CNS Therapeutics Xchange
East Coast 2021
May 24 & 26

Welcome to hubXchange’s East 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

DAY 1 (May 24, 2021)

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

9:10 – 10:10am

Preclinical Topic MD Biosciences Roundtable –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.

10:10 – 11:00am

BREAK

11:00 – 11:30am

1-2-1 Meetings

11:35am – 12:05pm

1-2-1 Meetings

12:05 – 1:00pm

BREAK

1:00 – 1:30pm

1-2-1 Meetings

1:40 – 2:40pm

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

Richard Malamut

Chief Medical Officer
Collegium Pharmaceuticals

Richard Malamut is currently Chief Medical Officer and Executive Vice President at Collegium Pharmaceuticals which is committed to be the leader in responsible pain management. He has also served as Chief Medical Officer for Braeburn Pharmaceuticals, Inc. where he was responsible for the company’s medical affairs, non-clinical and clinical development, clinical operations, research and development quality assurance, and pharmacovigilance functions. Prior to that, Richard had similar responsibilities as Chief Medical Officer at Avanir Pharmaceuticals and was Senior Vice President of Global Clinical Development at Teva Pharmaceutical Industries Ltd where he was responsible for Pain, Neuropsychiatry, Oncology, and New Therapeutic Entities. His experience also includes roles of increasing responsibility focusing on early clinical development and translational medicine in Neurology, Psychiatry and Analgesia at Bristol-Myers Squibb and AstraZeneca.

Richard earned his medical degree from Hahnemann University in Philadelphia and completed both a residency in Neurology and a fellowship in Neuromuscular disease. He worked as a board-certified academic and clinical neurologist for 17 years and has more than 50 publications in the fields of pain medicine, neuromuscular disease, autonomic disease, and neurodegenerative disease.

DAY 2 (May 26, 2021)

8:30 – 9:00am

Keynote Presentation:

Current Developments and Trends in Novel Therapeutic Classes for CNS Diseases

A review of recent scientific developments and emerging technologies
Review of the CNS deal space with a particular focus on neurodegenerative diseases and pain

Sigal Meilin, Chief Scientific Officer, MD Biosciences

9:10 – 10:10am

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

Huseyin Mehmet

Senior Vice President and Head of Research
Lucy Therapeutics

Huseyin Mehmet has over 20 years experience in basic research and drug discovery, spending half of his career in academic research (Imperial College London) and half in biopharma. He has led drug discovery groups at Merch, Proteostasis Therapeutics and Zafgen and has consulted for several biotech start ups under the Atlas Ventures umbrella. He has extensive experience in metabolic disease, immunology, neuroscience and cancer and has authored over 100 papers and patents.

10:10 – 11:00am

BREAK

11:00 – 11:30am

1-2-1 Meetings

11:35am – 12:05pm

1-2-1 Meetings

12:05 – 1:00pm

BREAK

1:00 – 1:30pm

Ulysses Neuroscience Poster

Interferon-Alpha as a Back-Translational Model of Neuroinflammation to Identify Novel Biomarkers and Targets

Neuroinflammation in psychiatric and neurodegenerative disorders
The interferon-alpha back-translational model of neuroinflammation
Translational central and peripheral biomarkers

Charlotte Callaghan

Chief Scientific Officer And Patient Insight Manager
Ulysses Neuroscience

Charlotte K. Callaghan, PhD, is a graduate of Trinity College Dublin, Ireland, where she also completed her post-doc embarking on an exciting scientific career undertaking a variety of academic-industrial research collaborations investigating the neuropsychopharmacology of affective disorders. Charlotte is currently Chief Scientific Officer and Patient insight manager at Ulysses Neuroscience Ltd., a private R&D organisation, which has been established with patient-centricity and translational research as its core research values. Ulysses Neuroscience multidisciplinary preclinical and clinical translational platforms investigates the molecular mechanisms leading to the synaptic pathology and neuroinflammatory events which accompany the development of psychiatric, neurodevelopmental and neurodegenerative disorders. The company has a collaborative and scientist-to-scientist approach in working with pharmaceutical companies on tailored research projects. Drug discovery in neuroscience has changed, Ulysses Neuroscience is part of the new journey against brain disorders.

