CNS Therapeutics Xchange
West Coast
San FranCisco 2021
September 22nd
Welcome to hubXchange’s West Coast CNS Therapeutics Xchange 2021, bringing together executives from pharma and biotech to address and find solutions to the key issues faced in developing CNS therapies.
Discussion topics will cover Novel Targets, Preclinical, Clinical Studies, Advanced Therapies and Drug Delivery.
Take advantage of this unique highly interactive meeting format designed for maximum engagement, collaboration and networking with your peers.
Novel Targets
- The Decentralized Landscape – Considering regulatory and operational features
- The Post-COVID-19 Landscape – Exploring risk mitigation, contingency, and AI strategies
- The Therapeutic Landscape – The advent of digital therapeutics at the expense of traditional drug therapy
Head, Neurology and Regenerative Medicine Center for Excellence
Biorasi
As head of Biorasi’s Neurology and Regenerative Medicine Centers of Excellence, Dr. El Hokayem oversees clinical projects related to neurological indications and therapies. His unique expertise in the basic, translational, and clinical research spectrum provides critical insights at strategic, operational, and managerial levels. He is an expert clinical trial decentralizer and an early adopter of digital technologies and digital therapeutics.
With over twenty years’ experience in both academia and clinical research, Dr. El Hokayem has focused his research on rare neurological diseases. He currently serves on the Scientific Advisory Board of the Foundation for Angelman Syndrome Therapeutics (FAST). Dr. El Hokayem has published over twenty peer reviewed papers and received multiple awards from international foundations and entities.
Dr. El Hokayem received his Ph.D. from University of Miami, Miller School of Medicine, in Biochemistry and Molecular Biology. He continued his training with post-doctoral work at the University’s Hussman Institute for Human Genomics.
9:05 – 10:05am
- What are the future exciting drug targets for pain, neurology and psychiatry?
- Discuss the current status of our existing “hot” CNS drug targets
- Explore the barriers to investigating a truly novel target
- Should we re-explore “failed” drug targets of the past
Vice President, Neuroscience
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.
10:50 – 11:50am
- Differences in sex with regards to therapeutic development
- Similarities of porcine skin, innervation and vascularization to humans
- Shortcomings of rodent models and the impact on success in CNS drug development
- Cost as a factor in choice of porcine models
Chief Scientific Officer
MD Biosciences
Sigal Meilin, PhD, is a graduate of Bar Ilan University in Tel Aviv, Israel. Prior to taking the helm as Chief Scientific Officer of MD Biosciences Innovalora, Ltd. in 2004, Sigal headed the preclinical program at an early-stage company. At MD Biosciences, Sigal established the full preclinical research program with well-known as well as novel rodent models. The research capabilities were expanded with new insight and understanding, eventually encompassing all aspects of in vitro testing, cell-based assays and electrophysiology. In vivo research capabilities were augmented with porcine models which have proven to be a new standard and milestone in translational development. As Chief Scientific Officer, Sigal Meilin has the privilege of seeing many novel therapeutic classes, many different pathways and administration routes, giving a unique perspective on development trends, successes and failures.
1-2-1 Meetings / Networking Break
1:25 – 2:25pm
Triaging Your Drug Targets to Find the Most Relevant Lead
- How to triage potential targets based on the most validated human biology and genetics evidence
- Explore the different approaches to defining standards for excluding drug targets
- Explore how community research experience and confidence in novel targets can de-risk development
- Discuss the risks of a research community-based approach to investigating new targets
President & Chief Executive Office
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.
3:45 – 4:45pm
Neuroinflammation- Identifying Biomarkers and Drug Targets
- Weighing up the evidence- is neuroinflammation cause of neurodegeneration or the symptoms?
- What are biomarkers faithfully recapitulate the CNS state and are easy to measure in the patient?
- What characteristics should biomarkers have to allow for accurate disease state tracking in patients?
- Discuss the current developments of drug targets for neuroinflammation
- How to effectively use biomarkers for patient identification, patient subgroups characterization and patient stratification?
