CNS Therapeutics Xchange

Biomarkers

Time

Titles and Bullets

Facilitator

8:30am – 9:00am

Opening Address & Keynote:

9:05am – 10:05am

New approaches for identifying neuroinflammation biomarkers

Non-invasive approaches
Revisiting old biomarkers with new approaches
Patient identification/stratification
Defining neuroinflammation

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.

10:10am – 10:40am

1-2-1 Meetings

10:45am – 11:45am

AI driven biomarker and imaging analysis

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

Thomas Westerling-Bui

Director, Scientific Strategy & Business Development
Aiforia

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

11:50am – 12:20pm

1-2-1 Meetings

12:20pm – 1:00pm

Lunch Break

1:00pm – 2:00pm

Applying precision medicine to CNS diseases: using genetics and biomarkers to select and monitor patients who will receive therapeutic benefit from treatment

Moving away from the classical “one-size-fits-all” approach towards more “molecularly” tailored therapy for truly effective CNS treatment options
Generating an “omic” profile using powerful methods for characterizing patients (proteomics, metabolomics, genomics)
Risk Assessment for Neurodegenerative and Neurodevelopmental Disorders Utilizing Polygenic Scores
Biomarkers for identifying and tracking patients – Fluid biomarkers for CNS-specific proteins (blood, CSF) and digital biomarkers (wearable sensor devices) could have the most impact in monitoring CNS diseases
Could CNS drugs achieve the same success as cancer drugs in gaining approval in biomarker defined (guided) subgroups?

Barry Ticho

Chief Medical Officer
Stoke Therapeutics

As Chief Medical Officer Dr. Ticho is responsible for Stoke’s efforts to develop first-in-class RNA based disease-modifying medicines to treat severe neurologic genetic diseases. Prior to joining Stoke Barry was Head of R&D for Cardiovascular and Metabolic Diseases at Moderna Therapeutics. He was previously Head of External R&D Innovation for Cardiovascular and Metabolic Diseases at Pfizer. Prior to that he was Vice President of Clinical Development at Biogen where he led clinical development for the Tysabri program for MS and led the aducanumab program for Alzheimer’s Disease. Barry obtained his M.D. and Ph.D. degrees from the University of Chicago and completed Pediatrics training at Northwestern University and a Cardiology fellowship at Children’s Hospital in Boston. He was on clinical staff at Harvard Medical School and Massachusetts General Hospital.

2:05pm – 2:35pm

1-2-1 Meetings

2:35pm – 3:05pm

1-2-1 Meetings

4:15pm – 4:45pm

Closing Keynote & Address:

Translational Research

Time

Titles and Bullets

Facilitator

8:30am – 9:00am

Opening Address & Keynote:

9:05am – 10:05am

Neuroinflammation – A new approach to neurodegeneration

Exploring new targets for neurodegeneration
Discussing new therapeutics for neurodegeneration and measuring the effect of these drugs
Identifying biomarkers of neurodegeneration

Michael Palfreyman

Chief Operating Officer & Director
Adelia Therapeutics

Michael Palfreyman is an internationally recognized leader in neurological drug development with a successful scientific and business career spanning 35 years. He brings to Adelia Therapeutics a distinguished track record of successful drug development and considerable expertise in CNS pharmacology, pharmacogenomics and rational drug design. Michael’s broad perspective on drug development derives from his experience as Chief Scientific Officer for Amorsa Therapeutics and President and CSO of Psychiatric Genomics; Senior Scientific Advisor to EnVivo Pharmaceuticals and Vitruvean; co-founder of NOVACE; Senior VP for Research and Development at Anadys (formerly Scriptgen); as well as senior executive and scientific leadership roles at Marion Merrell Dow (Sanofi-Aventis) and Beecham (GlaxoSmithKline). In these roles, he successfully developed and demonstrated clinical efficacy of several candidates across multiple therapeutic areas. Michael holds Ph.D. and D.Sc. degrees and is a member of the Royal Pharmaceutical Society. He continues to be an active scientific contributor with more than 150 publications and 35 issued patents.

