Takeda partners with JCR Pharmaceuticals to commercialise JR-141 protein

Takeda and JCR Pharmaceuticals yesterday announced an exclusive collaboration and license agreement to commercialise JR-141, an investigational, next-generation recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase (IDS) enzyme for treating hunter syndrome (also known as mucopolysaccharidosis type II or MPS II), said a statement by Takeda Pharma.

Hunter syndrome is caused by a deficiency of IDS and manifests in different forms. JR-141, applied with J-Brain Cargo, JCR’s proprietary blood-brain barrier (BBB) technology, is engineered to transport the therapeutic enzyme across the BBB to directly reach the brain and address both the somatic and neuronopathic manifestations of the disease, which can lead to progressive cognitive decline, it informed.

It also said that under the terms of the exclusive collaboration and license agreement, Takeda will exclusively commercialise JR-141 outside of the United States, including Canada, Europe and other regions (excluding Japan and certain other Asia-Pacific countries). JCR will receive an upfront payment for such ex-US licence, and is eligible to receive additional development and commercial milestones as well as tiered royalties on potential sales. The two companies will collaborate to bring this therapy to patients as quickly as possible upon completion of the global phase-III programme, which will be conducted by JCR.

Takeda receives an option under a separate option agreement, which allows it to acquire an exclusive license to commercialise JR-141 in the US upon completion of the phase-III programme, the statement added.

“Takeda is committed to continuously improve the way hunter syndrome is treated. JR-141 introduces a new way to deliver proteins across the blood-brain barrier, overcoming our current challenges to treat the underlying neuronopathic manifestations of hunter syndrome and help maintain or improve cognitive function in these patients,” said Dan Curran, MD, Head, Rare Genetics and Hematology Therapeutic Area Unit, Takeda.

He added, “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 as quickly as possible.”

In addition to it, Shin Ashida, President, Chairman, JCR, said, “JCR is pleased to have reached an agreement with Takeda that is well-placed to achieve our common goal of maximising the impact of JR-141. Our mission is to provide transformative treatment options as soon as possible to patients with Lysosomal Storage Disorders (LSDs) with central nervous system symptoms, such as hunter syndrome. JR-141 is the first-ever approved biopharmaceutical in Japan that penetrates the blood-brain barrier. I expect that we will be able to achieve this mission through our partnership with Takeda to deliver a new treatment option to hunter patients around the world as swiftly as possible.”

As per the statement, JR-141 met its primary endpoint in an open-label phase II/III clinical trial in Japan demonstrating significant reductions in heparan sulfate (HS) in the cerebrospinal fluid, a biomarker for assessing the drug’s effectiveness in reducing disease-causing substrate in the central nervous system, in all subjects for whom measurements were available after 52 weeks of treatment. Somatic disease control was maintained in patients who switched from standard Enzyme Replacement Therapy (ERT). The study also demonstrated an improvement in somatic symptoms in participants who had not previously received standard ERT prior to the beginning of the trial. Additionally, a neuro-cognitive development assessment demonstrated maintenance or improvement of age-equivalent function in 21 of the 25 patients at one year. There were no reports of serious treatment-related adverse events in the trial.