Piper Jaffray GenomeRx Symposium
M AY 2 0 – 2 1 , 2 0 1 5 I N N E W Y O R K C I T Y
Piper Jaffray GenomeRx Symposium M AY 2 0 – 2 1 , 2 0 1 5 I N N E W Y O R K C I T Y
P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
Event Map. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 3 Agenda. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4 Participating Companies. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 8 Participant Biographies . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10 Piper Jaffray Investment Research Biographies. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 15 Notes . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 18
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
Event Map The New York Palace, Fourth Floor
Holmes II
1 3
2
Holmes I
Apartment ↓
Kennedy I
4
Women’s Restroom
↑
Elevator
Kennedy II
Men’s Restroom
5 Kitchen
Foyer
The Apartment South
Elevator
↳
The Apartment Courtyard
1
Track
2
Coat Check
3
Registration
4
Food & Beverage
5
Reception
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
Agenda (as of May 13, 2015) 8:00 a.m.
Registration and Continental Breakfast
8:30 – 9:10 a.m.
Gene Editing: Tales of TALENs, Feelings on Fingers
Cellectis S.A. (CLLS), Julien Valton, Project Leader
Precision BioSciences, Inc. (private), Matthew Kane, CEO
Sangamo BioSciences, Inc. (SGMO), Edward Lanphier, President & CEO
Transposagen Biopharmaceuticals, Inc./ Poseida Therapeutics, Inc. (private), Eric Ostertag, CEO
9:10 – 9:50 a.m.
Cas-9: CRISPR is Heating Up
Caribou Biosciences, Inc. (private), Andrew May, Chief Scientific Officer
CRISPR Therapeutics AG (private), Rodger Novak, CEO
Intellia Therapeutics Inc. (private), Nessan Bermingham, CEO
Massachusetts Institute of Technology, Ann Ran, Ph.D.
Transposagen Biopharmaceuticals, Inc./ Poseida Therapeutics, Inc. (private), Eric Ostertag, CEO
9:50 – 10:20 a.m.
Investor Panel
Brookside Capital, LLC, Daniel Krizek, CFA, CAIA
Deerfield Management Company, L.P., Jean Kim, MBA
Venrock Partners Management, LLC, Bong Koh, M.D., MBA
10:20 – 10:25 a.m.
Break
10:25 – 11:10 a.m.
CAR-T/TCR: Engineering Lethal Lymphocytes
Adaptimmune Therapeutics plc. (ADAP), James Noble, CEO
Bellicum Pharmaceuticals, Inc. (BLCM), Kevin Slawin, M.D., CMO & CTO
Cellectis S.A. (CLLS), Julien Valton, Project Leader
Kite Pharma, Inc. (KITE), Jeffrey Wiezorek, VP, Clinical Development
Lion Biotechnologies, Inc. (LBIO), Elma Hawkins, President & CEO
Transposagen Biopharmaceuticals, Inc./ Poseida Therapeutics, Inc. (private), Eric Ostertag, CEO
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
11:10 – 11:50 a.m.
Beyond T-Cells: Cancer Killing with Killer Cells
Bellicum Pharmaceuticals, Inc. (BLCM), Kevin Slawin, M.D., CMO & CTO
Formula Pharmaceuticals, Inc. (private), Maurits Geerlings, M.D., MBA, CEO
Unum Therapeutics, Inc. (private), Charles Wilson, Ph.D., President & CEO
11:50 a.m. – 12:20 p.m.
Cellular Therapies: Toolkits and Platforms
Formula Pharmaceuticals, Inc. (private), Maurits Geerlings, M.D., MBA, CEO
Lion Biotechnologies, Inc. (LBIO), Elma Hawkins, President & CEO
MaxCyte, Inc. (private), Douglas Doerfler, President & CEO
Medgenics, Inc. (MDGN), Garry Neil, CSO
Miltenyi Biotec Inc. (private), Boro Dropulić, GM & Chief Science Officer
Miltenyi Biotec Inc. (private), Kai Pinkernell, Global Head Clinical Business
12:20 p.m.
