MEDIPAL HOLDINGS and JCR Pharmaceuticals sign global licensing and Japan co-development agreement for JR-479, a novel therapy for GM2 gangliosidosis
Tokyo and Hyogo, Japan – August 29, 2025 – MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the two companies have signed an exclusive global licensing deal and a co-development and commercialization partnership in Japan for JR-479, an investigational therapy for GM2 gangliosidosis, an ultra-rare lysosomal storage disorder.
GM2 gangliosidoses are a group of rare, inherited disorders caused by genetic mutations that prevent the normal breakdown of GM2 gangliosides in lysosomes, leading to their buildup, especially in neurons. Tay-Sachs disease and Sandhoff disease are the two most common types of GM2 gangliosidosis depending on the specific mutation. GM2 gangliosidosis affects an estimated one in 300,000 people worldwide and is considered an ultra-rare, life-threatening disorder for which no approved treatments currently exist.
JR-479 is a novel drug candidate developed with JCR’s proprietary J-Brain Cargo® platform, clinically validated through IZCARGO® for I.V. infusion 10mg, which gained approval in Japan in 2021 for the treatment of a lysosomal storage disorder. In preclinical studies,¹ JR-479 was delivered throughout the body, including to the central nervous system, reducing disease-related substrates and extending survival in animal models.
Under the global licensing deal, MEDIPAL will have exclusive worldwide rights (excluding Japan) to develop, manufacture, and commercialize JR-479, with sub-licensing rights. In Japan, through the co-development partnership, MEDIPAL will handle clinical trial logistics, support disease awareness, and assist with operations, while JCR leads commercialization and records domestic revenue. JCR will also receive an upfront payment, and royalties on overseas sales.
This agreement adds JR-479 to the strategic collaboration between MEDIPAL and JCR, launched in October 2022, to develop treatments for ultra-rare diseases. It follows JR-471 for fucosidosis² and JR-446 for mucopolysaccharidosis type IIIB,³ making JR-479 the third pipeline in the partnership.
Both companies have already factored the financial impact of this agreement into their consolidated forecasts for the fiscal year ending March 31, 2026.
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 rare lysosomal storage disorder.
About lysosomal storage diseases
Lysosomal storage diseases are genetic disorders in which certain enzymes or proteins that remove unwanted substances from cells do not work as they should. When that happens, those substances build up inside the lysosomes and damage cells and tissues. The symptoms differ depending on what builds up, and many of these conditions also affect the central nervous system.
About GM2 Gangliosidosis (Tay-Sachs, Sandhoff disease)
GM2 gangliosidosis is a group of rare, inherited lysosomal storage disorders caused by deficiency of β-hexosaminidase A (Hex A), an enzyme consisting of α and β subunits. Depending on whether the α- or β- subunit is deficient, GM2 gangliosidosis is classified as Tay-Sachs or Sandhoff disease, respectively. Deficiency of Hex A results in the progressive accumulation of GM2 gangliosides and other glycolipids, which leads to severe neurodegeneration. In severe cases, nearly half of affected children do not survive past the age of three.⁴ Currently, there are no approved treatments, highlighting the urgent need for new therapies.
About MEDIPAL HOLDINGS CORPORATION
MEDIPAL HOLDINGS CORPORATION is a holding company which controls, administers and supports the operating activities of companies in which it holds shares in the Prescription Pharmaceutical Wholesale Business; the Cosmetics, Daily Necessities and OTC Pharmaceutical Wholesale Business; and the Animal Health Products and Food Processing Raw Materials Wholesale and Related Business, and conducts business development for the MEDIPAL Group. For more information, visit https://www.medipal.co.jp/english/.
About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. is a global specialty pharmaceutical company dedicated that develops treatments that go beyond rare diseases to solve the world’s most complex healthcare challenges. JCR continues to build upon our 50-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. JCR’s innovative therapies address conditions like growth disorder, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. JCR is also developing treatments for rare diseases like MPS I, MPS II, MPS IIIA and B, and more.
Cautionary Statement Regarding Forward-Looking Statements
This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as “believe,” “estimate,” “anticipate,” “intend,” “plan,” “will,” “would,” “target” and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors’ pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.
