Immusoft to Announce Positive Phase 1 Data for First B Cell Therapy

Immusoft of CA, a subsidiary of Immusoft Corporation, revealed promising results from its Phase 1 trial of ISP-001 in a patient with mucopolysaccharidosis type I (MPS I). The trial is backed by an $8 million award from the California Institute for Regenerative Medicine (CIRM), a leading institute in regenerative medicine.

Sean Ainsworth, CEO of Immusoft, highlighted that while the primary focus of Phase 1 was safety, the trial showed significant pharmacodynamic and functional improvements even at low doses. The positive initial results will be discussed in detail during Immusoft's MPS I Clinical Update and Outreach webinar on October 14, 2024.

The trial involved the first-ever clinical use of engineered B cells, specifically ISP-001, in a human patient with the Hurler-Scheie form of MPS I. Observations included improvements in pharmacodynamics, function, quality of life, daily activities, and pain reduction, with no adverse events reported as of September 19, 2024. The administration of ISP-001 did not require the usual preconditioning regimen associated with other cell and gene therapies, and the infusion was well-tolerated.

Paul Orchard, MD, Principal Investigator and Professor at the University of Minnesota Medical School, expressed optimism about the trial's outcomes, noting unexpected improvements in an adult patient. Similarly, Paul Harmatz, MD, Pediatric Gastroenterologist at UCSF Benioff Children's Hospitals, emphasized the potential of ISP-001 in treating MPS I and other conditions.

The trial recruits patients with Hurler-Scheie or Scheie forms of MPS I and administers a single infusion of ISP-001. Assessments include biomarkers, functional outcomes, and patient-reported outcomes. Participants continue their standard care during part of the study. For more details on the trial protocol, visit clinicaltrials.gov (NCT05682144).

Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for ISP-001 in MPS I. Dr. Abla Creasey, Vice President of Therapeutics Development at CIRM, expressed enthusiasm about the collaboration with Immusoft, emphasizing the innovative platform technology's potential to address a broad range of unmet medical needs.

Immusoft is not only focusing on MPS I but also has programs targeting other lysosomal storage disorders like MPS II, as well as conditions affecting the central nervous system, metabolic diseases, and oncology.

Mucopolysaccharidosis type I (MPS I) is a rare genetic disorder that impairs the body's ability to produce the enzyme alpha-L-iduronidase (IDUA), essential for breaking down long-chain sugars inside cells. The accumulation of these sugars leads to progressive cellular damage, affecting various tissues, including the brain. Severe MPS I often results in a lifespan of less than ten years post-diagnosis, with symptoms appearing within the first year of life. The attenuated form of MPS I manifests later in childhood. The incidence of severe MPS I is about 1 in 100,000 births, while the attenuated form occurs in approximately 1 in 500,000 births.

Immusoft specializes in developing therapies for rare diseases using its Immune System Programming (ISP™) technology, which reprograms a patient's B cells to produce gene-encoded medicines. These engineered B cells act as miniature biofactories for protein therapeutics, potentially persisting for many years.

CIRM, created to accelerate stem cell treatments for patients with unmet medical needs, partners with academia and industry to develop promising stem cell technologies. With $5.5 billion in funding and over 150 active stem cell programs, CIRM is a leading force in regenerative medicine.