ALOPECIA AREATA RESEARCH SUMMIT Forging the Future

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ALOPECIA AREATA RESEARCH SUMMIT Forging the Future

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ALOPECIA AREATA RESEARCH SUMMIT Forging the Future PRELIMINARY PROGRAM Tuesday, December 4 Wednesday, December 5, 2018 Lerner Hall, Columbia University, New York City, USA Summit Co-Chairs Jerry Shapiro, MD NYU School of Medicine Natasha A. Mesinkovska, MD, PhD UC Irvine School of Medicine Angela M. Christiano, PhD Honorary: Columbia University Medical Center Hosted By

TUESDAY, DECEMBER 4 7:30 AM 8:00 AM Hang All Posters All abstracts selected for oral presentations must also be presented as posters. 8:00 AM 8:30 AM BREAKFAST 8:30 AM 9:05 AM Opening Session Welcome Address NAAF President and CEO, Dory Kranz will welcome participants and Summit Co-Chairs, Drs. Jerry Shapiro, Natasha Mesinkovska and Angela Christiano will summarize meeting objectives. Opening Keynote Coping with Stress: Stem Cells in Injury and Inflammation Understanding how stem cells remain quiescent during times of minimal wear and tear, how these cells become mobilized during the cyclical bouts of hair growth and wound-repair, and how the normal process of stem cell activation goes awry in inflammatory disorders such as alopecia areata. 9:05 AM 10:35 AM Session 1 Setting the Stage Summary of Preceding Alopecia Areata Research Summits Summary of outcomes resulting from past research summits and overview of the current state of alopecia areata research. Genetics and Immunology of Alopecia Areata Current Treatment of Alopecia Areata Overview of options available to treat pediatric and adult alopecia areata, including the risks and benefits of current and evolving off-label treatment options with the understanding that there is currently no treatment approved by the FDA for this disease. Alopecia Areata: Autoimmune Disease or Hair Follicle Response Pattern to Immunological Damage? Pre-clinical Research in Vivo: Pros and Cons of the C3H/HeJ versus the Humanized Mouse Model 10:35 AM 10:45 AM BREAK In order to probe the efficacy of new immune-intervention strategies in alopecia areata, the field can now choose from two mutually complementary mouse models. 2 2 0 1 8 Alopecia Areata Re search Summ it

10:45 AM 12:45 PM Session 2 Clinical Research in Alopecia Areata: Investigator Insights r JAK Inhibitors for the Treatment of Alopecia Areata in the Pediatric Population Review of the available data regarding the use of JAK inhibitors for alopecia areata in children. Responsiveness of Eyebrows and Eyelashes to Tofacitinib in Patients with Severe AA While JAK inhibitors have emerged as treatment of alopecia areata, studies have focused on scalp hair growth. In this study, we evaluated the response of eyebrows and eyelashes to tofacitinib. Long-Term Treatment for Severe Alopecia Areata with Oral Tofacitinib Citrate Results from Cleveland Clinic's retrospective study indicate tofacitinib to be a safe and viable treatment option for patients with severe alopecia areata. Platelet-Rich Plasma in the Treatment of Alopecia Areata A comprehensive review of data on efficacy, safety and mechanism of action of platelet-rich plasma (PRP) injections in treating alopecia areata. In Vivo Imaging in Alopecia Areata Patients Using Multiphoton Microscopy Exploring the utility of multiphoton microscopy (MPM) as an adjunct to histological studies and a diagnostic tool for alopecia areata. Cytokine Targeted Treatments for Alopecia Areata Overview of the cytokine pathways activated in alopecia areata and their therapeutic targeting using different agents. Alopecia Areata: What We Can Learn from Advances in Other Inflammatory Skin Diseases A review advances made in psoriasis and in atopic dermatitis and how those can be applied to plans to find a safe and effective treatment for alopecia areata. 12:45 PM 1:45 PM LUNCH AND KEYNOTE Alopecia Areata: A Patient s Perspective A viewpoint of alopecia areata from a patient, researcher in the immunology field, and advocate for the alopecia areata patient community. 2 0 1 8 Alopecia Areata Re search Summ it 3

1:45 PM 2:45 PM Session 3 Patient Involvement in Clinical Development: Industry Panel 2:45 PM 4:35 PM Session 4 Clinical Outcome Assessments r Objective Outcome Measures: Collecting Meaningful Data on Alopecia Areata Overview of assessment methods for alopecia areata including the SALT and ALODEX scoring systems, response criteria and potential global score. Importance of Addressing Eyebrows in Alopecia Areata Alopecia Areata Patient-Reported Outcome Consortium Lessons from the International Dermatology Outcome Measures IDEOM's achievements in establishing domains for clinical research in both psoriasis and hidradenitis suppurativa, including collaborations with the AAD to establish simple outcome measures for clinical practice and registries, will be discussed. 4:35 PM 5:30 PM COCKTAILS, NETWORKING AND POSTER VIEWING RECEPTION 6:00 PM 7:30 PM DINNER AND NETWORKING 4 2 0 1 8 Alopecia Areata Re search Summ it

