CNS Therapeutics Xchange
East Coast, Boston
May 18, 2022
Welcome to hubXchange’s East Coast Hybrid CNS Therapeutics Xchange 2022, 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 with a hybrid option so participants can also join virtually.
VENUE DETAILS: Hilton Boston Woburn Hotel, 2 Forbes Road, Woburn MA 01801
Novel Targets
8:30 – 9:00am
Decentralized Clinical Trials Are Here to Stay
- How the clinical landscape has looked prior to COVID-19
- Technology has been the catalyst for change in clinical research
- How the industry projects to grow in 2022
Manager, Program Development Neurology & Regenerative Medicine Center of Excellence, Biorasi
Ruslan Gorsky is a manager within the program development team at Biorasi, an award winning CRO headquartered in Miami, Florida. Ruslan and his team focus predominantly on Neurological clinical trials, ranging from Neurodevelopmental to Neurodegenerative studies across all clinical phases. He provides pharmaceutical companies tailored strategies that solidify clinical trial success by focusing on decentralized processes and engagement with key opinion leaders as well as clinical research centers. This engagement ensures the client receives a strategy that synthesizes time, risk and cost for their clinical trial across multiple stakeholders. In addition, Ruslan and the team provide continual support throughout the duration of the study, making any necessary adjustments to facilitate compliance and competitive recruitment. Throughout his time at Biorasi, Ruslan has been involved in numerous, complex phase II and phase III clinical trials in diseases such as Parkinson’s, Alzheimer’s and ALS involving groundbreaking biological investigational products and cell therapies.
9:05 – 10:05am
Opportunities from emerging therapeutic modalities: Target selection in an evolving druggability landscape
- Re-evaluation of what we can target in the CNS to develop medicines
- Therapeutic modalities most impactful to the target space of disease in the brain
- Where is genetic validation and human biology powering confidence in the next generation of targets?
- What CNS disorders will gene editing be best suited to address?
Director of Target Biology, Rgenta Therapeutics
Sam Hasson is currently the Director of Target Biology at Rgenta Therapeutics with a focus on the discovery of therapies for CNS diseases via modulation of RNA processing. Previously, he was an Associate Director at Voyager Therapeutics (Cambridge, Massachusetts). A major aim of his work at Voyager was the development of AAV-based gene therapy for the CNS to transform the treatment of unmet medical needs. Prior to joining Voyager, Sam led groups within Amgen Neuroscience and Pfizer Neuroscience that powered drug discovery programs via the deconvolution of human biology and disease genetics. He has been employing genome editing technologies and high throughout methods to enable more robust target selection and validation. As a postdoc, Sam trained with Richard Youle and Jim Inglese at the National Institutes of Health.
Networking Lunch
1:25 – 2:25pm
Novel therapies for complex neurological disorders: discovery of effective treatments
- Evaluating efficacy and potency of drug candidates on neurological conditions
- Novel and advanced therapeutic approaches in epilepsy
- Exosomes and secretome for treatment of neurodegenerative diseases
- Preclinical models and mechanisms: how well they correlate to clinical outcomes?
Director, Advanced Analytical Sciences, Noveome Biotherapeutics
Ziv Kirshner, is the Director of Advanced Analytical Sciences at Noveome Biotherapeutics, Inc. Noveome is advancing its ST266 platform biologic, a multi-targeted secretome containing many factors crucial to neuroprotection, the modulation of inflammation, cell recovery and healing. Ziv has extensive experience in the development of multiple cell and gene therapies across all stages of development and for a wide range of diseases. He was a lead scientist in Lonza Biologics and a scientist in Multi-Gene Vascular Systems Ltd. He holds a PhD in Pharmaceutical Sciences from the University of Pittsburgh and is dedicated to studying the mechanisms underlying the beneficial effects of complex and cell-secreted biologics.