1:40 – 2:40pm

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?

Jonathan Levenson

Vice President, Translational Biology
Tiaki Therapeutics

Jonathan Levenson is the Vice President of Translational Biology at Tiaki Therapeutics. Prior to joining Tiaki, he was the head of preclinical and nonclinical research and development at Proclara where he was key in moving two assets into the clinic, one for Alzheimer’s disease and one for systemic amyloidosis. Before that, Jonathan worked at Galenea where he held positions of increasing responsibility, culminating in the role of senior scientist. Jonathan was an assistant professor and the director of the rodent behavioral core at the University of Wisconsin-Madison. He was previously a consultant at Saegis Pharmaceuticals and an assistant professor at Baylor College of Medicine. He earned his doctorate in Biology from the University of Houston and did his post-doctoral training at the Baylor College of Medicine in the Department of Neuroscience. He has authored or co-authored over 70 research publications focused on neuropharmacology and the treatment of neurological diseases.

2:40 – 2:45pm

Closing Address

Preclinical

Time

Titles and Bullets

Facilitator

DAY 1 (May 24, 2021)

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

9:10 – 10:10am

Synapcell Roundtable –
The Current Role and Application of EEG in CNS Drug Discovery

Explore the translational aspects for both animal to human and preclinical to clinical
Discuss, where are the current limits of EEG applications?
What novel solutions do we need to develop with EEG in the near future?

Corinne Roucard

Chief Executive Officer
Synapcell

“In 2005 I co-founded and became C.E.O. at SynapCell, a drug discovery-focused CRO specialized in CNS disorders. Neuropsychiatric and neurodegenerative disorders are society’s burden today, and their incidence will continue to grow. It is our duty to assist the discovery of new treatments for patients, and I always keep in mind the need to develop innovative drug discovery solutions that are as translational as possible and inspired by a bed to benchside strategy. At SynapCell I am responsible for customer relations, and this mission allows me to actively participate in international networks, while also building strong relationships with our clients.”

Venceslas Duveau

Head of Science
Synapcell

“I am a neuroscientist with a strong interest and over 10 years of experience in drug discovery and brain disorders such as epilepsy, Parkinson’s and Alzheimer’s diseases. As SynapCell’s Head of science, I am at the cutting edge of Neuroscience research through the management and development of research programs for customers or for internal R&D projects.”

10:10 – 11:00am

BREAK

11:00 – 11:30am

1-2-1 Meetings

11:35am – 12:05pm

1-2-1 Meetings

12:05 – 1:00pm

BREAK

1:00 – 1:30pm

1-2-1 Meetings

1:40 – 2:40pm

Multi-Omics-Led Biomarker Discovery and Validation

How can you incorporate multi-omics profiles and patient data with AI & machine learning for next-generation CNS biomarker discovery?
How can we validate novel biomarkers and their clinical relevance at an early stage?
What are the advantages and drawbacks to the current modes of biomarker discovery?
How can we ensure that novel biomarkers have good clinical utility with strong translatability into the clinic?

Rebecca Evans

Senior Medical Director, Neuroscience
Takeda

DAY 2 (May 26, 2021)

8:30 – 9:00am

Keynote Presentation:

Current Developments and Trends in Novel Therapeutic Classes for CNS Diseases

A review of recent scientific developments and emerging technologies
Review of the CNS deal space with a particular focus on neurodegenerative diseases and pain

Sigal Meilin, Chief Scientific Officer, MD Biosciences

9:10 – 10:10am

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?

Marion Wittmann

Vice President of Translational Sciences
Praxis Precision Medicines

Marion Wittmann, Ph.D., has served as head of Translational Sciences at Praxis Precision Medicines since September 2020. Prior to her role leading the Translational Sciences team, Dr. Wittmann served as Vice President of Biology & Toxicology at Praxis from December 2016 to September 2020. Prior to joining Praxis, Dr. Wittmann was the Director of Alzheimer’s research at Biogen from October 2014 to November 2016 and the head of the electrophysiology core team at Biogen from July 2011 to October 2014. Prior to Biogen, Dr Wittmann worked at Merck & Co. where she led an in vitro and in vivo physiology and pharmacology group and conducted target discovery, drug discovery, and translational pharmacology research in the areas of Alzheimer’s disease, Parkinson’s disease, and schizophrenia. Dr. Wittmann has over 19 years of experience in CNS drug discovery and development with core expertise in animal models, translational biomarkers and small and large molecule drug discovery in CNS indications. Dr. Wittmann received an M.S. in biology and a Ph.D. in neurobiology from the Eberhard-Karls-University in Tuebingen, Germany.

