Collaborate 2 Cure
June 12, 2017


Location: Please note location change for June 12

Enterprise Center of Johnson County (ECJC)
4220 Shawnee Mission Parkway, Suite 350B, Fairway, KS 66205

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Please register to attend and select “Virtually by Zoom” and the link will be in your registration confirmation email.

Ameliorating Mitochondrial Dysfunction as a Therapeutic Strategy in Neurodegenerative Disease

Amylyx has developed a novel therapeutic, AMX0035, for the treatment of ALS. AMX0035 is a proprietary combination of two compounds, Sodium Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA). Amylyx discovered a synergy between these two compounds when administered together in a particular range of ratios across multiple preclinical models. In these models, AMX0035 blocked neuronal death and neurotoxic inflammation through simultaneous inhibition of ER stress and mitochondrial stress. AMX0035 entered its first clinical trial in ALS patients in mid-2017 (CENTAUR), with plans to enter clinical testing in Alzheimer’s disease patients in late 2017.
AMX0035 is designed to target mitochondrial and endoplasmic reticulum stress and has been evaluated in in vivo models of Alzheimer’s disease and ALS. Recently, AMX0035 was tested in cytoplasmic hybrids (cybrids) harboring patient-derived mtDNA mutations associated with mitochondrial diseases.  These mtDNA mutant cells exhibit OXPHOS defects compared with cybrids harboring wild type mtDNA (143B osteosarcoma cells).  AMX0035 was shown to be more effective in improving cell viability relative to treatment with the individual compounds, providing preliminary evidence of synergistic efficacy in the context of mitochondrial disease. Treatment with AMX0035 resulted in increased mitochondrial membrane potential and increased mitochondrial mass, providing potential mechanistic insight into the increased cell viability observed in cybrid and mouse embryonic fibroblast models. Our long-term goal is to clinically evaluate AMX0035 in a broad array of neurodegenerative diseases characterized by mitochondrial dysfunction.

Speaker- Kent Leslie

Kent Leslie serves as the Chief Scientific Officer at Amylyx Pharmaceuticals, a clinical stage pharmaceutical company focused on the treatment of neurodegenerative disease. Kent received his M.Sc for Brown University while completing research in the lab of Dr. Richard Bennett. Kent is a member of the American Society for Experimental Neurotherapeutics and the Alzheimer’s Association Business Consortium. Prior to receiving his Master’s degree, Kent received Bachelor degrees in Biology and Economics from Brown University.

Breezio Introduction

Speaker: Karen McCord

Karen McCord is Chief Executive Officer of Breezio, an online community engagement software company. Breezio’s patented technology allows organizations to curate, contribute to and grow knowledge. During the past year as CEO she was a finalist for the Start-up Maryland’s Contest, has been a panelist at scholarly publishing conferences and taken the company from pre-revenue to a revenue generating entity. Ms. McCord’s experience includes managing international teams for both technical and financial projects in global public health where she helped raise over $300 million towards HIV prevention efforts and most recently she was involved with an evaluation project of a large research enterprise in the Middle East. In September 2014, Karen won the “Make it Matter Challenge”, an innovation contest sponsored by LinkedIn and the Taproot Foundation, aimed at securing ideas to connect nonprofits to quality pro bono resources. Ms. McCord completed the Management and Technology Dual Degree Program at the University of Pennsylvania, with bachelor degrees from the School of Engineering and Applied Science and the Wharton School.