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June 19-20, 2014
Washington State
Convention Center
Seattle, WA

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LSINW 2013 Panelist and Presenter Interviews

Life Science Innovation Northwest – Session Summaries

If you were unable to make it to this year’s Life Science Innovation Northwest, we encourage you to read the podium presentation summaries below. Presenter slide decks are made available at the discretion of the presenting organization.

Advanced Medical Isotope Corporation (AMIC):

AMIC is a leader in the production of medical isotopes. Medical isotopes, also known as nuclear medicine, are often used for major diagnoses in cancer, neurological disorders and cardiovascular diseases. AMIC is seeking to develop diagnostic practices based on nuclear medicine that will be more effective and less toxic than what is currently available. AMIC is a very collaborative company; working with the University of Washington and the Pacific Northwest National Laboratory, AMIC has been able to develop a non-invasive diagnostic system based on the isotope Molybdenum (Mo)-99 and the isotope Yttrium (Y)-90. They use polymer seeds and radio-gel therapies to deliver these isotopes to the target and destroy cancer tumors. They have just completed animal studies and have obtained FDA approval to start studies with human subjects. AMIC has several patents and has been developing international collaborations, particularly with China, Russia and South Africa. AMIC is trying to establish a cyclotron facility in Richland, WA in order to create their own isotopes (because currently the US depends on foreign producers) and avoid clinical shortage. This will not only lower the cost of nuclear treatment, but the operation of the cyclotron will create a tremendous amount of jobs. Additionally, AMIC has a patent underway to convert nuclear waste (from nuclear reactors) into useful medical isotopes like (Y)-90. This is really great news for the environment and for future generations. - Graciela Matrajt, University of Washington

Alder Biopharmaceuticals:

The podium presentation of Alder Biopharmaceuticals was full of great scientific data and information. Alder is focusing its research goals on the development of antibodies to be used as a therapeutic method to combat inflammation caused by cancer. In the past, they developed an outstanding method to manufacture high quality antibodies. This method has resulted in therapeutic antibodies that are very efficient, which substantially lowers the price of treatment. The manufacturing system, called Mab Xpress, has allowed the company to enter more efficiently into new markets. In fact, Alder is well-funded: they just raised $38 million in April 2012. At this year's LSINW conference, Alder is presenting its newest development: two antibodies, called ALD403 and ALD317, which are targeted to reduce migraine and dyslipidemia, respectively. For migraines, they have been developing a therapeutic antibody whose target is CGRP (calcitonin gene related peptide). The drug acts in the periphery of the nervous system. So far, the drug has passed Phase I of R&D. During Phase I, Alder proved that the drug prevents the start of a migraine. The drug has 72% viability. This year, Alder is moving this drug into Phase II. The study design is now completed. Alder is still very proud of the outstanding success of their previous drug, Clazakizumab, which is very effective in treating rheumatoid arthritis, with 43% patient remission. - Graciela Matrajt, University of Washington

CEO Randall Schatzman presented Alder’s development of calcitonin gene-related peptide (CGRP) antibody, ALD403, for the treatment of high frequency migraines. Their work effectively builds on knowledge gained from a previous attempt to block the CGRP receptor using a small molecule developed by Merck. CGRP is released from trigeminal nerve endings and acts on smooth muscle cells of cerebral blood vessels, resulting in vasodilation, which is the underlying cause to migraine headaches. Although Merck’s therapy failed to reach the market due to liver toxicity, it provided an important proof of concept. Alder Biopharmaceuticals ALD403 monoclonal antibody is currently undergoing Phase 1 proof-of-concept clinical testing. Due to a long biological half-life of 28 days, ALD403 is well posed to effectively prevent the onset of migraine headaches. Its convenient administration via infrequent subcutaneous injections seem to provide additional potential advantage for this biologic. In preclinical testing ALD403 effectively reversed photophobia, one of the key attributes of migraines. In the initial Phase 1 study in human volunteers, ALD403 effectively blocked capsaicin-induced vasodilation for up to 3 months after a single dose. Another antibody in the Alder Biopharmaceuticals pipeline targets IL-6, a proinflammatory cytokine contributing to the clinical picture of rheumatoid arthritis (RA). Monoclonal antibody ALD518 blocking IL-6 activity successfully completed phase 2 studies for the RA indication; future Phase 3 studies will be conducted in collaboration with Bristol-Myers Squibb. Data from the Phase 2 studies show better efficacy than the established anti-TNF-alpha therapies after 16 weeks and two doses of ALD518 and 43% patients unresponsive to methotrexate therapy achieved ACR70 response. - Petra Volna, University of Washington

