RARE Daily

JCR Pharmaceuticals Enters Research Collaboration with Alexion to Develop Neurodegenerative Disease Therapy

April 4, 2023

Rare Daily Staff

JCR Pharmaceuticals said that it has signed a research collaboration, option, and license agreement with AstraZeneca’s rare disease subsidiary Alexion to develop an undisclosed initial therapeutic molecule that applies JCR’s proprietary blood-brain barrier penetration technology for the treatment of a neurodegenerative disease.

“The collaboration with Alexion is a milestone in the recognition of the J-Brain Cargo technology as it is for the first time applied for the treatment of a neurodegenerative disease,” said Shin Ashida, president and chairman of JCR. Further adding, “We are confident that this new partnership will lead to new therapies for the treatment of neurodegenerative disease an area with high unmet need and desperately needed therapeutic innovation.”

J-Brain Cargo allows the delivery of biotherapeutics into the central nervous system through a mechanism called receptor-mediated transcytosis. The first drug approved in Japan using the J-Brain Cargo technology is Izcargo for the treatment of a lysosomal storage disease with significant central nervous signs and symptoms.

The platform technology has broad applicability to many therapeutic effectors, and it has the potential to treat multiple CNS indications.

Under the terms of the agreement, the two companies will begin research and development of new treatments using the J-Brain Cargo technology in combination with an undisclosed effector molecule. During the research period, Alexion also has the option to nominate additional therapeutic protein candidates in undisclosed indications. Following pre-clinical development, Alexion may exercise its option to further develop and commercialize new drug candidates under this collaboration.

JCR will receive an immediate upfront payment from Alexion and has the right to receive certain research, development and commercial milestones and royalties upon the achievement of certain milestone events, including on post approval net sales.

Photo: Shin Ashida, president and chairman of JCR

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