Jcr Pharmaceuticals Co., Ltd. TSE:4552

JCR Pharmaceuticals : FY2025-Q2 Financial Report (Jul. 1-Sep. 30, 2025)(536KB)

Published

Source: MarketScreener

Note: This document has been translated from the Japanese original for reference purposes only. In the event of any discrepancy between this translated document and the Japanese original, the original shall prevail.

Financial Summary Consolidated Financial Results for the Six Months ended September 30, 2025 (FY2025) (Japanese standard)

October 30, 2025

Listed company name: JCR Pharmaceuticals Co., Ltd. Listed stock exchange: Tokyo Stock Exchange

Code number: 4552 URL: https://jcrpharm.com/

Representative: (Title) Representative Director, Chairman and President (Name) Shin Ashida

Person in charge of inquiries: (Title) Senior Corporate Officer, Executive Director, Corporate Strategy Division (Name) Yoh Ito TEL: 0797(32)1995

Scheduled date to file Quarterly Securities Report: November 14, 2025 Scheduled date to commence dividend payments: December 5, 2025 Preparation of supplemental information for this financial summary: Available IR Conference: To be held (for institutional investors and analysts)

(Fractions smaller than one million yen omitted)

  1. Consolidated Financial Results for 2Q FY2025 (April 1, 2025 to September 30, 2025)

    1. Consolidated Operating Results (Cumulative) (Percentage shows year-on-year changes.)

      Net sales

      Operating profit(loss)

      Ordinary profit(loss)

      Profit (loss) attributable to owners of parent

      Six Months Ended

      million yen

      %

      million yen

      %

      million yen

      %

      million yen

      %

      Sep. 30, 2025

      21,362

      28.2

      2,379

      -

      2,362

      -

      1,710

      -

      Sep. 30, 2024

      16,657

      (31.4)

      (739)

      -

      (1,621)

      -

      (691)

      -

      (Reference) Comprehensive income: Six months ended Sep. 30, 2025: 2,227 million yen (24.6%)

      Six months ended Sep. 30, 2024: 1,788 million yen ([69.6]%)

      Earnings per share (basic)

      Earnings per share (diluted)

      Six Months Ended

      yen

      yen

      Sep. 30, 2025

      14.03

      14.03

      Sep. 30, 2024

      (5.53)

      -

      (Note) "Earnings per share (diluted)" for six months ended Sep. 30, 2024 is not stated because there was a net loss per share, even though there were residual shares.

    2. Consolidated Financial Conditions

      Total assets

      Net assets

      Equity ratio

      As of

      million yen

      million yen

      %

      Sep. 30, 2025

      109,055

      48,517

      44.1

      Mar. 31, 2025

      104,855

      47,435

      44.8

      (Reference) Shareholders' equity: As of Sep 30, 2025: 48,080 million yen

      As of Mar. 31, 2025: 46,967 million yen

  2. Dividends

    Dividends per share

    1st quarter-end

    2nd quarter-end

    3rd quarter-end

    Year-end

    Annual

    FY2024 FY2025

    yen

    yen

    yen

    yen

    yen

    -

    10.00

    -

    10.00

    20.00

    -

    10.00

    FY2025 (Forecast)

    -

    10.00

    20.00

    (Notes) No revisions were made to the most recently announced dividend forecast.

  3. Consolidated Forecasts for the Fiscal Year Ending March 31, 2026 (April 1, 2025 to March 31, 2026)

    (Percentage figures for the fiscal year represent the changes from the previous year.)

    Net sales

    Operating profit

    Ordinary profit

    Profit attributable to owners of parent

    Earnings per share

    Year ending Mar. 31, 2026

    million yen

    %

    million yen

    %

    million yen

    %

    million yen

    %

    Yen

    37,800

    14.3

    2,600

    -

    2,400

    -

    3,000

    -

    24.22

    (Notes) No revisions were made to the most recently announced financial results forecast.

    *Note

    1. Changes in significant subsidiaries during the period: None

    2. Application of specific accounting practices for preparing quarterly consolidated financial statements: None

    3. Changes in accounting policy, changes in accounting estimates and restatements

      1. Changes in accounting policy due to the revision of accounting standards, etc. : None

      2. Changes in accounting principles other than 1. : None

      3. Changes in accounting estimates : None

      4. Restatements : None

    4. Number of shares outstanding (common stocks)

1. Number of shares outstanding at the end of the period (including treasury

stock)

As of Sep. 30, 2025

129,686,308 shares

As of Mar 31, 2025

129,686,308 shares

2. Number of shares treasury stock at the end of the period

As of Sep. 30, 2025

7,702,502 shares

As of Mar 31, 2025

7,851,002 shares

3. Average number of shares outstanding during the period

(quarterly cumulative amount)

As of Sep. 30, 2025

121,901,549 shares

As of Sep 30, 2024

125,010,957 shares

  • Review of accompanying quarterly consolidated financial statements by certified public accountants or auditing firms: None

  • Explanation on the appropriate use of forecasts of financial results and other comments (Note on forward-looking statements, etc.)

