Program
Tuesday, May 4, 2021 – all times in US Eastern Time
Session/Lecture
11:00 AM ET
11:15
11:55
11:55
12:00 PM
12:25
12:50
1:15
1:35
2:00
2:00
2:05
2:30
2:55
3:20
3:45
4:05
5:00
Chair/Speaker
11:00 AM ET
11:15
11:55
11:55
12:00 PM
12:25
12:50
1:15
1:35
2:00
2:00
2:05
2:30
2:55
3:20
3:45
4:05
5:00
Wednesday, May 5, 2021 – all times in US Eastern Time
Session/Lecture
11:00 AM ET
11:40
11:40
11:45
12:10
12:35
1:00
1:20
1:45
1:45
1:50
2:15
2:40
3:05
3:30
3:50
4:00
5:00
Chair/Speaker
11:00 AM ET
11:40
11:40
11:45
12:10
12:35
1:00
1:20
1:45
1:45
1:50
2:15
2:40
3:05
3:30
3:50
4:00
5:00
SCIENTIFIC ADVISORY COMMITTEE
Howard Fillit, MD—ADDF
Mark Forman, MD, PhD—ADDF
Suzana Petanceska, PhD—NIH/NIA
Andrew Pieper, MD—Harrington Discovery Institute
Edward Spack, PhD—Vector BioSolutions/Therini
Leticia Toledo-Sherman, PhD—Rainwater Charitable Foundation
Alessio Travaglia, PhD—ADDF
D. Martin Watterson, PhD—Northwestern University
CONFERENCE INTRODUCTION
Tuesday, May 4, 2021
11:00 US Eastern Time

Howard Fillit
Alzheimer’s Drug Discovery Foundation
Dr. Howard Fillit is ADDF’s Founding Executive Director and Chief Science Officer. His introduction will provide a welcome to our 15th Annual Drug Discovery for Neurodegeneration Workshop, a brief update on ADDF’s recent progress and will highlight the framework for this year’s meeting.
KEYNOTES


Matt Troyer
Denali
Bio
Matt Troyer, MD, leads Early Clinical development at Denali and is responsible for overseeing a team of physician-scientists who execute Denali’s early clinical programs in neurodegenerative disease. The Early Clinical group partners with cross functional teams to design biomarker-driven clinical development strategies and conducts first-in-human studies through investigations in Alzheimer’s disease, Parkinson’s disease, ALS and rare neurodegenerative disorders.
Dr. Troyer is a neurologist with subspecialty training in Parkinson’s disease and Movement Disorders and 15+ years of clinical development experience. Matt received a BA in biology from Illinois Wesleyan University and an MD from Stanford University. He completed neurology
residency training at Harvard Medical School in the Longwood Area Program and completed a fellowship in Parkinson’s disease and Movement disorders at the Institute of Neurology, Queen Square, London. Following his clinical fellowship, Matt did a postdoctoral research fellowship in the laboratory of Robert Edwards at UCSF, focusing on basic mechanisms of presynaptic function and Parkinson’s disease. Matt subsequently joined the Neurology faculty at UCSF with both clinical and research responsibilities. Matt later moved to industry where he led early stage neuroscience programs, both at Merck as Executive Director and Neuroscience Therapeutic Area Lead for Early Clinical Development, and then at Medivation, prior to joining Denali in 2016.
Susan Bates
Columbia University
Bio
Dr. Susan Bates received her MD degree from the University of Arkansas School of Medicine. She completed her clinical training in internal medicine at Georgetown University in Washington, DC, and in medical oncology at the National Cancer Institute (NCI) in Bethesda, MD. Dr. Bates was a Lead Clinical Investigator and Head of the Molecular Therapeutics Section in the Developmental Therapeutics Branch of the Center for Cancer Research before moving to Columbia University in August 2015.
During her years at the NCI, Dr. Bates led a highly successful translational research program focused on mechanisms of multidrug resistance and approaches to evaluate and improve the activity of epigenetic modifying agents. Her laboratory was among the first to clone the multidrug transporter ABCG2, eventually characterizing its function and its role in chemo-resistance and chemo-protection. This effort built upon earlier work elaborating the role of the multi-drug transporter P-glycoprotein that had defined the drug sensitivity profiles of cell lines in vitro, particularly in the NCI-60 cell line panel. The latter observation continues to impact how the NCI-60 cell line panel is used in drug discovery, and helped her identify a novel agent, at that time known as depsipeptide. Dr. Bates brought this drug to the clinic and after completing its phase I testing, served as Principal Investigator of a multi-institutional, international Phase II study of romidepsin (depsipeptide) in cutaneous and peripheral T-cell lymphoma. Working with Gloucester Pharmaceuticals, the data from this study were included in New Drug Applications (NDA) to the U.S. Food & Drug Administration (FDA). This partnership led to approval by the FDA of romidepsin for two indications – initially for cutaneous T-cell lymphoma and later for peripheral T-cell lymphoma.
Dr. Bates’ current interests are both laboratory and clinical in nature. Her laboratory efforts include laboratory and translational studies on drug resistance in T-cell lymphomas and advanced solid tumors including breast, pancreatic, neuroendocrine, renal and lung cancers. Her work is dedicated to new drug development, and finding antineoplastic agents that, alone or in combination with other anticancer agents, improve the options available for difficult to treat cancers. Emanating from the clinical and translational development of romidepsin, a histone deacetylase (HDAC) inhibitor, a current focus is on epigenetic therapies, and the development of combination therapies to use with HDAC inhibitors in refractory advanced cancers, including solid tumors. She also has a special interest in drug delivery and drug distribution and the role of the blood brain barrier in creating a sanctuary site for cancers that metastasize to the brain. Clinically, her goal has always been to translate ideas from the laboratory to clinical trials, an effort that has proven very successful. Clinically she seeks to develop combination therapies with histone deacetylase inhibitors for the therapy of solid tumors; and to develop therapies to treat central nervous system metastases, a complication of cancer that is becoming a greater problem as patients live longer with cancer.
Delivery of Protein Therapeutics to the CNS
Tuesday, May 4, 2021
11:15 US Eastern Time
Epigenetic Treatments for Neurodegenerative Diseases – What Can We Learn from Oncology?
Wednesday, May 5, 2021
11:00 US Eastern Time
SESSION I: EMBARKING ON A DRUG DISCOVERY CAMPAIGN
Tuesday, May 4, 2021
11:55 – 13:35 US Eastern Time



