Blast-induced Neurotrauma (BINT) simulator. The device allows for study of cellular response in mild traumatic brain injury (TBI) due to supersonic pressure waves caused by explosive blasts. Since 2001, 150,000 US military personnel have been diagnosed with mild blast-induced TBI (bTBI). Due to the many difficulties associated with diagnosing TBI, it often goes un- or misdiagnosed. There is also a lacking body of knowledge on a real-time cellular response, which has resulted in poor healthcare, a paucity of pharmaceuticals or therapies for treatment, and a need for research to find solutions for preventing such injuries. Although the device name suggests Neurotrauma, this technology can also be utilized for testing the response of other tissue types to damage. Research platforms, pharmaceuticals, and biomaterials are the three market segments that NeuroExP will target with the BINT simulator. An industry standard device will be manufactured in Philadelphia with University of Pennsylvania affiliated machine shops. The prototype will be tested with in vitro cell culture studies and as a high-throughput screening (HTS) platform for drug candidates. The device will be first marketed as a tool for Neurotrauma research to laboratories in academia and industry. The HTS capability of this device will be used for discovering small molecules that could treat the abnormal cellular response observed in bTBI. By comparing this platform with the currently available imaging and HTS platforms on the market, we project the device to cost over 100,000 dollars. Additionally, NeuroExP intends to develop a next-generation platform for the bioengineering industry, in order to develop novel biomaterials that could be used to prevent these injuries. The population who will benefit from such a device on the market will be patients diagnosed with bTBI, and especially those who haven’t yet been diagnosed or have been misdiagnosed. It will also encourage innovation to prevent such disorders. NeuroExP has a first-movers advantage to satisfy the tremendous need to study Neurotrauma as it happens.
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ImmunoMark is developing antibody-drug conjugates (ADC) for the treatment of brain cancer. Problem Despite advances in conventional therapies, brain cancer prognosis is still very poor. Of the 70,000 people diagnosed with brain tumors annually, only 13% reach 10-year survival. Surgery and radiation are invasive and cause off-target damage to healthy brain tissue. Additionally, chemotherapeutic drugs are not effective because they cannot cross the blood brain barrier (BBB). Thus, there is a high unmet need for minimally-invasive, targeted therapeutics that are not required to cross the BBB to kill brain tumors effectively. Solution Our lead product is an antibody-drug conjugate (ADC) that targets the blood vessels that feed the tumor rather than cancer cells. This eliminates the need to cross the BBB and reduces off-target neurological side effects. Competition Current competitors targeting tumor vasculature only slow down growth of new blood vessels without destroying existing ones and do not significantly improve patient survivability. Business Strategy We will take our lead compound from early discovery through Phase I clinical trials. To optimize capital utilization, we will outsource manufacturing and conduct clinical trials through a contract research organization. In parallel, we will maximize patent potential by developing ADCs targeting pathological vasculature for liver, lung, colorectal, and breast tumors. We intend to fund these non-core projects through SBIR grants and divest them early on to offset the costs of our initial focus, brain cancer therapies. Post-Phase I Exit Strategies: • Divesting lead asset • Strategic partnerships to continue lead compound development for market entry • Acquisition by established oncology-focused companies such as Genentech or Novartis Market Penetration Market analysis indicates billion dollar revenue potential for the next innovative therapeutic for brain cancer. We will capitalize on this opportunity by applying for orphan drug indication for adults suffering from Glioblastoma Multiforme, the most common and aggressive type of brain cancer. Our proposed strategic partnerships will provide resources and distribution channels to successfully bring our biologic to market with minimal investment risk. To maximize revenue we will seek approval for additional brain cancer indications. Ask $7.750MM, milestone-based Contact Us Email: info@immunomark.com Website: www.immunomark.com
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A median survival rate of 14.6 months underscores the desperate need for effective treatments of the brain tumor glioblastoma multiforme (GBM), as well as other deadly cancers. Such an advance is possible using the work of Drs. Zabow and Koretsky, whose multimodal MRI contrast agent based on magnetic microstructures enables differential tracking of GBM-specific CAR T-cells. This enhancement over fluorine and PET MRI may lead to reduced need for specialized equipment and remove the rapid signal decay that hinders personalized design of CAR T immunotherapies. Thus, our mission is to establish the clinical viability of this novel contrast agent for CAR T-cell tracking and to pursue its commercial development. Proof of the viability of this technology in mice and then in GBM patients will depend critically on experts who have expressed their intent to collaborate with this venture. These include Dr. Allan Johnson, PhD, Director of Duke’s Center for In Vivo Microscopy and Dr. John Sampson, MD, PhD, Chair of Neurosurgery at Duke and a leader in GBM treatment with CAR therapy. They will augment the existing management and advisory team that includes Dr. Nandan Lad, MD, PhD, Matthew Megaro, Duke Fuqua School of Business Executive in Residence and Dr. Jesko von Windheim, PhD, MBA; serial entrepreneurs with 25+ years of life sciences experience and deep connections to the venture community. Early work through proof of viability in animal models should provide the insight needed to demonstrate clinical and commercial potential to CAR therapy companies, who we will partner with for clinical studies. Several large entities have established franchises in the CAR therapy field and would greatly value a durable and specific contrast agent for its ability to synergistically improve their existing immunotherapies. Moreover, as similar nanoparticles are already well-established contrast agents, and based on the significant advances provided by this agent, we expect few obstacles in obtaining regulatory approval via an imaging drug application. This strategy will enable a rapid path to market and allow us to expand to additional cancers, resulting in a projected $6B market cap with a $1M/month burn rate.
