Slides
Page 1
Consolidated Financial Results for the Six-Months Period of Fiscal 2025 (Six-Months Period Ended September 30, 2025) November 11, 2025 KAKEN PHARMACEUTICAL CO., LTD.
Page 2
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 “Pipeline” 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
Page 3
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Agenda 1 Consolidated Financial Results for the Six-Months Period of FY2025 2 Overview of Pipeline Overview of Consolidated Financial Results for the Six-Months Period of FY2025 Consolidated Financial Forecast for FY2025 Initiatives for Rare Diseases 2
Page 4
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Overview of Consolidated Financial Results for the Six-Months Period of FY2025 3
Page 5
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Consolidated Statements of Income FY2024 2Q FY2025 2Q Change (yen) Change (％) Net Sales 51,372 39,356 ▲ 12,016 76.6 Cost of Sales 17,269 17,776 506 102.9 Gross Profit 34,103 21,580 ▲ 12,522 63.3 Selling, General and Administrative Expenses 14,523 21,377 6,854 147.2 R&D Expenses 5,973 11,672 5,698 195.4 Operating Profit 19,579 203 ▲ 19,376 1.0 Ordinary Profit 19,823 607 ▲ 19,216 3.1 Profit before Income Taxes 19,235 1,397 ▲ 17,837 7.3 Profit 14,286 992 ▲ 13,293 7.0 (amounts are rounded down to the nearest million yen) 4
Page 6
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Net Sales Bridge FY2024 2Q 36,171 ¥ FY2025 2Q 51,372 ▲12,016 YoY change 76.6 % ✓ Pharmaceuticals and medical devices (Domestic, overseas) ->Sales decreased due to the impact of the upfront payment received for the transfer of intellectual property (IP) of NM26. ->Sales increased due to the consolidation of Aadi, which added FYARRO sales to our results. ->Sales of Ecclock-related APIs and other materials increased in the U.S. market. ->Sales of Ecclock increased in the domestic market. ✓ Crop protection products ->Sales of Polyoxin, a major products in crop protection, increased due to the overseas sales increase. Pharmaceuticals and medical devices (Domestic, overseas) Crop protection products Real estate Others (amounts are rounded down to the nearest million yen) + 921 + 345 + 51 + 45 37,992 Upfront 13,380 51,372 39,356 2Q of FY2024 Excl. receipt of upfront payment FY2024 2Q FY2025 2Q Change (yen) Change （％） Pharmaceuticals 50,150 38,082 ▲ 12,067 75.9 Pharmaceuticals (Domestic, overseas) and medical devices 48,232 35,773 ▲ 12,459 74.2 Crop Protection products 1,872 2,218 345 118.5 Others 45 90 45 199.7 Real estate 1,222 1,274 51 104.2 Total 51,372 39,356 ▲ 12,016 76.6 5
Page 7
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved ✓ Cost of sales（increased by 506 million） ・Increase associated with higher sales . (FYARRO, overseas pharmaceuticals, etc.) ✓ SG&A（excluding R&D expenses） （increased by 1,155 million） ・Increase in Aadi’s stand-alone expenses . (other SG&A) ・Integration-related expenses incurred following the acquisition. ✓ R&D Expenses (increased by 5,698 million ） ・Upfront payments relating to in-licensing, etc. (Sebetralstat, Nabenibalt, etc.) ・Increased due to progress in development in various pipelines. ・Increase in Aadi’s standalone R&D expenses. Analysis of Changes 19,579 203 +1,155 ▲ 19,376 YoY change 1.0 ％ + 1,362 + 5,698 ※excl. R&D expenses 6,199 Upfront 13,380 Operating Profit Bridge 2Q of FY2024 (Excluding receipt of upfront payment) + 506 (amounts are rounded down to the nearest million yen) Net sales Cost of sales R&D expenses SG&A expenses※ FY2024 2Q FY2025 2Q 6
Page 8
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved ✓ SG&A Expenses （excluding R&D expenses） （increased by 1,155 million ） ・Increase in Aadi’s standalone expenses. (other SG&A) ・Integration-related expenses incurred following the acquisition. ✓ R&D Expenses (increased by 5,698 million ） ・Upfront payments relating to in-licensing, etc. (Sebetralstat, Navenibart, etc. ) ・Increased due to progress in development in various pipelines. ・Increased in Aadi’s stand-alone R&D expenses. Note: Upfront payments for Silk Elastin (FY2024) and gMSC1 (FY2025) are undisclosed and included in R&D expenses. Analysis of Changes （million yen） YoY Comparison of SG&A Expenses （ 2Q FY2024 vs 2Q FY2025 ） 0 5,000 10,000 15,000 20,000 25,000 2025年3月期 2026年3月期 + 1,155 + 5,698 Consoli dated SG&A R&D Upfront payments Aadi’s standalone R&D SG&A Aadi’s standalone SG&A R&D SG&A FY2024 2Q FY2025 2Q Consoli dated R&D 7
