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Consolidated Financial Results for the Fiscal Year 2024 (Year ended March 31, 2025) May 13, 2025 Kaken Pharmaceutical Co., Ltd.
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Cautionary notes regarding forward-looking statement • The performance forecasts described in this material are rational based on the information currently available and have been determined by the Company as reasonable. • Considerable financial investment and a long development time are required before a new drug is launched. The Company carefully develop new drugs by confirming their efficacy and safety. There is a possibility that their development may be discontinued before completion, in such case where anticipated efficacy has not been proven or safety issues have been identified. • The “Projects under Development” is based on the current development plans. The status may change depending on their progress. • The information about pharmaceutical products (including those under development) included in this material is not intended as an advertisement or medical advice. 1
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Agenda 1 Consolidated Financial Results for FY2024 3 Progress on “Long-Term Business Plan 2031” 5 Updates on Research and Development 4 Updates on “Actions to Implement Management that is Conscious of Cost of Capital and Stock Price” 2 Consolidated Financial Forecast for FY2025 2
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 1 Consolidated Financial Results for FY2024 3
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved FY2023 FY2024 YoY Change Change (%) Plan FY2024 * Change (%) Net Sales 72,044 94,035 21,990 130.5 94,000 100.0 Cost of Sales 33,505 35,502 1,997 106.0 ー ー Gross Profit 38,539 58,532 19,993 151.9 ー ー Selling, General and Administrative Expenses 29,025 37,497 8,471 129.2 ー ー R&D Expenses 12,543 18,725 6,182 149.3 ー ー Operating Profit 9,513 21,034 11,521 221.1 20,800 101.1 Ordinary Profit 9,951 21,279 11,328 213.8 21,000 101.3 Profit before Income Tax 9,796 19,435 9,639 198.4 ー ー Profit 8,025 13,945 5,919 173.8 14,000 99.6 Consolidated Statements of Income (amounts are rounded down to the nearest million yen) （*: Revised forecast announced on April 8, 2025） 4
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Net Sales Bridge FY2023 72,044 FY2024 94,035 +21,990 YoY change 130.5％ ✓ Pharmaceuticals and medical devices ・ Upfront payment from IP transfer and commercial option agreement for NM26 and license agreement for STAT6 inhibitors impacted overseas sales. ・ Mainly from Artz sales due to remaining impact of switchover from competing product, which sales had ended, in domestic sales. ✓ Crop protection products ・ Sales of Polyoxin, a major products in crop protection, decreased due to the impact of inventory adjustments at overseas customers. ✓ Real estate ・ About the same sales level +22,276 ▲348 +62 Pharmaceuticals and medical devices Crop protection products Real estate and others (amounts are rounded down to the nearest million yen) 5
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Operating Profit Bridge +11,521 YoY change 221.1％ +1,997 +2,289 +6,182 +21,990 ✓ Net sales (increase by ¥21,990 million) Increased due to overseas sales increase ✓ Cost of sales (increase by ¥1,997 million) Increased due to net sales increase ✓ SG&A (excl. R&D expenses) (increase by ¥2,289 million) Increased due to acquisition related cost ✓ R&D expenses (increase by ¥6,182 million) Increased due to progress in development in various pipelines. Upfront payment relating to in-licensing, etc. (ESK-001, ND081 and others) 9,513 21,034 FY2023 FY2024 Analysis of Changes Net sales Cost of sales SG&A (excl. R&D expenses) R&D expenses (amounts are rounded down to the nearest million yen) 6
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Consolidated Balance Sheets / Consolidated Statements of Cash Flows As of Mar. 31, 2024 As of Mar. 31, 2025 YoY change Current Assets 106,974 111,377 4,402 Non-current Assets 64,648 79,045 14,396 Total Assets 171,623 190,422 18,799 Current Liabilities 19,024 30,354 11,330 Non-current Liabilities 8,843 7,433 ▲1,410 Total Liabilities 27,868 37,787 9,919 Total Net Assets 143,755 152,634 8,879 Consolidated balance sheet As of Mar. 31, 2024 As of Mar. 31, 2025 YoY change Net cash provided by (used in) operating activities 2,577 29,780 27,203 Net cash provided by (used in) investing activities ▲5,854 ▲19,650 ▲13,795 Net cash provided by (used in) financing activities ▲5,658 ▲5,369 288 Cash and cash equivalents at end of period 65,325 70,085 4,760 【Major changes】 ◆ Total assets: Cash and cash equivalents: ¥70,085 million (increased by ¥4,760 million) Goodwill: ¥14,035 million (increased by ¥13,097 million) ◆ Total liabilities: Interest bearing debt: ¥3,850 million (no change) ◆ Total net assets: Retained earnings: ¥135,242 million (increased by ¥6,736 million) Treasury stock: ▲¥28,233 million (decreased by ¥380 million) 【Major changes】 ◆ Net cash provided by (used in) operating activities: Profit before income taxes: ¥19,435 million Change in A/R and inventories: ▲¥2,493 million ◆ Net cash provided by (used in) operating activities: Purchase of PP&E: ▲¥4,138 million Purchase of shares from acquisition: ▲¥15,378 million ◆ Net cash provided by (used in) operating activities: Change in treasury stock: ¥1,828 million Cash dividend paid: ▲¥7,198 million Consolidated statement of cash flows (amounts are rounded down to the nearest million yen) (amounts are rounded down to the nearest million yen) 7
