Orchard Therapeutics plc (NASDAQ:ORTX) announced its intention to focus its hematopoietic stem cell (HSC) gene therapy platform exclusively on neurometabolic diseases and early research programs.
- Orchard intends to discontinue its investment and seek alternatives for rare primary immune deficiency programs, including:
- OTL-103 in Wiskott-Aldrich syndrome (WAS).
- OTL-102 for X-linked chronic granulomatous disease (X-CGD).
- Strimvelis, a gammaretroviral vector-based gene therapy approved in Europe for adenosine deaminase severe combined immunodeficiency (ADA-SCID).
- Orchard will continue its investment in Libmeldy (atidarsagene autotemcel) / OTL-200 for metachromatic leukodystrophy (MLD).
- The Company also will continue to advance the clinical development of OTL-203 for mucopolysaccharidosis type I Hurler's syndrome (MPS-IH) and OTL-201 for mucopolysaccharidosis type IIIA (MPS-IIIA).
- Regarding the regulatory status of the OTL-103 program, Orchard recently received written feedback from the FDA. It believes the path to a potential BLA filing (late 2022 or early 2023) may require additional time and further investment.
- The Company will reduce its current workforce by approximately 30%, which will result in a restructuring charge in 2022.
- Cash, cash equivalents, and investments of approximately $220.1 million, with $33.0 million of debt outstanding, are expected to fund Orchard Therapeutics expenditure requirements into 2024.
- Price Action: ORTX shares are trading at $0.78 during the market session on the last check Wednesday.