Vice President, Translational Medicine and Nonclinical Development
Cerecin
Dr Morimoto has nearly 25 years of industry experience in leading project teams in the development of innovative medicines, providing guidance in the design and execution of preclinical, clinical and regulatory strategies with a therapeutic focus in Parkinson’s, Alzheimer’s and frontotemporal dementias. Previously, Bruce held leadership roles at Alkahest, Celerion and Allon Therapeutics, and works closely with the Michael J Fox Foundation, chairing one of their scientific review panels. He is an advisor to several biotech companies, contract research and conference organizations.
Bruce started his career on the faculty in the Chemistry Department at Purdue University where his independent research focused on neuronal signal transduction. Bruce earned his doctorate in biochemistry from UCLA and completed a postdoctoral fellowship at the University of California Berkeley.
Preclinical
- The Decentralized Landscape – Considering regulatory and operational features
- The Post-COVID-19 Landscape – Exploring risk mitigation, contingency, and AI strategies
- The Therapeutic Landscape – The advent of digital therapeutics at the expense of traditional drug therapy
Head, Neurology and Regenerative Medicine Center for Excellence
Biorasi
As head of Biorasi’s Neurology and Regenerative Medicine Centers of Excellence, Dr. El Hokayem oversees clinical projects related to neurological indications and therapies. His unique expertise in the basic, translational, and clinical research spectrum provides critical insights at strategic, operational, and managerial levels. He is an expert clinical trial decentralizer and an early adopter of digital technologies and digital therapeutics.
With over twenty years’ experience in both academia and clinical research, Dr. El Hokayem has focused his research on rare neurological diseases. He currently serves on the Scientific Advisory Board of the Foundation for Angelman Syndrome Therapeutics (FAST). Dr. El Hokayem has published over twenty peer reviewed papers and received multiple awards from international foundations and entities.
Dr. El Hokayem received his Ph.D. from University of Miami, Miller School of Medicine, in Biochemistry and Molecular Biology. He continued his training with post-doctoral work at the University’s Hussman Institute for Human Genomics.
Next Generation CNS Biomarkers Discovery – Current Opportunities with Multi-Omics, Patient Data and AI/ML
- How can we qualify novel biomarkers and their clinical relevance for use in early phase clinical trials?
- What are the advantages and disadvantages of current biomarker discovery platforms?
- What data are necessary to ensure that novel biomarkers have good clinical translatability?
Senior Director, Clinical Development and Translational Medicine
Asceneuron
Dr. Ryan Schubert is the Director of Clinical Development and Translational Medicine at Asceneuron. He is responsible for clinical strategy, medical oversight, operational support, and biomarkers for Asceneuron’s pipeline.
Dr. Schubert received his M.D. from the Stanford University School of Medicine and completed an Internal Medicine Internship and Neurology Residency at the University of California, San Francisco. Afterwards, he became a Post-Doctoral Fellow with laboratory and clinical responsibilities in the UCSF Division of Neuroinflammation and Glial Cell Biology. Ryan is a patent inventor, the author of over ten peer-reviewed publications, and holds a B.S. in Biology from the University of Southern California.
- Discuss current approaches to successfully identify and validate novel targets using genomics, genome engineering & iPS cells
- Modeling neurodegenerative disease at scale with precision engineering in iPS cells – fact or fantasy?
- Patient-derived iPSCs or Control iPSCs – A better starting point for disease models?
- High throughput neuronal differentiation – Addressing a downstream bottleneck
Vice President Commercial Development
Synthego
Abhi Saharia, Ph.D., is the VP of Commercial Development at Synthego. Over the past 12 years, Abhi has successfully developed and commercialized several innovative technologies with applications in research & drug discovery, ranging from RNAi, Zinc Finger Nucleases, cell-based assays to CRISPR-based cell engineering platforms. Abhi received his Ph.D. from Washington University in St Louis.
1-2-1 Meetings / Networking Break
- How can our current existing animal models be repurposed to have better clinical translatability?
- Can we achieve next-generation animal models by shifting focus towards disease mechanism instead of disease biology?
- We need models that represent the earliest stages of disease progression- how can this be achieved?