Forward-looking statements, such as forecasts of financial results, contained in this document are based on information currently available to the Company and certain assumption that are judged as rational. The Company does not assure the achievement of these forecasts. In addition, actual financial results may differ significantly from forecasts due to various reasons. For assumptions underlying forecasts of financial results and notes regarding the appropriate use of forecasts of financial results, please refer to "1. Overview of Financial Results, Etc., (3) Explanation on Projections such as Forecasts of Consolidated Financial Results" on page 3 of the attached material.

  • Table of Contents for Attached Material

    1. Overview of Financial Results, Etc. 2

      1. Overview of Quarterly Financial Results 2

      2. Overview of Quarterly Financial Conditions 3

      3. Explanation on Projections such as Forecasts of Consolidated Financial Results 3

    2. Quarterly Consolidated Financial Statements and Important Notes 4

      1. Quarterly Consolidated Balance Sheets 4

      2. Quarterly Consolidated Statements of Income and Consolidated Statements of Comprehensive Income 6

        (Quarterly Consolidated Statements of Income) 6

        (Quarterly Consolidated Statements of Comprehensive Income) 7

      3. Quarterly consolidated statements of cash flows 8

      4. Notes to Quarterly Consolidated Financial Statements 9

        (Notes on segment information) 9

        (Notes on any significant changes in the amount of shareholders' equity) 9

        (Notes on going concern assumption) 9

    3. Other 10

R&D Pipeline 10

  1. ‌Overview of Financial Results, Etc.

    1. ‌Overview of Quarterly Financial Results

      1. Financial results for Q2 FY2025

        Net sales amounted to 21,362 million yen (up 28.2% year on year).

        IZCARGO®10mg for intravenous infusion, a treatment for mucopolysaccharidosis type II, remained strong. Sales of GROWJECT®, a recombinant natural human growth hormone preparation, decreased due to a drug price revision in April 2025, which reduced product sales. However, total net sales increased compared to the same period last year as a result of an increase in income from contractual payment.

        R&D expenses totaled 7,835 million yen (up 1,258 million yen, or 19.1%, year on year) reflecting proactive R&D activities.

        As a result, the Company recorded an operating profit of 2,379 million yen (compared to an operating loss of 739 million yen in the same period of the previous fiscal year), an ordinary profit of 2,362 million yen (compared to an ordinary loss of 1,621 million yen in the same period of the previous fiscal year), and a profit attributable to owners of the parent of 1,710 million yen (compared to a loss attributable to owners of the parent of 691 million yen in the same period of the previous fiscal year).

        Previous quarterly consolidated results (cumulative)

        (April 1, 2024 to September 30, 2024)

        Current quarterly consolidated results (cumulative)

        (April 1, 2025 to September 30, 2025)

        Rate of change

        Amount (million yen)

        Amount (million yen)

        %

        Net sales

        16,657

        21,362

        28.2

        Operating profit (loss)

        (739)

        2,379

        -

        Ordinary profit (loss)

        (1,621)

        2,362

        -

        Profit (loss) attributable to owners of parent

        (691)

        1,710

        -

      2. Main components of sales

        Previous quarterly consolidated results (cumulative)

        (April 1, 2024 to September 30, 2024)

        Current quarterly consolidated results (cumulative)

        (April 1, 2025 to September 30, 2025)

        Rate of change

        Amount (million yen)

        Amount (million yen)

        %

        Human growth hormone product

        GROWJECT®

        9,401

        8,915

        (5.2)

        Treatment for mucopolysaccharidosis type II

        IZCARGO®for I.V. Infusion

        2,845

        3,354

        17.9

        Treatment for renal anemia Epoetin Alfa BS Inj. [JCR] Darbepoetin Alfa BS Inj. [JCR]

        1,764

        962

        801

        1,580

        296

        1,283

        (10.4)

        (69.2)

        60.2

        Regenerative medicine products

        TEMCELL®HS Inj.

