MEDIPAL HOLDINGS CORPORATION and JCR Pharmaceuticals dose first patient in global phase I/II trial of JR-446 for mucopolysaccharidosis type IIIB
– The study aims to evaluate the safety, tolerability, and preliminary efficacy of JR-446 for the treatment of people with MPS IIIB, for which there is no approved therapy –
Tokyo and Hyogo, Japan – September 11, 2026 – MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) announced today that the first participant has been dosed in the global Phase I/II trial of JR-446 for mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B; “MPS IIIB”). JR-446 is a blood-brain barrier-penetrating α-N-acetylglucosaminidase developed by JCR using its proprietary J-Brain Cargo® technology. MEDIPAL holds commercialization rights for JR-446 outside Japan.
MPS IIIB is a lysosomal storage disorder that causes severe central nervous system impairment. This ultra-rare disease is estimated to affect approximately 500 to 1,000 people worldwide,1 and there is currently no approved treatment.
“Dosing the first patient in this Phase I/II clinical trial is an important milestone for the MPS IIIB community, as there is no approved treatment for this devastating and life limiting lysosomal storage disorder that impacts patients’ neurocognitive development, independence, and quality of life,” said Dr. Irene Chang, Principal Investigator, Associate Professor of Pediatrics, Section of Biochemical Genetics, Division of Medical Genetics, at the University of California, San Francisco (UCSF). “Patients and families affected by this condition continue to face significant unmet medical needs, and we are encouraged to take this next step in evaluating the potential of this investigational therapy. We are grateful to the patients, caregivers, and study teams whose commitment made this milestone possible, and we are hopeful it may bring meaningful improvements in the quality of life for individuals living with MPS IIIB and their families.”
In September 2023, MEDIPAL and JCR entered into a licensing agreement for the commercialization of JR-446 for MPS IIIB outside Japan and a co-development and commercialization agreement in Japan.2 Under this agreement, MEDIPAL holds exclusive worldwide commercialization rights, excluding Japan, with the right to sublicense. On behalf of MEDIPAL, JCR is leading the global Phase I/II trial and development activities as the sponsor.
The global, multicenter, open-label trial is being conducted in patients with MPS IIIB under six years of age. Further information is available at ClinicalTrials.gov under identifier NCT07640984.
Nonclinical studies of JR-446 have demonstrated effects on symptoms associated with MPS IIIB. A separate Phase I/II trial, JR-446-101, is ongoing in Japan under the companies’ co-development agreement. In 2025, JR-446 received orphan drug designation in the United States, the European Union, and Japan.
MEDIPAL and JCR remain committed to advancing JR-446 with the goal of bringing a new treatment option to people with MPS IIIB as quickly as possible.
About Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)
Mucopolysaccharidosis type IIIB, or Sanfilippo syndrome type B, is an autosomal recessive disease caused by pathogenic mutations in the NAGLU gene, encoding a lysosomal enzyme involved in the degradation of heparan sulfate. With the accumulation of heparan sulfate in the central nervous system in the brain, individuals with this condition present rapid neurological decline, including sleep disorders, loss of speech, and behavioral changes, which may significantly affect the quality of life of patients and their families.
About the J-Brain Cargo® Platform Technology
JCR Pharmaceuticals has developed a proprietary blood-brain barrier (BBB)-penetrating technology, J-Brain Cargo®, to bring biotherapeutics into the central nervous system (CNS). The first drug developed based on this technology is IZCARGO™ (INN: pabinafusp alfa), which is approved in Japan and the United Arab Emirates for the treatment of a lysosomal storage disorder (LSD). With J-Brain Cargo®, JCR and MEDIPAL seek to address the unresolved clinical challenges of LSDs by delivering the enzyme to both the body and the brain.
About MEDIPAL HOLDINGS CORPORATION
MEDIPAL 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 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 its 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. Based on data from JCR’s own investigations, referring to the Ministry of Health, Labour and Welfare’s public research.
2. Press release on the licensing agreement for JR-446 between MEDIPAL and JCR (September 28, 2023).
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 joins “Relay For Life of Ashiya” in support of the cancer community
Hyogo, Japan – September 10, 2026 – JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) sponsored and participated in “Relay For Life of Ashiya,” held in Ashiya, Hyogo, on September 5 and 6. The community charity event is organized by local volunteers and managed by the Relay for Life Kansai Executive Committee and the Japan Cancer Society under license from the American Cancer Society.
Relay For Life brings communities together to support people living with cancer and their families. Funds raised through sponsorships and donations support Japan Cancer Society initiatives including research into new treatments and drug development, free cancer counseling, and efforts to encourage cancer screening. JCR continues to support the event through its sponsorship and participation.
This year, approximately 50 JCR employees and family members from across the company joined the Relay Walk and Candlelight Run. Walking through the night toward dawn, they reflected on what it means for patients to face cancer every day as they ran and walked forward together, one step at a time.