Lunch
12:30 – 1:00 p.m.
Gene Therapy for Muscle Disorders: Heavy Lifting for Success
Audentes Therapeutics, Inc. (private), John Gray, VP, R&D
Bamboo Therapeutics, Inc. (private), Jude Samulski, Ph.D.
Milo Biotechnology LLC (private), Al Hawkins, CEO
Nationwide Children’s Hospital, Inc. (private), Matthew McFarland, Dir, Research Technology & Commercialization
uniQure N.V. (QURE), Jörn Aldag, CEO
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
1:00 – 1:45 p.m.
Gene Therapy for Neuro/CNS Disorders: Thinking Through the Opportunity
AveXis, Inc. (private), Brian Kaspar, Ph.D., VP, R&D
Bamboo Therapeutics, Inc. (private), Larry Altstiel, M.D., Ph.D.
REGENX Biosciences, LLC (private), Ken Mills, President & CEO
uniQure N.V. (QURE), Jörn Aldag, CEO
Voyager Therapeutics, Inc. (private), Steven Paul, President & CEO
1:45 – 2:30 p.m.
Gene Therapy Vector Evolution: Mixing, Matching, Moving Ahead
4D Molecular Therapeutics (private), David Kirn, Co-Chairman & CEO
Applied Genetic Technologies Corp (AGTC), Susan Washer, President & CEO
Avalanche Biotechnologies Inc. (AAVL), Mehdi Gasmi, VP, Pharmceutical Development
Bamboo Therapeutics, Inc. (private), Aravind Asokan, Ph.D.
Switchback Bio (private), Joseph Glorioso, Ph.D.
University of Pennsylvania James Wilson, M.D., Ph.D.
2:30 – 3:15 p.m.
Gene Therapy: Ramping Up for Success
Audentes Therapeutics, Inc. (private), Matthew Patterson, President & CEO
Dimension Therapeutics, Inc. (private), Annalisa Jenkins, CEO
Kadmon Corporation, LLC (private), Zandy Forbes, CEO
REGENX Biosciences, LLC (private), Ken Mills, President & CEO
Voyager Therapeutics, Inc. (private), Steven Paul, President & CEO
3:15 p.m.
Break
3:20 – 4:00 p.m.
Gene Therapy: Prep for Launch
Access Bio, Inc. (private), Joy Cavagnaro, Ph.D.
bluebird bio, Inc. (BLUE), Nick Leschly, CEO
Spark Therapeutics, Inc. (ONCE), Jeff Marrazzo, Co-Founder & CEO
uniQure N.V. (QURE), Jörn Aldag, CEO
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
4:00 – 4:45 p.m.
Gene Therapy: Aligning the Industry’s Interests
bluebird bio, Inc. (BLUE), Nick Leschly, CEO
REGENX Biosciences, LLC (private), Ken Mills, President & CEO
Sangamo BioSciences Inc (SGMO), Edward Lanphier, President & CEO
University of North Carolina, Jude Samulski, Ph.D.
University of Pennsylvania James Wilson, M.D., Ph.D.
4:45 – 5:30 p.m.
Gene Therapy: The Bigger Players Step Up
Biogen (BIIB), Oliver Danos, Ph.D.
BioMarin Pharmaceutical Inc. (BMRN), Barrie Carter, VP, Vextor Biology
Bayer HealthCare Pharmaceuticals Inc. (private), Hans Duerr, VP, Global Head Hematology
University of North Carolina, Jude Samulski, Ph.D.
University of Pennsylvania, James Wilson, M.D., Ph.D.
5:30-7:30 p.m.
Reception
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
Participating Companies (as of May 13, 2015) Company Name
Ticker
4D Molecular Therapeutics
Private
Access Bio, Inc.
Private
Adaptimmune Therapeutics plc.
ADAP
Applied Genetic Technologies Corporation
AGTC
Audentes Therapeutics, Inc.
Private
Avalanche Biotechnologies, Inc.
AAVL
AveXis, Inc.
Private
Bamboo Therapeutics, Inc.