Bio
D. Martin Watterson
Northwestern University
CHAIR
Session Overview
Bio
Rouba Kozak obtained her PhD in neuroscience and eating behavior from Henri Poincare University in France, and has an extensive background in cognitive neuroscience with focus on translational research. Specifically, she has focused on bridging the gap between mechanistic studies in preclinical animal models, which provide exquisite detail on how brain circuits control discrete behaviors, and the measurement of functional impact of new therapies in clinical settings (e.g. neuro-degenerative and neuro-psychiatric patient populations).
Dr. Kozak’s scientific training ranged from studies on the mechanisms of obesity to cognition, employing state-of-the- art behavioral and neurochemical sampling techniques; providing her with a unique ability to conceptualize and design experiments in support of programs spanning the wide-ranging disease domains covered in the Takeda portfolio. She has utilized complex behavioral and neurochemical methods to test hypotheses regarding etiologies of neuropsychiatric disorders, as well as to develop translational models to test the efficacy of novel pharmacotherapeutics for the enhancement of cognitive function.
Rouba Kozak
Takeda
Bio
Dr. Tilmann Brotz is a Principal and senior Development Sciences Consultant at PharmaDirections. He has over 20 years of experience in the biopharmaceutical industry leading toxicology, DMPK, in-vivo pharmacology, clinical pharmacology and diagnostic groups.
Most recently Dr. Brotz was Senior Director, Development Sciences at Achaogen where he contributed to multiple antibiotics programs from lead optimization through Phase 3. Prior to Achaogen, he worked as head of preclinical development and as a consultant for numerous emerging companies, including VIA Pharmaceuticals where he led the non-clinical development and clinical biomarker efforts for a clinical Phase 2 5-lipoxygenase inhibitor.
Dr. Brotz is an advisor for the CLSI FAST Program and a member of the Scientific Review Board of the Alzheimer’s Drug Discovery Foundation.
Tilmann Brotz
PharmaDirections

