CNS Therapeutics
e-Xchange
(a virtual meeting)

Biomarkers

Time

Titles and Bullets

Facilitator

8:30am – 9:00am

Opening Address & Keynote:

9:05am – 10:05am

Strengthening and building more reliable biomarkers

The challenges of CNS biomarkers for supporting drug development for brain diseases: imaging and molecular markers
New opportunities for imaging biomarkers: MRI, PET, OCT
New technologies for developing molecular biomarkers for brain diseases: extracellular vesicles (exosomes) in blood and CSF

Pablo Villoslada

Founder & Chief Scientific Officer
Bionure

Pablo Villoslada is the Founder and Chief Scientific Officer at Bionure. He is a neurologist with more than 20 years of experience in translational medicine on biomarkers, and new therapeutics for neurological diseases (academic, biotech and big pharma).

He has worked at Genentech as Senior Medical Director, designing and monitoring Phase II and III trials in neuroscience and immunology. He has significant experience in drug development, diagnostics and medical devices, digital health, biomarkers and AI applied to health. Pablo is a serial entrepreneur: founder and advisor of several biotechs and medical devices start-ups in the field of CNS and Ophthalmology (Bionure, Health Engineering, QMenta, SpiralTx, Attune).

He has held several academic positions including Adjunct Professor of Neurology at University of California, San Francisco where he also obtained his scientific training; Professor in Neurobiology at University of Barcelona, Spain and Associate Professor of Neurology at University of Navarra, Spain.

Pablo has a PhD in Neuroscience from University Autonomous of Barcelona and an M.D. from the University of Santiago de Compostela in Spain.

10:10am – 10:40am

1-2-1 Meetings / Networking Break

10:45am – 11:45am

AI driven biomarker and imaging analysis

What are the current best CNS therapeutics biomarkers, and how many degrees of separation is there between readout and known clinical manifestation? Can we learn from the success stories?
How can we as a field collect data to support direct links between biomarkers and outcome?
What technologies are currently believable in accelerating CNS therapeutics?

Thomas Westerling-Bui

Director, Scientific Strategy & Business Development
Aiforia

Thomas received his PhD in cancer biology, focusing one cell cycle and transcription regulation, from the University of Helsinki Medical School in Finland. He completed a post-doctoral EMBO fellowship at the Dana Farber Cancer Institute in Boston, researching the genomics of breast and prostate cancer. Tom joined Aiforia to advance the new potential of cancer biology, pathology, big data, and artificial intelligence. At Aiforia Tom works with advanced Pharma and Clinical Projects Customers to bring the newest tools and solutions to the forefront of diagnosis, and therapeutics research and Development.

11:50am – 12:20pm

1-2-1 Meetings / Networking Break

12:20pm – 1:00pm

Lunch Break

1:00pm – 2:00pm

Working with increasingly sophisticated digital biomarkers

Understanding how big data sets can be used for better biomarker data
Exploring the use of voice and facial data
Discussing the regulatory pathways for digital biomarkers

Jane Tiller

Chief Medical Officer
BlackThorn Therapeutics

Jane Tiller has extensive clinical psychiatry and drug development experience. Prior to joining BlackThorn, she held several leadership positions at Bristol Myers Squibb (BMS), most recently as Head of European Markets Australia and Canada, where she oversaw all medical functions. She also served as VP Global Medical for Neuroscience, Virology & Immunoscience and as full development team lead for the Alzheimer’s program. Prior to joining BMS, she held positions of increasing responsibility at Cephalon. Prior to joining industry Jane was responsible for clinical services across several London teaching hospitals and served as a Consultant Psychiatrist (Attending) and Hon. Senior Lecturer. She is a Fellow of the Royal College of Psychiatrists, UK. Jane obtained her medical degree from Glasgow University, an executive MBA from Drexel University and a MPhil in Organizational Dynamics from the University of Pennsylvania. She trained at the Maudsley Hospital and Institute of Psychiatry in London and spent two years as visiting Faculty at Duke University.

