On September 1, Immusoft announced that the FDA has approved the clinical trial application of ISP-001 for mucopolysaccharidosis type I, which is the world’s first engineered B cell therapy to enter the clinical stage. It has previously received FDA orphan drug designation.
Mucopolysaccharidosis type I (MPS I) is a rare and fatal childhood genetic disorder caused by a mutation in the IDUA gene that results in a deficiency of α-L-iduronidase, an enzyme that helps break down intracellular Essential enzyme for mucopolysaccharides. When sugar chains cannot be degraded, they can accumulate in cells and cause progressive tissue or organ damage, which can be life-threatening in severe cases.
There are currently two main treatment options for MPS I, hematopoietic stem cell transplantation and BioMarin’s enzyme replacement therapy injection laronidase (laronidase). However, enzyme replacement therapy requires frequent infusions, which greatly affects quality of life. Immusoft’s ISP-001 therapy is designed to reprogram patients’ B cells to consistently produce therapeutic proteins, reducing the burden of frequent infusions and potentially improving patient outcomes.
Immusoft’s pipeline is deployed in a variety of rare diseases, cardiovascular and autoimmune diseases. The ISP-001 approved for clinical use this time is the one with the fastest progress.
The ISP platform pioneered by the company can genetically reprogram the patient’s own B cells, so that the B cells can be upgraded into “biofactories” (differentiated into plasma cells) after undergoing immune stimulation, and the plasma cells can be reinfused back into the patient’s body. Plasma cells will efficiently secrete tens of thousands of personalized antibodies, enabling sustained delivery of therapeutic proteins.
In October 2021, Immusoft and Takeda entered into a strategic collaboration to leverage Immusoft’s immune system programming technology platform to co-develop B-cell therapies for rare neurometabolic diseases. Under the terms of the agreement, Immusoft receives an undisclosed down payment and research funding, and is eligible to receive a combined option fee and mileage of more than $900 million.
“We are delighted that the FDA has approved the clinical application for ISP-001, our lead product candidate in the MPS I field. This is a huge achievement for the company and a historic moment in the cell and gene therapy arena.” Chief Executive Officer, Immusoft Sean Ainsworth, Chief Executive Officer and Chairman, said: “As we initiate Phase I clinical trials this year, our team is committed to developing engineered B-cell therapies for patients in need.”