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Glioblastoma is the most common and lethal cancer of the central nervous system, affecting hundreds of thousands of patients each year. Current diagnostic tools for glioblastoma require potentially dangerous biopsies and cost-intensive imaging. Existing therapeutic approaches are limited, with 95% of patients dying within five years. Our company, VasoMark, is developing platform technologies for the diagnosis and treatment of glioblastoma and other brain tumors, providing novel and essential tools to an underserved clinical market. Our approach utilizes tumor-associated blood vessels as both diagnostic markers and eventual therapeutic targets. VasoMark’s immediate objective is to conduct research and development to validate our technology and design a non-invasive diagnostic blood test. For improved therapeutic prediction our initial product will be two-tiered, detecting the patented biomarker ATIA in combination with tumor-specific vasculature markers. During development we will confer with clinicians for real-time voice-of-customer analysis and compliance experts to determine the proper regulatory pathway for our product. Beyond an initial market opportunity in glioblastoma, VasoMark plans to expand into companion diagnostics and develop applications for broader markets, including stroke and other cancers. With this approach, VasoMark takes an important step toward minimally invasive and personalized cancer care. VasoMark is comprised of academic researchers from the University of Rochester working to bring a higher quality of care to the medical field, while maintaining rigorous standards for scientific research. Our interdisciplinary team is ambitious and resourceful, actively pursuing expertise in business strategy. Based in Rochester, NY we are uniquely positioned to take advantage of a local environment supportive of biotech startups, strong in engineering, and on the leading edge of medical research. This provides us with a diverse group of leaders and collaborators, with extensive experience in developing diagnostics and leading a startup to success. Glioblastoma and other vasculature-associated neurological disorders remain a challenging, largely open frontier in biotech. At VasoMark, we intend to bring our extensive scientific training to bear in the diagnosis and treatment of these devastating and costly diseases.
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Pro-Arc Diagnostics offers an assay for the presence of virulent and non-virulent JC virus (JCV), allowing patients to safely receive monoclonal antibody (mAb) therapy. 50% of people in the US carry JCV, which is usually non-virulent. However, JCV spontaneously mutates into its virulent form in 30% of carriers. In a small percentage of these carriers, chronic immunomodulatory therapy (e.g. mAb therapy) or immunodeficiency (e.g. AIDS) triggers the fatal neurological disease Progressive Multifocal Leukoencephalopathy (PML). Tysabri® is a promising mAb drug treatment manufactured by Biogen for treatment of Multiple Sclerosis (MS). Because PML is a potential side effect of Tysabri use affecting 1/500 patients, it can only be administered as a last resort through the TOUCH program (Tysabri Outreach Unified Commitment to Health). This restricted prescription paradigm limits Biogen’s market share: less than 9% of eligible patients take Tysabri. Furthermore, since Tysabri more effectively reduces relapse rate and disease progression than first-line treatments for a similar price, this paradigm increases costs for patients and payers. Currently, providers in the TOUCH program do not have access to a diagnostic that differentiates between the virulent and non-virulent forms of JCV, meaning there is no effective way to screen for patients who can safely receive mAb therapy. Physicians are limited to monitoring Tysabri patients for PML symptoms. This causes unnecessary PML incidence in the patient population. We offer a multiplex qPCR assay kit that detects both virulent and non-virulent forms of JCV, allowing clinicians to determine which patients can safely receive mAb therapy and detect spontaneous JCV mutations before PML symptoms arise. We will introduce our diagnostic through the TOUCH program before scaling it out to other at-risk patient populations, including the 1.2 million people with HIV/AIDS. We anticipate that our assay will benefit 1.) Drug-makers, who will gain access to the 400,000 MS patients in the United States, representing $18 billion in potential revenue, and 2.) Payers, who could save $2,000 per additional Tysabri patient per year in reduced remission rates and disease progression costs. With our unique assay, Pro-Arc can increase the access that MS patients have to mAb therapies.
