 Regulatory and Clinical Progress in MLD, MPS-I and MPS-IIIA Illustrate Execution in Neurometabolic Disorders; EU Regulatory Decision for LibmeldyTM Expected in 2H 2020 MolMed and GSK Agreements Provide Near-term Manufacturing Capacity and Highlight Future Innovations Near-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 Orchards 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 Crohns disease, which we believe highlight the potential for HSC gene therapy beyond ultra-rare diseases.Recent Corporate Achievements Supporting New Strategic Plan Prioritize portfolio investments to realize opportunities for high need, high value indications, with an emphasis on neurometabolic conditions Libmeldy 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 globally Extended 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 innovations Entered 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:Second Quarter 2020 Financial ResultsFrank Thomas, president and chief operating officer added, We have successfully implemented many of the operational changes under our new strategic plan on schedule, including the decommissioning of the California site. We are seeing the financial benefits of a streamlined footprint and organization heading into the second half of 2020 with a disciplined approach to investing in the high value programs we prioritized under the new plan.Research and development expenses were 31.6 million for the second quarter of 2020 compared to 40.5 million in the same period in 2019. Research and development expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and milestone payments under the companys agreements with third parties, and personnel costs to support these activities. RD expenses in 2019 included a one-time 17.2 million in-process research and development charge associated with the in-licensing of our OTL-203 program for the treatment of MPS-I. In 2020, RD expenses included a non-cash restructuring charge of 5.6 million related to the consolidation and closure of our RD and manufacturing facilities in California. Excluding these charges, RD expenses increased by 2.7 million from 23.3 million in the second quarter of 2019 to 26.0 million in the second quarter of 2020.Selling, general and administrative expenses were 15.7 million for the second quarter of 2020 compared to 13.7 million in the same period in 2019. The increase was primarily due to increased launch readiness activities, personnel-related costs and share-based compensation expense.Net loss was 47.5 million for the second quarter of 2020 compared to 50.5 million in the same period in 2019. The decrease was primarily due to the in-process research and development expense for the MPS-I license deal signed in May 2019, offset by increases in RD and SGA expenses. The company had 97.5 million ordinary shares outstanding as of June 30, 2020.Cash, cash equivalents and investments as of June 30, 2020, were 228.7 million compared to 325.0 million as of December 31, 2019. The decrease was primarily driven by cash used to fund operations in the first half of 2020. The company expects that its existing cash, cash equivalents and investments will fund its anticipated operating and capital expenditure requirements into 2022. This excludes the 50 million expected to be available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers.Conference Call Webcast InformationOrchard will host a conference call and live webcast today at 8:00 a.m. ET to discuss its second quarter business highlights and financial results. The conference call will be broadcast live in listen-only mode under News Events in the Investors Media section of the companys website at and a replay will be archived on the Orchard website following the presentation. To ask a question, please dial 866 987-6504 U.S. domestic or 1 602 563-8620 international and refer to conference ID 7392139. Please dial in at least 15 minutes in advance to ensure a timely connection to the call.About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit and follow us on Twitter and LinkedIn.Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website the investor relations website ir.orchard-tx.com, and on social media Twitter and LinkedIn, including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. 