Page 9
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Domestic Pharmaceuticals & Medical Devices Sales by Products (Non-consolidated) FY2024 2Q FY2025 2Q Change (yen) Change （％） Clenafin Group 9,172 9,313 140 101.5 Artz 9,804 9,233 ▲ 571 94.2 Seprafilm 3,567 3,519 ▲ 47 98.7 Fiblast 1,249 1,143 ▲ 106 91.5 Ecclock 1,360 1,529 168 112.4 Regroth 463 434 ▲ 29 93.7 Hernicore 205 78 ▲ 126 38.3 Generic Products (total) (excluding AG) 4,279 3,731 ▲ 548 87.2 ◆ Clenafin Group Clenafin increased despite the NHI price revision, driven by AG launch. ◆ Artz Below expectations due to the 3.8% NHI price revision and limited market growth. ◆ Seprafilm Sales increased slightly due to a switchover from competing products. ◆ Ecclock Sales increased due to disease awareness activities that expanded the market. ◆ Generic Products Sales decreased due to competition of focused products. Main factors (amounts are rounded down to the nearest million yen) 8
Page 10
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved As of Mar.31, 2025 As of Sep.30,2025 Change (yen) Total assets 190,422 177,279 ▲ 13,143 Current assets 111,377 98,757 ▲ 12,619 Non-current assets 79,045 78,521 ▲ 523 Total liabilities 37,787 29,476 ▲ 8,310 Current liabilities 30,354 22,431 ▲ 7,922 Non-current liabilities 7,433 7,045 ▲ 388 Total net assets 152,634 147,802 ▲ 4,832 Consolidated Balance Sheet As of sep. 30,2024 As of Sep. 30,2025 Change (yen) Net cash provided by (used in) operating activities 23,932 ▲ 8,064 ▲ 31,997 Net cash provided by (used in) investing activities ▲ 2,432 429 2,861 Net cash provided by (used in) financing activities ▲ 2,839 ▲ 5,225 ▲ 2,386 Cash and cash equivalents at end of period 83,987 57,105 ▲ 26,881 ◆ Net cash provided by (used in) operating activities: Interim profit before income taxes : ¥1,397 million (▲17,837 million） Income tax paid, etc. ： ▲¥6,770 million（▲6,814 million） ◆ Net cash provided by (used in) investing activities: Income from sales of investment securities ：¥1,793 million（+1,793 million） Purchase of PP&E : ▲¥863 million（+1,098 million） ◆ Net cash provided by (used in) financing activities: Net change in treasury stock： ▲¥2,342 million (▲2,343 million) Cash dividend paid： ▲¥2,882 million (▲42 million) Goodwill： ¥13,420 million ( ▲614 million) ※（）indicate changes since the end of March 2025. Accrued Corporate Tax： ¥629 million (▲6,655 million） Consolidated Balance Sheets / Consolidated Statements of Cash Flows (amounts are rounded down to the nearest million yen) Consolidated Statement of Cash Flows Cash and cash equivalents : ¥57,105 million (＋12,980 million) Capital surplus ： ¥11,406 million （▲1,525 million） Retained earnings : ¥128,090 million (▲ 7,152 million) Treasury stock : ▲¥23,775 million (＋4,458 million) ※（）indicate changes since the end of September 2024. 9
Page 11
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Long-Term Business Plan 2031 （Unit: ¥100 million） Major Cash-Outflows and Cash and Cash Equivalents Balance Trend 139 162 173 384 116 81 69 56 131 52 746 742 653 700 571 0 100 200 300 400 500 600 700 800 2022年3月 2023年3月 2024年3月 2025年3月 2026年3月期 Strategic investment Shareholder Return Cash & CE 第2四半期 Only R&D Strategic investments: R&D (incl. upfront payments), M&A, etc. Shareholder returns: Dividends, share buybacks FY2021 FY2022 FY2023 FY2024 FY2025 2Q Steady Execution of Strategic Investments and Shareholder Returns; Cash Balance Trending Downward 10
Page 12
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Consolidated Financial Forecast for FY2025 11
