Codexis (CDXS) Stifel 2024 Healthcare Conference summary

Strategic direction and market outlook

• Focused on enzyme engineering for pharmaceutical manufacturing and expanding into siRNA manufacturing with the ECO Synthesis platform after a strategic review.

• siRNA therapeutics market is rapidly growing, with over 400–700 products in development and significant potential for large indications like cardiovascular and CNS diseases.

• Anticipates a major inflection point in siRNA production needs, paralleling the growth seen in monoclonal antibodies.

• Current chemical manufacturing methods for siRNA are capital intensive and not scalable for future demand, creating an opportunity for enzymatic solutions.

• Targeting both early and late-stage assets, with initial focus on integrating enzymatic ligation into existing chemical processes for easier regulatory adoption.

Technology development and customer engagement

• Demonstrated gram-scale synthesis of oligos and full-length siRNA assets, including successful presentations at TIDES US and Europe.

• Showcased flexibility by synthesizing siRNA using various combinations of chemical and enzymatic methods, meeting diverse customer needs.

• Customers can choose between fully enzymatic or hybrid approaches, with ligation being the simplest and most cost-effective entry point.

• No current technical disadvantages based on customer preferences for synthesis methods; future modifications may influence this.

• Scaling up production to 10–100 grams is the next milestone, critical for customer validation and GMP readiness.

Commercialization and partnership strategy

• Focusing on siRNA as the initial application, aiming to partner with major players with multiple assets in their pipelines.

• ECO Innovation Lab is set for completion by year-end, consolidating capabilities and improving efficiency.

• GMP manufacturing will be pursued via partnerships with CDMOs or by acquiring/building facilities in a capital-efficient manner.

• CDMOs are interested due to the scalability and cost-effectiveness of the enzymatic platform, with minimal CapEx required compared to chemical methods.

• Key requirements for CDMO adoption include secure raw material supply and clear cost-of-goods savings.