Corporate Events

JCR Pharmaceuticals Co., Ltd. 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. 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. 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. 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 genetic therapies. 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. 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®?.

JCR Pharmaceuticals Co., Ltd. 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 (AbO)-selective antibodies, which target toxic soluble AbOs, 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 AbO-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.

JCR Pharmaceuticals Co., Ltd., Q1 2026 Earnings Call, Jul 30, 2025

JCR Pharmaceuticals Co., Ltd. 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.

JCR Pharmaceuticals Co., Ltd. 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. 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.

JCR Pharmaceuticals Co., Ltd., Board Meeting, Jun 25, 2025. Agenda: To resolve to carry out a Disposition of Treasury Stock.

JCR Pharmaceuticals Co., Ltd. announced that, effective April 1, 2026, the company will implement a new leadership structure. The changes were approved at the Board of Directors meeting held earlier May 13, 2025. Under the new structure, Current Chairman, President and CEO, Shin Ashida, will transition to the role of Founder and Director.

JCR Pharmaceuticals Co., Ltd. announced that, effective April 1, 2026, the company will implement a new leadership structure. The changes were approved at the Board of Directors meeting held earlier May 13, 2025. Under the new structure, Toru Ashida will assume the role of Chairman, and Hiroyuki Sonoda will become President. Current Chairman, President and CEO, Shin Ashida, will transition to the role of Founder and Director. Changes in Representative Directors (including Chairman and President): New Assignments of Representative Directors: Toru Ashida- From Director, Senior Managing Executive Officer, Sales, Executive Director, Sales Division, To Representative Director Chairman; Hiroyuki Sonoda- From Director, Senior Managing Executive Officer, Research, Executive Director, Research Division, To Representative Director President; Shin Ashida: From Representative Director Chairman, President and CEO, To Director, Founder. Reason for Changes: Since founding JCR in September 1975, Shin Ashida has played a pivotal role in shaping the company's growth, grounded in a deep and enduring commitment to innovation in biopharmaceutical research and manufacturing. As the company marks its 50th anniversary this September, this leadership transition reflects a generational shift aimed at further strengthening the management structure and driving long-term corporate value. Details regarding other executive appointments will be announced once internal discussions have been concluded. Effective Date: April 1, 2026. Brief Personal History of New Chairman: Name: Toru Ashida Birthdate: October 31, 1968 (56 years old) Birthplace: Ashiya City, Hyogo Prefecture, Japan Education: March 1992 Graduated from Keio University, Faculty of Business and Commerce. Career: April 1992 Joined Nippon Life Insurance Company, April 2002 Appointed Representative Director and President at the establishment of JBS Co., Ltd. (clinical trial site support organization), January 2014 Joined the Company, July 2014 Appointed Corporate Officer of the Company Executive Director of Corporate Business Support Division and Director of Corporate Strategy Department of the Company, April 2016 Head of Office of the President of the Company, June 2018 Appointed Senior Executive Director of the Company Head of Quality Assurance Division, Corporate Planning Division, and Medical Affairs Department of the Company, June 2019 In charge of Corporate Strategy Head of Quality Assurance Division, Corporate Planning Division, Administration Division, and Medical Affairs Department of the Company, April 2020 Executive Director of Sales Division of the Company (to present), June 2020 Appointed Vice President of the Company and in charge of Sales Division, June 2021 Appointed Senior Vice President of the Company and in charge of Sales and Administration, April 2023 In charge of Sales (to present), June 2024 Appointed Director and Senior Managing Executive Officer of the Company (to present). Brief Personal History of New President Name: Hiroyuki Sonoda, Ph.D. Birthdate: June 15, 1978 (46 years old) Birthplace: Maizuru City, Kyoto Prefecture, Japan Education: September 2010 Graduated from Department of Chemical Science and Engineering Faculty of Engineering Graduate School of Engineering Kobe University (Doctor of Engineering). Career: April 2003 Joined the Company, April 2016 Director of Corporate Planning Division (In charge of Research) of the Company, October 2017 Leader of Frontier Research Unit and Director of Corporate Planning Division (In charge of Research) of the Company, April 2018 Executive Director of Research Planning Division of the Company, June 2018 Appointed Corporate Officer of the Company, June 2020 Appointed Senior Executive Director of the Company and in charge of Research and Development Division Executive Director of Research Division, and Director of Drug Discovery Research Institute, June 2021 Appointed Vice President of the Company and in charge of Research and Corporate Strategy Executive Director of Research Division (to present), October 2022 Representative Director and President of AlliedCel Corporation (Joint venture with Sysmex Corporation) (to present), April 2023 In charge of Research (to present), June 2024 Appointed Director and Senior Managing Executive Officer of the Company (to present).