Page 13
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Financial Performance Forecast FY2024 Revised plan FY2025 Change (yen) Change （％） Plan FY2025 Net Sales 94,035 86,300 ▲ 7,735 91.8 88,000 Operating Profit 21,034 2,100 ▲ 18,934 10.0 5,200 Ordinary Profit 21,279 2,800 ▲ 18,479 13.2 6,000 Profit 13,945 2,300 ▲ 11,645 16.5 3,400 Reason for revision to consolidated earnings forecast The forecast for net sales has been revised downward by JPY 1,700 million from the previous forecast to JPY 86,300 million, primarily due to the impact of competing products in the domestic pharmaceutical segment. Due to the upfront payment described in the press release announced on June 2, 2025, as well as the upfront payment of USD 16 million (approximately JPY 2,400 million) under the strategic collaboration announced on August 7, 2025, and R&D expenses are expected to exceed the initial plan; therefore, profit forecasts have been revised. SG&A Expenses 37,497 47,000 9,502 125.3 44,500 R&D Expenses 18,725 25,400 6,674 135.6 20,900 ※ Announced on September 26, 2025 (amounts are rounded down to the nearest million yen) 12
Page 14
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved FY2024 Revised plan FY2025 Change (yen) Change （％） Plan FY2025 Clenafin Group 16,866 13,200 ▲ 3,366 78.6 13,200 Artz 19,031 18,100 ▲ 931 95.1 18,500 Seprafilm 6,952 7,000 ▲ 47 100.7 7,000 Fiblast 2,432 2,200 ▲ 232 90.4 2,300 Ecclock 2,037 2,400 362 117.8 2,400 Regroth 922 900 ▲ 22 97.6 1,000 Hernicore 405 300 ▲ 105 74.1 400 Generic Products (total) (excluding AG) 8,497 7,800 ▲ 697 91.8 8,900 ◆ Clenafin Group • Promote AG’s market penetration and pursue volume growth. • Continue initiatives to improve access to care for undiagnosed and diagnosed patients. ◆ Artz • Emphasize the value of hyaluronic acid to expand the market. ◆ Seprafilm • Plan to increase sales from switchover and increase usage of anti-adhesives in unused operations. ◆ Ecclock • Plan to increase sales through higher disease awareness of doctors and patients, and market expansion. Initiatives for the fiscal year Domestic Pharmaceuticals & Medical Devices Sales Forecast by Products ※ announced on Novemver 10, 2025 (amounts are rounded down to the nearest million yen) 13
Page 15
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 14 10,252 9,795 9,633 9,199 9,172 9,313 8,940 8,654 8,352 7,917 7,694 3,887 19,192 18,449 17,985 17,116 16,866 13,200 0 5,000 10,000 15,000 20,000 F Y 2 0 2 0 F Y 2 0 2 1 F Y 2 0 2 2 F Y 2 0 2 3 F Y 2 0 2 4 F Y 2 0 2 5 Clenafin group 1H 2H AG Launch in Sep. 4,394 4,245 4,014 3,544 3,567 3,519 4,261 4,188 3,776 3,451 3,385 3,481 8,655 8,433 7,790 6,995 6,952 7,000 0 2,000 4,000 6,000 8,000 10,000 F Y 2 0 2 0 F Y 2 0 2 1 F Y 2 0 2 2 F Y 2 0 2 3 F Y 2 0 2 4 F Y 2 0 2 5 Seprafilm 1H 2H 9,225 9,536 8,673 8,929 9,804 9,233 9,664 9,317 8,389 9,111 9,227 8,867 18,889 18,853 17,062 18,040 19,031 18,100 0 5,000 10,000 15,000 20,000 25,000 30,000 35,000 40,000 F Y 2 0 2 0 F Y 2 0 2 1 F Y 2 0 2 2 F Y 2 0 2 3 F Y 2 0 2 4 F Y 2 0 2 5 Artz 1H 2H 0 557 823 1,387 1,360 1,529 170 393 434 425 677 871 170 950 1,257 1,812 2,037 2,400 0 1,000 2,000 3,000 4,000 5,000 6,000 F Y 2 0 2 0 F Y 2 0 2 1 F Y 2 0 2 2 F Y 2 0 2 3 F Y 2 0 2 4 F Y 2 0 2 5 Ecclock 1H 2H （Reference）Sales Trends of Domestic Pharmaceuticals & Medical Devices Launch in Nov. （M yen） （M yen） （M yen） （M yen） (Plan) (Plan) (Plan) (Plan)
Page 16
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 15 Initiatives for Rare Diseases
Page 17