WEDNESDAY, DECEMBER 5 7:30 AM 8:00 AM BREAKFAST AND OPENING SESSION Introduction and Review of Day One Discussion A review of key topics discussed on the first day. 8:00 AM 8:45 AM Session 5 Late Breaking Abstracts 8:45 AM 10:00 AM Session 6 The Basics: New Frontiers Mechanism of Hair Growth Pattern Formation A description of how all hair follicles on the body coordinate their regenerative activities to achieve efficient hair renewal. The Role of Regulatory T cells in Alopecia Areata Exploring our current understanding of the role of regulatory T cells (Tregs) in the pathogenesis of alopecia areata and the therapeutic potential of Treg augmentation for this disease. Involvement of SSEA-Positive Myeloid Cells in Driving AA in the Mouse Model Novel induction of alopecia areata in C3H/HeJ mice shows a potential role of previously unrecognized endogenous SSEA-positive myeloid cells in driving inflammatory cascade and hair loss mechanisms. A Protective Role of MicroRNA-486 in Alopecia Areata 10:00 AM 10:10 AM BREAK The activities of many genes implicated in the control of skin and hair follicle homeostasis are regulated by mirnas, which represent a new regulatory layer controlling an execution of lineage-specific differentiation programs. This study identified mir-486 that exerts anti-apoptotic and protective roles against the collapse of hair follicle immune privilege in anagen phase. 10:10 AM 12:00 PM Session 7 Epidemiology and Burden of Disease Using Big Data to Unravel Uncommon Diseases Observations in uncommon diseases are limited by small cohorts and selected samples which restrict the ability to make important observations on disease burden, disease course, co-morbid associations, disease outcomes, and treatment outcomes. The purpose of this talk is to describe the application of big data in overcoming such limitations. 2 0 1 8 Alopecia Areata Re search Summ it 5

Rare Genetic Mutations Contribute to Alopecia Areata Etiology Autoimmune diseases arise from complex aberrant interactions between the immune system and an end organ such as the hair follicle in the case of alopecia areata. While genome-wide association studies of common genetic variants in alopecia areata have highlighted etiological contributions from specific immune cells and pathways, exome studies of rare variants in patients and family members are implicating components of the hair follicle extracellular matrix, suggesting a crucial point of communication between the hair follicle and the immune system. Prevalence of Alopecia Areata in the U.S.: Results from a Cross-Sectional Survey Study The epidemiology of alopecia areata in the United States is not currently known. his cross-sectional study of 45,000 patients establishes the current point prevalence of alopecia areata among the general population. Willingness to Pay and Quality of Life in Alopecia Areata Broadening Diversity in Alopecia Areata Clinical Trial Participants The FDA plans to prioritize improvements in the quality of demographics subgroup data collection, reporting and analysis, encourages greater participation of diverse patients, and supports the transparency of subgroup data. To this end, ways to recruit, engage, educate, and study those of diverse backgrounds to alopecia areata trials will be discussed. The NAAF Patient Registry: Driving Towards a New Understanding of Alopecia Areata 12:00 PM 12:45 PM LUNCH Through its partners, NAAF is establishing a new advanced bio-repository capability for its patient registry that will enable researchers to access high quality annotated specimens for research. New specimen storage, processing and analysis coupled to state-of-the-art information systems and analytics will be used to help researchers gain a deeper understanding of alopecia areata. 12:45 PM 1:45 PM Session 8 Group Breakouts 1:45 PM 2:00 PM BREAK 2:00 PM 3:00 PM Session 9 Breakout Reports Breakout Topic 1 Report Breakout Topic 2 Report Breakout Topic 3 Report Breakout Topic 4 Report 6 2 0 1 8 Alopecia Areata Re search Summ it

3:00 PM 4:00 PM Closing Session NIAMS Funding Opportunities Meeting Summary, Outcomes and Action Plan 4:00 PM CLOSE SUMMIT 4:00 PM 5:00 PM Dismantle All Posters Posters not removed by 5:00 PM on Wednesday will be discarded. 2 0 1 8 Alopecia Areata Re search Summ it 7

INDUSTRY PARTNERS We gratefully acknowledge the following industry partners for their support of the 2018 Alopecia Areata Research Summit. Silver- $50,000 & Up Bronze- $25,000 & Up Other Support