- What measures from the animal models are translatable to humans?
- How can we develop better animal models and endpoints by back-translating clinical findings?
- How can multi-omics and patient data be utilized to better pinpoint the mechanism of disease progression?
Senior Director, Translational Sciences
Alkahest
Dr. S. Sakura Minami received her PhD in Neuroscience from Georgetown University, where she used in vitro and in vivo models to study multiple mechanisms underlying Alzheimer’s disease. She did her postdoctoral work at the Gladstone Institutes of Neurological Disease where she focused on the role of microglia in neurodegenerative disorders such as frontotemporal dementia and Alzheimer’s disease. She is currently Senior Director of Translational Sciences at Alkahest, a subsidiary of Grifols, where she leads a team of scientists to take preclinical findings from discovery research and translate mechanistic understandings into measurable biomarkers in clinical trials for diseases of aging.
3:45 – 4:45pm
Preclinical Development Strategies to De-Risk Clinical Translation
- How much confidence can you gain in the biology of a target using preclinical studies
- What value do genetics and data from human tissues bring
- What are the relevant human and clinical endpoints that are equivalent to your preclinical measurements?
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.
Clinical Studies
Registration
- The Decentralized Landscape – Considering regulatory and operational features
- The Post-COVID-19 Landscape – Exploring risk mitigation, contingency, and AI strategies
- The Therapeutic Landscape – The advent of digital therapeutics at the expense of traditional drug therapy
Head, Neurology and Regenerative Medicine Center for Excellence
Biorasi
As head of Biorasi’s Neurology and Regenerative Medicine Centers of Excellence, Dr. El Hokayem oversees clinical projects related to neurological indications and therapies. His unique expertise in the basic, translational, and clinical research spectrum provides critical insights at strategic, operational, and managerial levels. He is an expert clinical trial decentralizer and an early adopter of digital technologies and digital therapeutics.
With over twenty years’ experience in both academia and clinical research, Dr. El Hokayem has focused his research on rare neurological diseases. He currently serves on the Scientific Advisory Board of the Foundation for Angelman Syndrome Therapeutics (FAST). Dr. El Hokayem has published over twenty peer reviewed papers and received multiple awards from international foundations and entities.
Dr. El Hokayem received his Ph.D. from University of Miami, Miller School of Medicine, in Biochemistry and Molecular Biology. He continued his training with post-doctoral work at the University’s Hussman Institute for Human Genomics.
Best Approaches to Patient Stratification
- How to de-risk clinical development by ensuring your program’s trial patient population composition is correct
- How to effectively utilize biomarkers, multi-omics profiling and patient data to rationally define patient subgroups from heterogeneous patient populations?
- What are the current possibilities of AI and machine learning in identifying treatment responders and predicting disease progression?
Director, Clinical Development
Zogenix
Peter St. Wecker, Ph.D. is a Director of Clinical Science for Zogenix, a global pharmaceutical company developing therapies for central nervous system disorders that address specific clinical needs for people living with orphan diseases. Prior to Zogenix, Peter was a Senior Manager in Clinical Operations at Theravance, where he served as cross-functional project team lead for large Phase II and III US and International clinical studies.
Starting with a background in academic research, he has over 25 years of experience in CRO, service provider, biotech, and pharma company study management, protocol development, vendor selection, budgeting, contract negotiation, site monitoring, quality management, and oversight.
Peter received a B.A. in chemistry from Earlham College and a Ph.D. in neurophysiology from the University of North Carolina at Chapel Hill.
- Are existing, approved COVID-19 treatments efficient in treating COVID-19-related CNS disorders?
- Are past/current COVID-19 clinical trials taking CNS symptoms into consideration in the protocol/endpoints design?
- Is the industry planning future trials specifically targeting COVID-19-related CNS disorders in the post-COVID-19 era?
Head, Neurology and Regenerative Medicine Center for Excellence
Biorasi
As head of Biorasi’s Neurology and Regenerative Medicine Centers of Excellence, Dr. El Hokayem oversees clinical projects related to neurological indications and therapies. His unique expertise in the basic, translational, and clinical research spectrum provides critical insights at strategic, operational, and managerial levels. He is an expert clinical trial decentralizer and an early adopter of digital technologies and digital therapeutics.
