Slide 1: Regenerative Medicine to Cure Type 1 Diabetes
A P R I L 2 0 2 6 ( N A S D A Q : N C E L ) © 2 0 2 6 N EW C EL X L TD . REGENERATIVE MEDICINE TO CURE TYPE 1 DIABETES Leading The Evolution of Type 1 Diabetes Cure With a Scalable Platform Producing Enriched, High - quality Stem Cell – derived Islets
Slide 2: Disclaimer
Disclaimer This presentation contains express or implied forward - looking statements within the Private Securities Litigation Reform Act of 1995 and other U . S . Federal securities laws . For example, NewCelX Ltd . (“NewCelX,” “we,” “our” or “us”) is using forward - looking statements when we disc
Slide 3: Cure Type 1 Diabetes
Cure Type 1 diabetes We are a clinical stage regenerative medicine company focused on Type 1 Diabetes Cell Therapy using highly enriched stem cell - derived islets as scalable cell replacement therapy 3 3
Slide 4: The NewcelX Factor Today, Value Driven
The NewcelX Factor Today, Value Driven CORE PLATFORM Stem Cell Technology PRIMARY VALUE DRIVER Type 1 Diabetes PLATFORM VALIDATION Neurology – ALS AstroRx® ADJACENT CNS PLATFORM DOXA (Dual Orexin Agonist) STRATEGIC ALLIANCES • GMP Manufacturing • Clinical - Grade Bio - Banks • Off - the - Shelf Ther
Slide 5: Cell Therapy in Type 1 Diabetes: Unmet Need
Cell Therapy in Type 1 Diabetes: Unmet Need 5 Insulin therapy and glucose management manages symptoms, but are not a cure Cell therapy for diabetes = Cure Islet transplantation patients achieve complete insulin independence 1. Cell Metab . 2019 Mar 5;29(3):545 - 563. doi: 10.1016/j.cmet.2019.02.007.
Slide 6: Global Market and Facts For Type 1 Diabetes
Global Market and Facts For Type 1 Diabetes Living with Type 1 Diabetes worldwide 1 9.5 MILLION PEOPLE 1.8 MILLION Children and adolescents below 20 years have type 1 diabetes 2 174,000 Premature Deaths attributed to Type 1 Diabetes 3 813B$ Lifetime cost in US for health expenditure in Type1 diabete
Slide 7: An Active Market: Key Recent Transactions, Developments & Validations
An Active Market Key Recent Transactions, Developments & Validations 2019 Semma Therapeutics 2019 Acquired by Vertex $950M Preclinical islet cell therapy 1 Diabetes 2021 Diabetes and cell therapy Sana Biotechnology 2021 Aspect Biosystems /Novo Nordisk Lead asset licensing deal $75M upfr ont $750M to
Slide 8: Major Technology Competition – Type 1 Diabetes
Major Technology Competition – Type 1 Diabetes Company Product Development stage Cell source Technology Cell Therapy Technology Competition Liver infusion + immunosuppression Stem cells - derived Islets Phase 1 - 2 - 3 VX - 880 Vertex 1 Omentum + immunosuppression Stem cells - derived Islets Phase 1
Slide 9: NCEL-101: Enriched stem cells-derived islets
Pluripotent Definitive Endoderm Posterior Foregut Pancreatic progenitors Primitive gut tube Endocrine Immature Endocrine progenitors S 2 S 1 S 5 S 4 S 3 S 6 S 7 Differentiation of PSCs into islet - like clusters 7 - stage protocol, ~28 days for production Recapitulating pancreatic development stages
Slide 10: NCEL-101 With Eledon Pharmaceuticals
• Next generation immune supression drug: anti - CD40L monoclonal antibody, tegoprubart (AT - 1501) • Clinical experience from over 100 transplant patients treated with tegoprubart • The immune modulation component is intended to enhance durability and graft survival of the cells NCEL - 101 With Ele
Slide 11: Co-development & Projects Pipeline Developments
Co - development & Projects Pipeline Developments Type 1 Diabetes Indication NCEL - 201: In house product using encapsulation NCEL - 007: Collaboration project for hypo immune gene edited cells NCEL - 301: Collaboration project for device iTOL - 102 11
Slide 12: iTOL-102: Mechanism of Action
• Regenerative medicine without the need for life - long immunosuppression • Leverages the naturally occurring protein, Fas Ligand (FasL) • Creates localized immune privilege environment • BIRD Grant funded (~$1M) • Successful POC results (Q2 2024) • Breakthrough designation by DRI • Successful Pre
Slide 13: In House Product Development: NCEL-201 Preclinical Efficacy
• Long - term therapeutic effect was achieved in an immunocompetent animal model (C57BL/6 mice) • NCEL - 201 cells are well protected from host immune system In House Product Development: NCEL - 201 Preclinical Efficacy NCEL - 201 treated diabetic mice (STZ) demonstrated balanced and normal blood gl
Slide 14: Next Generation Cell Line NCEL-007 and NCEL-301