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 1st X R＆D Transformation Expansion into New Therapeutic Areas ～ Working on Rare Diseases ～ VISION PRIORITY MEASURES Expand into New Therapeutic Areas ✓Expand into new therapeutic areas where we have advantages, based on unmet medical needs Therapeutic areas where we have advantages Orthopedics Artz Hernicore Other Seprafilm Regroth Dermatology Clenafin Ecclock Fiblast New Therapeutic Areas ・Diseases with unmet medical needs at 10 years in the future ・Rare diseases 1st X R＆D Transformation Continuously launch innovative, world-class drugs in our three priority fields of drug discovery Expand pipeline • Increase the number of licensed products • Use external resources in early-stage development • Seek new indications and drug repositioning Working on Rare Diseases ⚫ Addressing Unmet Medical Needs and Contributing to Society ⚫ Maintaining and Enhancing Competitiveness New treatment options are essential for patients with rare diseases. Promote revolutionary drug development and fulfill our responsibility to deliver treatments. 16
Page 18
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Changes in the Pipeline Over Time ESK-001 FYARRO Navenibart ART-648 (discontinued development ） Tildacerfont Products In-Licensed from Eisai ND081 gMSC1 ART-001 （KP-001） KC-8025 (Seladelpar) （Merislon/ Myonal） Silk-Elastin Sebetralstat FY2021 FY2022 FY2023 FY2024 FY2025 Progress on in-licensing and marketing alliance Pipelines After the announcement the Long-Term Business Plan 2031, ten projects had been in-licensed as of September 2025. 7 8 8 9 12 (as of September 30) ：Rare diseases Long-Term Business Plan 2031 FY2021 FY2022 FY2023 FY2024 FY2025 (as of September 30) 17
Page 19
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved In-Licensed Two New Pipelines for HAE Treatment Hereditary Angioedema：HAE Sebetralstat Navenibart April FY2025 August October Establishment of a Dedicated Team for Rare Diseases ①Strength in Dermatology and Expansion into New Areas ●Often involves dermatological symptoms ⇒ Leveraging our strengths and experience in the Dermatology Area ●Handled across multiple medical specialties ⇒ Contributing to the expansion of our therapeutic areas ●Contributing to the development of other rare disease pipeline ②Track Record of Reaching to Untreated Patients ●Many patients remain untreated or undiagnosed ⇒ Promoting disease awareness through treatment for low-recognition diseases with many potential patients Background and Objectives of the Initiative Areas where Kaken can utilize our strengths • Rare genetic disorder, designated as an intractable disease in Japan • Characterized by recurrent edema attacks affecting the skin, gastrointestinal tract and airways, which can be life-threatening • Estimated 2,500 patients in Japan (approximately one in 50,000), with about 430 patients currently receiving treatment. 18
Page 20
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Sebetralstat 【Characteristics】 ・The world’s first oral on-demand therapy for HAE （previously only available as an injectable） →Potential to fulfill Unmet Medical Needs. ・Already marketed in the U.S. and other countries. （product name：Ekterly®） The upfront payment：$11 million Development Stage：Application for approval in Japan. Navenibart 【Characteristics】 ・Dosing every 3 or 6 months （Currently at most once a month in domestic ） →A reduction in patient visits can be expected. ・Fewer side effects, such as injection site pain. The upfront payment：$16 million Development Stage：Global phase 3 trials ongoing, including in Japan. Create synergy Dual approach to prevention and treatment for HAE patients Unmet Medical Needs in HAE On-demand therapy Length of dosing interval Unmet Medical Needs Long-term preventive therapy Limited options for administration 19
Page 21
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved SIGHTLINE Japan Awards 2025 – Double Award Winner 『Financing Deal of the Year』Finalist This award recognizes successful and creative financing achievements by pharma and biotech companies in Japan. Kaken was selected as a finalist for this award in recognition of the share transfer agreement concluded in FY2024 to acquire Aadi Subsidiary (now Aadi Biosciences, Inc.), through which Aadi became a wholly owned subsidiary of Kaken. 