NEURO NATIONAL CLINICAL TRIAL TRAINING PROGRAM · The Clinical Research Unit (CRU) manages clinical...

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NEURO NATIONAL CLINICAL TRIAL

TRAINING PROGRAM

May 23 & 24, 2019

de Grandpré Communications Centre The Neuro

3801 University Street, Montreal, Quebec

Message

Biographies

Program

Organizing Committe

Table of Contents

NEURO NATIONAL CLINICAL TRIAL TRAINING PROGRAM

Welcome


From the Clinical Research Unit

Angela GengeDirector

Clinical Research UnitThe Neuro

Building on the success our previous programs, we hope that you enjoy this year’s social events, exciting talks given by our invited speakers, and interactive discussions held by staff of the CRU. The Clinical Research Unit (CRU) manages clinical trials within The Neuro, the largest specialized neuroscience research and clinical centre in Canada. Under the direction of Dr. Angela Genge, the 2018 Forbes Norris award winner, the CRU’s 40 person team offers services in grant writing and trial development, trial recruitment and coordination, finance, ethics, biological sample processing, nursing, and medical writing. The CRU staff are dedicated to producing quality work, and committed to patient care and safety. Since 1992, the CRU has conducted over 300 trials, including all of the pivotal MS trials. The CRU has run every phase of industry sponsored clinical trials (I – IV), as well as investigator initiated clinical trials, bio-banking initiatives and patient registries. The CRU now includes a CNS- focused Phase 1 unit, a growing expertise in rare diseases and is expanding its presence to partner clinics affiliated with the Neuro.


Biographies


Jinsy A. Andrews, MD, MSc is an Assistant Professor of Neurology, in the Division of Neuromuscular Medicine and currently serves as the Director of Neuromuscular Clinical Trials in the Department of Neurology at Columbia University. In addition to overseeing neuromuscular clinical trials, she cares for patients with neuromuscular disorders, primarily with amyotrophic lateral sclerosis (ALS). Prior to this position, she helped develop and direct a clinical trials unit for neurological diseases at Hospital for Special Care/University of Connecticut. There, she served as the Director of Clinical Trials and Co-director of the MDA/ALS

multidisciplinary clinic. More recently, Dr. Andrews was Senior Director of Clinical Research and Development and Head of Neuromuscular Therapeutics at Cytokinetics where she focused on developing investigational therapies for neuromuscular diseases including Spinal Muscular Atrophy (SMA) and ALS. She has experience in conducting human clinical trials in neuromuscular disorders from Phase 1 to Phase 3 from both the academic and industry settings. She currently serves on the Executive Committee of the Northeastern ALS Consortium (NEALS).

Dr. Andrews received her BS from Union College, her MSc in Biostatistics (Patient-Oriented Research) from Columbia University’s Mailman School of Public Health, and her MD from Albany Medical College. Dr. Andrews completed her residency in Neurology at the University of Connecticut and completed fellowship training in both Neuromuscular Disease and Clinical Neurophysiology at Columbia University. Dr. Andrews is board certified in Neurology, Neuromuscular Disease, and Electrodiagnostic Medicine.

Marie-Hélène Bolduc is the Director of Mission at Muscular Dystrophy Canada. She is responsible for provincial advocacy, drug patient submissions and the delivery of MDCS’s program and services. This includes MDC’s system navigation program that enables Service Specialists to work with individuals and their families as well as, health care professionals to navigate complicated systems to access the right resources to enhance the quality of life for people affected by intramuscular disorders.

Jinsy A. Andrews

Marie-Hélène Bolduc

Dr. Jim Callaway has over three decades of biopharmaceutical development experience, serving as CEO for several privately held biotech companies targeting CNS therapeutics. Dr. Callaway and the KalGene team are advancing a clinical candidate into clinical studies studying Alzheimer’s Disease leveraging NRC technologies by both disrupting the amyloid oligomer and blood-brain barrier transport to provide an exceptional product profile. Jim previously led the Elan Alzheimer’s Immunotherapy program introducing the first disease-modifying biologics into clinical. In addition, as ArmaGen CEO he brought from the lab bench and into clinical studies the first biologics engineered to

cross the blood-brain barrier. Dr. Callaway has filed and defended numerous NDAs and INDs during his career, including shepherding the approval of MyoBloc® and the production of Tysabri®. He previously had senior development roles at Bayer Pharmaceuticals, SmithKline Beecham, and Ingene after receiving his Ph.D. in Biological Chemistry from UCLA.

