SEATTLE– Immusoft, a clinical-stage cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for ISP-001 in the treatment of MPS I. The open IND enables Immusoft to initiate a Phase 1 clinical study.
This milestone is both significant for Immusoft and historic in the field of cell and gene therapies, as ISP-001 will be the first engineered B cell therapy to enter into human clinical trials. Immusoft has pioneered the engineering of B cells to create biofactories for in vivo therapeutic protein delivery, leading the field with over 60 issued and pending patents. The company’s novel B cell platform was designed to circumvent immunogenicity associated with virus-delivered gene therapy and chemotherapy preconditioning associated with stem cell-mediated gene therapy, while enabling durable therapeutic delivery and the possibility to re-dose.
MPS I is a rare, childhood genetic disease that affects the body’s ability to produce an essential enzyme, resulting in progressive damage to tissues and organs. Children with MPS I require frequent infusions, which impacts quality of life and is expensive. Immusoft’s ISP™ approach is designed to enable reprogramming of the patient’s B cells for constant production of therapeutic proteins, mitigating the need for frequent infusions with the possibility to improve patient outcomes. ISP-001 represents the first product candidate developed using the company’s proprietary techniques.
“There is a clear need for additional therapies for MPS I, as current interventions are not curative and significant disease related morbidity still exists,” said Paul Orchard, M.D. Professor, Division of Pediatric Bone Marrow Transplantation, University of Minnesota Medical Center. “A non-viral gene delivery method such as ISP-001 holds great promise with the potential to provide definitive therapy.” Dr. Orchard serves as the principal investigator on the ISP-001 Phase 1 clinical trial.
“The ISP platform is a first-in-class technology and a pioneering new approach to treat human disease. It has been rewarding to collaborate with patients, the FDA, investigators, and the Immusoft organization to achieve this important milestone and to launch the first-in-human clinical study,” said Robert Sikorski, MD PhD, Chief Medical Officer, Immusoft.
With this IND clearance, Immusoft is now in a strong position to advance its broad pipeline of therapies toward the clinic, including near-term rare disease programs and expanding into areas including cardiovascular, autoimmune and CNS. The company has partnered with the California Institute for Regenerative Medicine (CIRM) in the development of its program for MPS II, a rare childhood genetic disease. Immusoft has also entered into a research collaboration and license option agreement with Takeda Pharmaceutical Company Limited aimed at delivering protein therapeutics across the blood-brain barrier to treat neurometabolic disorders.
“We are pleased with the FDA’s clearance of our IND application for our lead candidate ISP-001 in MPS I. This is a huge achievement for the Company and a historic moment in the field of cell and gene therapies,” said Sean Ainsworth, CEO and Chairman, Immusoft. “I look forward to working with our team and investigators to initiate our Phase I clinical trial later this year and to continue our commitment to the development of our engineered B cell platform for patients in need.”