Employees shared reflections such as: “Taking part gave me many new insights and much to learn. It was an invaluable opportunity to gain a firsthand sense of the thoughts and feelings of patients, their families and supporters, and the warmth of the connections among them.” “Running through the night was tough, but the encouragement all around the venue kept me going, one step at a time. I was glad to run even one kilometer farther and contribute more to the fundraising effort.”
JCR will continue to take on difficult healthcare challenges beyond rare disease, while strengthening ties with local communities and contributing to society.
About Relay For Life
Relay For Life began in 1985, when a surgeon in the United States ran around a track for 24 hours to raise donations for the American Cancer Society. His effort symbolized the reality that people with cancer face their illness every hour of every day. What started as a single act of solidarity has
grown into a global movement that inspires courage and hope by bringing people together to walk, talk, and share. Today, Relay For Life is held in approximately 1,700 locations across 37 countries worldwide.
In Japan, Relay For Life continues to expand nationwide, with approximately 52 events scheduled in 2026. Ashiya has officially hosted the event as an overnight gathering since 2007, and this year marks its 20th edition under the theme, “This is where I took a step forward. This is where I meet you again.”
For more information, please visit the official website (Japanese only):
Relay For Life of Ashiya: https://relayforlife.jp/ashiya/.
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.
Contact:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications / IR Office
ir-info@jp.jcrpharm.com
JCR Pharmaceuticals receives marketing authorization in the United Arab Emirates for IZCARGO™, a blood-brain barrier-penetrating enzyme replacement therapy for hunter syndrome
– This marketing authorization is the first for IZCARGOTM outside of Japan –
Hyogo, Japan – July 21, 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 IZCARGOTM (pabinafusp alfa, or JR-141), a blood-brain barrier-penetrating enzyme replacement therapy (ERT) for Hunter syndrome developed using JCR’s proprietary J-Brain Cargo® technology, received marketing authorization in the United Arab Emirates (UAE).
This marks the first marketing authorization for IZCARGOTM outside of Japan. IZCARGOTM was approved and launched in Japan in 2021 and is currently being evaluated in an ongoing global Phase III clinical trial.
JCR has been working to expand access to IZCARGOTM outside of Japan while advancing its global clinical development program. As part of this effort, JCR entered into a marketing and distribution agreement with Taiba Middle East FZ LLC (“Taiba”) and pursued regulatory approval in the UAE based on the product’s marketing authorization in Japan. Under this agreement, JCR will also seek marketing authorization for IZCARGOTM in countries across the MENA region, while Taiba will be responsible for sales and distribution in the relevant territories.
“We are very pleased to announce the approval of IZCARGOTM in the UAE,” said Hiroyuki Sonoda, Ph.D., President and Chief Scientific Officer of JCR Pharmaceuticals. “This is an important milestone for JCR, as it represents the first approval of IZCARGOTM outside of Japan. We look forward to working with Taiba to bring this therapy to more patients with Hunter syndrome and to continue expanding access to IZCARGOTM internationally.”
We are pleased to announce the first approval of IZCARGOTM in the MENA region in collaboration with JCR,” said Dr. Saif Al Hasani, Taiba group CEO. “This partnership reflects our shared commitment to supporting patients affected by Hunter disease and other rare conditions. IZCARGOTM brings a unique perspective by focusing on both innovation and accessibility, ensuring that meaningful solutions can reach those who need them. Together with JCR, we look forward to contributing to the advancement of rare disease care in the region”.
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 the J-Brain Cargo® Platform Technology
JCR Pharmaceuticals has developed a proprietary blood-brain barrier (BBB)-penetrating technology, J-Brain Cargo®, to bring biotherapeutics into the central nervous system (CNS). The first drug developed based on this technology is IZCARGOTM (INN: pabinafusp alfa), which is approved in Japan for the treatment of Hunter syndrome, 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 Hunter Syndrome (Mucopolysaccharidosis Type II, or MPS II)
Hunter syndrome (mucopolysaccharidosis type II or MPS II) is an X-linked recessive lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase, an enzyme that breaks down complex carbohydrates called glycosaminoglycans (GAGs, also known as mucopolysaccharides) in the body. Hunter syndrome, which affects an estimated 2,000-3,000 individuals worldwide (according to JCR research), gives rise to a wide range of somatic and neurological symptoms. The current standard of care for Hunter syndrome is enzyme replacement therapy. Central nervous system symptoms related to MPS II have been unmet medical needs so far.
About Pabinafusp Alfa
Pabinafusp alfa (JR-141) is a recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase, the enzyme that is missing or malfunctioning in subjects with Hunter syndrome. It incorporates J-Brain Cargo®, JCR’s proprietary blood-brain barrier (BBB)-penetrating technology, to cross the BBB through transferrin receptor-mediated transcytosis, and its uptake into cells is mediated through the mannose-6-phosphate receptor. This novel mechanism of action is expected to make pabinafusp alfa effective against the central nervous system (CNS) symptoms of Hunter syndrome.
