JCR Pharmaceuticals and Acumen Pharmaceuticals Forge Strategic Partnership to Develop a Blood-Brain Barrier-Penetrating Alzheimer’s Therapy Using J-Brain Cargo® Platform
JCR Pharmaceuticals Co., Ltd. (TSE: 4552), a Japan-based global specialty biopharmaceutical company renowned for its focus on rare and genetic diseases, has announced a significant strategic partnership with U.S.-based Acumen Pharmaceuticals. The two companies have entered into a joint collaboration, option, and license agreement aimed at developing an innovative therapeutic candidate for the treatment of Alzheimer’s disease (AD). Central to this effort is JCR’s proprietary blood-brain barrier (BBB)-penetrating technology platform, J-Brain Cargo®, which will be leveraged to deliver Acumen’s highly specific amyloid beta oligomer (AβO)-targeting antibodies into the brain.
The alliance combines the core strengths of each company—JCR’s advanced CNS delivery platform and Acumen’s expertise in targeting neurotoxic amyloid-beta species—with the shared goal of creating a new, more effective treatment for Alzheimer’s disease. This progressive neurological disorder affects over 50 million people globally and continues to represent one of the most significant unmet medical needs worldwide. A fundamental challenge in treating AD has been the delivery of therapeutic agents across the BBB, a highly selective barrier that blocks most biologics from reaching the brain. The new partnership aims to overcome this challenge by harnessing J-Brain Cargo® to transport Acumen’s AβO-selective antibodies, such as sabirnetug, across the BBB and directly to the site of disease pathology.
A New Frontier in Alzheimer’s Therapeutics
Under the terms of the agreement, JCR will receive an upfront payment from Acumen, as well as an additional option payment if Acumen chooses to exercise its exclusive option to develop, manufacture, and commercialize up to two therapeutic candidates emerging from the collaboration. Beyond this, JCR stands to receive up to $40 million in development milestones and up to $515 million in commercial sales milestones, totaling as much as $555 million (approximately JPY 80.5 billion). The company is also entitled to tiered royalty payments based on the net sales of any products that successfully advance to commercialization under the partnership.
This agreement underscores the growing global recognition of J-Brain Cargo® as a potentially transformative drug delivery platform. By enabling systemic biotherapeutics to penetrate the BBB via a mechanism known as receptor-mediated transcytosis, the platform represents a breakthrough for addressing complex central nervous system (CNS) disorders. It has already been clinically validated with IZCARGO®, a therapy for a lysosomal storage disorder with CNS involvement, which is approved for use in Japan. This milestone solidified J-Brain Cargo® as the first approved CNS-penetrating biologic platform therapy in Japan and demonstrated the platform’s scalability, clinical viability, and safety profile.
Leadership Perspectives: A Shared Vision for CNS Drug Innovation
Shin Ashida, Chairman, President, and CEO of JCR Pharmaceuticals, highlighted the significance of the collaboration in a statement:
“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. 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.”
Meanwhile, Daniel O’Connell, Chief Executive Officer of Acumen Pharmaceuticals, noted the strategic alignment between the two companies and their shared ambition to alter the trajectory of Alzheimer’s care:
“Acumen was founded on the belief that selectively targeting toxic forms of amyloid-beta can meaningfully change the course of Alzheimer’s disease. 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.”
Scientific Rationale and Disease Background
Alzheimer’s disease is marked by the gradual and irreversible degeneration of neurons, leading to cognitive decline, memory loss, and eventually the inability to perform basic activities of daily living. Among the most studied pathological features of AD is the accumulation of amyloid beta (Aβ) plaques in the brain. However, recent research indicates that soluble amyloid beta oligomers (AβOs)—smaller, more diffusible aggregates—may be even more toxic and central to the disease’s progression.
Acumen’s antibody, sabirnetug, is engineered to selectively bind to these toxic AβOs rather than to monomers or plaque-forming fibrils. This specificity offers the potential for a more targeted intervention that avoids some of the safety issues and limited efficacy seen with earlier amyloid-targeting approaches. However, these types of biologics face a major challenge in reaching the brain due to the restrictive nature of the blood-brain barrier.
JCR’s J-Brain Cargo® platform provides a solution to this problem. It employs a receptor-mediated transcytosis approach, wherein therapeutic antibodies are fused with ligands that bind to specific receptors expressed on the endothelial cells lining the BBB. Once bound, the complex is internalized and transported into the brain tissue. This method has not only demonstrated efficacy in preclinical studies but also achieved real-world validation with the regulatory approval of IZCARGO®.
Potential for Broad CNS Application
The implications of the collaboration extend far beyond a single product. The agreement covers up to two candidates, opening the door for Acumen and JCR to jointly pursue multiple antibody therapeutics optimized for CNS delivery. Given the increasing global burden of Alzheimer’s and related dementias—alongside a wave of aging populations—the market opportunity for safe and effective CNS-penetrant Alzheimer’s treatments is enormous. The Alzheimer’s Disease International organization estimates that the global cost of dementia was more than $1.3 trillion in 2023, with projections expected to soar past $2 trillion by 2030.
By aligning the precision of Acumen’s oligomer-selective antibodies with JCR’s proven BBB-penetrating technology, the two companies are poised to develop a class of therapeutics with potentially superior efficacy, CNS penetration, and safety profiles. This approach may also yield learnings that could be applied to a broader range of neurodegenerative conditions, including Parkinson’s disease, Huntington’s disease, and other protein misfolding-related disorders.
Financial and Market Outlook
The collaboration is not expected to impact JCR’s consolidated financial performance in the near term. The company noted that any earnings from the upfront payment and future milestone revenue have already been factored into its forecast for the fiscal year ending March 31, 2026.
However, the longer-term financial upside for both parties could be substantial. If one or both candidates under development reach the market and achieve commercial success, JCR could receive hundreds of millions of dollars in milestones and royalties, establishing a recurring revenue stream that supports its broader R&D and business operations. For Acumen, gaining access to a clinically validated BBB-penetration platform strengthens its drug development pipeline and differentiates its Alzheimer’s approach from competitors in a crowded and highly scrutinized field.
The collaboration between JCR Pharmaceuticals and Acumen Pharmaceuticals represents a convergence of two complementary technologies: one that excels in precision targeting of Alzheimer’s pathology, and another that solves the long-standing challenge of getting therapeutics across the brain’s formidable protective barrier. As both companies move forward with preclinical and potentially clinical development, the success of this partnership could redefine how biologics are delivered to the brain and reshape the future of Alzheimer’s treatment.
With a shared mission to improve the lives of millions affected by neurodegenerative diseases, JCR and Acumen are embarking on a high-potential endeavor that leverages innovation, validated platforms, and therapeutic precision. This alliance exemplifies how cross-border collaboration can accelerate the translation of scientific breakthroughs into meaningful patient outcomes—potentially ushering in a new era in the fight against Alzheimer’s disease.
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 mostcomplex 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/.
