JCR Pharmaceuticals Co., Ltd. (JCR), a leading specialty pharmaceutical company, has announced the achievement of a significant research milestone in its collaboration with Alexion, AstraZeneca Rare Disease (Alexion), focusing on the development of a novel therapeutic molecule for the treatment of a neurodegenerative disease. This development leverages JCR’s proprietary J-Brain Cargo® technology, designed to enhance the penetration of biotherapeutics across the blood-brain barrier (BBB). The accomplishment of this predefined research milestone has triggered a milestone payment to JCR from Alexion.
This recent progress strengthens the ongoing collaboration between JCR and Alexion, which expanded in December 2023 with a second research partnership. This additional collaboration aims to apply J-Brain Cargo® technology to oligonucleotide therapeutics, targeting unmet medical needs in neurodegenerative diseases.
The milestone payment received by JCR is anticipated to have a minor impact on the company’s financial results for the fiscal year ending March 31, 2024. However, it represents a significant step forward in leveraging J-Brain Cargo® technology for developing treatments for neurodegenerative conditions.
The J-Brain Cargo® platform is a pioneering technology allowing for the delivery of therapeutic agents directly into the central nervous system by overcoming the BBB. IZGARGO® (INN: pabinafusp alfa), the first drug developed utilizing this technology, has been approved in Japan for treating Mucopolysaccharidosis (MPS) type II, also known as Hunter Syndrome. Several other drugs based on the J-Brain Cargo® platform are in various stages of global clinical trials, targeting different forms of MPS and other lysosomal storage disorders (LSDs). JCR plans to initiate clinical trials for four additional programs from its LSD pipeline by FY2028.
JCR Pharmaceuticals is dedicated to redefining the treatment landscape for rare and genetic diseases worldwide, building upon a 49-year legacy in Japan while expanding globally. The company focuses on delivering next-generation therapies and has a portfolio that includes approved treatments for growth disorder, Fabry disease, acute graft-versus-host disease, MPS II, and renal anemia, with several investigational products in development for rare diseases.
This forward-looking statement highlights JCR’s commitment to medical innovation and its strategy to accelerate the development of new treatments for rare diseases at a global level, underscoring its mission to expand possibilities for patients while fostering medical progress.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.