10:10 – 11:00am

BREAK

11:00 – 11:30am

1-2-1 Meetings

11:35am – 12:00pm

1-2-1 Meetings

12:05 – 1:00pm

BREAK

1:00 – 1:30pm

Transpharmation Poster

Changes in Brain Oscillatory Activity Might Predict the Efficacy of Therapeutics in Neuropsychiatric Disorders

Disrupted sleep and abnormal brain oscillations are common early features of many neuropsychiatric disorders. Since sleep and EEG abnormalities often precede the clinical onset of these diseases, their possible involvement in the pathophysiological process is considered
Medications used for symptomatic treatment of neuropsychiatric disorders often affect sleep and brain oscillatory activity. Furthermore, altering brain oscillatory activity by non-pharmacological means can improve the symptoms of the diseases
Thus, the potential mechanistic relationship between changes in brain oscillatory activity and the pathophysiological process in these disorders will be discussed alongside with potential novel therapeutic targets

Sandor Kantor

Scientific Lead
Transpharmation

Sandor has over 20 years of experience in neuroscience research, published more than 20 research articles and was awarded the prestigious Pickwick Fellowship of National Sleep Foundation to study the neurobiology of narcolepsy at Harvard Medical School. Before taking up his post at Transpharmation, Sandor held a research role at Cambridge University where he identified the abnormal changes in sleep and brain oscillations in Huntington’s disease as well as the drugs that can reverse them. As a Principal Scientist at Transpharmation, he is responsible for directing sleep-wake profiling and EEG assessment of novel, CNS acting compounds.

1:40 – 2:40pm

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?

Philip Iredale

Vice President, Head of Biology
Cerevel Therapeutics

Phil did his PhD and a brief post doc in signal transduction mechanisms in CNS at the University of Nottingham in the UK. In 1994 he joined the Duman/Nestler lab at Yale investigating the molecular regulation of CRF receptors and, exploring the role the receptor plays in withdrawal from drugs of abuse. He joined Pfizer in 1997 as a lab head where he took on increasingly senior roles leading several programs incuding CB1 agonists/antagonists, psychosis disease space. In 2004 he joined the CNS management team and subsequently headed several programs in neuroscience, neurology and external collaborations which led to therapeutic area operational oversight as well as Kendall Sqauare MA site responsibilities. In 2018 Pfizer announced that it was exiting Neuroscience. Phil subsequently joined Cerevel Therapeutics as Head of Biology in October of 2018.

2:40 – 2:45pm

Closing Address

Clinical Studies

Time

Titles and Bullets

Facilitator

DAY 1 (May 24, 2021)

8:30 – 9:00am

Opening Address & Keynote:

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

9:10 – 10:10am

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

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.

10:10 – 11:00am

BREAK

11:00 – 11:30am

1-2-1 Meetings

11:35am – 12:05pm

1-2-1 Meetings

12:05 – 1:00pm

BREAK

1:00 – 1:30pm

1-2-1 Meetings

1:40 – 2:40pm

Best Approaches to Patient Stratification

How to de-risk clinical development by ensuring your program’s trial patient population composition is correct
What are the current possibilities of AI and machine learning in identifying treatment responders and predicting disease progression?
How can we best track patient disease progress using digital biomarkers?

Kemi Olugemo

Executive Medical Director, Neurology Clinical Development
Ionis Pharmaceuticals

Kemi Olugemo has an undergraduate degree in Clinical Laboratory Science from the University of Massachusetts (Magna cum Laude) and an MD from the University of Maryland School of Medicine where she remained for her Neurology Residency before undertaking an additional Fellowship in Neuroimmunology & Multiple Sclerosis (MS) at the Maryland Center for Multiple Sclerosis.

