News

Hyogo, Japan – July 15, 2026 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced its development plan in Japan for givinostat, a treatment for Duchenne muscular dystrophy (DMD), following consultation with the Pharmaceuticals and Medical Devices Agency (PMDA).

Based on the outcome of the consultation, JCR plans to:

• Submit a marketing authorization application in Japan in 2026, primarily using overseas clinical data
• Obtain approval and launch the treatment in Japan in 2027
• Conduct a separate clinical trial in Japanese patients with DMD to evaluate pharmacokinetics and safety

Givinostat is an oral histone deacetylase inhibitor licensed from Italfarmaco S.p.A. It has already been approved in multiple countries and regions, including the United States, the European Union, and the United Kingdom, for the treatment of DMD in patients aged 6 years and older, in accordance with local prescribing information. As announced in December 2025, JCR holds exclusive rights to develop and commercialize the treatment in Japan. Because its mechanism of action is not dependent on specific dystrophin gene mutations, givinostat has the potential to be used in patients with DMD regardless of underlying mutation type.

“Following our consultation with PMDA, we have established a specific development plan in Japan,” said Hiroyuki Sonoda, Ph.D., President and Chief Scientific Officer of JCR. “By using overseas clinical data, we believe this development strategy can help bring this treatment to patients with DMD in Japan more quickly. DMD remains an area of significant unmet medical need, and we will advance development with the goal of obtaining approval and launching it in Japan in 2027.”

JCR remains committed to advancing research and development to help meet the expectations of patients and families waiting for effective treatment options.

The impact of this announcement on JCR’s consolidated financial results for this fiscal year (ending March 31, 2027) is expected to be minor.

About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a rare, progressive neuromuscular disorder caused by mutations in the dystrophin gene. These mutations prevent the production of functional dystrophin, causing the dystrophin-associated protein complex (DAPC) to break down. This makes muscle fibres more vulnerable to damage and increases histone deacetylase (HDAC) levels in the muscle cells, blocking the activation of important genes needed for muscle maintenance and repair. As a result, muscle fibres experience ongoing damage, leading to chronic inflammation and poor regeneration. Over time, muscle cells die and are replaced by scar tissue and fat1-4. DMD primarily affects males, with symptoms typically appearing between the ages of two and five years. As the condition progresses, muscle weakness worsens, leading to difficulty walking and eventually loss of ambulation. Over time, the heart and respiratory muscles are also affected, which are the leading causes of premature death5. DMD is one of the most severe and common forms of childhood muscular dystrophy, with a global birth incidence of approximately 1 in 5,050 boys6. DMD affects an estimated 3,500 patients in Japan7.

About Givinostat
Givinostat (Duvyzat®) was discovered through Italfarmaco’s research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). Givinostat is an orally administered histone deacetylase (HDAC) inhibitor that regulates the excessive HDAC activity characteristic of DMD muscles. By doing so, it helps restore the expression of key genes and biological processes essential for muscle maintenance and repair. Its mechanism of action is independent of the specific dystrophin gene mutation causing the disease.

About Italfarmaco S.p.A.
Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development and approval of many pharmaceutical products around the world. The Italfarmaco group has operations in more than 90 countries through directly controlled or affiliated companies. The company is a leader in pharmaceutical research, product development, production and commercialisation with proven success in many therapeutic areas including immuno-oncology, gynaecology, neurology, cardiovascular disease and rare diseases. Italfarmaco’s rare disease unit includes programmes in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis and polycythaemia vera.

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.

References:

1. Sandonà M, Cavioli G, Renzini A, et al. Histone Deacetylases: Molecular Mechanisms and Therapeutic Implications for Muscular Dystrophies. Int J Mol Sci. 2023;24(5):4306. https://doi.org/10.3390/ijms24054306.
2. Consalvi S, Saccone V, Giordani L, Minetti G, Mozzetta C, Puri PL. Histone Deacetylase Inhibitors in the Treatment of Muscular Dystrophies: Epigenetic Drugs for Genetic Diseases. Mol Med. 2011;17(5):457–465. https://doi.org/10.2119/molmed.2011.00049.
3. Bez Batti Angulski A, Hosny N, Cohen H, et al. Duchenne muscular dystrophy: disease mechanism and therapeutic strategies. Front Physiol. 2023;14:1183101. https://doi.org/10.3389/fphys.2023.1183101.
4. Giuliani G, Rosina M, Reggio A. Signaling pathways regulating the fate of fibro/adipogenic progenitors (FAPs) in skeletal muscle regeneration and disease. FEBS J. 2022;289(21):6484–6517. https://doi.org/10.1111/febs.16080.
5. Walter MC, Reilich P. Recent developments in Duchenne muscular dystrophy: facts and numbers. J Cachexia Sarcopenia Muscle. 2017;8(5):681–685. https://doi.org/10.1002/jcsm.12245.
6. Crisafulli S, Sultana J, Fontana A, Salvo F, Messina S, Trifirò G. Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis. Orphanet J Rare Dis. 2020;15(1):141. https://doi.org/10.1186/s13023-020-01430-8.
7. Kawai M. The Number of Patients with Duchenne Muscular Dystrophy in Japan. No To Hattatsu. (Japanese) 2013;45(Supple.):S324.