With over twenty years’ experience in both academia and clinical research, Dr. El Hokayem has focused his research on rare neurological diseases. He currently serves on the Scientific Advisory Board of the Foundation for Angelman Syndrome Therapeutics (FAST). Dr. El Hokayem has published over twenty peer reviewed papers and received multiple awards from international foundations and entities.
Dr. El Hokayem received his Ph.D. from University of Miami, Miller School of Medicine, in Biochemistry and Molecular Biology. He continued his training with post-doctoral work at the University’s Hussman Institute for Human Genomics.
1-2-1 Meetings / Networking Break
- Discuss and review the clinical trial process disruptions bought on by the COVID-19 pandemic. What are the short and long term changes to conducting clinical trials?
- What are the best practices for home and remote patient monitoring?
- How can we maximize patient compliance whilst also minimizing patient burden?
- Discuss the regulator’s acceptance of clinical data captured from non-clinical settings
- How can you ensure effective collaboration between sponsors and clinical site in a virtual setting?
Senior Vice President
Alector
Sam Jackson, MD, MBA, is the Senior Vice President of Clinical Development at Alector where he works on therapies in development for FTD, Alzheimer’s Disease and other neurodegenerative conditions. Sam is a board-certified emergency physician with fellowship training in Medical Toxicology. Sam started his industry career at Amgen, moving to positions of growing responsibility at Genentech and Dynavax. He then served as the Chief Medical Officer of Alkahest where he designed and initiated four phase 2 trials in neurological and ophthalmologic diseases. Sam received his joint MD and MBA degrees from the University of Pennsylvania where he was a 21st Century Scholar. He lives in the San Francisco Bay Area where he makes a valiant attempt to keep up with his two teenage daughters.
1-2-1 Meetings / Networking Break
Afternoon Refreshments
1-2-1 Meetings / Networking Break
3:45 – 4:45pm
Selecting Clinical Endpoints and Assessing Clinical Outcomes
- How does one assess on-target effects in the CNS using imaging, fluid biomarkers and objective functional measures? What are the relevance of these measures to disease?
- Which digital biomarkers align with meaningful clinical endpoints?
- What are regulatory perspectives on the incorporation of objective biomarkers in the context of clinical endpoints?
- What are the best methods for tackling variability in clinical endpoints? Are there opportunities here for unique clinical designs, wearables, in home monitoring, AI or other approaches?
Chief Medical Officer
Allievex
Steve is the chief medical officer of Allievex Corporation, a clinical-stage pharmaceutical company developing therapies for Sanfilippo syndrome type B and other pediatric neurodegenerative disorders. He is a board-certified child neurologist and clinical development scientist with experience in rare disease therapeutic development and specific expertise in enzyme replacement and gene therapy. Steve previously worked in clinical development at BioMarin Pharmaceutical and has served on the Pediatrics and Child Neurology faculty at three world-renowned children’s hospitals. He was an NIH- and privately-funded basic science primary investigator who is internationally recognized for research in neural development. Steve is a recipient of the Child Neurology Society Philip R. Dodge Young Investigator Award for outstanding early career research.
Advanced Therapies
- The Decentralized Landscape – Considering regulatory and operational features
- The Post-COVID-19 Landscape – Exploring risk mitigation, contingency, and AI strategies
- The Therapeutic Landscape – The advent of digital therapeutics at the expense of traditional drug therapy
Head, Neurology and Regenerative Medicine Center for Excellence
Biorasi
As head of Biorasi’s Neurology and Regenerative Medicine Centers of Excellence, Dr. El Hokayem oversees clinical projects related to neurological indications and therapies. His unique expertise in the basic, translational, and clinical research spectrum provides critical insights at strategic, operational, and managerial levels. He is an expert clinical trial decentralizer and an early adopter of digital technologies and digital therapeutics.