Over her 12 years in clinical research, Kemi has been involved in all phases of clinical development, global medical strategy, business development, and innovations in lifecycle planning.

Prior to joining Akcea in 2019, she held increasing leadership roles at Parexel International and Questcor Pharmaceutical, Inc, and was a long-term consultant in Neurology with Quorum Independent Review Board. Kemi draws on a diverse set of experiences including being the current Director of Communications at Women of Color in Pharma (WOCIP) and serving on the Scientific Committee of the International Society for CNS Clinical Trials and Methodology (ISCTM).

DAY 2 (May 26, 2021)

8:30 – 9:00am

Keynote Presentation:

Current Developments and Trends in Novel Therapeutic Classes for CNS Diseases

A review of recent scientific developments and emerging technologies
Review of the CNS deal space with a particular focus on neurodegenerative diseases and pain

Sigal Meilin, Chief Scientific Officer, MD Biosciences

9:10 – 10:10am

Effective Planning and Execution of Decentralised Clinical Trials

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?

Simon Portsmouth

Executive Medical Director
Shionogi

Simon Portsmouth MD FRCP is head of medical Science at Shionogi Inc. Florham park new Jersey. Primarily working in infectious disease clinical development for the past 11 years following a 16 year career in the UK NHS as a HIV specialist clinician and researcher.

10:10 – 11:00am

BREAK

11:00 – 11:30am

1-2-1 Meetings

11:35am – 12:05pm

1-2-1 Meetings

12:05 – 1:00pm

BREAK

1:00 – 1:30pm

WCG Poster

CNS Trials in a (Post-) COVID World

How to meet or exceed enrollment timelines in a time of shut-downs
Remote assessment to keep trials moving in early phase and rare disease studies
Use of advanced analytic techniques to monitor patient-reported and clinician-rated outcomes to ensure accuracy for studies where every data point is precious

Mark G. A. Opler

Chief Research Officer
WCG MedAvante-ProPhase

Dr. Mark Opler serves as Chief Research Officer, directing scientific research and development at WCG MedAvante-ProPhase. Dr. Opler was the founder of ProPhase and served as its CEO and Chief Scientific Officer among other positions. He holds the titles of Adjunct Assistant Professor of Psychiatry at New York University and Assistant Professor of Clinical Neuroscience at Columbia University’s College of Physicians and Surgeons. His academic research focuses on the etiology, phenomenology, and treatment of serious and persistent mental disorders. He is also leading the development of the new upcoming edition of the PANSS Manual©.

Molly Hair

Director, Clinical Strategic Solutions and New Product Development
WCG ThreeWire

Molly is currently the Director of Clinical Strategic Solutions and New Product Development at WCG ThreeWire. With more than 14 years of experience in clinical trial patient recruitment, Molly works with clinical research sites to develop and deploy unique support strategies based on their individual needs. Prior to her time at WCG ThreeWire, Molly worked as a lead recruitment coordinator at an independent research unit, where she led recruitment efforts on more than 150 clinical trials in a wide range of therapeutic areas.

1:40 – 2:40pm

Selecting Clinical Endpoints and Assessing Clinical Outcomes

How to assess for 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?

Chris Winrow

Vice President, Head of Translational Medicine
Cyclerion Therapeutics

Christopher Winrow is the Senior Director, Clinical Development and Neuroscience Program Lead at Cyclerion Therapeutics in Cambridge, MA. Prior to Cyclerion, Dr. Winrow held roles of increasing responsibility at Merck & Co., Inc. and Ironwood Pharmaceuticals. He completed his Ph.D. at the University of Alberta and post-doc at the Salk Institute. Dr. Winrow has an extensive background in CNS diseases with areas of focus including Alzheimer’s disease, Parkinson’s disease, dementia, sleep disorders, autism, and orphan diseases. He authored more than 75 publications, is co-inventor on multiple patents, co-authored 10 eIND/INDs and has over 15 years of neuroscience drug discovery and development experience. Dr. Winrow delivered five CNS compounds from screening through successful clinical POC. He led the Belsomra® discovery team from screening to regulatory approval in less than 10 years, resulting in approvals by U.S. and international regulatory agencies. He currently leads multidisciplinary drug discovery and development teams for serious and orphan diseases at Cyclerion Therapeutics.

