Circio Holding ASA · OSL:CRNA
Circio Holding ASA — Investor Presentation (April 2023)
21 slides from Circio Holding ASA's investor presentation, April 2023
Slide 1: Building next generation RNA therapeutics
Building next generation RNA therapeutics Corporate presentation April 2023
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: mRNA was the top-selling drug class in 2022
mRNA was the Top 10 drugs by 2022 projected sales ($B) top-selling drug Comimaty (Pfizer) $29,1 class in 2022 Humira (Abbvie) $20,4 Keytruda (Merck & Co.) $19,6 Spikevax (Moderna) $19,6 Remarkable speed - first mRNA Eliquis (BMS/Pfizer) $11,9 therapeutics were approved in 2020 Revlimid (BMS) $11,3 $
Slide 4: Although mRNA is already a successful therapeutic class, several challenges remain unsolved
Although mRNA is already a successful therapeutic class, several challenges remain unsolved RNA is chemically unstable and immunogenic – mRNA vaccines have required significant modifications Circular RNA can Efficient delivery of RNA therapeutics is overcome these currently limited to LNP- based sys
Slide 5: Enhanced protein expression / Extra-cellular trafficking
Enhanced protein Extra-cellular expression trafficking CircRNA is resistant to exonuclease degradation, leading to vastly increased half-life microRNA Regulatory sponging functionality 5
Slide 6: The discoverers of circRNA work for Circio
The discoverers of circRNA work for Circio Dr Thomas B Hansen Dr Erik D Wiklund 6,373 citations 30 September 2011 922 citations 2,291 citations
Slide 7: Despite tough market conditions, RNA-based financings increased sharply in 2021-22
Despite tough market conditions, RNA-based financings increased sharply in 2021-22 Private Financing Total Deal value of $1,137M RNA- based Companies by RNA $976M Modality Subtype ($M) $162 $27 $24 $29 circRNA represented 40% $211 $24 of the total deal value of all private RNA financings $107 $291 i
Slide 8: circRNA is rapidly gaining momentum as an enhanced mRNA format
circRNA is rapidly gaining momentum as an enhanced mRNA format Licensing Series A+B $150M Upfront $490M $3,5B Total deal value May/Aug 2021 Aug 2022 Feb 2021 Jul 2022 Aug 2022 Oct 2022 $80M $42M $221M Undisclosed Series A Series A Series B M&A 8
Slide 9: circVec – Circio's proprietary vector system for in situ protein production
circVec – Circio´s proprietary vector system for in situ protein production circVec DNA vector DNA Inject circRNA biogenesis circRNA in situ protein production 9
Slide 10: circVec is a modular genetic cassette for in situ circRNA biogenesis and protein expression
circVec is a modular genetic cassette for in situ circRNA biogenesis and protein expression circVec - DNA Modular structure Genetic cassette Efficient circRNA biogenesis design In-built regulatory functionality Vector agnostic – applicable to a + variety of DNA and viral systems Long- half life Mult
Slide 11: Circio's circVec vector system is technologically differentiated and offers important advantages
Circio’s circVec vector system is technologically differentiated and offers important advantages Enhanced Does not Potent Delivery Available Delivery intra- cellular require vaccination to solid GMP manu- to liver stability packaging platform tumors facturing circVec vector approach Synthetic circRN
Slide 12: Circio's circRNA Development Plan
Circio’s circRNA Development Plan v v v v In vivo PoC tumor model In vivo PoC for model Target selection and T- cell CAR trans- Next infectious disease technical PoC in vitro First candidate ready for duction technical PoC Milestone antigen clinic by 2025 Injectable solid tumors, Infectious diseases
Slide 13: circAde concept for circRNA delivery to solid tumors
ONCOLOGY circAde concept for circRNA delivery to solid tumors: reduced technology risk through validated IT injection Prolonged protein circVec DNA expression cassette circAde viral vector microRNA sponging Multi- functional circRNA Regulatory functionality 1. Intra- tumoral injection 2. circRNA bio
Slide 14: The circAde concept is building on extensive clinical experience with first generation mRNA vector
ONCOLOGY The circAde concept is building on extensive clinical experience with first generation mRNA vector ONCOS-102 Benchmarking of published clinical data in PD-1 resistant melanoma CR PR 35% ORR First gen ONCOS- 1021 5% 30% (7/20 pts.) adenoviral mRNA 35% ORR vector expressing BNT111 0% 35% (6/1
Slide 15: circVac has potential as a highly potent and user friendly vaccine concept
VACCINES circVac has potential as a highly potent and user friendly vaccine concept for infectious disease antigen- circRNA Prolonged circVac circVec DNA antigen vector cassette expression Additional booster 1. Injection / delivery 2. circRNA biogenesis and antigen expression 3. Immune response 15
Slide 16: A systematic screen is ongoing to identify target rare diseases
A systematic screen is ongoing to identify target rare diseases that can be addressed by circVec Oncology Musculoskeletal GI and Metabolic disorders Diseases Respiratory and Endocrine and +300 Immunological disorders Hormonal Diseases Ophthalmic disorders Neurological disorders Rare Monogenic Cardio
Slide 17: circVec design established for efficient circRNA biogenesis and protein expression
circVec design established for efficient circRNA biogenesis and protein expression Multiple functional circVec vector design strategies established Superior circRNA design identified Protein payload expressed from circVec-encoded circRNA Protein expression from circRNA circVec circRNA Ctrl Mock desi
Slide 18: 15x prolonged half-life and increased protein expression for circRNA compared to mRNA in vitro
15x prolonged half-life and increased protein expression for circRNA compared to mRNA in vitro RNA Stability, nascent vs. total RNA Output quantification; circRNA and protein payload RT-qPCR, plasmid expressed RNA Plasmid-expressed circRNA, transfected cells Minutes Hours Days mRNA 530 8.8 0.37 circ
Slide 19: Circio has a unique edge in the emerging circRNA field
Circio has a unique edge in the emerging circRNA field World- leading experts in- house with over 10 years circRNA experience Led by circRNA discoverer and pioneer Dr. Thomas B Hansen circVec is broadly applicable to multiple vector systems and therapeutic areas Effective design, technical PoC estab
Slide 20: R&D pipeline and milestones
R&D pipeline and milestones Preclinical Clinical Product candidate Discovery In vivo Phase 1 Phase 2 Phase 3 / Milestones PoC pivotal Solid tumors 2H 2023 circAde In vivo proof-of-concept data circVec Infectious 2H 2023 circRNA vector disease, circVac In vivo proof-of-concept data Rare 2H 2023 disea
Slide 21: The circRNA wave is here, and Circio has a unique approach and delivery system
“ The circRNA wave is here, and Circio has a unique approach and delivery system providing a head-start in this emerging therapeutic area, with a high- caliber team aiming to build the leading circRNA biotech Dr Erik D Wiklund - CEO 21