JCR Pharmaceuticals Co., Ltd.

MEDIPAL HOLDINGS CORPORATION and JCR Pharmaceuticals Co., Ltd. announced that the first participant has been dosed in the global Phase I/II trial of JR-446 for mucopolysaccharidosis type IIIB, also known as Sanfilippo syndrome type B. JR-446 is a blood-brain barrier-penetrating alpha-N-acetylglucosaminidase developed by JCR using its proprietary J-Brain Cargo technology, and MEDIPAL holds commercialization rights for JR-446 outside Japan. The global, multicenter, open-label trial is enrolling patients with MPS IIIB under six years of age and is registered on ClinicalTrials.gov under identifier NCT07640984. MPS IIIB is an ultra-rare lysosomal storage disorder estimated to affect approximately 500 to 1,000 people worldwide, and there is currently no approved treatment. Under a September 2023 licensing agreement, MEDIPAL holds exclusive worldwide commercialization rights excluding Japan with the right to sublicense, while JCR leads the global Phase I/II trial and development activities as sponsor; a separate Phase I/II trial, JR-446-101, is ongoing in Japan under the companies' co-development agreement, and JR-446 received orphan drug designation in the United States, the European Union, and Japan in 2025.

Acumen Pharmaceuticals announced new data at the Alzheimer's Association International Conference 2026 showing its Enhanced Brain Delivery candidate ACU401 achieved up to 40-fold greater frontal cortex exposure in non-human primates compared to a native anti-AβO antibody. The bispecific antibodies, developed with JCR Pharmaceuticals, target both amyloid beta oligomers and the transferrin receptor, and demonstrated improved brain penetration without hematological safety signals. The company also presented patient experience findings from the ALTITUDE-AD Phase 2 trial, highlighting unmet needs in early Alzheimer's disease. Acumen is advancing sabirnetug in the ongoing ALTITUDE-AD study, with topline results expected in late 2026.

Acumen Pharmaceuticals will present three studies at the Alzheimer’s Association International Conference 2026 in London, highlighting progress on its Enhanced Brain Delivery technology and patient-perspective research from the ALTITUDE-AD trial. The presentations include an oral session on bispecific antibodies that bind Aβ oligomers and transferrin receptors, showing enhanced brain delivery in cynomolgus monkeys, and two posters—one on utilizing transferrin receptor-mediated transport for anti-Aβ oligomer antibodies and another on understanding early symptomatic Alzheimer’s disease through patient perspectives from the ALTITUDE-AD trial population. The EBD research is part of an ongoing collaboration with JCR Pharmaceuticals, leveraging Acumen’s AβO-selective antibody expertise and JCR’s J-Brain Cargo technology to improve brain delivery and develop more effective Alzheimer’s treatments. The conference runs July 12 to 15, 2026.