2:40 – 2:45pm

Closing Address

Advanced Therapies

Time

Titles and Bullets

Facilitator

DAY 1 (May 24, 2021)

8:30am – 9:00am

Opening Address & Keynote:

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

9:10 – 10:10am

Accelerate Early Clinical Development of Gene Therapies for CNS Diseases

Historical control – the role of natural history study
Interim analysis – for futility, stop early for success, and/or start early planning of subsequent studies
Master protocol – to increase efficiency of clinical trial design

Haichen Yang

Vice President, Global Therapeutics Lead, CNS, Drug Development Services
Icon

Dr Yang has 27 years of global drug development experience (22 with pharma and 5 with CRO), providing functional and strategic leadership. She has extensive expertise in overseeing and designing clinical programs across all phases of development and product support for various indications in the Neuroscience. She successfully directed multiple NDA/MAA submissions leading to worldwide drug and indication approvals.

At ICON, Dr. Yang provides strategic drug development expertise to client companies on full service, consulting, and due-diligence projects. Dr. Yang has contributed to multiple gene and cell therapy projects to advise clinical development strategy, provide study design input and protocol considerations. She is well published with 40+ articles in peer-reviewed journals and 2 book chapters. She serves as a committee member for several drug development societies and task forces.

10:10 – 11:00am

BREAK

11:00 – 11:30am

1-2-1 Meetings

11:35am – 12:05pm

1-2-1 Meetings

12:05 – 1:00pm

BREAK

1:00 – 1:30pm

1-2-1 Meetings

1:40 – 2:40pm

CNS Transplantations- Cells Alone is Not the Therapy

Discuss and define the mechanisms underlying immune rejection for allogeneic cell therapy and contrast with autologous therapy
Explore how healthy CNS circuity is formed after cell transplant treatment- what can be done to ensure the optimal engraftment and function?
Explore the neuro-transplantation biomarkers that can be measured in the periphery and via neuroimaging
What is the transferable experience from bone marrow transplantation? Can the CNS be conditioned before cell therapy administration to reduce neuroinflammation?

Thomas Hudzik

Director – Translational Neuroscience
BlueRock Therapeutics

Tom Hudzik is the Senior Director of Translational Neuroscience at BlueRock Therapeutics. He has worked in the Pharma and Biotech Industries for almost 30 years as a pharmacologist, toxicologist, manager, and project director who has shepherded 4 small molecule programs into clinical development and has assisted numerous other programs. He earned his PhD in Behavioral Neuroscience and has spent much of his career working on CNS indications in neurology, psychiatry, and substance use disorders – most recently in cell therapy for neurodegenerative disease. Tom has authored more than 70 original manuscripts and book chapters – most of which reflect cross-functional collaborations.

DAY 2 (May 26, 2021)

8:30 – 9:00am

Keynote Presentation:

Current Developments and Trends in Novel Therapeutic Classes for CNS Diseases

A review of recent scientific developments and emerging technologies
Review of the CNS deal space with a particular focus on neurodegenerative diseases and pain

Sigal Meilin, Chief Scientific Officer, MD Biosciences

9:10 – 10:10am

Gene Therapies- How Close Are We to Curing CNS Diseases

What drug delivery and manufacturing technologies will enable a non-invasive, blood system-administrated gene therapy that maintains efficacy with low off-target effects
How can we use combined scoring approaches to better characterise the clinical impact of treatment?
Can we accelerate phase 1 and 2 trials using pivotal studies that utilize well-designed and defined clinical endpoints?
How can you envisage treatment for pre-symptomatic patients?

Nathalie Cartier-Lacave

CNS Gene Therapy Lead
Asklepios BioPharmaceuticals

Nathalie Cartier-Lacave was the founder and CSO of BrainVectis which was acquired by AskBio. Research Director at INSERM, where she leads the group “Biotherapies of neurodegenerative diseases”, Nathalie is president of the European Society of Gene and Cell Therapy (ESGCT). Her research led to the first gene therapy clinical trial in humans, using HIV as a gene transfer vector (Science 2009). Thanks to this trial, four children suffering from adrenoleukodystrophy were cured.