3:45 – 4:15pm
- Integrating gene-disease annotations and biological networks from multiple sources
- Predicting novel gene-disease associations from known genetic associations
- Contextualising results with public knowledge in the Euretos platform
Head of Translational Science, Euretos
Sophie has a MSc degree in Pharmaceutical Sciences and a PhD in Medicine from Leiden University – studying cancer biology in model organisms. During her postdoctoral work at the Cancer Genome Project in the Sanger Institute and the Princess Maxima Center for pediatric oncology she has worked on understanding cancer biology from cancer genomics data. Within Euretos she heads up the translational science team – translating research questions from customers into data science solutions and/or user-friendly workflows in the Euretos AI Platform.
4:20 – 5:20pm
Use of tech in the pursuit of novel therapies
- Explore the use of technology that may help facilitate the
discovery and development of novel therapies - Understand how drug-tech combinations can target residual symptoms
and disability in CNS disease - Understand how drug-tech combinations can target the
neurobiological substrate underlying CNS disease
Chief Medical Officer, Click Therapeutics
Shaheen Lakhan, MD, PhD, FAAN, is a board-certified neurologist and
pain specialist with clinical training from Cleveland Clinic and
Massachusetts General Hospital. He is chief medical officer for Click
Therapeutics, and, with over 100 peer-reviewed papers, is a professor
of neurology and neuroscience at Western University and Virginia Tech, respectively. Previously, Dr. Lakhan has served as faculty of leading institutions (Harvard Medical School, Case Western Reserve
University), chief of pain management (Virginia Tech – Carilion Clinic), and executive/leadership positions in the life sciences (Sage Therapeutics, Zogenix, The Learning Corp, Fern Health). He was conferred the President’s Call to Service Award for Lifetime Achievement by George W. Bush. Recognized by his peers, he was inducted as Fellow of the American Academy of Neurology, the Academy’s highest designation.
Preclinical
8:30 – 9:00am
Opening Address & Keynote:
Decentralized Clinical Trials Are Here to Stay
- How the clinical landscape has looked prior to COVID-19
- Technology has been the catalyst for change in clinical research
- How the industry projects to grow in 2022
Manager, Program Development Neurology & Regenerative Medicine Center of Excellence, Biorasi
Ruslan Gorsky is a manager within the program development team at Biorasi, an award winning CRO headquartered in Miami, Florida. Ruslan and his team focus predominantly on Neurological clinical trials, ranging from Neurodevelopmental to Neurodegenerative studies across all clinical phases. He provides pharmaceutical companies tailored strategies that solidify clinical trial success by focusing on decentralized processes and engagement with key opinion leaders as well as clinical research centers. This engagement ensures the client receives a strategy that synthesizes time, risk and cost for their clinical trial across multiple stakeholders. In addition, Ruslan and the team provide continual support throughout the duration of the study, making any necessary adjustments to facilitate compliance and competitive recruitment. Throughout his time at Biorasi, Ruslan has been involved in innumerous, complex phase II and phase III clinical trials in diseases such as Parkinson’s, Alzheimer’s and ALS involving groundbreaking biological investigational products and cell therapies.
Are human-derived preparations a useful tool for neuroscience-related electrophysiology studies at the preclinical stages?
- Are there advantages in complementing in vitro electrophysiological studies performed in rodent tissues with data obtained from human neurons in R&D and preclinical studies?
- What progress have been achieved by using human iPSCs-derived neuronal cultures for in vitro electrophysiological studies?
- Can 3D brain organoids be a useful tool and present significant advantages to study neurodevelopment using electrophysiology?
- Could surgical wastes be valorized for in vitro electrophysiological studies in human tissues?
Chief Executive Officer, Ephyx Neuroscience
Txomin has over 8 years of experience planning and performing in vitro electrophysiology studies for R&D and preclinical projects.
He holds a PhD in neurobiology from McGill University (Montréal, QC, Canada), during which he obtained twice the Excellence award from the Integrated Program in Neuroscience. After postdoctoral experiences specialized in electrophysiology, he co-founded EphyX Neuroscience with the goal to apply his skills and expertise to boost neuroscience-related preclinical development by providing highly functional electrophysiological data from rodent and human neurons.