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Domestic Pharmaceuticals & Medical Devices Sales by Products FY2023 FY2024 Change Change (%) Clenafin 17,116 16,866 ▲249 98.5 Artz 18,040 19,031 990 105.5 Seprafilm 6,995 6,952 ▲42 99.4 Fiblast 2,626 2,432 ▲194 92.6 Ecclock 1,812 2,037 224 112.4 Regroth 898 922 24 102.7 Hernicore 382 405 22 106.0 Generic Products 7,992 8,497 504 106.3 (amounts are rounded down to the nearest million yen) ◆ Clenafin Sales declined slightly due to NHI price revision while the volume had increased ◆ Artz Sales increased due to remaining impact of switchover from competing products, which sales had ended ◆ Seprafilm Sales were at same level due to increase in usage of anti-adhesives in unused operations, while the high competition with other anti- adhesives ◆ Ecclock Sales increased due to market expansion through disease awareness of doctors and patients ◆ Generic Products Sales increased due to sales growth of focused products Main factors 8
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Topics Remarks IP transfer and commercial option agreement for NM26 • Entered agreement with Johnson & Johnson (J&J) and Numab. • Received an upfront payment of $86MM in total from J&J and Numab. • The rights to receive potential milestone payments based on the development progress and regulatory and sales milestone. • An option to negotiate co-promotion agreement in Japan. License agreement for STAT6 inhibitors • Entered agreement with J＆J • Received an upfront payment of $30MM • The rights to receive success-based payments based on development progress and sales milestone. • Retain commercialization rights in Japan • Size of the agreement is at a record high Major Topics for FY2024 R&D: Out-Licensing Topics Remarks Entered into a feasibility study agreement for the creation of SaMD for the treatment of orthopedic diseases • Consider the use of digital technology in the field of orthopedics through joint development of digital products. Agreement for exclusive marketing rights of Silk-Elastin Wound Healing Sheet, a novel wound treatment material • Japan’s first medical device using recombinant DNA technology. • Provide new treatment option in the field of wound treatment. Entered collaboration and option agreement for ND081 for treatment of inflammatory bowel disease • New multi-specific antibody for treatment of inflammatory bowel disease. • New co-development project with Numab following NM26. Collaboration and licensing agreement for ESK-001, a TYK2 inhibitor, for dermatology indication in Japan • Increase pipeline in dermatology area. R&D: In-Licensing 9
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Major Topics for FY2024 Topics Remarks Acquisition of Aadi Subsidiary, Inc. in the US. <What we earned from the acquisition> • Prescription drug for rare cancer, FYARRO® • Sales office in the US • sales platform and know-hows for orphan drugs Approval for marketing authorization in Italy of Jublia®, a topical formulation drug for onychomycosis. • First marketing authorization approval of efinaconazole in Europe. Overseas expansion 10
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Topics Remarks Announced the result of the “Large-scale Epidemiological Survey on the Actual Situation and Latent Morbidity of Tinea Pedis and Tinea Unguium (Foot Check 2023)”. • Cooperation with Japan Organization of Clinical Dermatologists. Construction of new fermentation plant in Shizuoka Site. • Preparation for increased demand of polyoxins. Entered into agreement with Yamagata City, Yamagata regarding health promotion and maintenance. • First cooperation agreement with local municipality. Disposal of treasury shares by third-party allotment. • With an aim of establishing a broader relationship with J&J. Survey on hyperhidrosis patients using big data relating to healthcare and weather. • Industry-academia collaboration with Nagasaki University. Certified as “A Plan to Establish Business Infrastructure based on MIDORI Strategy for Sustainable Food System” of MAFF. • Popularize and expansion of Polyoxins, natural pesticides derived from fermentation process of actinomycete. Certified as 2025 Certified KENKO Investment for Health Outstanding Organizations Recognition Program. • Certified consecutively for two years since 2024. Entered into business alliance agreement with Rakuten Mobile, Inc. for providing information for foot health. • Established “Project for Fulfilling Foot”. Entered into comprehensive agreement with Fujieda City, Shizuoka. • Maintaining and operating manufacturing and research facility in Fujieda City, Shizuoka since 1963, resulted the cooperation. Major Topics for FY2024 Others 11
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 2 Consolidated Financial Forecast for FY2025 12
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 13 Financial Performance Forecast FY2024 FY2025 Plan Change Change (%) Net Sales 94,035 88,000 ▲6,035 93.6 Operating Profit 21,034 5,200 ▲15,834 24.7 Ordinary Profit 21,279 6,000 ▲15,279 28.2 Profit 13,945 3,400 ▲10,545 24.4 Selling, General and Administrative Expenses 37,497 44,500 7,003 118.7 R&D Expenses 18,725 20,900 2,174 111.6 (amounts are rounded down to the nearest million yen) Main factors ◆ Sales / Profit ・Sales of FYARRO® will be added in net sales ・Reaction of upfront payment received in FY2024 ・Impact from Clenafin patent cliff ◆ SG&A (excl. R&D expenses) ・SG&A expenses for Aadi will be included ◆ R&D expenses ・expected to increase, as development of pipelines progress ・include upfront payment of ¥1,650 million for in-licensing sebetralstat.