        1,521

        1,582

        4.0

        Treatment for Fabry disease Agalsidase Beta BS I.V. Infusion [JCR]

        714

        426

        (40.3)

        Total

        16,246

        15,858

        (2.4)

        Income from contractual payment

        15

        5,015

        -

      3. The Status of Research and Development (R&D) [Lysosomal Storage Disorder (LSD) Treatments]

      • We are currently focusing on the research and development of over 17 LSD treatments utilizing our proprietary blood-brain barrier (BBB) penetration technology, J-Brain Cargo®.

      • For pabinafusp alfa (JR-141), a BBB-penetrating enzyme replacement therapy for Hunter syndrome, we are progressing with global Phase III clinical trials. These trials are progressing smoothly, and the target number of cases has been achieved. In addition, we held a meeting with the U.S. Food and Drug Administration (FDA) in June 2025 to discuss our strategy for new drug application (NDA).

      • For lepunafusp alfa (JR-171), a BBB-penetrating enzyme replacement therapy for mucopolysaccharidosis type I, we have completed a 13-week Phase I/II clinical trial and it's extension study in Japan, Brazil, and the U.S. We intend to develop this product through licensing out and are in ongoing negotiations with potential partners.

      • For posnafusp alfa (JR-441), a BBB-penetrating enzyme replacement therapy for mucopolysaccharidosis type IIIA, a Phase I/II clinical trial is underway in Germany, and the planned enrollment was completed. We have also enrolled the target number of patients for Phase I trials in Japan, and the trials are progressing smoothly. Additionally, the treatment has been granted orphan drug designation by the European Commission (EC) in January 2022, by the U.S. Food and Drug Administration (FDA) in December 2023, and the Japan's Ministry of Health, Labour and Welfare in December 2024.

      • For JR-446, a BBB-penetrating enzyme replacement therapy for mucopolysaccharidosis type IIIB, we entered into a licensing agreement for overseas commercialization and a co-development and commercialization agreement in Japan with MEDIPAL HOLDINGS CORPORATION in September 2023. In December 2024, administration of the investigational drug in a Phase I/II

        clinical trial began in Japan. Additionally, the drug received orphan drug designation from the FDA in April 2025, from the EC in June 2025, and from the Ministry of Health, Labour and Welfare of Japan in September 2025.

      • For JR-471, a BBB-penetrating enzyme replacement therapy candidate for fucosidosis using J-Brain Cargo®, we have granted MEDIPAL HOLDINGS CORPORATION an exclusive license, including sublicensing rights, for the research, development, manufacturing, and commercialization of the product outside Japan, under a licensing agreement signed in October 2022. We are currently conducting necessary studies in preparation for the initiation of clinical trials. Furthermore, in August 2025, the two companies have signed an exclusive global licensing deal and a co-development and commercialization partnership in Japan for JR-479, an investigational therapy for GM2 gangliosidosis.

        [Human Growth Hormone Products]

      • An extension study of redalsomatropin alfa (JR-142), a long-acting recombinant human growth hormone, began administration of the investigational drug in a Phase III clinical trial in Japan in December 2024. Additionally, it is underway as part of a Phase II clinical trial.

        [Creation of Platform Technologies] J-Brain Cargo®

      • In addition to expanding the applicability of JCR's proprietary J-Brain Cargo®technology to various modalities, we are focusing on licensing-out of this technology. In July 2025, we entered into an option agreement with Acumen Pharmaceuticals, Inc. for the licensing of the J-Brain Cargo®technology for the development of a blood-brain barrier-penetrating Alzheimer's disease treatment.

        JUST-AAV

      • We are focusing on creating new platform technologies beyond J-Brain Cargo®. One of the outcomes of these efforts is the creation of a new gene therapy technology called 'JUST-AAV' using adeno-associated virus (AAV) vectors. This technology not only enables efficient delivery of vectors to the specific tissues but also reduces vector accumulation in the liver, which is expected to mitigate side effects. It is currently under development as a new platform technology. In December 2023, we began joint research with Modalis Therapeutics Corporation to develop new gene therapies using this technology. In January 2025, due to the success of the partnership thus far, we concluded to proceed to the next phase of their research by entering into a new joint research agreement. In addition, in July 2025, we entered into a license agreement with Alexion, AstraZeneca Rare Diseases to license the JUST-AAV capsids for the development of new genomic medicines.

