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Orchard Therapeutics Advances New Strategic Plan and Reports Second Quarter 2020 Financial Results

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Regulatory and Clinical Progress in MLD, MPS-I and MPS-IIIA Illustrate Execution in Neurometabolic Disorders; EU Regulatory Decision for Libmeldy™ Expected in 2H 2020  
MolMed and GSK Agreements Provide Near-term Manufacturing Capacity and Highlight Future InnovationsNear-term Financial Position Secure with Cash and Investments of ~$230M and Runway into 2022BOSTON and LONDON, Aug. 06, 2020 (GLOBE NEWSWIRE) — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reviewed business updates and upcoming milestones supporting its new strategic plan and reported financial results for the quarter ended June 30, 2020.“Implementing Orchard’s new strategic plan yielded strong results in the second quarter as we marked important regulatory and clinical progress in our neurometabolic programs and strengthened our current and future manufacturing position,” said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. “Our commercial infrastructure and launch plans continue to develop with a focus on site qualification, diagnostic initiatives, pricing and reimbursement as we prepare for the potential approval of Libmeldy (formerly OTL-200) in the EU. Towards the end of the year, we plan to share more detail from our new research programs in FTD and Crohn’s disease, which we believe highlight the potential for HSC gene therapy beyond ultra-rare diseases.”Recent Corporate Achievements Supporting New Strategic PlanPrioritize portfolio investments to realize opportunities for high need, high value indications, with an emphasis on neurometabolic conditionsLibmeldy (OTL-200) for MLD:Submitted responses to the Day 120 questions from the European Medicines Agency (EMA) for the marketing authorization application (MAA) for Libmeldy (OTL-200) for the treatment of metachromatic leukodystrophy (MLD).OTL-203 for MPS-I:Received orphan drug and rare pediatric disease designations for the treatment of mucopolysaccharidosis type I (MPS-I) from the U.S. Food and Drug Administration (FDA).Presented interim data from the ongoing proof-of-concept trial of OTL-203 at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting. The first primary outcome measure of the study was met with all eight patients achieving hematologic engraftment within 45 days of treatment. In addition, clinical data from the first two patients treated have shown improved motor function, stable cognitive scores and continued normal growth after at least one year of follow-up. Link to full release here.OTL-201 for MPS-IIIA:Enrolled three patients in the proof-of-concept study of OTL-201 for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA).Establish focused commercial model for diagnosis and treatment of patients globallyExtended the gene therapy manufacturing relationship with MolMed S.p.A (an AGC Inc. company) for a period of five years through June 2025. MolMed will continue to support activities related to the development and manufacturing of vectors and drug product for multiple Orchard investigational programs, including for the potential upcoming commercial launch of Libmeldy (OTL-200) for MLD. Link to full release here.Invest in next-generation manufacturing technology and process innovationsEntered into worldwide royalty-bearing license agreements with GlaxoSmithKline plc (GSK) for use of their proprietary lentiviral stable cell line technology in gene therapy products for the treatment of Wiskott Aldrich syndrome (WAS) and transfusion-dependent beta thalassemia (TDT). Link to full release here.Planned Corporate MilestonesA Media Snippet accompanying this announcement is available by clicking on the image or link below:

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