CNS THERAPEUTICS XCHANGE
WEST COAST
San Francisco
September 27, 2023

Welcome to hubXchange’s CNS Therapeutics Xchange West Coast 2023, 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 and Drug Delivery.

Take advantage of this unique highly interactive meeting format designed for maximum engagement, collaboration and networking with your peers.

Please note this is an In-Person meeting.

VENUE DETAILS: DoubleTree by Hilton San Francisco Airport, 835 Airport Blvd, Burlingame CA 94010-9949

SNAPSHOTS OF DISCUSSION TOPICS

Track 1: Novel Targets

Novel therapies for complex neurological disorders: discovery of effective treatments
Oligonucleotides targeting microRNA: opportunities and challenges

Track 2: Preclinical

Methods to increase the success rates of drugs entering clinical phases
Novel strategies for reverse translation of temporal biomarkers for CNS drug development

Track 3: Clinical Studies

Fluid and digital biomarkers in neurodegenerative disorders
Utilizing clinical biomarkers to enhance drug development in neurodegenerative diseases

Track 4: Drug Delivery

Transcytosis-mediated brain delivery for proteins, antibodies, oligonucleotides, and small molecules
How AI can improve drug discovery

Full Xchange Agenda

Click on each track for detailed agenda

Novel Targets

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation by Axol Bioscience
Using Human iPSCs to Build a Clinical Trial in a Dish for Alzheimer’s Patient Stratification

At Axol Bioscience, we use human induced pluripotent stem cells (iPSCs) to build
better human disease models to support the drug discovery journey. With a specific interest in neurodegenerative diseases including Alzheimer’s and ALS, we have created in vitro models which act as human-like assay platforms for compound screening. Now with access to >100 donated samples from sporadic Alzheimer’s Disease patients, we’ve building towards a vision of bringing a new tool to patient stratification, with the aim of de-risking clinical trial selection and bolstering trial success.

Ashley Barnes

Chief Scientific Officer, Axol Bioscience

Ashley has over 30 years experience in drug discovery with the last 7 years focused on using iPSCs. Prior to this Ashley was at GlaxoSmithKline for 20 years where he led projects and teams across a range of diseases including Respiratory and Neuroscience.

9:05 – 10:05

Pills don’t teach skills – The promise of tech-augmented drug therapies for CNS

What are tech-augmented drug therapies and why should I care?
What is the value to biopharma and what are some examples?
What are key considerations in developing a Rx-DTx therapy? What are the known knowns, known unknowns and, likely, unknown unknowns?

Mikael Eliasson

Managing Partner, Redwood Group

Mikael Eliasson, MD, Ph.D., is an experienced healthcare innovator with a track record of transforming ideas into billion-dollar businesses in biotech, meditech, and diagnostics. He uses his in-depth expertise in both R&D and commercial to be an “innovation broker” that fosters interaction between ideas and people.

He has spearheaded technology integration into CNS drug development at Big Pharma and start-ups, incl. initiatives involving machine learning, digital therapeutics, and decentralized trials. At Roche, he created their first tech-augmented therapies for autism, and a rare neuromuscular disease.

Mikael trained as a neurosurgeon and neuroscientist before switching to venture capital and later industry.

10:10 – 10:40

1-2-1 Meetings/Networking Break

10:40 – 11:10

1-2-1 Meetings / Networking Break

11:10 – 11:20

Morning Refreshments

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Spotlight Presentation by Invicro

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:00 – 15:30

Poster Presentation by Lifescience Dynamics

Navigating neurological disorders: Advancements in biomarker discovery for diagnosis and targeted therapy

Explore the pivotal role of biomarkers in the dynamic landscape of neurology. This informative poster delves into the multifaceted applications of biomarkers, showcasing their potential to revolutionize the diagnosis, prognosis, and monitoring of disease progression and treatment response in neurology clinical practice.
From unravelling the mysteries of complex neurological disorders to guiding personalized therapeutic decisions, biomarkers offer a transformative lens through which we can understand and address these intricate conditions.