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved FY2024 FY2025 Plan Change Change (%) Clenafin 16,866 13,200 ▲3,666 78.3 Artz 19,031 18,500 ▲531 97.2 Seprafilm 6,952 7,000 47 100.7 Fiblast 2,432 2,300 ▲132 94.6 Ecclock 2,037 2,400 362 117.8 Regroth 922 1,000 77 108.4 Hernicore 405 400 ▲5 105.2 Generic Products 8,497 8,900 402 104.7 (amounts are rounded down to the nearest million yen) Domestic Pharmaceuticals & Medical Devices Sales Forecast by Products Main factors ◆ Clenafin • Expected to decline taking into account start selling AG and new entry of generic products. • Continue to increase treatment opportunities for untreated patients. ◆ Artz • Sales to decline due to NHI price revision. • Appeal for usage of hyaluronic acid in the shrinking market. ◆ Seprafilm • Plan to increase sales from switchover and increase usage of anti-adhesives in unused operations. ◆ Ecclock • Sales to increase through higher disease awareness of doctors and patients, and market expansion. ◆ Generic Products • Enhance sales of focused products. 14
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Consolidated Financial Performance 74,979 76,034 72,984 72,044 94,035 88,000 17,788 17,064 7,998 9,513 21,034 5,200 13,405 9,549 5,440 8,025 13,945 3,400 Sales Operating Profit Profit Mar. 2021 Mar. 2022 Mar. 2023 Mar. 2024 Mar. 2025 Mar. 2026 (plan) Annual Dividend 150 yen 150 yen 150 yen 150 yen 190 yen (Annual 150 yen) (Bonus 40 yen) 190 yen ROE (Return on Equity) 10.1% 7.0% 4.0% 5.7% 9.4％ --- Amount in Yen million 15
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 3 Progress on “Long-Term Business Plan 2031” 16
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved VISION and Strategy for Long-Term Business Plan 2031 VISION 1. A company that contributes to longer healthy life expectancy by developing and supplying innovative new drugs in a speedy manner. 2. A research-based pharmaceutical company with a global presence, primarily in the areas of dermatology and orthopedics. “3Xs” ～Three Transformations～ R&D Transformation 1. Utilize KAKEN’s own research base 2. Expand into new therapeutic areas 3. Take on challenges in new modalities 4. Actively invest in research and development Overseas Expansion Transformation 1. Enhance line-up of global products from in-house drug discovery and licensing of products already commercialized or under development 2. Maximize the value of global products through our own development and commercialization overseas Management Base Transformation 1. Develop human resources capable of pursuing transformation as professionals and improve the working environment 2. Create a corporate culture capable of continuous transformation by utilizing data and digital technologies 3. Maximize product value and establish reliable and high-quality manufacturing capabilities from the viewpoint of “patients first” 1st X 2nd X 3rd X 17
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 18 Progress on Long-Term Business Plan 2031 for FY2024 R&D Transformation 1. Improve Launch Probability > Projects using drug discovery AI are established and continue collaboration with drug discovery partner. 2. Expand Pipeline > In-licensed projects: four (Silk-Elastin, ND081, FYARRO® and ESK-001). > Pipelines: 10 (as of May 2025) 3. Address New Needs and Overseas Expansion > Exploration of original target using bioinformatics. > IP transfer and commercial option agreement for NM26 and license agreement for STAT6 inhibitors. 4. Take on Challenges in New Fields > Under consideration of potential collaboration with several companies with new modality and DTx tool. Overseas Expansion Transformation 1. Expand Global Products > Ecclock: Applied for marketing authorization approval in Korea. Clenafin: Obtained marketing authorization in Italy and waiting for an approval in Germany. 2. Establish our own Overseas Development Capabilities > In consultation with FDA for Phase III clinical trial of KP-001 in the US. 3. Establish Global Manufacturing and Commercialization Structure > Acquired Aadi for establishing our own commercialization structure. Management Base Transformation 1. Human Resources Strategy > Actively recruit mid-career professional with advanced expertise; enhanced training programs and provided opportunities for personal development; Certified as 2025 Certified KENKO Investment for Health Outstanding Organizations Recognition Program. 2. DX Strategy > Restructure digital platform for Customer Relationship Management and medical professionals for optimizing medical information provision, optimize supply management system etc.; DX Certification (security management) 3. Production Strategy > Preparation of manufacturing IND and commercial production for KP-001. > Construction of labor-saving and automated drug production machinery. Crop Protection Business 1. Growth in North America and New Market > Polyoxin: Expand into Indian market. 2. Growth by entering the EU Market > Polyoxin: Accepted application for setting a maximum imported crop residue level in Europe.
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Progress on Strategic Investment ◼ Spent approximately ¥72 billion in strategic investment for the past three years. Planned strategic investment (over the 10 years of the plan) 3 years result M&A, in-licensing R&D expenses (incl. CapEx and DX) ¥120 billion ¥80 billion ¥39 billion ¥33 billion ¥200 billion ¥72 billion R&D expenses ¥35 billion Capital expenditure (incl. DX investment) ¥4 billion ✓ Avg. R&D expenses: 1.6 times compared to three-year average of FY2019-2021 ✓ Launched: One (NexoBrid) ✓ Increase in pipelines: Seven (in FY2021) → 10 (in FY2024) ✓ In-licensed seven projects in three years ✓ Acquisition of Aadi M&A, in-licensing ¥33 billion 19
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Analysis of the Current Status of the Long-Term Business Plan 2031 ＊ Positive impact and negative impact for the achievement of the long-term business plan. Three years have passed since the formulation of the Long-Term Business Plan 2031. Need to respond to internal and external changes in the business environment. Analysis of the current status Progress on covering the patent cliff of Clenafin Impact of the "Elective Medical Care" system on long-term listed drug Soaring R&D costs Intensifying competition in the in-licensing and M&A market Increase in number of in-licensed products and projects under development Early realization of derivation Progress in overseas expansion Changes in the business environment＊ Partially reviewed the Long-Term Business Plan 2031 due to changes in the business environment. Revise Addition Revise 20