• Co - Development with TargetGene Biotechnologies Inc. Genome editing to evade immune system recognition allowing graft persistence. • Using precise, validated, gene editing platform, with minimal off - target editing • Enables the production of hypo immune cells for the treatment of Type 1 Diabete
Slide 15: Expansion and differentiation protocols
Expansion and differentiation protocols Potential wide range of cells treating a wide range of diseases. Pluripotent Stem Cell Expansion Bio - Banking GMP quality Starting Material. Clinical - grade Embryonic Stem Cell line Unlimited # of Pluripotent Stem Cell Bio - banks Large scale Key Platform At
Slide 16: Proof of platform product execution from bench to bed
Proof of platform product execution from bench to bed: FDA - approved IND, phase 2a using AstroRx® cell therapy product for clinical trial in ALS patients 16
Slide 17: AstroRx: Validated Platform for Neurodegenerative Diseases
Astrocytes Cell Therapy Treatment for Neurodegenerative Diseases a Validated Platform AstroRx® AstroRx® has Orphan drug designation from the U.S. Food and Drug Administration (“FDA”) 17
Slide 18: Why Use Astrocytes for ALS - AstroRx
Why Use Astrocytes for ALS - AstroRx ® In neurodegeneration, neurons die because their support cells (Astrocytes) fail AstroRx ® contains functional healthy astrocytes to protect ALS - diseased motor neurons using multiple mechanisms of action 1. 2. Izrael et al, 2020 Front Neurosci. 2020; 14: 824.
Slide 19: Follow-up Results Demonstrated a Clinically Meaningful Decline in Disease Progression
3 - month Follow - up Results Demonstrated a Clinically Meaningful Decline in Disease Progression Clinical results are consistent between Cohorts A and B ALSFRS - R slope difference between 3 months pre - and post - treatment in Cohorts A and B GMP manufacturing to human dosing achieved Favorable sa
Slide 20: DOXA (Dual Orexin-Receptor Agonist) Platform
Strategic Enhancements Positioned for potential out - licensing or strategic partnerships Target indications: • Targeting sleep – wake regulation • Rare Sleep Disorders (Narcolepsy, Idiopathic Hypersomnia) • Neurodegenerative / neuroinflammation diseases • Diabetes - Associated Neurological Disorder
Slide 21: Scalability & Clinical Manufacturing Capabilities
Scalability & Clinical Manufacturing Capabilities From Art to Industry Long - term success in cell therapy through reproducible expansion and differentiation technology, manufacturing systems that are scalable, and cost - efficient Manufacturing Strategy Allogeneic, Off - the - Shelf Model Enables s
Slide 22: NewcelX Pipeline Focus – Type 1 Diabetes & CNS Disorders
IND Preclinical Discovery Indication Program Priority Program Type 1 Diabetes NCEL - 101 Adjacent Programs Type 1 Diabetes ITOL - 102 NCEL - 201 NCEL - 301 NCEL - 007 ALS (Amyotrophic Lateral Sclerosis) AstroRx® (Platform Validation) Neurodegenerative Diseases (MS, PD) Narcolepsy, Sleep Disorders (P
Slide 23: Patent Protection
Patent Protection Patents in major countries/markets such as the US, EU and other countries providing NewCelX with robust technology protection Methods for differentiating and purifying pancreatic endocrine cells Insulin producing cells derived from pluripotent stem cells Methods of generating glial
Slide 24: The Partnership Blueprints Model
The Partnership "Blueprints" Model: We aim to monetize the platform in three ways: 1. Program Licensing: Regional deals for NCEL - 101/AstroRx ® 2. Platform Partnerships: Licensing the "Manufacturing Blueprints" to partners (e.g., Pharma wants Dopamine neurons; we build them) 3. Delivery Partnership
Slide 25: Leadership Team
Leadership Team Eric Konofal, MD, PhD Alex Zwyer, MBA Prof. Ariel Revel, MD Kfir Molakandov, PhD Omri Hagai, CPA Prof. Michel Revel MD, PhD Ronen Twito Director Translational Neuroscience Director & Chief Business Officer Medical Director Vice President of Research & Development Chief Financial Offi
Slide 26: Toward a Cure for Type 1 Diabetes
Hohstrasse 1 8302 Kloten Switzerland Headquarter in Switzerland :+41 44 512 21 50 Main office in Israel : +972 - 73 - 797 - 1613 info@newcelx.com T O W A R D A C U R E F O R T Y P E 1 D I A B 26
Slide 27: References
References page 27 Page 6 1. https:// www.diabetesresearchclinicalpractice.com/article/S0168 - 8227(25)00291 - 8/fulltext 2. https://diabetesatlas.org/data - by - indicator/type - 1 - diabetes - estimates/people - with - type - 1 - diabetes - 0 - 19 - y/ 3. https:// www.diabetesresearchclinicalpract