Protalix BioTherapeutics (PLX) Morgan Stanley 24th Annual Global Healthcare Conference summary

Company overview and technology

• Focuses on rare disease assets with two commercial products: Elelyso for Gaucher disease and Elfabrio for Fabry disease, both partnered with major global players.

• Holds a 27% market share in Brazil for Elelyso, generating $11 million in 2025 revenues.

• Utilizes the ProCellEx platform, a plant cell-based protein expression system, offering lower setup costs, room temperature manufacturing, and reduced contamination risk.

• Platform enables flexible scaling and was resilient during the COVID period, with no contamination issues.

• Maintains a strong balance sheet with no debt or warrants, and is well-funded for pipeline development.

Commercial performance and market outlook

• Elfabrio is the main growth driver, with global commercialization by Chiesi and expanding patient base and territories.

• Fabry market projected to grow from $2.2 billion in 2025 to $3.2 billion by 2031; Elfabrio aims to capture 15%-20% market share by 2031.

• Recent regulatory milestones include EU and UK approval for a four-week dosing regimen, reducing patient burden by 50%.

• Received a $25 million regulatory milestone and patent extension for Elfabrio until November 2035.

• Guidance for 2026: $78–$83 million total revenues, with $33–$35 million from Elfabrio, a 50% increase over 2025.

Pipeline and R&D strategy

• Lead pipeline product PRX-115 targets uncontrolled gout, with phase II topline results expected by end of 2027.

• Gout market is large and underserved, with PRX-115 aiming to differentiate through longer dosing intervals and no methotrexate.

• Phase I results showed patients maintained target uric acid levels for over 80 days at highest dose.

• Phase II is a double-blind study with five arms, designed to demonstrate differentiation from existing therapies.

• Financial strength allows for flexibility in advancing PRX-115, including potential self-commercialization.