10:10am – 10:40am

1-2-1 Meetings

10:45am – 11:45am

Biomarker topic Biomarker needs in Neurological diseases

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

Narges Rashidi

Director of Application Sciences, North America Olink Proteomics

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

11:50am – 12:20pm

1-2-1 Meetings

12:20pm – 1:00pm

Lunch Break

1:00pm – 2:00pm

Small molecule and cell therapy approaches for neuroinflammation

Inflammation is the common thread running through all neurodegenerative diseases
Using small molecules to alleviate neuroinflammation and develop curative therapies for Alzheimer’s and ALS
Exploring the latest innovations like CAR-Treg cell therapy to treat both rare and common neurodegenerative diseases

Philip Ashton-Rickardt

Senior Vice President, Immunology
AZ Therapies

Philip Ashton-Rickardt is an esteemed immunologist who was on the faculty at the University of Chicago and held the Chair in Immunology at the Imperial College, London. He has published many reviewed papers in over 30 academic. Many of his findings on T cell lymphocytes have been incorporated into leading immunology textbooks and he is the inventor on 6 patents. He has served as editor for several academic journals and has been a member of many grant review boards throughout the world.

Philip’s work on T lymphocytes has been recognized by his peers through the world of tenure from the University of Chicago and by his fellow citizens through the award of the Early Career Award for scientists and engineers from President Bill Clinton. Philip is the inventor of CAR-T reg targeting brain glial cells to shut down neuroinflammation which is a cause of all neurodegenerative diseases. He built Smith Therapeutics around immunotherapy in neurodegenerative diseases such as Alzheimer’s, Parkinson’s and ALS. It was acquired by AZ Therapies, a clinical stage biopharmaceutical company developing novel therapies for neurodegenerative diseases. Philip joined AZ Therapies to advance the CAR-T reg program into the clinic for the treatment of rare and common neurodegenerative diseases.

2:05pm – 2:35pm

1-2-1 Meetings

2:35pm – 3:05pm

1-2-1 Meetings

3:10pm – 4:10pm

De-risking preclinical models for better results

Exploring the full circle of data approaches over the last few years from complex behavioural biology to specific circuit based approaches
Understanding how much to invest in preclinical data generation to reduce risk of clinical failure
Comparing preclinical approaches for neurology and psychiatry

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.

4:15pm – 4:45pm

Closing Keynote & Address:

Clinical Studies

Time

Titles and Bullets

Facilitator

8:30am – 9:00am

Opening Address & Keynote:

9:05am – 10:05am

Choosing your patient population – how early should we go?

Selection of patients vs. healthy volunteers for Phase 1 studies
Optimizing study design to incorporate broad phenotypic expression without cannibalizing signal detection
Deciding when to intervene in the disease process
Combining pediatric and adult protocols
Chronic dosing and tolerance

Kemi Olugemo

Executive Medical Director, Neurology
Akcea Therapeutics

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).

10:10am – 10:40am

1-2-1 Meetings

11:50am – 12:20pm

1-2-1 Meetings

12:20pm – 1:00pm

Networking Lunch

1:00pm – 2:00pm

Developing a selection of meaningful endpoints for clinical design

How do you define a meaningful and quantifiable endpoint for patients?
How do incorporate patient input into this outcome measure?
How do systemise this process in your clinical development?

Asif Paker

Vice President, Clinical Development
Swan Bio Therapeutics

Asif Paker is VP Clinical Development at SwanBio therapeutics where he is responsible for developing gene therapies for rare neurological disorders. Asif has more than 10 years of drug development experience in rare diseases both in academia and the biopharmaceutical industry. Before joining SwanBio, Asif served as VP clinical development at Ovid Therapeutics where he led global clinical development for Ovid’s epilepsy franchise. Prior to Ovid, Asif led the clinical teams to develop therapies in Adrenoleukodystrophy and schizophrenia at Bluebird Bio and Alkermes respectively. In academia, he was a key team member on the clinical trials of Lorenzo oil in Adrenomyeloneuropathy patients, and DHA in children with peroxisomal disorders at Johns Hopkins Medical Institution. Prior to his career in clinical development, Asif was with UNICEF and Médecins du Monde where he served in various roles to advocate health policies to national governments and to support humanitarian aid programs. Asif is a neurologist and he completed his training at the University of Texas and at the Harvard University program at Massachusetts General, Brigham & Women’s and Boston Children’s Hospitals.

2:05pm – 2:35pm

1-2-1 Meetings

2:35pm – 3:05pm

1-2-1 Meetings

3:10pm – 4:10pm

Tailoring strategies for successful CNS clinical development

Optimal approaches for incorporating biomarkers into early phase development
Developing unique clinical designs for early proof of concept studies in CNS diseases
Incorporating translational studies for novel modalities including gene and cell therapies as CNS therapeutics

Christopher Winrow

Senior Director, Clinical Development, Neuroscience Program Lead
Cyclerion

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.

