Nxera Pharma Co., Ltd. · TSE:4565
Nxera Pharma — Investor Presentation (April 2026)
53 slides from Nxera Pharma's investor presentation, April 2026
Slide 1: Corporate Presentation
EXTERNAL NON-CONFIDENTIAL Corporate Presentation April 2026 | Nxera Pharma Co., Ltd. (TSE: 4565)
Slide 2: Disclaimer
The material that follows is a presentation of general background information about Nxera Pharma Co., Ltd and its subsidiaries (collectively, the “Company”) as of the date of this Disclaimer presentation. This material has been prepared solely for informational purposes and is not to be construed as
Slide 3: Agenda
Agenda 01 Business Overview 02 Strategic Roadmap 03 Japan/APAC Business 04 Our NxWave Platform 05 Financial Results 06 Appendix 3
Slide 4: Business Overview
Business Overview 01
Slide 5: Not a traditional Japanese pharma. We think and innovate globally, and specialize locally
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Not a traditional Japanese pharma. We think and innovate globally, and specialize locally Drug Discovery Platform Commercial CEO Research Finance Chief of Staff Legal Finance Operation Compliance Research & Early Clinical • Cryo-EM Nobel Prize winning fo
Slide 6: Nxera Pharma History
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Nxera Pharma History Since 1990: Growth Powered by Strategic Acquisitions JAPAN KOREA Sosei Group founded in Japan Acquisition of Arakis Commercial Acquisition of Idorsia Japan/Korea 1990 2004 2005 2015 2023 2024 Acquisition of Heptares Listed in Tokyo S
Slide 7: Major Pipeline Overview
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Major Pipeline Overview Discovery – Preclinical Phase1 Phase2 Phase3 Filed Commercial Vamorolone PIVLAZ® Duchenne Muscular Dystrophy Cerebral vasospasm Commercial Lucerastat QUVIVIQTM Fabry disease Insomnia Develop programs in-house up to a IN-HOUSE Best
Slide 8: Commercial Business
Commercial Business Commercial Two marketed products continue to grow, with a new product launch in early 2026 to support the next phase Vamorolone Sales Market Share North America (Catalyst) Europe (Santhera) (JPY million) +5% (JPY million) 547 (mUSD) +7% +30% 16,000 +14% 120% 13,800-14,200 +47%* 1
Slide 9: Platform Business
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Platform Business Platform Partner-led development progressing well; strong phase 1 readouts for in-house programs GPR52 ago | EP4 ago EP4 antag Muscarinic Agonist Portfolio Orexin Agonist Portfolio Licensing activities in progress Under development in-h
Slide 10: Financial Results/Strategic Roadmap
Financial Results/Strategic Roadmap 02
Slide 11: Key financial results
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Key financial results While the commercial business grew, milestones decreased year-on-year, resulting in a core operating loss (JPY million) Progress in 2025 28,835 29,615 30,000 2,293 2,329 ◼ Multiple milestone receipts 1,392 from Neurocrine, Centessa,
Slide 12: Breakdown of 2025 results
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Breakdown of 2025 results Strong profitability growth in the commercial business; accelerated clinical-trial investment in the platform business Consolidated Consolidated (JPY million) Platform*1 ＋ Commercial*2 = P&L (Core) ＋Non-core costs = P&L (IFRS) （
Slide 13: Priority objectives for FY2026
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Priority objectives JPY 19.5 billion+ Net product sales for FY2026 01 (PIVLAZ® plus QUVIVIQ®) Commercial Get one or more late-stage assets 02 for Japan and APAC (excl. China) Commercial 03 Sign one or more high-value partnership deals Platform 04 Initiat
Slide 14: Breakdown of 2026 guidance
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Breakdown of 2026 guidance (Without significant upfront from BD activity) Commercial business profitability has grown significantly. Platform business has reached breakeven on a core basis Consolidated Consolidated (JPY million) Platform*1 ＋ Commercial*2
Slide 15: Looking ahead to potential catalysts in 2026
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Looking ahead to potential catalysts in 2026* ✓ : Progress in 2026 PROGRAM PARTNER TIMING EVENT ✓ ORX142 (OX2 agonist) Feb 2026 Phase 2 study start ✓ ORX489 (OX2 agonist) Mar 2026 Phase 1 study start ORX750 (OX2 agonist) Q1 2026 Phase 2a data across NT1,
