Slide 1: FY2025 Q2 Financial Results Materials
FY2025 Q2 Financial Results Materials August 2025
Slide 2: Forward-looking statements disclaimer
◆ This document contains forward-looking statements, including our current outlook, forecasts, goals, and plans. These statements are based on information currently available and the judgment of our management. Therefore, actual results may differ significantly from these forecasts due to various ri
Slide 3: Overview of FY2025 Q2 Financial Results; FY2025 Q2 Topics
１ Overview of FY2025 Q2 Financial Results ２ FY2025 Q2 Topics ① HGF Gene Therapy Product ② NF-κB Decoy Oligo DNA ③ Tie2 Receptor Agonist AV-001 ④ Zokinvy ⑤ ACRL ⑥ EmendoBio Genome Editing Technology 3
Slide 4: Overview of FY2025 Q2 Financial Results
01 Overview of FY2025 Q2 Financial Results
Slide 5: FY2025 Q2 Consolidated Financial Results
FY2025 Q2 Consolidated Financial Results （Million Yen） Increase FY2024/2Q FY2025/2Q /Decrease Revenues 347 414 67 Business 5,455 2,815 △2,640 Expenses Operating △5,107 △2,400 2,707 Profit Non-operating 1,917 △1,498 △3,415 income/expenses Ordinary △3,190 △3,898 △708 Profit Extraordinary △227 0 227 in
Slide 6: Breakdown of Business Expenses
Breakdown of Business Expenses Business Business Expenses Expenses （million Yen） 5,455 2,815 6,000 199 2,245 Cost of sales: 254 million yen （YoY +55million yen） Increase in contracted testing service 162M yen (YoY+70M yen） Cost of goods sold by Zokinvy 92M yen 3,000 3,796百万円 254 10,783百万円 （YoY△7M ye
Slide 7: Consolidated Balance Sheet Highlights
Consolidated Balance Sheet Highlights (million yen) Increase FY2024 FY2025/2Q /Decrease Current Assets ■ Cash and deposits: 2,908 M yen Current assets 3,542 4,728 1,186 (up 1,200 M yen from FY2024) → 3,764 M yen raised through the 45th series of stock acquisition rights ■ Merchandise: 179 M yen Cash
Slide 8: Factors Affecting Net Profit/Loss for the Period
Factors Affecting Net Profit/Loss for the Period FY2025/2Q Results Factors for Increase/Decrease (YoY) million yen △3,500 △3,966 ・SG&A expenses decreased due to the elimination of goodwill amortization ・Non-operating loss was significantly impacted by foreign exchange effects 8
Slide 9: Performance Trends
Performance Trends Business Revenue Operating Profit/Loss million yen million yen 414 △2,400 FY2023-1H FY2023-2H FY2024-1H FY2024-2H FY2025-1H Operating revenue increased Operating profit/loss improved ・Increase in commissioned expanded newborn ・Cost reductions due to Emendo’s business screening tes
Slide 10: FY2025 Q2 Topics
02 FY2025 Q2 Topics
Slide 11: Status of Our Development Projects
Status of Our Development Projects Non- Clinical trials Licensing Formulati Basic Application Project Region /Partner Indication clinical Approval on Research trials Review Phase I Phase II Phase III Under consideration Chronic arterial while monitoring Japan － Injectable occlusion progress in the U
Slide 12: FY2025 Q2 Topics
FY2025 Q2 Topics 新型コロナウイルス DNAワクチン／治療薬の開発開 １ Progress of HGF Gene Therapy Product 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ２ Progress of NF-κB Decoy Oligo DNA 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ３ Progress of Tie2 Receptor Agonist 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ４ Sales of Zokinvy 始 新型コロナウイルス DNAワクチン／治療薬の開発開 5 Commissioned
Slide 13: FY2025 Q2 Topics
FY2025 Q2 Topics 新型コロナウイルス DNAワクチン／治療薬の開発開 １ Progress of HGF Gene Therapy Product 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ２ Progress of NF-κB Decoy Oligo DNA 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ３ Progress of Tie2 Receptor Agonist 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ４ Sales of Zokinvy 始 新型コロナウイルス DNAワクチン／治療薬の開発開 5 Commissioned
Slide 14: Development and Commercial Strategy in the U.S.
Development and Commercial Strategy in the U.S. The HGF gene therapy product has completed clinical trials in the U.S. and is now preparing for Biologics License Application (BLA) submission, aiming for early approval General process for application and approval Before Phase1 Phase2 Phase3 Applicati
Slide 15: Review of HGF Gene Therapy Product Development
Review of HGF Gene Therapy Product Development ＜Japan＞ ＜United States＞ November 2014 2014年 2014～2016 The conditional and time-limited Global Phase 3 Trial approval system was implemented Targeting patients at high risk of limb amputation in January 2018 15 countries (U.S., Europe, South America), bu
Slide 16: Scope of diseases in clinical trials in Japan and the U.S.
