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

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote:

What Will Neurology Trials Look Like in the Post-COVID-19 ERA?

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

Jimmy El Hokayem, Head, Neurology and Regenerative Medicine Center for Excellence, Biorasi

Jimmy El Hokayem

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

Novel Targets in CNS Research- Past, Present and Future?

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

Amy Pooler

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.

10:10 – 10:40am

1-2-1 Meetings / Networking Break

10:40 – 10:50am

Morning Refreshments

10:50 – 11:50am

Preclinical Models with Predictability and Human Translatability- Can Porcine Models Bridge this Gap?

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

Sigal Meilin

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.

11:50am – 12:50pm

Networking Lunch

12:50 – 1:20pm

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

Beth Hoffman

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.

2:30 – 3:00pm

1-2-1 Meetings / Networking Break

3:00 – 3:10pm

Afternoon Refreshments

3:10 – 3:40pm

1-2-1 Meetings / Networking Break

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?

Bruce Morimoto

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.

4:45 – 6:00pm

Closing Address/ Canape/ Drinks Reception

Preclinical

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote:

What Will Neurology Trials Look Like in the Post-COVID-19 ERA?

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

Jimmy El Hokayem, Head, Neurology and Regenerative Medicine Center for Excellence, Biorasi

Jimmy El Hokayem

Head, Neurology and Regenerative Medicine Center for Excellence
Biorasi

9:05 – 10:05am

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?

Ryan Schubert

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.

10:10 – 10:40am

1-2-1 Meetings / Networking Break

10:40 – 10:50am

Morning Refreshments

10:50 – 11:50pm

Novel Targets Topic:

Tackling Neurodegenerative Disease with Scalable Genome Engineering to Identify & Validate Novel Targets

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

Abhi Saharia

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.

11:50am – 12:50pm

Networking Lunch

12:50 – 1:20pm

1-2-1 Meetings / Networking Break

1:25 – 2:25pm

Defining Animal Models that will Close the Clinical Translational Gap

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?

Sakura Minami

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.

2:30 – 3:00pm

1-2-1 Meetings / Networking Break

3:00 – 3:10pm

Afternoon Refreshments

3:10 – 3:40pm

1-2-1 Meetings / Networking Break

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?

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.

4:45 – 6:00pm

Closing Address/ Canape/ Drinks Reception

Clinical Studies

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote:

What Will Neurology Trials Look Like in the Post-COVID-19 ERA?

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

Jimmy El Hokayem, Head, Neurology and Regenerative Medicine Center for Excellence, Biorasi

Jimmy El Hokayem

Head, Neurology and Regenerative Medicine Center for Excellence
Biorasi

9:05 – 10:05am

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?

Peter St.Wecker

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.

10:10 – 10:40am

1-2-1 Meetings / Networking Break

10:40 – 10:50am

Morning Refreshments

10:50 – 11:50am

COVID-19-Related CNS Syndromes in Neurology Clinical Trials

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?

Jimmy El Hokayem

Head, Neurology and Regenerative Medicine Center for Excellence
Biorasi

11:50am – 12:50pm

Networking Lunch

12:50 – 1:20pm

1-2-1 Meetings / Networking Break

1:25 – 2:25pm

Lessons Learnt from COVID 19 Operating and Executing Clinical Trials During the Pandemic

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?

Sam Jackson

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 21^st Century Scholar. He lives in the San Francisco Bay Area where he makes a valiant attempt to keep up with his two teenage daughters.

2:30 – 3:00pm

1-2-1 Meetings / Networking Break

3:00 – 3:10pm

Afternoon Refreshments

3:10 – 3:40pm

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?

Steve Maricich

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.

4:45 – 6:00pm

Closing Address/ Canape/ Drinks Reception

Advanced Therapies

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote:

What Will Neurology Trials Look Like in the Post-COVID-19 ERA?

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

Jimmy El Hokayem, Head, Neurology and Regenerative Medicine Center for Excellence, Biorasi

Jimmy El Hokayem

Head, Neurology and Regenerative Medicine Center for Excellence
Biorasi

9:05 – 10:05am

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

Ed Wirth

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.

10:10 – 10:40am

1-2-1 Meetings / Networking Break

10:40 – 10:50am

Morning Refreshments

10:50 – 11:50am

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

Ernesto Salegio

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.

11:50am – 12:50pm

Networking Lunch

12:50 – 1:20pm

Drug Delivery

Time

Titles and Bullets

Facilitator

8:00 – 8:30am

Registration

8:30 – 9:00am

Opening Address & Keynote:

What Will Neurology Trials Look Like in the Post-COVID-19 ERA?

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

Jimmy El Hokayem, Head, Neurology and Regenerative Medicine Center for Excellence, Biorasi

Jimmy El Hokayem

Head, Neurology and Regenerative Medicine Center for Excellence
Biorasi

9:05 – 10:05am

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

Hien Zhao

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.

10:10 – 10:40am

1-2-1 Meetings / Networking Break

10:40 – 10:50am

Morning Refreshments

10:50 – 11:50am

Medpace- Scientific and Operational Challenges to Blood-Brain Administration

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

Laura Heinichen

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.

11:50am – 12:50pm

Networking Lunch

12:50 – 1:20pm

1-2-1 Meetings / Networking Break

1:25 – 2:25pm

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?

Irit Rappley

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.

2:30 – 3:00pm

1-2-1 Meetings / Networking Break

3:00– 3:10pm

Afternoon Refreshments

3:10 – 3:40pm

1-2-1 Meetings / Networking Break

3:45 – 4:45pm

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

John Hoekman

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.

4:45 – 6:00pm

Closing Address/ Canape/ Drinks Reception

CNS Therapeutics Xchange
West Coast
San FranCisco 2021
September 22nd

Novel Targets

Preclinical

Clinical Studies

Advanced Therapies

Drug Delivery

Partners

© Copyright 2023 by Weblify & Webtec

CNS Therapeutics XchangeWest CoastSan FranCisco 2021September 22nd

Novel Targets

Preclinical

Clinical Studies

Advanced Therapies

Drug Delivery

Partners

CNS Therapeutics Xchange
West Coast
San FranCisco 2021
September 22nd