Aminex Therapeutics:

Many will agree with President & CEO Mark Burns that the ideal cancer therapeutic would combine all of the following attributes: capable of targeting hard-to-treat and genetically diverse tumors, achieving durable therapeutic response and financially affordable. Aminex Therapeutics based its approach to cancer therapy on blocking the polyamine transport to the tumor cells by Polyamine-Blocker Therapy (PaBTTM), or AMXT 1501, in combination with DMFO (difluoromethylornithine or elfornithine, marketed as Ornidyl). Polyamines have been revealed as natural suppressors of the immune system, and the polyamine rich microenvironment surrounding tumors likely contributes to the tumors’ immune evasion. Aminex’s strategy to combine its proprietary PaBTTM to block the polyamine transport and DMFO to block the polyamine biosynthesis inside the tumor cells showed promise in preclinical experiments in mice. In skin cancer models bearing the Ker/Ras transformed keratinocytes, the combination therapy of PaBTTM and DMFO achieved a durable response also after the therapy was stopped. Aminex is planning to file an IND in 2013 to start the initial Phase 1 clinical study for subcutaneously delivered AMXT 1501 plus oral DMFO. The study design includes a single AMXT 1501 dose in week 1 to study the pharmacokinetics and pharmacodynamics of this agent and the combination therapy for weeks 2 through 4 to examine its safety and efficacy. - Petra Volna, University of Washington

Aqueduct Neurosciences Inc.:

At LSINW, Aqueduct Neurosciences discussed exciting new technology developments. This year, Aqueduct Neurosciences presented their new medical device, SED; its purpose is to automatically control pressure and CSF flow in hospitalized patients with drainage problems. Current devices do not allow the patient to have mobility, which means the patient needs constant assistance. The new device developed by Aqueduct Neurosciences is a drain device controlled automatically, allowing the patient to keep his/her mobility and be more autonomous. Consequently, patients will feel better during their recovery, they will be safer (because the drainage is controlled automatically) and this patient autonomy will improve the hospital's economics. Aqueduct Neurosciences is looking to partner with someone for the distribution of this SED device. The technology was developed at the University of Washington, where the licensing is being issued. Their plan is to make clinical trials post market. Aqueduct Neurosciences is seeking to raise $3 million to be able to afford operation costs through 2014. Their next step is to test the device for its usefulness in the diagnosis of NPH (Normal Pressure Hydrocephalus). - Graciela Matrajt, University of Washington

CEO, Tom Clements M.S., M.B.A, engaged the audience and took advantage of their participation to demonstrate the advantages of the company's Smart Shunt, a CSF drainage device they consider to be superior to current market standards. The Aqueduct device would allow for remote monitoring and automatic draining height adjustment, based on intracranial pressure measurements. - Marie-Laure Crouch, Senior Scientist, Drug Discovery/Life Sciences

Atossa Genetics Inc.:

Kyle Guse, Chief Financial Officer of Atossa Genetics Inc., presented the company’s recent platform developments.  Atossa is singularly focused on breast health. The Atossa platform uses breast aspirate as a sample to analyze a woman’s risk of getting breast cancer. This platform is designed for the investigation of precancerous changes in breast cells and is marketed to women ages 17-83. Breast aspirate gets collected by a nurse or a physician assistant with a handheld breast pump and is then sent to the Seattle-based lab. The test is non-invasive and fairly painless.  The data analysis facilitates IBIS algorithms that use lab test results and a patient’s family history to evaluate her risk of getting breast cancer. The result then categorizes patients in three groups of normal risk (<15%), intermediate risk (15-20%) and high risk (>20%). Each group then has to take respective suitable further steps.  The MSRP for this test is $1300. Atossa has recently partnered with major medical distributers, e.g. Thermo Fisher, and currently serves 61% of the US population. Atossa is also developing a treatment product that uses micro-catheters to deliver the cancer therapeutics to the cancer site through the nipple. This spares the breast’s healthy parts from chemotherapy, radiation and surgery.  - Rosa Daneshvar, Bioengineer/Chemical Engineer 