Contact:
Investors & Media:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
ir-info@jp.jcrpharm.com

– Strategic partnership, announced in July 2025, utilizes JCR’s J-Brain Cargo® to develop a novel blood-brain barrier-penetrating therapy targeting a pathological driver of Alzheimer’s disease

– Option exercise puts Acumen’s Enhanced Brain Delivery (EBD™) program on track for IND filing in mid-2027

Hyogo, Japan – June 17, 2026 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; JCR), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today announced that Acumen Pharmaceuticals, Inc. (NASDAQ: ABOS) exercised its exclusive option to develop, manufacture, and commercialize, on a global basis, up to two candidates for development in the treatment of Alzheimer’s disease (AD), enabled by JCR’s proprietary blood-brain barrier (BBB)-penetrating technology platform, J-Brain Cargo®.

The partnership, announced in July 2025, focuses on developing a BBB-penetrating treatment for AD 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 AD. The collaborative program aims to demonstrate the feasibility of applying J-Brain Cargo® in delivering sabirnetug, Acumen’s targeted immunotherapy drug candidate, and other AβO-selective antibodies across the BBB to slow the progression of AD pathology. Earlier this year, Acumen presented non-clinical data from a mouse model of AD at the International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders, which demonstrated that using AβO-selective antibodies with J-Brain Cargo® technology increased brain exposure while preserving target engagement as demonstrated by AβO selectivity. Acumen plans to file an investigational new drug application for this program in mid-2027.

Under the terms of the agreement, JCR will receive an option payment with Acumen’s decision to exercise its exclusive option to develop, manufacture, and commercialize, on a global basis, 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 JPY83.2 billion converted at the exchange rate of JPY 150 to USD). In addition, JCR is entitled to receive tiered royalties based on net sales for any products that emerge from the collaboration.

“We’re pleased that our strategic partner, Acumen Pharmaceuticals, opted to exercise its exclusive option to develop, manufacture, and commercialize, on a global basis up to two candidates for the treatment of Alzheimer’s disease,” said Hiroyuki Sonoda, Ph.D., President and Chief Scientific Officer of JCR Pharmaceuticals. “Acumen continues to progress the collaborative development program in Alzheimer’s disease, and the non clinical data that Acumen reported earlier this year are truly encouraging. 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 to potentially improve the ability to treat AD safely and effectively.”

“As we build on the strength of our sabirnetug program, we’re pleased to exercise our exclusive option to develop, manufacture, and commercialize, on a global basis up to two candidates for the treatment of Alzheimer’s disease,” said Daniel O’Connell, Chief Executive Officer of Acumen Pharmaceuticals. “By pairing our AβO-selective antibody expertise with JCR’s blood-brain barrier-penetrating technology, J-Brain Cargo®, we have the potential to deliver a next-generation therapeutic which could further expand and enhance the treatment paradigm for people living with Alzheimer’s disease. We look forward to sharing updates when they are available.”

AD is a progressive neurodegenerative disorder affecting more than 50 million people globally and is a leading form 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 central nervous system (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. The J-Brain Cargo® technology platform has a well-established safety profile through several non-clinical studies, clinical trials and marketing experience in Japan.

The impact of this announcement on JCR’s consolidated financial results for the fiscal year ending March 31, 2027, 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 features is the accumulation of amyloid-beta (Aβ) in the brain, and increasing evidence highlights soluble Aβ oligomers as a particularly neurotoxic species that are thought 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), which was approved in Japan for the treatment of a lysosomal storage disorder (LSD). With J-Brain Cargo®, JCR seeks to address the unresolved clinical challenges of LSDs by delivering the enzyme to both the body and the brain.

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.

About Sabirnetug (ACU193)
Sabirnetug (ACU193) is a humanized monoclonal antibody (mAb) discovered and developed based on its selectivity for soluble amyloid beta oligomers (AβOs), which are a highly toxic and pathogenic form of Aβ, relative to Aβ monomers and amyloid plaques. Soluble AβOs have been observed to be potent neurotoxins that bind to neurons, inhibit synaptic function, and induce neurodegeneration. By selectively targeting toxic soluble AβOs, sabirnetug aims to address the hypothesis that soluble AβOs are an early and persistent underlying cause of the neurodegenerative process in Alzheimer’s disease (AD). Sabirnetug has been granted Fast Track designation for the treatment of early AD by the U.S. Food and Drug Administration and is currently being evaluated in a Phase 2 study in patients with early AD.

About Acumen Pharmaceuticals, Inc.
Acumen Pharmaceuticals is a clinical-stage biopharmaceutical company developing a novel therapeutic that targets toxic soluble amyloid beta oligomers (AβOs) for the treatment of Alzheimer’s disease (AD). Acumen’s scientific founders pioneered research on AβOs, which a growing body of evidence indicates are early and persistent triggers of Alzheimer’s disease pathology. Acumen is currently focused on advancing its investigational product candidate, sabirnetug (ACU193), a humanized monoclonal antibody that selectively targets toxic soluble AβOs, in its ongoing Phase 2 clinical trial ALTITUDE-AD (NCT06335173) in early symptomatic AD, following positive results in its Phase 1 trial INTERCEPT-AD. Acumen is investigating a subcutaneous formulation of sabirnetug using Halozyme’s proprietary ENHANZE® drug delivery technology. Acumen is also collaborating with JCR Pharmaceuticals to develop an Enhanced Brain Delivery™ (EBD™)-enabled therapy for Alzheimer’s disease utilizing a transferrin-receptor-targeting blood-brain barrier-penetrating technology. The company is headquartered in Newton, Mass. For more information, visit www.acumenpharm.com.

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.

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.

Contact:
Investors & Media:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications
ir-info@jp.jcrpharm.com