Latest news with #JCR
Business Wire
6 days ago
- Business
- Business Wire
JCR Pharmaceuticals Selected for Japan's Regenerative Medicine CDMO Subsidy Program
HYOGO, Japan--(BUSINESS WIRE)-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; 'JCR'), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, has been selected for the Ministry of Economy, Trade and Industry's 'Regenerative CDMO Subsidy' as of July 15, 2025. The subsidy will support facility upgrades and equipment installation to expand biomanufacturing capacity for regenerative, cell, and gene therapies. Project Overview 1) Project Title JCR Regenerative Medicine CDMO Enhancement 2) Description Building on its expertise in regenerative medicine, JCR will strengthen its biomanufacturing capability through facility upgrades and workforce training. 3) Period Approval date – December 31, 2027 Background and Rationale Since the 2015 approval of TEMCELL ® HS Inj., JCR has steadily built expertise in regenerative medicine while ensuring a stable, long-term product supply. The company is now developing an AAV gene therapy platform, JUST-AAV, based on its proprietary J-Brain Cargo ® technology for delivery across the blood-brain barrier. To leverage these assets as a development and manufacturing partner for regenerative medicine products, JCR applied for this government subsidy to support manufacturing-related capital investment. Looking Ahead With this subsidy, JCR will invest not only in its own pipeline but also in new partnerships, strengthen as its role in biomanufacturing in regenerative and gene/cell therapies. About the Regenerative CDMO Subsidy Program This subsidy program supports the development of domestic CDMO (Contract Development and Manufacturing Organization) facilities and talent to ensure Japan's capacity to manufacture regenerative, cell, and gene therapies. For details, visit: (Japanese only). 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 ' J CR' ' U ltimate destination of organ' ' S afeguarding against off-target delivery' and ' T ransformative 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 partners, patients and employees. 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: 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.
Zawya
6 days ago
- Business
- Zawya
Africa Finance Corporation (AFC) Assigned A+ Rating with Stable Outlook by Japan Credit Rating Agency, Strengthening Access to Asian Capital Markets
Africa Finance Corporation (AFC) ( the continent's leading infrastructure solutions provider, has been assigned a long-term Issuer credit rating of A+ with a stable outlook by Japan Credit Rating Agency, Ltd (JCR). This rating will enable AFC to continue growing its footprint in Asian capital markets. 'The credit rating reflects AFC's leading role in infrastructure development in Africa, the strong support from its member states and shareholders, the benefits of Preferred Creditor Status (PCS), its conservative financial policy, and its strong capital base,' JCR stated in its report.' AFC employs diverse funding channels, including Eurobond issuance in international capital markets; borrowing from MDBs such as the African Development Bank, PROPARCO, DEG/FMO, KFW group, Export-Import Bank of China, Korea Development Bank, etc.; and financing from African, Chinese, European, Indian, Japanese and Middle Eastern private financial institutions.' The Japan Credit Rating Agency's A+ rating reflects AFC's continued demonstration of solid capital adequacy, maintaining a Capital Adequacy Ratio of 33.6% and improving its Cost-to-Income Ratio to 17.3% in FYE2024. In 2024, AFC delivered remarkable financial results, posting a 22.8% increase in revenue to surpass US$1 billion for the first time, as well as a 16.7% rise in total assets to US$14.41 billion. Liquidity buffers remain well above prudential thresholds, with a liquidity coverage ratio of 194% under normal conditions and 191% on a stressed basis, underscoring AFC's resilience. JCR's rating decision supports the Corporation's ability to secure competitive borrowing costs. This financial strength underpins AFC's ability to deliver transformational infrastructure projects across power, natural resources, transport and logistics, heavy industry, telecommunications, and technology—driving industrialisation and job creation across the continent. A notable example is the Lobito Corridor, where AFC serves as lead developer. Positioned to become one of Africa's most strategic economic arteries, the corridor will connect Angola's Port of Lobito on the Atlantic coast to Zambia through modernised rail infrastructure, enhancing regional trade, unlocking mineral value chains, and catalysing cross-border economic integration. Other key AFC transactions include a US$150 million investment in the Kamoa-Kakula Copper Complex—Africa's largest and one of the world's most sustainable copper producers and leading the commercial financing of a €381.5 million package for the engineering, procurement, and construction of 186 bridges and critical upgrades to Angola's road network, which will improve connectivity and boost regional trade. Leading Japanese financial institutions—Mizuho Bank, MUFG Bank, and Sumitomo Mitsui Banking Corporation have been critical partners supporting AFC on its journey of transforming Africa, participating in multiple funding transactions including bilateral, syndicated and Samurai facilities. This partnership has extended beyond AFC's own capital-raising efforts to broader support for African issuers. A notable example is the Arab Republic of Egypt's inaugural Samurai Bond, where AFC acted as re-guarantor and SMBC served as guarantor, facilitating a successful JPY 75 billion private placement. 'Amidst a challenging global macroeconomic backdrop, this endorsement by JCR affirms AFC's financial strength and credibility, enhancing our ability to mobilise competitively priced capital for transformative infrastructure projects across Africa,' said Banji Fehintola, Executive Board Member&Head, Financial Services at AFC. 'It reinforces our position as a reliable institutional partner for Japan and a key driver of Africa-Japan cooperation.' 'In the challenging business environment, with increasing geopolitical instability in some African countries, AFC's role in advancing infrastructure development in Africa as an MDB established by African countries is becoming more important, and support from member states and shareholders is expected to strengthen,' JCR analysts said, commending the Corporation. 'AFC conducts appropriate risk management in the challenging business environment in Africa, ensuring strong profitability and building a sound financial structure. AFC has established risk management policies for various risks associated with its operations, including credit risk, market risk, liquidity risk, operational risk, assets and liabilities management (ALM) risk, and environmental/social policy risks,' they further reported. Some of AFC's landmark funding initiatives include the successful issuance of its US$500 million perpetual hybrid bond, the closing of a US$400 million Shariah-compliant Commodity Murabaha, and leading Nigeria's inaugural domestic dollar bond issuance, which raised over US$900 million, with an oversubscription rate of 180%. These transactions underscore the Corporation's innovative approach to capital markets, diversifying funding sources and enhancing its ability to finance transformational infrastructure projects across Africa. For the full statement from Japan Credit Rating Agency, please click here ( Distributed by APO Group on behalf of Africa Finance Corporation (AFC). Media Enquiries: Yewande Thorpe Communications Africa Finance Corporation Mobile: +234 1 279 9654 Email: About AFC: AFC was established in 2007 to be the catalyst for pragmatic infrastructure and industrial investments across Africa. AFC's approach combines specialist industry expertise with a focus on financial and technical advisory, project structuring, project development, and risk capital to address Africa's infrastructure development needs and drive sustainable economic growth. Eighteen years on, AFC has developed a track record as the partner of choice in Africa for investing and delivering on instrumental, high-quality infrastructure assets that provide essential services in the core infrastructure sectors of power, natural resources, heavy industry, transport, and telecommunications. AFC has 45 member countries and has invested over US$15 billion in 36 African countries since its inception.