With over twenty years’ experience in both academia and clinical research, Dr. El Hokayem has focused his research on rare neurological diseases. He currently serves on the Scientific Advisory Board of the Foundation for Angelman Syndrome Therapeutics (FAST). Dr. El Hokayem has published over twenty peer reviewed papers and received multiple awards from international foundations and entities.
Dr. El Hokayem received his Ph.D. from University of Miami, Miller School of Medicine, in Biochemistry and Molecular Biology. He continued his training with post-doctoral work at the University’s Hussman Institute for Human Genomics.
CNS Cell Therapies- Current Opportunities and Limitations
- Discuss the latest targets for curative cell therapies
•Discuss the promise of current pipelines of cell therapy candidates - Contrast the approaches for hereditary and acquired diseases
- Explore the ethics around the permanent modification to patients with CNS advanced therapies
Chief Medical Officer
Aspen Neuroscience
Dr. Edward Wirth completed the M.D./Ph.D. program at the University of Florida (UF) in 1994. He elected to remain at UF to conduct postdoctoral research, and subsequently joined the faculty there in 1996. From 1997 to 2002, Dr. Wirth led the UF team that performed the first human embryonic spinal cord transplant in the United States. This pilot study demonstrated the feasibility and safety of implanting embryonic spinal cord cells into patients with post-traumatic syringomyelia (a complication of spinal cord injury). From 2002 to 2004, Dr. Wirth held academic appointments at RushPresbyterian St. Luke’s Medical Center and at the University of Chicago. From 2004-2011, he served as Medical Director for Regenerative Medicine at Geron Corporation, where he led the world’s first clinical trial of a human embryonic stem cell (hESC)-derived product. He subsequently served as Chief Science Officer at InVivo Therapeutics from 2011-2012.
In 2013 he joined several former Geron colleagues at Asterias Biotherapeutics, which had acquired the stem cell assets of Geron. He served as Asterias’ Chief Translational Officer from 2013-2015 and Chief Medical Officer from 2015-2019. At Asterias he led the expansion and completion of the former Geron clinical trial, which demonstrated the initial clinical safety and activity of OPC1 (formerly GRNOPC1 & AST-OPC1), which contains hESC-derived oligodendrocyte progenitor cells (OPCs), in patients with subacute spinal cord injuries. He next served at Chief Medical Officer at Lineage Cell Therapeutics (formerly BioTime), which acquired Asterias in March 2019.
Dr. Wirth joined Aspen Neuroscience as Chief Medical Officer in January, 2020.
Drug Delivery Topic
Considerations for Intraparenchymal Delivery of Therapeutics
- Cannula design and placement within regions of interest
- Differences between brain structures (e.g., putamen, thalamus, cerebellum)
- Infusion monitoring and coverage of regions of interest
Vice President, Translational & Pre-Clinical Research
Clearpoint Neuro
Ernesto brings over 20 years of experience in translational neuroscience to ClearPoint, with preclinical neurosurgery expertise delivering therapeutics to the brain, spinal cord, and cerebrospinal fluid. He has over 16 years of experience in AAV gene therapy, cell therapy, image-guided platforms, and neurodegenerative disorders. He has served as a study director for more than 11 years in nonGLP and safety/tox GLP studies. During his career, he has led cross-functional teams, strategic partnerships, studies investigating novel therapeutics, biomarker identification, and device development. He has authored over 32 peer-reviewed publications, and holds a Ph.D. in Neuroscience from Flinders University of South Australia.
