JCR Pharmaceuticals Completes Phase I Trial Notification in Japan for JR-441 in MPS IIIA

JCR Pharmaceuticals Co., Ltd. (TSE 4552), based in HYOGO, Japan, has announced the successful completion of the regulatory review by the Pharmaceuticals and Medical Devices Agency (PMDA) for its clinical trial notification for the Phase I study of JR-441. This study targets individuals with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A.

JR-441 is a recombinant form of heparan N-sulfatase developed using JCR's proprietary J-Brain Cargo® technology, which allows the drug to penetrate the blood-brain barrier (BBB). This technology is significant because it addresses the neurological symptoms of MPS IIIA by delivering treatment directly to the central nervous system, a critical area affected by the disease.

MPS IIIA is a rare and severe lysosomal storage disorder caused by mutations in the SGSH gene, which encodes an enzyme necessary for the breakdown of heparan sulfate. The accumulation of this substance leads to severe neurological deterioration, cognitive impairment, and a shortened lifespan, with no current approved treatments available.

Shin Ashida, Chairman and President of JCR Pharmaceuticals, emphasized the importance of this development. He expressed the company's commitment to advancing the JR-441 clinical development program to address the unmet medical needs of those affected by MPS IIIA. Ashida also highlighted the progression of the clinical trial and the company's dedication to providing updates as they become available.

JR-441 has already received orphan drug designation from the European Commission (EC) and the U.S. Food and Drug Administration (FDA) for the treatment of MPS IIIA. In addition to the Japanese Phase I study, JR-441 is being evaluated in an ongoing Phase I/II clinical trial in Germany under the identifier JR-441-101, NCT06095388.

JCR Pharmaceuticals has indicated that the financial impact of this announcement for the current fiscal year, ending March 2025, is expected to be minor.

JCR Pharmaceuticals Co., Ltd. is a specialty pharmaceuticals company with a global presence, focusing on rare and genetic diseases. The company has a 49-year legacy in Japan and is expanding its reach into the United States, Europe, and Latin America. JCR Pharmaceuticals aims to improve patient outcomes through its scientific expertise and innovative technologies, developing next-generation therapies.

The company offers approved treatments in Japan for conditions such as growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus-host disease, and renal anemia. Additionally, the company is developing investigational products for a range of rare diseases, including MPS I (Hurler, Hurler-Scheie, and Scheie syndrome), MPS II, and MPS IIIA and B (Sanfilippo syndrome types A and B).

JCR Pharmaceuticals is dedicated to advancing medical science on a global scale, driven by core values of reliability, confidence, and persistence. These values benefit all stakeholders, including employees, partners, and patients. The company remains committed to its mission of expanding possibilities for patients with rare diseases and accelerating medical advancements worldwide.