Yahoo
08-07-2025
- Business
- Yahoo
JCR Pharmaceuticals Enters License Agreement with Alexion for Proprietary JUST-AAV Capsids to be Used in the Development of Genomic Medicines
HYOGO, Japan, July 08, 2025--(BUSINESS WIRE)--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 Pharmaceuticals. "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-AAVJUST-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 TechnologyJCR 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., 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 the global website: Cautionary Statement Regarding Forward-Looking StatementsThis 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. View source version on Contacts Investors & Media:JCR Pharmaceuticals Co., Communicationsir-info@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
08-07-2025
- Business
- Business Wire
JCR Pharmaceuticals Enters License Agreement with Alexion for Proprietary JUST-AAV Capsids to be Used in the Development of Genomic Medicines
HYOGO, Japan--(BUSINESS WIRE)-- 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 Pharmaceuticals. '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 ' J CR' ' U ltimate destination of organ' ' S afeguarding against off-target delivery' and ' T ransformative 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. For more information, please visit the global website: 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.
Associated Press
02-07-2025
- Business
- Associated Press
JCR Pharmaceuticals Announces the Achievement of Enrollment in the JR-141 Global Phase III Clinical Trial
HYOGO, Japan--(BUSINESS WIRE)--Jul 2, 2025-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; JCR) announced that it achieved the enrollment of the target number of participants in the global Phase III clinical trial of JR-141 (INN: pabinafusp alfa), which is in development for the treatment of mucopolysaccharidosis type II (MPS II, also known as Hunter syndrome). The Phase III clinical trial is ongoing in the United States, Latin America, and Europe. ( JR-141-GS31 ) 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 people with Hunter syndrome. JR-141 was developed using J-Brain Cargo ®, JCR's proprietary blood-brain barrier (BBB)-penetrating technology, which is designed to deliver biotherapeutics across the BBB into the central nervous system (CNS) to address the neurological symptoms of Hunter syndrome. 'This achievement is a milestone in the JR-141 clinical development program, as the Hunter syndrome community needs a therapy that treats the cognitive symptoms of this devastating and life-threatening disease for which there are inadequate treatment options available,' said Shin Ashida, Chairman, President and CEO of JCR Pharmaceuticals. 'We are making good progress in this global Phase III clinical trial, and we look forward to sharing the clinical data as they are available. Thank you to all the participants who are part of this clinical trial.' In March 2021, the Ministry of Health, Labour and Welfare (MHLW) in Japan approved JR-141 (also known by the brand name IZCARGO ® ) for a lysosomal storage disorder. JR-141 is the first-ever approved ERT in the world that penetrates the BBB. About JR-141 JR-141 (INN: pabinafusp alfa) 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 JR-141 effective against the central nervous system (CNS) symptoms of Hunter syndrome. In non-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 syndrome. 1,2 In several clinical trials of pabinafusp alfa, JCR obtained evidence of reducing heparan sulfate concentrations in the cerebrospinal fluid, a biomarker for assessing effectiveness against CNS symptoms; these results were consistent with those obtained in pre-clinical studies. 3 Clinical studies have also demonstrated the positive effects of pabinafusp alfa on CNS symptoms. 4,5,6 About Mucopolysaccharidosis Type II (Hunter Syndrome) Mucopolysaccharidosis type II (MPS II, or Hunter syndrome) 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 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: 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. 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. View source version on CONTACT: Investors & Media: JCR Pharmaceuticals Co., Ltd. Corporate Communications [email protected] KEYWORD: UNITED STATES JAPAN NORTH AMERICA ASIA PACIFIC INDUSTRY KEYWORD: RESEARCH NEUROLOGY CLINICAL TRIALS BIOTECHNOLOGY HEALTH PHARMACEUTICAL GENERAL HEALTH OTHER SCIENCE SCIENCE SOURCE: JCR Pharmaceuticals Co., Ltd. Copyright Business Wire 2025. PUB: 07/02/2025 11:12 AM/DISC: 07/02/2025 11:12 AM
