Protalix Biotherapeutics

EMA validates variation submission for pegunigalsidase alfa to label a 2 mg/kg dose every four weeks in adult Fabry disease patients, supported by clinical data from Phase 3 studies.

EMA validates Chiesi and Protalix's submission for pegunigalsidase alfa, proposing a 2 mg/kg dose every four weeks for Fabry disease treatment, compared to the current 1 mg/kg every two weeks regimen.

PRX-115, a PEGylated uricase therapy, showed dose-dependent urate-lowering responses and was well-tolerated in a first-in-human study of gout patients. PRX-115 catalyzes urate breakdown into allantoin, designed to improve stability and reduce immunogenicity. Preliminary results suggest PRX-115 could be a promising uric-acid lowering treatment, with plans for a phase II trial in 2025.

Protalix Biotherapeutics announced 3Q24 financial results, with $18M revenue (+74% YoY), driven by Chiesi revenues. Net income was $3.2M, or $0.03 per share. PRX-115 Phase I trial in gout completed, showing dose-dependent urate reduction and safety. Phase II planned for 2H25.

Protalix BioTherapeutics reports financial results for Q3 2024, highlighting completion of phase I PRX-115 study for gout treatment. Preliminary results show PRX-115's potential as an effective uric acid-lowering treatment. The company plans to initiate a phase II clinical trial in H2 2025. Financial highlights include $17.8M in revenue, a $3.2M net income, and $27.4M in cash and cash equivalents.

Elfabrio (pegunigalsidase alfa) administered every 4 weeks at 2 mg/kg appears safe and effective for stable Fabry disease adults, according to a Phase 3 study. The study, involving 30 patients, showed no severe side effects and stable kidney function, though more research is needed.

Sangamo Therapeutics' shares surged 69% after FDA alignment on an Accelerated Approval pathway for its Fabry disease gene therapy, isaralgagene civaparvovec (ST-920), using data from its Phase I/II STAAR trial. The company plans to submit a BLA in the second half of 2025, three years ahead of previous estimates.

Protalix BioTherapeutics (PLX) is a clinical-stage biopharmaceutical company developing therapeutic proteins using its ProCellEx® system. Key products include Taliglucerase Alfa for Gaucher disease and Elfabrio® for Fabry disease, with strategic partnerships with Pfizer and Chiesi Farmaceutici S.p.A. The company's robust pipeline includes PRX-115 for severe gout and PRX-119 for NETs-related diseases. Protalix maintains strong financial resilience and operational stability despite regional security challenges.

Protalix BioTherapeutics to present phase I PRX-115 trial data at ACR Convergence 2024, showcasing prolonged plasma urate-lowering effect for uncontrolled gout treatment.