Private
Bayer HealthCare Pharmaceuticals Inc.
Private
Bellicum Pharmaceuticals, Inc.
BLCM
Biogen BIIB BioMarin Pharmaceutical Inc. bluebird bio, Inc.
BMRN BLUE
Brookside Capital, LLC
Private
Caribou Biosciences, Inc.
Private
Cellectis S.A.
CLLS
CRISPR Therapeutics AG
Private
Deerfield Management Company, L.P.
Private
Dimension Therapeutics, Inc.
Private
Formula Pharmaceuticals, Inc.
Private
Intellia Therapeutics Inc.
Private
Kadmon Corporation, LLC
Private
Kite Pharma, Inc.
KITE
Lion Biotechnologies, Inc.
LBIO
Massachusetts Institute of Technology
Private
MaxCyte, Inc.
Private
Medgenics, Inc.
MDGN
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
Company Name
Ticker
Milo Biotechnology LLC
Private
Miltenyi Biotec Inc.
Private
Nationwide Children’s Hospital, Inc.
Private
Precision BioSciences, Inc
Private
REGENX Biosciences, LLC
Private
Sangamo BioSciences, Inc.
SGMO
Spark Therapeutics, Inc.
ONCE
Switchback Bio
Private
Transposagen Biopharmaceuticals, Inc./ Poseida Therapeutics, Inc
Private
uniQure N.V.
QURE
University of North Carolina
Private
University of Pennsylvania
Private
Unum Therapeutics, Inc.
Private
Venrock Partners Management, LLC
Private
Voyager Therapeutics, Inc.
Private
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
Participant Biographies Joy Cavagnaro, Ph.D. President Access BIO Joy Cavagnaro, Ph.D., is the president of Access BIO, where she consults on development strategies and therapeutic safety for novel drug, biologics and device combinations. Her career spans academia, contract research organization industries, biotechnology industries and government. During her tenure at the Center for Biologics Evaluation and Research (CBER) within the FDA, Cavagnaro was appointed to the Senior Biomedical Research Service (SBRS) and served as the FDA’s safety topic lead and rapporteur for ICH S6. She was the first to advocate the case-by-case, science-based approach to preclinical safety evaluation, which is consistent with traditional principles but embraces new practices, is questions-based, data-driven and targeted based on product attributes. The approach is designed to obtain maximum information with judicious animal use. Prior to joining the FDA, Cavagnaro was principal study director for biotechnology products at Covance. Cavagnaro serves various roles on multiple scientific advisory boards, including founder, past chair and current ex officio member of the leadership committee of BioSafe; member of the Translational Science and Product Development Committee of the American Society of Gene & Cell Therapy; advisor and member of the Grants Working Group of the California Institute of Regenerative Medicine; and chair of Center for Research in Regulated Industries. In 2011, Cavagnaro received The Society of Toxicology’s Biotechnology Specialty Section first Career Achievement Award. She consults and lectures internationally on translation and risk assessment of novel therapies. She has co-authored numerous white papers and chapters related to various aspects of preclinical safety assessment. The book she edited, Preclinical Safety Evaluation of Biopharmaceuticals: A Science-Based Approach to Facilitating Clinical Trials, published by John Wiley & Sons, is commonly referred to as the “BioBible.”
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
Matthew McFarland, R.Ph., Ph.D. Director, Office of Technology Commercialization The Research Institute at Nationwide Children’s Hospital Matthew McFarland, R.Ph., Ph.D., joined Nationwide Children’s Hospital as the director of the office of technology commercialization in the spring of 2012. As director, he works closely with Nationwide Children’s Hospital’s faculty and staff to identify intellectual property with commercial potential and to facilitate the transfer of new technologies to outside partners, ultimately for the benefit and enhancement of pediatric care. McFarland has a diverse background in technology transfer, technology valuation and licensing, academic research and pharmacy practice. Prior to joining Nationwide Children’s Hospital, McFarland was the associate director of commercialization in the office of technology commercialization at the Purdue Research Foundation. McFarland has authored several articles for peer-reviewed journals, including Molecular Pharmacology, Journal of Biological Chemistry and Medical Innovation & Business. He also has received the JenkinsKnevel Award for Excellence in Research and the Albert and Anna Kienly Award for Excellence in Teaching from the College of Pharmacy at Purdue University. McFarland received a bachelor’s degree in pharmacy from Ohio Northern University as well as a doctorate in medicinal chemistry and molecular pharmacology from Purdue University. He also completed a postdoctoral research fellowship in translational genetics and pharmacogenomics of neuropsychiatric disorders at the Institute of Psychiatric Research – Indiana University Medical School.
