Codexis (CDXS) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Business model evolution and enzyme engineering

• Shifted focus from general pharma manufacturing to RNA production, especially siRNA, leveraging enzyme engineering expertise.

• Enzymatic approaches are favored for longer RNA constructs and complex modifications, offering advantages over chemical synthesis.

• Developed cascades of enzymes to enable efficient RNA construct production.

Market trends and siRNA opportunity

• siRNA is emerging as a major drug class, with over 400 candidates in development and 30-40 in late-stage trials.

• siRNA therapies offer convenient dosing and strong efficacy, especially for liver-targeted drugs.

• Anticipated surge in demand for siRNA will outpace current chemical manufacturing capacity.

Manufacturing challenges and ECO Synthesis platform

• Current siRNA production relies on phosphoramidite chemistry, which is inefficient, wasteful, and capital-intensive.

• Enzymatic, aqueous-based solutions are seen as necessary for future scalability and sustainability.

• ECO Synthesis platform enables flexible production strategies, including chemical, enzymatic, and hybrid approaches.

• Demonstrated ability to produce identical siRNA molecules via multiple routes, supporting transition from chemical to enzymatic methods.