US-based cell therapy company Immusoft has reported positive results from a Phase I trial of its engineered B-cell therapy, ISP-001, designed to treat mucopolysaccharidosis type I (MPS I).
The trial is being supported by a $8m grant from the California Institute for Regenerative Medicine.
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MPS I is a rare genetic disease in children that affects the body’s ability to break down long-chain sugars inside cells, causing sugar chains to accumulate in cells and damage them.
Subjects in the trial have received a single infusion of the therapy, with participant assessments comprising biomarkers of disease, functional outcomes and patient-reported outcomes.
Immusoft dosed the first subject with ISP-001 in December 2023. The patient received the lowest dose planned for the study and continued on standard care for part of the trial.
The first adult patient in the trial suffering from the Hurler-Scheie form of MPS I reported pharmacodynamic and functional improvements.
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By GlobalDataThis patient had also seen improvements in quality of life and daily functioning, as well as reduced pain associated with the disease.
ISP-001’s administration did not require any preconditioning regimen, as is common with other cell and gene therapies.
The infusion was reported to be well-tolerated, with no adverse events observed to date.
The study is currently recruiting participants with the Hurler-Scheie or Scheie forms of MPS I.
ISP-001 has received orphan drug and rare paediatric disease designations for MPS I from the US Food and Drug Administration (FDA).
Immusoft CEO Sean Ainsworth said: “Although the Phase I focus is safety, we have observed activity and functional improvements, even at the low dose, far beyond our initial expectations, and are very excited by this data.”
Beyond MPS I, Immusoft is extending its research to other lysosomal storage disorders, including mucopolysaccharidosis type II.
The company has active programmes targeting central nervous system disorders, metabolic diseases and oncology.
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