MPS IIIA is a lysosomal storage disorder represented by various somatic and neurological signs and symptoms. Credit: Gorodenkoff / Shutterstock.com.

JCR Pharmaceuticals has dosed the first patient in the Phase I/II clinical trial of JR‑441 for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome Type A).

The trial is carried out at the International Center for Lysosomal Disorders (ICLD) of the University Hospital of Hamburg Eppendorf (UKE) in Germany.

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The single-centre study aims to enrol 12 patients aged one to 18 years at different stages of disease progression.

Meanwhile, the open-label trial compares two different JR-441 doses for 52 weeks.

Its main objective is to establish the safety of chronic dosing in MPS IIIA patients, assess the pharmacokinetics of JR-441, and explore early efficacy signs.

After completing the treatment period, patients taking part in the trial will have a chance to enter an extension study.

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JCR Pharmaceuticals chairman, president and CEO Shin Ashida said: “JR-441 is the third programme based on the J-Brain Cargoplatform that enters the clinical stage, following JR-141 and JR-171.”

“It is another testament to our efforts to bring our innovation to patients across the globe. My deepest respect goes to the families, Dr Muschol, and all the physicians and clinical staff who are so committed to changing the future of individuals affected with this disease.”

Developed using the company’s J-Brain CargoBBB-penetrating technology, JR‑441 is a blood-brain barrier penetrating form of heparan N-sulfatase.

It is a recombinant fusion protein of antibody fragment designed to target the human transferrin receptor and heparan N-sulfatase, the enzyme which is missing or malfunctioning in MPS IIIA patients.

MPS IIIA is a lysosomal storage disorder characterised by various somatic and neurological signs and symptoms with no standard of care that is established.