2:05pm – 2:35pm

1-2-1 Meetings

2:35pm – 3:05pm

1-2-1 Meetings

3:10pm – 4:10pm

Exploring new biomarkers for neurodegeneration, particularly NfL

Challenge of biomarker development for neuro – lack of availability human tissue to assess
Imaging, CSF sampling challenges
Sensitivity of blood sampling generally lacking, correlation to CNS difficult to evaluate
Example of success across indications – Neurofilament light chain shows promise as potential prognostic and predictive marker – more widespread study dependent of sensitive techniques to measure in circulation

Sarah Harris

Senior Director, Translational Medicine
Bristol-Myers Squibb

Sarah Harris currently leads TM activities at Science Park, SD site for BMS, in immunology and neurology disease areas. She has been the translational medicine and bioanalytical (PK/ADA) lead for numerous compounds in oncology, vaccines, immunology and neurology therapeutic areas. She has supported compounds from IND through post-marketing including Prevnar 13 (Wyeth/Pfizer), and Rituxan, Zevalin, Tysabri (Idec/Biogen Idec), Zeposia (BMS). Sarah has led the US pharma division at a small CRO (Intertek) for 2 years supporting PK, ADA, and biomarker assay development, validation and sample testing for pharma companies in GLP environment. She has served as scientific advisor for multiple non-profit family foundations with focus on genetic neurodegenerative disorders in children (cln1 and cln6 Batten’s Disease, PTHS).

Sarah has a Ph.D. in Immunology from University of Rochester School of Medicine and Dentistry, Rochester, NY.

4:15pm – 4:45pm

Closing Keynote & Address:

Genomics in Drug Discovery

Time

Titles and Bullets

Facilitator

8:30am – 9:00am

Opening Address & Keynote:

9:05am – 10:05am

How can we leverage oncology innovation to advance CNS drug development

Gain an understanding on why oncology drug development has succeeded compared to CNS drug development and what can be translated.
Focus on CNS biomarkers which are rarely obvious before clinical development with hypotheses often developed during Phase 2 and what can we do to accelerate these
How has CNS drug discovery utilized findings related to genes, the genome, and proteome to advance biomarker research?
Where have we been able to successfully utilize predictive, prognostic, or pharmacogenomic biomarkers in CNS drug development?
Discuss the most exciting opportunities where oncology development has laid a path for CNS development

Glenn Morrisson

Vice President, Global Clinical Development
Zogenix

Glenn Morrison, a neurobiologist by training, is VP, Global Clinical Development at Zogenix.

He has more than 20 year’s experience in drug development spanning multiple therapeutic areas, with a specific focus on the CNS.

His areas of interest include rare epilepsy disorders, neurodegeneration, and cognitive impairment.

Glenn has a MSc and PhD from the University of Toronto.

10:10am – 10:40am

1-2-1 Meetings

10:45am – 11:45am

Biomarker topic Biomarker needs in Neurological diseases

Where is the biggest needs for biomarkers in neurological settings? Predictive, Prognostic, Response therapy, others?
What are the current modes of biomarker discovery in neurological diseases and what are their benefits and challenges?
Which one is a more suitable matrix, CSF, or plasma?
Where do we want to be in 10 years and how do we get there as a community? (Collaborations, Research Network, Consortia)

Narges Rashidi

Director of Application Sciences, North America
Olink Proteomics

Narges Rashidi received her Ph.D. in Systems Biology from the University of Southern California. She completed a postdoc at Imperial College London where she studied stem cells in the context of Leukemia. She then moved to Boston to take over the management of the mass cytometry core lab at MGH. Capitalizing on her expertise in proteomics and imaging, she was recruited by Fluidigm as an Application Scientist, playing a critical role in the support and deployment of their Helios and Hyperion Imaging system. She joined Olink in 2017 and has been heading the Scientific Applications team in North America since January.

11:50am – 12:20pm

1-2-1 Meetings

12:20pm – 1:00pm

Lunch Break

1:00pm – 2:00pm

The gap between animal models and patient outcome

What is the best role of animal models in research? therapeutic efficacy? safety? pharmacology?
What are lessons we can learn from disease models that have translated to clinical patients?
What lessons have we learned from animal models that have failed to translate to clinical patients?
What lessons have we learned from safety issues discovered in patients that were not present in animal models?

