Targeting Ocular Disorders

Transcription

1 Targeting Ocular Disorders final agenda October 6-7, 2014 Westin Boston Waterfront Hotel Boston, MA Register Today to Reserve Your Place! Preserving Vision through Innovative Drug Discovery and Development Topics Include: New disease pathways Alternative drugs Anti-inflammatories Biologics Bi-specific therapies Novel therapeutics Anti-angiogenics Drugs in clinical trials Corporate Sponsors Organized by Cambridge Healthtech Institute

2 Targeting Ocular Disorders October 6-7, 2014 Westin Boston Waterfront Hotel Boston, MA With the aging of the population, the need for treatments of ocular diseases and disorders has become more important than ever. Increasingly high incidences of age-related macular degeneration (AMD), glaucoma, diabetic retinopathy and ocular inflammatory diseases demand better, more effective and innovative treatments. If we are to maintain the quality of life for this aging population, the preservation of vision is critical. This conference will address targeting of ophthalmic medications and treatments and will provide the latest results in the field, along with novel and emerging targets that hold promise in treating these quality-of-life threats. MONDAY, OCTOBER 6 1:00 pm Registration 2:00 Opening Remarks by Chairperson»» KEYNOTE PRESENTATION 2:10 Systemic Administration of Anti-Ab mab Reduces Retinal Deposition of Abeta and Activated Complement C3 in Age-Related Macular Degeneration Mouse Model Ian R. Catchpole, Ph.D., Topical BioPharm Discovery Research and Development, Glaxo-Smith Kline Cfh-/- mice were treated systemically in both prophylactic andtherapeutic regimes with an anti-ab monoclonal antibody (mab), 6F6, todetermine the effect on the cfh-/- retinalphenotype. Prophylactic treatment with 6F6 demonstrated a dose dependent reduction in the accumulation of both Aband activated C3 deposition. SerumAb levels after systemic administration of 6F6 show accumulation of Ab in the periphery suggestive of a peripheral sinkmechanism. In summary, anti-ab mab treatment can partially prevent or reverse ocular phenotypes of the cfh-/- mouse. The data support this therapeutic approach in humans potentially modulating two key elements in the pathogenesis of AMD Ab and activated, complement C3. EMERGING THERAPIES FOR RETINAL AND MACULAR DISORDERS 2:40 Oral Therapies for Retinal Degeneration: Case Study of ALK-001 Leonide Saad, Ph.D., CEO, Alkeus Pharmaceuticals, Inc. Safely attaining biologically active sustained amounts of a compound in the retina is notoriously difficult. Here we present how compounds delivered orally can find their way to the retina. We further discuss the science behind ALK-001, an oral investigational new drug designed to inhibit the formation of toxic byproducts thought to be responsible for major retinal degenerations such as AMD, Stargardt, Best disease or autosomal recessive Retinitis Pigmentosa. 3:10 Wnt Pathway Inhibition for Diabetic Retinopathy James Larrick, Ph.D., Founder, Managing Scientific Director, CMO, Panorama Research Institute, Wntgen LLC & Velocity Pharmaceutical Development Activation of the Wnt pathway via LRP6 cell surface signaling contributes to the severe retinal/choroidal neovascularization observed in Vldlr-/- mice. Elevated Wnt signaling is also observed in human retinas of patients with diabetic retinopathy. We identified a novel murine monoclonal antibody that antagonizes LRP6 activity which demonstrates significant activity in numerous animal models of retinal disease. A humanized form of this antibody may be a novel therapy for diabetic retinopathy. 3:40 MANF - A Novel Neurotrophic Factor for Sponsored by the Treatment of Retinal Disorders Roman Urfer, Ph.D., Chief Development Officer, NeuroAssets Sarl Mesencephalic astrocyte-derived neurotrophic factor is the prototype of an emerging family of neurotrophic factors with evolutionary highly conserved structure and function. MANF is expressed in response to cellular stress and during retinal development. MANF s activity in genetic and pharmacolocical models of retinal disorders will be presented. 