Sponsored Spotlight Presentation
1:25 – 2:25pm
- Necessity of pre-screening patients for pre-existing anti-AVV antibodies for different CNS Routes of Administration (ROAs) and diseases (such as with comprised Blood Brain Barrier)
- Monitoring treatment-induced peripheral and CNS local immune response for different ROAs and the impact on safety and efficacy
- Effective mitigation strategies for both pre-existing and treatment-induced CNS immune responses
Vice President, Biomarker and BioAnalytical Sciences
Sangamo Therapeutics
Dr. Yanmei Lu is currently the Vice President of Biomarker and BioAnalytical Sciences at Sangamo Therapeutics. The functions of her group include biomarker discovery, biomarker strategy, bioanalysis and companion diagnostic develop to support gene and cell therapy programs at both preclinical and clinical stages. Prior to joining Sangamo, Dr. Lu acquired extensive experience at Genentech in developing PK, biomarker, immunogenicity and functional assays for antibody screening, candidate selection and in vivo studies to evaluate PK/PD, efficacy and safety of large molecule therapeutics in disease areas of , Neurosciences, Immunology, Oncology and Ophthalmology. Dr. Lu holds a PhD in Biochemistry and Molecular Biology from University of South Florida and has published over 40 peer-reviewed articles/book chapters.
3:45 – 4:45pm
- What drug delivery and novel technologies will enable gene therapy that maintains efficacy with low off-target effects
- How can we use combined scoring approaches and novel biomarkers to better characterise the clinical impact of the treatment and accelerated 1-3 trials?
- How can you envisage treatment for pre-symptomatic patient
- How can you manage immune responses to gene therapy
Vice President, Research & Development
Locanabio
Ron Batra joined Locanabio in March 2018 and serves as Vice President, Research and Development. Dr. Batra was a significant contributor to the foundational work of RNA targeting CRISPR therapeutics for microsatellite expansion diseases, which served as the basis for the founding of Locanabio. Prior to joining Locanabio, Ron worked at Verily. Dr. Batra is an expert in RNA splicing and development of therapeutics of RNA mediated diseases including myotonic dystrophy and C9orf72 ALS. He was awarded the Myotonic Dystrophy Foundation (MDF) postdoctoral fellowship for his work at the laboratory of Gene Yeo Ph.D. at UC San Diego. During his time at UC San Diego, he also worked with the department of Neurology studying pathology and disease mechanisms of sporadic and C9orf72 ALS. Prior to UC San Diego, Dr. Batra studied the mechanisms of C9orf72 ALS at the Mayo Clinic. Dr. Batra received his bachelor’s degree in pharmacy (Pharm. D equivalent) at the University of Delhi in India. He received a Master’s in pharmacology at Ohio State University. Dr. Batra completed his Ph.D. degree in genetics at University of Florida where he studied RNA biology and gene therapy of neuromuscular diseases. Ron was the 2019 recipient of Biocom Catalyst award for biotech leaders under 40 in California and 2021 recipient of the Endpoints 20 under 40 mention.
Drug Delivery
- The Decentralized Landscape – Considering regulatory and operational features
- The Post-COVID-19 Landscape – Exploring risk mitigation, contingency, and AI strategies
- The Therapeutic Landscape – The advent of digital therapeutics at the expense of traditional drug therapy
Head, Neurology and Regenerative Medicine Center for Excellence
Biorasi
As head of Biorasi’s Neurology and Regenerative Medicine Centers of Excellence, Dr. El Hokayem oversees clinical projects related to neurological indications and therapies. His unique expertise in the basic, translational, and clinical research spectrum provides critical insights at strategic, operational, and managerial levels. He is an expert clinical trial decentralizer and an early adopter of digital technologies and digital therapeutics.
With over twenty years’ experience in both academia and clinical research, Dr. El Hokayem has focused his research on rare neurological diseases. He currently serves on the Scientific Advisory Board of the Foundation for Angelman Syndrome Therapeutics (FAST). Dr. El Hokayem has published over twenty peer reviewed papers and received multiple awards from international foundations and entities.
Dr. El Hokayem received his Ph.D. from University of Miami, Miller School of Medicine, in Biochemistry and Molecular Biology. He continued his training with post-doctoral work at the University’s Hussman Institute for Human Genomics.
Exploring the Current Opportunities of Shuttle Vehicles and Transcytosis Drug Delivery Technologies
- Discuss the most CNS disease relevant drug targets for targeted brain delivery
- Explore the current technologies for shuttling drugs across the BBB
- Explore alternative non-chemically altering drug delivery technologies
- Discuss which of these technologies can rescue existing drug candidates
Director of Neuroscience Research
Ionis Pharmaceuticals
Dr. Zhao is a director in the Department of Neuroscience Drug Discovery at Ionis Pharmaceuticals, Inc., a company pioneering in RNA therapeutics. She leads target discovery and validation efforts for Alzheimer’s and Parkinson’s Disease, rare neurogenetic diseases and peripheral neuropathies. Dr. Zhao is passionate about her work and impact on patients.