Orion Keifer

Vice President, Discovery & Translation
CODA Biotherapeutics

Orion Keifer is the Vice President of Discovery and Translation at CODA Biotherapeutics Inc. He is a neuroscientist with extensive translational training in small and large animal models, focused on small molecule, cell therapy, and gene therapies for neurological diseases. Before joining CODA, he worked as a consultant translational scientist and surgeon for several different biotechnology companies helping to design and execute proof-of-concept and IND-enabling preclinical studies for neurological disorders. Orion earned an M.D. and a Ph.D. in neuroscience from Emory University, an M.S. in brain and cognitive neurosciences and B.S. degrees in biomedical engineering, applied psychology and applied biology with Highest Honors from Georgia Tech. He completed his post-doctoral training in the Department of Neurosurgery at Emory University.

2:05pm – 2:35pm

1-2-1 Meetings

2:35pm – 3:05pm

1-2-1 Meetings

3:10pm – 4:10pm

Challenges and opportunities in therapeutic development for rare diseases

Discussion of clinical design challenges (recruitment, lack of natural history data, disease heterogeneity)
Potential ways to address these challenges (multidomain responder indices and composite endpoints, how to leverage preclinical data)
Review of regulatory opportunities (accelerated approval based on biomarkers, Phase 2/3 trial designs)

Steve Maricich

Chief Medical Officer
Allievex

Steve Maricich is the Chief Medical Officer of Allievex Pharmaceuticals, a clinical stage company that develops therapies for rare childhood neurological disorders. Prior to his move to Allievex, Steve led the tralesinidase alfa enzyme replacement therapy clinical development program for the treatment of Sanfilippo Syndrome Type B and contributed to other preclinical and clinical stage programs at BioMarin Pharmaceutical.

Prior to working at BioMarin, Steve enjoyed an 8-year academic career on the Pediatrics and Child Neurology faculty at Baylor/Texas Children’s, Case Western/Rainbow Babies and the University of Pittsburgh/UPMC Children’s Hospital of Pittsburgh, three world-renowned children’s hospitals. He was an NIH- and privately-funded primary investigator who has authored over 35 scientific articles, reviews and book chapters. Steve received the Child Neurology Society’s Young Investigator award and is internationally recognized for his lab’s research in auditory and somatosensory development.

Steve is a Board-certified child neurologist who obtained his MD and PhD (neuroscience) degrees from Case Western Reserve University in Cleveland, Ohio.

4:15pm – 5:15pm

Closing Keynote & Address:

Clinical Trials

Time

Titles and Bullets

Facilitator

8:30am – 9:00am

Opening Address & Keynote:

9:05am – 10:05am

Choosing your patient population – how early should we go?

Patient selection in clinical trials for primary or secondary prevention or in manifest disease
Clinical and biomarker endpoints for use in primary or secondary prevention or in manifest disease
Leveraging regulatory guidance on trial design for the different types of clinical trials

Enchi Liu

Vice President, Global Program Team Leader
Annexon BioSciences

Enchi Liu has extensive experience in drug development, including therapies targeting CNS disorders (mood, pain, sleep), neurodegenerative diseases (Alzheimer’s disease, Amyotrophic Lateral Sclerosis, Huntington’s disease, Parkinson’s disease), and rare diseases (AL and ATTR amyloidosis, neuronopathic lysosomal storage disorders). Her expertise extends to implementation of biomarkers (CSF, blood, imaging, pharmacogenomics) for translational research and to support development candidate registration. Prior to Annexon, she served as VP Head of Clinical Development and Translational Biomarkers at E-Scape Bio, Program Team Leader for a rare disease program at Prothena Biosciences, Alzheimer’s disease (AD) Biomarker Team Leader at Janssen Alzheimer Immunotherapy and Janssen Research and Development (Johnson and Johnson), and Senior Director responsible for leading the AD clinical development and operations teams at Elan Pharmaceuticals.

Enchi received her Ph.D. in Neurobiology and Behavior from Cornell University where she conducted research on cellular and molecular biology of neurotransmitter receptors. She completed post-doctoral neurogenetics research on CNS reorganization of Drosophila melanogaster during metamorphosis at the University of Arizona.

10:10am – 10:40am

1-2-1 Meetings

11:50am – 12:20pm

1-2-1 Meetings

12:20pm – 1:00pm

Networking Lunch

1:00pm – 2:00pm

What tools are available to accurately diagnose early disease?