JCR Pharmaceuticals Co., Ltd. announced that they will report Q1, 2026 results on Jul 30, 2025

JCR Pharmaceuticals Co., Ltd. provided consolidated earnings guidance for the fiscal year ending March 31, 2026. For the full year, the company expected net sales of JPY 37,800 million, operating profit of JPY 2,600 million, profit attributable to owners of parent of JPY 3,000 million and basic earnings per share of JPY 24.22.

JCR Pharmaceuticals Co., Ltd. announced that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy research programs at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, being held May 13-17, 2025, in New Orleans, LA. In an oral presentation, the JCR researcher reported that the Company's proprietary J-Brain Cargo®? (JBC) technology enables the efficient delivery of an adeno-associated virus ("AAV") gene therapy across the blood-brain barrier (BBB) and into the central nervous system (CNS) in mice, monkeys, and several animal models of CNS diseases. The company has successfully developed JUST-AAV, a novel AAV vector platform technology designed to enhance targeted delivery and reduce liver tropism, thereby improving safety and efficacy of AAV-based gene delivery technologies to the CNS. JUST-AAV encompasses a range of vector types optimized for various target tissues, including liver-sparing, muscle-targeting, and brain-targeting variants. This enhanced safety profile greatly increases the potential for clinical translation. This research is a crucial step in ongoing commitment to developing innovative solutions for unmet medical needs. JCR showcased the following presentation: Incorporation of transferrin receptor binder and surface mutations into AAV enables efficient brain delivery and reduced liver tropism. Presenter: Yuhei Ashida (JCR Pharmaceuticals) Researchers successfully created a brain-targeting AAV vector (brain-targeting JUST-AAV) by incorporating a miniaturized antibody that binds to the transferrin receptor, into the AAV capsid. Application of JUST-AAV to a mouse model of neuronal ceroid lipofuscinosis resulted in the disappearance of symptoms such as seizures and prolonged lifespan to an extent of a functional cure. These results suggest that the newly developed JUST-AAV technology offers the potential for safer and more efficient gene therapy than conventional gene delivery vectors.

JCR Pharmaceuticals Co., Ltd. expected to report Q1 2026 results on July 25, 2025. This event was calculated by S&P Global (Created on May 14, 2025).

JCR Pharmaceuticals Co., Ltd., Annual General Meeting, Jun 25, 2025.

JCR Pharmaceuticals Co., Ltd., Board Meeting, May 13, 2025. Agenda: To discuss a year-end dividend.

JCR Pharmaceuticals Co., Ltd. Presents at American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, May-14-2025 01:30 PM. Venue: New Orleans Ernest N. Morial Convention Center, New Orleans, Louisiana, United States. Speakers: Yuhei Ashida.

JCR Pharmaceuticals Co., Ltd., Board Meeting, Apr 25, 2025. Agenda: To consider Election of Candidate for New Audit & Supervisory Board Members.

