JCR Pharmaceuticals Co., Ltd.

Expect to report topline results for ALTITUDE-AD, a Phase 2 study to investigate sabirnetug (ACU193) for the treatment of early Alzheimer’s disease, in late 2026 Two candidates nominated for development in Acumen’s Enhanced Brain Delivery™ (EBD™) program; lead clinical candidate IND filing for the EBD program targeted for mid-2027 Cash, cash equivalents and marketable securities of $110.2 million as of June 30, 2026, expected to support current clinical and operational activities into early 2027 Company to host conference call and webcast today at 8:00 a.m. ET NEWTON, Mass., Aug. 12, 2026 (GLOBE NEWSWIRE) -- Acumen Pharmaceuticals, Inc. (NASDAQ: ABOS) (“Acumen” or the “Company”), a clinical-stage biopharmaceutical company developing novel therapeutics that target toxic soluble amyloid beta oligomers (AβOs) for the treatment of Alzheimer’s disease (AD), today reported financial results for the second quarter of 2026 and provided a business update. “This quarter we have been laser-focused on the readout preparations for our ALTITUDE-AD Phase 2 study investigating the efficacy, safety and tolerability of sabirnetug for the treatment of early AD, with topline results expected later this year. We are excited about the potential for sabirnetug to emerge as a potential treatment of choice with a differentiated benefit-to-risk pro its unique product attributes as an anti-AβO, IgG2 monoclonal,” said Daniel O’Connell, Chief Executive Officer of Acumen. “In the second quarter, we also announced the nomination of two Enhanced Brain Delivery™ candidates for the treatment of Alzheimer’s Disease, representing the only program combining a validated blood-brain barrier-penetrating technology with an AβO-selective therapeutic antibody. We continue to advance IND-enabling activities toward a mid-2027 IND submission for a lead candidate, and are excited about the optionality this innovative product approach adds to our pipeline and the potential it holds to deliver a next-generation treatment for AD.” Recent Highlights In June 2026, the Company announced the nomination of two EBD™ development candidates for the treatment of AD. ACU301 and ACU401 were nominated in the EBD program as part of the exercise of Acumen’s option under its license agreement with JCR Pharmaceuticals. Acumen’s EBD candidates are designed to achieve higher brain penetration with potential for improved safety compared to native antibodies and delivered in a stable, low volume subcutaneous administration format. In July 2026, the Company announced EBD program data and early AD insights at the Alzheimer’s Association International Conference (AAIC ® ) 2026. EBD bispecific antibodies targeting both AβOs and hTfR (human transferrin receptor) showed improved brain penetration compared to the native anti-AβO antibodies, which may enable administration with a low-volume delivery device and reduce the delivered dose required for efficacy. EBD candidate ACU401 achieves up to 40-fold greater frontal cortex exposure in non-human primates and significant increase in exposures in deep brain regions. No hematological safety signals, including no effects on reticulocytes, were observed in non-human primates, demonstrating low potential for anemia. ALTITUDE-AD patient experience data highlight unmet need in early Alzheimer's disease. To access the material, please visit the Publications section of the Acumen website . Anticipated Milestones ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); The Company expects topline results from ALTITUDE-AD in late 2026. ALTITUDE-AD is a Phase 2 study investigating sabirnetug in 542 participants (N=180 per cohort) for the treatment of early Alzheimer’s disease. ALTITUDE-AD includes two active doses (35 mg/kg, 50 mg/kg, IV Q4W) vs. placebo over an 18-month double-blinded period. Topline results are expected to include iADRS (Integrated Alzheimer’s Disease Rating Scale), our primary clinical efficacy endpoint, as well as key secondary endpoints, such as CDR-SB (Clinical Dementia Rating – Sum of the Boxes), certain safety measures such as adverse event rates, including ARIA rates, and key biomarkers. The Company is targeting the submission of an IND filing with respect to a lead clinical candidate in its EBD program in mid-2027. Second Quarter 2026 Financial Results Cash Balance. As of June 30, 2026, cash, cash equivalents and marketable securities totaled $110.2 million compared to cash, cash equivalents and marketable securities of $128.4 million as of March 31, 2026. Cash is expected to support current clinical and operational activities into early 2027. Research and Development (R&D) Expenses. R&D expenses were $27.8 million for the quarter ended June 30, 2026, compared to $37.1 million for the quarter ended June 30, 2025. The decrease was primarily due to reductions in manufacturing and materials costs, and CRO costs, which are both associated with our ALTITUDE-AD clinical trial. General and Administrative (G&A) Expenses. G&A