Bill & Melinda Gates Foundation:

The Bill and Melinda Gates Foundation is one of the world’s leading philanthropic institutes with an annual give of three billion dollars. The foundation focuses on three different areas: Global Health, Global development and US programs. Global health focuses on the utilization of science and technology to create high quality and affordable vaccines for developing nations. Global development focuses on getting this science and technology to these targets areas (e.g. how we finance these ventures). US programs focuses on the success of college ready/ post-secondary students. Their podium presentation focused on the concept of bringing more business principles to philanthropic work through their program-related investments initiative strategy; started in 2011 as a way in which private foundations can make investments for a charitable goal. This program utilizes low interest loans to NGOs, financial institutions or companies, direct equity investments, as well as fund investments. The criteria for the program includes programmatic impact, leveraging external capital, scalable solutions, passing the “but for” test (project would not be possible but for this investment), appropriate level of subsidy, project is within portfolio limits, acceptable portfolio management burden and the size of investment. An example of this program in action would be the leveraging of a company’s funds as a volume guarantee to bring down the price of birth control for women from $40 to $20. - Alec Lindberg, University of Washington

CMC Biologics, Inc.:

CMC Biologics, Inc. is a contract manufacturing organization (CMO) specializing in contract development and manufacturing of protein-based medicines from the preclinical development stage through scale up manufacturing for clinical testing and commercialization phases. CMC Biologics is based in Copenhagen, Denmark and has 2 other manufacturing facilities in Bothell, WA and Berkeley, CA. It has already served over 60 different customers from the US, Canada, Europe and Australia and has generated over $100 million in revenues. The company has signed its first Commercial Supply Agreement with a European company and two more are expected to be signed in 2013, generating a stable supply of revenue. CMC Biologics proprietary technology CHEF1® expression platform enables standardized cell line development. The company’s recently acquired facility in Berkeley, CA has a capacity of 3000 L of mammalian cell culture and claims to be the fastest development platform for monoclonal antibodies in the world. The platform enables researchers to develop up to 500 g of pure monoclonal antibody in GMP standard quality from the initial DNA construct in just 12 months. As of July, CMC Biologics has developed over 11 biosimilars, with one expected to receive marketing authorization approval in late 2013. In addition to mammalian expression system, the company operates microbial expression manufacturing platforms and has the capacity for cGMP cell banking. - Petra Volna, University of Washington

GE Healthcare-Life Sciences:

Rick Loya, Senior Sales Manager, presented on the original equipment manufacturer (OEM) services that the company provides. Originally known as Applied Precision, the company was established 30 years ago by three engineers in Issaquah, Washington. In 2010, it was acquired by GE. GE Healthcare provides the expertise for design, integration and manufacturing instruments in a wide range of applications. Some examples of previous products are: microscopy, cellular imaging, genomic imaging, cell analysis, etc. Applied Precision is Nikon’s largest consumer of microscope lenses west of Mississippi. The company now follows the GE quality management system and benefits from GE’s world-wide infrastructure. - Rosa Daneshvar, Bioengineer/Chemical Engineer


Heapsylon is a small startup based in Redmond Washington. Wearable technology is a growing market; however, the foot is underserved. Heapsylon’s solution to this gap is Sensoria, a device that interfaces with three separate components: a smart-fabric sock (washable & dryable) that contains E-textile sensors, an anklet that contains additional sensors and battery and a tracking App for mobile devices. Sensoria is extremely accurate and more informative than current wearable running-technologies, as it is able to count steps and identify signal patterns. The information gathered by Sensoria is widely applicable and could be used in multiple scenarios ranging from medical uses to fitness. Heapsylon is looking for a limited number of partners to help create custom APIs to best utilize this technology, due to its potential application in multiple fields. - Alec Lindberg, University of Washington

