Takeda, JCR ink deal to commercialize Hunter syndrome drug in key markets

Takeda HQ
JCR will receive an upfront payment from Takeda and will be eligible for development and commercial milestones as well as royalties on potential sales. (Takeda)

Six months after gaining approval in Japan for its Hunter syndrome treatment Izcargo, JCR Pharmaceuticals has made a move to prepare for an international rollout. Thursday, Takeda inked a deal with JCR to handle commercialization of the drug in Canada, Europe and other countries outside of the U.S. and Japan should it gain approval in those regions. 

JCR will receive an upfront payment that was not disclosed and will be eligible for development and commercial milestones as well as royalties on potential sales, the companies said. The goal is to quickly bring the therapy to patients following completion of a global late-stage trial conducted by JCR.

Izcargo, also known by its investigational name JR-141, received fast-track designation by the FDA in February of this year.

“We will work closely with JCR to apply our expertise in enzyme replacement therapies with the hope of bringing this potentially transformative therapy to patients,” Dan Curran, M.D., Takeda’s chief of rare genetics and hematology, said in a statement announcing the partnership.

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Hunter syndrome, also known as mucopolysaccharidosis type II, is an inherited lyosomal disease that's caused by a deficiency of an enzyme needed to break down sugar molecules in the body. A buildup of these molecules in tissues and organs leads to debilitating symptoms and cognitive decline. Hunter syndrome affects 1 in 162,000 infants, almost all male. Those with it typically die between the ages of 10 and 20. 

JR-141 is an infused, next-generation enzyme that can penetrate the blood-brain barrier and is engineered to address the symptoms, including neurologic effects, of Hunter syndrome. In a late-stage trial in Japan, HR-141 demonstrated reductions in heparan sulfate in the cerebrospinal fluid, a biomarker for assessing the drug’s effectiveness in the central nervous system. After 52 weeks, the reductions were evident in all patients who switched from standard enzyme replacement therapy.

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JCR's global trial is targeted for completion in 2024.  

The Takeda partnership is part of JCR’s global expansion push. In 2018, the company established JCR USA, based in California, to explore commercial opportunities for its pipeline drugs. JCR’s profile increased recently when AstraZeneca tapped its manufacturing expertise to help produce COVID-19 vaccines for Japan.