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Orchard Therapeutics Announces U.S. FDA Clearance of IND Application for OTL-203 in MPS-IHBOSTON and LONDON, Jan. 05, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for OTL-203, a hematopoietic stem cell (HSC) gene therapy being developed for the treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The company expects to initiate a global registrational trial evaluating the efficacy and safety of OTL-203 compared to standard of care in the second half of 2023. “Based on data from the proof-of-concept trial, treatment with a single administration of OTL-203 has the potential to address a range of multisystemic manifestations of MPS-IH,” said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics. “Our recent interactions with the FDA have been productive and we look forward to advancing this registrational study designed to generate the data necessary to enable global regulatory submissions and potentially provide an urgently needed new treatment option for the MPS-IH community.” The study is a multi-center, randomized, active controlled clinical trial designed to evaluate the efficacy and safety of OTL-203 in patients with MPS-IH compared to standard of care with allogeneic hematopoietic stem cell transplant (HSCT). A total of 40 patients with a confirmed diagnosis of MPS-IH who meet the study inclusion criteria will be randomized 1:1 to receive either OTL-203 or allogeneic HSCT. The study is powered to demonstrate superiority of OTL-203 over HSCT. The primary endpoint, which will be measured at two years post-treatment, comprises a composite of clinically meaningful outcomes, including death, the need for rescue treatment, treatment failure, immunological complications, as well as severe cognitive and growth impairment. Secondary endpoints include biochemical markers, additional clinical assessments, as well as safety and tolerability. The company expects to open up to six sites in the United States and Europe. “The complications associated with MPS-IH involve multiple organ systems and have a lasting impact on patients’ quality of life despite treatment with allogeneic HSCT,” said Paul Orchard, M.D., a study investigator and professor in the Division of Pediatric Blood and Marrow Transplantation and Cellular Therapy Program at the University of Minnesota Medical School. “Existing therapeutic options are associated with significant morbidity and mortality. There is experience acquired over decades that treatment with allogeneic HSCT does not adequately impact manifestations of the disorder such as growth and other skeletal issues. Furthermore, patients can still experience an irreversible decline in neurocognitive function. We look forward to facilitating this study to characterize the potential clinical impact of OTL-203 for MPS-IH patients.” In an earlier, single center proof-of-concept study, eight patients diagnosed with MPS-IH were treated with investigational OTL-203 between July 2018 and December 2019. Previously published results showed all patients had stable cognitive development post-treatment. In addition, at last follow-up (ranging from 12-24 months), all participants had progressed along expected growth percentiles of healthy children and exhibited longitudinal growth that was considered within the normal range adjusted for age and gender. Treatment with OTL-203 was generally well-tolerated with no indication of insertional oncogenesis and no evidence of clonal dominance due to integration into oncogenes reported to date. “MPS-IH is a devastating disease that places a significant burden on affected children and their families,” said Terri Klein, president and chief executive officer of the National MPS Society. “New treatment options are desperately needed to better address some of the more severe symptoms of the disease. We are encouraged by today's announcement and the hope that a one-time gene therapy could offer this community if approved.” About MPS-I Treatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have limitations, such as inadequate impact on some of the more severe manifestations of disease, as well as significant morbidity and mortality. At present, Newborn Screening (NBS) for MPS-I has been established in multiple geographies, including the United States and Europe. About OTL-203 About Orchard Therapeutics In 2018, the company acquired GSK’s 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. Today, Orchard is advancing a pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society 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 www.orchard-tx.com, and follow us on Twitter and LinkedIn. Availability of Other Information About Orchard Forward-looking Statements Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchard’s most recent annual or quarterly report filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchard’s views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law. Dr. Paul Orchard is a paid consultant for Orchard Therapeutics.  Contacts Investors Renee Leck Senior Director, Investor Relations +1 862-242-0764 [email protected] Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 [email protected]
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