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Long-Term Business Plan 2031 -Concept of the Revision- Concept of the Revision Major Items ➢ Increase strategic investment for continuous launch of innovative new drugs to further increase corporate value. ➢ Maintain financial discipline taking into account increasing volatility and uncertainty in financial performance and cash flows. ➢ Enhance shareholder return for the stakeholders. A) Revision and addition to “3Xs” Three Transformations. B) Revision of amount of strategic investment. C) Revision of shareholder return policy. D) Revision of cash flow allocation taking into account the financial discipline together with B) and C) above. 21
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Long-Term Business Plan 2031 - Revision and Addition to Strategies - ◼ Additional measures and revisions to further pursue Three Transformations Items Priority Measures of the Plan Addition and Revision 1st x ➢ Secure pipelines capable of launching eight new products over the 10 years of the plan (maintain at least six pipelines in Phase I or later phases at any given time). ➢ Secure at least one new licensed products or new marketing alliance every year. ➢ Secure pipelines capable of launching eight new products over the 10 years of the plan (maintain at least eight pipelines in Phase I or later phases at any given time). ➢ Secure at least one new licensed products or new marketing alliance every year expanding global products as target. 2nd x ➢ overseas sales ratio of 25% or higher. • Expand global products. • Establish our own overseas development capabilities. • Establish global manufacturing and commercialization structure. ➢ Succession and stable operation of FYARRO® business. ➢ Consideration of European expansion policy. ➢ Procure products for overseas expansion in anticipation of synergies with Aadi. 3rd x ➢ Increase corporate value by establishing a strong organizational base that can flexibly respond to change and by improving operational efficiency. ➢ Actively recruit mid-career professionals with advanced expertise. ➢ Introduce Employee Stock Ownership Plan (J-ESOP). ➢ Maximize products value through optimizing medical information provision activities using Customer Relationship Management Revision Addition Addition, Specified 22
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved ◼ Revision of strategic investment amount and shareholder return policy Items KPIs for FY2031 Revision and additions Strategic Investment ➢ ¥200 billion or more over the 10 years of the plan ➢ ¥260 billion or more over the 10 years of the plan Shareholder Return Policy ➢ Dividend payout ratio: 30％ or higher ➢ Shareholder return ratio: 50％ or higher ➢ Minimum of ¥190 per share dividend of FY2024 taking into account dividend payout ratio of 30％ or higher and shareholder return ratio of 50％ or higher. ➢ Cumulative shareholder return of ¥50 billion or more for the next seven years. Others ➢ Consider changing accounting standard to IFRS. Revision Revision Addition 23 Long-Term Business Plan 2031 - Revision and Addition to Strategies -
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Revision of Image of Cash Flow Allocation (FY2025 – FY2031) ◼ Generate sources for strategic investment and shareholder return through operating cash flows and balance sheet management. Cash flows from operating activities ¥190 billion ¥100 billion Balance Sheet Management Approx. ¥30 billion ¥190 billion ¥50 billion Cash required for business activities Strategic Investment Maintenance CapEx Shareholder Return Cash-outflow Cash-inflow Cash-outflow of ¥260 billion or more ➢ Prioritize excess cash for strategic investment ➢ Consider utilizing indebtedness taking financial discipline into account ➢ Generate cash through disposal of assets, etc. * Figures in this page are estimates. * Cash flows from operating activities exclude R&D investment and one-time fees for in-licensing. 24 ¥20 billion
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 4 Updates on “Actions to Implement Management that is Conscious of Cost of Capital and Stock Price” 25
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved What Kaken Aim for Kaken aim to increase mid- to long-term corporate value through execution of strategies to achieve VISION for Long-Term Business Plan 2031 Achieve ROE target of 10% through allocating cash as priority source for strategic investments and continuous launch of revolutionary drugs Reduce cost of capital and expand pipelines to improve PER Realize PBR of 1.0x or higher 26
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 27 Target and Initiatives ◼ Target and Initiatives to achieve PBR of 1.0x or higher Target ROE PER PBR 10％ or higher Further improvement (lowering cost of capital) 1.0x or higher Initiatives A) Pursue 3Xs strategy to expand pipelines and increase net sales and profits B) Prioritize strategic investment based on cash flow allocation C) Set reduction policy of cross-shareholding D) Communication with investors by Corporate Communications Department E) Reset materiality issues and hold investors’ meeting on sustainability F) Revision of stock compensation plan and director’s compensation plan
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved C) Set reduction policy of cross-shareholding 28 Progress on Initiatives Initiatives Major progress ◆ Increased number of pipelines in Phase I or later phases from six to eight. ◆ Pipelines: 10; In-licensing: Four ◆ IP transfer and commercial option agreement for NM26 and license agreement for STAT6 inhibitors; acquisition of Aadi. ◆ Increased strategic investment amount to ¥260 billion over the 10 years of the plan. ◆ Minimum of ¥190 per share dividend annually. ◆ Spent ¥38.5 billion for strategic investment in FY2024. ◆ Reduced cross-shareholdings based on the reduction policy. ◆ Details in “Communication with Shareholders and Investors” ◆Increased relevance of the Long-Term Business Plan 2031 ◆Held Sustainability Meeting in May 2024 ◆ Revision of stock compensation plan and director’s compensation plan. ◆ Resolved introduction of ESOP. A) Pursue 3Xs strategy to expand pipelines and increase net sales and profits Increase pipelines B) Prioritize strategic investment based on cash flow allocation Increase strategic investment Enhance shareholder return D) Communication with investors by Corporate Communications Department E) Reset materiality issues and investors’ meeting on sustainability F) Revision of stock compensation plan and director’s compensation plan Revision Revision Revision ◆ Revision of Initiatives ◆ Achievement