Can AI be used to help in the diagnosis of patient with early disease and subsequently, the selection process?
Patient genome or biomarker profiling – how can these tools be used to identify suitable patient populations

Pamela Garzone

Chief Medical Officer
Calibr

Pamela Garzone is a respected pharmaceutical executive and currently serves as Chief Medical Officer of Calibr, a division of Scripps Research. In her role as Chief Medical Officer, she leverages drug development expertise in the therapeutic areas of oncology, hematology, immunology, neuroscience, cardiovascular, and infectious disease to strategically guide Calibr to achieve its mission. Prior to joining Calibr, she was Vice President, Group Asset Team Lead at Pfizer, serving on multiple leadership teams in an early oncology clinical research department. Pamela received the Pittsburgh School of Pharmacy Distinguished Alumni Award in 2019.

She has comprehensive experience providing trusted leadership on boards and committees for a variety of organizations, including outstanding achievements as National Sponsorship Chair of Women in BioScience. Pamela has been profiled in Clinical Leader and served as a member of its Advisory Board. She currently is on the scientific advisory board of Temple Therapeutics, a private clinical stage biotechnology company.

She earned a Ph.D. in Clinical Science and M.Sc. in Pharmacy from the University of Pittsburgh and a B.Sc. in Pharmacy from Purdue University.

2:05pm – 2:35pm

1-2-1 Meetings

2:35pm – 3:05pm

1-2-1 Meetings

3:10pm – 4:10pm

Strategies for early clinical trials: selection of subjects and outcome measures

Exploring benefits of jumping straight to patients for Phase I trials
Where to use a combined Ph I/PhII trial in patients
Secondary measures that can make or break clinical trials
Biomarkers – to validate or not to validate

Beth Hoffman

Chief Executive Officer
Origami Therapeutics

Beth Hoffman, Ph.D. is Founder, President and Chief Executive Officer at Origami Therapeutics where she is creating a new approach to treat neurodegenerative diseases. Prior to Origami, Beth was the Vice President, Discovery Biology for Vertex Pharmaceuticals where she made significant contributions to the launch of first-in-class drugs for Cystic Fibrosis (CF) that created a paradigm shift in the treatment of CF by focusing on the underlying cause of the disease to modulate protein folding and conformation. Prior to Vertex, Beth was a Research & Development executive at Amgen and Eli Lilly. She has led and overseen programs from target identification to Phase II proof-of-concept, with major contributions to 30 assets that have advanced to the clinic in CF, neurology, pain and psychiatry.

Beth serves on the Board of Directors for the San Diego Chapter of the Huntington’s Disease Society of America (HDSA). She received an A.B. in Molecular Biology from Wellesley College, a Ph.D. in Cell Biology from the Johns Hopkins University and was Chief of the Molecular Pharmacology Unit at the National Institute of Mental Health, Bethesda, MD.

4:15pm – 5:15pm

Closing Keynote & Address:

Managing Regulatory Pathways

Time

Titles and Bullets

Facilitator

8:30am – 9:00am

Opening Address & Keynote:

9:05am – 10:05am

Regulation – Fine turning gene expression in the brain for neurological diseases

Discussing gene protein regulation technology, including new innovations for improving specificity, precision and efficiency
Provide specific examples of defined therapeutic applications including the generation of ZFP-TFs suitable for use in neurodegenerative disorders

Amy Pooler

Senior Director, Neurology
Sangamo Therapeutics

Amy Pooler is Vice President of Neuroscience at Sangamo Therapeutics, a genomics medicine company based in San Francisco, California area. At Sangamo she is leading the effort to bring novel, gene therapy approaches to the clinic for CNS disorders. She obtained her PhD from the Massachusetts Institute of Technology, for research into regulation of amyloid formation. She moved to University of Oxford as a Blaschko European Visiting Research Fellow in the MRC Anatomical Neuropharmacology Unit before joining the Institute of Psychiatry at King’s College London. She obtained a fellowship from Alzheimer’s Research UK to investigate mechanisms of neuronal dysfunction in Alzheimer’s disease. She spent a year at Massachusetts General Hospital and Harvard Medical School exploring synaptic degeneration in dementia before joining Nestlé Research in Lausanne, Switzerland as a Brain Health Specialist.