Scope of diseases in clinical trials in Japan and the U.S. Based on advice from the lead investigator that “it is important to treat patients before the condition becomes severe,” the U.S. Phase 2b clinical trial targeted patients with mild to moderate CLTI (Chronic Limb-Threatening Ischemia) Chroni
Slide 17: FY2025 Q2 Topics
FY2025 Q2 Topics 新型コロナウイルス DNAワクチン／治療薬の開発開 １ Progress of HGF Gene Therapy Product 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ２ Progress of NF-κB Decoy Oligo DNA 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ３ Progress of Tie2 Receptor Agonist 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ４ Sales of Zokinvy 始 新型コロナウイルス DNAワクチン／治療薬の開発開 5 Commissioned
Slide 18: NF-κB Decoy Oligo DNA
NF-κB Decoy Oligo DNA In 2025, a paper was published in The SPINE JOURNAL, issued by the North American Spine Society (NASS), detailing the results of a Phase 1 clinical trial conducted in the U.S. since February 2018 targeting patients with discogenic low back pain 【Results of the Phase 1b Clinical
Slide 19: FY2025 Q2 Topics
FY2025 Q2 Topics 新型コロナウイルス DNAワクチン／治療薬の開発開 １ Progress of HGF Gene Therapy Product 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ２ Progress of NF-κB Decoy Oligo DNA 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ３ Progress of Tie2 Receptor Agonist 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ４ Sales of Zokinvy 始 新型コロナウイルス DNAワクチン／治療薬の開発開 5 Commissioned
Slide 20: Tie2 Receptor Agonist (AV-001)
Tie2 Receptor Agonist (AV-001) As a treatment for acute respiratory distress syndrome (ARDS) Designated as Fast Track by the US FDA What is Fast Track? A system that prioritizes the review of drugs for serious diseases or unmet medical needs To deliver promising drugs to patients more quickly Benefi
Slide 21: FY2025 Q2 Topics
FY2025 Q2 Topics 新型コロナウイルス DNAワクチン／治療薬の開発開 １ Progress of HGF Gene Therapy Product 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ２ Progress of NF-κB Decoy Oligo DNA 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ３ Progress of Tie2 Receptor Agonist 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ４ Sales of Zokinvy 始 新型コロナウイルス DNAワクチン／治療薬の開発開 5 Commissioned
Slide 22: About Zokinvy
About Zokinvy Sales began on May 27, 2024 Application for Exclusive distribution manufacturing and agreement with Eiger marketing approval of Sales launched in Japan BioPharmaceuticals for Japan 'Zokinvy' in Japan 2022 May 2023 March May 2024 January May Manufacturing Orphan drug Orphan drug and mar
Slide 23: FY2025 Q2 Topics
FY2025 Q2 Topics 新型コロナウイルス DNAワクチン／治療薬の開発開 １ Progress of HGF Gene Therapy Product 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ２ Progress of NF-κB Decoy Oligo DNA 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ３ Progress of Tie2 Receptor Agonist 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ４ Sales of Zokinvy 始 新型コロナウイルス DNAワクチン／治療薬の開発開 5 Commissioned
Slide 24: Status of Expanded Newborn Screening at ACRL
Status of Expanded Newborn Screening at ACRL The number of expanded newborn screening tests continues to increase In FY2024, approximately 50,000 tests were commissioned In FY2025, the number of commissioned tests is expected to double 120,000 110,000 FY2025 100,000 90,000 80,000 FY2024 70,000 50,00
Slide 25: Biomarker testing services are being prepared
Biomarker testing services are being prepared Rare genetic disease testing Genetic testing Biomarker testing Screening test （Definitive test） （Monitoring of treatment efficacy） Test to check whether If the screening test suggests the If the screening test suggests the newborns have a possibility pos
Slide 26: One-Stop Testing Services for Rare Genetic Diseases
One-Stop Testing Services for Rare Genetic Diseases At ACRL, we aim to build a system that can provide comprehensive testing services for rare genetic diseases. Present Medical institution Hygiene Inspection Center A Hygiene Inspection Center B Hygiene Inspection Center C To examine the presence or
Slide 27: FY2025 Q2 Topics
FY2025 Q2 Topics 新型コロナウイルス DNAワクチン／治療薬の開発開 １ Progress of HGF Gene Therapy Product 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ２ Progress of NF-κB Decoy Oligo DNA 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ３ Progress of Tie2 Receptor Agonist 始 新型コロナウイルス DNAワクチン／治療薬の開発開 ４ Sales of Zokinvy 始 新型コロナウイルス DNAワクチン／治療薬の開発開 5 Commissioned
Slide 28: Joint research with Stanford University
Joint research with Stanford University Using the OMNI nuclease developed by EmendoBio Launch of joint research and development of a novel cancer therapy with Stanford University EmendoBio Stanford University More precise than conventional Cas9 Technology for delivering drugs OMNI nuclease enables m
Slide 29: Leading Global in Gene Medicine
Leading Global in Gene Medicine AnGes, Inc. https://www.anges.co.jp/en