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 77% 8% 15% ◼ Conducted communication with shareholders and investors through investor meeting and individual meeting. Number of investors meeting conducted FY2024: 80 times (47 investors) (reference) FY2023: 38 times (28 investors) Main attendants Investor meetings Individual meetings Shareholders meeting All directors (excluding outside directors) Director in charge of investor relations GM of Corporate Communications Department All directors and audit & supervisory board members Attribute of investors * * Include sell side analysts Topics and themes in the meeting ✓ Current business situation ◼ Financial performance and external and market environment ◼ Progress on development of pipelines ◼ Countermeasures for Clenafin patent cliff ✓ Corporate actions in FY2024 ◼ Acquisition of Aadi, third-party allotment of treasury shares ◼ Out-licensed STAT6 and transferred IP , NM26 ✓ Mid- to long-term management principles ◼ Progress on Long-Term Business Plan 2031 ◼ Growth and R&D strategies ◼ Progress on strategic investment and cash spending including shareholder return ◼ Thoughts about financial discipline ✓ About disclosure ◼ Expectation on expansion of disclosures and IR activities ◼ Status on ESG activities Communication with Shareholders and Investors Domestic/ Foreign attributes Buy side analysts Person in charge of exercising voting right Sell side analysts Domestic 45% Foreign 55% 29
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 0 1 2 3 4 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024 0 10 20 30 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024 9.4% 0.0% 10.0% 20.0% 30.0% 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024 30 Progress on Targets ◼ ROE has improved to 9.4% and PBR exceeded 1.0x Cost of Capital ROE (%) PER (multiple) 12.26x PBR (multiple) 1.12x
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 31 Appendix
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 8 8 10 FY2022 FY2023 FY2024 Pipelines 32 (Reference) Progress on In-Licensing and Pipelines 【 1st X R&D Transformation】 ESK-001 Marketing alliances from Eisai FYARRO® Tildacerfont ND081 Seladelpar （Merislon®/Myonal®） Silk-Elastin FY2022 FY2023 FY2024 Progress on in-licensing and marketing alliances Pipelines * Include NM26, Sebetralstat In-licensed seven projects as of March 2025 *as of May 2025, see Appendix for more information
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 33 (Reference) Progress on Out-Licensing Out-licensing ✓ Entered into IP transfer and commercial option agreement for NM26. ✓ Entered into licensing agreement for STAT6 inhibitor. (Disposed treasury shares through a third-party allotment to JJDC.) 【 1st X R&D Transformation】 <Reference> Received an upfront payment of $86MM in total from Numab and Johnson & Johnson for the transfer of IP and licensing rights in FY2024. Expects potential payments totaling up to $252.4MM in total for the achievement of certain regulatory and sales milestones as well as single to lower double-digit percent royalties on net sales in Asia. Received an upfront payment of $30MM from Johnson & Johnson by granting an exclusive license for the worldwide development, manufacturing and commercialization of STAT6 program in FY2024. Expect for success-based payments of up to $1,217.5MM based on development progress and sales milestones, as well as single to lower double-digit percent royalties on sales worldwide. ■ IP transfer and commercial option agreement for NM26 ■ License agreement for STAT6 inhibitors
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 34 (Reference) Progress on Overseas Expansion 【 2nd X Overseas Expansion Transformation】 Overseas Expansion ✓ Acquisition of Aadi Subsidiary, Inc. in the US. ✓ Dong Wha Pharma filed for marketing authorization of “Sofpironium Bromide” in Korea. ✓ For marketing authorization of “efinaconazole”, filed in Germany and obtained approval in Italy ✓ Entered Phase III clinical trial of “efinaconazole” in China by AIM <Reference> ■Acquisition of Aadi Subsidiary, Inc. in the US Entered into a stock purchase agreement for the acquisition of Aadi Subsidiary, Inc for $100MM, to make it a wholly-owned subsidiary, in December 2024 and completed the acquisition on March 26, 2025. Expected effects from the acquisition ➢ A step towards building own sales structure overseas under the Overseas Expansion Transformation of Long-Term Business Plan 2031. ➢ Aadi obtains sales platform and know-hows for orphan drugs, which is critical for the success of KP- 001 business in the US. ➢ Potential procurement of global products through synergies with Aadi, together with the sales of “FYARRO🄬”
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 35 (Reference) Revision of Amounts of Strategic Investments (by FY2031) ◼ Allocate ¥260 billion by FY2031 increase by ¥60 billion from initial plan. Initial M&A, in-licensing R&D expenses (incl. CapEx and DX) ¥120 billion ¥80 billion ¥200 billion After revision M&A, in-licensing R&D expenses (incl. CapEx and DX) ¥90 billion ¥260 billion Expected investment amount for the next seven years M&A, in-licensing R&D expenses (incl. CapEx and DX) ¥130 billion ¥60 billion ¥190 billion ¥39 billion ¥33 billion Three-year Results • Aim in-license one pipeline per year • Expand global products ¥170 billion R&D expenses Capital expenditure (incl. DX investment) : ¥110 billion : ¥20 billion * Figures in this page are estimates.