Immune Design:

Immune Design’s focus includes cancer immunotherapy, infectious disease and allergy. For cancer immunotherapy, the company aims to boost the immune response by priming dendritic cells to stimulate cytotoxic T lymphocytes (CTL) response. While Dendreon builds on the same concept in its prostate cancer therapy Provenge, Immune Design combines a lentiviral vector mediated delivery of the target tumor-related antigen directly to dendritic cells via DC-SIGN receptor and TLR4 receptor agonist, GLA, as a powerful novel adjuvant. Major advantages of this approach include the vaccine-like delivery to the tumor draining lymph node and a single therapeutic suitable for treating all patients diagnosed with a certain type of cancer. Studies in animal models showed in vivo activity of VP02 lentiviral vector plus TLR4 agonist limited to the tumor draining lymph nodes and no activity in the control lymph nodes and off-target organs. The therapy also showed increased counts of CD8 positive T-cells nine days after the delivery of this vaccine-like therapy and improved survival in experimental animal models. Immune Design has already filed the first IND for its VP02 plus GLA cancer immunotherapy intended for treatment of Merkel Cell Carcinoma, and is expected to file another IND later this year. The company decided not to publically reveal the target cancer antigens used to prime the dendritic cells and delivered via the lentiviral vectors. In addition, the GLA adjuvant is claimed to be best-in-class molecular vaccine adjuvant and much more effective than the GlaxoSmithKline proprietary adjuvant MPL. To this end, Immune Design is aiming to develop further collaborations through its infectious disease program for the use of GLA in pandemic flu and other vaccines. - Petra Volna, University of Washington


Theresa Seldon Ph.D., Director of product development at Immunexpress, presented on a new molecular diagnostic test for sepsis, a long neglected diagnostic and therapeutic area. Immunexpress currently has a series B financing option open for enrollment. - Marie-Laure Crouch, Senior Scientist, Drug Discovery/Life Sciences

M3 Biotechnology:

Dr. Lewis Rumpler, CEO of M3 biotechnology, presented on the social and personal impact of neurodegenerative disease (NDD); current nationwide investments in NDD therapeutic research and the success rate of outcomes were highlighted. The prescribed medication for current Parkinson patients was developed forty years ago, indicating slow progress in this area of therapeutics. M3 is taking a new approach on NDD therapeutics. The M3 discovery platform targets growth factor multimerization. They have developed a small molecule, MM-201, which is mimetic of Hepatocyte Growth Factor (HGF). This blood-brain barrier permeant molecule facilitates the regrowth and restoration of neuro-tissue at a faster rate than the disease can destroy. Rat-rope-hang and gape study results for proof of concept were presented. In both studies, results for lesioned and treated and sham surgery animals were compared. Video from the rope-hang-study showed that lesioned and treated animals could stay on the rope twice as long as the sham surgery animal.  MM-201 for Parkinson disease is M3’s major therapeutic development plan going forward. - Rosa Daneshvar, Bioengineer/Chemical Engineer

Lewis Rumpler, CEO of M3 Biotechnology, described the exciting results obtained with their lead small molecule, MM201, in the treatment of neurodegenerative diseases, in particular Parkinson’s disease. Most impressive, was the video showing that, in the rat hang rope model, the treated rat held on for 35-seconds compared to the 15 and 5-seconds of the healthy and untreated diseased rat, respectively. - Marie-Laure Crouch, Senior Scientist, Drug Discovery/Life Sciences


MalarVx CEO, James Davie, gave an overview of the global problems associated with malaria infections and the efforts by MalarVx to develop a genetically attenuated live parasite vaccine. - Marie-Laure Crouch, Senior Scientist, Drug Discovery/Life Sciences

Nanofacture Inc.:

Sherb Edmonson, CEO of Nanofacture Inc., presented the capabilities of their hand held device for the isolation of DNA from field samples, which would allow for later processing in the laboratory. Nanofacture is currently searching for either a distribution partner, or a companion assay to their device for a planned 2014 commercialization into an estimated $200 million market. - Marie-Laure Crouch, Senior Scientist, Drug Discovery/Life Sciences

Nova TheraNostics LLC:

Robert Abbott, CEO, presented the company’s product and described how it differentiates from its competitors. NTN-101 is a lipid-based nanoparticle used as a contrast agent in MRI imaging. There are nine approved contrast agents currently being used for MRI. However, these contrast agents have limitations that consequently limit the application of MRI in comparison to other imaging tools; small molecules that escape vasculature, causing signal to noise deterioration rapidly within imaging time. Moreover, all agents except one are cleared by the kidney and subsequently obscure renal imaging due to the brightness of accumulated agents. NTN-101 has four attributes that distinguishes it from other existing agents. It is vigorously intravascular, yielding high signal to noise ratio. It has a very long half-life of six hours versus the thirty-minute half-life of existing agents. It is mainly liver excreted, which facilitates clear kidney imaging. Finally, it is brighter in comparison to existing agents. The aforementioned properties open up the possibility of using MRI in new areas, which have not been possible using existing agents. - Rosa Daneshvar, Bioengineer/Chemical Engineer

Robert Abbott, President and CEO of Nova TheraNostics, gave an introduction to the impressive imaging capabilities of their new lipid based and brighter MRI contrast agent: Nanobright. With its six hour half-life and 97% liver excretion, Nanobright could provide the means for real-time endovascular and renal imaging studies. - Marie-Laure Crouch, Senior Scientist, Drug Discovery/Life Sciences


Obenomics president, Yansong Gu, Ph.D., described the exciting activity of their lead molecule, OB1201, a first-in-class intervention to reduce the risk of breast cancer in obese post-menopausal women. OB1201, a SIRT-1 modulator, targets adipose tissues, including breast tissue, and is being proposed as a future replacement of tamoxifen in the treatment of breast cancer. - Marie-Laure Crouch, Senior Scientist, Drug Discovery/Life Sciences

Omeros Corporation:

The Omeros pipeline is divided into three major areas: pharmacosurgery, G protein-coupled receptors (GPCR) and other. In his presentation, Chairman & CEO Gregory Demopulos showed the results from Phase 3 clinical trials in ophthalmology surgery for OMS 302, a combination of phenylephrine and ketorolac. Phenylephrine is considered standard of care premedication to produce mydriasis (pupil dilation) for intraocular lens replacement and/or cataract extraction. The evidence from clinical trials showed that the addition of ketorolac effectively blocks the reactive constriction of the pupil during the surgery by blocking the production and release of prostaglandin A in the eye. Ketorolac provided statistically significant and clinically meaningful improvement in intraoperative miosis, pupil constriction and postoperative pain. In addition, the drug was well-tolerated; producing no difference in adverse reactions compared to the placebo (here meaning the standard therapy). Omeros is preparing to file NDA within several weeks and is expected to file MAA in Europe later in 2013, and begin marketing the product in 2014. Omeros also operates an automated screening platform for GPCRs drug targets generating further value in intellectual property. Other programs include OMS 824, phosphodiesterase 10 (PDE 10), an inhibitor targeting primarily Huntington’s disease and schizophrenia, and phosphodiesterase 7 (PDE 7), an inhibitor for the treatment of cocaine and nicotine addictions. Omeros is also developing OMS 271, an antibody blocking MASP-2 protein, which was involved in the activation of complement system and human plasmin inhibitor, Kunitz Domain 1 (KD1) of Human Tissue Factor Pathway Inhibitor-2, for which the company plans to file IND in 2013. - Petra Volna, University of Washington

OncoGenex Pharmaceuticals Inc.:

Scott Cormack, CEO, presented the current state of ten ongoing OncoGenex clinical trials. The current focus of the company is on the development of new therapeutics addressing the treatment resistance of cancerous tumors. Cancer tumor cells change during the course of cancer treatment and develop resistance to therapeutics. OncoGenex is conducting ten clinical trials for pancreas, lung, prostate and bladder cancer with two developed molecules: Custirsen and OGX-427. A majority of the clinical trials are in phase 2 and 3 clinical development stage.  Among these trials, Synergy, a prostate cancer trial, is the most well-known. Synergy is a phase 3 prostate cancer clinical trial which ended last year with 1,000 patients. Phase 2 clinical trial results for prostate cancer has shown a 39% increase in patient survival rate. - Rosa Daneshvar, Bioengineer/Chemical Engineer