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Our company, angioClast, will provide a new approach for clinicians to treat brain cancer. Using 13 newly patented markers of cancer-specific blood vessels, identified by Dr St Croix of the NCI Center for Cancer Research, we will develop clinical grade antibodies to carry drugs directly to kill cancer blood vessels and prevent cancer progression. A handful of current FDA-approved cancer drugs can hamper the formation of new blood vessels (angiogenesis), but these are not specific to cancer and can cause complications in vital processes such as heart and kidney function and wound healing. In contrast, our new invention has the advantage of disrupting both newly forming and existing blood vessels specifically in tumours. Currently, only a few drugs in Phase I/II of clinical trials are designed to preferentially target cancer blood vessels. This project will start with several stages of R&D. We will first develop and verify the antibodies, which we will later modify to carry specific drugs such as Auristatin and Maytansine (compounds shown to destroy targets in antibody-conjugated forms). Initial capital will be raised from VC’s/Angel investors. After scientifically verifying the viability of the project, we will apply for IP protection in key markets and seek regulatory approval thereafter. In the product development stage, our business model implies the outsourcing of manufacturing and distributary operations, with the support of in-house coordinators. The company will set up an in-house global marketing team, responsible for key market developments, and an R&D facility, to support improvements and further product developments. To fund setup investments and initial WC needs, the company will raise additional capital through a second stage of VC financing. After one year of stable cash flow, we will prepare for initial public offering. This project will be managed by our Cambridge team consisting of seasoned entrepreneurs, with extensive experience in launching startup companies, and scientists and clinicians with cumulative research experience of more than 10 years. Additionally, the board of advisors, including both experts in antibody therapeutics and active clinicians, will support business development.
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Mission: Neurono aims to preclude ~8,600 potential US brain cancer patients from undergoing unnecessary brain biopsies annually by leveraging a new proprietary diagnostic blood test. Specifically, JK Neurotech has developed an assay that detects levels of Anti-TNF Induced Apoptosis (ATIA) protein, which is released specifically by glioblastoma multiforme (GBM). Serum ATIA can predict presence of GBM with 100% specificity and 70% sensitivity. Competitive analysis: ~20,000+ patients have a new brain mass detected on imaging annually. They require brain biopsy to determine tumor type to have the optimal treatment administered. Biopsy is the current standards, and carries substantial risk, including 1.3% risk of death and 6.9% risk of morbidity. Neurono assay can preclude ~8,600 of these biopsies. No other promising diagnostic GBM serum biomarkers have been identified. Value proposition: Neurono provides both clinical (i.e., mortality/morbidity) and economic (e.g., $1,250 for neurosurgeon time, additional for operating room time and hospital stay) benefit to patients and other stakeholders involved in the care of potential brain cancer patients by avoiding brain biopsy. Milestones and strategic vision: - Scientific: The key next step is to validate the ATIA assay in human blood samples. Current data is based on tissue samples – human blood samples from GBM patients are being obtained from a collaborating Chinese laboratory. Mouse models and ATIA’s 70KD size suggest that it will be found at elevated serum levels in GBM patients. - Commercialization: A strategic partner will be required to market and mass-produce the assay given the relatively small patient base. This partner may also provide an option for an exit. - Funding: Funding is needed for the additional validation described above as well as gathering data for the FDA 510(k) application, which takes ~6-12 months for approval. Team: Neurono is led by Josh and Keyvan, two MD/MBA candidates at UPenn Med and Wharton/Harvard. Expert advisors include Drs. Nichtberger and Litt, who have 40+ years combined healthcare entrepreneurial experience. Notably, Dr. Nichtberger has had successful exits, including a $230M IPO (TNGN) while Dr. Litt, in addition to entrepreneurial endeavors, is also an active neurology researcher and clinician as the Director of the Center of Neuroengineering and Therapeutics at UPenn.
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Cephalocare Inc., offers an inexpensive and rapid DNA based chip technology to identify virulent forms of John Cunningham virus (JCV) that causes Progressive multifocal leukoencephalopathy (PML). Multiple Sclerosis (MS) treatment drugs, Tysabri and Avonex, have shown to activate the virulent forms of JCV. Cephalocare Inc will help clinicians segregate existing patient groups to enable safe treatment with MS drugs. With the advent of new JCV diagnostic methods, Cephalocare Inc. believes that by working alongside other companies, it could potentially boost the worldwide market for Tysabri and Avonex from 10 to 20 % in 2016 accounting for $2billion USD. Cephalocare Inc. may help Tysabri and Avonex compete with other MS treatment drugs such as Copaxone, Rebif, Betaseron and Gilenya. Pro forma projections estimated a capture of at least 10% of the global PML diagnostic market and 10% of the global MS market that accounts for $7.5 billion USD. The competitive landscape of Cephalocare Inc includes Quest Diagnostics Inc., which generated revenue of $7.1 billion in 2013 and other PML diagnostic companies. In 2010, Biogen Idec, introduced Tysabri in India, and Cephalocare Inc. will reach out to collaborate with Biogen Idec to capture the expanding MS market in India and North America (NA). Cephalocare Inc., has made collaborations with Sensoreal Inc., (Canada), and Ancil labs Inc., and Get well soon Healthcare Inc., (India) which are biochip and drug companies respectively. Cephalocare Inc., is seeking $1.3m to manufacture biochips, establish its offices and payroll, run its sales and marketing campaigns and other capital requirements. Pro forma projections estimated that cash and accounts receivable could reach $1.5m USD by the end of year. The company attracted an MS patient clinic and a biomedical engineering laboratory at McGill University for research and development. Cephalocare Inc., is headed by Ravishankar Palanisamy who is the President and CEO., and the team involved in the development of the diagnostic tool will own the technology. Cephalocare Inc., aims to adopt “owner buyout” as a primary exit strategy and plans to strike an agreement with the investors and stockholders.
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