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 36 (Reference) Revision of Materiality Business Philosophy “Three Joys” Priority (Material) Issues “Joy for patients” We strive to create and supply efficacious drugs that satisfy the needs of patients and medical professionals ❶Contributing to medical solutions • Creation of innovative new drugs that satisfy unmet medical needs • Provision of pharmaceuticals with proven health economics • Partnerships with domestic and overseas companies and others ❷Fulfilling responsibilities as a pharmaceutical company • Stable supply of high -quality pharmaceuticals with proven safety • Activities to provide accurate information • Intellectual properties strategies “Joy for society” We recognize our social responsibility as a pharmaceutical company, engage in all activities with high ethical standards, and aspire to earn society’s trust ❸Strengthening corporate governance • Strengthening relationships with stakeholders • Promotion of compliance • Risk management to ensure business continuity ❹Consideration for the environment • Proper management of waste and wastewater • Reduction of CO 2 emissions • Efficient use of water and other resources • Supply of eco -friendly crop protection products “Joy for employees” Our objective is to become a company with vitality and presence whose employees enjoy and take pride in their work ❺Creating fulfilling workplaces • Generating and maintaining employment opportunities • Work-style reform and improved productivity • Employees’ health, occupational safety and welfare • Diversity • Development of the next generation of human resources • Respect for human rights Broken down into “business” and “business foundation” groups and organized to 12 items from 19 items :S:E :G 1st X Business Philosophy "Three Joys" ❶ Creation of innovative new drugs that satisfy unmet medical needs ❷ Expansion of access to healthcare and pharmaceuticals ❸ Stable and sustainable supply of pharmaceuticals ❺ Contribution to sustainable agriculture ❻ Pursue environmental management ❼ Strengthen corporate governance ❾ Strengthen relationship with stakeholders to achieve sustainability ❿ Respect for human rights ⓬ Strengthen development of human resources Joy for employees ⓫ Create fulfilling workplaces ❹ Appropriate provision of pharmaceutical information Joy for patients Materiality Materiality related to business Joy for society Joy for patients Joy for society Joy for employees Materiality related to business foundation ❽ Promotion of compliance 2nd X Others 3rd X 3rd X
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 5 Updates on Research and Development 37
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Topics of Research and Development Development Pipelines and Progress Forecasts Sebetralstat: World's First Oral On-Demand Therapy for HAE (Hereditary Angioedema) ESK-001: An Innovative TYK2 Inhibitor for the Treatment of Psoriasis 38
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Development Pipelines and Progress Forecasts 39
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Pipelines Development Code Development Stage * Therapeutic Area Planned Indication Remarks KAR P III Dermatology Head lice In-licensed from Arbor Pharmaceuticals, LLC Product name in the US: Sklice® KP-001 P III Plastic Surgery, Pediatrics, Dermatology Refractory vascular malformations ー KC-8025 （Seladelpar） P III Gastroenterology, Hepatology, Internal Medicine primary biliary cholangitis In-licensed from CymaBay Therapeutics, Inc. CymaBay was acquired by Gilead Sciences, Inc. in February 2024 ESK-001 P III Dermatology Plaque Psoriasis In-licensed from Alumis Inc. Alumis Inc. is conducting an international Phase III clinical trial, including Japan. KP-483 P I --- Solid Tumors (immuno-oncology) Product discovered in-house KP-910 P I Neurology, Orthopedics, Pain Medicine Peripheral neuropathic pain Product discovered in-house Tildacerfont P I Pediatrics, Internal Medicine congenital adrenal hyperplasia In-licensed from Spruce Biosciences, Inc. KP-001 (US) P I Plastic Surgery, Pediatrics, Dermatology Refractory vascular malformations Consulting with FDA on PIII plan ■ In-house discovered and In-licensed Drugs * as of May 12, 2025 includes preparation stage of clinical trials 40
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Product Planned Indication Development Stage Remarks Sebetralstat Hereditary Angioedema Filed KalVista Pharmaceuticals, the licensor, is currently applying for manufacturing and marketing approval. We hold exclusive marketing rights in Japan NM26 Atopic dermatitis P II Clinical trial underway after IP transfer to J&J ■ Status of the other pipelines Generic name (domestic sales name) Planned Indication Development Stage Out-licensed to (Developing country) Efinaconazole (Clenafin) Onychomycosis Filed Almirall S.A. (Europe)；in Italy, manufacturing and marketing approval was obtained in March 2025 Efinaconazole (Clenafin) Onychomycosis P III AIM (China) Sofpironium bromide (Ecclock) Primary axillary hyperhidrosis Filed Dong-Wha Pharm Co., Ltd. (Korea) ※The medical device “Silk-Elastin Wound Healing Sheet”, for which manufacturing and marketing approval had been submitted by the licensor Sanyo Chemical Industries, Ltd., received regulatory approval on April 21, 2025. ■Status of out-licensed products overseas as of May 12, 2025 as of May 12, 2025 Pipelines 41
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 2024 2026 20272025 2028 2029 2030 2031 2032 KP-483 (PⅠ) KP-910 (P I) KP-001 (PⅢ) KC-8025(Seladelpar PⅢ） Tildacerfont (P I) KP-001 (PⅠ, 米国) 難治性脈管奇形/経口剤 Primary biliary cholangitis /oral preparation NM26-2198 JNJ-5939（P２） Efinaconazole EU (Filed) Sofpironium bromide KR (Filed) 2022 2023 KAR (PⅢ) NexoBrid: Approved in 2022 Silk-Elastin: Approved in 2025 Solid Tumors (immuno-oncology)/oral preparation Peripheral neuropathic pain/oral preparation Congenital adrenal hyperplasia/oral preparation (IP transferred) Atopic Dermatitis/antibody (injection) CN (P III) Development Pipelines and Progress Forecasts ESK-001(PⅢ） ＊ HAE/ oral preparationSebetralstat （Filed) Italy: Approved in 2025 Refractory vascular malformations/oral preparation plaque psoriasis /oral preparation Primary axillary hyperhidrosis /topical medication Onychomycosis/topical medication Onychomycosis/topical medication ＊ Based on our forecasts as of May 2025 42 Refractory vascular malformations/oral preparation Head lice/topical medication
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Sebetralstat: World's First Oral On-Demand Therapy for HAE (Hereditary Angioedema) 43
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Hereditary Angioedema (HAE): Disease Overview and Unmet Medical Needs About HAE ➢ Designated intractable disease (primary immunodeficiency syndrome) ➢ Estimated 2,500 patients in Japan (1 in 50,000) ➢ Most cases are caused by genetic abnormalities of the complement C1 inhibitor, leading to deficiency or dysfunction of C1 inhibitor in the blood. ➢ It causes recurrent edema attacks affecting the skin, gastrointestinal tract, and airways, which can be life-threatening. ➢ Symptoms typically peak within 24 hours and resolve within a few days. Kininogen Bradykinin KallikreinC1 inhibitor Unmet Medical Needs in HAE Treatment ➢ The on-demand treatment is only available as an injectable formulation. ➢ Lack of early prediction and monitoring technologies for attacks ➢ Quality of life impacts (anxiety about attacks, limitations in social and professional life) HAE Treatment ➢ On-demand treatment: C1 inhibitor products, kallikrein inhibitors (e.g., ecallantide), bradykinin receptor antagonists (e.g., icatibant) ➢ Prophylaxis: C1 inhibitor replacement therapy, kallikrein inhibitors (e.g., lanadelumab, berotralstat) Created by Kaken Pharmaceutical Co., Ltd. 44
