MeiraGTx Holdings (NasdaqGS:MGTX) FY Conference Transcript

Summary of Conference Call on Gene Therapy for Neurodegenerative Diseases Companies Involved - MeiraGTx Holdings - uniQure - Passage Bio Key Points and Arguments MeiraGTx Holdings - Focuses on genetic medicines for inherited and larger indications, utilizing local delivery of small doses to enhance safety and reduce costs [2][3] - Owns in-house manufacturing facilities, which supports the rapid development of their programs [2] - Two pivotal studies and two awaiting BLA filings, including treatments for rare and common eye diseases and a Parkinson's treatment that has shown three positive studies [3][4] - Partnership with an AI company to analyze data from the largest neurohospital in Europe, demonstrating physiological changes in the brain with their Parkinson's treatment [4][24] uniQure - A pioneer in genomic medicine with two approved AAV gene therapies: Glybera for lipoprotein lipase deficiency and Hemgenix for hemophilia B [6] - Lead program focuses on Huntington's disease, utilizing a platform called MyCure to suppress the aberrant huntingtin protein [9] - Clinical testing of AMT-130 has shown a 75% statistically significant slowing of disease progression over three years [10] - Plans to hold a pre-BLA meeting in Q4 and submit a BLA in Q1 of the following year [10] Passage Bio - Focuses on gene therapy for frontotemporal dementia patients with GRN mutations, using AAV1 delivered via a minimally invasive procedure [15] - Demonstrated high levels of target engagement and stabilization of plasma neurofilaments [15] Challenges in Gene Therapy for Neurodegenerative Diseases - Delivery to the right location in the brain remains a significant challenge, especially for diseases like Huntington's [18][19] - Slow progression of neurodegenerative diseases complicates the design of clinical studies to detect meaningful changes [19][20] - Heterogeneity among patients complicates the assessment of treatment effects [32][34] Safety Considerations - Local delivery methods may reduce systemic exposure and associated risks compared to systemic therapies [39][40] - Smaller doses and targeted delivery are believed to enhance safety profiles [42] Regulatory Aspects - The FDA is showing increased flexibility in evaluating gene therapies for rare diseases, allowing for single-arm studies with supportive evidence [44][45] - Emphasis on using natural history data and biomarkers to assess treatment effectiveness [31][33] Access and Implementation Challenges - The complexity of procedures may limit access, but strong data can encourage training and adoption among healthcare providers [53][55] - Existing surgical techniques can facilitate the implementation of new therapies, particularly in diseases with significant unmet needs [55][56] Other Important Insights - AI technology is being utilized to analyze large datasets to identify physiological changes and improve the robustness of clinical trial endpoints [25][35] - The potential for gene therapy to address multiple neurodegenerative diseases by altering brain circuitry is highlighted [22][24]