Thomas Durcan is an assistant Professor at The Neuro, McGill University. As an assistant professor at the Neuro and McGill University, his research focus is on applying patient-derived stem cells towards the development of phenotypic discovery assays and 3D mini-brain models for both neurodegenerative and neurodevelopmental disorders. As group leader of the iPSC platform at the Neuro, Tom Durcan oversees a team of 30 research staff and students and growing, committed to applying novel stem cell technology, combined with CRISPR genome editing, mini-brain models and new microfluidic technologies

towards elucidating the underlying causes of these complex disorders. Leading the SGC tissue platform, NeuroSGC; Tom oversees a team committed to assay development, in parallel with leading the establishment of NeuroCDRD, towards the automation of our open assays for small molecule screens. In addition, He is also part of antibody validation efforts between the Neuro, Oxford and the Karolinska to generate CRISPR KO cell-lines for validating commercial antibodies against high-value ALS targets. Combined with new approaches in the group towards building multiomic profiles on the patient-derived IPSC cells within the group, the long-term strategy over the coming years is to identify new personalized precision therapies that can be applied towards building clinical trials on a dish.


Jim Callaway

Thomas Durcan

Joshua Galanter is a Safety Science Associate Medical Director at Genentech, a member of the Roche group, where he works in the licensing and early clinical development safety group (LEAD). There he leads safety strategy for Phase I and II molecules in diverse clinical programs including ALS, rheumatoid arthritis, lupus, asthma and other allergic diseases. Prior to Genentech, he was an Assistant Professor in the Department of Medicine and the School of Pharmacy at UCSF. He received his medical degree from Harvard Medical School. He trained as a resident in Internal Medicine at UCLA and completed fellowships in Pulmonary and Critical Care Medicine and Clinical Pharmacology and a master’s

degree in Clinical Research at UCSF.

Having funded his PhD (Biophysics) as research assistant at Guys Hospital London, Ignazio Di Giovanna went on to work in the CRO and pharmaceutical industry since 1986. As a CRA, Ignazio spent over 12 years as site manager and monitoring for clinical trials in oncology, cardiology and neurology, and 10 years as Project Manager on oncology, neurology and neuromuscular degenerative studies. Ignazio has contributed to developing and promoting clinical research internationally; as a board member (and Chair) of the Institute of Clinical Research, and as President of the Clinical & Contract Research Association (UK’s national

CRO body). Ignazio has taken part in a number of research related initiatives with the UK’s Department of Health, Research and Development Departments, the Association of British Pharmaceutical Industry and various UK advisory groups, including Biotechnology & Pharmaceuticals Sector Advisory Group, advising and influencing the UK’s Trade and Industry Department on sector-related matters to help improve the UK’s research offering. Ignazio was owner manager of CCA Clinical Research from 1999 until its acquisition by Atlantic Research Group in Dec 2018, and is now ARG’s Vice-President of European Operations.


Joshua Galanter

Ignazio Di Giovanna

Marie Hirtle is a lawyer with graduate training in law and ethics. She specializes in health issues ranging from community-based health and social services, to tertiary and quaternary care, biomedical research and public health. As the current Manager of the Centre for Applied Ethics at the McGill University Health Centre (MUHC), she leads a team of ethics professionals who provide clinical, organizational and research ethics services to the MUHC community. Benefiting from 25 years experience on REBs, she is current Chair of the MUHC Research Ethics Board (REB) as well as of the Central REB of the Québec Minister of Health and Social services. She was recently appointed to the FRQ Working Group

on Artificial Intelligence and is a member of the Observatoir de l’IA.

Chris Kent has served as the CEO of ODS Medical since its’ founding in 2015. ODS Medical is a medical device company commercializing an exclusive Raman spectroscopy system for real-time, intra-operative tumor margin detection during surgical oncology procedures. Prior to ODS Medical, he worked with Dr. Kevin Petrecca, building a translational research program at The Neuro focused on working with industry partners to accelerate the discovery and pre-clinical development of a wide variety of small molecules and biologics for treatment of brain cancers.

He has published numerous peer-reviewed journal articles. Prior to his work at The Neuro, Chris was involved with a start-up that specialized in the application of stimuli-responsive polymer materials to a wide range of industries. As Director of Business Development and IP strategy, he developed the company’s business model, leveraging strategic industry collaborations in sectors such as packaging, textiles, energy and life sciences, into a growing patent portfolio, which through subsequent development and out-licensing deals grew the company’s revenue ten-fold.