- Disease modifying therapeutics for CNS indications may not be informed from preclinical models
- Mechanism of disease causation (etiology) may be distinct from those driving pathogenesis
- Early mechanistic causation may be clinically silent (both preclinical and early prodromal)
- Delivery of therapeutics, notably biologics, is an important facet of drug development for many CNS indications
President, Director & CEO, Brooklyn ImmunoTherapeutics
Howard Federoff, MD, PhD is CEO, President and director of Brooklyn ImmunoTherapeutics. He was recently a distinguished professor of neurology at the University of California, Irvine. He is the former CEO of UCI Health, vice chancellor for Health Affairs and dean of the UCI School of Medicine. Prior to joining UCI Health, Howard was executive vice president of Health Sciences and executive dean at Georgetown University. Howard has published more than 275 peer-reviewed and invited articles, and serves on editorial boards of five journals. He co-founded MedGenesis Therapeutix and Brain Neurotherapy Bio, both advancing therapeutics for neurologic diseases. He became CEO of the regenerative medicine company, Aspen Neuroscience, Inc, in San Diego. He is an elected Fellow of the American Association for the Advancement of Science and the National Academy of Inventors. He received his MD, MS and PhD in biochemistry from the Albert Einstein College of Medicine in New York. He completed his residency and clinical and research fellowships at Massachusetts General Hospital and Harvard Medical School.
Clinical Studies
Registration
8:30 – 9:00am
Opening Address & Keynote:
Decentralized Clinical Trials Are Here to Stay
- How the clinical landscape has looked prior to COVID-19
- Technology has been the catalyst for change in clinical research
- How the industry projects to grow in 2022
Manager, Program Development Neurology & Regenerative Medicine Center of Excellence, Biorasi
Ruslan Gorsky is a manager within the program development team at Biorasi, an award winning CRO headquartered in Miami, Florida. Ruslan and his team focus predominantly on Neurological clinical trials, ranging from Neurodevelopmental to Neurodegenerative studies across all clinical phases. He provides pharmaceutical companies tailored strategies that solidify clinical trial success by focusing on decentralized processes and engagement with key opinion leaders as well as clinical research centers. This engagement ensures the client receives a strategy that synthesizes time, risk and cost for their clinical trial across multiple stakeholders. In addition, Ruslan and the team provide continual support throughout the duration of the study, making any necessary adjustments to facilitate compliance and competitive recruitment. Throughout his time at Biorasi, Ruslan has been involved in numerous, complex phase II and phase III clinical trials in diseases such as Parkinson’s, Alzheimer’s and ALS involving ground-breaking biological investigational products and cell therapies.
Impact of wearables/passive
monitoring for CNS trials
- Key advantages of integrating wearables/passive monitoring into clinical CNS studies
- What type of studies are most amenable to wearable/passive monitoring approaches?
- What are the preferred technologies, platforms and paradigms currently being applied (actigraphy, EEG, ERPs, sleep…)?
- Any specific examples of how these approaches have impacted your studies? Any lessons learned?
- Are there challenges/opportunities integrating wearables/passive monitoring with other remote assessments, such as cognitive testing?
Vice President & Head, Translational Medicine, Cyclerion Therapeutics
Christopher Winrow is Vice President and Head of Translational Medicine at Cyclerion Therapeutics, a clinical stage biopharmaceutical company located in Cambridge, MA. Prior to Cyclerion, Chris held roles of increasing responsibility at Ironwood Pharmaceuticals and Merck &Co., Inc. He completed his Ph.D. at the University of Alberta and post-doc at the Salk Institute. Chris has an extensive background in CNS diseases with areas of focus including Alzheimer’s disease, Schizophrenia, 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. Chris 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 CNS diseases at Cyclerion Therapeutics.
Challenges in neurology trials during the COVID/post-Covid era
- How COVID-19 has impacted patient engagement and retention during clinical trials.
- Clinical trial decentralization impact on patient compliance and the patient/PI relationship.
- Will decentralization methodologies stay in the clinical trial setting post COVID?