    2. ‌Overview of Quarterly Financial Conditions

      1. Status of Assets, Liabilities, and Net Assets

        As of September 30, 2025, total assets amounted to 109,055 million yen (an increase of 4,199 million yen from March 31, 2025), total liabilities were 60,537 million yen (an increase of 3,117 million yen from March 31, 2025), and net assets were 48,517 million yen (an increase of 1,082 million yen from March 31, 2025).

        Current assets increased by 4,776 million yen from March 31, 2025 to 55,833 million yen mainly due to increases in cash and deposits, accounts receivable - trade, and contract assets, and inventories. Non-current assets decreased by 576 yen million from March 31, 2025 to 53,221 yen million due to factors including an increase in investment securities and decreases in property, plant and equipment, intangible assets, and deferred tax assets.

        Current liabilities increased by 3,520 million yen from March 31, 2025 to 47,508 million yen mainly due to increases in short-term borrowings and accounts payable - trade. Non-current liabilities decreased by 402 million yen from March 31, 2025 to 13,028 million yen, mainly due to a decrease in long-term borrowings.

        Net assets increased by 1,082 million yen from March 31, 2025 to 48,517 million yen due to factors including dividend payments, the recording of a profit attributable to owners of parent, and an increase in valuation difference on available-for-sale securities.

        As a result, the equity ratio as of September 30, 2025 was 44.1%, down 0.7 percentage points from March 31, 2025.

        In order to achieve sustainable global growth, we need to secure a flexible and stable source of funds. We concluded commitment line agreements with our financial institutions for a total of 49,500 million yen for the purpose of securing operating funds.

        Of this amount, 26,500 million yen has been concluded to finance the construction of a new plant for drug product filling and finishing. The construction of this new plant has been adopted by the Ministry of Economy, Trade and Industry (METI) under the project of Establishing Biopharmaceutical Manufacturing Sites to Strengthen Vaccine Production, and the subsidy from this project will be used to construct the new plant. The purpose of the commitment line agreement is to provide the necessary funds until we receive the subsidy.

      2. Status of Cash Flows

      Cash and cash equivalents as of September 30, 2025 increased by 2,425 million yen from March 31, 2025 to 15,621 million yen. The details of the cash flows and their primary causes are as follows:

      (Cash Flow from Operating Activities)

      Operating activities resulted in a cash inflow of 2,323 million yen, an increase of in inflow of 3,432 million yen from the same period last year. This was primarily due to a profit before income taxes of 2,541 million yen and depreciation of 1,459 million yen while inventories increased by 1,655 million yen.

      (Cash Flow from Investing Activities)

      Investing activities resulted in a cash outflow of 706 million yen, a decrease in outflow of 2,256 million yen from the same period last year. This was mainly driven by the purchase of property, plant and equipment of 911 million yen.

      (Cash Flow from Financing Activities)

      Financing activities resulted in a cash inflow of 770 million yen, a decrease in inflow of 3,081 million yen from the same period last year. This was primarily due to the repayment of long-term borrowings of 2,200 million yen, a dividend payment of 1,220 million yen, and a net increase in short-term borrowings of 4,200 million yen.

    3. ‌Explanation on Projections such as Forecasts of Consolidated Financial Results

    Looking at consolidated financial results for the six months ended September 30, 2025, net sales and profits increased year on year. There have been no changes to the forecasts for the fiscal year ending March 31, 2026 announced on May 13, 2025.

  2. ‌Quarterly Consolidated Financial Statements and Important Notes

    1. ‌Quarterly Consolidated Balance Sheets

      (Millions of yen)

      As of March 31, 2025 As of September 30, 2025

      Assets

      Current assets

      Cash and deposits

      13,196

      15,621

      Accounts receivable - trade, and contract assets

      12,236

      13,645

      Merchandise and finished goods

      2,571

      2,182

      Work in process

      6,388

      7,528

      Raw materials and supplies

      12,799

      13,702

      Other

      3,866

      3,152

      Total current assets

      51,056

      55,833

      Non-current assets

      Property, plant and equipment

      Buildings and structures, net

      13,229

      12,746

      Land

      10,587

      10,587

      Construction in progress

      9,495

      10,254

      Other, net

      4,097

      3,542

      Total property, plant and equipment

      37,410

      37,131

      Intangible assets

      Patent right

      1,881

      1,743

      Other

      1,079

      962

      Total intangible assets

      2,960

      2,705

      Investments and other assets

      Investment securities

      9,629

      10,404

      Other

      3,803

      2,984

      Allowance for doubtful accounts

      (4)

      (4)