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F. Ann Ran Postdoctoral Fellow Lab of Feng Zhang at the Broad Institute of MIT and Harvard F. Ann Ran is a postdoctoral fellow in the lab of Feng Zhang at the Broad Institute of MIT and Harvard in Cambridge, Massachusetts. Ran received her doctorate in biochemistry from Harvard in 2014. Prior to that, she earned a master’s degree from Harvard and a bachelor’s degree in biology, with honors, from Yale. In Zhang’s lab, Ran and colleagues pioneered the use of the CRISPR/ Cas9 technology for genome editing in mammalian cells. Efficient and versatile, this technology has since emerged as one of the most important scientific breakthroughs in recent years with broad applications for basic science, medicine and biotechnology. Ran has presented her work at numerous workshops and conferences in North America, Europe and Asia. She is currently a junior fellow at the Harvard Society of Fellows, a small group of researchers selected for their extraordinary scholarly potential.
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
R. Jude Samulski, Ph.D. Director of Gene Therapy University of North Carolina at Chapel Hill R. Jude Samulski, Ph.D., has served as director of gene therapy at the University of North Carolina at Chapel Hill since 1993. Having received his doctorate in medical microbiology and immunology from the University of Florida, followed by postdoctoral training at Princeton, Samulski initiated his independent lab at the University of Pittsburgh, where his research has primarily focused on the study of the human non-pathogenic parvovirus adeno-associated virus (AAV). He has long pioneered methodologies for using viruses to deliver genes effectively and safely to various targets in the body, including the brain, lungs, heart and muscle. As a graduate student at the University of Florida, Samulski’s thesis project was initial cloning and development of AAV as a vector for therapeutic genes. This work eventually led to development of AAV type-2 as a viral vector, which has been used for gene therapy trials in cystic fibrosis, hemophilia, Parkinson’s disease, retinal disorders and in several other settings, including the first clinical trial of gene therapy for muscular dystrophy in the United States. Development of self-complementary AAV vectors as well as first use of chimeric AAV vectors in clinical setting has continued to provide and advanced the gene transfer community in treatment of orphan diseases with these novel therapeutics. Based on these accomplishments, Samulski has received various awards, including Hemophilia Research of the Year, the first recipient of American Society of Cell and Gene Therapy (ASCGT) Outstanding Achievement Award, and the recent Gene Therapy Pioneer Award. In addition to serving on many relevant committees (e.g. RAC, FDA advisory board), Samulski served as president of ASCGT and initiated the first U.S./China gene therapy symposium. In addition to his work at the University of North Carolina at Chapel Hill and abroad, Samulski has founded the Chapel Hill Project, a non-profit organization focused on facilitating gene therapy clinical trials for orphan diseases.
