FDA Clears PRX-115 IND for Phase 2 Activation as Protalix Appeals Elfabrio Dosing Opinion
Protalix’s IND for PRX-115 in uncontrolled gout became effective after FDA’s 30-day review, activating Phase 2 sites following Phase 1 data showing up to 12-week urate reduction. Protalix and Chiesi have filed an appeal against EMA’s negative opinion on Elfabrio’s 2 mg/kg every-four-weeks dosing, with a decision expected in Q1 2026.
1. 2026 Strategic Priorities and Pipeline Advancement
In his January 5 letter to stockholders, CEO Dror Bashan outlined Protalix’s 2026 roadmap, emphasizing a dual focus on commercial execution with existing partners and advancement of its internal pipeline targeting high-need rare diseases. The company will prioritize Phase 2 initiation of PRX-115 for uncontrolled gout, following Phase 1 data demonstrating rapid urate reduction below 6 mg/dL across all cohorts and durable effects lasting up to 12 weeks at higher doses. An active IND (NCT05745727) allows U.S. sites to enroll patients, positioning PRX-115 as potentially best-in-class with once-every-four-weeks dosing. Concurrently, Protalix is accelerating preclinical work on PRX-119, a PEGylated DNase I designed to degrade neutrophil extracellular traps and reduce inflammation and fibrosis in rare renal diseases.
2. Commercial Execution and Market Positioning
Protalix’s near-term revenue foundation rests on partnered products Elfabrio® (pegunigalsidase alfa) and Elelyso® (taliglucerase alfa). With Chiesi Global Rare Diseases, Elfabrio launch metrics—treated patient counts and market share across the U.S., EU and additional territories—are tracking to plan. The global Fabry market is projected to reach approximately $3.4 billion by 2030; Protalix forecasts Elfabrio could capture 15%–20% of that value. An appeal of the EMA’s November 2025 negative opinion on a 2 mg/kg every-four-weeks dosing regimen is underway, with an outcome expected in Q1 2026; the approved bi-weekly label remains unaffected. Elelyso® contributions, supported by collaborations with Pfizer and Brazil’s Fiocruz, continue to underpin operating resilience.
3. Partnerships and Long-Term Value Creation
Protalix is leveraging strategic collaborations to fortify its pipeline and accelerate innovation. The RNA-based alliance with Secarna Pharmaceuticals will utilize Secarna’s AI-driven OligoCreator® platform to generate novel oligonucleotide therapeutics for rare renal indications. Commercial partnerships remain core to value creation: Chiesi drives Elfabrio’s global roll-out, Pfizer manages Elelyso® worldwide outside Brazil, and Fiocruz facilitates Brazilian access. This blended model of partnered commercialization and internal R&D is designed to limit downside risk while preserving upside, as Protalix scales PRX-115 into Phase 2, advances PRX-119 toward IND readiness, and explores further programmatic expansion in areas of high unmet need.