JCR Pharmaceuticals Co., Ltd. Presents at World Orphan Drug Congress USA 2025, Apr-24-2025 12:50 PM. Venue: Boston Convention & Exhibition Center, Boston, Massachusetts, United States. Speakers: Anne Bechet, Sr. Exe. Offi., Exe. Dir. Dev. Div, GM of JCR EU. B.V, JCR USA Inc, Dir. of JCR Luxemburg S.A.

JCR Pharmaceuticals Co., Ltd., Q4 2025 Earnings Call, May 13, 2025

JCR Pharmaceuticals Co., Ltd. revised consolidated earnings guidance for the fiscal year ended March 31, 2025. For the year, the company expects net sales to be JPY 33,000 million against previous guidance of JPY 39,000 million. Operating loss to be JPY 6,000 million against previous guidance of operating profit of JPY 1,400 million. Loss attributable to owners of parent of JPY 4,300 million against previous guidance of profit attributable to owners of parent of JPY 2,200 million. Loss per share to be JPY 34.73 against previous guidance of earnings per share of JPY 17.77. Reason for the revision: Net sales have been adjusted downward by JPY 6 billion to JPY 33 billion, as a licensing agreement that had been expected to close during the current fiscal year is now unlikely to be finalized before year-end. Cost of sales and R&D expenses have increased following a closer review of inventories, including raw materials and investigational drugs, with write-offs for unused items to be recorded in the current fiscal year. These adjustments are expected to impact cost of sales by JPY 200 million and R&D expenses by JPY 1.1 billion. In addition, JCR had planned to record approximately JPY 1.5 billion in extraordinary income tied to a government subsidy related to the construction of its API facility at the Kobe Science Park Center (completed in November 2022). However, the official approval of the subsidy is now expected to occur in the next fiscal year. As a result, the anticipated gain will not be reflected in this term's results. As a result, operating profit has been revised downward by JPY 7.4 billion to operating loss of JPY 6 billion, ordinary profit by JPY 7.45 billion to ordinary loss of JPY 6.7 billion, and profit attributable to owners of parent by JPY 6.5 billion to loss attributable to owners of parent of JPY 4.3 billion.

JCR Pharmaceuticals Co., Ltd. announced that they will report fiscal year 2025 results at 3:00 PM, Tokyo Standard Time on May 13, 2025

On February 27, 2025, Mycenax Biotech Inc., cancelled the transaction. The termination has been approved by the shareholders of the company.

JCR Pharmaceuticals Co., Ltd. announced that its Board of Directors resolved (Changes: As of April 1, 2025)) at a meeting held on February 27, 2025 that Kinki Sales Department" will be newly established to facilitate prompt decision-making in the formulation of area strategies and the strengthening of activities. A part of the groups within the Sales Promotion Department will be promoted, and a new "Distribution Measures Department" will be established.

JCR Pharmaceuticals Co., Ltd. announced reorganization of Sales Division (As of April 1, 2025). The areas in charge of "Tokai Keiji Hokuriku Sales Department" and "West Japan Sales Department" will be reorganized, and "Tokai Keiji Hokuriku Sales Department" will be renamed "Central Japan Sales Department.

JCR Pharmaceuticals Co., Ltd. announced that its Board of Directors resolved at a meeting held February 27, 2025 to change in duties of corporate officers, organizational and personnel changes as follows. Change in Duties of Corporate Officers (As of April 1, 2025): Makoto Ashida from Current Area of Supervision: Senior Director of Production Division to New Area of Supervision: Senior Director of Production Division and Director of Production Management Unit. Organizational Changes: Personnel Changes (As of April 1, 2025): Shuichi Yasumoto: New title: Director, Central Japan Sales Development, Sales Division; Current Title: Director, West Japan Sales Development, Sales Division. Hiroyuki Ozaki: New title: Director, Kinki Sales Development, Sales Division; Current Title: Director, Tokai Keiji Hokuriku Sales Development, Sales Division. Hironori Tokura: New title: Director, West Japan Sales Development, Sales Division; Current Title: Manager, North Kanto Koshinetsu area, East Japan Sales Development, Sales Division. Masamitsu Kubo: New title: Director, Distribution Measures Development, Sales Division; Current Title: Deputy Director, Sales Promotion Development, Sales Division.