Manager, Program Development Neurology & Regenerative Medicine Center of Excellence, Biorasi
Ruslan Gorsky is a manager within the program development team at Biorasi, an award winning CRO headquartered in Miami, Florida. Ruslan and his team focus predominantly on Neurological clinical trials, ranging from Neurodevelopmental to Neurodegenerative studies across all clinical phases. He provides pharmaceutical companies tailored strategies that solidify clinical trial success by focusing on decentralized processes and engagement with key opinion leaders as well as clinical research centers. This engagement ensures the client receives a strategy that synthesizes time, risk and cost for their clinical trial across multiple stakeholders. In addition, Ruslan and the team provide continual support throughout the duration of the study, making any necessary adjustments to facilitate compliance and competitive recruitment. Throughout his time at Biorasi, Ruslan has been involved in numerous, complex phase II and phase III clinical trials in diseases such as Parkinson’s, Alzheimer’s and ALS involving groundbreaking biological investigational products and cell therapies.
Attaining accelerated approval in rare neurological diseases
- Are certain surrogate endpoints more likely to predict clinical benefit in patients?
- Regulatory thresholds by region/agency – opportunities and best practices
- Role of patient advocacy groups in orphan disease drug development
- Design and conduct of Phase 4 confirmatory trials
Vice President & Head, Clinical Development, Laronde (a Flagship Pioneering company)
Kemi Olugemo is currently Vice President and Head of Clinical Development at Laronde, a Flagship Pioneering Company.
Over her 2 decades in clinical research and practice, Kemi has been involved in all phases of clinical development, global medical strategy, business development, and innovations in lifecycle planning. Her current role at Laronde is focused on advancing a new class of RNA medicines for all patients, and to have a global impact on access to innovative lifesaving therapies.
Prior to joining Laronde, Kemi held progressive leadership roles at Ionis Pharmaceuticals, Akcea Therapeutics, Parexel International and Questcor Pharmaceuticals, Inc, and was a long-term consultant in Neurology with Quorum Independent Review Board (now Advarra).
Kemi draws on a diverse set of experiences including being the current Director of Communications at Women of Color in Pharma (WOCIP), Board Member at Watermark, serving on the Leadership Council at CNS Summit and on the Scientific Committee of the International Society for CNS Clinical Trials and Methodology (ISCTM).
Kemi had as undergraduate degree in Clinical Laboratory Science from the University of Massachusetts (magna cum laude) and a medical degree at the University of Maryland School of Medicine in Baltimore.
4:20 – 5:20pm
Utilizing clinical biomarkers to enhance drug development in neurodegenerative diseases
- Important things to consider when choosing biomarkers to include in clinical trials
- Expectations with biomarkers – what do they tell us and how can they be used to support development in various phases of development
- Will we ever have a truly convincing surrogate biomarker in neurodegeneration? If not, how do we get there?
Chief Medical Officer, Arkuda Therapeutics
Serena is the Chief Medical Officer at Arkuda Therapeutics, a biotechnology company that focuses on lysosomal dysfunction in neurodegenerative diseases. She joined Arkuda in April 2020. She is a board-certified neurologist with subspecialty training in movement disorders. She has approximately 20 years of clinical research experience both in industry and academia, including leadership roles in Phase I through Phase III development with successful INDs, BLA/NDAs. She has experience in multiple therapeutic areas in CNS including Parkinson’s disease and other movement disorders, Huntington’s disease, dementia, ALS, ataxia, and multiple sclerosis. Prior to Arkuda, Serena was Senior Director of Neurology in clinical development leading all CNS programs. Before that she was at Biogen where she led the clinical development efforts in Movement Disorders. Before joining industry, she had an academic appointment as Associate Professor of Neurology at the Medical College of Wisconsin.