Slide 16: Our 2030 vision is to build a high growth, highly profitable Japanese biopharma
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Our 2030 vision is to build a high growth, highly profitable Japanese biopharma JPY Bn Royalties from WAVE1 + WAVE2 create step-change WAVE2 WAVE2 Royalty Royalty Potential royalty from next generation Only 1-2 launches from seven pipelines. WAVE1 drives
Slide 17: Japan/APAC Business
Japan/APAC Business Deliver innovation to patients in Japan/APAC 03
Slide 18: Japan will serve as our base to expand across APAC markets
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Japan will serve as our base to expand across APAC markets Commercial Japan is an attractive, established market with strong volumes Japan is the third largest Favourable JP market APAC is the second highest pharma market (ex-China) environment growth ph
Slide 19: PIVLAZ (clazosentan, an endothelin A antagonist)
PIVLAZ® (clazosentan, an endothelin A antagonist) Commercial Our first commercially available product for the prevention of cerebral vasospasm in patients with Aneurysmal Subarachnoid Haemorrhage (aSAH) PIVLAZ® sales growth 2025 PIVLAZ® highlights (million JPY) Net Sales Market Share(patient base) ✓
Slide 20: QUVIVIQ (daridorexant, dual orexin antagonist DORA)
QUVIVIQ® (daridorexant, dual orexin antagonist “DORA”) Commercial DORA is rapidly establishing its position in the treatment paradigm for insomnia Domestic Market Size for DORA QUVIVIQ® Annual Sales and Growth Rate (Bn JPY) 90 45% Daridorexant (Quviviq) 83Bn Suvorexant (Belsomra) 5.0 – 6.0 Bn JPY 79
Slide 21: QUVIVIQ Business structure
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX QUVIVIQ® Business structure Commercial Royalty profits initiated and supply margin expected in a few years Sales structure Profit structure for Nxera Supply chain optimization Product net sales Comprehensive strategy to optimize the end-to-end supply Roy
Slide 22: In-licensing of vamorolone (AGAMREE) for DMD
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX In-licensing of vamorolone (AGAMREE®) for DMD Commercial There is no established therapy for DMD other than corticosteroids in Japan Vamorolone (AGAMREE®) Duchenne Muscular Dystrophy (DMD) • First-in-class drug candidate that binds • DMD is a rare and li
Slide 23: Vamorolone (AGAMREE) addresses the need for a tolerable steroid
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Vamorolone (AGAMREE®) addresses the need for a tolerable steroid Commercial Compared with conventional corticosteroid therapy, the risk of treatment-related adverse events is reduced • Vamorolone confronts the limitations of standard Consensus sales fore
Slide 24: Synergy with Pivlaz
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Synergy with Pivlaz Commercial DMD treatment is concentrated in a limited number of centers and there is approximately 70% commercial overlap with PIVLAZ, creating significant sales synergies Proportion of prescription volume by hospital Pivlaz® target U
Slide 25: Our NxWave Platform
Our NxWave Platform Cutting-edge Science 04
Slide 26: Renewed R&D focus where the science is strongest and the opportunity is greatest
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Renewed R&D focus where the science is strongest and the opportunity is greatest Platform IN-HOUSE PORTFOLIO - R&D FOCUS AND PROGRAM PRIORITISATION Discovery Phase 2 ready Phase 2 NXE’149 NXE’744 NXE’732 Discovery candidates GPR52 agonist EP4 agonist EP4
Slide 27: NXE'744: EP4 agonist for IBD – target engagement & Phase 2 ready
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX NXE’744: EP4 agonist for IBD* – target engagement & Phase 2 ready Platform A first-in-class GI-targeted agent to promote mucosal healing in IBD EP4 AGONIST OFFERS A DIFFERENTIATED MOA TO CURRENT SOC: • All elements of the first-in-human study have now Mo
Slide 28: NXE'149: GPR52 agonist for schizophrenia – Phase 2 ready
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX NXE’149: GPR52 agonist for schizophrenia – Phase 2 ready Platform A novel first-in-class mechanism to treat positive, negative & cognitive domains of schizophrenia Phase 1 highlights: Phase 2 enablement: ✓ Safe and well tolerated • 3 month GLP toxicology
