Orchard Therapeutics announced today that it intends to focus its hematopoietic stem cell (HSC) gene therapy platform exclusively on severe neurometabolic diseases and early research programs.
Orchard CEO Bobby Gaspar/GEN
Orchard Therapeutics announced Wednesday that it intends to focus its hematopoietic stem cell (HSC) gene therapy platform exclusively on severe neurometabolic diseases and early research programs. In addition to refining its portfolio, the company said it will reduce its current workforce by 30%, noting that such changes will extend its cash runway into 2024.
Orchard’s portfolio focuses on the curative potential of HSC gene therapy, which uses blood stem cells to help a patient’s body create new blood cells of all types to help eliminate certain diseases. The company stated that moving forward, it will continue its investment in Libmeldy (atidarsagene autotemcel) for metachromatic leukodystrophy (MLD), a rare hereditary disorder that causes lipids to build up in cells of the brain causing a broad spectrum of neurological symptoms.
Libmeldy has had recent commercial momentum in Europe, with the company announcing in February that it had reached an agreement with the National Health Service that provides access to Libmeldy for all children with MLD in England and Wales. Orchard is expected to initiate regulatory filings for the commercialization of the therapeutic in the U.S. as well.
In addition to developing Libmeldy, Orchard stated it plans to continue to advance the clinical development of its therapeutic OTL-203 for mucopolysaccharidosis type I Hurler’s syndrome (MPS-IH) and OTL-201 for mucopolysaccharidosis type IIIA (MPS-IIIA), genetic disorders that cause lysosomes to aggregate in cells enlarging tissues and organs. Focusing on neurometabolic disorders allows Orchard to channel its energies into conditions with immense unmet needs and leverage clinical validation of HSC therapies for such disorders.
“In light of our experiences and knowledge gained in this current and rapidly evolving market environment for gene therapy, our plan is to concentrate resources on programs that have the potential to make a remarkable difference to patients while also providing sustainable value to the business to enable the achievement our long-term vision,” said Bobby Gaspar, M.D., Ph.D., CEO of Orchard. “As launch momentum for Libmeldy continues to build in Europe and we prepare for a regulatory filing in the U.S., a focused strategy that utilizes a common infrastructure for future neurometabolic disease launches is critical to our success as a commercial gene therapy company.”
Along with this portfolio refinement, Orchard’s workforce will be cut by 30%, resulting in restructuring changes that will take place this year. The announcement of these cuts comes after the U.S. Food and Drug Administration provided written feedback to Orchard regarding its Biologics License Application (BLA) for OTL-103, a therapeutic intended to treat Wiskott Aldrich Syndrome (WAS), a life-threatening inherited immune disorder.
Orchard stated that the written feedback from the FDA prompted the reduction in its workforce. The path to a potential BLA filing may require more time and investment than initially thought, so the company has decided to abandon the program and find alternatives. OTL-103 joins several other programs in the pile of abandoned programs including OTL-102 for X-linked chronic granulomatous disease (X-CGD) and Strimvelis for adenosine deaminase severe combined immunodeficiency (ADA-SCID).
Orchard also anticipates reporting preclinical data for its therapeutic program investigating OTL-104 in the treatment of Crohn’s Disease by the end of 2022, with plans to file an Investigational New Drug (IND) application in 2024.