3:55 Refreshment Break in the Exhibit Hall with Poster Viewing 4:25 Nanoliter Scaled Immunoassays: Enabling Sponsored by Biomarker Assay Development in Low Volume Ocular Samples Robert A. Durham, Ph.D., Director, Field Applications North America, Gyros US, Inc. Biotherapeutic approaches that target diseases of the eye pose a bioanalytical challenge due to the small sample volume of relevant matrices in the target organ. To this end we have evaluated the feasibility of using a nanoliter-scaled immunoassay on the Gyrolab for pharmacokinetic and biomarker assays to support ocular diseased projects. This presentation will review data from Biomarker and PK measurements made in vitreous humor, and tear samples. The results show that the Gyrolab platform enables development of low volume assays for PK and biomarker measurements in the eye. 4:55 Combination Therapy in Diseases of Retinal Origin: A New Paradigm David Sherris, Ph.D., CSO, RestorGenex, Corp. Neovascular retinal and subretinal diseases as in age-related macular degeneration and proliferative diabetic retinopathy occur through a cascade of events over time. Numerous cytokines have shown involvement throughout the disease process. Anti-VEGF technologies inhibit edema and hemorrhage due to their ability to reduce vascular permeability and to some extent inhibit neovascularization. However, VEGF is not the only cytokine in these pathologies. Here we will discuss the influence of a cytokine storm in back-of-the-eye diseases with the example of P529, a first-in-class, allosteric, dual dissociative inhibitor of the TORC1 and TORC2 complexes within the PI3K/Akt/mTOR pathway in combination with anti-vegf. 5:25 Welcome Reception in the Exhibit Hall with Poster Viewing 6:25 Close of Day Present a Poster CHI encourages attendees to gain further exposure by presenting their work in the poster sessions. Your poster will be exposed to our international delegation Receive $50 off your registration Your poster abstract will be published in our conference materials Your research will be seen by leaders from top pharmaceutical, biotech, academic and government institutes To secure a poster board and inclusion in the conference materials, your abstract must be submitted, approved and your registration paid in full by September 5, Targeting Ocular Disorders

3 TUESDAY, OCTOBER 7 8:15 am Morning Coffee NOVEL PATHWAYS AND STRATEGIES 8:45 Chairperson Remarks 8:50 A Synthetic Chemically Modified sirna Targeting Caspase-2 as a Therapeutic Agent for Ocular Neuroprotection Elena Feinstein, M.D., Ph.D., CSO, Quark Pharmaceuticals Caspase-2 is a pro-apoptotic gene specifically expressed and activated in retinal ganglion cells following acute optic nerve injury or intraocular pressure increase. Intravitreal administration of QPI1007 (a syntehticsirna targeting caspase-2) demonstrated local RNA interference, lack of inflammatory effects and excellent preclinical and clinical safety profiles. Neuroprotective activity of QPI1007 has been demonstrated in five preclinical animal models involving RGC loss. In human Phase I trials in NAION patients, a single intravitreal injection of QPI1007 within 2 weeks following disease onset prevented further visual deterioration. 9:20 Nanobodies as Next-Generation Therapeutics for Ocular Applications Tony De Fougerolles, Ph.D., CSO, Ablynx NV Next-generation ophthalmic therapeutics must aim at being superior to the currently available agents with regards to efficacy and improved drug delivery. We show here that Nanobodies deliver these properties and present a novel class of highly potent and specific drug candidates for the treatment of eye disorders. 9:50 Local Inhibition of Cytokine Signaling to Treat Anterior and Posterior Ocular Disorders Eric Furfine, Ph.D., CSO, Eleven Therapeutics Cytokines, chemokines, and growth factors mediate anterior and posterior eye diseases. Our lead product, the IL-1 receptor inhibitor EBI-005, was designed and engineered for the topical treatment of dry eye disease and was biologically active in subjects with dry eye disease. In addition, we engineered an IL-6 inhibitor with potential for local treatment diabetic macular edema. Finally, a novel soluble receptor inhibitor of cytokines IL-17A and IL-17F was engineered for the local treatment of uveitis. Both IL-6- and IL-17-targeted drugs were designed and engineered for intravitreal administration. 10:20 Coffee Break in the Exhibit Hall with Poster Viewing 10:50 Topical Aganirsen in Eye Diseases: From Phase III Results in the Cornea to Retinopathies - A New Revolution Eric Thorin, Chief Development Officer, Gene Signal International SA The antisense aganirsen inhibits the expression of Insulin Receptor Substrate-1 (IRS-1). The phase III showed that its topical application induces regression of pathological corneal neovascularization and reduces the need for corneal transplantation. Topical aganirsen reaches the retina, which is at the basis of its clinical development for the treatment of AMD and DME. Aganirsen should become the first therapy active by topical application for the treatment of angiogenesis-based retinopathies. 