10:10 – 11:00am

BREAK

11:00 – 11:30am

1-2-1 Meetings

11:35am – 12:05pm

1-2-1 Meetings

12:05 – 1:00pm

BREAK

1:40 – 2:40pm

Atuka Poster Preclinical Topic-

Non-Human Primate Models in the Development of Parkinson’s Disease Therapeutics: Highlights of Atuka’s Recent Experience
• State of the marketplace – Atuka’s analysis of late-stage preclinical activity and experience
• The latest news on our non-human primate model of synucleinopathy in Parkinson’ disease.
• Experience with evaluating LRRK2 target engagement and efficacy: does non-human primate data add value to rodent data as a prelude to initiating clinical development?
• Building an efficacy dataset in non-human primates to empower a clinical program

James Koprich

Chief Technology Officer
Atuka

Dr. James Koprich is Chief Technology Officer at Atuka Inc. He received his PhD in Neuroscience from Rush University Medical Center (Chicago, IL) and completed a post-doctoral fellowship at Harvard Medical School (Boston, MA) where he also joined their faculty. Dr. Koprich’s research interests include animal models of neurodegenerative disease, biodistribution and use of viral vectors and the preclinical evaluation of novel disease modification and symptomatic therapeutics. In recent years, his work has led to the development of progressive rodent and non-human primate models of Parkinson’s disease alpha-synucleinopathy. These and other studies are detailed in over 50 peer reviewed publications. In addition to his role at Atuka, Dr. Koprich is visiting Professor of Neurology at Huashan Hospital/ Fudan University and occasionally serves as a lecturer at the University of Toronto.

1:40 – 2:40pm

Preparing Your Development Strategies for the Next Wave- Gene Therapies for Common CNS Diseases

Developing Gene Therapies for common neurological disorders requires a fundamental shift on the way that we develop and evaluate these technologies. Are we coordinating early enough in the development process between key stakeholders to ensure commercial success?
What are the healthcare infrastructure considerations needed once gene therapies are commercially available in common diseases?
As Gene Therapies become available for larger volume of patients, how can we avoid potential roadblocks with payers?
With the curative nature of advanced therapies, how can you maintain patient engagement for the next 2-3 years to gather phase IV data
How do you capture the value of advanced therapies with the nature of one-time high expensive treatment vs conventional long, sustained costs to healthcare?

Vanessa Almendro

Head of Strategy and Operations
Eisai, G2D2

Vanessa Almendro, Ph.D, MBA, is the Head of Strategy and External Innovation at G2D2, Eisai, where she manages innovation initiatives inside the organization and identifies strategies, business opportunities and new technologies that could support or complement the current business goals. She is also in charge of identifying new business models and new industry structures that are better suited to support those opportunities. Prior to Eisai, Vanessa was the Director for Strategy and Operations at Cogen Immune Medicine, where she led the development of the Disease Area Strategy including building the Clinical Collaborations unit, the Competitive Intelligence efforts and leading New Product Planning. Prior to Cogen, Vanessa served in different leadership roles at Vertex Pharmaceuticals Inc., where she was Global Commercial Brand lead for the pain portfolio and responsible of providing insights to shape the development path of clinical candidates and provide input to develop the pipeline strategy. Prior to joining the Commercial Department, Vanessa served as the Head of Exploratory Biology at the same company where she was in charge of identifying new oncology targets for therapeutic intervention. Prior to industry, she held several positions in academic institutions, including the Hospital Clinic in Barcelona, Spain, and the Dana-Farber Cancer Institute in Boston. At the Dana-Farber Cancer Institute she was a pioneer in the study of breast cancer intra-tumor heterogeneity, incorporating mathematical modeling with tumor topological features to predict tumor evolution. As a Group Leader at the Hospital Clinic in Spain, her research interests included understanding mechanisms of resistance to conventional chemotherapy, including acquired and adaptive resistance, to identify novel targets for the treatment of cancer. Vanessa has authored multiple articles in top-tier journals, and received several research awards. During her career, Vanessa has served as consultant for Novartis, Prous Science, AB Therapeutics and the National Brain Tumor Society in the US. She holds a doctorate in Biochemistry and Molecular Biology from the University of Barcelona, a post-doc from Harvard Medical School, and an Executive MBA from the Sloan School at MIT.