10:10am – 10:40am

1-2-1 Meetings

11:50am – 12:20pm

1-2-1 Meetings

12:20pm – 1:00pm

Lunch Break

Targeted Therapies

Time

Titles and Bullets

Facilitator

8:30am – 9:00am

Opening Address & Keynote:

10:10am – 10:40am

1-2-1 Meetings

10:45pm – 11:45pm

Gene therapy for rare neurological disorders

Overview of gene therapy candidates in rare neurological disorders
What are the bioanalytical and biomarker considerations for GT trials?
What are the non-clinical studies needed to support an IND?

Jim McNally

Chief Scientific Officer
BioAgilytix

Jim McNally has an extensive background in bioanalytical assay development and program leadership spanning nearly 20 years working in the pharmaceutical and biotechnology industry.Prior to joining BioAgilytix, Dr. McNally was Executive Director at a large pharmaceutical company, where he lead a team of scientists to develop a portfolio of assays to support development of gene-based therapeutic candidates throughout their lifecycle. He has previously held roles at CRISPR Therapeutics, Genzyme, Pfizer, EMD Serono, and Shire which have given him broad experience in the development of large molecule, gene therapy, and cell therapy biotherapeutics.

11:50am – 12:20pm

1-2-1 Meetings

12:20pm – 1:00pm

Lunch Break

1:00pm – 2:00pm

Where is the disruption? Leveraging AI and Machine Learning in clinical development

Why has AI and Machine Learning not had greater adoption in Clinical Development to-date?
How has the COVID-19 pandemic changed how we leverage technology in conducting clinical trials?

Michael Ropacki

Vice President, Clinical & Product Development
Oryzon

Michael Ropacki is the Vice President of Clinical and Product Development and US Site Head for Oryzon Genomics, a public clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need. In addition, he serves as the Founder and President of Strategic Global Research & Development (SG R&D) based out of San Francisco that collaborates with sponsors developing and executing Clinical Development Plans to maximize meaningful and productive regulatory interactions, as well as increase probability of technical and regulatory success. Michael also serves as Co-Chair of a Scientific Advisory Group for the Innovative Medicines Initiative-European Prevention of Alzheimer’s Dementia (IMI-EPAD) program and as a National Institute of Health (NIH) advisor. He is a member of the Critical Path Institute’s Critical Path for Alzheimer’s Disease (CPAD) and digital Drug Development Tool (dDDT) Team. Previously, he was Co-Chair of the CAMD Preclinical Cognitive Outcome Assessment (COA) team working with the Food and Drug Administration’s Drug Development Tool, COA Qualification Program. Michael has authored or co-authored of dozens of published manuscripts and abstracts and was an Assistant Clinical Professor of Neurology at the Loma Linda University School of Medicine.

2:05pm –2:35pm

1-2-1 Meetings

2:35pm – 3:05pm

1-2-1 Meetings

3:10pm – 4:10pm

Novel targets and therapeutic approaches in neuroinflammation

Exploring novel targets in neuroinflammation
Identifying early signs of neuroinflammation in neurodegenerative disorders
Discussing biomarkers and imaging agents to track neuroinflammation
Analysing the role of innate immunity in neuroinflammation
Overcoming delivery challenges to address neuroinflammation

Edward Spack

Co-Founder & Head of Strategic Projects
Therini Bio

Edward Spack is a cellular immunologist focused on neuroinflammation with over 25 years of biotech translational experience, including preclinical development of drug candidates for multiple sclerosis, nosocomial infection, and biodefense. Most recently he co-founded Therini Bio Inc. (formerly MedaRed Inc. The company is developing a lead monoclonal antibody that inhibits fibrin-induced neuroinflammation in neurodegenerative disorders including Alzheimer’s Disease (AD) and Multiple Sclerosis (MS).

Edward currently chairs an NIA U01 AD Drug Development Review Panel and is also a member of the NIH Small Business Review on Drug Discovery for Aging, Neuropsychiatric and Neurological Disorders (SBIR) panel. He is also a faculty member for the NINDS and ASENT supported Training in Neurotherapeutics Discovery and Development course and a member of the Scientific Advisory Committee for the Alzheimer’s Drug Discovery Foundation Annual Drug Discovery for Neurodegeneration Conference. Edward received his doctoral degree from The Johns Hopkins University and conducted postdoctoral studies at Stanford University.

4:15pm – 5:15pm

Closing Keynote & Address:

CNS Therapeuticse-Xchange (a virtual meeting)

Biomarkers

Partners

CNS Therapeutics
e-Xchange
(a virtual meeting)