Drug Delivery
8:30 – 9:00am
Opening Address & Keynote:
Decentralized Clinical Trials Are Here to Stay
- How the clinical landscape has looked prior to COVID-19
- Technology has been the catalyst for change in clinical research
- How the industry projects to grow in 2022
Manager, Program Development Neurology & Regenerative Medicine Center of Excellence, Biorasi
Ruslan Gorsky is a manager within the program development team at Biorasi, an award winning CRO headquartered in Miami, Florida. Ruslan and his team focus predominantly on Neurological clinical trials, ranging from Neurodevelopmental to Neurodegenerative studies across all clinical phases. He provides pharmaceutical companies tailored strategies that solidify clinical trial success by focusing on decentralized processes and engagement with key opinion leaders as well as clinical research centers. This engagement ensures the client receives a strategy that synthesizes time, risk and cost for their clinical trial across multiple stakeholders. In addition, Ruslan and the team provide continual support throughout the duration of the study, making any necessary adjustments to facilitate compliance and competitive recruitment. Throughout his time at Biorasi, Ruslan has been involved in numerous, complex phase II and phase III clinical trials in diseases such as Parkinson’s, Alzheimer’s and ALS involving ground-breaking biological investigational products and cell therapies.
Transcytosis-mediated brain delivery for proteins, antibodies, oligonucleotides
and small molecules
- Explore various types of transcytosis to cross the blood brain barrier (BBB)
- Discuss BBB drug delivery systems (DDSs) that are transcytosis-mediated
- Consider DDS attributes that provide unique solutions for CNS delivery
- Decipher differences in delivery for various compounds
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 42 scientific conferences, w/posters at 8 scientific meetings, made Lauren Sciences 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.
High molecular weight drug delivery – lessons from SARS-COV-2
- Olfactory Nerve Pathways
- Bypassing the Blood Brain Barrier
- Blood Brain Barrier breakdown due to disease or injury
- Nanoparticle carriers to bypass Blood Brain Barrier
Executive Vice President, R&D & 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, Pittsburgh, PA, USA. He manages Noveome’s scientific research and development pathway, working closely with the regulatory group in all FDA related submissions and the company’s executive leadership team. 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 Childrens Hospital. Previously, he served as VP Research at Baxter Healthcare Corporation and in scientific leadership roles at several startup biotech companies. Larry has over 100 publications and patents in the field of biotechnology and novel drug delivery systems. He maintains strong academic collaborations with MIT’s Deshpande Center for technological innovation.
Poster Session: Maximizing intracranial drug concentration via active fluid exchange delivery mechanism
- The need for controlled, optimized delivery of therapeutic medication is well recognized due to challenges crossing blood brain barrier
- The IRRAflow system from IRRAS is cleared in the US and Europe and commercially available for intracranial fluid management
- IRRAflow’s recurring irrigation provides controlled fluid introduction that prevents catheter occlusion and ensures patency
- Clinical experience using the system to deliver thromblytic, antibiotic, and anti-spasm medication has been published, and other drug delivery possibilities currently being explored
Director, Clinical Affairs, IRRAS
As head of IRRAS’ clinical affairs and drug delivery programs, John Unser directs ongoing clinical projects that are designed to document the clinical utility of the company’s IRRAflow system. Building upon his early experience as a clinical nurse, his 15+ years of industry experience at Volcano Corporation and Philips Healthcare have built clinical trial, product development, and marketing experience that he now combines to provide a unique mastery of launching innovative technologies into the market. Mr. Unser joined IRRAS in 2018, and, during his time with the company, he has played an integral role in developing and executing the company’s global launch strategy as well as providing strategic input to key acquisitions and expansion into adjacent areas, such as intracranial drug delivery.
How do you efficiently deliver vectors to a large volume of parenchyma?
- For many brain disorders, therapeutics delivery selectively to key brain regions with little systemic exposure is desirable
- Today, many CNS targeting drugs achieve broad systemic exposure but often limited brain exposure
- Future therapeutics for brain disorders should optimize brain over systemic exposure
- Different modalities and routes of administration towards this goal will be discussed
Chief Medical Officer, Head of R&D, Triplet Therapeutics
Irina Antonijevic, MD, PhD is a board-certified psychiatrist and neuroscientist. For the last 21 years, she has held R&D roles of increasing responsibilities in small biotech and large pharma organizations, such as Sanofi, and Wave Life Sciences, advancing new and potentially better therapeutic modalities to address unmet medical needs in neuropsychiatric disorders, with a focus on rare and genetic neurological disorders. Currently, she is leading R&D at Triplet Therapeutics, developing nucleic acid therapeutics for HD and other genetically driven CNS disorders.