Dr. Zhao received her Ph.D in Neuroscience from Washington University in St Louis, St Louis, Missouri, and her B.S in Biochemistry from the University of Houston, Houston, Texas. Dr. Zhao received her postdoctoral training in Dr. Virginia Lee’s lab, focusing on immunotherapeutic approaches to treat Parkinson’s Disease.
- Discuss the scientific challenges to delivering macromolecules and advanced therapeutics to the brain: vascular, LP and transcranial
- Exploring the operational site oversights and training requirements for single administrations and implantable infusion pumps
- Considerations for maintaining the blind: sham controls, imaging, coordination with pharmacy, and blinding plans
- How should procedural and investigational product related adverse events be managed and documented
Associate Director/Senior Clinical Trial Manager
Medpace
Laura Heinichen, PharmD, RPh, is Associate Director/Sr. Clinical Trial Manager with a demonstrated history in clinical research with experience managing complex Phase I-IV global clinical trials. Her experience includes managing multiple global rare disease/orphan indication clinical trials in pediatric and adult populations as well as managing a Specialty Pharmacy servicing patients with chronic illnesses. Laura received her Doctor of Pharmacy from University of Southern California and her Bachelor of Arts in Psychology and Biology from Dartmouth College.
1-2-1 Meetings / Networking Break
What is the impact of an accelerated approval pathway on biomarker selection for clinical trials?
This is not intended to be a debate about the validity of the accelerated approval pathway for CNS therapeutics. Rather, now that this pathway has been made available, we will discuss how it affects planning for future trials. Topics could include:
- What biomarkers, beyond Abeta and Tau, could be used to support a mechanism-based accelerated approval for CNS therapeutics?
- What assays or tools are needed in order to enable the use of such biomarkers?
- Are there existing preclinical models for any CNS indications that could support a translational prediction of clinical benefit based on pharmacodynamic readouts?
Scientific Director, Discovery and Translational Research, Neuroscience
Bristol Myers Squibb
Irit Rappley is a neurobiologist with expertise in protein homeostasis pathways and translational biomarkers. She is the biomarkers lead for all BMS Neuroscience programs as they head into the clinic, leads several external collaborations across indications and modalities, and serves as a subject matter expert for evaluation of business development opportunities. Irit helped to develop the strategy upon which the Neuroscience group at Celgene (now BMS) was founded. Prior to her work in Neuroscience, she was part of the Protein Homeostasis group where she contributed to several hematology/oncology programs that are now in the clinic.
Irit earned dual SB degrees from the Massachusetts Institute of Technology, and a PhD in Neurobiology from Harvard University.
Best Approaches in Intranasal CNS Delivery of Large Molecular Weight Therapeutic Proteins
- Discuss the current and latest approaches in intranasal CNS drug administration
- Discuss the intranasal drugs’ mechanisms in bypassing the BBB and blood-CSF barrier
- Explore the preclinical models for testing the method of drug delivery
- Contrast the advantages and limitations of intranasal delivery to other approaches
Founder and Chief Scientific Officer
Impel NeuroPharma
John Hoekman, Ph.D. is co-founder and CSO at Impel NeuroPharma. He co-founded Impel while in graduate school to develop tools, devices, and formulations to overcome the inherent variability and efficiency limitations of traditional nasal drug delivery. At Impel, John has been involved in nasal delivery studies of small molecules, peptides, and protein molecules with a focus on biodistribution to the CNS. Additionally, he has experience developing both drug and formulation aspects of nasal drug device combination products from concept through Phase 3. Impel currently has 3 pipeline assets including its lead pipeline candidate, Trudhesa, which is currently under NDA review by the FDA. John is an inventor on over 25 issued patents.