Marie Hirtle

Chris Kent

Nadine Kolas is the policy lead for advanced cell therapies in Health Canada’s Biologics and Genetic Therapies Directorate. Prior to joining the Branch in 2017 she worked on the “Science Review” with an expert panel to review the federal ecosystem supporting fundamental research in Canada; and developed policy for knowledge translation and research governance for management of Health Canada’s regulatory research programs including chemicals, nanomaterials, Northern contaminants and air pollution. As a former researcher, she received her PhD in molecular genetics from Albert Einstein College of Medicine in New York City, and conducted post-doctoral work at the

Lunenfeld-Tanenbaum Research Institute in Toronto. With expertise including reproductive biology and early mechanisms of cancer development using mouse models and genomics approaches, her work has been published in journals including Science and Cell. Nadine has held fellowships from CIHR, The Terry Fox Research Institute, EIRR21, and won the 2008 Polanyi Award for medicine/physiology from the Ontario government.

Martine Lafrance, PhD, is the Manager, Program Design and Delivery Branch, Research Programs Portfolio, at the Canadian Institutes of Health Research. Since joining the Canadian Institutes of Health Research (CIHR) in 2005, Martine has managed the competition delivery of various open and strategic grant programs and initiatives, including the Project Grant program.

Before joining CIHR, Martine was a researcher at the Institute of Biological Sciences, National Research Council of Canada in Ottawa. She obtained her Ph.D. in Biomedical Sciences (Endocrinology) from the University of Montreal, and pursued postdoctoral studies in Endocrinology at Cornell University (New York) and the University of Ottawa.


Nadine Kolas

Martine Lafrance

Marie-Claude Marchand is based in Montreal from where she performs business development, market & technology advisory services with a focus on diagnostics for TVM Capital.

Marie-Claude is an experienced biotechnology executive and global commercial strategy leader with an extensive track record in large organizations as well as in start-up environments. She currently also lead Business development efforts as Vice-President for Optina Diagnostics, a start-up company developing retinal biomarkers with first indications in Alzheimer Disease.

During her many years at QIAGEN Marie-Claude has lead teams around the world establishing new markets and innovative technologies. One of the teams led by Marie-Claude, recognized the power of precision diagnostics very early and managed to enter into various Pharma partnerships in order to drive the development of dedicated biomarkers tests. These continue to offer safer, and more effective therapies for many patients all over the world.

Norman Putzki, based in New York City is Vice President Neuroscience Global Drug Development for Novartis Pharmaceuticals. After studying human medicine in Germany, Switzerland and the UK, Norman graduated with an MD and PhD from University of Essen, Germany. Norman’s background is in clinical neurology. During the course of his academic career in Neurology in various settings across several geographies, his focus was on headache disorders and inflammatory disorders of the central nervous system such as Multiple Sclerosis (MS). While his pre-clinical focus was on T regulatory lymphocytes’

role in autoimmunity, his groups also focused on outcomes and epidemiological research in MS. He led clinical and research groups in two academic centers before joining pharma industry in 2010.

In 2013, Norman joined Novartis and held various leadership positions in the global Neuroscience Development organization headquartered in Basel (Switzerland); very recently, he served as Head of the US medical Neuroscience organization (2016-2019).

He is author/co-author of > 50 peer reviewed publications and he has authored four books.

He is a fellow of the American Academy of Neurology.


Marie-Claude Marchand

Norman Putzki

Kristiana Salmon, CRC is the Team Leader for the ALS & Neuromuscular division of the MNI’s Clinical Research Unit. She has been involved with all of the major, international ALS trials over the past 6 years, often enabling the CRU to be the top global recruiter. Her other duties within the CRU include overseeing training and education of new staff, maintaining the site’s SOPs, and overseeing audit preparations. In addition to conducting the industry-sponsored trials, Kristiana has fostered an invaluable relationship with the MNI/H’s multidisciplinary ALS clinic. She is considered a member of the multidisciplinary team, and has encouraged research initiatives amongst its members.

Kristiana is also a contributing member of the ALS Untangled group and a member of the NEALS PEACe Committee. She has recently completed an Executive MBA at the John Molson School of Business, Concordia University.