『Pharma Company of the Year』 This award is presented to pharmaceutical companies that have demonstrated outstanding achievement in Japan during the qualifying period. The following initiatives were highly recognized, leading to Kaken’s receipt of this award. • Collaboration for multi-specific antibody "ND081" for the treatment of inflammatory bowel disease • Out-licensing of the STAT6 program • In-licensing of the TYK2 inhibitor "ESK-001". • Completion of acquisition of Aadi Bioscience, Inc.(U.S.) • Out-licensing of the STAT6 program • In-licensing of "Sebetralstat" , a treatment for hereditary angioedema 『Licensing Deal of the Year』 This award recognizes licensing agreements in which a specific drug, project, or portfolio of R&D assets transferred from one company to another for further development and/or commercialization. In December 2024, Kaken entered into a license agreement with Johnson & Johnson for the STAT6 program, including the STAT6 inhibitor (Kaken’s development code: "KP-723") that Kaken has been developing as a next-generation oral treatment for Type 2 inflammatory diseases (such as atopic dermatitis and asthma). This agreement represents the largest deal in Kaken’s history since its founding in 1948. This major out-licensing deal was highly recognized, leading to this award. ＊About Citeline Japan Awards : Citeline Japan Awards was established by Citeline (UK) as the Japanese edition of the well-known "Scrip Awards" from the UK. It recognizes companies and organizations that have made significant contributions in areas such as research and development (R&D), partnerships, business strategy, and social impact in the global life sciences field. 20
Page 22
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 2 Overview of Pipeline 21
Page 23
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Agenda Pipeline Progress Forecast Highlights KP-001 KAR／KC-8025 gMSC1／NM26 22
Page 24
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Pipeline Progress Forecast 23
Page 25
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Pipelines ■In-house discovered and in-licensed projects Development code Developm entStage * Therapeutic Area Planned Indication Remarks KAR PIII Dermatology, Pediatrics Head lice In-licensed from Arbor Pharmaceuticals, LLC Product name in the US: Sklice® KP-001 PIII Pediatrics, Plastic Surgery, Hematology/Oncology, Dermatology Refractory vascular malformations Product discovered in-house Announced the topline data from the confirmatory trial KC-8025 (Seladelpar) PIII Gastroenterology, Hepatology, Internal Medicine Primary biliary cholangitis In-licensed from CymaBay Therapeutics, Inc.(Currently, Gilead Sciences, Inc.) Product name in US, UK︓Livdelzi® Product name in EU : Lyvdelzi® ESK-001 PIII Dermatology Plaque psoriasis In-licensed from Alumis Inc. Under global P III (incl. Japan) Navenibart PIII Allergy, Dermatology, Internal Medicine, ENT Hereditary angioedema In-licensed from Astria Therapeutics, Inc. Under global P III (incl. Japan) KP-483 PI — Solid tumors (Immuno-oncology) Product discovered in-house KP-910 PI Orthopedics, Pain Clinic, Diabetology Peripheral neuropathic pain Product discovered in-house Tildacerfont PI Pediatrics, Internal Medicine Congenital adrenal hyperplasia In-licensed from Spruce Biosciences, Inc. Strategy under Cconsideration KP-001 (US) PIII Same as above Refractory vascular malformations In-house discovery Preparing for P III trial Note: Kaken will advance KP-723, an oral STAT6 inhibitor in preclinical development, to the completion of Phase I clinical trials, which had entered into a licence agreement with J&J. As of November 10, 2025 (*) The development stage includes the preparatory period for clinical trials. 24
Page 26