2:40 – 2:45pm

Closing Address

Drug Delivery

Time

Titles and Bullets

Facilitator

DAY 1 (May 24, 2021)

8:30am – 9:00am

Opening Address & Keynote:

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

9:10 – 10:10am

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

Richard Scheyer

Vice President, Medical Department
Medpace

Dr. Richard Scheyer is a board-certified neurologist with over 30 years of professional medical experience which includes 20 years dedicated to clinical drug development in academia, Pharma/Biotech, and CRO. He is a highly-regarded author with over 60 manuscripts and abstracts, with a focus on clinical pharmacology and therapeutic activity. He received his B.S. in Physics from Stanford University, and his M.D. from The State University of New York, Upstate Medical University. He completed his residency in neurology and fellowship training in epilepsy at Yale University before joining the Yale faculty, serving as Associate Professor of Neurology.

Suzanne Kempisty

Senior Director, Clinical Trial Management
Medpace

Suzanne Kempisty, BSN, RN, CCRP is Senior Director of Trial Management with a demonstrated history of working in academic research, as well as, pharmaceutical and device industries. She is Registered Nurse with bedside neurological intensive care experience. Ms. Kempisty is also certified as a Clinical Research Professional in Clinical Research from University of Cincinnati. Prior to joining Medpace, she served as Associate Professor and Director of the Division of Clinical Research in Neurosurgery at University of Cincinnati and Mayfield Clinic. She received her Bachelor of Science in Nursing from Temple University in Philadelphia, PA.

10:10 – 11:00am

BREAK

11:00 – 11:30am

1-2-1 Meetings

11:35am – 12:05pm

1-2-1 Meetings

12:05 – 1:00pm

BREAK

1:00 – 1:30pm

1-2-1 Meetings

1:40 – 2:40pm

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

Susan Rosenbaum

Founder, Chairman & Chief Executive Officer
Lauren Sciences

Susan Rosenbaum, J.D., is Founder, Chairman & CEO of Lauren Sciences; 10 years successful biotech visionary, entrepreneurial leader, strategic executive, motivated manager w/experience and expertise. Licensed breakthrough V-Smart® nanotechnology for BBB delivery from BGU, Israel; built Lauren Sciences LLC, multiple medical and scientific collaborations, 5-member MAB/SAB of KOLs, 12 foundation grant awards, innovative V-Smart® platform applications, transformative V-Smart® pipeline products in pre-clinical development for CNS; co-authored DD&D March 2018 cover feature, presented at 36 scientific conferences, w/posters at 7 scientific meetings, made Lauren Sciences a BIO 2018 winner and 1st Place Winner Global Healthcare Innovation at BIO 2019. Founder (present): Lauren Sciences LLC; Maya Sciences LLC; Founder (past): investment banking firm (Rosenbaum & Co.), corporate finance law firm (Rosenbaum, P.C.); Private and public company BODs; Nat’l/internat’l author/speaker.

2:40 – 2:45pm

DAY 2 (May 26, 2021)

8:30 – 9:00am

Keynote Presentation:

Current Developments and Trends in Novel Therapeutic Classes for CNS Diseases

A review of recent scientific developments and emerging technologies
Review of the CNS deal space with a particular focus on neurodegenerative diseases and pain