JCR Pharmaceuticals Co., Ltd., Board Meeting, Feb 27, 2025. Agenda: To consider and approve change in duties of corporate officers, organizational and personnel changes.

JCR Pharmaceuticals Co., Ltd. announced that the Company has started construction of a new plant for drug product filling and finishing in the Kobe Science Park Center. At the Kobe Science Park Center, construction of an Active Pharmaceutical Ingredient (API) plant was completed in November 2022 and the plant is now in operation. JCR, in cooperation with the adjacent API plant, will construct this new plant with the aid of subsidies from "Project for Establishing Biopharmaceutical Manufacturing Sites to Strengthen Vaccine Production" selected by the Ministry of Economy, Trade and Industry (METI). The objective is establishment of an integrated plant system for contract manufacturing of vaccines in case of a pandemic emergency.

SanBio Co., Ltd. announced on July 31, 2025 that it has entered into a contract manufacturing agreement with JCR Pharmaceuticals Co., Ltd. for the trial production of the human (allogeneic) cell therapy product AKUUGO Suspension for Intracranial Implantation ("AKUUGO") to assess the feasibility of commercial manufacturing. By entering into this agreement, SanBio and JCR Pharma aim to explore future contract manufacturing opportunities to ensure the stable production of AKUUGO as a commercial product and to enhance the stability of and diversify its supply framework in anticipation of indication expansion for conditions such as ischemic stroke and market entry into the US.

JCR Pharmaceuticals Co., Ltd. announced the presentation of two datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs) at the 21 Annual WORLDSymposium 2025. JCR is presenting new data from a pair of its programs that apply its J-Brain Cargo platform, a proprietary technology developed by JCR, to deliver medicines across the blood-brain barrier (BBB) through poster presentations this week. The first presentation highlights non-clinical data from the JR-479 clinical development program. JR-479 is an investigational BBB-penetrating ß-Hexosaminidase A (rDNA origin) enzyme replacement therapy (ERT) that JCR is developing for the treatment of people with GM2 gangliosidoses. The second presentation focuses on the long-term safety and efficacy and behavioral effects of JR-141 (pabinafusp alfa) for mucopolysaccharidosis type II (MPS II, or Hunter syndrome). JR-141 is a recombinant iduronate-2-sulfatase (I2S) ERT that was approved in March 2021 by the Ministry of Health, Labour and Welfare (MHLW) in Japan, where it is marketed as IZCARGO for the treatment of people with MPS II. JR-479 GM2 Gangliosidosis Dataset: This late-breaking poster presentation provides non-clinical data from the JR-479 clinical development program investigating GM2 gangliosidoses: Presentation Title: Treatment with a transferrin receptor-targeted ß-hexosaminidase A prolongs life span of GM2 gangliosidosis mice (Poster Number LB-33). Presenter: Kazuki Miyauchi, Scientist (JCR Pharmaceuticals)This poster presentation reported non-clinical data on JR-479, a fusion protein of anti-human transferrin receptor 1 (TfR) antibody and human ß-hexosaminidase A (Hex A) designed to cross the BBB, taking advantage of the mechanism of receptor-mediated transcytosis of transferrin. After a single intravenous administration, JR-479 was distributed in the brain of mice, confirming that it crosses the BBB to reach the central nervous system (CNS). In addition, the pharmacodynamics in an animal model of GM2 gangliosidosis was evaluated. The results included reduced substrate concentrations in both peripheral, and CNS tissues and histopathological findings such as neuronal vacuolation in the brain of GM2 gangliosidosis mice were suppressed. Furthermore, JR-479 dramatically prolonged the lifespan of GM2 gangliosidosis mice. The disease control mice died around 120 postnatal days, whereas more than 90% of JR-479-treated animals were still alive at 270 days of age. These results confirm the non-clinical proof-of-concept of CNS delivery and efficacy of the transferrin receptor-targeted Hex A. JR-479 may be a promising drug candidate for the treatment of people with GM2 gangliosidoses. JR-141 MPS II Dataset: The following poster presentation provides additional evidence and context for the use of JR-141 in the treatment of MPS II: Presentation Title: Long-term impact of pabinafusp alfa on disease burden in hunter syndrome: a 4-year follow-up of patient-reported outcomes (Poster Number 222). Presenter: Ana Maria Martins, M.D., Ph.D. (Federal University of São Paulo)In this retrospective case study, they investigated patient-reported outcomes to assess the impact of pabinafusp alfa on people with MPS II for four years. Of the nine participating patients at the beginning of the study, two passed away due to progression of the underlying disease between the 104 and 208 week. The remaining seven participants received pabinafusp alfa intravenously for at least 208 weeks. The results from the study include increased muscle strength in 71% of the participants, and 42% also showed improved general motor skills (gross and fine motor skills); in addition, 57% of the participants showed cognitive and gait improvement, with 28% also demonstrating improved speech; half of the participants showed behavioral improvement, and all of the participants showed improvements in facial expression, general appearance, and breathing parameters. The caregivers also reported increased ability to express emotions through smiling, establishing eye contact, and giving hugs. During the four-year follow-up, the participants not only maintained the previously observed improvements, but also continued to show new progress, particularly in neurodevelopmental milestones and fine motor skills. Future studies (including a global Phase III, which is already in place) should be conducted to clarify the mechanisms underlying these beneficial effects.