11:20 NLRP3 Inflammasome Activation in Retinal Pigment Epithelial Cells by Lysosomal Destabilization: Implications for Age-Related Macular Degeneration Bruce R. Ksander, Ph.D., Associate Professor, Ophthalmology, Schepens Eye Research Institute/Massachusetts Eye and Ear NLRP3 upregulation occurs in the RPE during the pathogenesis of advanced AMD, in both geographic atrophy and neovascular AMD. Destabilization of RPE lysosomes induces NLRP3 inflammasome activation, which may contribute to AMD pathology through the release of the proinflammatory cytokine IL-1β and through caspase-1-mediated cell death, known as pyroptosis. 11:50 Maximizing Chances of Success in the Sponsored by Clinic: The Role of Animal Models in the Development of Ocular Therapeutics Claire M. Gelfman, Ph.D., Director, Pre-Clinical Services, Ora Achieving proof-of-concept in animal models of ophthalmic disease is a commonly desired prerequisite for advancing drugs into the clinic. The choice of endpoints for evaluation should mirror, when possible, those that will be evaluated in the clinic. In this presentation, we will review several models that recapitulate specific features of human ophthalmic disease, and discuss the integration of that information into the larger clinical development plan in order to maximize chances of success. 12:20 pm Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own 1:50 Chairperson s Remarks CELL-BASED AND BIOLOGICAL THERAPIES 1:55 AAV-Based Gene Therapy for Genetic Ocular Diseases Sue Washer, President and CEO, Executive, AGTC The utility of AAV-based gene therapy for the treatment of ocular disorders has been validated in human clinical trials for Leber congenital amaurosis and wet AMD. Given their safety and persistent expression, AAV vectors are an optimal approach to providing long-lasting treatment for genetic eye diseases such as archromatopsia, X-linked retinoschisis, and X-linked retinitis pigmentosa. Current investigational AAV-based therapies for these indications will be discussed. 2:25 Porous Poly({varepsilon}-caprolactone) Scaffolds for Retinal Pigment Epithelium Transplantation Magali Saint Geniez, Ph.D., Assistant Scientist, Schepens Eye Research Institute; Assistant Professor, Harvard Medical School Retinal pigment epithelium (RPE) transplantation is a promising strategy for the treatment of dry age-related macular degeneration. However, previous attempts at subretinal RPE cell transplantation have experienced limited success due to poor survival on aged or diseased Bruch s membrane. To improve the clinical outcome of RPE transplantation, we are developing biomimetic and biocompatible scaffolds able to not only support RPE survival but also significantly improve cell morphology, gene expression and function. 2:55 Stem Cell-Based Therapeutic Strategies for Understanding and Treating Ocular Surface Failure Sajjad Ahmad, M.D., Consultant Ophthalmic Surgeon, St. Paul s Eye Unit, Royal Liverpool University Hospital The cornea is the clear front of the eye and its clarity is vital for visual function. The corneal surface is maintained by stem cells and these fail in the disease of limbal stem cell deficiency resulting in painful blindness. Stem cell-based cellular therapies are increasingly being used to treat patients with this debilitating disease. I have developed an animal-free culture system for expanding human limbal stem cells that has been used to successfully treat patients with limbal stem cell deficiency. In addition, using in vitro disease models, I am currently investigating the role of the limbal stem cell microenvironment in contributing to disease progression so that therapeutic strategies can be developed earlier on in the disease process. 