Vicki Tsao

Consultant, Lifescience Dynamics

Vicki is a Consultant with 5+ years in neuroscience research and 3+ years in life science consulting. Her prior research focused on neurotransmitter receptors uncovering molecular mechanisms underlying ion channel function and drug modulation. Vicki has work experience in drug development and business strategies with early-stage biotechnology companies. At Lifescience Dynamics, Vicki has worked and managed multiple projects, including neuroscience, helping clients understand the current and emerging competitive landscapes and assess companies’ clinical development and commercial activities.
Vicki has a PhD in Physiology, University of Wisconsin-Madison and a BSc in Life Science, National Taiwan University

Peter Kelly

Principal, Lifescience Dynamics

Peter is a Principal with 10+ years in biomedical science and 7+ years in the financial services consulting at the Bank of England. He is published in a wide range of scientific fields while at Stanford, Memorial Sloan-Kettering, and University College London. At the Bank, Peter led teams responsible for supervising systemically important financial institutions, which involved business model and strategic analysis. At Lifescience Dynamics, Peter has designed and led consulting projects is a wide range of therapy areas, including neurology, to help clients navigate commercial and clinical challenges and implement a successful strategy.
Peter has a BA in Molecular & Cellular Biology, University of California, Berkeley and a PhD Biochemistry, University College London.

15:30 – 15:40

Afternoon Refreshments

15:40 – 16:40

Novel therapies for complex neurological disorders: discovery of effective treatments

The challenges and opportunities for human IPSCs as a platform for novel target discovery
The rise, fall, and resurrection of organoids as a platform for novel target discovery and validation
Emerging platforms for novel target discovery

Orion Keifer

Senior Medical Director, Head of Translational Medicine – Neuroscience, Insitro

16:40 – 17:40

Drinks-Canape Reception

Preclinical

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation by Axol Bioscience
Using Human iPSCs to Build a Clinical Trial in a Dish for Alzheimer’s Patient Stratification

Ashley Barnes

Chief Scientific Officer, Axol Bioscience

09:05 – 10:05

Methods to increase the success rates of drugs entering clinical phases

What is the biggest hurdle so far to increase the success rate of drugs entering clinical trials?
What are we doing to overcome the hurdles right now?
What lesson have we learned from working on tackling the hurdles?
What can we do in the next 3-5, or 10 years to increase the success rate?
Any new methods, approaches, or technologies you want to share that can improve the success rate?

Tao Huang

President and Chief Executive Officer, NeuCyte

Tao Huang is President and CEO of NeuCyte, a biotechnology company that focuses on developing treatments for those suffering from neurological and neurodegenerative disorders. NeuCyte develops translatable cell-based assays for modeling CNS-related disorders using patient-derived and genetically engineered neural cell types to discover novel therapeutics. Based on SynFire® technology, its proprietary human neural in vitro platforms assess cell autonomous, non-cell autonomous and complex electrophysiological and morphological readouts suited for target identification and validation, as well as drug efficacy testing. It is actively pursuing drug discovery programs on Alzheimer’s disease, ALS/FTD, Autism, and Epilepsy.
Tao obtained his Ph.D. in Biochemistry from Peking Union Medical College, and J.D. from the University of Michigan Law School. He received his post-doctoral trainings from the University of Colorado School of Medicine and Princeton University.

10:10 – 10:40

1-2-1 Meetings/Networking Break

10:40 – 11:10

1-2-1 Meetings / Networking Break

11:10 – 11:20

Morning Refreshments

11:20 – 12:20

How can EEG and novel cutting-edge techniques transform the way drug candidates are developed until clinical phase

Pitfalls in drug development: Are they evolving?
Making the fields of EEG and drug development more objective
What are the most promising areas for this marriage of AI and EEG?
Do precision medicines require precision measurements?

Julien Volle

Head of Technology, SynapCell

Julien holds a PhD in Neuroscience from the University of Lyon, France. Julien joined SynapCell in 2017 after two post-docs in different structures in Toronto. Since then, he manages R&D Preclinical projects and develops new innovative solutions for sponsors. With over 12 years’ experience in the field, he is well-versed in the emerging technologies and industry trends.

Hugo Monchal

Scientific Sales Manager, SynapCell

After a MSc in Neuroscience and in international business in science, Hugo now manages the USA and LATAM zone. His background allows him to understand the current needs of the biopharma industry towards drug discovery for CNS disorders, and how to address them.

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Spotlight Presentation by Invicro

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:00 – 15:30

Poster Presentation by Altasciences

The nonhuman primate model of CNS therapies and utility of adeno-associated viral (AAV) vectors in gene therapy: From discovery to IND-enabling studies

Making the transition from exploratory discovery to GLP studies requires close coordination between discovery and toxicology. Discovery require specialized delivery techniques and fewer animals; IND-enabling studies transition to standard routes of administration. MRI-guided technologies allow delivery into specific areas of the brain, while intrathecal dosing allows delivery to reach the cerebrospinal fluid via injection into the spinal cord. In this presentation, we will provide examples of the specialized delivery methods in the nonhuman primate model of gene and cell therapy. We reviewed data collected in a large number of studies conducted in the past few years with the aims of (1) establishing ranges for the number of animals to screen for nAb; (2) establishing the dosage range for commonly used corticosteroid given prior to AAV vector administration. Approximately 37% (182/491) of animals screened for nAb against AAV8 were suitable for study assignment based on established criteria for negative or low viral titers by AAV neutralizing antibody assay (≤ 5 nAb₅₀ in HEK293 cells). Pretreatment with 2 mg/kg of dexamethasone at approximately 1-2 hours prior to AAV administration was adequate to mediate immune related responses.

Norbert Makori

Norbert Makori, Vice President of Toxicology, Altasciences

Norbert Makori is Vice President of Toxicology, Altasciences. He has over 20 years of industry experience as a toxicologist (general and reproductive toxicology) and immunotoxicology. Prior to joining Altasciences, Norbert was Director of the General Toxicology group at Charles River Laboratories in Ashland, Ohio. Prior to that, he held the position of Director of Toxicology and Immunotoxicology at WIL Research Laboratories. Norbert is a Diplomate of the American Board of Toxicology (DABT), trained as a veterinarian and has a PhD in Comparative Pathology from the University of California in Davis.

15:30 – 15:40

Afternoon Refreshments

16:40 – 17:40

Drinks-Canape Reception

Clinical Studies

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation by Axol Bioscience
Using Human iPSCs to Build a Clinical Trial in a Dish for Alzheimer’s Patient Stratification

Ashley Barnes

Chief Scientific Officer, Axol Bioscience

09:05 – 10:05

Emerging novel biomarkers and technology platforms bringing precision medicine to neurology

How (and why) has the recent emergence of precision biomarkers in neurology changed the regulatory landscape and drug approval process?
What type(s) of precision biomarkers are most critical for de-risking programs early in the clinic?
How can we think about developing novel precision biomarkers and biological definitions for neurological diseases?

Sean Pintchovski

Vice President of Biology, Acurex Biosciences

Sean Pintchovski, PhD, has over 15 years of experience in the pharmaceutical industry. During this time, he successfully led pre-clinical research teams to discover, validate, and advance several small molecules into the clinic, including PTC857 (GBA-Parkinson’s disease and Amyotrophic Lateral Sclerosis) and PTC743 (Friedreich’s ataxia). Subsequently, Dr. Pintchovski contributed within cross-functional clinical teams to identify novel biomarkers and next-generation technologies needed to advance the use of precision medicine in neurology. As VP of Biology at Acurex Biosciences, he now merges these experiences to effectively lead a biology team focused on the rapid development of first-in-class drug-responsive peripheral biomarkers and novel therapeutics for the early diagnosis and treatment of Parkinson’s disease. Dr. Pintchovski received a BS with Honors from the California Institute of Technology and earned his PhD in Neuroscience from the University of California, San Francisco.

10:10 – 10:40

1-2-1 Meetings/Networking Break

10:40 – 11:10

1-2-1 Meetings / Networking Break

11:10 – 11:20

Morning Refreshments

11:20 – 12:20

Building a clinical trial in a dish for Alzheimer’s patient stratification using human iPSCs

How to build a clinical trial in a dish using iPSCs – patient access and manufacturing and technical expertise
How to use a clinical trial in a dish – strategy for patient stratification and compound testing
Collaboration and partnership

Ashley Barnes

Chief Scientific Officer, Axol Bioscience

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Spotlight Presentation by Invicro

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:30 – 15:40

Afternoon Refreshments

15:40 – 16:40

How AI can improve clinical trials

How will AI impact different stages if a clinical trial
Leveraging AI to improve clinical trial design
Risk-based monitoring (RBM)
Drug discovery
Feasibility analysis using AI/ML models
Clinical trial protocol digitization using AI
How to enable AI-based solutions at enterprise level
Change management

Amir Emadzadeh

Director of Software Engineering in the Digital Transformation Office (DTO), Genentech

Amir holds a PhD in Electrical Engineering from the University of California at Los Angeles (UCLA). Over his 13 years of work experience, he has been granted an equal number of patents while at top Silicon Valley companies, including Qualcomm, Nvidia, and Google. He is a Director of software in the Digital Transformation Office (DTO) at Genentech (Roche). He is also an adjunct Professor at California Science and Technology University (CSTU). Most recently, he was a Director of data science at Gilead sciences, where he led a team of data scientists and software developers dedicated to improving different aspects of clinical trials. Amir is very passionate about employing AI/ML techniques and automation to tackle pharma/healthcare industry challenges. Some of his recent work includes data-driven feasibility analysis for clinical trials (e.g., clinical trial site/investigator ranking, country assessment, diversity analysis & patient enrollment forecasting), extracting insights from clinical trial documents and sentiment analysis using NLP technology, and medical image segmentation/ classification using deep neural networks.