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved About HAE (Hereditary Angioedema) •Designated intractable disease (primary immunodeficiency syndrome) in Japan; estimated 2,500 patients domestically (1 in 50,000 people). •Most cases are caused by genetic abnormalities of the complement C1 inhibitor, leading to deficiency or dysfunction of C1 inhibitor in the blood. •It causes recurrent edema attacks in areas such as the skin, gastrointestinal tract, and airways. These can sometimes lead to life-threatening symptoms such as abdominal pain or respiratory distress due to laryngeal edema. •Symptoms typically peak within 24 hours and resolve within a few days. Treatment of HAE •On-demand treatment for attacks: C1 inhibitor replacement products, kallikrein inhibitors (e.g., ecallantide), bradykinin receptor antagonists (e.g., icatibant) •Prophylactic treatment: C1 inhibitor replacement therapy, kallikrein inhibitors (e.g., lanadelumab, berotralstat) (According to the 2023 Revised Clinical Guidelines for Hereditary Angioedema) Unmet Medical Needs in HAE Treatment •On-demand treatment options are limited to injectable formulations. •Lack of early prediction and monitoring technologies for attacks. •Impact on quality of life (anxiety over attacks, limitations in social and professional life). Excerpt from KalVista Pharmaceuticals, Inc. 45
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Sebetralstat Kininogen Bradykinin KallikreinSebetralstataSebetralstat (KVD900) Phase 3 Trial: KONFIDENT Overview (NCT05259917) Objective: •To evaluate the efficacy and safety of the first oral on-demand therapy for HAE attacks Study Design: •Randomized, double-blind, crossover trial •Each patient takes a single dose of 300 mg, 600 mg, or placebo at the time of an attack Subjects: •≥12 years of age, 136 HAE patients Endpoints: •Primary: Time to symptom relief (PGI-C) •Secondary: Attack severity, time to resolution, safety Targeted Attacks: •All HAE attacks, including laryngeal and breakthrough attacks during prophylaxis ➢ Developer: KalVista Pharmaceuticals, Inc. (USA) ➢ Mechanism of Action: Inhibits plasma kallikrein to suppress excessive bradykinin production ➢ Administration: Oral, taken at the time of attack Created by Kaken Pharmaceutical Co., Ltd. Excerpt from KalVista Pharmaceuticals, Inc. 46
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Sebetralstat (KVD900) Phase 3 Trial: KONFIDENT Results Efficacy: ➢ Primary Endpoint (Time to symptom relief): •300 mg: Median 1.61 hours (95% CI: 1.28–2.27) •600 mg: Median 1.79 hours (95% CI: 1.33–2.27) •Placebo: Median 6.72 hours (95% CI: 2.33–>12) ➢ Secondary Endpoints: •Significant reduction in time to symptom severity relief compared to placebo •Significant reduction in time to complete symptom resolution compared to placebo Safety: ➢ No discontinuations due to adverse events; no serious treatment-related adverse events ➢ Incidence of side effects: 300 mg (2.3%), 600 mg (2.2%), placebo (4.8%) Clinical Significance: ➢ First oral on-demand treatment, offering a non-injectable alternative ➢ Contributes to improved patient convenience and quality of life Excerpt from KalVista Pharmaceuticals, Inc. 47
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Sebetralstat: World's First Oral On-Demand Therapy for HAE (Hereditary Angioedema) Mechanism of Action ➢ Oral drug that selectively inhibits plasma kallikrein ➢ Prevents excessive bradykinin generation by suppressing plasma kallikrein activation, thereby reducing vascular permeability, the cause of HAE attacks ➢ Expected to improve convenience as an oral alternative to injectable therapies Phase 3 KONFIDENT Trial Summary •Population: 136 patients with moderate to severe HAE •Dosing: Oral sebetralstat 300 mg or 600 mg at the time of an attack •Primary Endpoint: Time to onset of symptom relief •300 mg group: Median 1.61 hours (vs. 6.72 hours for placebo) •600 mg group: Median 1.79 hours •Secondary endpoints: Significant reduction in attack severity and time to complete resolution •Safety: Favorable profile comparable to placebo Potential ➢ As the world’s first oral on-demand therapy, sebetralstat may enable early treatment, improve quality of life, and increase access to care ➢ Rapid symptom relief may reduce anxiety during attacks ➢ Designated as an orphan drug by both the U.S. FDA and European Medicines Agency (EMA); U.S. approval expected in June 2025 ➢ Also designated as an orphan drug in Japan, with a New Drug Application (NDA) submitted in January 2025 48
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved ESK-001: An Innovative TYK2 Inhibitor for the Treatment ofPsoriasis 49
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved ESK-001 ESK-001 is a highly selective, oral, allosteric small molecule TYK2 inhibitor ➢ ESK-001 is potent and intrinsically selective for TYK2. ➢ ESK-001 allosterically inhibits TYK2, with maximal target inhibition maintained over 24 hours with 40 mg BID dose. Allosteric binding site JH2domain Kinase active site JH1domain TYK2 enzyme ESK-001 Inflammatory cytokines IL-23,IL-12,IFN ESK-001 IL-23/12 pathway IFN pathway IL-23/12/Th17 driven disease IFN driven disease ➢ Developer: Alumis Inc. (USA) ➢ Mechanism of Action: Highly selective inhibition of TYK2 (Tyrosine Kinase 2) ➢ Administration: Oral (twice daily; once-daily modified-release formulation also under development) Development Status: •Moderate to Severe Plaque Psoriasis (Phase 3 trial) •Systemic Lupus Erythematosus (SLE) (Phase 2 trial) European Academy of Dermatology & Venereology Congress, September 25-28, 2024, P1004 50
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Phase 2 Clinical Trial of ESK-001, an Oral Allosteric TYK2 Inhibitor for the Treatment of Plaque Psoriasis Secondary Endpoints ➢PROs ▪ DLQI ▪ NRS ➢Achievement of PASI-90 ➢Achievement of sPGA 0/1 Primary Endpoint ➢Achievement of PASI-75 Study Design ➢ STRIDE is a randomized, double-blinded, placebo-controlled Phase II study in adults with moderate-to-severe plaque psoriasis (PASI ≥12, sPGA ≥3, BSA ≥10%) (NCT05600036). PASI：Psoriasis Area and Severity Index DLQI：Dermatology life quality index NRS：Numerical Rating Scale sPGA：Static Physician's Global Assessment European Academy of Dermatology & Venereology Congress, September 25-28, 2024, P1004 51