Slide 29: NXE'732: EP4 antagonist is our novel immunotherapy for solid tumors
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX NXE’732: EP4 antagonist is our novel immunotherapy for solid tumors Platform Phase 2a expansion in process in combination with atezolizumab Disease Rationale Phase 1 trial results • When EP4 is activated, it • The emerging data for NXE-732 points to a po
Slide 30: Leveraging our GPCR expertise: next-generation small molecules for obesity
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX We can make a huge impact by leveraging our GPCR expertise in the areas of highest unmet Platform medical need: next-generation small molecules for obesity, metabolic and endocrine disorders Unparalleled GPCR SBDD capabilities • Launched broad new pipeli
Slide 31: Hundreds of millions of dollars received, billions of dollars in potential to come
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX … hundreds of millions of dollars received, billions of dollars in potential to come Platform Upfront and Initial Potential Total Partner Execution Program Therapeutic Area(s) Milestones Milestone1 Collaboration and exclusive option-to- March 2024 Schizo
Slide 32: Neurocrine is advancing the world's most comprehensive portfolio of muscarinic agonists
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Neurocrine is advancing the world’s most comprehensive portfolio of Platform muscarinic agonists to treat neuropsychiatric disorders Stage of Program Mechanism Disease State Development Schizophrenia Phase 3 Direclidine M4 Agonist Bipolar Mania Phase 2 D
Slide 33: Muscarinic program development
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Muscarinic program development. Platform Ph3 ongoing for our product NBI'568, which aims to be best-in-class, owing to its predecessor Cobenfy First clinical evidence for benefit of Positive KarXT Phase2 clinical Cerevel M4 PAM Phase1b Positive KarXT Pha
Slide 34: Topline Results for Phase 2 Trial of M4 Agonist
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Topline Results for Phase 2 Trial of M4 Agonist Platform Efficacy confirmed at 20 mg. Statistically significant difference in both PANSS and CGI-S compared to placebo. “The effects with the 20-milligram dose, both PANSS and CGI-S scores consistently show
Slide 35: Comparison of Study Sites and Duration with Known Muscarinic Programs
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Comparison of Study Sites and Duration with Known Muscarinic Programs Platform Mentioned in a presentation Phase 3 of NBI-568 will be one to one randomization and around 20 sites per study Neurocrine/Nxera Neurocrine/Nxera BMS/Karuna AbbVie/Cerevel Compo
Slide 36: Placebo effect
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Data comparison of placebo effects (Total PANSS) Platform Large number of study sites in a clinical trial of muscarinic program may be linked to a higher placebo response. 20 Emraclidine (Empower-3) 15 Emracridine (Empower-2) KarXT (Emergent-3) Placebo e
Slide 37: Safety: Adverse Events Risk
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Safety: Adverse Events Risk Platform The gastrointestinal and cardiovascular adverse events were higher than placebo in Cobenfy, but not with NBI-568 Safety Dietary Number of Gastrointestinal Cardiovascular Restriction doses Others (M2) (M3) NBI- 568 Sim
Slide 38: Centessa is advancing ORX750, a potential best-in-class Orexin Receptor 2 agonist
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Centessa is advancing ORX750, a potential best-in-class Orexin Receptor Platorm 2 agonist for treatment of NT1, NT2 and IH Potential BIC for NT1, NT2 and IH Phase 2a study update Endpoints Maintenance of >20 min change at 1.5mg vs baseline (with half of
Slide 39: Data on OX2 agonist competitors
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Data on OX2 agonist competitors Platform ORX750 reported favorable safety and efficacy results in Phase 1b trials TAK-861 ALKS2680 ORX750 *MWT (mins) % Side Effects *MWT (mins) % Side Effects *MWT (mins) % Side Effects 40 100 40 100 40 37.9 100 Effect TE