Robert J. Schott, is a cardiologist who joined Lilly in April of 2014 as the launch leader for Evacetrapib. Rob is a Michigan native, and completed his undergraduate and medical studies in a combined program followed by 3 years of training in Internal Medicine at the University of Michigan, where he also took a Masters of Public Health degree in epidemiology and biostatistics. Rob became interested in basic heart disease research and pursued post-doctoral work in cardiovascular science at the University of Michigan in the Thoracic Surgery lab, followed by 2 years at the Max Planck Institute for Experimental Cardiology in Bad Nauheim, Germany. He returned to the US to train in invasive cardiology at

the Massachusetts General Hospital and did further research in cardiac molecular biology as a Burgher Fellow at Harvard University.

Rob practiced cardiology for many years in Northern California serving as a Department Chief and the Vice President of Medical Affairs at Sutter Medical Center in Sacramento California. He left clinical practice in 2010 to become the Medical Director at the Profil Institute, which specialized in early phase clinical research in diabetes and dyslipidemias. In 2012 he became the Vice President of Medical Affairs for diaDexus in San Francisco, a company which developed biomarkers for ischemic heart disease and heart failure. He was recruited from that position to Lilly in his current role.

Rob divides his time between Indianapolis and San Diego, where most of his free time is devoted to riding around a bicycle as well as recovering from injuries sustained after falling off his bicycle.


Kristiana Salmon

Robert J. Schott

Leonard H. van den Berg is a Professor of Neurology at the University Medical Center Utrecht and director of the Netherlands ALS Center. At the UMC Utrecht outpatient clinic for patients suspected of ALS or other motor neuron, 90% of ALS patients (>300 patients/year) in The Netherlands are diagnosed. Improved awareness of ALS through media and education resulted in a mean diagnostic delay from onset of symptoms of less than 8.5 months. ALS care has been organized through a Dutch network of ALS care providers, consisting of home care teams and 40 specialized multidisciplinary ALS teams in Rehabilitation Centres or Hospitals. He is the chairman of ENCALS

(European Network for the Cure of ALS), which is a Europe-wide network of >50 ALS Care and Research Centres. ENCALS fosters high standards of care and research collaborations, and holds a European ALS meeting annually. He founded TRICALS, a European ALS trial consortium of certified ALS centers, embedded in ENCALS; it provides multiple European sites with experienced certified (medical) staff and services, to accelerate the conduct of clinical research. TRICALS facilitates academic and industry initiatives to investigate the efficacy of potential compounds in ALS treatment. He established a research group focused on translational research into ALS to develop effective treatment for patients with ALS by delineating molecular pathways that initiate/drive motor neuron degeneration using data obtained from patients, transgenic and neuronal cell culture models (iPSC technology). For his role in ALS care and research he has received the ‘Sheila Essey Award’ from the American Academy of Neurology and the ‘Forbes Norris Award’ from the International Alliance of ALS/MND Associations.

Daria Wojtal is the Director of Research at Muscular Dystrophy Canada where she is working to develop MDC’s research portfolio including restructuring of research funding programs, researcher, regulatory and pharmaceutical stakeholder engagement, as well as leading knowledge translation initiatives aimed at helping patients understand new research and medical advances in the neuromuscular field. Prior to this she worked in the laboratory of Dr. Ronald Cohn at The Hospital for Sick Children in Toronto and her PhD thesis focuses on developing CRISPR gene editing technology as a potential treatment for neuromuscular disorders.


Leonard H. van den Berg

Daria Wojtol

Dr. Angela Genge has been the Director of the Clinical Research Unit at The Neuro since 2004, and Director of the ALS clinic and research program at the Neuro since 1998. The clinical research unit develops and executes over 75 clinical trials in various neurological conditions with an emphasis on ALS, MS, Neuromuscular diseases and primary brain tumors. Sponsors include the entire range of groups including large pharma, industry sponsored trials, small biotech, federally funded trials and private donors. She currently manages a group of 26 coordinators, nurses and support staff, in addition to supporting

the principal investigators of the department of neurology and neurosurgery of McGill University. On the clinical side, Dr. Genge is the director of the ALS clinical program, with a team of 12 professionals. Current goals are the development of investigator-initiated trials in ALS and an ALS Biobank in collaboration with Dr. Guy Rouleau.

Nationally, Dr. Genge is the Chair of the ALS working group of the CNDR registry and a member of the Governance and Administrative committee of the new CAN-NMD network. At the Neuro, she is a member of the Neuromuscular Research Group. She is an executive member of the CALS network and a board member of both ALS Canada and ALS Quebec.

