Core Insights - Protalix BioTherapeutics reported total revenues of $43.6 million for the first nine months of 2025, a 24% increase compared to the same period in 2024, while third-quarter revenues were $17.9 million, reflecting a 1% decrease year-over-year [2][13] - The company is advancing its recombinant PEGylated uricase candidate, PRX-115, for uncontrolled gout, with plans to initiate a phase 2 clinical trial later this year based on promising phase 1 data [2][5] - Protalix's financial results indicate a net income of approximately $2.4 million for the third quarter of 2025, down from $3.2 million in the same quarter of 2024 [6] Financial Highlights - Total revenues for the nine months ended September 30, 2025, were $43.1 million, up from $34.8 million in 2024, with significant contributions from sales of Elfabrio, Elelyso, and alfataliglicerase [13] - Cost of goods sold increased by 10% to $22.4 million for the nine months ended September 30, 2025, primarily due to increased sales to Chiesi and Pfizer [13] - Research and development expenses rose by 58% to approximately $13.9 million for the nine months ended September 30, 2025, driven by preparations for the phase 2 clinical trial of PRX-115 [13][18] Operational Updates - The company submitted an Investigational New Drug (IND) application to the FDA for PRX-115, which became effective after the standard 30-day review period [5] - Protalix is collaborating with Chiesi Global Rare Diseases to address a negative opinion from the EMA regarding Elfabrio's dosing regimen, while the currently approved regimen remains unaffected [5] - As of September 30, 2025, Protalix had $29.4 million in cash and cash equivalents, sufficient to meet capital needs for at least the next 12 months [8]

Core Viewpoint - Chiesi Global Rare Diseases and Protalix BioTherapeutics have requested a re-examination of the negative opinion from the CHMP regarding the proposed dosing regimen for Elfabrio® (pegunigalsidase alfa) [1][2] Company Overview - Chiesi Global Rare Diseases is a business unit of the Chiesi Group, focused on delivering innovative therapies for rare diseases [16] - Protalix BioTherapeutics specializes in developing recombinant therapeutic proteins using its proprietary plant cell-based expression system, ProCellEx [18] Product Information - Elfabrio® is indicated for the treatment of adults with confirmed Fabry disease [3] - The current approved dosing regimen is 1 mg/kg every 2 weeks, while a new regimen of 2 mg/kg every 4 weeks is under review [2] Clinical Trial Insights - In clinical trials, 14% of patients treated with Elfabrio experienced hypersensitivity reactions, with 3% experiencing anaphylaxis [6] - Infusion-associated reactions were reported in 29% of patients, including symptoms such as nausea, chills, and rash [7][9] Regulatory Context - The existing marketing authorization for Elfabrio remains effective while the re-examination process is ongoing [2] - The outcome of the re-examination will influence the final decision by the European Commission [2] Disease Background - Fabry disease is a rare inherited lysosomal storage disorder caused by mutations in the GLA gene, leading to enzyme deficiency and accumulation of globotriaosylceramide (GL-3) [11][12] - Symptoms include fatigue, chronic pain, and increased risk of serious complications such as kidney failure and stroke [11][12]

Core Opinion - The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a negative opinion regarding the revised dosing schedule for Elfabrio, a treatment for Fabry disease, proposed by Protalix BioTherapeutics and Chiesi Group [1][2]. Company Overview - Protalix BioTherapeutics and Chiesi Group are facing selling pressure following the CHMP's decision [2][5]. - The companies had requested approval for a new dosing regimen of 2 mg/kg body weight infused every 4 weeks, in addition to the currently approved regimen of 1 mg/kg body weight infused every 2 weeks [2]. Clinical Data - The CHMP review was based on data from the BRIGHT trial and an ongoing open-label extension study, which collectively provided a median exposure of almost six years [3][4]. - The data from these studies, along with modeling and exposure-response analyses from prior trials, were deemed insufficient to demonstrate similar efficacy for the new dosing schedule [4]. Market Impact - Following the negative opinion, Protalix BioTherapeutics' stock price decreased by 29.83%, closing at $1.68 [5].