JCR Pharmaceuticals Co., Ltd. revised consolidated earnings guidance for the fiscal year ending March 31, 2025. For the period, the company expected net sales of JPY 39,000 million, operating profit of JPY 1,400 million, profit attributable to owners of parent of JPY 2,200 million and earnings per share of JPY 17.77 compared to previous guidance of net sales of JPY 41,300 million, operating profit of JPY 5,400 million, profit attributable to owners of parent of JPY 3,700 million and earnings per share of JPY 29.65. Reason for the revision: Net sales have been revised to JPY 39,000 million, down JPY 2,300 million from the previous forecast. While product sales remain largely on track, income from contractual payment was revised downward because an overseas licensing agreement will not be concluded for JR-171 within this fiscal year. Despite improvements in cost efficiency and a favorable product mix, higher disposal costs for manufacturing materials are expected. Accordingly, the cost of sales has increased by JPY 700 million from the previous forecast. Expenses were increased by JPY 400 million allocated to selling, general and administrative expenses, and JPY 600 million to R&D expenses, based on results until the third quarter, etc. As a result, operating profit has been revised down by JPY 4,000 million to JPY 1,400 million. Ordinary profit was revised down by JPY 3,850 million to JPY 750 million, and net profit attributable to owners of the parent was reduced by JPY 1,500 million to JPY 2,200 million.

JCR Pharmaceuticals Co., Ltd. Presents at 21st Annual WORLDSymposium 2025, Feb-03-2025 through Feb-07-2025. Venue: San Diego, California, United States. Presentation Date & Speakers: Feb-04-2025, Kazuki Miyauchi, Scientist. Feb-06-2025, Ana Maria Martins.

JCR Pharmaceuticals Co., Ltd. Presents at 6th CNS Drug Delivery Summit, Dec-03-2024 through Dec-05-2024. Venue: Westin Seaport, 425 Summer St, Boston, Massachusetts, United States. Presentation Date & Speakers: Dec-03-2024, Mathias Schmidt, Executive Advisor to the President, Chairman & CEO. Dec-04-2024, Mathias Schmidt, Executive Advisor to the President, Chairman & CEO.

JCR Pharmaceuticals Co., Ltd. expected to report Fiscal Year 2025 results on May 13, 2025. This event was calculated by S&P Global (Created on April 8, 2025).

Hanson Wade Limited, 6th CNS Drug Delivery Summit, Dec 03, 2024 through Dec 05, 2024. Venue: Westin Seaport, 425 Summer St, Boston, Massachusetts, United States.

JCR Pharmaceuticals Co., Ltd. and Modalis Therapeutics Corporation announced that they have validated the initial proof of concept in a joint research program for the development of a novel gene therapy for a central nervous system disease. Due to the success of the partnership thus far, Modalis and JCR have agreed to proceed to the next phase of their research by entering into a new joint research agreement. The purpose of this joint agreement is to conduct pre-clinical studies for the development of a new gene therapy for the undisclosed CNS disease by applying J-Brain Cargo, JCR’s proprietary technology that is able to cross the blood-brain barrier, and a gene therapy payload based on CRISPR-GNDM (Guide Nucleotide-Directed Modulation), Modalis’ proprietary epigenome modulation technology which does not cleave or alter DNA sequences. The two companies began a joint research collaboration in December of 2023 to evaluate the drug delivery technology of the gene therapy to the CNS. As a result of this research, the initial proof of concept has been validated. The next phase of the agreement is to jointly develop a novel and innovative gene therapy to provide patients with improved efficacy, safety, and less burden via intravenous injection in a minimally invasive and efficient manner.

JCR Pharmaceuticals Co., Ltd., Q3 2025 Earnings Call, Jan 31, 2025

JCR Pharmaceuticals Co., Ltd. announced that the first patient has been dosed in the Phase III clinical trial of JR-142 (INN: redalsomatropin alfa) in Japan, marking a significant milestone in the development of this innovative treatment. JR-142 is a long-acting growth hormone therapy and is being studied in patients with pediatric growth hormone deficiency. The trial involves 54 pediatric patients and will compare the efficacy of JR-142 to JCR's existing product, Growject®. Patients will receive treatment over a 52-week period, with growth outcomes serving as the primary measure of success. Detailed trial information is available through Japan Register of Clinical Trials (jRCT2031240282). About Growject® Growject® is an injectable preparation containing recombinant human growth hormone, first launched in Japan by JCR in 1995. The dosage is administered subcutaneously 6­7 times per week, adjusted according to the indicated condition.

JCR Pharmaceuticals Co., Ltd. announced that they will report Q3, 2025 results on Jan 31, 2025

MediPal Holdings Corporation and JCR Pharmaceuticals Co., Ltd. announced the initiation of the Phase I/II clinical trial of JR-446 in Japan following the dosing of the first individual from the trial. JR-446 is a proprietary blood-brain barrier (BBB)-penetrating -N-acetylglucosaminidase in development for the treatment of mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B or MPS IIIB). MPS IIIB affects an estimated 500 to 1,000 individuals worldwide1, causing severe central nervous system (CNS) symptoms. Despite the dire need, there are currently no approved treatments available for this condition. JR-446, developed using JCR's proprietary J-Brain Cargo® technology, has shown promising non-clinical results in addressing the symptoms of this challenging disorder. This Phase I/II clinical trial is an open-label, single-arm, multi-center study that includes individuals under 18 years of age who are diagnosed with MPS IIIB. The study aims to evaluate the safety, tolerability, and exploratory efficacy of JR-446, while also assessing the optimal dosage through the administration of multiple doses. For more details on the trial, visit the Clinical Research Submission and Disclosure System (JR-446-101, jRCT2071240043). In September 2023, MEDIPAL and JCR entered into a licensing agreement in which MEDIPAL will commercialize JR-446 outside of Japan. In addition, MEDIPAL will support JCR in the clinical development of JR-446 in Japan, including the distribution of investigational drugs, disease awareness, and clinical trial advancement2. This collaboration highlights the commitment of MEDIPAL and JCR to pioneer treatments for ultra- rare diseases. By advancing therapies like JR-446, the company aim to bring hope to patients and their families, while enhancing corporate value and contributing to a society where everyone can live with physical and mental well-being. 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. 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.

American Society of Gene & Cell Therapy, American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, May 13, 2025 through May 17, 2025. Venue: New Orleans Ernest N. Morial Convention Center, New Orleans, Louisiana, United States.

R&D Meeting

The company closed its plan on November 1, 2024.

From October 30, 2024 to November 1, 2024, the company has repurchased 3,672,000 shares, representing 2.93% for ¥2,530.01 million. With this, the company has completed the repurchase of 3,672,000 shares, representing 2.93% for ¥2,530.01 million under the buyback announced on October 30, 2024.

JCR Pharmaceuticals Co., Ltd. announced the initiation of the first patient dosing in Japan in the Phase I clinical trial of JR-441, an investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo Syndrome Type A). JR-441 is a proprietary recombinant heparan N-sulfatase capable of crossing the blood-brain barrier (BBB). MPS IIIA is a rare genetic disorder characterized by severe central nervous system (CNS) symptoms, for which there is currently no approved treatment. Preclinical studies have demonstrated the potential of JR-441 to address CNS-related symptoms associated with MPS IIIA. This open-label, multicenter, single-arm study aims to assess the safety profile, biological effect, and pharmacokinetic profile of JR-441 in patients aged 1 to under 18 years with MPS IIIA. JR-441 received Orphan Drug Designation from both the European Commission (EC) and the U.S. Food and Drug Administration (FDA), with a Phase I/II trial underway in Germany, which began in 2023 (JR-441-101, NCT06095388).

JCR Pharmaceuticals Co., Ltd., ¥ 10.0, Cash Dividend, Mar-28-2025

The Board of Directors of JCR Pharmaceuticals Co., Ltd. has authorized a buyback plan on October 30, 2024.

JCR Pharmaceuticals Co., Ltd. expected to report Q3 2025 results on January 24, 2025. This event was calculated by S&P Global (Created on October 30, 2024).

JCR Pharmaceuticals Co., Ltd., Board Meeting, Oct 30, 2024. Agenda: To consider and approve to distribute retained earnings with the record date being September 30, 2024.

JCR Pharmaceuticals Co., Ltd. announced that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy research programs in a poster session at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress, being held October 22-25, 2024, in Rome, Italy. JCR leverages its proprietary J-Brain Cargo®? (JBC) technology for the delivery of adeno-associated virus (AAV) gene therapy to the body and the brain to potentially address the central nervous system (CNS) symptoms by crossing the blood-brain barrier (BBB).

JCR Pharmaceuticals Co., Ltd., Q2 2025 Earnings Call, Oct 30, 2024

JCR Pharmaceuticals Co., Ltd. announced that the completion of the regulatory review by the Pharmaceuticals and Medical Devices Agency (PMDA) for the clinical trial notification for the Phase I study of JR-441 in individuals with mucopolysaccharidosis type IIIA (MPS IIIA; Sanfilippo syndrome type A). JR-441 is a recombinant form of heparan N-sulfatase that is developed using JCR's proprietary J-Brain Cargo blood-brain barrier (BBB)-penetrating technology. JR-441 addresses a significant unmet medical need by crossing the BBB and targeting the neurological symptoms of MPS IIIA within the central nervous system. The European Commission (EC) and the U.S. Food and Drug Administration (FDA) granted JR- 441 orphan drug designation (ODD) for the treatment of MPS IIIA. JR-441 is currently being studied in an ongoing Phase I/II clinical trial in Germany (JR-441-101, NCT06095388), apart from this Japanese Phase I study.

JCR Pharmaceuticals Co., Ltd. announced that they will report Q2, 2025 results on Oct 30, 2024