Immusoft's Phase 1 trial of ISP-001, an engineered B cell therapy, has yielded positive initial results in a patient with mucopolysaccharidosis type I (MPS I). The data, which will be presented on October 14, 2024, showcase pharmacodynamic effects and functional improvements observed up to nine months following a single dose of the therapy. This trial is supported by an $8 million grant from the California Institute for Regenerative Medicine (CIRM).
The study participant, an adult with the Hurler-Scheie form of MPS I, experienced notable improvements in quality of life, activities of daily living, and a reduction in pain associated with the disease. Importantly, the administration of ISP-001 did not require any preconditioning, a common necessity in other cell and gene therapies, and was well-tolerated, with no adverse events reported as of the data cutoff date of September 19, 2024.
Clinical Observations and Expert Commentary
"The data from this Phase 1 trial are highly encouraging. We have observed pharmacodynamic improvements as well as functional improvements that were unanticipated in an adult patient, where some manifestations were not expected to be reversible," stated Paul Orchard, MD, Principal Investigator, Professor, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy University of Minnesota Medical School.
Paul Harmatz, MD, Pediatric Gastroenterologist, UCSF Benioff Children’s Hospitals, added, "The data to date point to tremendous potential in the treatment of MPS I, as well as numerous other applications. I am excited to follow and be a part of the progress in this current study and future developments."
Trial Design and Patient Enrollment
The Phase 1 trial (NCT05682144) involves a single infusion of ISP-001, with assessments including biomarkers of disease, functional outcomes, and patient-reported outcomes. The study is currently recruiting patients with the Hurler-Scheie or Scheie forms of MPS I. The first patient received the lowest dose planned in this study and remained on standard of care for a portion of the study.
About Mucopolysaccharidosis Type I
MPS I is a rare, inherited lysosomal storage disorder caused by a deficiency in the enzyme alpha-L-iduronidase (IDUA), which is essential for breaking down long-chain sugars called glycosaminoglycans (GAGs). The accumulation of GAGs leads to progressive cellular damage, affecting various tissues and organs, including the brain. The severe form of MPS I typically manifests within the first year of life, with affected children rarely surviving beyond ten years. Attenuated forms of MPS I present later in childhood.
Immusoft's Innovative Approach
Immusoft's Immune System Programming (ISP™) platform modifies a patient's B cells to produce gene-encoded medicines. These reprogrammed B cells act as miniature protein therapeutic biofactories, offering the potential for sustained therapeutic protein delivery over many years. The company has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for ISP-001 in MPS 1.
Future Directions
Immusoft is also pursuing programs in other lysosomal storage disorders, including MPS II, as well as active programs in CNS, metabolic disease, and oncology. According to Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM, this collaboration with Immusoft leverages the first-ever engineered B cell platform technology to create novel solutions for this severe rare disease, and this innovative platform technology may provide real solutions for unmet medical needs across a broad number of indications.