Scott Cormack, President and CEO of OncoGenex Pharmaceuticals, gave an impressive overview of 10 different clinical trials, either recently completed or ongoing, for their two lead molecules, curstisen and OGX-427, targeting bladder, lung and pancreatic cancers. - Marie-Laure Crouch, Senior Scientist, Drug Discovery/Life Sciences

Paw Print Genetics:

Paw Print Genetics is the brainchild of Lisa G. Shaffer, a geneticist who brings her years of expertise to the developing field of canine genetics. The U.S. pet industry is a $53 billion industry and Dr. Schaffer recognized an opportunity to tap into an underserved market. Years of inbreeding have led to a host of fixed mutations in the canine population, resulting in a myriad of costly and undesirable inherited conditions. Paw Print Genetics developed a model using human based diagnostic standards, coupled with free genetic counseling, to help breeders, owners and trainers screen for 100 different canine disorders with the goal of decreasing the long-term cost of pet ownership. In its first year, Paw Print Genetics conducted 46,000 tests and is currently seeking partners. Additional congratulations to Dr. Shaffer for being a recipient of the 2013 LSINW Women to Watch in Life Science award. - Eleni Adams, Substantial Inc.

Philips Healthcare:

The podium presentation of Philips Healthcare was outstanding; the speaker was excellent and the content was full of innovative ideas. This year, Philips Healthcare is presenting their newest technology, a medical imaging device designed to do ultrasound at a lower cost and with much more reproducibility. It is called Anatomically Intelligent Ultrasound (AIUS) and it is truly an innovative idea. Based on the fact that ultrasound has a lower cost than other diagnostic technologies (NMR, PCT), and that it is vastly used in several branches of medicine (cardiovascular, gastrointestinal and OB/GYN), there is a need to advance its technology. One of the most apparent limitations of today's ultrasound technology is the lack of reproducibility. Not being able to perform a trusted and reproducible measurement may result in an incorrect diagnosis, which can have serious ramifications. It is important to be able to make reproducible measurements in order to deliver the best diagnosis and treatment. The functionality of the AIUS device is based on a model that will automatically adapt to the patient's own variable anatomy. Within the proposed model, the device can automatically align and segment ultrasound images and perform automated measurements. The device can “learn” about specific features in the anatomy of the patient and can make a personalized interpretation of the results. A specific device used in cardiology ultrasound is now available and it is called Xmatrix. Philips is investigating whether it can also be used in renal ultrasound. By developing an automated device, it is easier and faster to carry out these procedures, which decreases the need for expensive staff training (because it is easy to operate), lowers the amount of dollars spent per exam (because it works faster), and lessens the need to repeat exams (because it is more reliable). Overall, the AIUS will allow for better therapy and outcomes. - Graciela Matrajt, University of Washington


Christian Burks Ph.D., CEO of Pregenen, described their DisplayArray™ and TrueCut™ platforms, which allow for the generation of custom engineered high-specificity nucleases. Pregenen is entering preclinical validation of PGN-312, which targets CCR5 for the treatment of HIV. - Marie-Laure Crouch, Senior Scientist, Drug Discovery/Life Sciences

Presage Biosciences:

Dr. Nathan Caffo, President of Presage Biosciences, presented Presage’s recent progress on cancer drug development. Low efficacy of the current cancer treatment procedures, underdeveloped genomic approaches in personalized medicine and the adverse effect of cancer drug toxicity were highlighted. The in-vivo results of an injected cancer drug into several areas of a cancer tumor were presented. The data showed different localized responses caused by the complex and heterogeneous structure of the cancer tumor. The Presage CIVOTM platform is designed to improve treatment decisions by assessing the therapeutic effect of multiple drugs simultaneously by microinjecting specific doses of the drugs in a particular location of a tumor; the feasibility trial is ongoing with lymphoma patients. The goal of the trial is the toxicity-sparing pretreatment assessment of the drug response. Microinjection of the drugs through local anesthesia takes 20 seconds. Afterwards, the histological process is conducted on the whole tumor to address the drug response. To date, four patients have been dosed. - Rosa Daneshvar, Bioengineer/Chemical Engineer


