- Strategic partnership utilizes JCR’s J-Brain Cargo® to develop a novel blood-brain barrier-penetrating therapy targeting a pathological driver of Alzheimer’s disease -

JCR Pharmaceuticals Co., Ltd. (TSE 4552; JCR), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced a joint collaboration, option and license agreement with Acumen Pharmaceuticals to develop a novel therapeutic candidate for the treatment of Alzheimer’s disease (AD), enabled by JCR’s proprietary blood-brain barrier (BBB)-penetrating technology platform, J-Brain Cargo®.

The collaboration focuses on developing a blood-brain barrier-penetrating treatment for Alzheimer’s that combines JCR’s J-Brain Cargo® with Acumen’s amyloid beta oligomer (AβO)-selective antibodies, which target toxic soluble AβOs, a key pathological driver in the onset and progression of Alzheimer’s disease. The program aims to demonstrate the feasibility of J-Brain Cargo® in delivering sabirnetug and other AβO-selective antibodies across the BBB to slow the progression for AD pathology.

Under the terms of the agreement, JCR will receive an upfront payment from Acumen and will be eligible for an additional option payment should Acumen exercise its exclusive option to develop, manufacture, and commercialize worldwide up to two candidates from the collaboration. JCR will also be eligible to receive future milestone payments of up to USD 40 million related to development, and up to USD 515 million related to sales, for a total of up to USD 555 million (approximately JPY80.5 billion converted at the exchange rate of JPY 145 to USD). In addition, JCR is entitled to receive tiered royalties based on net sales for any products that emerge from the collaboration.

“Alzheimer’s disease remains one of the greatest unmet needs in medicine, and the collaboration with Acumen marks a significant milestone in our effort to address it,” said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals. “By combining our J-Brain Cargo® platform with Acumen’s novel, AβO-selective antibodies, we aim to overcome the challenge of delivering biologics to the brain, which has limited progress in treating neurodegenerative disease safely and effectively. This collaboration not only advances a promising approach in Alzheimer’s but also reinforces J-Brain Cargo® as a clinically validated, scalable platform for CNS drug development.”

Alzheimer’s disease is a progressive neurodegenerative disorder affecting more than 50 million people globally and is a leading cause of dementia. One of its defining pathological hallmarks is the accumulation of Aβ in the brain, which are believed to trigger a cascade of events leading to neuronal damage, cognitive decline, and memory loss.1 Delivering biologics across the BBB remains a fundamental obstacle to effectively treating the disease.

J-Brain Cargo® is JCR’s proprietary platform that enables the systemic delivery of biotherapeutics to the CNS through a mechanism known as receptor-mediated transcytosis. The technology has been clinically validated with IZCARGO®, the first approved drug in Japan to use the platform, for the treatment of a lysosomal storage disorder with CNS involvement. With broad potential to deliver a wide range of biologic therapies across the BBB, J-Brain Cargo® is a versatile platform for advancing treatments for complex CNS conditions, including neurodegenerative diseases. Through preclinical and clinical trials, the J-Brain Cargo® technology platform has an established safety profile.

“Acumen was founded on the belief that selectively targeting toxic forms of amyloid-beta can meaningfully change the course of Alzheimer’s disease,” said Daniel O’Connell, Chief Executive Officer of Acumen Pharmaceuticals. “This collaboration with JCR allows us to explore a powerful new approach to delivering therapies directly to the brain. By pairing our AβO-selective antibody expertise with JCR’s J-Brain Cargo® platform, we have the potential to advance a differentiated therapeutic that could change the treatment paradigm for people living with Alzheimer’s disease.”

The impact of this announcement on JCR's consolidated financial results for the fiscal year ending March 31, 2026, has already been factored into the current earnings forecast.

About Alzheimer’s Disease

Alzheimer’s disease is a progressive neurodegenerative disorder affecting more than 50 million people globally and is a leading cause of dementia. One of its defining pathological hallmarks is the accumulation of amyloid-beta Aβ plaques in the brain, which are believed to trigger a cascade of events leading to neuronal damage, cognitive decline and memory loss.1

About the J-Brain Cargo® Platform Technology

JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology J- Brain Cargo®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology is IZCARGO® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder.

About JCR Pharmaceuticals Co., Ltd.

JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that develops treatments that go beyond rare diseases to solve the world’s most complex healthcare challenges. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the U.S., Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. Our core values – Putting people first, Forging our own path, Always advancing, and Committed to excellence – mean that the work we do benefits all our stakeholders, including employees, partners, and patients. We strive to expand the possibilities for patients while accelerating medical advancement at a global level. For more information, please visit JCR’s global website: https://jcrpharm.com/.

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Reference

1. Sadigh-Eteghad S, Sabermarouf B, Majdi A, Talebi M, Farhoudi M, Mahmoudi J. Amyloid-beta: a crucial factor in Alzheimer's disease. Med Princ Pract. 2015;24(1):1-10. www.doi.org/10.1159/000369101.

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“Alzheimer’s disease remains one of the greatest unmet needs in medicine, and the collaboration with Acumen marks a significant milestone in our effort to address it,” said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals.