Cell therapy biotech Immusoft will partner Takeda to develop new cell therapies to target rare inherited metabolic disorders with central nervous system (CNS) manifestations and complications in a deal that could be worth more than $900M if all targets are met.
Immusoft was founded in 2009 and is headquartered in Seattle. It has pioneered an approach the company calls ‘Immune System Programming’ that involves extraction of B cells from the patient’s blood, engineering these cells to express proteins lacking in specific diseases, expansion of cells and re-infusion. Essentially a non-viral, cell-based gene therapy.
The U.S. biotech has several products in development in the rare and common disease space, but its most advanced candidate to date is targeting Hurler syndrome or MPS-I, a rare and lethal childhood genetic disease where specific sugars cannot be broken down properly and so accumulate in cells causing damage. This candidate therapy has not yet gone into Phase I trials, which are predicted to start sometime in the next 6 months. It received Rare Pediatric Disease Designation from the FDA for it in fall 2018.
The next two most advanced candidates from the company are a treatment for muscle wasting in the neurological rare diseases amyotrophic lateral sclerosis (ALS) and Duchenne muscular dystrophy (DMD) and for Hunter syndrome or MPS-II. However, it also has others in the discovery phase.
The deal with Takeda, the biotech’s first with big pharma, will involve using Immusoft’s platform to develop new treatments for rare diseases in the metabolic and CNS space focusing on getting proteins past the blood brain barrier. Rare diseases are something that Takeda has had a strong focus on since its acquisition of Shire in 2019.
The initial payment was not disclosed by the companies, but Immusoft will receive initial payment and funding for research and will be eligible for future option fees and milestone payments worth more than $900M if they are successful. This funding builds on Immusoft’s Series B round, which raised $20M in 2019.
“This advances our leadership position in B cells as biofactories for therapeutic protein delivery, a novel approach that Immusoft has pioneered. This partnership provides Immusoft with significant resources to further develop our Immune System Programming technology platform, and therapies in diseases for which patients have limited options,” said Sean Ainsworth, Chief Executive Officer, Immusoft, in a press statement.
As part of the partnership, Takeda will be able to license programs from Immusoft at the preclinical stage, taking responsibility for further development, and Immusoft will then receive later royalties should the candidate therapy receive market approval.
“We continue to build our internal capabilities as well as partner with innovative companies early on in the discovery process to advance our next-generation gene and cell therapy ambitions for rare genetic and hematologic diseases,” said Takeda Rare Diseases Drug Discovery Unit Head Madhu Natarajan. “Working together with Immusoft, we hope to validate their ISP technology for CNS delivery of innovative therapeutics for rare neurometabolic diseases.”