3:25 Interim Analysis of the Safety and Preliminary Efficacy of Human Neural Stem Cells (HuCNS-SC) in Geographic Atrophy (dry AMD) Joel Naor, M.D., M.B.A., M.Sc., Vice President, Clinical Development, Ophthalmology, StemCells, Inc. A multi-center dose-escalation Phase I/II trial (clinicaltrials.gov: NCT ) for patients with the dry form of AMD is investigating the safety and preliminary efficacy of 200,000 and 1 million cell doses administered to the subretinal space of the study eye. Post-transplant assessments include tests of visual acuity, fluorescein angiography, spectral domain ocular coherence tomography (OCT), microperimetry, multifocal electroretinography, and contrast sensitivity. Interim results confirm the safety and tolerability of HuCNS-SC cell transplantation into the subretinal space and show slowing of disease progression and improvement in visual function / contrast sensitivity. 3:55 Decellularized Retina Matrix: Natural Substrates for Human Retinal Progenitor Cells Joydip Kundu, Ph.D., Associate Research Scientist, Department of Chemical Engineering, Northeastern University Development of a biomimetic platform that is permissive to hrpc (human retinal progenitor cell) survival, differentiation, and integration within the host retina to restore vision. Decellularized retina (decell-retina) mimics the environment of retina, and thus we hypothesize will promote hrpc survival, attachment, migration, differentiation and integration into the host retina. We explored the prospect of decell-retina based substrates as delivery vehicle of hrpcs to subretinal space for retinal repair. 4:25 Closing Remarks Targeting Ocular Disorders 3

4 SPONSORSHIP, EXHIBIT OPPORTUNITIES + LEAD GENERATION CHI offers comprehensive sponsorship packages which include presentation opportunities, exhibit space, branding and networking with specific prospects. Sponsorship allows you to achieve your objectives before, during, and long after the event. Any sponsorship can be customized to meet your company s needs and budget. Signing on early will allow you to maximize exposure to qualified decision-makers. Podium Presentations Available Within the Main Agenda! Showcase your solutions to a guaranteed, highly-targeted audience. Package includes a 15 or 30-minute podium presentation within the scientific agenda, exhibit space, on-site branding and access to cooperative marketing efforts by CHI. Breakfast & Luncheon Presentations Opportunity includes a 30-minute podium presentation. Boxed lunches are delivered into the main session room, which guarantees audience attendance and participation. A limited number of presentations are available for sponsorship and they will sell out quickly. Sign on early to secure your talk! Invitation-Only VIP Dinner/Hospitality Suite Sponsors will select their top prospects from the conference pre-registration list for an evening of networking at the hotel or at a choice local venue. CHI will extend invitations and deliver prospects, helping you to make the most out of this invaluable opportunity. Evening will be customized according to sponsor s objectives i.e.: Looking for additional ways to drive leads to your sales team? One move can make all the difference! CHI s Lead Generation Programs will help you obtain more targeted, quality leads throughout the year. We will mine our database of 800,000+ life science professionals to your specific needs. We guarantee a minimum of 100 leads per program! Opportunities include: Whitepapers Web Symposia Custom Market Research Surveys Podcasts Advertising opportunities such as marketing and promotional s are also available. For sponsorship and exhibit information, please contact: Carolyn Benton Business Development Manager cbenton@healthtech.com Purely social Focus group Reception style Plated dinner with specific conversation focus Exhibit Exhibitors will enjoy facilitated networking opportunities with qualified delegates. Speak face-to-face with prospective clients and showcase your latest product, service, or solution. *Inquire about exhibit space, branding and more! MEDIA PARTNERS Official Media Partner: Lead Sponsoring Publications: Sponsoring Publications: Web Partner: 4 Targeting Ocular Disorders