expenses were $4.7 million for the quarter ended June 30, 2026, compared to $4.6 million for the quarter ended June 30, 2025. Loss from Operations. Loss from operations was $32.6 million for the quarter ended June 30, 2026, compared to $41.8 million for the quarter ended June 30, 2025. This decrease was due to the decreased R&D expenses over the prior year period. Net Loss. Net loss was $32.7 million for the quarter ended June 30, 2026, compared to $41.0 million for the quarter ended June 30, 2025. Conference Call Details Acumen will host a conference call and live audio webcast today, August 12, 2026, at 8:00 a.m. ET. To participate in the live conference call, please register using this link . After registration, you will be informed of the dial-in numbers including PIN. Please register at least one day in advance. The webcast audio will be available via this link . An archived version of the webcast will be available for at least 30 days in the Investors section of the Company's website at . About Sabirnetug (ACU193) Sabirnetug (ACU193) is a humanized monoclonal antibody (mAb) discovered and developed based on its selectivity for soluble amyloid beta oligomers (AβOs), which are a highly toxic and pathogenic form of Aβ, relative to Aβ monomers and amyloid plaques. Soluble AβOs have been observed to be potent neurotoxins that bind to neurons, inhibit synaptic function and induce neurodegeneration. By selectively targeting toxic soluble AβOs, sabirnetug aims to address the hypothesis that soluble AβOs are an early and persistent underlying cause of the neurodegenerative process in Alzheimer’s disease (AD). Sabirnetug has been granted Fast Track designation for the treatment of early AD by the U.S. Food and Drug Administration and is currently being evaluated in a Phase 2 study in patients with early AD. About ALTITUDE-AD (Phase 2) Initiated in 2024, ALTITUDE-AD is a Phase 2, multi-center, randomized, double-blind, placebo-controlled clinical trial designed to evaluate the efficacy and safety of sabirnetug (ACU193) infusions administered once every four weeks in slowing cognitive and functional decline as compared to placebo in participants with early Alzheimer's disease. The study has enrolled 542 individuals with early Alzheimer’s disease (mild cognitive impairment or mild dementia due to AD) at multiple investigative sites located in the United States, Canada, the European Union and the United Kingdom. Topline results are expected in late 2026. More information can be found on NCT identifier NCT06335173. About Acumen Pharmaceuticals, Inc. Acumen Pharmaceuticals is a clinical-stage biopharmaceutical company developing novel therapeutics that target toxic soluble amyloid beta oligomers (AβOs) for the treatment of Alzheimer’s disease (AD). Acumen’s scientific founders pioneered research on AβOs, which a growing body of evidence indicates are early and persistent triggers of Alzheimer’s disease pathology. Acumen is currently focused on advancing its lead investigational product candidate, sabirnetug (ACU193), a humanized monoclonal antibody that selectively targets toxic soluble AβOs, in its ongoing Phase 2 clinical trial ALTITUDE-AD (NCT06335173) in early symptomatic Alzheimer’s disease patients, following positive results in its Phase 1 trial INTERCEPT-AD. Acumen is also investigating a subcutaneous formulation of sabirnetug using Halozyme’s proprietary ENHANZE ® drug delivery technology. Acumen is also collaborating with JCR Pharmaceuticals to develop an Enhanced Brain Delivery™ (EBD™) therapy for Alzheimer’s disease utilizing a transferrin-receptor-targeting blood-brain barrier-penetrating technology. The company is headquartered in Newton, Mass. For more information, visit . Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Any statement describing Acumen’s goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Words such as “believes,” “expects,” “anticipates,” “could,” “should,” “would,” “seeks,” “aims,” “plans,” “potential,” “will,” “milestone” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements include statements concerning Acumen’s business, and Acumen’s ability to achieve its strategic and financial goals, including its projected use of cash, cash equivalents and marketable securities and the expected sufficiency of its cash resources into early 2027, the therapeutic potential of Acumen’s product candidate, sabirnetug (ACU193), including against other antibodies, the timing of anticipated topline results of ALTITUDE-AD, Acumen’s plans to develop a candidate to treat Alzheimer's Disease utilizing EBD technology, including its expectations with respect to timing for the submission of an IND, the potential of its EBD candidates and the potential for developing a best-in-class therapeutic candidate for people living with Alzheimer’s Disease. These statements are based upon the current beliefs and expectations of Acumen management, and are subject to certain factors, risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing safe and effective human therapeutics. Such risks may be amplified by the impacts of geopolitical events and macroeconomic conditions, such as rising inflation and interest rates, supply disruptions and uncertainty of credit and financial markets. These and other risks concerning Acumen’s programs are described in additional detail in Acumen’s filings with the Securities and Exchange Commission (“SEC”), including in Acumen’s most recent Annual Report on Form 10-K, and in subsequent filings with the SEC. Copies of these and other documents are available from Acumen. Additional information will be made available in other filings that Acumen makes from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and Acumen expressly disclaims any obligation to update or revise any forward-looking statement, except as otherwise required by law, whether, as a result of new information, future events or otherwise. CONTACTS: Investors: Alex Braun abraun@acumenpharm.com Media: ICR Healthcare AcumenPR@icrhealthcare.com Acumen Pharmaceuticals, Inc. Condensed Balance Sheets (in thousands, except share and per share data) June 30, December 31, 2026 2025 (unaudited) ASSETS Current assets Cash and cash equivalents $ 59,443 $ 53,989 Marketable securities, short-term 50,754 62,876 Prepaid expenses and other current assets 5,316 5,387 Total current assets 115,513 122,252 Restricted cash 231 231 Other assets, long-term 263 350 Total assets $ 116,007 $ 122,833 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities Accounts payable $ 13,379 $ 554 Accrued clinical trial expenses 6,263 10,616 Accrued expenses and other current liabilities 7,533 10,072 Debt, short-term 19,673 8,765 Total current liabilities 46,848 30,007 Debt, long-term 11,896 22,396 Total liabilities 58,744 52,403 Commitments and contingencies Stockholders' equity Preferred stock, $0.0001 par value; 10,000,000 shares authorized and no shares issued and outstanding as of June 30, 2026 and December 31, 2025 - - Common stock, $0.0001 par value; 300,000,000 shares authorized as of June 30, 2026 and December 31, 2025; 72,314,714 and 60,575,369 shares issued and outstanding as of June 30, 2026 and December 31, 2025, respectively 7 6 Additional paid-in capital 557,186 516,803 Accumulated deficit (499,917 ) (446,462 ) Accumulated other comprehensive (loss) income (13 ) 83 Total stockholders' equity 57,263 70,430 Total liabilities and stockholders' equity $ 116,007 $ 122,833 Acumen Pharmaceuticals, Inc. Condensed Statements of Operations and Comprehensive Loss (in thousands, except share and per share data) (unaudited) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 Operating expenses Research and development $ 27,809 $ 37,125 $ 44,293 $ 62,391 General and administrative 4,747 4,625 9,412 9,729 Total operating expenses 32,556 41,750 53,705 72,120 Loss from operations (32,556 ) (41,750 ) (53,705 ) (72,120 ) Other income (expense) Interest income 1,111 2,015 2,175 4,486 Interest expense (1,094 ) (1,046 ) (2,163 ) (2,069 ) Change in fair value of embedded derivatives (160 ) (40 ) 300 150 Other expense, net (19 ) (129 ) (62 ) (193 ) Total other (expense) income (162 ) 800 250 2,374 Net loss (32,718 ) (40,950 ) (53,455 ) (69,746 ) Other comprehensive gain (loss) Unrealized (loss) gain on marketable securities (34 ) 8 (96 ) 71 Comprehensive loss $ (32,752 ) $ (40,942 ) $ (53,551 ) $ (69,675 ) Net loss per common share, basic and diluted $ (0.45 ) $ (0.68 ) $ (0.79 ) $ (1.15 ) Weighted-average shares outstanding, basic and diluted 72,250,694 60,573,425 67,759,940 60,549,658 Acumen Pharmaceuticals, Inc. Statements of Cash Flows (in thousands) (unaudited) Six Months Ended June 30, 2026 2025 Cash flows from operating activities Net loss $ (53,455 ) $ (69,746 ) Adjustments to reconcile net loss to net cash used in operating activities: Depreciation 23 31 Stock-based compensation expense 4,920 4,972 Amortization of premiums and accretion of discounts on marketable securities, net (161 ) (644 ) Change in fair value of embedded derivatives (300 ) (150 ) Amortization of right-of-use asset 68 61 Realized gain on marketable securities (6 ) (3 ) Noncash interest expense 708 613 Changes in operating assets and liabilities: Prepaid expenses and other current assets 71 1,714 Other long-term assets (4 ) 18 Accounts payable 12,825 (3,545 ) Accrued clinical trial expenses (4,353 ) (4,236 ) Accrued expenses and other liabilities (2,539 ) 4,962 Net cash used in operating activities (42,203 ) (65,953 ) Cash flows from investing activities Purchases of marketable securities (40,823 ) (38,056 ) Proceeds from maturities and sales of marketable securities 53,016 105,316 Purchases of property and equipment - (88 ) Net cash provided by investing activities 12,193 67,172 Cash flows from financing activities Proceeds from private placement, net of offering costs 35,441 - Proceeds from exercise of stock options 172 37 Repurchase of common shares to pay employee withholding taxes (149 ) (73 ) Net cash provided by (used in) financing activities 35,464 (36 ) Net change in cash and cash equivalents and restricted cash 5,454 1,183 Cash and cash equivalents and restricted cash at the beginning of the period 54,220 35,859 Cash and cash equivalents and restricted cash at the end of the period $ 59,674 $ 37,042