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Other Development Programs Development code Planned Indication Development Stage Remarks Sebetralstat Hereditary angioedema Authorization application ・Applied by KalVista Pharmaceuticals, Inc. ・Obtained approval in the US and Europe （Product name︓Ekterly®） ・Exclusive sales rights in Japan gMSC1 Knee osteoarthrits with focal defect Preparing for P III ・Allogenic synovial mesenchymal stem cell-derived three-dimensional artificial tissue ・Development by the licensor, TWOCELLS Co., Ltd. ・Joint development and exclusive sales rights in the area of orthopedics in Japan NM26 Atopic dermatitis PⅡ ・Clinical trial underway by J&J after IP transfer ・Option rights to negotiate a sales partnership agreement in Japan ■Other As of November 10, 2025 Generic name (domestic sales name） Planned indication Out-licensed Developing country Efinaconazole (Clenafin) Onychomycosis AIM China : Phase III Efinaconazole (Clenafin) Onychomycosis Almirall S.A. Germany : Approved in August 2025 Italy : Approved in March 2025 Sofpironium Bromide (Ecclock) Primary axillary Hyper hidrosis Dong-Wha Pharm Co., Ltd. Korea : Approved in August 2025 ■Status of Out-Licensed Products Overseas 25
Page 27
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Pipeline Status (In-house Discovered & In-licensed Projects) PⅠ PⅡ PⅢ Application Submitted Development Phase KP-001 (Refractory vascular malformations) In-house discovered KP-001 ( Refractory vascular malformations) KP-483 (Solid tumors) KP-910 (Peripheral neuropathic pain) In-licensed products KAR (Head lice) KC-8025 (Primary biliary cholangitis) ESK-001 (Plaque psoriasis) Tildacerfont (Congenital adrenal hyperplasia) Navenibart (Hereditary angioedema) In-licensed in August KP-723 is in pre-Phase I. Announced the topline data from the confirmatory trial Preparing for P III 26
Page 28
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Pipeline Status (Other Development Products) Sebetralstat (Hereditary angioedema) Partner- developed Products gMSC1 (Knee osteoarthritis with focal defect) NM26 (Atopic dermatitis) Out- licensed Products (Overseas) Preparing for P III Efinaconazole (Clenafin) Onychomycosis Approved in Italy and Germany in 2025 AIM China EU Dong-Wha Korea Sofpironium bromide (Ecclock) Primary axillary hyperhidrosis Approved in August 2025 Efinaconazole (Clenafin) Onychomycosis PⅠ PⅡ PⅢ Application Submitted Development Phase TWOCELLS COMPANY, LIMITED 27
Page 29
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved 2024 2025 2026 2027 2028 2029 2030 2031 2032 KAR KP-001 KC-8025 ESK-001 Navenibart KP-001 KP-483 KP-910 Tildacerfont Head lice 2024 2025 2026 2027 2028 2029 2030 2031 2032 Sebetralstat gMSC1 NM26 Refractory vascular malformations Primary biliary cholangitis Solid tumors (Immuno-oncology) Peripheral neuropathic pain Congenital adrenal hyperplasia Refractory vascular malformations Knee osteoarthritis with focal defect Atopic dermatitis Hereditary angioedema Pipeline Progress Forecast Application Submitted PⅢ PⅡ PⅠ Plaque psoriasis Hereditary angioedema ※ Considering Out-licensing Going Forward ※ Strategy Under Consideration （CY） 28
Page 30
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Development code Indication 2025 2026 2027 H1 H2 H1 H2 H1 H2 KAR Head lice Phase III Topline Data Disclosure KP-001 Refractory vascular malformations Phase III Top- line Data Disclosure KC-8025 Primary biliary cholangitis Phase Ⅲ Completion ESK-001 Plaque psoriasis Phase III Topline Data Disclosure (PsO) Phase Ⅱb Topline Data Disclosure （SLE） Navenibart Hereditary angioedema Phase III Topline Data Disclosure KP-483 Solid tumors (Immuno-oncology) Phase I Completion KP-910 Peripheral neuropathic pain Phase I Completion Tildacerfont Congenital adrenal hyperplasia Phase I Completion KP-001 Refractory vascular malformations Preparing for Phase III Pipeline Progress Forecast – Planned Disclosure of Upcoming Milestones ※ Data Analysis Underway for the Next Phase ※ Strategy Under Consideration Preparing for PhaseⅠKP-723 Type 2 Inflammatory Diseases ※ Preparing for P III trial 29
Page 31