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved * 0.0 19.4 100 90 80 70 60 50 40 30 *** 64.1*** *** 56.4 56.4 *** ** 33.3 Proportion of Patients with Response (%) 20 10 0 38 36 36 39 39 39 Primary endpoint at Week 12 ESK-001 inhibition of Type 1 IFN gene signature* ESK-001 reduced skin inflammatory markers to unaffected skin levels PASI-75 Blood and Skin 20 mg BID 40mg QD 40 mg BID *In blood by RNA-seq from STRIDE psoriasis study; blood sampled at baseline and pre-dose (trough) at Weeks 2 & 12. Placebo 10 mg QD 20 mg QD *p<0.05; **p< 0.005; ***p<0.001. P-value: Proportion of responders of each active arm vs placebo. Missing data was imputed based on non-responder imputation (NRI). Timepoints: Baseline nonlesional (Unaffected skin), baseline lesional (Affected skin), Week 12 lesional (ESK-001 Rx). ｎ European Academy of Dermatology & Venereology Congress, September 25-28, 2024, P1004 52
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved OLE Study: thorough dose selection to identify maximum benefit/risk for evaluation of long-term safety/efficacy of ESK-001 STRIDE Phase 2 Study Patient Screening + Treatment Assignment Open Label Extension (OLE) Study 4-week Safety Follow-up ESK-001 40 mg QD (N=39) ESK-001 40 mg BID (N=39) Double-blinded Period Placebo (N=38) ESK-001 10 mg QD (N=36) ESK-001 20 mg QD (N=36) ESK-001 20 mg BID (N=39) ESK-001 40 mg BID (N=83*) ESK-001 40 mg QD (N=82) STRIDE Ph2 Dose OLE Dose 40 mg QD 40mg BID Placebo 17 (21%) 9 (11%) 10 mg QD 30 (37%) 20 mg QD 26 (32%) 20 mg BID 30 (36%) 40 mg QD 9 (11%) 17 (21%) 40 mg BID 27 (33%) Dose Assignment from STRIDE to OLE Baseline Primary STRIDE Wk 16/ Wk Wk Wk Wk Wk Wk Wk Quarterly End of Study endpoint Day 1 OLE 2 4 8 12 16 28 52 visits Wk 12 (ongoing) Study Design ➢ OLE is an ongoing open-label extension study in patients with plaque psoriasis who have completed STRIDE study (NCT05739435) Secondary Endpoints ➢long-term efficacy of ESK-001(changes in skin and PROs) ➢clinical response ➢PK Primary Endpoint ➢Long-term safety and tolerability • 95% of eligible STRIDE patients rolled over to OLE study • Two highest dose arms from STRIDE, 40 mg QD and 40 mg BID, with QD patients switching to 40 mg BID arm after Week 28** European Academy of Dermatology & Venereology Congress, September 25-28, 2024, P1004 53
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved OLE Safety profile of ESK-001 ESK-001 40 mg QD (N=82) ESK-001 40 mg BID* (N=147) Overall (N=164) n (%) EAIR n (%) EAIR n (%) EAIR Subjects with ≥ 1 TEAE 50 (61) 122.76 73 (50) 102.33 108 (66) 108.18 Subjects with ≥ 1 TE SAE 2 (2) 3.10 4 (3) 3.39 6 (4) 3.29 Subjects with TEAE relatedto study drug 12 (15) 20.19 15 (10) 13.83 26 (16) 15.70 Subjects with SAE related to study drug 2 (2) 3.10 2 (1) 1.67 4 (2) 2.17 Subjects with TEAE leading to death 0 - 0 - 0 - Subjects with TEAE leading to study drug discontinuation 1 (1) 1.55 5 (3) 4.19 6 (4) 3.26 Subjects with TEAE ≥ Grade 3 3 (4) 4.66 6 (4) 5.12 8 (5) 4.42 Most frequent TEAEs (≥5% in any treatment group) Nasopharyngitis 10 (12) 16.88 6 (4) 5.15 14 (9) 8.09 Upper respiratory tract infection 3 (4) 4.71 13 (9) 11.66 16 (10) 9.20 Headache 5 (6) 8.28 5 (3) 4.28 10 (6) 5.71 COVID-19 3 (4) 4.74 8 (5) 6.88 11 (7) 6.17 Data are based on the safety analysis population (all treated patients). Safety data displayed are based on 06 SEP 2024 data cut of ongoing OLE study. *Includes subjects who were randomized to ESK-001 40 mg BID from the start of OLE and who switched from 40 mg QD to 40 mg BID. TEAE, treatment-emergent adverse event; SAE, serious adverse event; EAIR, exposure-adjusted incidence rate per 100 patient years. European Academy of Dermatology & Venereology Congress, September 25-28, 2024, P1004 54
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved ESK-001 response over 52 weeks of treatment: increasing PASI-90 and PASI-100 responses over time (mNRI*) 40 mg BID includes only patients originally assigned to 40 mg BID at the start of OLE 0 20 40 60 80 100 Baseline STRIDE W12 Day 1 W2 W4 W8 W28 W40 W52 Patients with Response (%) PASI-75 (40 mg QD) PASI-90 (40 mg QD) PASI-100 (40 mg QD) PASI-75 (40 mg BID) PASI-90 (40 mg BID) PASI-100 (40 mg BID) 82 82 81 79 81 81 81 80 80 - - 82 82 82 76 81 82 82 81 81 82 80 40 mg QD 40 mg BID W12 W16 25.6 4.9 1.2 19.8 6.2 1.2 67.5 45.0 18.8 53.7 24.4 11.0 46.3 23.2 6.1 82.7 63.0 30.9 77.5 38.8 61.3 WashoutSTRIDE OLE1 NUMBER OF SUBJECTS 1Patients switched from 40 mg QD to BID at Week 40 and onwards. As a result, the QD series are plotted up to Week 28 only. European Academy of Dermatology & Venereology Congress, September 25-28, 2024, P1004 55
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Patient-reported outcomes throughout Week 52 for original 40 mg BID Robust and rapid improvements in quality of life and control of itch maintained over time 58.5 61.3 0 20 40 60 80 100 Proportionof Patients (%) 73.2 81.3 0 20 40 60 80 100Proportionof Patients (%) DLQI 0/1 Average pruritus NRS score <4 STRIDE W12 (N=82) OLE W52 (N=80) STRIDE W12 (N=82) OLE W52 (N=80) DLQI, Dermatology Life Quality Index; NRS, numerical rating scale. European Academy of Dermatology & Venereology Congress, September 25-28, 2024, P1004 56
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Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved ESK-001: An Innovative TYK2 Inhibitor for the Treatment of Psoriasis Phase 2 Trial (Moderate to Severe Plaque Psoriasis): ➢ Population: Patients with moderate to severe plaque psoriasis ➢ Administration: Oral ESK-001 twice daily (multiple doses) ➢ Results: Dose-dependent increase in PASI-75 achievement rate at Week 12 -> PASI-75 achievement rate exceeded 70% in the highest dose group ➢ Response rates increased from Week 12 to Week 24, with durability in PASI response rates observed in a 52-week OLE (Open-Label Extension) study -> PASI-100 achievement rate was 39% in the OLE study ➢ Adverse Events: Just state that to date a low rate of adverse events has been observed Mechanism of Action: ➢ An oral inhibitor with intrinsic selectivity for TYK2 (Tyrosine Kinase 2) ➢ TYK2 is involved in the signaling of cytokines such as IL-23, IL-12, and Type I IFNs, which play key roles in autoimmune diseases Potential: ➢ High selectivity and efficacy ➢ Favorable safety profile ➢ Ongoing Phase 3 trials in psoriasis; potential for expansion into multiple autoimmune indications beyond psoriasis 57