Slide 1: Targovax Biotech Showcase January 2023
° targovax Biotech showcase January 2023
Slide 2: Forward-looking statements
This report contains certain forward-looking statements based on uncertainty, since they relate to events and depend on circumstances that will occur in the future and which, by their nature, will have an impact on the results of operations and the financial condition of Targovax and the Targovax Gr
Slide 3: Remarkable success given first mRNA-based therapeutics were approved in 2020
Remarkable success given first USD 50b total 2022 mRNA-based therapeutics were sales for COVID approved in 2020 mRNA vaccines mRNA outcompeted more established concepts in COVID vaccine race However, challenges remain… SOURCE: Statista analysis January 2023 3
Slide 4: RNA is chemically unstable – mRNA vaccines have required heavy modifications
RNA is chemically unstable – o Circular RNA is resistant to intra- mRNA vaccines have required cellular RNA degradation mechanisms heavy modifications o Expressing RNA from a DNA vector solves the stability issue Efficient delivery of RNA therapeutics is currently limited to o Clinically validated D
Slide 5: The circRNA discoverers work for Targovax
The circRNA discoverers work for Targovax Dr Thomas Hansen Dr Erik D Wiklund 5
Slide 6: Product candidates and milestones
Preclinical Clinical Product candidate Discovery IND- Phase 1 Phase 2 Phase 3 / Milestones enabling pivotal PD-1 Resistant Melanoma 1H 2023 Re-challenge combination w/aPD-1 & CTLA-4 Initiation of phase 2 trial (USA) ONCOS-102 Mesothelioma 1H 2023 Combination w/Standard-of-Care (SoC) Publication in o
Slide 7: Technical proof-of-concept data, Team, Summary
2. Technical proof-of-concept data 3. Team 4. Summary 7
Slide 8: circRNA provides a toolbox to create a novel class of medicines
circRNA provides a toolbox to create a novel class of medicines Extra-cellular trafficking Ob 2D CircRNA is resistant to exonuclease degradation, leading to much longer half-life and thus stronger and prolonged therapeutic effects eo . O O Onco-microRNA sponging —_ Immune response booster O A
Slide 9: Enhanced intra-cellular stability
Enhanced Does not Delivery Existing Delivery Vaccination intra-cellular require to solid GMP manu- to liver platform stability packaging tumors facturing Targovax circAde Synthetic circRNA Synthetic mRNA o Targovax has the only vector to deliver to solid tumors o cGMP vector manufacturing available
Slide 10: Synthetic circRNA manufacturing process
Synthetic circRNA manufacturing process1 Targovax manufacturing process Currently only small-scale, no commercial systems Using commercially available equipment In vitro RNA Master Cell Bank transcription Circularization Cell expansion, harvest and Issue: formation of linear cell lysis by-products,
Slide 11: Technical proof-of-concept data
Technical proof-of- concept data 3. Summary
Slide 12: Highly versatile – Multi-modal MoA – Excellent stability
Therapeutic product Inject Circular RNA Gene therapy / Inside the Onco-microRNA protein translation patient sponge Encode Immunological antigens activation Highly versatile – Multi-modal MoA – Excellent stability 12
Slide 13: Functional vector established
Functional vector established Highly effective circRNA design identified Payload expressed from vector-encoded circRNA Plasmid-expressed circRNA, transfected cells Insertion Vector Protein expression strategy function V1 V2 ⍺-payload 1 V3 ⍺-payload 1 V4 ⍺-Loading Ctrl o circRNA design strategy is cr
Slide 14: Increase in circRNA level from 48 to 96h
Output quantification; circRNA and protein payload Plasmid-expressed circRNA, transfected cells o circRNA expression o Increase in higher, even at 48hr circRNA level from time point 48 → 96h o circRNA protein o In same period, level builds from 48 >80% drop in → 96h, increasing mRNA level difference
Slide 15: Synthetic circRNA versus vector-based circRNA
Synthetic circRNA: One circRNA per experiment – oRNA approach Laborious approach Requires circRNA purification Sensitive to RNA yield, LNP formulation and transfection efficiencies One-by-one evaluation to pick top hits One sequence = One assay versus Vector-based circRNA: Parallel circRNA screening
Slide 16: Proof-of-concept summary
✓Functional vector design established ✓Superior circRNA design identified ✓Extended circRNA half-life confirmed ✓Enhanced and prolonged circRNA protein expression vs. mRNA 16
Slide 17: Focus for oncology and expansion opportunities
High-potency neoantigen immunization Immuno-stimulatory payloads for validated targets Focus for Oncology Vector I with systemic toxicity issues in-house Solid tumors Long-term supply of proteins or peptides to target development key cancer signalling and metabolic pathways Vaccines Higher and long-
Slide 18: Summary
Summary
Slide 19: Summary of key strengths
World-leading experts in-house with over 10 years circRNA experience Led by circRNA pionéer Dr. Thomas Hansen Unique circAde vector system for circRNA delivery to solid tumors Technical PoC established, vector turns cancer cells into circRNA factory GMP manufacturing at scale using commercially avai
Slide 20: Want to learn more?
.e) targovax Want to learn more? CFO Lubor Gaal Contact lubor.gaal@targovax.com www.targovax.com