Richard Watson, CSO of Revalesio, presented the company’s recent progress in anti-inflammatory therapeutics. Revalesio’s core technology, charge stabilized nanostructure (CSN), is a stabilized nano-bubble in solution.  The therapeutic CSN candidate, RNS60, is comprised of normal saline and medical grade oxygen.  The super saturation of liquid with the gas and stability is achieved by a combination of manufacturing properties and the properties of the solution itself.  RNS60 has been used in clinical trials of asthma, multiple sclerosis, Alzheimer’s disease, Parkinson’s disease and acute myocardial infarctions. - Rosa Daneshvar, Bioengineer/Chemical Engineer

Seattle Genetics:

COO Eric Dobmeier described Seattle Genetics’ proprietary technology of antibody drug conjugates (ADC) as a way of “empowering antibodies.” In ADCs the company uses potent synthetic drugs that are easy and cheap to manufacture and are attached to the antibody by a stable linker. Once the ADC is delivered to the inside of the target cell, the linker is readily cleaved by an enzyme, thus releasing the potent drug. Currently, Adcetris is marketed for the treatment of CD30 positive relapsed Hodgkin’s lymphoma and Anaplastic Large Cell Lymphoma. Multiple clinical trials are ongoing to expand the indication to other CD30 positive hematological malignancies such as B-cell lymphomas, Diffused Large B-cell Lymphoma (DLBCL) and as a frontline therapy for patients with Hodgkin’s lymphoma who are over 50-years-old and would otherwise receive bone marrow transplants. The results from clinical studies are encouraging with complete response rates of 34% for T-cell lymphomas, 44% for DLBCL and 50% in mature T/NK cells neoplasm. For the approved indications, Adcetris generated over $215 million in net sales since its approval in August 2011. In addition, a permanent J-code, effective from January 2013, was secured for Adcetris, achieving a positive reimbursement environment for the drug. Seattle Genetics has partnered with Takeda and its subsidiary Millennium to operate in Europe and other markets. The company partners are currently negotiating reimbursement deals in Europe. Seattle Genetics has three more ADCs in clinical testing and four others are expected to enter clinical trials in 2013. - Petra Volna, University of Washington

Tekmira Pharmaceuticals Corporation:

Mark Murray, CEO, presented updates on the company’s two major products: TKM-PLK1 (oncology candidate) and TKM-Ebola (virus infection candidate). Tekmira’s business is based on RNA interference (RNAi) technology. The RNAi system is comprised of natural mechanism mammalian cells used to eliminate unwanted proteins. Tekmira is producing compounds that trigger RNAi. They have developed a technology for delivering these compounds to cells called lipid nanoparticle (LNP). LNP is a lipid technology, where a small sphere completely encapsulates and protects the active molecule that triggers RNAi. Tekmira’s oncology candidate, TKM-PKL1, is named after its target enzyme: PKL1. PKL1 is key regulator in cell mitosis. PLK1 is up-regulated in many tumor types. The TKM-PLK1 agent is designed to effect tumor cell expression of PLK1. TKM-PKL1 is currently being employed at a phase one clinical trial where, out of nine dosed patients, four have achieved clinical benefit. Tekmira is planning to start phase 2 clinical trials later this year. The company’s second candidate, TKM- Ebola, is an agent that is delivered to infected cells to trigger RNAi against the deferent part of the Ebola genome. This program is funded by the U.S. Department of Defense. A $140 million contract is in place to develop this agent as an FDA approved therapeutic to be used in Ebola virus infection therapeutics. Tekmira’s revenue partially comes from the royalties received from partners who use their technology. - Rosa Daneshvar, Bioengineer/Chemical Engineer

Tekmira Pharmaceuticals President and CEO, Mark Murray Ph.D., gave an overview of their lead clinical candidates, TKM-PLK1 and TKM-Ebola, which both take advantage of Tekmira’s lipid nanoparticle (LNP) delivery platform for RNAi. - Marie-Laure Crouch, Senior Scientist, Drug Discovery/Life Sciences


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