Protalix BioTherapeutics (PLX) Investor Day 2024 summary

Future plans and strategic direction

• Focus will be on expanding the rare disease pipeline, especially in renal diseases, leveraging the ProCellEx platform and plant-based drug delivery systems.

• Phase II of the PRX-115 gout program is planned to initiate mid next year, following completion of Phase I and regulatory consultations.

• Continued partnerships with Chiesi, Pfizer, and the Brazilian government are expected to drive revenue growth and support operations.

• Strategic emphasis on both organic R&D and targeted M&A to broaden the innovative rare disease portfolio.

• Aim to achieve a fully integrated company with end-to-end capabilities and a robust commercial infrastructure.

Financial guidance

• End of Q1 cash position was $48 million, with sufficient runway to support debt repayment and ongoing operations, including Phase II gout trial.

• Debt will be fully repaid by September, leaving the company debt-free by year-end.

• Three revenue streams (Chiesi, Pfizer, Brazil) are expected to grow, supporting self-financing of pipeline development.

• Breaking $100 million in revenues is anticipated within a few years, requiring at least a 15% market share.

New business developments

• Elfabrio, a new enzyme replacement therapy for Fabry disease, was approved in the US and EU in May 2023 and is positioned as a strong alternative in a $2–3 billion market.

• PRX-115 for uncontrolled gout completed Phase I with positive safety and efficacy signals; Phase II is in planning.

• Ongoing evaluation of plant-based drug delivery systems and chemical modifications to enhance the ProCellEx platform.

• Early-stage and preclinical programs are in development, with updates to be provided as milestones are reached.

• Real-world data and expanded indications are being pursued for both Fabry and gout programs.