MeiraGTx (MGTX) Investor Presentation - Slideshow

Company Overview - MeiraGTx is a vertically integrated, clinical-stage gene therapy company developing cost-effective treatments for a range of serious disorders[3] - The company has a diversified pipeline of gene therapy candidates and a platform of core viral vector engineering capabilities[3] - MeiraGTx possesses cGMP manufacturing capacity with a commercial-ready scalable process[3] - The company is developing a next-generation gene therapy riboswitch-based gene regulation platform[3] Pipeline and Programs - MeiraGTx has six ongoing clinical programs focusing on inherited retinal diseases, salivary gland disorders, and Parkinson's disease[3] - In ocular, botaretigene sparoparvovec for X-linked RP is in Phase 3, with Phase 1/2 randomized expansion cohort data to be presented in 1H 2022 and Phase 3 enrollment in 2022[5, 6] - AAV-GAD for Parkinson's disease is expected to file an IND and initiate a clinical study in 1H 2022[6] - AAV-hAQP1 for Grade 2/3 RIX (radiation-induced xerostomia) has Phase 1 dose escalation data expected by YE 2022, and a randomized, multi-dose Phase 2 trial is planned to initiate by YE 2022[6] Gene Regulation Platform - The company's proprietary technology allows precise control of gene therapy expression levels via dose response to orally delivered small molecules, with an unprecedented dynamic range of >5000[3, 22] - MeiraGTx has created a gene regulation cassette controlled by a riboswitch, offering high expression when on and low expression when off[28] - The gene regulation system can control any gene, any vector, cell therapy, and gene editing[22] Manufacturing Capabilities - MeiraGTx has a fully integrated manufacturing ecosystem, including a non-GMP vector core in Amsterdam for preclinical supply[9] - The company has flexible and scalable GMP manufacturing facilities for clinical and commercial production, including a 150,000 sq ft facility in Shannon, Ireland, and a 29,000 sq ft facility in London, UK[10] - The Shannon facility has up to 12 viral vector suites with 2x 500L bioreactors per suite, while the London facility has 2 cell suites and 3 viral vector suites[10]