5 HOTEL + TRAVEL INFORMATION CONFERENCE VENUE AND HOST HOTEL FLIGHT DISCOUNTS Westin Boston Waterfront Hotel 425 Summer St. Boston, MA Phone: Special discounts have been established with American Airlines for this conference. Discounted Room Rate: Discounted Cut-off Date: $269 s/d September 8, 2014 Please visit our conference website to make your reservations on-line, or call the hotel directly to reserve your sleeping accommodations. You will need to identify yourself as a Cambridge Healthtech Institute conference attendee to receive the discounted room rate with the host hotels. Reservations made after the cut-off date or after the group room block has been filled (whichever comes first) will be accepted on a space-and-rate-availability basis. Rooms are limited, so please book early. Call American Airlines and use Conference code 23H4BR Go to and enter Conference code 23H4BR in promotion discount box Contact our dedicated travel agent, Rona Meizler, at (617) or rona.meizler@protravelinc.com CAR RENTAL DISCOUNTS Special discount rentals have been established with Hertz for this conference. Call Hertz and use our Hertz Convention Number (CV): 04KL0005 Go to and use our Hertz Convention Number (CV): 04KL0005 TOP REASONS TO STAY AT THE WESTIN BOSTON WATERFRONT HOTEL: Just three miles from Boston s Logan International Airport Complimentary wireless internet access in guest rooms No Commute, since meeting takes place at hotel A short walk, bus, taxi or train ride to Boston s historic sites and family attractions Minutes from some of Boston s finest restaurants and shops Targeting Ocular Disorders 5

6 Targeting Ocular Disorders October 6-7, 2014 Westin Boston Waterfront Hotel Boston, MA Complimentary news delivered to your inbox Pricing and Registration Information Subscribe to New Bulletins or the Weekly Update Newsletter at Bio-ITWorld.com Clinical Trials to the Clinic, subscribe at ClinicalInformaticsNews.com CONFERENCE PRICING PREMIUM PACKAGE Commercial Academic, Government, (Includes access to Targeting Ocular Disorders, and a copy of Insight Pharma Reports Hospital-affiliated Ocular Disorders: Rising Therapeutics, Technologies, and Devices) Registrations after August 22 and on-site $2549 $1329 STANDARD PACKAGE Commercial Academic, Government, (Includes access to Targeting Ocular Disorders, excludes report) Hospital-affiliated Registrations after August 22 and on-site $1849 $829 CONFERENCE DISCOUNTS Poster Submission - Discount ($50 Off): Poster abstracts are due by September 5, Once your registration has been fully processed, we will send an containing a unique link allowing you to submit your poster abstract. If you do not receive your link within 5 business days, please contact jring@healthtech.com. *CHI reserves the right to publish your poster title and abstract in various marketing materials and products. REGISTER 3-4th IS FREE: Individuals must register for the same conference or conference combination and submit completed registration form together for discount to apply. Alumni Discount: Cambridge Healthtech Institute (CHI) appreciates your past participation in Targeting Ocular Disorders. As a result of the great loyalty you have shown us, we are pleased to extend to you the exclusive opportunity to save an additional 20% off the registration rate. Alumni and Register 3-4th is free discounts cannot be combined Group Discounts: Discounts are available for multiple attendees from the same organization. For more information on group rates contact David Cunningham at If you are unable to attend but would like to purchase the Targeting Ocular Disorders CD for $350 (plus shipping), please visit healthtech.com/targeting-ocular-disorders. Massachusetts delivery will include sales tax. How to Register: reg@healthtech.com P: or Toll-free in the U.S Please use keycode EYE F when registering A series of diverse reports designed to keep life science professionals informed of the salient trends in pharmaceutical technology, business, clinical development, and therapeutic disease markets. For a detailed list of reports, visit InsightPharmaReports.com, or contact Rose LaRaia, rlaraia@healthtech.com, Barnett is a recognized leader in clinical education, training, and reference guides for life science professionals involved in the drug development process. For more information, visit barnettinternational.com. ADDITIONAL REGISTRATION DETAILS Each registration includes all conference sessions, posters and exhibits, food functions, and access to the conference proceedings link. Handicapped Equal Access: In accordance with the ADA, Cambridge Healthtech Institute is pleased to arrange special accommodations for attendees with special needs. All requests for such assistance must be submitted in writing to CHI at least 30 days prior to the start of the meeting. To view our Substitutions/ Cancellations Policy, go to Video and or audio recording of any kind is prohibited onsite at all CHI events. Please use keycode EYE F when registering! Please refer to the Registration Code below: Cambridge Healthtech Institute 250 First Avenue, Suite 300, Needham, MA Fax: