Circio Holding Asa · OSL:CRNA
Circio Holding Asa — FY2024 Earnings Presentation
23 slides from Circio Holding Asa's earnings presentation, Mar 26, 2025
Slide 1: Circular RNA expression systems for enhanced gene and cell therapies
Circular RNA expression systems for enhanced gene and cell therapies 2024 report & R&D update 26 March 2025
Slide 2: Important notice and disclaimer
Important notice and disclaimer 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
Slide 3: Market update; circVec technology update; 2024 financials
1 Market update 2. circVec technology update 3. 2024 financials 3
Slide 4: Continued high deal activity in circular RNA space
Continued high deal activity in circular RNA space Licensing Series A+B Series A $150M Upfront $490M $3,5B deal value $270M M&A $65M Upfront May/Aug 2021 Aug 2022 April 2023 May 2024 Jan 2025 Feb 2021 Jul 2022 Aug 2022 Oct 2022 Aug 2023 $80M $42M $221M Undisclosed $30M Series A Series A Series B M&A
Slide 5: Two recent circRNA and gene therapy deals highlight substantial commercial opportunity
Two recent circRNA and gene therapy deals highlight substantial commercial opportunity in Circio areas Licensing M&A $65m up-front $1.1b + up to $635m in milestones in up-front + milestones LNP-circRNA for in vivo AAV gene therapy for muscular gene editing in blood diseases dystrophies circRNA (oRNA
Slide 6: Circio has built broad visibility in scientific literature, life science media and industry conferences
Circio has built broad visibility in scientific literature, life science media and industry conferences January 2025 6
Slide 7: circVec technology update
2 circVec technology update 3. 2024 financials 7
Slide 8: Novel circVec 3.0 generation adds power to the platform
Novel circVec 3.0 generation adds power to the platform circVec generation 1.X – 3.X, design schematics circVec protein quantification, Western blot circVec 1.1 ~27x increase for SA SD IR IRES + ORF IR circVec 3.0 vs 1.1 Relative protein quantity ~4x circVec 2.0 IRES + ORF ~2x circVec 2.1 ~3x IRES +
Slide 9: circVec 3.0 validated in several vector formats
circVec 3.0 validated in several vector formats plasmid Technical development DNA Non-therapeutic applications ✓ next.gen Repeat-dosable non-viral DNA gene therapy ✓ Repeat-dosable gene therapy ✓ linear DNA Vaccines Classic viral gene therapy AAV Ongoing Single dose only 9
Slide 10: LNP-formulated circVec 2.1 accumulates in spleen, potentially valuable opportunity in cell therapy
LNP-formulated circVec 2.1 accumulates in spleen, potentially valuable opportunity in cell therapy LNP-mVec (mRNA), luminescence LNP-circVec 2.1 (circRNA), luminescence Systemic I.V. delivery, single dose on Day 0 Systemic I.V. delivery, single dose on Day 0 Mainly short-term liver expression No liv
Slide 11: 3D imaging confirms spleen-specific circVec expression
3D imaging confirms spleen-specific circVec expression Highly specific, strong signal in spleen 11 Example animal, spleens harvested for cellular and molecular analyses
Slide 12: circVec for gene therapy – enhancing AAV vectors
circVec for gene therapy – enhancing AAV vectors AAV protein expression, in vitro f-Luc AAV vector AAV-circVec 2.0 AAV-mRNA ~2x Standard circVec mRNA approach circRNA approach Enhanced RNA durability Enhanced Protein expression circVec-AAV feasibility validated, testing and optimization of construct
Slide 13: Muscle-specific AAV-circVec 2.0 vs. mRNA in vivo expression by systemic I.V. injection
Muscle-specific AAV-circVec 2.0 vs. mRNA in vivo expression by systemic I.V. injection AAV-circVec technical IVIS, Low-dose (LD) optimization in progress IVIS signal quantification, low dose, in vivo f-Luc AAV9-circVec 2.0-FL AAV9-mVec-FL n = 4 per group circVec expression advantage from D60, +50% a
Slide 14: Broad circVec DNA and AAV in vivo program ongoing, multiple read-outs during Q2 2025
Broad circVec DNA and AAV in vivo program ongoing, multiple read-outs during Q2 2025 JAN´25 MAR´25 MAY´25 JUL´25 circVec 2.1, Certest LNP, I.V. ✓ Feasibility study on LNP:DNA delivery, strong spleen signal, Completed, tissue analysis post-mortem tissue analysis ongoing circVec 2.1/3.0, non-viral vec
Slide 15: Several external collaborations starting / ongoing to test DNA circVec 2.1 & 3.0 delivery
Several external collaborations starting / ongoing to test DNA circVec 2.1 & 3.0 delivery in vivo JAN´25 MAR´25 MAY´25 JUL´25 LNP-circVec 2.1, I.V. delivery, w/undisclosed Ongoing; data expected in April LNP-circVec 2.1, I.M. delivery, w/4BaseBio Ongoing, data during April circVec 2.1/3.0, new splee
Slide 16: circVec R&D top priorities and next steps
circVec R&D top priorities and next steps circVec 3.0 optimization for multiple vector formats circVec circVec 3.0 testing in vivo, multiple vector formats platform Two new patent filings at drafting / planning stage Construct and validate superior circVec-AAV designs Gene therapy Testing tissue-spe
Slide 17: 2024 financials
2024 financials 17 ¢ircio
Slide 18: Circio's financial position was substantially strengthened during 2024
Circio´s financial position was substantially strengthened during 2024 NOK 30m raised in equity financing in 2024 NOK 19.6m from Rights Issue in July Equity NOK 10.7m from warrants exercise in December: Financing − Warrants: NOK 7.8m (93% of warrants exercised) − NOK 2.9m from parallel private place
Slide 19: 2024 financials – Strong cost reduction
2024 financials1 – Strong cost reduction NOK m 2023 2024 Total revenue 0 0 Transition from clinical R&D expenses2 -50 -12 to preclinical: - 75% Payroll and related expenses -34 -22 Payroll cost: - 35% Other operating expenses3 -16 -9 Total operating expenses -101 -43 Other operating: - 45% Operating
Slide 20: Resources focused to maximize R&D output
Resources focused to maximize R&D output Lean & efficient team (10 FTEs) Organization Most FTEs are R&D related positions Steady state reached for current funding level Generate data package for transactions R&D focus Active collaborations to generate new data Enabling asset development for partneri
Slide 21: Pursuing various options to strengthen the shareholder base and financial outlook
Pursuing various options to strengthen the shareholder base and financial outlook Continuously exploring multiple financing options Financing Accessing new groups of potential future investors Atlas Agreement to extend the Financing commitment beyond facility June 2025 Structural Evaluating opportun
Slide 22: Upcoming milestones and timelines: rich pipeline of R&D milestones with multiple shots on goal
Upcoming milestones and timelines: rich pipeline of R&D milestones with multiple shots on goal ASGCT 2025 conference, mid-May Value inflection point 1: Value inflection point 2: Premier global gene therapy event Data for gene therapy BD deal Disease model validation Data from ongoing in vivo program
Slide 23: Q & A Session
Q & A Session 2024 report & R&D update 26 March 2025