      Total investments and other assets

      13,427

      13,384

      Total non-current assets

      53,798

      53,221

      Total assets

      104,855

      109,055

      Liabilities

      Current liabilities

      Accounts payable - trade

      590

      1,052

      Short-term borrowings

      26,055

      28,455

      Income taxes payable

      36

      365

      Special suspense account for tax purpose reduction entry

      11,996

      11,996

      Provision for bonuses

      1,089

      1,368

      Provision for bonuses for directors (and other officers)

      127

      62

      Other

      4,093

      4,207

      Total current liabilities

      43,988

      47,508

      Non-current liabilities

      Long-term borrowings

      12,050

      11,650

      Provision for employee stock ownership plan

      120

      109

      Retirement benefit liability

      966

      1,002

      Other

      294

      266

      Total non-current liabilities

      13,431

      13,028

      Total liabilities

      57,420

      60,537

      (Millions of yen)

      As of March 31, 2025 As of September 30, 2025

      Net assets

      Shareholders' equity

      Share capital

      9,061

      9,061

      Capital surplus

      10,392

      10,378

      Retained earnings

      31,191

      31,681

      Treasury shares

      (5,066)

      (4,976)

      Total shareholders' equity

      45,579

      46,144

      Accumulated other comprehensive income

      Valuation difference on available-for-sale securities

      937

      1,515

      Deferred gains or losses on hedges

      2

      1

      Foreign currency translation adjustment

      393

      371

      Remeasurements of defined benefit plans

      53

      47

      Total accumulated other comprehensive income

      1,387

      1,935

      Share acquisition rights

      75

      75

      Non-controlling interests

      392

      361

      Total net assets

      47,435

      48,517

      Total liabilities and net assets

      104,855

      109,055

    2. ‌Quarterly Consolidated Statements of Income and Consolidated Statements of Comprehensive Income (Quarterly Consolidated Statements of Income)‌

      (Millions of yen)

      Six months ended September 30, 2024

      Six months ended September 30, 2025

      Net sales

      16,657

      21,362

      Cost of sales

      4,330

      4,323

      Gross profit

      12,326

      17,038

      Selling, general and administrative expenses

      13,066

      14,659

      Operating profit (loss)

      (739)

      2,379

      Non-operating income

      Interest income

      68

      38

      Dividend income

      17

      21

      Gain on sale of investment securities

      0

      41

      Foreign exchange gains

      -

      158

      Compensation income

      -

      47

      Other

      48

      45

      Total non-operating income

      134

      353

      Non-operating expenses

      Share of loss of entities accounted for using equity method

      398

      91

      Interest expenses

      63

      157

      Commission expenses

      41

      31

      Depreciation

      98

      77

      Foreign exchange losses

      404

      -

      Other

      9

      13

      Total non-operating expenses

      1,016

      370

      Ordinary profit (loss)

      (1,621)

      2,362

      Extraordinary income

      Gain on sale of investment securities

      -

      209

      Gain on reversal of share acquisition rights

      393

      -

      Gain on cancellation of contract

      627

      -

      Other

      44

      -

      Total extraordinary income

      1,065

      209

      Extraordinary losses

      Loss on disposal of non-current assets

      0

      31

      Total extraordinary losses

      0

      31

      Profit (loss) before income taxes

      (556)

      2,541

      Income taxes - current

      24

      252

      Income taxes - deferred

      100

      581

      Total income taxes

      124

      833

      Profit (loss)

      (680)

      1,707

      Profit (loss) attributable to non-controlling interests

      10

      (3)

      Profit (loss) attributable to owners of parent

      (691)

      1,710

      ‌(Quarterly Consolidated Statements of Comprehensive Income)

      (Millions of yen)

      Six months ended September 30, 2024

      Six months ended September 30, 2025

      Profit (loss)

      (680)

      1,707

      Other comprehensive income

      Valuation difference on available-for-sale securities

      1,863

      578

      Deferred gains or losses on hedges

      8

      (1)

      Foreign currency translation adjustment

      208

      (50)

      Remeasurements of defined benefit plans, net of tax

      (12)

      (6)

      Share of other comprehensive income of entities accounted for using equity method

      401

      -

      Total other comprehensive income

      2,469

      520

      Comprehensive income

      1,788

      2,227

      Comprehensive income attributable to

      Comprehensive income attributable to owners of parent

      1,740

      2,258

      Comprehensive income attributable to non-controlling interests

      48 (30)

    3. ‌Quarterly consolidated statements of cash flows

      (Millions of yen)