16:40 – 17:40

Drinks-Canape Reception

Drug Delivery

Time

Titles and Bullets

Facilitator

08:00 – 08:30

Registration

08:30 – 09:00

Opening Address & Keynote Presentation by Axol Bioscience
Using Human iPSCs to Build a Clinical Trial in a Dish for Alzheimer’s Patient Stratification

Ashley Barnes

Chief Scientific Officer, Axol Bioscience

09:05 – 10:05

Non-invasive methods to increase brain drug uptake

Blood brain barrier excludes many drugs and therapies from targeting CNS.
Many CNS diseases are poorly understood.
CNS diseases are becoming a larger societal problem with an aging population.

John Hoekman

Chief Technology and Development Officer, Impel Pharmaceuticals

John Hoekman is the Founder and Chief Technology and Development Officer at Impel Pharmaceuticals and previously served as Chief Scientific Officer since July 2008. John has spent the last 14 years investigating intranasal drug delivery of therapeutics targeting the central nervous system. John co-founded Impel while attending graduate school at the University of Washington in 2008. He is the lead inventor of the POD technology and has made fundamental contributions to understanding intranasal drug delivery. John conceived of Impel’s lead product, Trudhesa, and subsequently development from preclinical stages through FDA approval. John has spent the last 10 years working across all functions to grow Impel from a pre-clinical device focused company to a commercial stage pharmaceutical company. He holds a BS in Physics from the University of Minnesota and a Ph.D. in Pharmaceutics from the University of Washington.

10:10 – 10:40

1-2-1 Meetings/Networking Break

10:40 – 11:10

1-2-1 Meetings / Networking Break

11:10 – 11:20

Morning Refreshments

12:20 – 13:20

Networking Lunch

13:20 – 13:50

Spotlight Presentation by Invicro

13:55 – 14:25

1-2-1 Meetings / Networking Break

14:25 – 14:55

1-2-1 Meetings / Networking Break

15:30 – 15:40

Afternoon Refreshments

15:40 – 16:40

Considerations in evaluating the efficiency of CNS drug delivery by different routes of administration: PK and fluid-based biomarkers

How are currently approved CNS biotherapeutics administered and where are their target sites?
What are the major physiological and anatomical factors governing CNS biodistribution across species?
What considerations govern the interpretation of PK/PD in biofluids for CNS drug delivery (e.g. how might CSF PK and biomarker readouts like NfL inform on CNS target engagement across species and different routes of administration?)

Robert G. Thorne

Denali Fellow, Denali Therapeutics

Adjunct Associate Professor, University of Minnesota

Dr. Thorne joined Denali Therapeutics as a Denali Fellow in 2018 after over 10 years on the faculty at NYU School of Medicine and the University of Wisconsin-Madison. His research interests include the biology of CNS barriers, routes of administration bypassing the blood-brain barrier (BBB), CNS biodistribution, and receptor-mediated transcytosis across the BBB. Dr. Thorne is currently the President of the International Brain Barriers Society (IBBS). Previously, he has served as chair / lead organizer for the 2016 ‘Barriers of the CNS’ Gordon Research Conference, 2019 AAPS-IBBS CNS delivery workshop, and 2023 ‘Drug Delivery to the Brain’ Keystone Symposium.

16:40 – 17:40

Drinks-Canape Reception

CNS THERAPEUTICS XCHANGE
WEST COAST
San Francisco
September 27, 2023

SNAPSHOTS OF DISCUSSION TOPICS

Full Xchange Agenda

Novel Targets

Preclinical

Clinical Studies

Drug Delivery

Partners

© Copyright 2023 by Weblify & Webtec

CNS THERAPEUTICS XCHANGEWEST COASTSan FranciscoSeptember 27, 2023

SNAPSHOTS OF DISCUSSION TOPICS

Full Xchange Agenda

Novel Targets

Preclinical

Clinical Studies

Partners

CNS THERAPEUTICS XCHANGE
WEST COAST
San Francisco
September 27, 2023