References
1. Press release on research presentations at WORLDSymposium™ 2025 (February 5, 2025).
2. Press release on the exclusive negotiation rights for global commercialization targeting four ultra-rare diseases and the licensing contract for the fucosidosis therapeutic between MEDIPAL and JCR (October 31, 2022).
3. Press release on the licensing agreement for JR-446 between MEDIPAL and JCR (September 28, 2023).
4. Bley AE, et al. Natural history of infantile G(M2) gangliosidosis. Pediatrics. 2011;128(5): e1233-1241.
Contacts
MEDIPAL HOLDINGS CORPORATION
Public Relations Department
TEL: (+81)-3-3517-5171
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
E-mail：ir-info@jp.jcrpharm.com
JCR Pharmaceuticals and Acumen Pharmaceuticals announce collaboration to develop therapy for Alzheimer’s disease, enabled by J-Brain Cargo® technology platform
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
Hyogo, Japan – July 15, 2025 - 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. ADD 1 SUP
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 partners, patients and employees. We strive to expand the possibilities for patients while accelerating medical advancement at a global level.
Cautionary Statement Regarding Forward-Looking Statements
This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as “believe,” “estimate,” “anticipate,” “intend,” “plan,” “will,” “would,” “target” and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors’ pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.
Contact:
Investors & Media:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
ir-info@jp.jcrpharm.com
JCR Pharmaceuticals enters license agreement with Alexion for proprietary JUST-AAV capsids to be used in the development of genomic medicines
Hyogo, Japan – July 8, 2025 - JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) today announced that it has entered into a license agreement with Alexion, AstraZeneca Rare Disease (“Alexion”), for JCR’s new, proprietary JUST-AAV capsids to develop genomic medicines.
JUST-AAV encompasses a range of vector types optimized for various target tissues—including liver-sparing, muscle-targeting, and brain-targeting variants—to expand the potential of AAV-based gene therapy.
Under the terms of the agreement, Alexion may use the licensed capsids, which are part of the JUST-AAV platform, in up to five of Alexion's genomic medicines programs. JCR will receive an upfront payment from Alexion. JCR is eligible to receive milestone payments of up to USD 225 million related to research and development, and up to USD 600 million related to sales, for a total of up to USD 825 million (approximately JPY 120 billion converted at the exchange rate of JPY 145 to USD), which reflects the aggregate milestones if all five targets are exercised. In addition, JCR is entitled to receive tiered royalties based on net sales.
This agreement marks the third partnership between JCR and Alexion, following research collaborations involving J-Brain Cargo® technology—the first announced in March 2023 for neurodegenerative disease and the second announced in December 2023 for the discovery of oligonucleotide therapeutics.
“I am very pleased to announce the signing of this license agreement for our proprietary AAV capsid platform, JUST-AAV” said Shin Ashida, Chairman, President and CEO of JCR. “I believe JUST-AAV has the potential to make a real difference in the development of gene therapies for rare diseases, and I look forward to its application across a broader range of conditions. JCR values its partnership with Alexion, and we are pleased to build on the collaboration we have developed over the years.”
The impact of this agreement on JCR’s consolidated financial results for the fiscal year ending March 31, 2026, has already been factored into the current earnings forecast.
About JUST-AAV
JUST-AAV is a proprietary platform technology that utilizes modified adeno-associated virus (AAV) vectors. The technology entails insertion of miniaturized antibodies against receptors on selected tissues, organs or the blood-brain barrier onto the capsid surface, enhancing targeted delivery to those tissues and organs. Further capsid modifications minimize off-target effects and improve safety. The name is derived from “JCR” “Ultimate destination of organ” “Safeguarding against off-target delivery” and “Transformative technology” reflecting its potential for broad application across various diseases.
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 and approved in Japan for the treatment a lysosomal storage disorder is IZCARGO® (INN: pabinafusp alfa).
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.
Cautionary Statement Regarding Forward-Looking Statements
This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as “believe,” “estimate,” “anticipate,” “intend,” “plan,” “will,” “would,” “target” and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors’ pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.
Contact:
Investors & Media:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
ir-info@jp.jcrpharm.com