Bio
Susan Catalano
Cognition Therapeutics
SESSION II: REPURPOSING AND REPOSITIONING
Tuesday, May 4, 2021
14:00 – 16:05 US Eastern Time



Bio
Leticia Toledo-Sherman
Rainwater Charitable Foundation
CHAIR
Session Overview
Bio
Clive Ballard
University of Exeter
Bio
Arnab K. Chatterjee completed a Bachelor of Arts in Chemistry and minor in Business from Northwestern University in 1997 and a brief period as an IT consultant, he proceeded on to the Caltech to conduct his chemistry doctoral thesis research in the laboratory of Professor Robert H. Grubbs in from 1999-2002 (Professor Grubbs was awarded the Nobel Prize in Chemistry in 2005 for his work in the area olefin metathesis). Arnab joined the Genomics Institute of the Novartis Research Foundation (GNF) as a Principal Investigator in the chemistry department. His work over of 9+ years has been focused on hit-to-lead and lead optimization in several medicinal chemistry projects ranging a variety of therapeutic areas (neuroscience, oncology, respiratory disease and infectious diseases). The project teams he has worked with have produced 8 preclinical candidates optimized for both inhaled and oral formulations with three novel first-in-class compounds in clinical trials including KAF156 currently in Phase 2b a first-in-class antimalarial.
Since May 2012 he has been responsible for setting up and leading the chemistry group at the Calibr in La Jolla, CA working across a wide-variety of disease areas including three compounds in clinical trials and three in IND-enabling studies in the areas of malaria, HIV, regenerative medicine and most recently for SARS-2. His research interests include application of novel synthetic methods to expedite the structural diversification in medicinal chemistry, cell-based lead optimization, advanced formulation methods for delivery of small molecules and drug repurposing.
Arnab Chatterjee
Calibr


Bio
Sharon Rosenzweig-Lipson
AgeneBio
Bio
Rob Berman, MD
BioHaven Pharmaceuticals
SESSION III: BIOMARKERS TO ENABLE PRECLINICAL AND CLINICAL PROOF OF CONCEPT STUDIES
Wednesday, May 5, 2021
11:40 – 13:20 US Eastern Time



Bio
Dr. Edward (Ted) Spack has over 25 years of biotech translational experience, including preclinical development of drug candidates for multiple sclerosis, nosocomial infection, and biodefense. At SRI International, Dr. Spack directed the PharmaSTART program (a consortium of SRI, Stanford, UC Berkeley, UC San Diego, and UC San Francisco), drafting preclinical development blueprints that led to several major grants and new biotech companies. He has consulted with the NIH translational core services committee and several NIH institutes on preclinical development and serves on several study sections, including the NIA Alzheimer’s Disease Drug Development review panel, the NIH Small Business Review on Drug Discovery for Aging, Neuropsychiatric and Neurological Disorders, and the Falk Trust Catalyst and Transformational Award programs.
As Managing Director of an innovative partnership between the National Multiple Sclerosis Society and EMD Serono he supported innovative early stage MS drug discovery and development projects in academic labs and biotech companies. Through the California Life Sciences Institute (CLSI) FAST program and the SRI Innovation program he mentors SF Bay area and international academic and industry teams in biotech company formation and pitch decks.
Dr. Spack received his doctoral degree from The Johns Hopkins University and his postdoctoral fellowship in cellular immunology at Stanford University.
Edward (Ted) Spack
Vector BioSolutions/Therini
CHAIR
Session Overview
Bio
Darrell A. Henze, PhD, is the Director of Neuroscience, leading the Sensory Biology and Neuropharmacology group in the Department of Neuroscience at Merck & Co., Inc. Dr. Henze graduated Magna Cum Laude from The University of Rochester and earned his PhD from the University of Pittsburgh in 1998. His PhD work focused on understanding the synaptic properties of the hippocampal Mossy Fiber synapse. He completed a post-doctoral fellowship at Rutgers, The State University of New Jersey working in the laboratory of Dr. Gyorgy Buzsaki focusing on in vivo electrophysiological techniques to study how single cells contribute to network activity.
In 2002, he joined Merck where he worked on discovery programs in the therapeutic areas of schizophrenia, Parkinson’s disease, and Alzheimer’s disease. In 2006, he moved to join the pain and migraine research group providing discovery project leadership. In 2013, he took on the lead role for the Late Discovery Pain and Migraine Department. In 2016, he transitioned to leading an experienced group of in vivo pharmacologists providing pharmacodynamic and translational biomarker support across the entire neuroscience portfolio, leveraging rodent, dog, and primate models to support the discovery and development of therapeutics for neurodegeneration, cognition, psychiatry, and pain. Most recently, in the fall of 2020, he has resumed responsibility for the discovery portfolio for pain and itch as well as leading in vitro electrophysiology and in vivo pharmacology teams supporting a range of neuroscience programs including neurodegeneration.
Darrell Henze
Merck
Bio
Michael Gold, MS, MD, is currently Vice-President, Development Neurosciences at AbbVie. Before this, Dr. Gold spent time at PPD, UCB Bioscience and Allon Therapeutics. At UCB, he served as vice president of and head of CNS practice, and at Allon Therapeutics, he served as Chief Medical Officer. From 2005-2011, Dr. Gold was with GSK, where he served as medicines development leader for Alzheimer’s disease and vice president of neurosciences, medicines development center. Among his many responsibilities at GSK, he was accountable for all aspects of the execution of the clinical development plans for two late stage assets in the area of Alzheimer’s disease. Earlier in his career, he held positions of increasing responsibility within clinical research at J&J and Bristol-Myers Squibb.
Michael Gold
AbbVie

Bio
Philip Scheltens, MD, is Professor of Cognitive Neurology and the Director of the Alzheimer Center at the VU University Medical Center in Amsterdam. He is also the initiator and Vice President of Deltaplan Dementia Netherlands. In 2011, Dr. Scheltens was appointed scientific director at the Pearlstring Institute and elected a Member of the Royal Netherlands Academy of Arts and Sciences, where he is now a member of the Board. He is an Honorary Professor of Neurology at University College London.
His main clinical and research interests are Alzheimer’s disease, vascular dementia, frontotemporal dementia, magnetic resonance imaging, PET imaging and biomarkers for disease detection and early diagnosis. He is active in the field of biomarkers and clinical trials and has been the national Primary Investigator for many trials, including phase 1-3 international multicenter clinical trials.
Dr. Scheltens is an active collaborator and contributor to many fields and is currently a member of various research societies including the European Academy of Neurology, the Dutch Society for Neurology, the American Academy of Neurology, the Alzheimer Imaging Consortium, and the ISTAART Consortium. He is also a jury member of the Brain Prize and a member of the organizing committee for the VASCOG conference 2016 in Amsterdam. From 2017 he is (again) a member of the AAIC program committee.
Dr. Scheltens received his MD in 1984 and his PhD in 1993 from the VU University in Amsterdam.
Philip Scheltens
VUMC Alzheimer’s Center
A Path Toward Precision Medicine in Alzheimer’s Disease
SESSION IV: CASE STUDIES – CHASING NEW PATHWAYS
Wednesday, May 5, 2021
13:45 – 15:50 US Eastern Time



Bio
Andrew Pieper, MD, PhD, is a board-certified psychiatrist and neuroscientist in the Department of Psychiatry at Case Western Reserve University and University Hospitals Cleveland Medical Center, where he serves as Morley Mather Chair in Neuropsychiatry. He also serves as Psychiatrist at the Louis Stokes Cleveland VA Medical Center (VAMC), as well as Investigator in the University Hospitals Harrington Discovery Institute, where he is the Director of Neurotherapeutic Discovery.
Dr. Pieper is devoted to patient care and basic science research applied to disease, most keenly focused on neurodegeneration. Clinically, he maintains an active Adult Psychiatry Outpatient Clinic at the VAMC. His goal is to understand and investigate human disorders in order to foster development of new pharmacologic treatments for patients.
Andrew Pieper
Harrington Discovery Institute
CHAIR
Session Overview
Bio
Siew Peng Ho obtained her PhD in synthetic organic chemistry with Donald Cram at UCLA. Her post-doctoral training was with Bill DeGrado on de novo protein design at the Central Research Department at DuPont. Soon after, she began working on nucleic acid therapeutics first at DuPont Pharmaceuticals and later at Bristol-Myers Squibb. She is currently a consultant in nucleic acid therapeutics, largely with Harrington Discovery Institute.
Siew’s comprehensive expertise over 30 years encompasses design, synthesis, cellular and in vivo studies with these molecules. Her group validated over 40 different gene targets in in vivo models for drug discovery programs using antisense and siRNA oligonucleotides in cardiovascular diseases, metabolic diseases, neuroscience and virology. She was also intimately involved in BMS’ therapeutic collaborations with Ionis (drug candidate advanced to Phase 1) and Santaris (now part of Roche).
Siew Peng Ho
Harrington Discovery Institute
Bio
Dr. Travis Dunckley obtained his undergraduate degree in molecular and cellular biology and biochemistry from the University of Arizona. His doctoral work was completed at the University of Arizona in the field of molecular and cellular biology. Prior to joining TGen, he studied gene expression at the level of mRNA stability. In 2000, Dr. Dunckley expanded his training to the field of Neurobiology during a fellowship position at Barrow Neurological Institute where he studied structure/function interactions in nicotinic acetylcholine receptors (nAChR) and identified novel genes regulated by nAChR activity. In 2003 he transitioned to the Neurogenomics division at TGen to leverage the rapidly developing genomics field for the identification of relevant therapeutic targets and biomarkers for Parkinson’s disease and Alzheimer’s disease. He has developed efficient single cell isolation and transcriptome profiling methods, high-throughput functional assays directly applicable to relevant phenotypic endpoints in neurodegenerative diseases – including α-synuclein accumulation and tau hyperphosphorylation, and he has performed large scale association studies to identify DNA variants underlying neurodegenerative disease.
Dr. Dunckley has recently moved to the Neurodegenerative Disease Research Center at the Biodesign Institute at Arizona State University where ongoing work involves the role of epigenetics as a potentially modifiable factor contributing to neurodegeneration and the development of novel small molecule inhibitors of the DYRK1A kinase for potential therapeutic applications in Alzheimer’s disease and Down syndrome.
Travis Dunckley
Arizona State University


Bio
Toren Finkel received his undergraduate degree in Physics and his MD and PhD degree from Harvard Medical School. Following a residency in Internal Medicine at the Massachusetts General Hospital, he completed a fellowship in Cardiology at Johns Hopkins Medical School. In 1992, after completing his clinical training, he came to the NIH as an Investigator within the Intramural Research Program of the National Heart, Lung and Blood Institute (NHLBI). Over the next 25 years at the NIH, he held various positions including Chief of the Cardiology Branch and Chief of the Center for Molecular Medicine within the NHLBI. He has been inducted into the American Society for Clinical Investigation (ASCI) and the Association of American Physicians (AAP). Toren is also a Fellow of the American Association for the Advancement of Science (AAAS) and an elected member of the National Academy of Medicine. He serves on numerous editorial boards including currently serving on the Board of Reviewing Editors for Science.
As of Sept 1, 2017, Dr. Finkel assumed the role of the Director of the Aging Institute, and the G. Nicholas Beckwith III and Dorothy B. Beckwith Endowed Chair of Translational Medicine at the University of Pittsburgh/UPMC. Over the last three decades, his laboratory has made fundamental contributions in our understanding of the role of reactive oxygen species and mitochondrial function in aging and age-related diseases. According to Google Scholar, he currently is one of the highest cited authors in both cardiovascular disease and in aging. Toren also co-founded and is the CSO of Generian Pharmaceuticals. With his colleagues Bill Chen and Yuan Liu he identified a small molecule activator of TFEB which is anticipated to be in Phase I testing within 12 months and will be of potential benefit for a range of age-related disorders including neuro-degenerative diseases.
Toren Finkel
Generian
Small Molecules to Modulate Autophagy and Lysosomal Biogenesis in LBD and AD
Bio
Jamie Bilsland, PhD, is the Chief Scientific Officer of AstronauTx, a biotechnology firm dedicated to unlocking the therapeutic potential of astrocytes in neurodegenerative disease.
Dr. Bilsland has worked in the field of neuroscience drug discovery for over 25 years, with a focus on neurodegenerative diseases and pain. He spent over 20 years in large pharmaceutical companies (Merck, Pfizer) then moved into academic drug discovery as Head of Neurodegeneration Biology at the Alzheimer’s Research UK funded Drug Discovery Institute at UCL. He joined AstronauTx in 2019.
Jamie Bilsland
AstronauTx