NEWTON, Mass., Aug. 05, 2026 (GLOBE NEWSWIRE) -- Acumen Pharmaceuticals, Inc. (NASDAQ: ABOS) (“Acumen” or the “Company”), a clinical-stage biopharmaceutical company developing novel therapeutics that target toxic soluble amyloid beta oligomers for the treatment of Alzheimer’s disease, today announced that the Company will report second quarter financial results on Wednesday, Aug. 12, 2026. The Company will host a conference call and live audio webcast at 8:00 a.m. ET to provide a business and financial update. To participate in the live conference call, please register using this link. After registration, you will be informed of the dial-in numbers including PIN. Please register at least one day in advance.   The webcast audio will be available via this link. An archived version of the webcast will be available for at least 30 days in the Investors section of the Company's website at www.acumenpharm.com. About Acumen Pharmaceuticals, Inc. Acumen Pharmaceuticals is a clinical-stage biopharmaceutical company developing a novel therapeutic that targets toxic soluble amyloid beta oligomers (AβOs) for the treatment of Alzheimer’s disease (AD). Acumen’s scientific founders pioneered research on AβOs, which a growing body of evidence indicates are early and persistent triggers of Alzheimer’s disease pathology. Acumen is currently focused on advancing its investigational product candidate, sabirnetug (ACU193), a humanized monoclonal antibody that selectively targets synaptotoxic AβOs, in its ongoing Phase 2 clinical trial ALTITUDE-AD (NCT06335173) in early symptomatic Alzheimer’s disease patients, following positive results in its Phase 1 trial INTERCEPT-AD. Acumen is also investigating a subcutaneous formulation of sabirnetug using Halozyme’s proprietary ENHANZE® drug delivery technology. Acumen is also collaborating with JCR Pharmaceuticals to develop an Enhanced Brain Delivery™ (EBD™) therapy for Alzheimer’s disease utilizing a transferrin-receptor-targeting blood-brain barrier-penetrating technology. The company is headquartered in Newton, Mass. For more information, visit www.acumenpharm.com. Investors: Alex Braun abraun@acumenpharm.com Media:  ICR Healthcare AcumenPR@icrhealthcare.com 

- This marketing authorization is the first for IZCARGO ™ outside of Japan - 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, today announced that IZCARGO™ (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 IZCARGO™ outside of Japan. IZCARGO™ 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 IZCARGO™ outside of Japan while advancing its global clinical development program. As part of this effort, JCR entered into a 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 IZCARGO™ in countries across the Middle East and North Africa (MENA) region, while Taiba will be responsible for sales and distribution in the relevant territories. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); “We are very pleased to announce the approval of IZCARGO™ 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 IZCARGO™ 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 IZCARGO™ internationally.” “We are pleased to announce the first approval of IZCARGO™ 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. IZCARGO™ 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.” JCR does not expect a material impact on FY2026 consolidated results. 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 IZCARGO™ (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 syndrome. 1,2 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 studies. 3 Clinical studies have also demonstrated the positive effects of pabinafusp alfa on CNS symptoms. 4,5,6 Pabinafusp alfa was approved in Japan by the Ministry of Health, Labour and Welfare and marketed since May 2021 under the brand name “IZCARGO™ I.V. Infusion 10mg.” Important Safety Information INDICATION: IZCARGO™ is indicated for the treatment of mucopolysaccharidosis type II (MPS II), which is also known as Hunter syndrome. IZCARGO™ is approved in Japan and the UAE. CONTRAINDICATION: IZCARGO™ 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 IZCARGO™, 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 IZCARGO™ should be discontinued, and appropriate actions should be taken. When IZCARGO™ 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 IZCARGO™ 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. IZCARGO™ 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 IZCARGO™. 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 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. 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. Contacts Investors & Media: JCR Pharmaceuticals Co., Ltd. Corporate Communications ir-info@jp.jcrpharm.com