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Development code Indication 2025 2026 2027 H1 H2 H1 H2 H1 H2 Sebetralstat Hereditary angioedema Marketing Approval Granted gMSC1 Knee osteoarthritis with focal defect Preparing for P III NM26 Atopic dermatitis Phase Ⅱ Completion (Projection) Status of Other Development Products (Upcoming Milestones) Generic name Indication Out- licensed 2025 2026 2027 H1 H2 H1 H2 H1 H2 Efinaconazole (Clenafin) Onychomycosis Almirall S.A. Marketing Approval Granted Efinaconazole Onychomycosis AIM Sofpironium Bromide Primary axillary hyperhidrosis Dong-Wha Marketing Approval Granted ※ Phase III Trial Currently Ongoing. Detailed Completion Timeline Not Disclosed Status of Overseas Out-licensed Products (Upcoming Milestones) 30
Page 32
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Highlights 31
Page 33
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Highlights – KP-001 ◼ U.S. Phase III Trial – Advancement to Phase III Stage ・Planning documents to be submitted to the FDA promptly in preparation for trial initiation ・Trial expected to begin within the current fiscal year ◼ JP Phase III Trial – Completion and Topline Data Acquisition (Long-term Dosing Trial Ongoing) Target Patients Treatment-resistant vascular malformations (venous malformations, lymphatic malformations, Klippel–Trénaunay syndrome) Design Open-label, non-controlled, multi-center study Primary Endpoint Response rate of target lesions based on MRI images at the final evaluation point after 24 weeks of administration * *Percentage of subjects whose target lesion volume decreased by 20% or more compared to pre-treatment Result: Primary endpoint achieved The response rate significantly exceeded the pre-specified rate based on the natural history of untreated patients. Safety evaluation Result: No adverse events were observed that would pose a development concern. Trial Overview Regulatory submission targeted for FY2026, driven by these positive outcomes. 32
Page 34
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Highlights ～KAR・ KC-8025～ ◼ KAR Phase III Trial – Completed, Topline Data to Be Announced ◼ KC-8025 Phase III Trial – Enrollment Completed Target Patients Head lice infestation with insufficient response to existing treatments Design Placebo-controlled, randomized, double-blind, parallel-group comparison Primary Endpoint Treatment success in index subjects* (maintenance of a state with no live head lice) *Index subjects were pre-identified within each household for evaluation purposes Trial Overview Results expected to be announced within this year Target Patients Primary biliary cholangitis (PBC) with inadequate response or intolerance to ursodeoxycholic acid Design Open-label, non-controlled, multi-center study Primary Endpoint Achievement of composite endpoint at Week 52 post-treatment (based on clinical laboratory values**) **Alkaline phosphatase (ALP) < 1.67 × upper limit of normal, ≥15% reduction from baseline ALP , and total bilirubin ≤ upper limit of normal Trial Overview Confirmatory study expected to conclude in FY2026 33
Page 35
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved Highlights –gMSC1・ND081 ◼ gMSC1 – Joint Development Initiated with Two Cells Co., Ltd. ・Three-dimensional artificial tissue derived from allogeneic (donor) synovial mesenchymal stem cells, created by Two Cells ・Being developed as a regenerative medicine product for cell therapy to repair damaged cartilage ◆ Allogeneic cell-based product expected to enable stable supply ◆ Easy to handle during surgery, allowing simple and efficient procedures ◆ Two Cells has conducted a Phase III trial targeting “traumatic cartilage injury and osteochondritis dissecans” ◆ gMSC judged to have high potential ⇒ Plan to conduct a Phase III trial targeting “knee osteoarthritis with focal defect” Key Features ◼ ND081 – Advancing Toward Joint Development The novel multi-specific antibody drug candidate ND081, currently under joint research with Numab Therapeutics AG for inflammatory bowel disease, is aiming for early progression into the development stage. 34
Page 36
Copyright © KAKEN PHARMACEUTICAL CO., LTD. ALL Rights Reserved R&D Briefing Session – December 10 (Wed) 13:30–14:30 (Tentative) 35