4:15pm – 5:15pm

Closing Keynote & Address:

Targeted Therapies

Time

Titles and Bullets

Facilitator

8:30am – 9:00am

Opening Address & Keynote:

9:05am – 10:05am

Translating the biology of target pathways into therapeutics

How do we identify a potential therapeutic target?
What are our expectations for animal studies as a means of increasing probability of success in humans?
How does the identification of a biomarker impact clinical studies
How successful has translational studies been in Neuroscience?

Richard Malamut

Chief Medical Officer
Collegium Pharmaceutical

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.

10:10am – 10:40am

1-2-1 Meetings

10:45am – 11:45am

Gene therapy for rare neurological disorders

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

Jim McNally

Chief Scientific Officer
BioAgilytix

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

11:50am – 12:20pm

1-2-1 Meetings

12:20pm – 1:00pm

Lunch Break

1:00pm – 2:00pm

Developing gene-targeted therapy approaches for CNS disorders

The challenges of cell and gene therapy
The opportunities cell and gene therapy
How to select targets
Matching MoA to indication

Brendon Boot

Senior Medical Director
Blue Rock Therapeutics

Brendon Boot, is a clinician-scientist with Australian board-certification in Neurology and Internal Medicine. He is a neurologist at Brigham and Women’s Hospital in Boston and an Assistant Professor of Neurology at Harvard Medical School. Brendon has 15 years’ experience in design and implementation of clinical trials in academic and industry settings. For the last five years, he was the Medical Director of high-profile pre-clinical and phase 1-4 programs at Biogen, Voyager Therapeutics, SSI Strategy and BlueRock Therapeutics focusing on Alzheimer’s Disease, Parkinson’s Disease and Immunological programs. He publishes on the clinical and genetic risk factors, biomarkers, progression and treatment of these diseases.

He is a member of the Alzheimer’s Association Medical and Scientific Committee (New England), and serves on the Scientific Advisory Board for the XDP Consortium, Blackfynn, Bioverge and the Boston Harbor Angels.

Brendon attended medical school at The University of Sydney and did his residency in Sydney and in Toulouse, France, with fellowships at the Royal Prince Alfred Hospital, Sydney, and Mayo Clinic, MN.

2:05pm – 2:35pm

1-2-1 Meetings

2:35pm – 3:05pm

1-2-1 Meetings

3:10pm – 4:10pm

Exploring the potential & current limitations of cell & gene therapies

What’s the status of the current technology and what can be achieved today and in the near future?
Comparing approaches for genetic diseases versus acquired conditions
Exploring the ethics around permanent modification

Olivier Danos

Senior Vice President & Chief Scientific Officer Regenx Bio

Olivier Danos is a pioneer in the field of gene therapy and has dedicated his career to advancing the use of this technology to develop life-saving therapies for patients. Olivier joined REGENXBIO in 2017 from Biogen where he was a Senior Vice President in charge of Cell and Gene Therapy. Over the past twenty years, he has played leadership roles in cell and gene therapy as Director of the Gene Therapy Consortium of the University College of London, at the Necker Hospital – Enfants Malades in Paris, as Chief Scientific Officer of Genethon and as senior director of research at Somatix Therapy Corporation. He has held senior research positions in France at the Centre National de la Recherche Scientifique and at the Institut Pasteur. Olivier is the former President and a founding member of the European Society of Gene and Cell Therapy.

Olivier received a Master’s in Genetics and Molecular Biology at University of Paris Orsay, and his Ph.D. at the Pasteur Institute and University of Paris Diderot.

4:15pm – 4:45pm

Closing Keynote & Address:

Managing Regulatory Pathways

Time

Titles and Bullets

Facilitator

8:30am – 9:00am

Opening Address & Keynote:

3:10pm – 4:10pm

The evolution of substantial evidence for drug registration and labeling

The standard evidence consisting of 2 adequate and well controlled investigations is changing
Emerging criteria to demonstrate effectiveness based on a single trial with confirmatory evidence
What this confirmatory evidence can be and the flexibility shown by the Agency on how and when this criteria is applied

Douglas Harnish

Vice President Regulatory Affairs, Head Neurology, Pain & Migraine
Teva Pharmaceuticals

4:15pm – 4:45pm

Closing Keynote & Address:

CNS Therapeutics Xchange

Biomarkers

Translational Research

Clinical Studies

Targeted Therapies

Managing Regulatory Pathways

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