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
James M. Wilson, M.D., Ph.D. Director, Gene Therapy Program Director, Orphan Disease Center Professor, Pathology & Laboratory Medicine Perelman School of Medicine, University of Pennsylvania James M. Wilson, M.D., Ph.D., is a professor in the Perelman School of Medicine at the University of Pennsylvania, where he has led an effort to develop the field of gene therapy. Dr. Wilson began his work in gene therapy during his graduate studies at the University of Michigan over 30 years ago. He then moved to Boston to do a residency in internal medicine at the Massachusetts General Hospital and continued his work in gene therapy at MIT. Dr. Wilson has been at the nexus of this emerging therapeutic area from its birth. He initially focused on clinical translation of existing gene transfer technologies but soon redirected his efforts to the development of second- and third-generation gene transfer platforms, the first of which was licensed to a biotechnology company he started that resulted in the first, and, at this point only, commercially approved gene therapy in the Western Hemisphere. More recently, his laboratory discovered a family of viruses from primates that could be engineered to be very effective gene transfer vehicles. These so-called “vectors” have become the technology platform of choice and have set the stage for the recent resurgence of the field of gene therapy. Dr. Wilson has also been active in facilitating the commercial development of these new gene therapy platforms through the establishment of several biotechnology companies. He is leading a national dialogue on the challenges of commercializing these potentially lifesaving treatments due to the disruptive nature they will have on traditional business models. Throughout his career, the focus of Dr. Wilson’s research has been rare inherited diseases, ranging from cystic fibrosis to dyslipidemias to a variety of metabolic disorders. Dr. Wilson has published more than 550 papers, reviews, commentaries and editorials in the peer-reviewed literature and is an inventor on over 117 patents. Dr. Wilson was the 2014 recipient of the William Osler Patient Oriented Research Award of the University of Pennsylvania and received the 2015 Scientific Achievement Award from Pennsylvania Bio.
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Piper Jaffray Investment Research Biographies Charles Duncan, Ph.D. Sr. Research Analyst Piper Jaffray & Co. +1 212 284-5025 charles.c.duncan@pjc.com Charles Duncan, Ph.D., joined Piper Jaffray in 2012 as a managing director and senior research analyst focused on small- and mid-cap emerging growth biotechnology companies. Duncan brings more than 20 years of sell-side experience during which he has covered a broad range of biopharma companies, most recently serving as an analyst at JMP Securities since 2002. Previously, Duncan covered the sector at Dresdner Kleinwort Wasserstein, Vector Healthcare Group - Prudential Securities, Tucker Anthony Cleary Gull and Chatfield Dean & Co. Duncan has been recognized by industry sources, including the Financial Times and StarMine Analyst Awards, as being among the best analysts for his fundamental and timely analysis. Duncan began his career as a manager of clinical development at Global Drug Development, Inc., a pharmaceutical development consulting firm, and he also launched InfusionVision Medical, a venture-backed start-up medical device company. He is a graduate of the University of Wisconsin–Madison and holds a doctorate in pharmaceutical sciences with a concentration in neuropharmacology from the University of Colorado.
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
Joshua Schimmer, M.D. Sr. Research Analyst Piper Jaffray & Co. +1 212 284-9322 joshua.e.schimmer@pjc.com Josh Schimmer is a managing director and senior research analyst focused on biotechnology companies. Schimmer has been on the Street for more than 10 years. Prior to joining Piper Jaffray in 2013, he worked in equity research at Lazard Capital Markets, Leerink Swann and Cowen and Company. In addition, he spent time on the buy-side at Davidson Kempner as part of the healthcare investing team. Schimmer holds a doctorate from the University of Toronto, where he specialized in internal medicine and sub-specialized in rheumatology. He also holds a Master of Business Administration degree from Harvard Business School.
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
Edward Tenthoff Sr. Research Analyst Piper Jaffray & Co. +1 212 284-9403 edward.a.tenthoff@pjc.com Ted Tenthoff is a managing director and senior research analyst at Piper Jaffray, focused on the drug discovery sector of the biotech industry. In April 2013, Tenthoff moderated a cardiovascular medicine panel at the International Adult Stem Cell Conference at the Vatican. He was ranked No. 2 stock picker in biotechnology by the 2012 Wall Street Journal “Best on the Street” survey and was ranked No. 1 stock picker for the life science tools and services sector in the 2006 StarMine Analyst Awards. Prior to joining Piper Jaffray in 2003, he covered the genomics sector at Robertson Stephens & Company and was a medical technology analyst at Lehman Brothers. Tenthoff received a bachelor’s degree in international relations from the University of Pennsylvania.
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Notes
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P IP ER J A FFR AY GEN O MER X S Y MP O SI U M M AY 2 0 – 21, 2 015
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