In pre-clinical trials, JCR has confirmed both high-affinity binding of pabinafusp alfa to transferrin receptors and passage across the BBB into neuronal cells. In addition, JCR has confirmed enzyme uptake in various brain tissues. The company has also confirmed a reduction of substrate accumulation in the CNS and peripheral organs in an animal model of Hunter syndrome1,2.
In several clinical trials of pabinafusp alfa, JCR obtained evidence of reducing heparan sulfate (HS) concentrations in the cerebrospinal fluid (CSF), a biomarker for assessing effectiveness against CNS symptoms; these results were consistent with those obtained in pre-clinical studies3. Clinical studies have also demonstrated the positive effects of pabinafusp alfa on CNS symptoms4,5,6.
Pabinafusp alfa was approved in Japan by the Ministry of Health, Labour and Welfare and marketed since May 2021 under the brand name “IZCARGOTM I.V. Infusion 10mg.”
Important Safety Information
INDICATION:
IZCARGOTM is indicated for the treatment of mucopolysaccharidosis type II (MPS II), which is also known as Hunter syndrome. IZCARGOTM is approved in Japan and the UAE.
CONTRAINDICATION:
IZCARGOTM is contraindicated in patients with a history of anaphylactic shock to its components.
WARNINGS AND PRECAUTIONS:
Warnings
Since serious anaphylaxis and shock may occur with use of IZCARGOTM, adequate emergency measures should be made ready for execution before initiation of administration, and the patient should be closely monitored during and after the administration. If a serious infusion associated reaction (IAR) occurs, administration of IZCARGOTM should be discontinued, and appropriate actions should be taken.
When IZCARGOTM is administered to patients with severe respiratory failure or acute respiratory disease, an IAR may lead to acute exacerbation of symptoms. A patient’s condition should be closely monitored, and appropriate actions should be taken as needed.
Precautions for Use
IZCARGOTM is a protein medicinal product and may cause anaphylactic shock, for which close monitoring is required. If any signs of anaphylaxis are noted, discontinue the infusion, and take appropriate actions. Considering the onset of such symptoms, emergency measures should be made ready for execution.
IZCARGOTM may cause IARs such as headache, chills, syncope, fatigue, dizziness, pyrexia, rash, erythema, urticaria, or other symptoms. If an IAR occurs, reduce the rate or temporarily discontinue the infusion, and initiate appropriate drug treatment (e.g., corticosteroids, antihistamines, antipyretic analgesics, anti-inflammatory drugs) or emergency procedures (e.g., oxygen administration, securing of airway, adrenaline administration). Premedication with antihistamines, corticosteroids, etc., should be considered for the subsequent infusion of IZCARGOTM.
ADVERSE REACTIONS:
The most commonly reported adverse reactions were pyrexia and urticaria.
About Taiba Middle East FZ LLC
Taiba is a regional leader in the marketing, distribution, and commercialization of orphan and rare disease therapies across the Middle East and North Africa (MENA). With two decades of trusted collaboration with clinicians, hospitals, and global partners.
Taiba combines deep regional expertise with robust commercial execution—from regulatory approvals and patient access programs to strategic partnerships and long-term brand lifecycle management, the company connects healthcare systems with breakthrough treatments through early access and commercialization initiatives. Driven by a mission to close the treatment gap.
Taiba empowers medical institutions with cutting-edge solutions and exceptional service, ensuring patients with unmet medical needs gain timely and sustainable access to life-changing therapies. please visit http://www.taibarare.com/.
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: Sonoda, et al. A blood-brain-barrier-penetrating anti-human transferrin receptor antibody fusion protein for neuronopathic mucopolysaccharidosis II. Mol. Ther. 2018; 26(5): 1366-1374.
2: Morimoto, et al. Clearance of heparin sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice. Mol. Ther. 2021; 29(5): 1853-1861.
3: Okuyama, et al. Iduronate-2-sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial. Mol Ther. 2020; 27(2): 456-464.
4: Okuyama, et al. A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II. Mol Ther. 2021; 29(2): 671-679.
5: Giugliani, et al. Iduronate-2-sulfatase fused with anti-human transferrin receptor antibody, pabinafusp alfa, for treatment of neuronopathic and non-neuronopathic mucopolysaccharidosis II: Report of a phase 2 trial in Brazil. Mol Ther. 2021; 29(7): 2378-2386.
6: Giugliani, et al. Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II; an Integrated Analysis of Preclinical and Clinical Data. Int. J. Mol. Sci. 2021, Volume 22, Issue 20, 10938.
Contact:
JCR Pharmaceuticals Co., Ltd.
Corporate Communications / IR Office
ir-info@jp.jcrpharm.com
JCR Pharmaceuticals announces development plan in Japan for givinostat for duchenne muscular dystrophy
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