      Six months ended September 30, 2024

      Six months ended September 30, 2025

      Cash flows from operating activities

      Profit (loss) before income taxes

      (556)

      2,541

      Depreciation

      1,667

      1,459

      Loss (gain) on sale of investment securities

      0

      (251)

      Share of loss (profit) of entities accounted for using equity method

      398

      91

      Gain on reversal of share acquisition rights

      (393)

      -

      Increase (decrease) in retirement benefit liability

      21

      34

      Increase (decrease) in provision for bonuses

      162

      278

      Share-based payment expenses

      9

      27

      Interest and dividend income

      (85)

      (59)

      Interest expenses

      63

      157

      Foreign exchange losses (gains)

      457

      (81)

      Decrease (increase) in trade receivables

      2,579

      (1,409)

      Decrease (increase) in accounts receivable - other

      69

      512

      Decrease (increase) in inventories

      (1,830)

      (1,655)

      Increase (decrease) in trade payables

      (350)

      462

      Increase (decrease) in accounts payable - other

      159

      (190)

      Increase (decrease) in accrued consumption taxes

      (1,818)

      453

      Other, net

      (228)

      (738)

      Subtotal

      325

      1,633

      Interest and dividends received

      85

      59

      Interest paid

      (68)

      (152)

      Income taxes refund (paid)

      (1,451)

      783

      Net cash provided by (used in) operating activities

      (1,109)

      2,323

      Cash flows from investing activities

      Purchase of property, plant and equipment

      (2,992)

      (911)

      Proceeds from sale and redemption of investment securities

      2

      293

      Other, net

      27

      (88)

      Net cash provided by (used in) investing activities

      (2,962)

      (706)

      Cash flows from financing activities

      Net increase (decrease) in short-term borrowings

      5,112

      4,200

      Proceeds from long-term borrowings

      300

      -

      Repayments of long-term borrowings

      (300)

      (2,200)

      Net decrease (increase) in treasury shares

      15

      17

      Dividends paid

      (1,248)

      (1,220)

      Other, net

      (26)

      (27)

      Net cash provided by (used in) financing activities

      3,852

      770

      Effect of exchange rate change on cash and cash equivalents

      (264)

      38

      Net increase (decrease) in cash and cash equivalents

      (484)

      2,425

      Cash and cash equivalents at beginning of period

      18,756

      13,196

      Cash and cash equivalents at end of period

      18,271

      15,621

    4. ‌Notes to Quarterly Consolidated Financial Statements

    ‌(Notes on segment information)

    Segment information is not disclosed since the Group has only one segment, Pharmaceuticals.

    ‌(Notes on any significant changes in the amount of shareholders' equity)

    Not applicable.

    ‌(Notes on going concern assumption) Not applicable.

  3. ‌Other

‌R&D Pipeline

Recombinant drug products

Code Nonproprietary Name

Status

Indication

Remarks

JR-141

BBB-Penetrating Iduronate-2-sulfatase

Global: Clinical Phase III trials

Mucopolysaccharidosis II (Hunter syndrome)

ERT

(rDNA origin)

J-Brain Cargo®

JR-142

Long-acting Growth hormone

(rDNA origin)

Japan: Clinical Phase III trials

Pediatric growth hormone deficiency

J-MIG System®

JR-171

Mucopolysaccharidosis I (Hurler syndrome, etc.)

BBB-Penetrating

α-L-Iduronidase

Global: Clinical

Phase I/II trials

ERT

J-Brain Cargo®

(rDNA origin)

J-MIG System®

JR-441

BBB-Penetrating heparan N-sulfatase

(rDNA origin)

Germany: Clinical Phase I/II trials Japan: Clinical Phase I trials

Mucopolysaccharidosis IIIA (Sanfilippo syndrome type A)

ERT

J-Brain Cargo®

JR-446

Mucopolysaccharidosis IIIB (Sanfilippo syndrome type B)

BBB-Penetrating

α-N-acetylglucosaminidase

(rDNA origin)

Japan: Clinical Phase I/II trials

ERT

J-Brain Cargo®

JR-471

Fucosidosis

BBB-penetrating

α-L-fucosidase

Preclinical

ERT

(rDNA origin)

J-Brain Cargo®

JR-479

GM2 gangliosidosis (Tay-Sachs disease, Sandhoff disease)

BBB-penetrating

β-Hexosaminidase A

Preclinical

ERT

(rDNA origin)

J-Brain Cargo®

(Note) ERT= Enzyme Replacement Therapy