Pharmacodynamic effects and functional improvements were observed through nine months post-dosing with a single administration of ISP-001.
As of the data cutoff date of September 19, 2024, no adverse events have been reported.
Data for ISP-001 for Mucopolysaccharidosis Type I to be presented on October 14, 2024 (details below).
SAN FRANCISCO – September 23, 2024 – Immusoft of CA, a wholly owned subsidiary of Immusoft Corporation (“Immusoft”), a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced positive results from the Phase 1 ISP-001 trial evaluating the therapy in a patient with mucopolysaccharidosis type I (MPS I).
The clinical trial is supported by an $8 million award from the California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutes dedicated to regenerative medicine.
“Although the Phase 1 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,” said Sean Ainsworth, Immusoft Chief Executive Officer.
Initial ISP-001 trial results will be presented during Immusoft’s MPS I Clinical Update and Outreach webinar.
Title: ISP-001: Data from the first-ever engineered B cell in a human clinical trialDate: Monday, October 14, 2024, 4:00 pm ET / 1:00 pm PTWebinar Registration
Agenda:
The first adult patient to receive ISP-001 in this study, with the Hurler-Scheie form of MPS I, experienced pharmacodynamic improvements, functional improvements, as well as reported improvements in quality of life, activities of daily living, and reductions in pain associated with MPS I.
The administration of ISP-001 required no preconditioning regimen typically associated with other cell and gene therapeutics, and the infusion was well-tolerated. There have been no adverse events reported as of September 19, 2024.
“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,” said Paul Orchard, MD, Principal Investigator, Professor, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy University of Minnesota Medical School.
“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,” said Paul Harmatz, MD, Pediatric Gastroenterologist, UCSF Benioff Children’s Hospitals.
In the ISP-001 trial, eligible participants receive a single infusion of ISP-001, with assessments that include biomarkers of disease, functional outcomes, and patient-reported outcomes. This 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. For additional details on the trial protocol, please see clinicaltrials.gov (NCT05682144). For patients interested in participating, please visit the patient recruitment website. The company has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for ISP-001 in MPS I.
“We are enthusiastic about this collaboration with Immusoft leveraging the first-ever engineered B cell platform technology to create novel solutions for this severe rare disease. We believe this innovative platform technology may provide real solutions for unmet medical needs across a broad number of indications,” said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM.
In addition to its clinical MPS I program, Immusoft has programs in other lysosomal storage disorders including MPS II as well as active programs in CNS, metabolic disease, and oncology.
About MPS I (Mucopolysaccharidosis type I)MPS I (Mucopolysaccharidosis type I) is a rare, lethal childhood genetic disease that affects the body’s ability to produce IDUA (alpha-L-iduronidase), an essential enzyme in the breakdown of long-chain sugars inside cells. When the sugar chains cannot be broken down and disposed of, they accumulate in the cells and cause progressive damage. This accumulation can happen in the tissues, including the brain. In its most severe form, children affected rarely live longer than ten years after diagnosis. Severe MPS I occurs in about 1 in 100,000 births and symptoms appear within a child’s first year of life. In what is referred to as attenuated MPS I, symptoms appear later in childhood. It occurs in about 1 in 500,000 births.
About ImmusoftImmusoft of CA is a wholly owned subsidiary of Immusoft Corporation. Immusoft is a clinical-stage cell therapy company focused on developing novel therapies for rare diseases using a sustained delivery of protein therapeutics from a patient’s own cells. The company has developed a technology platform called Immune System Programming (ISP™), which modifies a patient’s B cells and instructs the cells to produce gene-encoded medicines. The B cells that are reprogrammed using ISP become miniature protein therapeutic biofactories that are expected to persist for many years. For more information, visit www.immusoft.com.
About the California Institute for Regenerative Medicine (CIRM)
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast-track the development of today’s most promising stem cell technologies.
With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.
Media:Susan RobertsRoberts Communicationssr@roberts-communications.com202-779-0929
First-in-human clinical trial of genetically engineered B cells: Application to the treatment of mucopolysaccharidosis type I
