Axovant Gene Therapies to Host Virtual Parkinson’s Disease R&D Day on October 30, 2020
- Agenda to feature presentations from leading clinical researchers in Parkinson’s disease and the
Michael J. Fox Foundation
- Individual patient-level data from Cohort 2 of the Phase 2 SUNRISE-PD study to be shared
Axovant’s Parkinson’s disease R&D Day will be moderated by Chief R&D Officer,
Charles Adler, M.D., Ph.D., Professor at Mayo Clinic College of Medicinein Arizona Jamie Eberling, Ph.D., Vice President of Research Programs at the Michael J. Fox Foundation Stéphane Palfi, M.D., Ph.D., Professor of Neurosurgery and Head of the Neurosurgery Departmentat Henri Mondor Medical Center, Paris University(UPEC)
In addition, the Company will present data from the second cohort of the Phase 2 SUNRISE-PD trial for AXO-Lenti-PD including:
- Summary of available data from the second dose cohort at the 6-month timepoint following one-time dosing with AXO-Lenti-PD gene therapy
- Individual patient-level data at 6-months after dosing:
○ Hauser diary “Good ON time” and “OFF time” changes from baseline for all 4 patients
○ Levodopa-equivalent daily dose (LEDD) changes from baseline for all 4 patients
○ UPDRS Part II and Part III “OFF” scores for the 2 evaluable patients in the cohort
- 12-month evaluation for the first patient treated in Cohort 2, who was assessed remotely
Drs. Adler, Palfi, and Eberling will be joined by
AXO-Lenti-PD is the only investigational gene therapy for Parkinson’s disease that delivers three genes via a lentiviral vector to encode a set of critical enzymes required for endogenous dopamine synthesis, with the goal of improving motor function and restoring steady, tonic levels of dopamine in the brain. The gene therapy aims to provide patient benefit for years following a single administration.
To register for the R&D webcast, please click here.
A live audio webcast of the R&D Day can be accessed through the Events & Presentations section of the company's website at investors.axovant.com. An archived replay of the webcast will be available on the company's website following the event.
Biographies of R&D Day Panelists:
Charles H. Adler, M.D., Ph.D. is currently Consultant and Professor in the Department of Neurologyat Mayo Clinic College of Medicinein Arizona. He is also Co-Principal Investigator at both Arizona Parkinson's Disease Consortiumand Arizona Study of Aging and Neurodegenerative Disorders in Scottsdale, Arizona. Dr. Adlerreceived his Ph.D. in pharmacology and his medical degree, from the New York University School of Medicine. He did his neurology residency at the University of Pennsylvaniaand a fellowship in movement disorders at Graduate Hospital/ University of Pennsylvaniain Philadelphia. He then joined the staff at Mayo Clinicin Scottsdale, Arizona. Dr. Adlerhas received numerous grants to investigate experimental treatments for Parkinson's disease, essential tremor, dystonia, restless legs syndrome, and chronic traumatic encephalopathy (CTE). He serves as an advisory member to many different international medical societies such as the International Parkinson and Movement Disorder Society, MDS Industry Education and Services Committee, and the American Academy of Neurology Section of Movement Disorders. Dr. Adlerhas a commitment to education having trained residents, 14 fellows and graduate students, and has given many invited lectures. Dr. Adler’s main research interests are investigating tissue diagnostic tests for Parkinson’s disease, biomarkers for an early diagnosis of Parkinson’s disease and PD with dementia, and identification of new treatments for PD and PD with dementia. He also has been investigating essential tremor, restless legs syndrome, and dystonia. He has published over 400 research papers and reviews, and edited a book entitled Parkinson's Disease and Movement Disorders: Diagnosis and Treatment Guidelines for the Practicing Physician. In 2006, Dr. Adlerwas awarded the Mayo Clinic Distinguished Investigator of the Year Award. Stéphane Palfi, M.D., Ph.D. is a Professor of Neurosurgery and Head of the Neurosurgery Departmentat Henri Mondor Medical Center, Paris University(UPEC). His interests are in developmental therapeutics for Parkinson’s disease, Huntington’s disease, tremor, dystonia, and psychiatric disorders. He has worked extensively in the area of electrical neuromodulation of the brain in movement disorders, gene therapy for Parkinson’s disease, cell grafting for Huntington’s and Parkinson’s disease as well as primate models of neurodegenerative disorders. Dr. Palfihas published extensively on trophic factor- and enzyme-based gene therapy in Parkinson’s disease and Huntington’s disease. He is a principal investigator on numerous preclinical and clinical studies and has been involved in studies of many novel agents including implanted brain devices, optogenetic, homeoprotein, trophic factors GDNF, CNTF and dopamine lentiviral vectors. Jamie Eberling, Ph.D. joined the Michael J. Fox Foundation(MJFF) in 2009. As Vice President, Research Programs, Dr. Eberlingstays closely linked to the Parkinson’s research community in order to develop an aggressive and innovative agenda for accelerating research and drug development for Parkinson’s disease. This ensures that MJFF research priorities reflect and best serve the ultimate needs of patients. Dr. Eberlingregularly meets with academic and industry researchers around the world to identify promising proposals to support, providing troubleshooting and ongoing management of projects as they go forward. She also supports the Foundation’s priority interests in translating promising new therapies into the clinic. Jamie’s primary responsibilities are managing the PET imaging program, including alpha-synuclein tracer development, the non-motor symptomatic program aimed at developing new therapies for non-motor symptoms, and the clinical trials that are funded by MJFF. Dr. Eberlingearned undergraduate and graduate degrees in biological psychology from the University of Californiaat Berkeley, later moving to the Lawrence Berkeley National Laboratorywhere she developed expertise in neuroimaging techniques and gene therapy approaches for Parkinson’s disease. Gavin Corcoran, M.D. joined Axovantas Chief R&D Officer. In his career, he has overseen successful drug development across multiple therapeutic areas, including neurology and psychiatry, and was previously Chief Medical Officer of Allergan plc and Actavis. Dr. Corcoranwas Executive Vice President for Global Medicines Developmentat Forest Laboratoriesprior to the acquisition of Forest Laboratoriesby Actavis. Dr. Corcoranalso served as Head of Late Stage Clinical Developmentfor Inflammation and Immunology at Celgene, and as Chief Scientific Officer and head of R&D at Stiefel Laboratories. Earlier in his career he held leadership roles in clinical development and regulatory affairs at Amgen, Schering-Plough, and Bayer. He received his MB BCh from the University of the Witwatersrandin South Africaand completed his clinical training in internal medicine and infectious diseases at the University of Texas Health Science Center at San Antonio.
About Axovant Gene Therapies
Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinson’s disease.
Axovant Gene Therapies Ltd.