Internationally, Dr. Genge is an active member of NEALS and WALS, 2 US based ALS clinical trial networks. She is a sought-after consultant in clinical trial design for ALS and other orphan neuromuscular diseases. She is particularly interested in working with biotech companies working with new approaches to these currently untreatable conditions. In her role as Medical Director of the CRU, she has also developed a presence in the multiple sclerosis clinical trial community as she has participated in the landmark trials dating back to 1992.

Dr. Genge earned her medical degree from the Memorial University of Newfoundland. She completed her Canadian and American certifications in Internal Medicine and Neurology prior to completing a fellowship in neuromuscular diseases at McGill University. She also completed an executive MBA program through Lancaster University.

She has received numerous awards throughout her career, most recently the DIVA of distinction award and the Governor General Diamond Jubilee award. Dr. Genge is also committed to community initiatives to help ALS and NM patients and their families as well as other groups in need.

Angela Genge



Program

May 22, 2019

6pm– 10pm Welcome Event – Pointe-à-Callière Museum, 350 rue Place Royale, Montreal

Cocktail – Dinatoire + Museum tour

May 23, 2019

8:00Continental Breakfast

8:30Welcoming Remarks

Angela Genge, MD, FRCPDirector, Clinical Research Unit, The Neuro, Canada

8:45Development Path of the Raman Probe Technology

Christopher Kent, PhDPresident & CEO, ODS Medical, Canada

9:15Modelling Disorders of the Brain through Stem Cells

Thomas Durcan, PhDAssistant Professor, The Neuro, Canada

9:45A Tale of Two Worlds: Investment & Entrepreneurship in Diagnostics

Marie-Claude Marchand, BSc, MBASenior Advisor, TVM Life Science Management Inc., Canada

10:15Panel - Q & A

10:30 Refreshment break

10:45Consortiums & Clinical Trials Leonard van den Berg, MD

Professor of Neurology, Director Netherlands ALS Center, Chair of ENCALS University Medical Center Utrecht, Netherlands

11:15The Chorus Model: The Benefits of Therapeutic Cross-Pollination Design

Robert Schott, MD, MPH, FAACChief Medical Officer, Lilly – Chorus, USA

11:45

An Entrepreneur’s Guide to Addressing Unmet Medical NeedsJim Callaway, PhD

CEO, KalGene Pharmaceuticals, Canada

12:15 Panel – Q & A

12:30 Lunch

13:30Medical Monitoring in Clinical Trials

Jinsy Andrews, MD, MSc.Assistant Professor of Neurology, Columbia University, USA

14:00Drug Safety Monitoring

Joshua Galanter, MD, MASSafety Science Associated Medical Director, LEAD/Early Development Safety

Genentech Inc., USA

14:30 Ethics of AI & Gene Therapy Research in Medicine

Marie Hirtle, LLB, LLMManager Center of Applied Ethics – MUHC, Canada

15:00 Panel Q & A

15:15 Refreshment break

15:30CIHR Initiatives That Support Drug Development

Martine Lafrance, PhDManager of Program Design & Delivery Branch, Research Programs Portfolio

Canadian Institutes of Health Research (CIHR), Canada

16:00 Patients before Costs: A Balanced Approach to Drug Development

Daria Wojtal, PhDDirector of Research, Muscular Dystrophy Canada

&Marie-Hélène Bolduc

Director of Mission, Muscular Dystrophy Canada

16:30 Regulation of Regenerative Medicines in Canada –

Overview of the Regulatory Framework for Cell and Gene TherapiesNadine Kolas, MD

Senior Policy Analyst, Policy & International Collaboration, Health Canada

17:00 Panel Q & A

17:15 Closing Remarks & Discussion

Dr. Angela Genge

18:00 Dinner – Joiea Sociale

656 Wellington Street, Montréal

May 24 , 2019

8:30 Continental Breakfast

9:00Welcoming Remarks


9:15Drug Development in the Digital Age – A Perspective on Neuroscience

Norman Putzki, MD, PhDVice President Global Drug Development, Novartis Pharma AG, USA

9:45CRO-Sponsor Relationships

Ignazio Di Giovanna, PhDVice President, European Operations, Atlantic Research Group

10:15New Tech in Trials

(via video conference) Jesse Cedarbaum, MDVice President, Neurology Early Clinical Development, Biogen Idec, USA

10:45Panel Q & A

11:00

Refreshment break

11:15Phase 1 Units in Neuroscience


&Kristiana Salmon, BSc, EMBA

Assistant Manager, Clinical Research Unit, The Neuro, and Hospital, Canada

12:30Closing Remarks

Angela Genge

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