Sigal Meilin, Chief Scientific Officer, MD Biosciences

9:10 – 10:10am

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

Larry Brown

Executive Vice President & Chief Scientific Officer
Noveome Biotherapeutics

Larry Brown, Sc.D., serves as the Executive Vice President of R&D and Chief Scientific Officer of Noveome Biotherapeutics. He obtained his doctorate in Biochemistry and Bioengineering at the Massachusetts Institute of Technology under Prof. Robert Langer and trained in the surgical research laboratory of the late Dr. Judah Folkman at the Boston’s Children’s Hospital. Currently, Dr. Brown manages Noveome’s scientific research and development team, working closely with the regulatory group for all FDA related submissions and the company’s executive management leadership team. He has broad experience in large pharmaceutical and start-up venture capital-based companies which have generated over a hundred publications and patents. Previously, he served as Vice President of Research at Baxter Healthcare Corporation and in scientific leadership roles at several startup biotech companies. He has significant interdisciplinary experience in drug delivery systems particular for therapeutic protein and nucleic acid molecules and has conceived of and developed 8 novel product formulations opportunities that have reached the clinic. He maintains strong academic collaborations with MIT’s Deshpande Center for technological innovation.

10:10 – 11:00am

BREAK

11:00 – 11:30am

1-2-1 Meetings

11:35am – 12:05pm

1-2-1 Meetings

12:05 – 1:00pm

BREAK

1:00 – 1:30pm

Clinical Studies Topic: Science 37 Poster
The Promise Of Telepsychiarty: Implications for Clinical Research
•The promise of telepsychiatry: background and potential
•Real-life design and delivery: the patient journey for a major depressive disorder trial

Chris Reist

Medical Director, Psychiatry and Behavioral Science
Science 37

Christopher Reist serves as the Medical Director for Psychiatry and Behavioral Science.
Prior to joining Science 37, Dr. Reist served for 17 years as Assistant Dean in the School of
Medicine at UC Irvine and the Director of Medical Research for the Long Beach Veterans
Affairs Healthcare System. Prior to this role he was the Chief of Mental Health,
overseeing full service mental health care delivery to over 45,000 enrollees at the Long
Beach VA Healthcare System. Since 1999 he also served as Associate Director of the
Southern California VA Mental Illness Research, Education and Clinical Center (MIRECC)
which is focused on improving long term outcomes in chronic psychotic disorders through
basic/clinical research and dissemination of evidence based practices. Dr. Reist has
extensive experience directing clinical trials sponsored by pharma, DoD, NIH and the VA.
PTSD has been the focus of the recent past with trials completed in the use of cognitive
enhancers and virtual reality technology to complement psychotherapy, acupuncture,
stellate ganglion block, and medications.

1:40 – 2:40pm

Bypassing the Blood-Brain-Barrier- Direct Brain Administration Strategies

Discuss the potentials of utilizing an intrathecal, intraparenchymal or intracerebroventricular routes of administration for drug delivery
Explore the opportunities of repurposing CNS drug candidates through a drug-device combination
Explore the current limitations and opportunities of conjugation chemistries, intranasal and other direct to CNS administration routes
Discuss the current biopharma industry approach to drug delivery design for CNS therapeutics

Lisa Shafer

Chief Scientific Officer
Cerebral Therapeutics

Dr Lisa Shafer is the Chief Scientific Officer of Cerebral Therapeutics. Dr. Shafer’s technical and leadership experience in R&D, strategy, and business development spans medical device, biotechnology and pharmaceutical industries. Prior to joining Cerebral Therapeutics, Dr. Shafer served in R&D leadership positions at start-up, mid-size and large global pharmaceutical companies. Prior to joining the pharmaceutical side, Dr. Shafer spent over 10 years in the medical device industry. While at Medtronic Dr. Shafer held various roles in new ventures, drug-device combination product development, neuromodulation research, and management. Dr Shafer’s foundational research and leadership resulted in seminal publications regarding mechanistic, proof of concept and clinical evidence for a portfolio of translational and clinical research programs to advance brain targeted drug delivery. Dr. Shafer began her career at 3M in the Pharmaceutical and Drug Delivery Division and holds a B.S in Genetics and Cell Biology and a PhD in Neuroimmunology.

2:40 – 2:45pm

Closing Address

CNS Therapeutics Xchange
East Coast 2021
May 24 & 26

Novel Targets

Preclinical

Clinical Studies

Advanced Therapies

Drug Delivery

Partners

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CNS Therapeutics XchangeEast Coast 2021May 24 & 26

Novel Targets

Preclinical

Clinical Studies

Advanced Therapies

Drug Delivery

Partners

CNS Therapeutics Xchange
East Coast 2021
May 24 & 26