Slide 40: NxWave: Proprietary structure-based drug design delivering proven pipeline impact
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX NxWave : Proprietary structure-based drug design delivering proven pipeline impact Platform Target ID and NxStaR NxHit NxDesign Translational Med. Validation Identifying the Stabilising the Identifying the Selecting the Testing the best targets right tar
Slide 41: Number of structures solved and deposited in PDB, resolution by technology
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Number of structures solved and deposited in PDB, resolution by technology Platform The number of structures solved using Cryo-EM is increasing, X-ray crystallography has extremely high resolution Number of structures solved by technology Resolution by t
Slide 42: Our platform enables precise design of GPCR models
OVERVIEW FINANCIALS JP/APAC PLATFORM APPENDIX Our platform enables precise design of GPCR models Platform Only by performing detailed structural analysis can we design great drugs. Imprecise GPCR model: Standard Medicine Precise GPCR model: Optimized Medicine Poorly understood GPCRs (locks) led to s
Slide 43: Appendix
Appendix 05
Slide 44: Partnered pipeline (1/2)
Partnered pipeline (1/2) Compound Target / Mechanism of Action Modality Indication Partner Disc. PCC Ph1 Ph2 Ph3 App Mkt Partnered Seebri® Breezhaler® LAMA SME COPD Ultibro® Breezhaler® LAMA+LABA SME COPD Enerzair® Breezhaler® LAMA+LABA+ICS SME Asthma ORAVI® Antifungal agent miconazole SME Oropharyn
Slide 45: Partnered pipeline (2/2)
Partnered pipeline (2/2) Compound Target / Mechanism of Action Modality Indication Partner Disc. PCC Ph1 Ph2 Ph3 App Mkt Co-development KY1051 CXCR4 mAb mAb Immuno-oncology (Not disclosed) AI-Augmented Drug Discovery SME Neurology diseases (Not disclosed) Multi targe SME/LME Immune / Neurology disea
Slide 46: In-house pipeline
In-house pipeline Compound Target / Mechanism Modality Indication Partner Disc. PCC Ph1 Ph2 Ph3 App Mkt In-house Programs PIVLAZ® ETA antagonist SME Cerebral vasospasm QUVIVIQ® Dual Orexin antagonist SME Insomnia NXE0048149 1 GPR52 agonist SME Neurology diseases NXE0039732 2 EP4 antagonist SME Immun
Slide 47: Clinical Trials
Clinical Trials Compound MoA Condition Phase Size Patient Start Completion* Last Update Link (main/latest) Link (others) NBI-1117568 M4 agonist Schizophrenia Ph2 210 Yes 2022-10-04 2024-07-10 2025-07-11 NCT05545111 - NBI-1117568 M4 agonist Schizophrenia Ph3 284 Yes 2025-05-08 2027-10 2025-12-15 NCT0
Slide 48: Estimation of potential market size
Estimation of potential market size Multi-billion USD annual peak sales potential for our post-pre-clinical pipeline Peak Sales Category Indication2 Number of Patients Candidates Market Size Individual Products Dementia ~55 million $7.3 billion (2010) $3.9 billion (2009/Aricept) M1 ag, M1/M4 ag Schi
Slide 49: Exclusive Opt-in Rights And ROFN/ROFR
Exclusive Opt-in Rights And ROFN/ROFR1 Option to develop up to five clinical programs for Japan and APAC (ex-China) from Idorsia Program Mechanism of Action Indication Stage Region Exclusive Lucerastat Glucosylceramide synthase inhibitor Fabry disease Phase 3 Opt-in Right Multiple sclerosis and ACT-
Slide 50: Core Operating Profit - Definition
Core Operating Profit - Definition Core Operating Profit/Loss – a financial indicator closer to the reality of our business Operating Profit Operating Profit “Core” “IFRS” ◼ Core Operating Profit/ Loss is a key ◼ Financial results recorded and financial indicator that highlights the + Material Non-c
Slide 51: Glossary
Glossary Basic Terminology/Technology GPCR G Protein-Coupled Receptor There are about 800 types of GPCRs in the human body. While 400 of them are known to be potential drug targets, about 300 of them are not yet drugged Nxera’ proprietary technology to stabilize a GPCR by engineering a small number
Slide 52: Locations
Locations Midtown East, F17, 410 Teheran- Steinmetz Building Burleigh-on-the-Strand Office 26.01 9-7-2 Akasaka Ro Granta Park, 355-359 Strand Flexoffice Messteurm London 26th Floor Minato-ku GangNam-Gu Cambridge Messeplatz 10 Tokyo 107-0052 Seoul 06192 WC2R 0HS CB21 6DG CH-4058 Basel Japan South Kor
Slide 53: Thank you
Thank you BREAKTHROUGHS IN PROGRESS ● BREAKTHROUGHS IN PROGRESS ● BREAKTHROUGHS IN PROGRESS